Journal of Market Access & Health Policy Latest open access articles published in J. Mark. Access Health Policy at https://www.mdpi.com/journal/jmahp https://www.mdpi.com/journal/jmahp MDPI en Creative Commons Attribution (CC-BY) MDPI support@mdpi.com JMAHP, Vol. 14, Pages 32: Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies https://www.mdpi.com/2001-6689/14/2/32 Background: Real-world evidence (RWE) can complement clinical trials to inform health technology assessments (HTAs). This study examined the extent to which RWE is considered in HTAs of non-oncology orphan medicinal products across six agencies globally. Methods: Published European Medicines Agency decisions were reviewed to identify approved non-oncology orphan medicinal products (2018–2023) that included RWE within their submission package, which was anticipated to align with the inclusion of RWE in HTA submissions. Data were extracted from the corresponding HTA reports published by six national agencies (Australia, Canada, France, Germany, Sweden, and the UK). Results: RWE was included in 105 regulatory submissions and 52.6% of the corresponding HTA reports (range: 29.9% [Germany] to 78.8% [Canada]), nearly 90% of which received a positive decision (range: 44.4% [Australia] to 100.0% [Germany]). RWE was derived from a variety of study designs and commonly supported clinical efficacy across many therapeutic areas. Conclusions: RWE commonly supports HTAs of recently approved non-oncology orphan medicinal products, strengthening the evidence base and contributing to demonstration of product value. 2026-05-21 JMAHP, Vol. 14, Pages 32: Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020032

Authors: Oliver Blandy Pierluigi Lembo Rebecca Folorunso Karl-Johan Myren Helene Chevrou-Severac Simu K. Thomas

Background: Real-world evidence (RWE) can complement clinical trials to inform health technology assessments (HTAs). This study examined the extent to which RWE is considered in HTAs of non-oncology orphan medicinal products across six agencies globally. Methods: Published European Medicines Agency decisions were reviewed to identify approved non-oncology orphan medicinal products (2018–2023) that included RWE within their submission package, which was anticipated to align with the inclusion of RWE in HTA submissions. Data were extracted from the corresponding HTA reports published by six national agencies (Australia, Canada, France, Germany, Sweden, and the UK). Results: RWE was included in 105 regulatory submissions and 52.6% of the corresponding HTA reports (range: 29.9% [Germany] to 78.8% [Canada]), nearly 90% of which received a positive decision (range: 44.4% [Australia] to 100.0% [Germany]). RWE was derived from a variety of study designs and commonly supported clinical efficacy across many therapeutic areas. Conclusions: RWE commonly supports HTAs of recently approved non-oncology orphan medicinal products, strengthening the evidence base and contributing to demonstration of product value.

]]> Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies Oliver Blandy Pierluigi Lembo Rebecca Folorunso Karl-Johan Myren Helene Chevrou-Severac Simu K. Thomas doi: 10.3390/jmahp14020032 Journal of Market Access & Health Policy 2026-05-21 Journal of Market Access & Health Policy 2026-05-21 14 2 Article 32 10.3390/jmahp14020032 https://www.mdpi.com/2001-6689/14/2/32 JMAHP, Vol. 14, Pages 31: EU-HTA—Why Do Words Matter? https://www.mdpi.com/2001-6689/14/2/31 Words matter because they have profound power to shape thoughts, emotions, actions, and social realities [...] 2026-05-18 JMAHP, Vol. 14, Pages 31: EU-HTA—Why Do Words Matter?

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020031

Authors: Mondher Toumi Bruno Falissard Steven Simoens Maarten Postma Marta Wilk Laurent Boyer Renato Bernardini Stefano Capri Jaime Espin Jürgen Wasem Pascal Auquier

Words matter because they have profound power to shape thoughts, emotions, actions, and social realities [...]

]]> EU-HTA—Why Do Words Matter? Mondher Toumi Bruno Falissard Steven Simoens Maarten Postma Marta Wilk Laurent Boyer Renato Bernardini Stefano Capri Jaime Espin Jürgen Wasem Pascal Auquier doi: 10.3390/jmahp14020031 Journal of Market Access & Health Policy 2026-05-18 Journal of Market Access & Health Policy 2026-05-18 14 2 Editorial 31 10.3390/jmahp14020031 https://www.mdpi.com/2001-6689/14/2/31 JMAHP, Vol. 14, Pages 30: Between Rigor and Relevance: Why the EU HTA Guidelines on Indirect Comparisons Miss the Mark https://www.mdpi.com/2001-6689/14/2/30 Indirect treatment comparisons (ITCs) are essential in the context of joint clinical assessments (JCAs) under Regulation (European Union [EU]) 2021/2282, bridging evidence gaps where head-to-head data are lacking and enabling assessment across diverse national patient, intervention, comparator, and outcome (PICO) requirements. This paper critically reviews the EU Health Technology Assessment Coordination Group’s (HTACG) guidelines on direct and indirect comparisons, with particular focus on ITCs. While the guidelines promote transparency and rigorous evaluation of assumptions, they adopt a restrictive stance on assumption violations, the use of unanchored comparisons, and population-adjusted methods such as matching-adjusted indirect comparisons (MAIC) and simulated treatment comparisons (STC). The guidance shows limited support for Bayesian methods and undervalues meta-regression in favor of subgroup analyses. Operational implications for health technology developers (HTDs) are substantial, including new requirements for dual systematic reviews, multiple network structures, and shifted null hypothesis testing. Moreover, the guidelines effectively dissuade the use of non-randomized comparisons in rare or rapidly evolving indications and may inadvertently hinder access to effective treatments. Emerging practices such as external control arms (ECA) or target trial emulation are underdeveloped. Notably, there is no indication that the guidelines are grounded in systematic methodological validation studies. As JCAs evolve, greater methodological flexibility, empirical grounding, and clear operational guidance will be essential. Refining the guidelines along these principles would enhance their practical utility, mitigate intrinsic assessment variability, support consistent assessments across Member States (MS), and ultimately improve patient access to innovative therapies. 2026-05-07 JMAHP, Vol. 14, Pages 30: Between Rigor and Relevance: Why the EU HTA Guidelines on Indirect Comparisons Miss the Mark

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020030

Authors: Samuel Aballéa Mondher Toumi Piotr Wojciechowski Emilie Clay Bruno Falissard Steven Simoens Pascal Auquier Stefano Capri Renato Bernardini Joerg Ruof Frank-Ulrich Fricke Oriol Sola Morales Laurent Boyer

Indirect treatment comparisons (ITCs) are essential in the context of joint clinical assessments (JCAs) under Regulation (European Union [EU]) 2021/2282, bridging evidence gaps where head-to-head data are lacking and enabling assessment across diverse national patient, intervention, comparator, and outcome (PICO) requirements. This paper critically reviews the EU Health Technology Assessment Coordination Group’s (HTACG) guidelines on direct and indirect comparisons, with particular focus on ITCs. While the guidelines promote transparency and rigorous evaluation of assumptions, they adopt a restrictive stance on assumption violations, the use of unanchored comparisons, and population-adjusted methods such as matching-adjusted indirect comparisons (MAIC) and simulated treatment comparisons (STC). The guidance shows limited support for Bayesian methods and undervalues meta-regression in favor of subgroup analyses. Operational implications for health technology developers (HTDs) are substantial, including new requirements for dual systematic reviews, multiple network structures, and shifted null hypothesis testing. Moreover, the guidelines effectively dissuade the use of non-randomized comparisons in rare or rapidly evolving indications and may inadvertently hinder access to effective treatments. Emerging practices such as external control arms (ECA) or target trial emulation are underdeveloped. Notably, there is no indication that the guidelines are grounded in systematic methodological validation studies. As JCAs evolve, greater methodological flexibility, empirical grounding, and clear operational guidance will be essential. Refining the guidelines along these principles would enhance their practical utility, mitigate intrinsic assessment variability, support consistent assessments across Member States (MS), and ultimately improve patient access to innovative therapies.

]]> Between Rigor and Relevance: Why the EU HTA Guidelines on Indirect Comparisons Miss the Mark Samuel Aballéa Mondher Toumi Piotr Wojciechowski Emilie Clay Bruno Falissard Steven Simoens Pascal Auquier Stefano Capri Renato Bernardini Joerg Ruof Frank-Ulrich Fricke Oriol Sola Morales Laurent Boyer doi: 10.3390/jmahp14020030 Journal of Market Access & Health Policy 2026-05-07 Journal of Market Access & Health Policy 2026-05-07 14 2 Perspective 30 10.3390/jmahp14020030 https://www.mdpi.com/2001-6689/14/2/30 JMAHP, Vol. 14, Pages 29: Impact of a Digital Leakage Notification System on Healthcare Resource Utilisation and Costs for People with Stomas in the United Kingdom: Analysis of Interim Results from a Prospective Longitudinal Study https://www.mdpi.com/2001-6689/14/2/29 Background/Objectives: Leakage of stomal effluent adversely affects the quality of life for people living with a stoma and increases healthcare resource utilisation and costs. The Heylo™ digital leakage notification system (DLNS) detects early signs of leakage under the baseplate, enabling proactive stoma management. We evaluated the impact of the DLNS on stoma-related costs in a real-world UK setting. Methods: This costing analysis used data from the first 100 DLNS users with 6 months of follow-up in an ongoing longitudinal observational study (NCT06554015). Costs were calculated as 3-month totals before DLNS (baseline): Month 3, and Month 6 for stoma-related healthcare provider consultations, hospitalisations, and ostomy solution components (pouching systems, supporting products, and DLNS sensor layers). Least squares (LS) means were estimated using a mixed model. Results: Total LS mean costs for stoma-related consultations plus ostomy solution use decreased significantly from baseline by 21.9% at Month 3 (−£304 [95% CI: −£456, −£153]; p = 0.001) and 24.5% at Month 6 (−£340 [95% CI: −£504, −£175]; p < 0.001). Stoma-related consultation costs decreased by 50.7% at Month 3 (−£309 [95% CI: −£447, 171]; p < 0.001) and 57.9% at Month 6 (−£353 [95% CI: −£493, −£213]; p < 0.001), driven by fewer physician and stoma care nurse consultations. Mean ostomy solution costs remained similar from baseline (£784/person) to Month 6 (£782/person; p = 0.955) as DLNS sensor layer costs were offset by less use of other ostomy products. Conclusions: Initiation of the Heylo™ DLNS was associated with significant cost savings from reduced stoma-related consultations, pouching systems, and supporting product use. 2026-05-06 JMAHP, Vol. 14, Pages 29: Impact of a Digital Leakage Notification System on Healthcare Resource Utilisation and Costs for People with Stomas in the United Kingdom: Analysis of Interim Results from a Prospective Longitudinal Study

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020029

Authors: Alexandra Baxter Esben Bo Boisen Majken Linnemann Jensen Aamir Shaikh

Background/Objectives: Leakage of stomal effluent adversely affects the quality of life for people living with a stoma and increases healthcare resource utilisation and costs. The Heylo™ digital leakage notification system (DLNS) detects early signs of leakage under the baseplate, enabling proactive stoma management. We evaluated the impact of the DLNS on stoma-related costs in a real-world UK setting. Methods: This costing analysis used data from the first 100 DLNS users with 6 months of follow-up in an ongoing longitudinal observational study (NCT06554015). Costs were calculated as 3-month totals before DLNS (baseline): Month 3, and Month 6 for stoma-related healthcare provider consultations, hospitalisations, and ostomy solution components (pouching systems, supporting products, and DLNS sensor layers). Least squares (LS) means were estimated using a mixed model. Results: Total LS mean costs for stoma-related consultations plus ostomy solution use decreased significantly from baseline by 21.9% at Month 3 (−£304 [95% CI: −£456, −£153]; p = 0.001) and 24.5% at Month 6 (−£340 [95% CI: −£504, −£175]; p < 0.001). Stoma-related consultation costs decreased by 50.7% at Month 3 (−£309 [95% CI: −£447, 171]; p < 0.001) and 57.9% at Month 6 (−£353 [95% CI: −£493, −£213]; p < 0.001), driven by fewer physician and stoma care nurse consultations. Mean ostomy solution costs remained similar from baseline (£784/person) to Month 6 (£782/person; p = 0.955) as DLNS sensor layer costs were offset by less use of other ostomy products. Conclusions: Initiation of the Heylo™ DLNS was associated with significant cost savings from reduced stoma-related consultations, pouching systems, and supporting product use.

]]> Impact of a Digital Leakage Notification System on Healthcare Resource Utilisation and Costs for People with Stomas in the United Kingdom: Analysis of Interim Results from a Prospective Longitudinal Study Alexandra Baxter Esben Bo Boisen Majken Linnemann Jensen Aamir Shaikh doi: 10.3390/jmahp14020029 Journal of Market Access & Health Policy 2026-05-06 Journal of Market Access & Health Policy 2026-05-06 14 2 Article 29 10.3390/jmahp14020029 https://www.mdpi.com/2001-6689/14/2/29 JMAHP, Vol. 14, Pages 28: Assessment and Appraisal of Drug Innovativeness in Italy: Ultimate Evidence on Key Drivers and Consistency https://www.mdpi.com/2001-6689/14/2/28 This study aims to update and integrate empirical evidence on the key drivers and consistency of the appraisals of drug innovativeness in Italy by the Italian Medicines Agency (AIFA), and discuss if this evidence is supportive of the reform and requirements implemented in 2025. Appraisals from July 2017 to December 2024 were retrieved from the AIFA website. The association between the innovativeness appraisal, the innovativeness domains (unmet need/added therapeutic value/quality of evidence) and disease/drug/evidence-specific variables was assessed using odds ratios (ORs) from binary/multinomial logistic regression models. Innovativeness status was strongly associated with added therapeutic value (OR > 70). Medicines for rare diseases were more likely to receive conditional innovativeness (OR = 2.95). Full innovativeness was more frequently recognized for indications including paediatric patients (OR = 3.60). References to severe diseases and patient-reported outcomes (PROs) had a higher, not statistically significant, likelihood of innovativeness, whereas reference to indirect treatment comparisons had a lower likelihood (OR = 0.18). The appraisal process showed high internal consistency, but its regulation needs more specific guidance. The innovativeness regulation was reformed in July 2025, including specific recommendations on the criteria to identify the alternative treatments; the role and robustness of indirect comparisons; and the role and requirements for PROs. Our evidence provides an empirical rationale for this reform. 2026-05-02 JMAHP, Vol. 14, Pages 28: Assessment and Appraisal of Drug Innovativeness in Italy: Ultimate Evidence on Key Drivers and Consistency

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020028

Authors: Alvise Verde Federica Turati Clara Trimarchi Carlotta Galeone Claudio Jommi

This study aims to update and integrate empirical evidence on the key drivers and consistency of the appraisals of drug innovativeness in Italy by the Italian Medicines Agency (AIFA), and discuss if this evidence is supportive of the reform and requirements implemented in 2025. Appraisals from July 2017 to December 2024 were retrieved from the AIFA website. The association between the innovativeness appraisal, the innovativeness domains (unmet need/added therapeutic value/quality of evidence) and disease/drug/evidence-specific variables was assessed using odds ratios (ORs) from binary/multinomial logistic regression models. Innovativeness status was strongly associated with added therapeutic value (OR > 70). Medicines for rare diseases were more likely to receive conditional innovativeness (OR = 2.95). Full innovativeness was more frequently recognized for indications including paediatric patients (OR = 3.60). References to severe diseases and patient-reported outcomes (PROs) had a higher, not statistically significant, likelihood of innovativeness, whereas reference to indirect treatment comparisons had a lower likelihood (OR = 0.18). The appraisal process showed high internal consistency, but its regulation needs more specific guidance. The innovativeness regulation was reformed in July 2025, including specific recommendations on the criteria to identify the alternative treatments; the role and robustness of indirect comparisons; and the role and requirements for PROs. Our evidence provides an empirical rationale for this reform.

]]> Assessment and Appraisal of Drug Innovativeness in Italy: Ultimate Evidence on Key Drivers and Consistency Alvise Verde Federica Turati Clara Trimarchi Carlotta Galeone Claudio Jommi doi: 10.3390/jmahp14020028 Journal of Market Access & Health Policy 2026-05-02 Journal of Market Access & Health Policy 2026-05-02 14 2 Article 28 10.3390/jmahp14020028 https://www.mdpi.com/2001-6689/14/2/28 JMAHP, Vol. 14, Pages 27: Primary Care or Primary Problem? Aligning Access Pathways with Patient Needs Across the Care Continuum https://www.mdpi.com/2001-6689/14/2/27 In the United States, access to healthcare is shaped not only by patient need but also by payer policies that determine which providers are reimbursable, how care is sequenced, and what constitutes a legitimate entry point into the system. These gatekeeping functions, while valuable for supporting clinical prioritization, risk stratification, and continuity of care, can also unintentionally reinforce structural inequities and credential hierarchies that delay or limit timely and equitable care, particularly for historically marginalized populations. While reform efforts often focus on expanding benefits or provider networks, fewer address the underlying design of access itself or the rules that govern how patients enter care. It is argued in this paper that a more equitable and efficient healthcare system requires multi-entry care models, in which nurses, behavioral health clinicians, pharmacists, and community health workers may serve as condition-appropriate, reimbursable first points of contact within coordinated care teams. Drawing on evidence from Medicare, Medicaid, the Veterans Health Administration, and commercial payers, these models may support cost containment, improve care coordination, facilitate appropriate utilization, and promote earlier patient engagement. While findings from these models are not uniform across all settings, evidence suggests that outcomes are highly dependent on implementation context, system design, and supporting infrastructure. When implemented with appropriate safeguards (such as interoperable health records, team-based care requirements, and coordinated referral tracking), multi-entry systems can preserve continuity while expanding access. Payers are uniquely positioned to lead this transformation by aligning reimbursement policy with patient needs, supporting team-based care infrastructure, and embedding accountability into access pathways, thereby creating a system that can be more responsive, inclusive, and sustainable. 2026-05-01 JMAHP, Vol. 14, Pages 27: Primary Care or Primary Problem? Aligning Access Pathways with Patient Needs Across the Care Continuum

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020027

Authors: Gregory J. Privitera James J. Gillespie Alexa Walton

In the United States, access to healthcare is shaped not only by patient need but also by payer policies that determine which providers are reimbursable, how care is sequenced, and what constitutes a legitimate entry point into the system. These gatekeeping functions, while valuable for supporting clinical prioritization, risk stratification, and continuity of care, can also unintentionally reinforce structural inequities and credential hierarchies that delay or limit timely and equitable care, particularly for historically marginalized populations. While reform efforts often focus on expanding benefits or provider networks, fewer address the underlying design of access itself or the rules that govern how patients enter care. It is argued in this paper that a more equitable and efficient healthcare system requires multi-entry care models, in which nurses, behavioral health clinicians, pharmacists, and community health workers may serve as condition-appropriate, reimbursable first points of contact within coordinated care teams. Drawing on evidence from Medicare, Medicaid, the Veterans Health Administration, and commercial payers, these models may support cost containment, improve care coordination, facilitate appropriate utilization, and promote earlier patient engagement. While findings from these models are not uniform across all settings, evidence suggests that outcomes are highly dependent on implementation context, system design, and supporting infrastructure. When implemented with appropriate safeguards (such as interoperable health records, team-based care requirements, and coordinated referral tracking), multi-entry systems can preserve continuity while expanding access. Payers are uniquely positioned to lead this transformation by aligning reimbursement policy with patient needs, supporting team-based care infrastructure, and embedding accountability into access pathways, thereby creating a system that can be more responsive, inclusive, and sustainable.

]]> Primary Care or Primary Problem? Aligning Access Pathways with Patient Needs Across the Care Continuum Gregory J. Privitera James J. Gillespie Alexa Walton doi: 10.3390/jmahp14020027 Journal of Market Access & Health Policy 2026-05-01 Journal of Market Access & Health Policy 2026-05-01 14 2 Commentary 27 10.3390/jmahp14020027 https://www.mdpi.com/2001-6689/14/2/27 JMAHP, Vol. 14, Pages 26: Real-World Shelf Life of Adrenaline Auto-Injectors at Pharmacies in Denmark, Finland, Sweden, and Norway https://www.mdpi.com/2001-6689/14/2/26 There is a well-documented gap between the prescription of adrenaline auto-injectors (AAIs) and their real-world use during anaphylaxis. Although several aspects of AAI underuse have been investigated, the potential role of shelf life in influencing patient adherence has not been quantified. This study assessed the real-world remaining shelf life of AAIs available at pharmacies in Denmark, Finland, Sweden, and Norway, using pharmacy-level stock data and pharmacy employee-reported perceptions. Across Denmark, Finland, and Sweden, the average remaining shelf life was 9.6 months, and in Norway it was 10.5 months at the point of dispensing. In Denmark, Finland and Sweden, 100%, 91%, and 94% of employees, respectively, considered shelf life an important or very important factor when dispensing AAIs to patients. Our findings suggest that patients and caregivers filling prescriptions for AAIs frequently receive devices with limited remaining shelf life, which may necessitate multiple renewals per year. This has potential implications in terms of adherence to clinical guidelines, dependence of expired devices during emergencies, patient cost, caregiver burden, and overall societal expenditure. These results highlight an unmet need for emergency treatment options with longer shelf life to better support continuous access to life-saving medicine during anaphylaxis. 2026-05-01 JMAHP, Vol. 14, Pages 26: Real-World Shelf Life of Adrenaline Auto-Injectors at Pharmacies in Denmark, Finland, Sweden, and Norway

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020026

Authors: Jesper Nørregaard Christoffer Mertz Anne Danø Jeppe Hæstrup Kamstrup Mille Vang Lybech

There is a well-documented gap between the prescription of adrenaline auto-injectors (AAIs) and their real-world use during anaphylaxis. Although several aspects of AAI underuse have been investigated, the potential role of shelf life in influencing patient adherence has not been quantified. This study assessed the real-world remaining shelf life of AAIs available at pharmacies in Denmark, Finland, Sweden, and Norway, using pharmacy-level stock data and pharmacy employee-reported perceptions. Across Denmark, Finland, and Sweden, the average remaining shelf life was 9.6 months, and in Norway it was 10.5 months at the point of dispensing. In Denmark, Finland and Sweden, 100%, 91%, and 94% of employees, respectively, considered shelf life an important or very important factor when dispensing AAIs to patients. Our findings suggest that patients and caregivers filling prescriptions for AAIs frequently receive devices with limited remaining shelf life, which may necessitate multiple renewals per year. This has potential implications in terms of adherence to clinical guidelines, dependence of expired devices during emergencies, patient cost, caregiver burden, and overall societal expenditure. These results highlight an unmet need for emergency treatment options with longer shelf life to better support continuous access to life-saving medicine during anaphylaxis.

]]> Real-World Shelf Life of Adrenaline Auto-Injectors at Pharmacies in Denmark, Finland, Sweden, and Norway Jesper Nørregaard Christoffer Mertz Anne Danø Jeppe Hæstrup Kamstrup Mille Vang Lybech doi: 10.3390/jmahp14020026 Journal of Market Access & Health Policy 2026-05-01 Journal of Market Access & Health Policy 2026-05-01 14 2 Article 26 10.3390/jmahp14020026 https://www.mdpi.com/2001-6689/14/2/26 JMAHP, Vol. 14, Pages 25: Impact of Market Access Delays on Time to Patient Access: Multi-Country Comparative Analysis Assessing the First Commercial Launch Indications for Five Oncology Medicines Across Europe and Canada https://www.mdpi.com/2001-6689/14/2/25 Background: The benefit of pharmaceutical innovation manifests when patients access treatment. Following regulatory approval in Europe and Canada, reimbursement decisions depend on health technology assessments (HTAs), which can be prolonged. To quantify the impact of delays on patients, we evaluated market access timelines for olaparib, osimertinib, durvalumab, acalabrutinib, and trastuzumab deruxtecan across six high-income countries with established HTA systems (Canada, England, France, Germany, Italy, Spain). Methods: Time to access was from regulatory approval to reimbursement. Survival benefit was median overall survival (OS) and progression-free survival (PFS) assessed versus the comparator at approval and the latest data cut-off. The number of eligible patients per year multiplied by the years to patient access and survival benefit reflects the lost survival benefit. Results: Efficacy benefits observed at approval continued to the latest data cut-offs. The mean time to patient access was 18 months. Although this varied by country and treatment, with England and Germany typically being the fastest and France and Spain the slowest, timelines often exceeded the 180-day EU target despite identical evidence used in HTA submissions. This resulted in an estimated mean of 2836 patients being unable to access treatment and 3391 OS-derived and 2739 PFS-derived life-years lost. Conclusions: Access processes must evolve to ensure the timely realization of new medicines’ benefits. 2026-04-28 JMAHP, Vol. 14, Pages 25: Impact of Market Access Delays on Time to Patient Access: Multi-Country Comparative Analysis Assessing the First Commercial Launch Indications for Five Oncology Medicines Across Europe and Canada

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020025

Authors: Barry Crean David Parry Alison Horsfield James Ryan Nektarios Oraiopoulos

Background: The benefit of pharmaceutical innovation manifests when patients access treatment. Following regulatory approval in Europe and Canada, reimbursement decisions depend on health technology assessments (HTAs), which can be prolonged. To quantify the impact of delays on patients, we evaluated market access timelines for olaparib, osimertinib, durvalumab, acalabrutinib, and trastuzumab deruxtecan across six high-income countries with established HTA systems (Canada, England, France, Germany, Italy, Spain). Methods: Time to access was from regulatory approval to reimbursement. Survival benefit was median overall survival (OS) and progression-free survival (PFS) assessed versus the comparator at approval and the latest data cut-off. The number of eligible patients per year multiplied by the years to patient access and survival benefit reflects the lost survival benefit. Results: Efficacy benefits observed at approval continued to the latest data cut-offs. The mean time to patient access was 18 months. Although this varied by country and treatment, with England and Germany typically being the fastest and France and Spain the slowest, timelines often exceeded the 180-day EU target despite identical evidence used in HTA submissions. This resulted in an estimated mean of 2836 patients being unable to access treatment and 3391 OS-derived and 2739 PFS-derived life-years lost. Conclusions: Access processes must evolve to ensure the timely realization of new medicines’ benefits.

]]> Impact of Market Access Delays on Time to Patient Access: Multi-Country Comparative Analysis Assessing the First Commercial Launch Indications for Five Oncology Medicines Across Europe and Canada Barry Crean David Parry Alison Horsfield James Ryan Nektarios Oraiopoulos doi: 10.3390/jmahp14020025 Journal of Market Access & Health Policy 2026-04-28 Journal of Market Access & Health Policy 2026-04-28 14 2 Article 25 10.3390/jmahp14020025 https://www.mdpi.com/2001-6689/14/2/25 JMAHP, Vol. 14, Pages 24: Introducing an Expanded Value Framework in Health Technology Assessment of Vaccines https://www.mdpi.com/2001-6689/14/2/24 Health Technology Assessment (HTA) frameworks increasingly recognize the broader value elements of vaccines; however, their adoption remains inconsistent across jurisdictions and often incomplete in practice. Many HTA processes continue to prioritize narrow clinical outcomes and direct costs, leading to the underrepresentation of the full preventive and long-term benefits of vaccination. Building on the ISPOR “Elements of Value” framework and recent evidence, this study adapts and expands existing models specifically for vaccines to enhance HTA applicability in both high-income and resource-limited settings. We introduce an updated vaccine value framework comprising 21 distinct value elements. Notably, the original model was expanded by introducing four entirely new value drivers: (1) real-world evidence; (2) control of antimicrobial resistance; (3) health system strengthening; and (4) environmental impact. Additionally, existing elements were refined, such as broadening “fear of contagion” to “peace of mind” and expanding “productivity” to capture education and leisure gains. We map these elements to potential data sources and methodological tools to facilitate their inclusion in HTA. This study offers an operational, holistic, and context-sensitive framework that reflects current advancements in assessment. By capturing the full spectrum of vaccine value, this framework aims to support more comprehensive, transparent, and equitable HTA decision-making for global immunization programs, while considering conceptual overlap between value elements to reduce the risk of double counting. 2026-04-28 JMAHP, Vol. 14, Pages 24: Introducing an Expanded Value Framework in Health Technology Assessment of Vaccines

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020024

Authors: Farzaneh Eslami Thi Hao Pham Angga P. Kautsar Cao Ba Khuong Cornelis Boersma Mondher Toumi Jurjen van der Schans Maarten J. Postma

Health Technology Assessment (HTA) frameworks increasingly recognize the broader value elements of vaccines; however, their adoption remains inconsistent across jurisdictions and often incomplete in practice. Many HTA processes continue to prioritize narrow clinical outcomes and direct costs, leading to the underrepresentation of the full preventive and long-term benefits of vaccination. Building on the ISPOR “Elements of Value” framework and recent evidence, this study adapts and expands existing models specifically for vaccines to enhance HTA applicability in both high-income and resource-limited settings. We introduce an updated vaccine value framework comprising 21 distinct value elements. Notably, the original model was expanded by introducing four entirely new value drivers: (1) real-world evidence; (2) control of antimicrobial resistance; (3) health system strengthening; and (4) environmental impact. Additionally, existing elements were refined, such as broadening “fear of contagion” to “peace of mind” and expanding “productivity” to capture education and leisure gains. We map these elements to potential data sources and methodological tools to facilitate their inclusion in HTA. This study offers an operational, holistic, and context-sensitive framework that reflects current advancements in assessment. By capturing the full spectrum of vaccine value, this framework aims to support more comprehensive, transparent, and equitable HTA decision-making for global immunization programs, while considering conceptual overlap between value elements to reduce the risk of double counting.

]]> Introducing an Expanded Value Framework in Health Technology Assessment of Vaccines Farzaneh Eslami Thi Hao Pham Angga P. Kautsar Cao Ba Khuong Cornelis Boersma Mondher Toumi Jurjen van der Schans Maarten J. Postma doi: 10.3390/jmahp14020024 Journal of Market Access & Health Policy 2026-04-28 Journal of Market Access & Health Policy 2026-04-28 14 2 Perspective 24 10.3390/jmahp14020024 https://www.mdpi.com/2001-6689/14/2/24 JMAHP, Vol. 14, Pages 23: When Do Annuity-Based Payments Help to Address the Affordability Challenge of Funding Advanced Therapies? Insights from a Budget Impact Simulation https://www.mdpi.com/2001-6689/14/2/23 Spreading payments over time by means of annuities has been proposed as a means to address the affordability challenge of funding very expensive advanced therapies, especially within managed entry agreements. This study aims to examine when annuities (in contrast with a single upfront payment) offer a viable solution for both healthcare payers and manufacturers to fund one-time advanced therapies. We put forward four conditions under which annuity-based payments can be considered an acceptable payment strategy: (1) excessive budget impact, (2) cost equivalence with upfront payment, (3) compensation for financial risk and (4) a limited annuity period. We develop an exploratory model that simulates how the budget impact of annuity-based payments for advanced therapies meets these conditions across several economic and epidemiological scenarios. Given our model parameter values, results suggest that annuity-based payments are most suitable when the initial patient volume (prevalence) significantly exceeds annual new cases (incidence), and when the financial risk premium for the annuity-based payment scheme does not exceed the social discount rate. While further refinement of the model is needed, this study demonstrates that annuity-based payments can only help control the annual budget need when the focus is on a high-prevalence disease, and the therapy is financed through health impact bonds issued by a governmental payer. This arrangement ensures a low-risk premium, which is typically only available to public payers. 2026-04-20 JMAHP, Vol. 14, Pages 23: When Do Annuity-Based Payments Help to Address the Affordability Challenge of Funding Advanced Therapies? Insights from a Budget Impact Simulation

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020023

Authors: Walter Van Dyck Sissel Michelsen David Veredas Isabelle Huys Jeroen Luyten Steven Simoens

Spreading payments over time by means of annuities has been proposed as a means to address the affordability challenge of funding very expensive advanced therapies, especially within managed entry agreements. This study aims to examine when annuities (in contrast with a single upfront payment) offer a viable solution for both healthcare payers and manufacturers to fund one-time advanced therapies. We put forward four conditions under which annuity-based payments can be considered an acceptable payment strategy: (1) excessive budget impact, (2) cost equivalence with upfront payment, (3) compensation for financial risk and (4) a limited annuity period. We develop an exploratory model that simulates how the budget impact of annuity-based payments for advanced therapies meets these conditions across several economic and epidemiological scenarios. Given our model parameter values, results suggest that annuity-based payments are most suitable when the initial patient volume (prevalence) significantly exceeds annual new cases (incidence), and when the financial risk premium for the annuity-based payment scheme does not exceed the social discount rate. While further refinement of the model is needed, this study demonstrates that annuity-based payments can only help control the annual budget need when the focus is on a high-prevalence disease, and the therapy is financed through health impact bonds issued by a governmental payer. This arrangement ensures a low-risk premium, which is typically only available to public payers.

]]> When Do Annuity-Based Payments Help to Address the Affordability Challenge of Funding Advanced Therapies? Insights from a Budget Impact Simulation Walter Van Dyck Sissel Michelsen David Veredas Isabelle Huys Jeroen Luyten Steven Simoens doi: 10.3390/jmahp14020023 Journal of Market Access & Health Policy 2026-04-20 Journal of Market Access & Health Policy 2026-04-20 14 2 Article 23 10.3390/jmahp14020023 https://www.mdpi.com/2001-6689/14/2/23 JMAHP, Vol. 14, Pages 22: Household Out-of-Pocket Burden Costs for Pediatric Pneumonia in Low- and Middle-Income Countries: Evidence Review and Econometric Framework https://www.mdpi.com/2001-6689/14/2/22 Pediatric pneumonia remains a major cause of morbidity and mortality in low- and middle-income countries (LMICs), imposing both health and financial burdens. While the clinical aspects of pediatric pneumonia are well-studied, less attention has been paid to its economic implications for households, particularly regarding out-of-pocket (OOP) expenditure. This paper synthesizes current evidence from Kenya, India, Bangladesh, and Vietnam and introduces a proposed econometric framework designed to identify cost determinants and model policy interventions. The framework integrates microeconomic data, identifies cost determinants, and models the effects of clinical and policy factors (e.g., intensive care, vaccination, insurance coverage) on household expenditures. Simulated results illustrate potential findings from such an approach. Existing studies show substantial variability in hospitalization costs, with OOP payments ranging from US$30 to US$250 per episode, often exceeding 20% of monthly household income. Econometric modeling using generalized linear models (GLMs) and difference-in-differences (DiD) can disentangle the impact of hospital practices, disease severity, and policy interventions. Simulated regression results demonstrate that length of stay, intensive care admission, and absence of insurance significantly increase household costs, while pneumococcal conjugate vaccine (PCV) introduction reduces both admissions and financial burden. Hospitalization for pediatric pneumonia imposes significant OOP costs on households in LMICs. An econometric framework provides rigorous tools to estimate cost drivers, evaluate policy impacts, and guide equitable health financing reforms. 2026-04-13 JMAHP, Vol. 14, Pages 22: Household Out-of-Pocket Burden Costs for Pediatric Pneumonia in Low- and Middle-Income Countries: Evidence Review and Econometric Framework

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020022

Authors: Ioannis Smaraidos Maria Kyrmanidou Asterios Kampouras

Pediatric pneumonia remains a major cause of morbidity and mortality in low- and middle-income countries (LMICs), imposing both health and financial burdens. While the clinical aspects of pediatric pneumonia are well-studied, less attention has been paid to its economic implications for households, particularly regarding out-of-pocket (OOP) expenditure. This paper synthesizes current evidence from Kenya, India, Bangladesh, and Vietnam and introduces a proposed econometric framework designed to identify cost determinants and model policy interventions. The framework integrates microeconomic data, identifies cost determinants, and models the effects of clinical and policy factors (e.g., intensive care, vaccination, insurance coverage) on household expenditures. Simulated results illustrate potential findings from such an approach. Existing studies show substantial variability in hospitalization costs, with OOP payments ranging from US$30 to US$250 per episode, often exceeding 20% of monthly household income. Econometric modeling using generalized linear models (GLMs) and difference-in-differences (DiD) can disentangle the impact of hospital practices, disease severity, and policy interventions. Simulated regression results demonstrate that length of stay, intensive care admission, and absence of insurance significantly increase household costs, while pneumococcal conjugate vaccine (PCV) introduction reduces both admissions and financial burden. Hospitalization for pediatric pneumonia imposes significant OOP costs on households in LMICs. An econometric framework provides rigorous tools to estimate cost drivers, evaluate policy impacts, and guide equitable health financing reforms.

]]> Household Out-of-Pocket Burden Costs for Pediatric Pneumonia in Low- and Middle-Income Countries: Evidence Review and Econometric Framework Ioannis Smaraidos Maria Kyrmanidou Asterios Kampouras doi: 10.3390/jmahp14020022 Journal of Market Access & Health Policy 2026-04-13 Journal of Market Access & Health Policy 2026-04-13 14 2 Review 22 10.3390/jmahp14020022 https://www.mdpi.com/2001-6689/14/2/22 JMAHP, Vol. 14, Pages 21: Accessibility of Biologic Drugs in Morocco: Comparison with FDA and EMA Approvals (2015–2025) https://www.mdpi.com/2001-6689/14/2/21 This study aims to evaluate the rates and timeframes of the availability and reimbursement of biologic drugs in Morocco, after approval by the Food and Drug Administration (FDA) or the European Medicines Agency (EMA). The results will help to identify disparities in access and promote rapid access to these innovative treatments. This descriptive study established an international reference list of biological medicines, based on data from the FDA and EMA for the period from 2015 to 2025. An analysis was conducted using national sources, focusing on the availability, reimbursement rates, and timeframes for each listed biological drug. Of the 233 listed biological drugs, only 13.7% (32/233) of those approved between 2015 and 2025 are available in Morocco. Of these, 87.5% (28/32) have been priced, and only 10.7% (3/28) have been approved for reimbursement. The average time between FDA/EMA approval and pricing in Morocco is 3.75 years and 3.41 years, respectively, while the average reimbursement approval time is 2.74 years. This study highlights the delay and limited access for Moroccan patients to internationally approved biologic drugs. 2026-04-09 JMAHP, Vol. 14, Pages 21: Accessibility of Biologic Drugs in Morocco: Comparison with FDA and EMA Approvals (2015–2025)

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020021

Authors: Mounir Charrak Yahia Cherrah Samira Serragui

This study aims to evaluate the rates and timeframes of the availability and reimbursement of biologic drugs in Morocco, after approval by the Food and Drug Administration (FDA) or the European Medicines Agency (EMA). The results will help to identify disparities in access and promote rapid access to these innovative treatments. This descriptive study established an international reference list of biological medicines, based on data from the FDA and EMA for the period from 2015 to 2025. An analysis was conducted using national sources, focusing on the availability, reimbursement rates, and timeframes for each listed biological drug. Of the 233 listed biological drugs, only 13.7% (32/233) of those approved between 2015 and 2025 are available in Morocco. Of these, 87.5% (28/32) have been priced, and only 10.7% (3/28) have been approved for reimbursement. The average time between FDA/EMA approval and pricing in Morocco is 3.75 years and 3.41 years, respectively, while the average reimbursement approval time is 2.74 years. This study highlights the delay and limited access for Moroccan patients to internationally approved biologic drugs.

]]> Accessibility of Biologic Drugs in Morocco: Comparison with FDA and EMA Approvals (2015–2025) Mounir Charrak Yahia Cherrah Samira Serragui doi: 10.3390/jmahp14020021 Journal of Market Access & Health Policy 2026-04-09 Journal of Market Access & Health Policy 2026-04-09 14 2 Article 21 10.3390/jmahp14020021 https://www.mdpi.com/2001-6689/14/2/21 JMAHP, Vol. 14, Pages 20: Mapping the Use of Real-World Evidence Across the EU Health Technology Assessment Regulation: Methodological Considerations, Challenges, and Opportunities for Harmonization https://www.mdpi.com/2001-6689/14/2/20 Methodological guidelines for real-world evidence (RWE) in European Union (EU) joint clinical assessments (JCA) are lacking. This manuscript explores RWE potential in EU health technology assessment (HTA) and offers recommendations for generating high-quality RWE. An environmental scan of peer-reviewed and gray literature was conducted to review RWE frameworks and documents in EU regulatory and HTA decision-making. Extraction elements were standardized across key RWE themes: data quality, methodological rigor, stakeholder engagement, and applications. In JCA, RWE has multiple uses, including informing PICO simulation exercises, understanding disease landscape, identifying prognostic factors and effect modifiers, and directly or indirectly informing comparative clinical assessments. Methodological guidance from the HTA Coordination Group is limited to cases in which evidence from non-randomized studies is used as direct inputs in comparative assessments. Individual HTA bodies provide more detailed guidance, missing an opportunity to leverage RWE within JCAs that can offer insight for local Member State submissions. Generating high-quality RWE that is credible, actionable, and acceptable for JCA submissions and local HTA bodies requires careful attention to methodological considerations and early planning. Broader RWE integration that reflects patient journeys is needed. Expanding the HTA Coordination Group guidance can unlock RWE’s full potential in supporting EU JCA submissions. 2026-04-08 JMAHP, Vol. 14, Pages 20: Mapping the Use of Real-World Evidence Across the EU Health Technology Assessment Regulation: Methodological Considerations, Challenges, and Opportunities for Harmonization

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020020

Authors: Grammati Sarri Bengt Liljas Keith R. Abrams Stephen J. Duffield Murtuza Bharmal

Methodological guidelines for real-world evidence (RWE) in European Union (EU) joint clinical assessments (JCA) are lacking. This manuscript explores RWE potential in EU health technology assessment (HTA) and offers recommendations for generating high-quality RWE. An environmental scan of peer-reviewed and gray literature was conducted to review RWE frameworks and documents in EU regulatory and HTA decision-making. Extraction elements were standardized across key RWE themes: data quality, methodological rigor, stakeholder engagement, and applications. In JCA, RWE has multiple uses, including informing PICO simulation exercises, understanding disease landscape, identifying prognostic factors and effect modifiers, and directly or indirectly informing comparative clinical assessments. Methodological guidance from the HTA Coordination Group is limited to cases in which evidence from non-randomized studies is used as direct inputs in comparative assessments. Individual HTA bodies provide more detailed guidance, missing an opportunity to leverage RWE within JCAs that can offer insight for local Member State submissions. Generating high-quality RWE that is credible, actionable, and acceptable for JCA submissions and local HTA bodies requires careful attention to methodological considerations and early planning. Broader RWE integration that reflects patient journeys is needed. Expanding the HTA Coordination Group guidance can unlock RWE’s full potential in supporting EU JCA submissions.

]]> Mapping the Use of Real-World Evidence Across the EU Health Technology Assessment Regulation: Methodological Considerations, Challenges, and Opportunities for Harmonization Grammati Sarri Bengt Liljas Keith R. Abrams Stephen J. Duffield Murtuza Bharmal doi: 10.3390/jmahp14020020 Journal of Market Access & Health Policy 2026-04-08 Journal of Market Access & Health Policy 2026-04-08 14 2 Article 20 10.3390/jmahp14020020 https://www.mdpi.com/2001-6689/14/2/20 JMAHP, Vol. 14, Pages 19: When Activism Becomes Survival: The Mental Health Costs of Constant Resistance in the Digital Era in the Balkans—A Health Policy Perspective https://www.mdpi.com/2001-6689/14/2/19 Activism exposes individuals to sustained harassment, threat and psychological strain in contexts marked by discrimination and weak institutional protection. For LGBTQ communities, public engagement frequently increases vulnerability to both offline and digital harm, with cumulative consequences for mental health. Using the Balkans as a case example, this perspective sees activist mental health through a public health and health policy lens, framing distress not as an individual coping failure but as an outcome of structural barriers and minority stress processes, including inadequate legal protection, limited access to culturally competent mental health care and insufficient accountability for platform-mediated harm. This article highlights the population-level implications of unaddressed structural stressors, like burnout, disengagement and reduced sustainability of civil society participation, by situating activist mental health within broader questions of health system performance, access to care and governance. Upstream policy responses that strengthen institutional protection, ensure equitable access to mental health services and promote safer digital environments would address these challenges, positioning activist mental health as a critical public health policy issue. 2026-04-02 JMAHP, Vol. 14, Pages 19: When Activism Becomes Survival: The Mental Health Costs of Constant Resistance in the Digital Era in the Balkans—A Health Policy Perspective

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020019

Authors: Aleksandar Sič Svetozar Mijuskovic Nebojsa Brezic

Activism exposes individuals to sustained harassment, threat and psychological strain in contexts marked by discrimination and weak institutional protection. For LGBTQ communities, public engagement frequently increases vulnerability to both offline and digital harm, with cumulative consequences for mental health. Using the Balkans as a case example, this perspective sees activist mental health through a public health and health policy lens, framing distress not as an individual coping failure but as an outcome of structural barriers and minority stress processes, including inadequate legal protection, limited access to culturally competent mental health care and insufficient accountability for platform-mediated harm. This article highlights the population-level implications of unaddressed structural stressors, like burnout, disengagement and reduced sustainability of civil society participation, by situating activist mental health within broader questions of health system performance, access to care and governance. Upstream policy responses that strengthen institutional protection, ensure equitable access to mental health services and promote safer digital environments would address these challenges, positioning activist mental health as a critical public health policy issue.

]]> When Activism Becomes Survival: The Mental Health Costs of Constant Resistance in the Digital Era in the Balkans—A Health Policy Perspective Aleksandar Sič Svetozar Mijuskovic Nebojsa Brezic doi: 10.3390/jmahp14020019 Journal of Market Access & Health Policy 2026-04-02 Journal of Market Access & Health Policy 2026-04-02 14 2 Perspective 19 10.3390/jmahp14020019 https://www.mdpi.com/2001-6689/14/2/19 JMAHP, Vol. 14, Pages 18: Cost-Effectiveness of 3D-Printed Patient-Specific Versus Off-the-Shelf Interbody Cages in Lumbar Spinal Fusion: A Markov Model Cost-Utility Analysis https://www.mdpi.com/2001-6689/14/2/18 The aim of the present study was to compare the cost-effectiveness of 3DMorphic’s spinal 3DFusion Lumbar (3DFL) cages versus Off-The-Shelf (OTS) cages for patients undergoing lumbar interbody fusion in an Australian healthcare setting. 3DFL cages differ from generic OTS cages in that they are Patient-Specific Interbody Cages (PSICs). While several studies have discussed the clinical benefits of PSIC versus OTS cages, no studies have evaluated the cost-effectiveness of this technology. Without a direct randomised controlled trial between the two implant categories, an indirect treatment comparison was performed. The indirect comparison was informed by a clinical trial of 3DFL cages, the Australian Spine Registry and an analysis of reoperation rates for patients undergoing spinal fusion in an Australian cohort. In conclusion, the PSICs were demonstrated to be clinically superior to OTS cages as measured by Health Related Quality of Life (HRQoL) and reoperation rates. The cost-utility analysis demonstrated that 3DFL cages were cost-effective compared to OTS cages in an Australian healthcare setting. 2026-03-25 JMAHP, Vol. 14, Pages 18: Cost-Effectiveness of 3D-Printed Patient-Specific Versus Off-the-Shelf Interbody Cages in Lumbar Spinal Fusion: A Markov Model Cost-Utility Analysis

Journal of Market Access & Health Policy doi: 10.3390/jmahp14020018

Authors: Jackson C. Hill Ralph J. Mobbs Marc Coughlan Kevin A. Seex Chloe A. Amaro William R. Walsh William C. H. Parr

The aim of the present study was to compare the cost-effectiveness of 3DMorphic’s spinal 3DFusion Lumbar (3DFL) cages versus Off-The-Shelf (OTS) cages for patients undergoing lumbar interbody fusion in an Australian healthcare setting. 3DFL cages differ from generic OTS cages in that they are Patient-Specific Interbody Cages (PSICs). While several studies have discussed the clinical benefits of PSIC versus OTS cages, no studies have evaluated the cost-effectiveness of this technology. Without a direct randomised controlled trial between the two implant categories, an indirect treatment comparison was performed. The indirect comparison was informed by a clinical trial of 3DFL cages, the Australian Spine Registry and an analysis of reoperation rates for patients undergoing spinal fusion in an Australian cohort. In conclusion, the PSICs were demonstrated to be clinically superior to OTS cages as measured by Health Related Quality of Life (HRQoL) and reoperation rates. The cost-utility analysis demonstrated that 3DFL cages were cost-effective compared to OTS cages in an Australian healthcare setting.

]]> Cost-Effectiveness of 3D-Printed Patient-Specific Versus Off-the-Shelf Interbody Cages in Lumbar Spinal Fusion: A Markov Model Cost-Utility Analysis Jackson C. Hill Ralph J. Mobbs Marc Coughlan Kevin A. Seex Chloe A. Amaro William R. Walsh William C. H. Parr doi: 10.3390/jmahp14020018 Journal of Market Access & Health Policy 2026-03-25 Journal of Market Access & Health Policy 2026-03-25 14 2 Article 18 10.3390/jmahp14020018 https://www.mdpi.com/2001-6689/14/2/18 JMAHP, Vol. 14, Pages 17: Comparative Cost Evaluation of Managed Entry Agreement Techniques Using Real-World Data from High-Cost Anticancer Drugs in Thailand https://www.mdpi.com/2001-6689/14/1/17 High-cost innovative anticancer drugs pose challenges for health systems in balancing timely patient access with long-term financial sustainability. In Thailand, reliance on Health Technology Assessment for reimbursement decisions may delay access, highlighting the potential role of Managed Entry Agreements (MEAs) as complementary policy instruments to manage uncertainty related to price, effectiveness, and use; however, MEA application remains limited and lacks an analytical framework for technique selection. This study used real-world data from Thammasat University Hospital to examine and compare the cost-saving performance of five MEA techniques—discount, free initiation treatment, utilization cap, conditional treatment continuation, and pay-by-result—across six high-cost anticancer drugs representing dominant uncertainty characteristics. Drug procurement costs were modeled over a 24-month horizon from the payer’s perspective, and one-way sensitivity analyses were conducted using ±10% variation in median progression-free survival. Free initiation treatment generated the highest cost savings across uncertainty types, followed by conditional treatment continuation, while utilization cap and discount produced more moderate savings. Pay-by-result demonstrated the lowest cost-saving potential. Sensitivity analyses confirmed the robustness of comparative rankings. Overall, the findings indicate that MEA performance varies according to dominant sources of drug-related uncertainty and support a more structured, context-appropriate approach to MEA selection to strengthen market access and value-based pricing in Thailand. 2026-03-20 JMAHP, Vol. 14, Pages 17: Comparative Cost Evaluation of Managed Entry Agreement Techniques Using Real-World Data from High-Cost Anticancer Drugs in Thailand

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010017

Authors: Piyapat Owat Chaoncin Sooksriwong Hataiwan Ratanabunjerdkul Tuangrat Phodha

High-cost innovative anticancer drugs pose challenges for health systems in balancing timely patient access with long-term financial sustainability. In Thailand, reliance on Health Technology Assessment for reimbursement decisions may delay access, highlighting the potential role of Managed Entry Agreements (MEAs) as complementary policy instruments to manage uncertainty related to price, effectiveness, and use; however, MEA application remains limited and lacks an analytical framework for technique selection. This study used real-world data from Thammasat University Hospital to examine and compare the cost-saving performance of five MEA techniques—discount, free initiation treatment, utilization cap, conditional treatment continuation, and pay-by-result—across six high-cost anticancer drugs representing dominant uncertainty characteristics. Drug procurement costs were modeled over a 24-month horizon from the payer’s perspective, and one-way sensitivity analyses were conducted using ±10% variation in median progression-free survival. Free initiation treatment generated the highest cost savings across uncertainty types, followed by conditional treatment continuation, while utilization cap and discount produced more moderate savings. Pay-by-result demonstrated the lowest cost-saving potential. Sensitivity analyses confirmed the robustness of comparative rankings. Overall, the findings indicate that MEA performance varies according to dominant sources of drug-related uncertainty and support a more structured, context-appropriate approach to MEA selection to strengthen market access and value-based pricing in Thailand.

]]> Comparative Cost Evaluation of Managed Entry Agreement Techniques Using Real-World Data from High-Cost Anticancer Drugs in Thailand Piyapat Owat Chaoncin Sooksriwong Hataiwan Ratanabunjerdkul Tuangrat Phodha doi: 10.3390/jmahp14010017 Journal of Market Access & Health Policy 2026-03-20 Journal of Market Access & Health Policy 2026-03-20 14 1 Article 17 10.3390/jmahp14010017 https://www.mdpi.com/2001-6689/14/1/17 JMAHP, Vol. 14, Pages 16: Validation of a De Novo Health Economic Model for Finerenone in Heart Failure with Left Ventricular Ejection Fraction ≥40% https://www.mdpi.com/2001-6689/14/1/16 This study aimed to validate the health economic model for finerenone in the treatment of patients with heart failure (HF) and left ventricular ejection fraction (LVEF) ≥40% in the United Kingdom. A Markov model informed by the pivotal FINEARTS-HF trial compared finerenone + standard of care (SoC) to SoC alone. Cross-validation was performed on the results (life years [LYs] and quality adjusted life years [QALYs]) for the SoC arm against three models in HF with LVEF >40%. External validation compared cardiovascular (CV) mortality and the number of total HF events (hospitalisation for heart failure [HFF] and urgent heart failure visit [UHFV]) against FINEARTS-HF. The model estimated similar discounted outcomes to other models in HF (6.47 vs. 6.63–7.91 LYs and 4.78 vs. 4.63–5.27 QALYs). CV deaths (22 vs. 27) and UHFV events (60 vs. 61) avoided with finerenone were similar between the model and FINEARTS-HF. The broad estimated range of avoided HHF events (205–303 vs. 219 in FINEARTS-HF) was largely driven by baseline patient age. This comprehensive validation exercise demonstrated that the finerenone model accurately estimated observed clinical data and was well aligned in its projections with previous models assessing similar populations. 2026-03-11 JMAHP, Vol. 14, Pages 16: Validation of a De Novo Health Economic Model for Finerenone in Heart Failure with Left Ventricular Ejection Fraction ≥40%

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010016

Authors: Tobiasz Lemański Kerstin Folkerts Phil McEwan Paul Mernagh Mateusz Robert Żemojdzin Michał Pochopień

This study aimed to validate the health economic model for finerenone in the treatment of patients with heart failure (HF) and left ventricular ejection fraction (LVEF) ≥40% in the United Kingdom. A Markov model informed by the pivotal FINEARTS-HF trial compared finerenone + standard of care (SoC) to SoC alone. Cross-validation was performed on the results (life years [LYs] and quality adjusted life years [QALYs]) for the SoC arm against three models in HF with LVEF >40%. External validation compared cardiovascular (CV) mortality and the number of total HF events (hospitalisation for heart failure [HFF] and urgent heart failure visit [UHFV]) against FINEARTS-HF. The model estimated similar discounted outcomes to other models in HF (6.47 vs. 6.63–7.91 LYs and 4.78 vs. 4.63–5.27 QALYs). CV deaths (22 vs. 27) and UHFV events (60 vs. 61) avoided with finerenone were similar between the model and FINEARTS-HF. The broad estimated range of avoided HHF events (205–303 vs. 219 in FINEARTS-HF) was largely driven by baseline patient age. This comprehensive validation exercise demonstrated that the finerenone model accurately estimated observed clinical data and was well aligned in its projections with previous models assessing similar populations.

]]> Validation of a De Novo Health Economic Model for Finerenone in Heart Failure with Left Ventricular Ejection Fraction ≥40% Tobiasz Lemański Kerstin Folkerts Phil McEwan Paul Mernagh Mateusz Robert Żemojdzin Michał Pochopień doi: 10.3390/jmahp14010016 Journal of Market Access & Health Policy 2026-03-11 Journal of Market Access & Health Policy 2026-03-11 14 1 Article 16 10.3390/jmahp14010016 https://www.mdpi.com/2001-6689/14/1/16 JMAHP, Vol. 14, Pages 15: Addressing Unmet Medical Needs in Drug Development: Assessment and Implications for Regulatory and Clinical Development Strategies https://www.mdpi.com/2001-6689/14/1/15 Unmet need is a core component of many Health Technology Assessment (HTA) processes at EU and national level. Most visibly, it is a core selection criterion for Joint Scientific Consultations (JSC) and Joint Clinical Assessment (JCA) for medical devices. This qualitative study explored how Unmet Medical Needs (UMNs) are understood and applied in drug development, with an emphasis on the European regulatory, HTA and access context, and examined their impact on regulatory and clinical development strategies. Twenty semi-structured interviews were conducted with representatives from regulatory authorities, HTA bodies, clinical development, industry, and patient insight roles. Data was analyzed using a thematic content approach combining deductive and inductive coding. Thematic analysis revealed general agreement on the importance of addressing UMNs, but also substantial variation in how they are defined and prioritized. Regulators often stressed disease severity and clinical evidence, while patients and clinicians emphasized quality of life. HTA representatives highlighted comparative benefit and long-term outcomes. These differing perspectives shaped how UMNs were integrated into development strategies, trial design, and regulatory planning. The findings indicate that clearer yet adaptable criteria could support earlier and more consistent alignment. Based on the analysis, a five-part roadmap to guide drug development is proposed, focusing on internal coordination, structured stakeholder engagement, collaboration between regulators and HTA bodies, adaptable definitions, and transparent decision-making. Together, these elements aim to support more systematic and predictable approaches to identifying and addressing unmet needs in drug development. 2026-03-09 JMAHP, Vol. 14, Pages 15: Addressing Unmet Medical Needs in Drug Development: Assessment and Implications for Regulatory and Clinical Development Strategies

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010015

Authors: Carla Domingo-Esteban Inka Heikkinen Nanco Hefting

Unmet need is a core component of many Health Technology Assessment (HTA) processes at EU and national level. Most visibly, it is a core selection criterion for Joint Scientific Consultations (JSC) and Joint Clinical Assessment (JCA) for medical devices. This qualitative study explored how Unmet Medical Needs (UMNs) are understood and applied in drug development, with an emphasis on the European regulatory, HTA and access context, and examined their impact on regulatory and clinical development strategies. Twenty semi-structured interviews were conducted with representatives from regulatory authorities, HTA bodies, clinical development, industry, and patient insight roles. Data was analyzed using a thematic content approach combining deductive and inductive coding. Thematic analysis revealed general agreement on the importance of addressing UMNs, but also substantial variation in how they are defined and prioritized. Regulators often stressed disease severity and clinical evidence, while patients and clinicians emphasized quality of life. HTA representatives highlighted comparative benefit and long-term outcomes. These differing perspectives shaped how UMNs were integrated into development strategies, trial design, and regulatory planning. The findings indicate that clearer yet adaptable criteria could support earlier and more consistent alignment. Based on the analysis, a five-part roadmap to guide drug development is proposed, focusing on internal coordination, structured stakeholder engagement, collaboration between regulators and HTA bodies, adaptable definitions, and transparent decision-making. Together, these elements aim to support more systematic and predictable approaches to identifying and addressing unmet needs in drug development.

]]> Addressing Unmet Medical Needs in Drug Development: Assessment and Implications for Regulatory and Clinical Development Strategies Carla Domingo-Esteban Inka Heikkinen Nanco Hefting doi: 10.3390/jmahp14010015 Journal of Market Access & Health Policy 2026-03-09 Journal of Market Access & Health Policy 2026-03-09 14 1 Article 15 10.3390/jmahp14010015 https://www.mdpi.com/2001-6689/14/1/15 JMAHP, Vol. 14, Pages 14: Can We Trust PAICs in Rare Diseases? Methodological Challenges and Limitations https://www.mdpi.com/2001-6689/14/1/14 Population-adjusted indirect comparisons (PAICs), including Matching-Adjusted Indirect Comparison and Simulated Treatment Comparison, are increasingly used to inform health technology assessments. These methods offer a pragmatic approach to generating comparative evidence between treatments when head-to-head trial data are unavailable and standard indirect treatment comparison methods are unfeasible. In rare diseases, however, PAICs often face substantial methodological challenges arising from small sample sizes, limited covariate overlap, and the frequent use of unanchored comparisons that rely on unverifiable assumptions. These limitations can lead to unstable estimates, reduced precision, and bias that may undermine the reliability of findings. Methodological refinements—such as optimized weighting, Bayesian approaches, and doubly robust estimators—provide some improvements but do not resolve these fundamental issues. Current European Joint Clinical Assessment guidance recommends that anchored PAICs be applied with great caution, while unanchored PAICs are considered highly problematic, and other methods should be used instead. We argue that PAICs can play a supportive role within a multidimensional and deliberative HTA process, contributing to comparative assessment alongside other evidence sources when available data are limited. However, their results require careful interpretation and transparent communication of uncertainty. Future research should prioritize the further development of formal frameworks to quantify bias and systematically assess robustness, thereby preventing overstatement of the credibility of PAIC-derived evidence in rare disease contexts. 2026-03-06 JMAHP, Vol. 14, Pages 14: Can We Trust PAICs in Rare Diseases? Methodological Challenges and Limitations

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010014

Authors: Mikolaj Parkitny Samuel Aballéa Piotr Wojciechowski Mondher Toumi

Population-adjusted indirect comparisons (PAICs), including Matching-Adjusted Indirect Comparison and Simulated Treatment Comparison, are increasingly used to inform health technology assessments. These methods offer a pragmatic approach to generating comparative evidence between treatments when head-to-head trial data are unavailable and standard indirect treatment comparison methods are unfeasible. In rare diseases, however, PAICs often face substantial methodological challenges arising from small sample sizes, limited covariate overlap, and the frequent use of unanchored comparisons that rely on unverifiable assumptions. These limitations can lead to unstable estimates, reduced precision, and bias that may undermine the reliability of findings. Methodological refinements—such as optimized weighting, Bayesian approaches, and doubly robust estimators—provide some improvements but do not resolve these fundamental issues. Current European Joint Clinical Assessment guidance recommends that anchored PAICs be applied with great caution, while unanchored PAICs are considered highly problematic, and other methods should be used instead. We argue that PAICs can play a supportive role within a multidimensional and deliberative HTA process, contributing to comparative assessment alongside other evidence sources when available data are limited. However, their results require careful interpretation and transparent communication of uncertainty. Future research should prioritize the further development of formal frameworks to quantify bias and systematically assess robustness, thereby preventing overstatement of the credibility of PAIC-derived evidence in rare disease contexts.

]]> Can We Trust PAICs in Rare Diseases? Methodological Challenges and Limitations Mikolaj Parkitny Samuel Aballéa Piotr Wojciechowski Mondher Toumi doi: 10.3390/jmahp14010014 Journal of Market Access & Health Policy 2026-03-06 Journal of Market Access & Health Policy 2026-03-06 14 1 Perspective 14 10.3390/jmahp14010014 https://www.mdpi.com/2001-6689/14/1/14 JMAHP, Vol. 14, Pages 13: Structural Enablers of Rare Disease Treatment Coverage in Latin America and the Caribbean: Lessons from Emicizumab https://www.mdpi.com/2001-6689/14/1/13 We examine how structural characteristics of health systems in Latin America and the Caribbean (LAC) shape access to innovative therapies, using emicizumab for hemophilia A as a case study. Although the therapy is available in the region, access remains uneven and constrained by high costs and fragmented health system arrangements. Using a descriptive structural approach, we characterize the health system configurations associated with financial coverage of emicizumab across 16 LAC countries, representing more than 85% of the regional population. Regulatory approval timelines and coverage status were described, and principal component analysis (PCA) was applied to synthesize multiple indicators into a Global Characteristics Index capturing five core health system functions: resource generation, financing, service delivery, general governance, and therapy-specific governance. Coverage is defined as formal access with explicit financial protection provided by the health system. Substantial heterogeneity was observed across countries. Regulatory approval was often achieved relatively rapidly, but this did not consistently translate into timely or comprehensive coverage. Countries with stronger structural characteristics—particularly in resource generation, service delivery, and governance—tended to achieve broader and more sustained coverage, although institutional capacity alone was not sufficient in all cases. Our results emphasize the need to strengthen health governance and adopt specific policies for rare diseases in the region. 2026-02-25 JMAHP, Vol. 14, Pages 13: Structural Enablers of Rare Disease Treatment Coverage in Latin America and the Caribbean: Lessons from Emicizumab

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010013

Authors: Daniela Sugg Herrera Dino Sepúlveda Viveros Moisés Russo Namias Natalia Garrido

We examine how structural characteristics of health systems in Latin America and the Caribbean (LAC) shape access to innovative therapies, using emicizumab for hemophilia A as a case study. Although the therapy is available in the region, access remains uneven and constrained by high costs and fragmented health system arrangements. Using a descriptive structural approach, we characterize the health system configurations associated with financial coverage of emicizumab across 16 LAC countries, representing more than 85% of the regional population. Regulatory approval timelines and coverage status were described, and principal component analysis (PCA) was applied to synthesize multiple indicators into a Global Characteristics Index capturing five core health system functions: resource generation, financing, service delivery, general governance, and therapy-specific governance. Coverage is defined as formal access with explicit financial protection provided by the health system. Substantial heterogeneity was observed across countries. Regulatory approval was often achieved relatively rapidly, but this did not consistently translate into timely or comprehensive coverage. Countries with stronger structural characteristics—particularly in resource generation, service delivery, and governance—tended to achieve broader and more sustained coverage, although institutional capacity alone was not sufficient in all cases. Our results emphasize the need to strengthen health governance and adopt specific policies for rare diseases in the region.

]]> Structural Enablers of Rare Disease Treatment Coverage in Latin America and the Caribbean: Lessons from Emicizumab Daniela Sugg Herrera Dino Sepúlveda Viveros Moisés Russo Namias Natalia Garrido doi: 10.3390/jmahp14010013 Journal of Market Access & Health Policy 2026-02-25 Journal of Market Access & Health Policy 2026-02-25 14 1 Article 13 10.3390/jmahp14010013 https://www.mdpi.com/2001-6689/14/1/13 JMAHP, Vol. 14, Pages 12: Updated Conversion Table for the Multiple Criteria Qualitative Value-Based Pricing Framework “MARIE” https://www.mdpi.com/2001-6689/14/1/12 Background: Value-based pricing has the potential to contribute to the appropriate allocation of healthcare expenditures. We developed the “MARIE”, a qualitative scheme that evaluates broad value elements without requiring a comparator to estimate new drug prices. In this study, we updated the conversion table that monetizes points calculated from value elements. Methods: We investigated and calculated the daily drug prices at the time of listing for drugs containing new active ingredients that were included in the National Health Insurance Drug Price List from fiscal year 2015 to 2024, summarizing the data using descriptive statistics. Results: New drug prices are listed annually in Japan, so we updated the conversion table using current drug prices to maintain continuity. This study also observed a trend where the median daily price tended to be higher as the maximum number of patients decreased. Conclusions: The MARIE method, our developed framework to qualitatively evaluate the various values of drugs, monetizing points calculated based on the elements of value via the conversion table, plays a crucial role. Updating the conversion table based on the latest data maintains continuity with the current drug pricing system and is considered to contribute to the social implementation of the MARIE method. 2026-02-25 JMAHP, Vol. 14, Pages 12: Updated Conversion Table for the Multiple Criteria Qualitative Value-Based Pricing Framework “MARIE”

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010012

Authors: Akina Takami Ataru Igarashi

Background: Value-based pricing has the potential to contribute to the appropriate allocation of healthcare expenditures. We developed the “MARIE”, a qualitative scheme that evaluates broad value elements without requiring a comparator to estimate new drug prices. In this study, we updated the conversion table that monetizes points calculated from value elements. Methods: We investigated and calculated the daily drug prices at the time of listing for drugs containing new active ingredients that were included in the National Health Insurance Drug Price List from fiscal year 2015 to 2024, summarizing the data using descriptive statistics. Results: New drug prices are listed annually in Japan, so we updated the conversion table using current drug prices to maintain continuity. This study also observed a trend where the median daily price tended to be higher as the maximum number of patients decreased. Conclusions: The MARIE method, our developed framework to qualitatively evaluate the various values of drugs, monetizing points calculated based on the elements of value via the conversion table, plays a crucial role. Updating the conversion table based on the latest data maintains continuity with the current drug pricing system and is considered to contribute to the social implementation of the MARIE method.

]]> Updated Conversion Table for the Multiple Criteria Qualitative Value-Based Pricing Framework “MARIE” Akina Takami Ataru Igarashi doi: 10.3390/jmahp14010012 Journal of Market Access & Health Policy 2026-02-25 Journal of Market Access & Health Policy 2026-02-25 14 1 Article 12 10.3390/jmahp14010012 https://www.mdpi.com/2001-6689/14/1/12 JMAHP, Vol. 14, Pages 11: Optimising Investment in Health Innovations in Europe https://www.mdpi.com/2001-6689/14/1/11 European citizens made it clear in 2024 that healthcare should be the EU’s top priority for shaping the future of Europe. This sentiment reflects the escalating health challenges facing the region, driven by ageing populations, rising chronic disease burdens, and persistent disparities in access to healthcare. Despite these growing needs, the most recent data on health spending as a share of gross domestic product (GDP) is just slightly above the pre-COVID-19 pandemic level, and spending on pharmaceuticals specifically has remained a stable proportion of healthcare spending over the last 20 years. Austerity measures have profoundly impacted the health sector and pharmaceutical industry, more so than any other sectors, despite the wide range of health and socioeconomic benefits medicines bring to patients, the health system, and society. Such trends are not keeping pace with evolving population demographics and disease prevalence. To secure a healthier, more equitable future, Europe must urgently increase health investments, optimise health systems, and address unmet needs by supporting fast uptake of pharmaceutical innovations. Policymakers must work with all stakeholders to ensure stronger and sustained investments in health innovations by (i) adopting a long-term vision, moving away from short-term thinking and valuing health appropriately to drive economic growth; (ii) implementing transformative policies that eliminate ineffective and wasteful spending; (iii) promoting value-based approaches to improve patient access and system sustainability, and (iv) creating incentives that attract greater investments to strengthen Europe’s competitiveness and safeguard against health threats. 2026-02-13 JMAHP, Vol. 14, Pages 11: Optimising Investment in Health Innovations in Europe

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010011

Authors: Tosin Adeyemo Tim Wilsdon Christina Vandorou Fiona Davies Annabelle Godet

European citizens made it clear in 2024 that healthcare should be the EU’s top priority for shaping the future of Europe. This sentiment reflects the escalating health challenges facing the region, driven by ageing populations, rising chronic disease burdens, and persistent disparities in access to healthcare. Despite these growing needs, the most recent data on health spending as a share of gross domestic product (GDP) is just slightly above the pre-COVID-19 pandemic level, and spending on pharmaceuticals specifically has remained a stable proportion of healthcare spending over the last 20 years. Austerity measures have profoundly impacted the health sector and pharmaceutical industry, more so than any other sectors, despite the wide range of health and socioeconomic benefits medicines bring to patients, the health system, and society. Such trends are not keeping pace with evolving population demographics and disease prevalence. To secure a healthier, more equitable future, Europe must urgently increase health investments, optimise health systems, and address unmet needs by supporting fast uptake of pharmaceutical innovations. Policymakers must work with all stakeholders to ensure stronger and sustained investments in health innovations by (i) adopting a long-term vision, moving away from short-term thinking and valuing health appropriately to drive economic growth; (ii) implementing transformative policies that eliminate ineffective and wasteful spending; (iii) promoting value-based approaches to improve patient access and system sustainability, and (iv) creating incentives that attract greater investments to strengthen Europe’s competitiveness and safeguard against health threats.

]]> Optimising Investment in Health Innovations in Europe Tosin Adeyemo Tim Wilsdon Christina Vandorou Fiona Davies Annabelle Godet doi: 10.3390/jmahp14010011 Journal of Market Access & Health Policy 2026-02-13 Journal of Market Access & Health Policy 2026-02-13 14 1 Opinion 11 10.3390/jmahp14010011 https://www.mdpi.com/2001-6689/14/1/11 JMAHP, Vol. 14, Pages 10: Adopting a Societal Perspective in Health-Economic Evaluation: Analysis of Nine HTA Methodological Guidelines on How to Integrate Societal Costs https://www.mdpi.com/2001-6689/14/1/10 Objective: The perspective applied in Health Technology Assessment (HTA) has recently been presented as a central methodological debate in HTA. Beyond expected effects on patients’ health and quality of life, health technologies can affect broader societal domains, such as the labor market and informal care. Ensuring comparability between treatments when estimating their impact on society relies strongly on transparency regarding the methodology used to measure and value societal costs. This study aims to describe and summarize current recommendations of HTA bodies on adopting societal perspectives in HTA. Methods: A scoping review of HTA guidelines was conducted, and findings were validated through semi-structured interviews with academic and industry HTA experts. Guidelines from nine countries (Australia, Canada, France, Germany, The Netherlands, Spain, Sweden, United Kingdom, United States of America) were analyzed using an extraction grid covering four domains: out-of-pocket/copayment costs, informal care costs, productivity losses, and unrelated health care costs. Exploratory analyses (multiple factor analysis and clustering) assessed whether recommendations for a societal perspective as a base case were homogeneous across countries. Results: A societal perspective was recommended as a base case in 56% of the guidelines. Marked cross-country heterogeneity was observed for measurement methods. Only The Netherlands recommended a specific instrument for both informal care and productivity losses (iPCQ). The most frequently cited valuation approaches were the opportunity cost method (informal care) and the friction cost method (productivity losses). Three clusters of countries were identified: The Netherlands/Canada (1); US/Sweden/UK/Germany (2); and Spain/France/Australia (3). Exploratory analyses indicated limited alignment between endorsing the inclusion of societal costs and recommending the societal perspective as base case. Conclusions: This preliminary work highlights the need for explicit methodological guidance on societal cost estimation within HTA. HTA bodies could draw on the Netherland’s guidelines and cost database and develop a national “societal perspective doctrine”—including core cost sets, standardized measurement tools (e.g., iPCQ; diary methods), and structured involvement of patients and caregivers—to enhance comparability and decision relevance. 2026-02-10 JMAHP, Vol. 14, Pages 10: Adopting a Societal Perspective in Health-Economic Evaluation: Analysis of Nine HTA Methodological Guidelines on How to Integrate Societal Costs

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010010

Authors: Chloé Gervès-Pinquié Hortense Nanoux Sarah Akarkoub Rémi Brazeilles Henri Bonnabau Katell Le Lay

Objective: The perspective applied in Health Technology Assessment (HTA) has recently been presented as a central methodological debate in HTA. Beyond expected effects on patients’ health and quality of life, health technologies can affect broader societal domains, such as the labor market and informal care. Ensuring comparability between treatments when estimating their impact on society relies strongly on transparency regarding the methodology used to measure and value societal costs. This study aims to describe and summarize current recommendations of HTA bodies on adopting societal perspectives in HTA. Methods: A scoping review of HTA guidelines was conducted, and findings were validated through semi-structured interviews with academic and industry HTA experts. Guidelines from nine countries (Australia, Canada, France, Germany, The Netherlands, Spain, Sweden, United Kingdom, United States of America) were analyzed using an extraction grid covering four domains: out-of-pocket/copayment costs, informal care costs, productivity losses, and unrelated health care costs. Exploratory analyses (multiple factor analysis and clustering) assessed whether recommendations for a societal perspective as a base case were homogeneous across countries. Results: A societal perspective was recommended as a base case in 56% of the guidelines. Marked cross-country heterogeneity was observed for measurement methods. Only The Netherlands recommended a specific instrument for both informal care and productivity losses (iPCQ). The most frequently cited valuation approaches were the opportunity cost method (informal care) and the friction cost method (productivity losses). Three clusters of countries were identified: The Netherlands/Canada (1); US/Sweden/UK/Germany (2); and Spain/France/Australia (3). Exploratory analyses indicated limited alignment between endorsing the inclusion of societal costs and recommending the societal perspective as base case. Conclusions: This preliminary work highlights the need for explicit methodological guidance on societal cost estimation within HTA. HTA bodies could draw on the Netherland’s guidelines and cost database and develop a national “societal perspective doctrine”—including core cost sets, standardized measurement tools (e.g., iPCQ; diary methods), and structured involvement of patients and caregivers—to enhance comparability and decision relevance.

]]> Adopting a Societal Perspective in Health-Economic Evaluation: Analysis of Nine HTA Methodological Guidelines on How to Integrate Societal Costs Chloé Gervès-Pinquié Hortense Nanoux Sarah Akarkoub Rémi Brazeilles Henri Bonnabau Katell Le Lay doi: 10.3390/jmahp14010010 Journal of Market Access & Health Policy 2026-02-10 Journal of Market Access & Health Policy 2026-02-10 14 1 Article 10 10.3390/jmahp14010010 https://www.mdpi.com/2001-6689/14/1/10 JMAHP, Vol. 14, Pages 9: Pulmonary Arterial Hypertension and Cancer: Unveiling Parallels in Epidemiology, Clinical Pathways, and Therapeutic Strategies https://www.mdpi.com/2001-6689/14/1/9 Pulmonary arterial hypertension (PAH) and cancer share high mortality and complex prognoses. Due to PAH’s rarity, these parallels may be underrecognized by healthcare stakeholders. This study explored similarities between PAH and cancer across epidemiological, clinical, therapeutic, and healthcare resource utilization (HCRU) considerations. A four-step approach was employed: (1) inclusion/exclusion criteria were applied to identify potential PAH cancer analogs; (2) characteristics for comparison were categorized as epidemiologic, clinical, therapeutic landscape, and HCRU; (3) a targeted literature review extracted data on disease characteristics; (4) a similarity ranking was calculated as the absolute difference between each cancer’s and PAH’s characteristics. Fourteen cancers met the inclusion criteria. Well-differentiated thyroid cancer (WDTC) had the highest number (5) of characteristics closest to PAH. WDTC and medullary thyroid cancer were most similar to PAH in epidemiology; gastrointestinal stromal tumor was most similar in clinical and HCRU characteristics, and anaplastic lymphoma kinase-positive (ALK+) non-small-cell lung cancer and renal cell carcinoma were most similar in therapeutic landscape. Although no single cancer fully mirrors PAH, the identification of multiple analogs underscores PAH’s multidimensional complexity and confirms its overlap with oncological conditions. Cancer analogs could serve as a valuable framework for enhancing recognition of PAH’s clinical, therapeutic, and HRCU implications among healthcare stakeholders. 2026-02-06 JMAHP, Vol. 14, Pages 9: Pulmonary Arterial Hypertension and Cancer: Unveiling Parallels in Epidemiology, Clinical Pathways, and Therapeutic Strategies

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010009

Authors: Karim El-Kersh Nadine Zawadzki Catelyn Coyle Shurui Zhang Dhruv Dalal Anna Watzker Dominik Lautsch Jason Shafrin

Pulmonary arterial hypertension (PAH) and cancer share high mortality and complex prognoses. Due to PAH’s rarity, these parallels may be underrecognized by healthcare stakeholders. This study explored similarities between PAH and cancer across epidemiological, clinical, therapeutic, and healthcare resource utilization (HCRU) considerations. A four-step approach was employed: (1) inclusion/exclusion criteria were applied to identify potential PAH cancer analogs; (2) characteristics for comparison were categorized as epidemiologic, clinical, therapeutic landscape, and HCRU; (3) a targeted literature review extracted data on disease characteristics; (4) a similarity ranking was calculated as the absolute difference between each cancer’s and PAH’s characteristics. Fourteen cancers met the inclusion criteria. Well-differentiated thyroid cancer (WDTC) had the highest number (5) of characteristics closest to PAH. WDTC and medullary thyroid cancer were most similar to PAH in epidemiology; gastrointestinal stromal tumor was most similar in clinical and HCRU characteristics, and anaplastic lymphoma kinase-positive (ALK+) non-small-cell lung cancer and renal cell carcinoma were most similar in therapeutic landscape. Although no single cancer fully mirrors PAH, the identification of multiple analogs underscores PAH’s multidimensional complexity and confirms its overlap with oncological conditions. Cancer analogs could serve as a valuable framework for enhancing recognition of PAH’s clinical, therapeutic, and HRCU implications among healthcare stakeholders.

]]> Pulmonary Arterial Hypertension and Cancer: Unveiling Parallels in Epidemiology, Clinical Pathways, and Therapeutic Strategies Karim El-Kersh Nadine Zawadzki Catelyn Coyle Shurui Zhang Dhruv Dalal Anna Watzker Dominik Lautsch Jason Shafrin doi: 10.3390/jmahp14010009 Journal of Market Access & Health Policy 2026-02-06 Journal of Market Access & Health Policy 2026-02-06 14 1 Article 9 10.3390/jmahp14010009 https://www.mdpi.com/2001-6689/14/1/9 JMAHP, Vol. 14, Pages 8: A Cost-Effectiveness Analysis of the Sentio Bone Conduction Hearing Implant System in the Australian Healthcare Setting https://www.mdpi.com/2001-6689/14/1/8 Bone conduction hearing implant systems (BCHIs) are established treatments for patients with conductive or mixed hearing loss or single-sided deafness when conventional hearing aids are unsuitable. This study evaluated the cost-effectiveness of the active transcutaneous system Sentio versus a similar system, i.e., Osia in an Australian setting. Scenario analyses also compared Sentio to other systems, i.e., Ponto and Baha Attract. A Markov cohort model was adapted from a previously published source to reflect Australian practice, incorporating device acquisition, surgery, maintenance, battery replacement and adverse event management over a 15-year horizon from a healthcare perspective. Effectiveness inputs were derived from published evidence using a naïve indirect comparison. Extensive sensitivity analyses and external validation tested robustness. In the base case, Sentio was associated with lower costs and a small modelled incremental quality-adjusted life years (QALYs) gain versus Osia. Scenario analyses confirmed cost-effectiveness relative to Ponto and Baha Attract, with outcomes below the Australian willingness-to-pay threshold. Health state utility, device price and reimplantation assumptions were the most influential drivers, yet Sentio remained cost-effective in over 95% of simulations. These findings support Sentio as a clinically and economically efficient BCHI in Australia and highlight the need for direct utility and long-term durability data. 2026-01-27 JMAHP, Vol. 14, Pages 8: A Cost-Effectiveness Analysis of the Sentio Bone Conduction Hearing Implant System in the Australian Healthcare Setting

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010008

Authors: Magnus Värendh Ida Haggren Helén Lagerkvist Maria Åberg Håkansson Jonas Hjelmgren

Bone conduction hearing implant systems (BCHIs) are established treatments for patients with conductive or mixed hearing loss or single-sided deafness when conventional hearing aids are unsuitable. This study evaluated the cost-effectiveness of the active transcutaneous system Sentio versus a similar system, i.e., Osia in an Australian setting. Scenario analyses also compared Sentio to other systems, i.e., Ponto and Baha Attract. A Markov cohort model was adapted from a previously published source to reflect Australian practice, incorporating device acquisition, surgery, maintenance, battery replacement and adverse event management over a 15-year horizon from a healthcare perspective. Effectiveness inputs were derived from published evidence using a naïve indirect comparison. Extensive sensitivity analyses and external validation tested robustness. In the base case, Sentio was associated with lower costs and a small modelled incremental quality-adjusted life years (QALYs) gain versus Osia. Scenario analyses confirmed cost-effectiveness relative to Ponto and Baha Attract, with outcomes below the Australian willingness-to-pay threshold. Health state utility, device price and reimplantation assumptions were the most influential drivers, yet Sentio remained cost-effective in over 95% of simulations. These findings support Sentio as a clinically and economically efficient BCHI in Australia and highlight the need for direct utility and long-term durability data.

]]> A Cost-Effectiveness Analysis of the Sentio Bone Conduction Hearing Implant System in the Australian Healthcare Setting Magnus Värendh Ida Haggren Helén Lagerkvist Maria Åberg Håkansson Jonas Hjelmgren doi: 10.3390/jmahp14010008 Journal of Market Access & Health Policy 2026-01-27 Journal of Market Access & Health Policy 2026-01-27 14 1 Article 8 10.3390/jmahp14010008 https://www.mdpi.com/2001-6689/14/1/8 JMAHP, Vol. 14, Pages 7: Evaluating [18F]-DCFPyL for Detecting Prostate Cancer Recurrence: A Cost–Consequence Comparison with Alternative PET Radiotracers in Spain https://www.mdpi.com/2001-6689/14/1/7 Introduction: [18F]-DCFPyL (Piflufolastat [18F]) is a prostate-specific membrane antigen (PSMA)-targeted position emission tomography (PET) radiotracer for detecting the biochemical recurrence (BCR) of prostate cancer (PCa). This study evaluates its economic impact compared with [68Ga]-PSMA-11, [18F]-FCH, and [18F]-PSMA-1007 from the Spanish National Healthcare System’s perspective. Methods: A cost–consequence model, over a 5-year time horizon, simulated the diagnostic and treatment pathway based on radiotracer-specific accuracy and disease localization. Treatment options included a radical prostatectomy, radiation therapy, androgen deprivation therapy (ADT), and radiation therapy + ADT. Costs were calculated for true/false positives and negatives. Due to limited data availability, key inputs were informed by expert opinions, supported by published meta-analyses, public sources, and literature. Officially published Spanish prices were applied: EUR 2000 for [18F]-DCFPyL, [68Ga]-PSMA-11, and [18F]-PSMA-1007, and EUR 1144 for [18F]-FCH. Results: The use of [18F]-DCFPyL led to fewer unnecessary therapies; specifically, it led to 11,229 (74%) fewer than [68Ga]-PSMA-11, and 5180 (56%) and 7771 (66%) fewer than [18F]-FCH and [18F]-PSMA-1007, respectively. It achieved significant cost savings for repeated testing: EUR 15M (43%) versus [68Ga]-PSMA-11, EUR 37M (65%) versus [18F]-FCH, and EUR 27M (58%) versus [18F]-PSMA-1007. Cost savings for false positives were EUR 15M (50%) against [68Ga]-PSMA-11, EUR 22M (60%) versus [18F]-FCH, and EUR 29M (66%) compared with [18F]-PSMA-1007. The cost per correct diagnosis was reduced by EUR 198 (8%) compared with [68Ga]-PSMA-11 and EUR 377 (15%) relative to [18F]-PSMA-1007, while showing a EUR 635 (40%) increase compared with [18F]-FCH. Conclusions: [18F]-DCFPyL offers a cost-saving option for BCR detection within the Spanish National Healthcare System by reducing the number of unnecessary therapies, the cost of false positives, and repeat testing compared with alternative radiotracers. These improvements support the potential for better diagnostic outcomes and more informed downstream clinical decision-making. 2026-01-23 JMAHP, Vol. 14, Pages 7: Evaluating [18F]-DCFPyL for Detecting Prostate Cancer Recurrence: A Cost–Consequence Comparison with Alternative PET Radiotracers in Spain

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010007

Authors: Tiago Matos Mrunmayee Godbole Rithvik Badinedi Madhusubramanian Muthukumar Marina Hodolic Nicolas Tchouen Anthony Berthon

Introduction: [18F]-DCFPyL (Piflufolastat [18F]) is a prostate-specific membrane antigen (PSMA)-targeted position emission tomography (PET) radiotracer for detecting the biochemical recurrence (BCR) of prostate cancer (PCa). This study evaluates its economic impact compared with [68Ga]-PSMA-11, [18F]-FCH, and [18F]-PSMA-1007 from the Spanish National Healthcare System’s perspective. Methods: A cost–consequence model, over a 5-year time horizon, simulated the diagnostic and treatment pathway based on radiotracer-specific accuracy and disease localization. Treatment options included a radical prostatectomy, radiation therapy, androgen deprivation therapy (ADT), and radiation therapy + ADT. Costs were calculated for true/false positives and negatives. Due to limited data availability, key inputs were informed by expert opinions, supported by published meta-analyses, public sources, and literature. Officially published Spanish prices were applied: EUR 2000 for [18F]-DCFPyL, [68Ga]-PSMA-11, and [18F]-PSMA-1007, and EUR 1144 for [18F]-FCH. Results: The use of [18F]-DCFPyL led to fewer unnecessary therapies; specifically, it led to 11,229 (74%) fewer than [68Ga]-PSMA-11, and 5180 (56%) and 7771 (66%) fewer than [18F]-FCH and [18F]-PSMA-1007, respectively. It achieved significant cost savings for repeated testing: EUR 15M (43%) versus [68Ga]-PSMA-11, EUR 37M (65%) versus [18F]-FCH, and EUR 27M (58%) versus [18F]-PSMA-1007. Cost savings for false positives were EUR 15M (50%) against [68Ga]-PSMA-11, EUR 22M (60%) versus [18F]-FCH, and EUR 29M (66%) compared with [18F]-PSMA-1007. The cost per correct diagnosis was reduced by EUR 198 (8%) compared with [68Ga]-PSMA-11 and EUR 377 (15%) relative to [18F]-PSMA-1007, while showing a EUR 635 (40%) increase compared with [18F]-FCH. Conclusions: [18F]-DCFPyL offers a cost-saving option for BCR detection within the Spanish National Healthcare System by reducing the number of unnecessary therapies, the cost of false positives, and repeat testing compared with alternative radiotracers. These improvements support the potential for better diagnostic outcomes and more informed downstream clinical decision-making.

]]> Evaluating [18F]-DCFPyL for Detecting Prostate Cancer Recurrence: A Cost–Consequence Comparison with Alternative PET Radiotracers in Spain Tiago Matos Mrunmayee Godbole Rithvik Badinedi Madhusubramanian Muthukumar Marina Hodolic Nicolas Tchouen Anthony Berthon doi: 10.3390/jmahp14010007 Journal of Market Access & Health Policy 2026-01-23 Journal of Market Access & Health Policy 2026-01-23 14 1 Article 7 10.3390/jmahp14010007 https://www.mdpi.com/2001-6689/14/1/7 JMAHP, Vol. 14, Pages 6: Enhancing Patient-Centered Health Technology Assessment: A Modified Delphi Panel for PICOS Scoping in Spinal Muscular Atrophy https://www.mdpi.com/2001-6689/14/1/6 Objectives: This study explores the feasibility and value of integrating structured patient input into the PICOS (Population, Intervention, Comparator, Outcome, Study design) scoping process for Joint Clinical Assessments under the EU Health Technology Assessment Regulation. Methods: A modified Delphi panel, led by a steering committee composed of two clinicians, one patient expert, and one policy expert, engaged 12 individuals representing patient organizations across 12 European Member States to reach consensus on PICOS elements for CAR-T therapy in pediatric spinal muscular atrophy. Results: The Delphi process effectively facilitated PICOS consolidation and consensus among the 12 patient experts representing diverse EU contexts. Through 3 iterative rounds integrating quantitative rankings and qualitative feedback, the panel achieved strong agreement on key outcomes, intervention delivery, and study design elements, with population eligibility and comparator selection showing heterogeneity. Patient engagement was central: participants emphasized inclusive eligibility criteria, shared decision-making, and the inclusion of caregiver perspectives. The integration of qualitative insights allowed nuanced interpretation of dissent, distinguishing between genuine disagreement and framing effects, thereby enhancing transparency and scientific validity. Importantly, the process revealed patient priorities for outcomes, treatment burden, and evidence trade-offs, informing both PICOS refinement and future health technology assessment (HTA) strategies. This structured, participatory approach demonstrates the feasibility and value of incorporating patient voices systematically into early-stage EU HTA, fostering robust, credible, and context-sensitive consensus on complex rare-disease interventions. Conclusions: The study demonstrates the potential of consensus-building methodologies to enhance transparency, reduce heterogeneity, and support patient-centered evidence generation and decision-making in HTA. 2026-01-19 JMAHP, Vol. 14, Pages 6: Enhancing Patient-Centered Health Technology Assessment: A Modified Delphi Panel for PICOS Scoping in Spinal Muscular Atrophy

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010006

Authors: Emanuele Arcà Adele Barlassina Adaeze Eze Valentina Strammiello

Objectives: This study explores the feasibility and value of integrating structured patient input into the PICOS (Population, Intervention, Comparator, Outcome, Study design) scoping process for Joint Clinical Assessments under the EU Health Technology Assessment Regulation. Methods: A modified Delphi panel, led by a steering committee composed of two clinicians, one patient expert, and one policy expert, engaged 12 individuals representing patient organizations across 12 European Member States to reach consensus on PICOS elements for CAR-T therapy in pediatric spinal muscular atrophy. Results: The Delphi process effectively facilitated PICOS consolidation and consensus among the 12 patient experts representing diverse EU contexts. Through 3 iterative rounds integrating quantitative rankings and qualitative feedback, the panel achieved strong agreement on key outcomes, intervention delivery, and study design elements, with population eligibility and comparator selection showing heterogeneity. Patient engagement was central: participants emphasized inclusive eligibility criteria, shared decision-making, and the inclusion of caregiver perspectives. The integration of qualitative insights allowed nuanced interpretation of dissent, distinguishing between genuine disagreement and framing effects, thereby enhancing transparency and scientific validity. Importantly, the process revealed patient priorities for outcomes, treatment burden, and evidence trade-offs, informing both PICOS refinement and future health technology assessment (HTA) strategies. This structured, participatory approach demonstrates the feasibility and value of incorporating patient voices systematically into early-stage EU HTA, fostering robust, credible, and context-sensitive consensus on complex rare-disease interventions. Conclusions: The study demonstrates the potential of consensus-building methodologies to enhance transparency, reduce heterogeneity, and support patient-centered evidence generation and decision-making in HTA.

]]> Enhancing Patient-Centered Health Technology Assessment: A Modified Delphi Panel for PICOS Scoping in Spinal Muscular Atrophy Emanuele Arcà Adele Barlassina Adaeze Eze Valentina Strammiello doi: 10.3390/jmahp14010006 Journal of Market Access & Health Policy 2026-01-19 Journal of Market Access & Health Policy 2026-01-19 14 1 Article 6 10.3390/jmahp14010006 https://www.mdpi.com/2001-6689/14/1/6 JMAHP, Vol. 14, Pages 5: Development of a Performance Measurement Framework for European Health Technology Assessment: Stakeholder-Centric Key Performance Indicators Identified in a Delphi Approach by the European Access Academy https://www.mdpi.com/2001-6689/14/1/5 Background: The objective of this work was to support the implementation of the European Health Technology Assessment Regulation (EU HTAR) and optimize performance of the evolving EU HTA system. Therefore, an inclusive multi-stakeholder framework of key performance indicators (KPI) for success measurement was developed. Methods: A modified Delphi-procedure was applied as follows: (1) development of a generic KPI pool at the Fall Convention 2024 of the European Access Academy (EAA); (2) review of initial pool and identification of additional KPIs; (3) development of prioritized KPIs covering patient, clinician, Health Technology Developer (HTD), and System/Member State (MS) perspectives, and (4) consolidation of the stakeholder-centric KPIs after EAA’s Spring Convention 2025. Results: Steps 1 and 2 of the Delphi procedure revealed 14 generic KPI domains. Steps 3 and 4 resulted in four prioritized KPIs for patients (patient input; utilization of patient-centric outcome measures; time to access; equity); six for clinicians (population/intervention/comparator/outcomes (PICO); addressing uncertainty; clinician involvement; transparency; equity and time to access); four for HTDs (PICO; joint scientific consultation (JSC) process; joint clinical assessment (JCA) process; time to national decision making); five from a system/MS perspective (PICO; learning and training the health system; reducing duplication; equity and time to access). The scope of, e.g., the PICO-related KPI, differed between stakeholder groups. Also, several KPIs intentionally reached beyond the remit of EU HTA as they are also dependent on MS-specific factors including national health systems and budgets. Discussion and Conclusions: The KPI framework developed here presents a step towards the generation of systematic multi-stakeholder evidence to support a successful implementation of the EU HTAR. The relevance of the identified stakeholder-centric KPIs is confirmed by their alignment with the Health System Goals suggested in the context of “Performance measurement for health improvement” by the World Health Organisation. Implementation of the framework, i.e., measurement of KPIs, is envisioned to provide evidence to inform the 2028 revision of the EU HTAR. 2026-01-15 JMAHP, Vol. 14, Pages 5: Development of a Performance Measurement Framework for European Health Technology Assessment: Stakeholder-Centric Key Performance Indicators Identified in a Delphi Approach by the European Access Academy

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010005

Authors: Elaine Julian Nicolas S. H. Xander Konstantina Boumaki Maria João Garcia Evelina Jahimovica Joséphine Mosset-Keane Monica Hildegard Otto Mira Pavlovic Giovanna Scroccaro Valentina Strammiello Renato Bernardini Stefano Capri Ruben Casado-Arroyo Thomas Desmet Walter Van Dyck Frank-Ulrich Fricke Fabrizio Gianfrate Oriol Solà-Morales Jürgen Wasem Bernhard J. Wörmann Jörg Ruof

Background: The objective of this work was to support the implementation of the European Health Technology Assessment Regulation (EU HTAR) and optimize performance of the evolving EU HTA system. Therefore, an inclusive multi-stakeholder framework of key performance indicators (KPI) for success measurement was developed. Methods: A modified Delphi-procedure was applied as follows: (1) development of a generic KPI pool at the Fall Convention 2024 of the European Access Academy (EAA); (2) review of initial pool and identification of additional KPIs; (3) development of prioritized KPIs covering patient, clinician, Health Technology Developer (HTD), and System/Member State (MS) perspectives, and (4) consolidation of the stakeholder-centric KPIs after EAA’s Spring Convention 2025. Results: Steps 1 and 2 of the Delphi procedure revealed 14 generic KPI domains. Steps 3 and 4 resulted in four prioritized KPIs for patients (patient input; utilization of patient-centric outcome measures; time to access; equity); six for clinicians (population/intervention/comparator/outcomes (PICO); addressing uncertainty; clinician involvement; transparency; equity and time to access); four for HTDs (PICO; joint scientific consultation (JSC) process; joint clinical assessment (JCA) process; time to national decision making); five from a system/MS perspective (PICO; learning and training the health system; reducing duplication; equity and time to access). The scope of, e.g., the PICO-related KPI, differed between stakeholder groups. Also, several KPIs intentionally reached beyond the remit of EU HTA as they are also dependent on MS-specific factors including national health systems and budgets. Discussion and Conclusions: The KPI framework developed here presents a step towards the generation of systematic multi-stakeholder evidence to support a successful implementation of the EU HTAR. The relevance of the identified stakeholder-centric KPIs is confirmed by their alignment with the Health System Goals suggested in the context of “Performance measurement for health improvement” by the World Health Organisation. Implementation of the framework, i.e., measurement of KPIs, is envisioned to provide evidence to inform the 2028 revision of the EU HTAR.

]]> Development of a Performance Measurement Framework for European Health Technology Assessment: Stakeholder-Centric Key Performance Indicators Identified in a Delphi Approach by the European Access Academy Elaine Julian Nicolas S. H. Xander Konstantina Boumaki Maria João Garcia Evelina Jahimovica Joséphine Mosset-Keane Monica Hildegard Otto Mira Pavlovic Giovanna Scroccaro Valentina Strammiello Renato Bernardini Stefano Capri Ruben Casado-Arroyo Thomas Desmet Walter Van Dyck Frank-Ulrich Fricke Fabrizio Gianfrate Oriol Solà-Morales Jürgen Wasem Bernhard J. Wörmann Jörg Ruof doi: 10.3390/jmahp14010005 Journal of Market Access & Health Policy 2026-01-15 Journal of Market Access & Health Policy 2026-01-15 14 1 Article 5 10.3390/jmahp14010005 https://www.mdpi.com/2001-6689/14/1/5 JMAHP, Vol. 14, Pages 4: Conformity Assessment of Medical Devices: An Overview from a Notified Body https://www.mdpi.com/2001-6689/14/1/4 This perspective provides an in-depth analysis of the role and tasks of Notified Bodies (NBs) under the Medical Device Regulation (MDR) and In Vitro Diagnostic Regulation (IVDR). It explores the conformity assessment process and highlights typical issues encountered. It provides background on the legal framework and roles and tasks of Notified Bodies (NBs). It further explores the seven-step conformity assessment process which aims to ensure that medical devices meet European Union (EU) safety and performance standards. Finally, we highlight typical issues encountered during the process and re-cent developments in the area and conclude with an outlook for the implementation of the MDR and IVDR. 2026-01-08 JMAHP, Vol. 14, Pages 4: Conformity Assessment of Medical Devices: An Overview from a Notified Body

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010004

Authors: Andreas F. Stange Elaine Julian

This perspective provides an in-depth analysis of the role and tasks of Notified Bodies (NBs) under the Medical Device Regulation (MDR) and In Vitro Diagnostic Regulation (IVDR). It explores the conformity assessment process and highlights typical issues encountered. It provides background on the legal framework and roles and tasks of Notified Bodies (NBs). It further explores the seven-step conformity assessment process which aims to ensure that medical devices meet European Union (EU) safety and performance standards. Finally, we highlight typical issues encountered during the process and re-cent developments in the area and conclude with an outlook for the implementation of the MDR and IVDR.

]]> Conformity Assessment of Medical Devices: An Overview from a Notified Body Andreas F. Stange Elaine Julian doi: 10.3390/jmahp14010004 Journal of Market Access & Health Policy 2026-01-08 Journal of Market Access & Health Policy 2026-01-08 14 1 Communication 4 10.3390/jmahp14010004 https://www.mdpi.com/2001-6689/14/1/4 JMAHP, Vol. 14, Pages 3: European Joint Clinical Assessment PICO Scoping Process: Analysis of Current Approaches and Recommendations https://www.mdpi.com/2001-6689/14/1/3 The PICO framework determines the scope of the Joint Clinical Assessment (JCA) under the EU HTA Regulation (EU HTAR), with PICO consolidation being a critical final step of the scoping process. Due to limited clarity on how consolidation works in practice, Health Technology Developers (HTDs) may simulate PICO scoping as a strategic tool to guide the development of robust JCA submissions. A review of 14 publications, representing 35 individual PICO exercises across 20 indications (74% in oncology), showed an average of 7 countries participating per exercise and 8 consolidated PICOs per analysis. A separate PICO scoping simulation focused on a first-line immuno-oncology treatment for metastatic non-small cell lung cancer (mNSCLC) generated 67 PICOs, reflecting the anticipated perspectives of 25 countries, largely driven by biomarker and histology-based sub-populations. The limited number of published examples and country participation restricts the ability to draw clear conclusions or confidently predict the output of PICO scoping in a real life JCA processes. The simulation also raised questions about whether all sub-populations should be included or consolidated further. Overall, there is a need for greater clarity in the JCA PICO scoping process, in particular the consolidation step, to facilitate high-quality evidence generation and support the EU HTAR to meet its goals of efficiency, transparency, and equity in health technology evaluation across Europe, along with more consistent patient access. 2025-12-29 JMAHP, Vol. 14, Pages 3: European Joint Clinical Assessment PICO Scoping Process: Analysis of Current Approaches and Recommendations

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010003

Authors: Kalpana D’Oca Eline Darquennes Chloé Garrigues Aristeidis Draganigos Natalie Steck

The PICO framework determines the scope of the Joint Clinical Assessment (JCA) under the EU HTA Regulation (EU HTAR), with PICO consolidation being a critical final step of the scoping process. Due to limited clarity on how consolidation works in practice, Health Technology Developers (HTDs) may simulate PICO scoping as a strategic tool to guide the development of robust JCA submissions. A review of 14 publications, representing 35 individual PICO exercises across 20 indications (74% in oncology), showed an average of 7 countries participating per exercise and 8 consolidated PICOs per analysis. A separate PICO scoping simulation focused on a first-line immuno-oncology treatment for metastatic non-small cell lung cancer (mNSCLC) generated 67 PICOs, reflecting the anticipated perspectives of 25 countries, largely driven by biomarker and histology-based sub-populations. The limited number of published examples and country participation restricts the ability to draw clear conclusions or confidently predict the output of PICO scoping in a real life JCA processes. The simulation also raised questions about whether all sub-populations should be included or consolidated further. Overall, there is a need for greater clarity in the JCA PICO scoping process, in particular the consolidation step, to facilitate high-quality evidence generation and support the EU HTAR to meet its goals of efficiency, transparency, and equity in health technology evaluation across Europe, along with more consistent patient access.

]]> European Joint Clinical Assessment PICO Scoping Process: Analysis of Current Approaches and Recommendations Kalpana D’Oca Eline Darquennes Chloé Garrigues Aristeidis Draganigos Natalie Steck doi: 10.3390/jmahp14010003 Journal of Market Access & Health Policy 2025-12-29 Journal of Market Access & Health Policy 2025-12-29 14 1 Article 3 10.3390/jmahp14010003 https://www.mdpi.com/2001-6689/14/1/3 JMAHP, Vol. 14, Pages 2: Overcoming Patient Access Barriers in Complex Conditions: Lessons from Schizophrenia for Broader Healthcare Applications https://www.mdpi.com/2001-6689/14/1/2 Patient access to innovative care for complex conditions like schizophrenia remains limited by systemic, clinical and policy-level barriers. Cognitive impairment associated with schizophrenia (CIAS) illustrates how critical symptom domains are often overlooked despite their impact on long-term outcomes. This study examines how systemic, infrastructural and economic factors shape access to CIAS care across eight mid-sized European countries to identify shared constraints and opportunities for improvement. Semi-structured interviews were conducted with 32 healthcare professionals and 9 health policy experts. Thematic analysis identified consistent barriers across countries, including fragmented care pathways, insufficient capacity for cognitive assessment, underdeveloped community-based rehabilitation services and reimbursement structures that favour pharmacological over psychosocial interventions. Variability across countries was shaped by differences in community infrastructure, professional training and the breadth of health technology assessment perspectives applied to non-pharmacological care. Countries with stronger community infrastructure and broader reimbursement frameworks were better positioned to deliver comprehensive care. These findings highlight that structural constraints, rather than clinical uncertainty, are the primary impediments to care in complex therapeutic areas. Addressing them will require coordinated reforms that strengthen early identification, expand multidisciplinary rehabilitation capacity and align reimbursement with functional and long-term outcomes. 2025-12-23 JMAHP, Vol. 14, Pages 2: Overcoming Patient Access Barriers in Complex Conditions: Lessons from Schizophrenia for Broader Healthcare Applications

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010002

Authors: Saartje Burgmans Anne Rieper Hald Sayagi Tina Markandaier Nicolas Hall Rafael Loiseau Xandra Lie Bregt Kappelhoff

Patient access to innovative care for complex conditions like schizophrenia remains limited by systemic, clinical and policy-level barriers. Cognitive impairment associated with schizophrenia (CIAS) illustrates how critical symptom domains are often overlooked despite their impact on long-term outcomes. This study examines how systemic, infrastructural and economic factors shape access to CIAS care across eight mid-sized European countries to identify shared constraints and opportunities for improvement. Semi-structured interviews were conducted with 32 healthcare professionals and 9 health policy experts. Thematic analysis identified consistent barriers across countries, including fragmented care pathways, insufficient capacity for cognitive assessment, underdeveloped community-based rehabilitation services and reimbursement structures that favour pharmacological over psychosocial interventions. Variability across countries was shaped by differences in community infrastructure, professional training and the breadth of health technology assessment perspectives applied to non-pharmacological care. Countries with stronger community infrastructure and broader reimbursement frameworks were better positioned to deliver comprehensive care. These findings highlight that structural constraints, rather than clinical uncertainty, are the primary impediments to care in complex therapeutic areas. Addressing them will require coordinated reforms that strengthen early identification, expand multidisciplinary rehabilitation capacity and align reimbursement with functional and long-term outcomes.

]]> Overcoming Patient Access Barriers in Complex Conditions: Lessons from Schizophrenia for Broader Healthcare Applications Saartje Burgmans Anne Rieper Hald Sayagi Tina Markandaier Nicolas Hall Rafael Loiseau Xandra Lie Bregt Kappelhoff doi: 10.3390/jmahp14010002 Journal of Market Access & Health Policy 2025-12-23 Journal of Market Access & Health Policy 2025-12-23 14 1 Article 2 10.3390/jmahp14010002 https://www.mdpi.com/2001-6689/14/1/2 JMAHP, Vol. 14, Pages 1: Knowledge Spillover and Innovation in Healthcare: A Comparative Study of Social Media Interaction Between Medical Technology Companies and Healthcare Professionals in Austria https://www.mdpi.com/2001-6689/14/1/1 Background: Interactions between healthcare professionals and medical technology companies on social media are central to understanding how variations in knowledge spillover and innovation performance vary. Aim: This study investigates how social media facilitates knowledge exchange between these two stakeholder groups in Austria, drawing on a cross-sectional online survey of 97 participants (45 healthcare professionals and 52 medical technology company representatives). Data were analyzed using Confirmatory Factor Analysis (CFA), Exploratory Structural Equation Modeling (ESEM), and independent-samples t-tests. The comparative approach enabled the identification of structural differences across stakeholder groups and regions within Austria. Methodology: The study adopts a comparative analysis framework to explore geographic differences within Austria and to examine how social media interaction strengthens dense and strong network ties between healthcare professionals and medical technology companies, thereby enhancing information-processing capabilities. Results: The findings underscore the pivotal role of social media in bridging geographic boundaries, fostering dense and strong network ties, and enhancing information-processing capacities. Conclusions: This study advances the understanding of how digital interaction mechanisms shape knowledge exchange and innovation performance in healthcare outcomes. Practical Implications: The findings suggest that social media promotes stronger professional relationships and deeper customer engagement. The results may assist policymakers and industry leaders aiming to design effective digital strategies for innovation and improved healthcare outcomes. 2025-12-22 JMAHP, Vol. 14, Pages 1: Knowledge Spillover and Innovation in Healthcare: A Comparative Study of Social Media Interaction Between Medical Technology Companies and Healthcare Professionals in Austria

Journal of Market Access & Health Policy doi: 10.3390/jmahp14010001

Authors: Mariella Zilahi-Lugbauer Harald Stummer

Background: Interactions between healthcare professionals and medical technology companies on social media are central to understanding how variations in knowledge spillover and innovation performance vary. Aim: This study investigates how social media facilitates knowledge exchange between these two stakeholder groups in Austria, drawing on a cross-sectional online survey of 97 participants (45 healthcare professionals and 52 medical technology company representatives). Data were analyzed using Confirmatory Factor Analysis (CFA), Exploratory Structural Equation Modeling (ESEM), and independent-samples t-tests. The comparative approach enabled the identification of structural differences across stakeholder groups and regions within Austria. Methodology: The study adopts a comparative analysis framework to explore geographic differences within Austria and to examine how social media interaction strengthens dense and strong network ties between healthcare professionals and medical technology companies, thereby enhancing information-processing capabilities. Results: The findings underscore the pivotal role of social media in bridging geographic boundaries, fostering dense and strong network ties, and enhancing information-processing capacities. Conclusions: This study advances the understanding of how digital interaction mechanisms shape knowledge exchange and innovation performance in healthcare outcomes. Practical Implications: The findings suggest that social media promotes stronger professional relationships and deeper customer engagement. The results may assist policymakers and industry leaders aiming to design effective digital strategies for innovation and improved healthcare outcomes.

]]> Knowledge Spillover and Innovation in Healthcare: A Comparative Study of Social Media Interaction Between Medical Technology Companies and Healthcare Professionals in Austria Mariella Zilahi-Lugbauer Harald Stummer doi: 10.3390/jmahp14010001 Journal of Market Access & Health Policy 2025-12-22 Journal of Market Access & Health Policy 2025-12-22 14 1 Article 1 10.3390/jmahp14010001 https://www.mdpi.com/2001-6689/14/1/1 JMAHP, Vol. 13, Pages 61: How Patients Can Contribute to the Assessments of Health Technologies https://www.mdpi.com/2001-6689/13/4/61 In the process of determining whether a health technology should be covered by healthcare systems, patients and their representatives were initially excluded from both evaluations and decision-making. In Europe, direct dialogue between patient organisations and regulatory authorities—particularly in the pharmaceutical sector—began in the early 1990s. It was only decades later, as the high cost of medicines created new challenges, that authorities recognised the necessity of engaging with patients. Patients’ contributions to the assessment of a health technology begin with discussions about the need for the technology in question. Initially, these discussions involve the developer, and later—after research and development—regulators, HTA assessors, and payers. Given that multiple technologies may be under development, patients and their organisations often prioritise those that generate the most interest within the patient community. They can then share their perspectives with evaluators during the horizon-scanning phase. Another key contribution is the role patients play in guiding clinical research by participating in scientific advice. Finally, during the assessment and appraisal stages, various methods are used to gather their views. 2025-12-15 JMAHP, Vol. 13, Pages 61: How Patients Can Contribute to the Assessments of Health Technologies

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040061

Authors: François Houÿez Julien Delaye

In the process of determining whether a health technology should be covered by healthcare systems, patients and their representatives were initially excluded from both evaluations and decision-making. In Europe, direct dialogue between patient organisations and regulatory authorities—particularly in the pharmaceutical sector—began in the early 1990s. It was only decades later, as the high cost of medicines created new challenges, that authorities recognised the necessity of engaging with patients. Patients’ contributions to the assessment of a health technology begin with discussions about the need for the technology in question. Initially, these discussions involve the developer, and later—after research and development—regulators, HTA assessors, and payers. Given that multiple technologies may be under development, patients and their organisations often prioritise those that generate the most interest within the patient community. They can then share their perspectives with evaluators during the horizon-scanning phase. Another key contribution is the role patients play in guiding clinical research by participating in scientific advice. Finally, during the assessment and appraisal stages, various methods are used to gather their views.

]]> How Patients Can Contribute to the Assessments of Health Technologies François Houÿez Julien Delaye doi: 10.3390/jmahp13040061 Journal of Market Access & Health Policy 2025-12-15 Journal of Market Access & Health Policy 2025-12-15 13 4 Perspective 61 10.3390/jmahp13040061 https://www.mdpi.com/2001-6689/13/4/61 JMAHP, Vol. 13, Pages 60: The Pharmaceutical Innovativeness Index: Supporting Value-Based Economic Regulation of Innovative Medicines https://www.mdpi.com/2001-6689/13/4/60 The regulation of new medicine prices must balance financial sustainability with equitable access to innovation. Value-Based Pricing (VBP) strategies seek to align drug prices with their clinical and societal impact. The Pharmaceutical Innovativeness Index (PII) is a transparent and reproducible tool proposed to assess the degree of innovativeness of new medicines, with potential to support pricing decisions within economic regulation frameworks. An exploratory qualitative study was conducted through a focus group study was conducted with experts in health economics and pharmaceutical regulation to evaluate the applicability of the PII and to discuss key domains relevant to the assessment of pharmaceutical innovation. Responses were collected anonymously using an interactive digital platform and analyzed through inductive thematic content analysis. Based on these findings, the research team developed a conceptual pricing model integrating the PII with additional value-based criteria. Two hypothetical case studies were created to explore its practical feasibility. Participants identified Added Therapeutic Value (ATV) and Unmet Therapeutic Need (UTN) as the most relevant domains, while Methodological Quality (MQ) and Study Design (SD) were also recognized as essential to ensure rigor and reduce bias. The PII scores showed strong alignment with expert judgment in the illustrative case studies. The proposed model incorporates international best practices—such as the efficiency frontier approach—and additional dimensions including safety and incremental innovation. Overall, the PII demonstrated potential to enhance transparency, consistency, and regulatory efficiency in drug pricing decisions in Brazil. However, it should currently be regarded as an exploratory framework requiring further empirical validation and regulatory adaptation before implementation. 2025-12-08 JMAHP, Vol. 13, Pages 60: The Pharmaceutical Innovativeness Index: Supporting Value-Based Economic Regulation of Innovative Medicines

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040060

Authors: Ludmila Peres Gargano Marcus Carvalho Borin Wallace Mateus Prata Juliana Alvares-Teodoro Francisco de Assis Acurcio Roberto F. Iunes Augusto Afonso Guerra

The regulation of new medicine prices must balance financial sustainability with equitable access to innovation. Value-Based Pricing (VBP) strategies seek to align drug prices with their clinical and societal impact. The Pharmaceutical Innovativeness Index (PII) is a transparent and reproducible tool proposed to assess the degree of innovativeness of new medicines, with potential to support pricing decisions within economic regulation frameworks. An exploratory qualitative study was conducted through a focus group study was conducted with experts in health economics and pharmaceutical regulation to evaluate the applicability of the PII and to discuss key domains relevant to the assessment of pharmaceutical innovation. Responses were collected anonymously using an interactive digital platform and analyzed through inductive thematic content analysis. Based on these findings, the research team developed a conceptual pricing model integrating the PII with additional value-based criteria. Two hypothetical case studies were created to explore its practical feasibility. Participants identified Added Therapeutic Value (ATV) and Unmet Therapeutic Need (UTN) as the most relevant domains, while Methodological Quality (MQ) and Study Design (SD) were also recognized as essential to ensure rigor and reduce bias. The PII scores showed strong alignment with expert judgment in the illustrative case studies. The proposed model incorporates international best practices—such as the efficiency frontier approach—and additional dimensions including safety and incremental innovation. Overall, the PII demonstrated potential to enhance transparency, consistency, and regulatory efficiency in drug pricing decisions in Brazil. However, it should currently be regarded as an exploratory framework requiring further empirical validation and regulatory adaptation before implementation.

]]> The Pharmaceutical Innovativeness Index: Supporting Value-Based Economic Regulation of Innovative Medicines Ludmila Peres Gargano Marcus Carvalho Borin Wallace Mateus Prata Juliana Alvares-Teodoro Francisco de Assis Acurcio Roberto F. Iunes Augusto Afonso Guerra doi: 10.3390/jmahp13040060 Journal of Market Access & Health Policy 2025-12-08 Journal of Market Access & Health Policy 2025-12-08 13 4 Article 60 10.3390/jmahp13040060 https://www.mdpi.com/2001-6689/13/4/60 JMAHP, Vol. 13, Pages 59: Cost-Effectiveness of Electrical Stimulation Therapy in the Treatment of Chronic Wounds: A Systematic Review, Meta-Analysis and Economic Analysis https://www.mdpi.com/2001-6689/13/4/59 Hard-to-heal wounds are a major burden to healthcare systems. Electrical stimulation therapy (EST) is known to improve clinical outcomes, but cost-effectiveness analysis is lacking. The aim was to explore the cost-effectiveness of EST with standard of care (SoC) versus SoC alone. A systematic review and meta-analysis of randomised controlled studies (RCTs) were conducted. Fourteen RCTs were identified, representing 783 patients. EST + SoC, versus SoC alone, significantly increased the proportion of wounds healed (odds ratio [OR] 2.46 [95% CI, 1.75–3.46], p < 0.0001) and significantly decreased the mean time to healing (−2.67 weeks (95% CI, 1.49–3.84, p < 0.00001). A cost-effectiveness model was developed based on these findings and on the usage and cost of the EST device used in the largest included RCT. Weekly costs of community wound care were taken from published estimates and inflated to 2024 levels, reflecting costs in the UK. In a hypothetical cohort of 100 patients treated over 12 weeks, EST + SoC was estimated to save over GBP 38,000 overall, reduce nursing visits by 385 and lead to 154 more ulcer-free weeks. In conclusion, EST used in the community is a cost-effective addition to SoC with the ability to improve outcomes and reduce human and financial burden of hard-to-heal wounds. 2025-11-24 JMAHP, Vol. 13, Pages 59: Cost-Effectiveness of Electrical Stimulation Therapy in the Treatment of Chronic Wounds: A Systematic Review, Meta-Analysis and Economic Analysis

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040059

Authors: Jennifer M. Smith John Posnett Emma J. Woodmansey

Hard-to-heal wounds are a major burden to healthcare systems. Electrical stimulation therapy (EST) is known to improve clinical outcomes, but cost-effectiveness analysis is lacking. The aim was to explore the cost-effectiveness of EST with standard of care (SoC) versus SoC alone. A systematic review and meta-analysis of randomised controlled studies (RCTs) were conducted. Fourteen RCTs were identified, representing 783 patients. EST + SoC, versus SoC alone, significantly increased the proportion of wounds healed (odds ratio [OR] 2.46 [95% CI, 1.75–3.46], p < 0.0001) and significantly decreased the mean time to healing (−2.67 weeks (95% CI, 1.49–3.84, p < 0.00001). A cost-effectiveness model was developed based on these findings and on the usage and cost of the EST device used in the largest included RCT. Weekly costs of community wound care were taken from published estimates and inflated to 2024 levels, reflecting costs in the UK. In a hypothetical cohort of 100 patients treated over 12 weeks, EST + SoC was estimated to save over GBP 38,000 overall, reduce nursing visits by 385 and lead to 154 more ulcer-free weeks. In conclusion, EST used in the community is a cost-effective addition to SoC with the ability to improve outcomes and reduce human and financial burden of hard-to-heal wounds.

]]> Cost-Effectiveness of Electrical Stimulation Therapy in the Treatment of Chronic Wounds: A Systematic Review, Meta-Analysis and Economic Analysis Jennifer M. Smith John Posnett Emma J. Woodmansey doi: 10.3390/jmahp13040059 Journal of Market Access & Health Policy 2025-11-24 Journal of Market Access & Health Policy 2025-11-24 13 4 Systematic Review 59 10.3390/jmahp13040059 https://www.mdpi.com/2001-6689/13/4/59 JMAHP, Vol. 13, Pages 58: Network Meta-Analysis of Bevacizumab Gamma Versus Competing Interventions for Treating Neovascular Age-Related Macular Degeneration in the United Kingdom https://www.mdpi.com/2001-6689/13/4/58 This study aimed to determine the relative efficacy of bevacizumab gamma (an ophthalmic formulation of bevacizumab) versus alternative interventions relevant to the treatment of neovascular age-related macular degeneration (nAMD) in the United Kingdom (UK) via a systematic literature review (SLR) and network meta-analysis (NMA). An SLR was conducted to identify randomized controlled trials (RCTs) of anti-vascular endothelial growth factor (anti-VEGF) therapies for the treatment of nAMD in adult patients relevant to the UK context. The included anti-VEGF treatments were ranibizumab, aflibercept, faricimab, and bevacizumab gamma. Bayesian NMA models were used to estimate relative efficacy in terms of change from baseline (CFB) in best-corrected visual acuity (BCVA) at 12 months, the proportion of patients gaining 15 or more letters at 12 months, and the proportion of patients losing less than 15 letters at 12 months. Twenty-two relevant RCTs were included in the NMA. At 12 months, all anti-VEGF treatments were similarly efficacious to ranibizumab 0.5 mg every four weeks (Q4W) in terms of CFB in BCVA, the proportion of patients gaining 15 or more letters, and the proportion of patients losing less than 15 letters (except for ranibizumab 0.5 mg every 12 weeks [Q12W] and ranibizumab 0.5 mg pro re nata [PRN]). Bevacizumab gamma provided similar improvements in visual acuity to other anti-VEGF treatments. 2025-11-19 JMAHP, Vol. 13, Pages 58: Network Meta-Analysis of Bevacizumab Gamma Versus Competing Interventions for Treating Neovascular Age-Related Macular Degeneration in the United Kingdom

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040058

Authors: Maria Lorenzi Stephen Ebohon Jennifer Kissner Jedd Comiskey Mayke Paap Christine Bouchet Andy Garnham Erika Wissinger

This study aimed to determine the relative efficacy of bevacizumab gamma (an ophthalmic formulation of bevacizumab) versus alternative interventions relevant to the treatment of neovascular age-related macular degeneration (nAMD) in the United Kingdom (UK) via a systematic literature review (SLR) and network meta-analysis (NMA). An SLR was conducted to identify randomized controlled trials (RCTs) of anti-vascular endothelial growth factor (anti-VEGF) therapies for the treatment of nAMD in adult patients relevant to the UK context. The included anti-VEGF treatments were ranibizumab, aflibercept, faricimab, and bevacizumab gamma. Bayesian NMA models were used to estimate relative efficacy in terms of change from baseline (CFB) in best-corrected visual acuity (BCVA) at 12 months, the proportion of patients gaining 15 or more letters at 12 months, and the proportion of patients losing less than 15 letters at 12 months. Twenty-two relevant RCTs were included in the NMA. At 12 months, all anti-VEGF treatments were similarly efficacious to ranibizumab 0.5 mg every four weeks (Q4W) in terms of CFB in BCVA, the proportion of patients gaining 15 or more letters, and the proportion of patients losing less than 15 letters (except for ranibizumab 0.5 mg every 12 weeks [Q12W] and ranibizumab 0.5 mg pro re nata [PRN]). Bevacizumab gamma provided similar improvements in visual acuity to other anti-VEGF treatments.

]]> Network Meta-Analysis of Bevacizumab Gamma Versus Competing Interventions for Treating Neovascular Age-Related Macular Degeneration in the United Kingdom Maria Lorenzi Stephen Ebohon Jennifer Kissner Jedd Comiskey Mayke Paap Christine Bouchet Andy Garnham Erika Wissinger doi: 10.3390/jmahp13040058 Journal of Market Access & Health Policy 2025-11-19 Journal of Market Access & Health Policy 2025-11-19 13 4 Systematic Review 58 10.3390/jmahp13040058 https://www.mdpi.com/2001-6689/13/4/58 JMAHP, Vol. 13, Pages 57: Criterion-Related Validity and Reliability of a Measurement Tool for Medical Doctors’ Work-Related Quality of Life in Japan https://www.mdpi.com/2001-6689/13/4/57 Objective: This confirmatory survey aimed to verify the criterion-related validity and reliability of the final version of the Medical Doctors’ Work-Related Quality of Life Questionnaire (WQMD-9), following partial revision of its content. This study also explored the questionnaire’s structure and scoring methods. Method: From June to July 2022, the WQMD-9 was administered to 98 MDs selected to match the statistical distribution of MDs in Japan. Criterion-related validity was evaluated using a visual analogue scale (VAS) as the reference standard, and reliability was examined using inter-dimension correlations and Cronbach’s α. Results: The correlation coefficient between the VAS score and the simple sum of WQMD-9 dimensions scores was 0.7891, supporting criterion-related validity. Cronbach’s α was 0.87, indicating acceptable reliability. Conclusions: The profile-type WQMD-9 consists of nine dimensions—“Workload,” “Working time,” “Collaboration,” “Clinical practice,” “Working conditions,” “Working environment,” “Feelings of fatigue,” “Work-life balance,” and “Career”—with five levels. In the confirmatory survey population, the WQMD-9 demonstrated criterion-related validity and reliability, suggesting that it can be utilized with simple total scoring approach. 2025-11-14 JMAHP, Vol. 13, Pages 57: Criterion-Related Validity and Reliability of a Measurement Tool for Medical Doctors’ Work-Related Quality of Life in Japan

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040057

Authors: Miyuki Ezura Katsuhiko Sawada Yusuke Takushima Lida Teng Ataru Igarashi

Objective: This confirmatory survey aimed to verify the criterion-related validity and reliability of the final version of the Medical Doctors’ Work-Related Quality of Life Questionnaire (WQMD-9), following partial revision of its content. This study also explored the questionnaire’s structure and scoring methods. Method: From June to July 2022, the WQMD-9 was administered to 98 MDs selected to match the statistical distribution of MDs in Japan. Criterion-related validity was evaluated using a visual analogue scale (VAS) as the reference standard, and reliability was examined using inter-dimension correlations and Cronbach’s α. Results: The correlation coefficient between the VAS score and the simple sum of WQMD-9 dimensions scores was 0.7891, supporting criterion-related validity. Cronbach’s α was 0.87, indicating acceptable reliability. Conclusions: The profile-type WQMD-9 consists of nine dimensions—“Workload,” “Working time,” “Collaboration,” “Clinical practice,” “Working conditions,” “Working environment,” “Feelings of fatigue,” “Work-life balance,” and “Career”—with five levels. In the confirmatory survey population, the WQMD-9 demonstrated criterion-related validity and reliability, suggesting that it can be utilized with simple total scoring approach.

]]> Criterion-Related Validity and Reliability of a Measurement Tool for Medical Doctors’ Work-Related Quality of Life in Japan Miyuki Ezura Katsuhiko Sawada Yusuke Takushima Lida Teng Ataru Igarashi doi: 10.3390/jmahp13040057 Journal of Market Access & Health Policy 2025-11-14 Journal of Market Access & Health Policy 2025-11-14 13 4 Article 57 10.3390/jmahp13040057 https://www.mdpi.com/2001-6689/13/4/57 JMAHP, Vol. 13, Pages 56: Patient Perspectives on Health Insurance Design: A Mixed-Methods Analysis https://www.mdpi.com/2001-6689/13/4/56 There remains a persistent lack of patient-centered evidence on insurance reform and real-world experiences of patients with chronic disease. This study gathered insights around insurance design from chronic disease beneficiaries. This mixed-methods analysis comprised an online survey and virtual focus group sessions (August to December 2023) involving US residents with chronic disease and health insurance. Patients’ perspectives on insurance design were explored. Survey data were analyzed descriptively. Key themes were identified from focus group transcripts and direct observations. In total, 146 patients across 15 chronic diseases completed the survey; 29 then participated in focus groups. Although most beneficiaries were satisfied with their health plan, concerns centered on prescription medication affordability due to high deductibles and cost exposure, the disproportionate effects of cost exposure based on income, and the unpredictability of out-of-pocket costs. For some, the financial burden led to financial debt, therapy abandonment, mental health issues, and/or worsening of their condition. Overall, there was broad support for policy solutions to redesign insurance and adjust cost exposure for patients with chronic disease. This research offers valuable patient insights into health insurance design in the US to ensure patients’ needs are addressed. 2025-11-14 JMAHP, Vol. 13, Pages 56: Patient Perspectives on Health Insurance Design: A Mixed-Methods Analysis

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040056

Authors: Bridget Doherty Kimberly Hooks Ulrich Neumann Wesley Peters Steven Zona Lisa Shea

There remains a persistent lack of patient-centered evidence on insurance reform and real-world experiences of patients with chronic disease. This study gathered insights around insurance design from chronic disease beneficiaries. This mixed-methods analysis comprised an online survey and virtual focus group sessions (August to December 2023) involving US residents with chronic disease and health insurance. Patients’ perspectives on insurance design were explored. Survey data were analyzed descriptively. Key themes were identified from focus group transcripts and direct observations. In total, 146 patients across 15 chronic diseases completed the survey; 29 then participated in focus groups. Although most beneficiaries were satisfied with their health plan, concerns centered on prescription medication affordability due to high deductibles and cost exposure, the disproportionate effects of cost exposure based on income, and the unpredictability of out-of-pocket costs. For some, the financial burden led to financial debt, therapy abandonment, mental health issues, and/or worsening of their condition. Overall, there was broad support for policy solutions to redesign insurance and adjust cost exposure for patients with chronic disease. This research offers valuable patient insights into health insurance design in the US to ensure patients’ needs are addressed.

]]> Patient Perspectives on Health Insurance Design: A Mixed-Methods Analysis Bridget Doherty Kimberly Hooks Ulrich Neumann Wesley Peters Steven Zona Lisa Shea doi: 10.3390/jmahp13040056 Journal of Market Access & Health Policy 2025-11-14 Journal of Market Access & Health Policy 2025-11-14 13 4 Article 56 10.3390/jmahp13040056 https://www.mdpi.com/2001-6689/13/4/56 JMAHP, Vol. 13, Pages 55: The Impact of Copay Accumulators and Maximizers on Treatment Patterns, Adherence, and Costs Among Patients with Major Depressive and Bipolar Disorders Treated with Branded Therapies https://www.mdpi.com/2001-6689/13/4/55 Copay accumulator (CA) and copay maximizer (CM) programs in the United States, which prevent manufacturer copay assistance from counting toward deductibles or out-of-pocket (OOP) maximums, are increasingly used, raising concerns about costs and outcomes for patients with major depressive disorder (MDD) or bipolar disorder (BPD) treated with branded atypical antipsychotics (AAPs) and/or antidepressants (ADs). This retrospective claims study used Kythera commercial data (2020–2024) in the United States to identify adults with MDD or BPD who had at least 1 diagnosis and one branded AAP or AD prescription between 2021 and 2023, requiring 12 months’ continuous enrollment pre- (2020–2021) and post-index (2023–2024) and at least three months of post-index branded medication use. This retrospective claims study used Kythera commercial data (2020–2024) to identify adults with MDD or BPD who had at least one diagnosis and one branded AAP or AD prescription between 2021 and 2023, requiring 12 months’ continuous enrollment pre- and post-index and at least 3 months of post-index branded medication use. Patients were stratified into CA, CM, or standard copay plan (SCP) cohorts, and propensity score matching was used to compare treatment patterns and costs. Both CA and CM groups had significantly higher median OOP costs than SCPs (e.g., $75/$60 vs. $16 for MDD+AAP; p < 0.0001), and higher pharmacy costs among adherent patients. CA patients had poorer adherence and persistence, shorter treatment duration, and higher discontinuation and abandonment rates than SCPs. These findings highlight higher OOP burden and adherence challenges with CA and CM programs, underscoring the need for careful benefit design for US mental health patients. 2025-11-07 JMAHP, Vol. 13, Pages 55: The Impact of Copay Accumulators and Maximizers on Treatment Patterns, Adherence, and Costs Among Patients with Major Depressive and Bipolar Disorders Treated with Branded Therapies

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040055

Authors: Onur Baser Katarzyna Rodchenko Heidi C. Waters Matthew Sullivan Lixuan Wu Shuangrui Chen Madeline Shurtleff Cynthia Bigley Rashmi Patel

Copay accumulator (CA) and copay maximizer (CM) programs in the United States, which prevent manufacturer copay assistance from counting toward deductibles or out-of-pocket (OOP) maximums, are increasingly used, raising concerns about costs and outcomes for patients with major depressive disorder (MDD) or bipolar disorder (BPD) treated with branded atypical antipsychotics (AAPs) and/or antidepressants (ADs). This retrospective claims study used Kythera commercial data (2020–2024) in the United States to identify adults with MDD or BPD who had at least 1 diagnosis and one branded AAP or AD prescription between 2021 and 2023, requiring 12 months’ continuous enrollment pre- (2020–2021) and post-index (2023–2024) and at least three months of post-index branded medication use. This retrospective claims study used Kythera commercial data (2020–2024) to identify adults with MDD or BPD who had at least one diagnosis and one branded AAP or AD prescription between 2021 and 2023, requiring 12 months’ continuous enrollment pre- and post-index and at least 3 months of post-index branded medication use. Patients were stratified into CA, CM, or standard copay plan (SCP) cohorts, and propensity score matching was used to compare treatment patterns and costs. Both CA and CM groups had significantly higher median OOP costs than SCPs (e.g., $75/$60 vs. $16 for MDD+AAP; p < 0.0001), and higher pharmacy costs among adherent patients. CA patients had poorer adherence and persistence, shorter treatment duration, and higher discontinuation and abandonment rates than SCPs. These findings highlight higher OOP burden and adherence challenges with CA and CM programs, underscoring the need for careful benefit design for US mental health patients.

]]> The Impact of Copay Accumulators and Maximizers on Treatment Patterns, Adherence, and Costs Among Patients with Major Depressive and Bipolar Disorders Treated with Branded Therapies Onur Baser Katarzyna Rodchenko Heidi C. Waters Matthew Sullivan Lixuan Wu Shuangrui Chen Madeline Shurtleff Cynthia Bigley Rashmi Patel doi: 10.3390/jmahp13040055 Journal of Market Access & Health Policy 2025-11-07 Journal of Market Access & Health Policy 2025-11-07 13 4 Article 55 10.3390/jmahp13040055 https://www.mdpi.com/2001-6689/13/4/55 JMAHP, Vol. 13, Pages 54: How the Stakeholders’ Perception Contributes to the Pharmaceutical Strategies: A Regional Case Study in Latin America https://www.mdpi.com/2001-6689/13/4/54 Background: Stakeholders’ perception plays a crucial role in shaping pharmaceutical strategies. Stakeholders are groups interested in pharmaceutical companies’ success and outcomes. Stakeholders’ perceptions are multifaceted and impact pharmaceutical strategies, from shaping research to enhancing market access, pricing, and corporate reputation. Understanding and actively managing stakeholders’ perceptions is vital for pharmaceutical companies to succeed in an increasingly complex and competitive industry. Methods: In this case study, knowledge contributions from stakeholders offered insights and strategies for application in the pharmaceutical sector. Results: Qualitative, exploratory research was conducted, which included the participation of sixteen stakeholders from different countries in Latin America, who responded to a semi-structured interview script, whose data were understood through lexical analysis in the Interface de R pour les Analyses Multimensionnelles de Texts et de Questionnaires (IRaMuTeQ). Conclusions: The results of this study underscore the importance of regulatory knowledge for professionals’ support and implementation of international strategies. Regulatory knowledge provides professionals with tools and insights to navigate complex regulatory environments, make informed decisions, and enhance organizational performance in global markets. 2025-10-23 JMAHP, Vol. 13, Pages 54: How the Stakeholders’ Perception Contributes to the Pharmaceutical Strategies: A Regional Case Study in Latin America

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040054

Authors: Talita da Silva Ferreira Giovanni M. Pauletti Luis Vázquez-Suárez

Background: Stakeholders’ perception plays a crucial role in shaping pharmaceutical strategies. Stakeholders are groups interested in pharmaceutical companies’ success and outcomes. Stakeholders’ perceptions are multifaceted and impact pharmaceutical strategies, from shaping research to enhancing market access, pricing, and corporate reputation. Understanding and actively managing stakeholders’ perceptions is vital for pharmaceutical companies to succeed in an increasingly complex and competitive industry. Methods: In this case study, knowledge contributions from stakeholders offered insights and strategies for application in the pharmaceutical sector. Results: Qualitative, exploratory research was conducted, which included the participation of sixteen stakeholders from different countries in Latin America, who responded to a semi-structured interview script, whose data were understood through lexical analysis in the Interface de R pour les Analyses Multimensionnelles de Texts et de Questionnaires (IRaMuTeQ). Conclusions: The results of this study underscore the importance of regulatory knowledge for professionals’ support and implementation of international strategies. Regulatory knowledge provides professionals with tools and insights to navigate complex regulatory environments, make informed decisions, and enhance organizational performance in global markets.

]]> How the Stakeholders’ Perception Contributes to the Pharmaceutical Strategies: A Regional Case Study in Latin America Talita da Silva Ferreira Giovanni M. Pauletti Luis Vázquez-Suárez doi: 10.3390/jmahp13040054 Journal of Market Access & Health Policy 2025-10-23 Journal of Market Access & Health Policy 2025-10-23 13 4 Article 54 10.3390/jmahp13040054 https://www.mdpi.com/2001-6689/13/4/54 JMAHP, Vol. 13, Pages 53: A Data-Driven Approach for Estimating Type 2 Diabetes-Related Costs in Greece https://www.mdpi.com/2001-6689/13/4/53 Type 2 diabetes (T2D) constitutes a major health problem, reaching alarming rates over the last decades, especially due to contemporary lifestyle and associated obesogenic environments, as well as the aging population. Diabetes not only causes social consequences but also leads to increasing healthcare costs, posing a significant challenge for the health system. This paper applies a five-step approach for estimating T2D-related costs in Greece. The approach initially estimates the T2D-related ICD10 prevalence and the target population. Next it applies the appropriate therapeutic protocols to identify the most appropriate treatments. Subsequently, it calculates the total cost of medical treatments for each target population, based on the distribution of patients between the different treatments and treatment lines. Finally, based on the diagnostic and treatment protocols, it calculates the annual direct costs associated with the cost categories. Using the estimated future population of the country, the proposed methodology can also project the budget required, under certain conditions, to deal with T2D. The analysis estimated that T2D-related costs in 2021 under rational use of resources were EUR 1,397,871,172.55 billion and EUR 1,512,934,947.63 billion projected in the year 2030 considering the aging effect, per cost category, and in total, presenting an increase of approximately 115 million euros in 2030 compared to 2021. The term “rational use of resources” in this study refers to the use of internationally recognized, evidence-based diagnostic and therapeutic protocols, as adopted by the Greek Ministry of Health. This scenario represents an idealized standard of care rather than actual real-world adherence and is used to estimate the potential resource needs under optimal medical practice conditions. An inflation rate of 4.2% was applied to costs between 2021 and 2030. The analysis showed that the highest percentage (39%) of the total T2D-related healthcare expenditures is associated with complications that occur in T2D patients. Despite a comparatively modest prevalence of T2D in Greece relative to other European and Mediterranean countries, the economic burden associated with its management remains high. The aging of the population will lead to an increase in the total cost of T2D. The applied methodology of estimating budgets by aggregating categories of expenses under a specific disease (ICD10), instead of dividing budgets into categories of expenses, can successfully lead to the optimization and rationalization of expenses according to actual needs. The findings underline the significant economic burden of T2D in Greece, particularly due to complications and population aging. These results emphasize the urgent need for health policy strategies focusing on prevention, early intervention, and the efficient allocation of healthcare resources. The methodology applied can serve as a decision-making tool for forecasting healthcare budgets and optimizing expenditures under different population and treatment scenarios. 2025-10-15 JMAHP, Vol. 13, Pages 53: A Data-Driven Approach for Estimating Type 2 Diabetes-Related Costs in Greece

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040053

Authors: Elisavet Nika Thomas Tsiampalis Athanasios Sachlas Evangelos Liberopoulos Sotirios Bersimis Dimitrios Georgakellos

Type 2 diabetes (T2D) constitutes a major health problem, reaching alarming rates over the last decades, especially due to contemporary lifestyle and associated obesogenic environments, as well as the aging population. Diabetes not only causes social consequences but also leads to increasing healthcare costs, posing a significant challenge for the health system. This paper applies a five-step approach for estimating T2D-related costs in Greece. The approach initially estimates the T2D-related ICD10 prevalence and the target population. Next it applies the appropriate therapeutic protocols to identify the most appropriate treatments. Subsequently, it calculates the total cost of medical treatments for each target population, based on the distribution of patients between the different treatments and treatment lines. Finally, based on the diagnostic and treatment protocols, it calculates the annual direct costs associated with the cost categories. Using the estimated future population of the country, the proposed methodology can also project the budget required, under certain conditions, to deal with T2D. The analysis estimated that T2D-related costs in 2021 under rational use of resources were EUR 1,397,871,172.55 billion and EUR 1,512,934,947.63 billion projected in the year 2030 considering the aging effect, per cost category, and in total, presenting an increase of approximately 115 million euros in 2030 compared to 2021. The term “rational use of resources” in this study refers to the use of internationally recognized, evidence-based diagnostic and therapeutic protocols, as adopted by the Greek Ministry of Health. This scenario represents an idealized standard of care rather than actual real-world adherence and is used to estimate the potential resource needs under optimal medical practice conditions. An inflation rate of 4.2% was applied to costs between 2021 and 2030. The analysis showed that the highest percentage (39%) of the total T2D-related healthcare expenditures is associated with complications that occur in T2D patients. Despite a comparatively modest prevalence of T2D in Greece relative to other European and Mediterranean countries, the economic burden associated with its management remains high. The aging of the population will lead to an increase in the total cost of T2D. The applied methodology of estimating budgets by aggregating categories of expenses under a specific disease (ICD10), instead of dividing budgets into categories of expenses, can successfully lead to the optimization and rationalization of expenses according to actual needs. The findings underline the significant economic burden of T2D in Greece, particularly due to complications and population aging. These results emphasize the urgent need for health policy strategies focusing on prevention, early intervention, and the efficient allocation of healthcare resources. The methodology applied can serve as a decision-making tool for forecasting healthcare budgets and optimizing expenditures under different population and treatment scenarios.

]]> A Data-Driven Approach for Estimating Type 2 Diabetes-Related Costs in Greece Elisavet Nika Thomas Tsiampalis Athanasios Sachlas Evangelos Liberopoulos Sotirios Bersimis Dimitrios Georgakellos doi: 10.3390/jmahp13040053 Journal of Market Access & Health Policy 2025-10-15 Journal of Market Access & Health Policy 2025-10-15 13 4 Article 53 10.3390/jmahp13040053 https://www.mdpi.com/2001-6689/13/4/53 JMAHP, Vol. 13, Pages 52: EU Joint Clinical Assessment: A Framework for Optimising Use with Cost-Effectiveness Decision-Making https://www.mdpi.com/2001-6689/13/4/52 The introduction of the European Union (EU) Joint Clinical Assessment (JCA) under Regulation (EU) 2021/2282 marks a transformative step in harmonizing health technology assessments (HTAs) across EU member states. This article explores the implications of JCA, particularly in oncology, for member states who utilize cost-effectiveness (CE) analysis and health technology developers (HTDs) who produce this evidence. The JCA framework attempts to standardise the assessment of relative clinical effectiveness and safety across the EU to input into national appraisals. Importantly, it excludes economic evaluations that may be required nationally, necessitating HTDs to align their CE models with the JCA PICO (Population/Intervention/Comparator/Outcome) parameters outlined by member states. This article discusses the challenges and opportunities for aligning JCA and CE modelling outcomes, contrasting evidence requirements between JCA and CE frameworks. It highlights the potential increase in complexity due to the diverse comparators in PICO surveys, necessitating the use of indirect comparison methodologies. It further underscores the importance of early communication between HTDs and HTA bodies to ensure timely, relevant, and pragmatic decision-making. By sharing national PICOs upfront to support national evidence generation, the JCA framework’s potential to aid high-quality decision-making and improve patient access to innovative medicines can be maximised. 2025-10-09 JMAHP, Vol. 13, Pages 52: EU Joint Clinical Assessment: A Framework for Optimising Use with Cost-Effectiveness Decision-Making

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040052

Authors: Adam Johns André Andrade Sukhvinder Johal James Ryan

The introduction of the European Union (EU) Joint Clinical Assessment (JCA) under Regulation (EU) 2021/2282 marks a transformative step in harmonizing health technology assessments (HTAs) across EU member states. This article explores the implications of JCA, particularly in oncology, for member states who utilize cost-effectiveness (CE) analysis and health technology developers (HTDs) who produce this evidence. The JCA framework attempts to standardise the assessment of relative clinical effectiveness and safety across the EU to input into national appraisals. Importantly, it excludes economic evaluations that may be required nationally, necessitating HTDs to align their CE models with the JCA PICO (Population/Intervention/Comparator/Outcome) parameters outlined by member states. This article discusses the challenges and opportunities for aligning JCA and CE modelling outcomes, contrasting evidence requirements between JCA and CE frameworks. It highlights the potential increase in complexity due to the diverse comparators in PICO surveys, necessitating the use of indirect comparison methodologies. It further underscores the importance of early communication between HTDs and HTA bodies to ensure timely, relevant, and pragmatic decision-making. By sharing national PICOs upfront to support national evidence generation, the JCA framework’s potential to aid high-quality decision-making and improve patient access to innovative medicines can be maximised.

]]> EU Joint Clinical Assessment: A Framework for Optimising Use with Cost-Effectiveness Decision-Making Adam Johns André Andrade Sukhvinder Johal James Ryan doi: 10.3390/jmahp13040052 Journal of Market Access & Health Policy 2025-10-09 Journal of Market Access & Health Policy 2025-10-09 13 4 Opinion 52 10.3390/jmahp13040052 https://www.mdpi.com/2001-6689/13/4/52 JMAHP, Vol. 13, Pages 51: Examining State Policies and Administrative Factors as Determinants of Consumer-Reported Unmet Service Needs in Publicly Funded Home- and Community-Based Services in the United States https://www.mdpi.com/2001-6689/13/4/51 Access to home- and community-based services (HCBSs) varies substantially between states. Yet, it is unknown how state-level policies and administrative factors impact consumer-reported unmet service needs, an important indicator of HCBS access and quality. Using the National Core Indicators—Aging and Disability Adult Consumer Survey (2016–2019; n = 13,654 community-dwelling older adults, 13 states), we examined associations between unmet HCBS needs with four state-level factors: HCBS spending relative to institutional care spending, HCBS spending per client, percentage of Medicaid beneficiaries in managed care, and Medicaid expansion; and funding program. In the adjusted logistic regression model, the odds of overall unmet HCBS needs were lower with higher percentage Medicaid beneficiaries in managed care (adjusted odds ratio [aOR], 0.92; 95% confidence interval [CI], 0.89–0.96) and Medicaid expansion (aOR, 0.80; 95% CI, 0.73–0.87) but greater with higher HCBS spending relative to institutional care spending (aOR, 1.19; 95% CI, 1.11–1.28). Compared to Medicaid waiver, odds of unmet HCBS needs were significantly lower among consumers in Managed Long-Term Services and Supports (aOR, 0.67; 95% CI, 0.61–0.74) and Program of All-Inclusive Care for the Elderly (PACE; aOR, 0.39; 95% CI, 0.31–0.49). State policies and administrative factors are important place-based determinants of HCBS consumers’ unmet HCBS needs/access; and warrant consideration in HCBS quality assurance and improvement. 2025-10-02 JMAHP, Vol. 13, Pages 51: Examining State Policies and Administrative Factors as Determinants of Consumer-Reported Unmet Service Needs in Publicly Funded Home- and Community-Based Services in the United States

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040051

Authors: Romil R. Parikh Tetyana P. Shippee Benjamin Langworthy Zheng Wang Stephanie Giordano Eric Jutkowitz

Access to home- and community-based services (HCBSs) varies substantially between states. Yet, it is unknown how state-level policies and administrative factors impact consumer-reported unmet service needs, an important indicator of HCBS access and quality. Using the National Core Indicators—Aging and Disability Adult Consumer Survey (2016–2019; n = 13,654 community-dwelling older adults, 13 states), we examined associations between unmet HCBS needs with four state-level factors: HCBS spending relative to institutional care spending, HCBS spending per client, percentage of Medicaid beneficiaries in managed care, and Medicaid expansion; and funding program. In the adjusted logistic regression model, the odds of overall unmet HCBS needs were lower with higher percentage Medicaid beneficiaries in managed care (adjusted odds ratio [aOR], 0.92; 95% confidence interval [CI], 0.89–0.96) and Medicaid expansion (aOR, 0.80; 95% CI, 0.73–0.87) but greater with higher HCBS spending relative to institutional care spending (aOR, 1.19; 95% CI, 1.11–1.28). Compared to Medicaid waiver, odds of unmet HCBS needs were significantly lower among consumers in Managed Long-Term Services and Supports (aOR, 0.67; 95% CI, 0.61–0.74) and Program of All-Inclusive Care for the Elderly (PACE; aOR, 0.39; 95% CI, 0.31–0.49). State policies and administrative factors are important place-based determinants of HCBS consumers’ unmet HCBS needs/access; and warrant consideration in HCBS quality assurance and improvement.

]]> Examining State Policies and Administrative Factors as Determinants of Consumer-Reported Unmet Service Needs in Publicly Funded Home- and Community-Based Services in the United States Romil R. Parikh Tetyana P. Shippee Benjamin Langworthy Zheng Wang Stephanie Giordano Eric Jutkowitz doi: 10.3390/jmahp13040051 Journal of Market Access & Health Policy 2025-10-02 Journal of Market Access & Health Policy 2025-10-02 13 4 Article 51 10.3390/jmahp13040051 https://www.mdpi.com/2001-6689/13/4/51 JMAHP, Vol. 13, Pages 50: Vaccine Development, Its Implementation and Price Setting: A Historical Perspective with Proposed Ways to Move Forward https://www.mdpi.com/2001-6689/13/4/50 Vaccination has resulted in substantial public health benefits for human populations worldwide since it was first introduced more than a century ago. This article presents an overview of the history of vaccine development, its implementation, and price setting, the latter mainly from a developed world perspective. It considers potential issues and challenges. Over time, vaccine development and production has evolved to a market-driven approach, conducted largely by private commercial entities. The complex processes of identifying potential vaccine targets and developing and producing vaccines at scale have now become more efficient. However, vaccine pricing is an emerging concern. The elements that maximize the overall health benefit of vaccination include high volume, high coverage, and rapid initial implementation to achieve the high coverage with the vaccine as quickly as possible. It therefore requires substantial initial investment. Consequently, the price set for the vaccine should be reasonable to avoid limiting the coverage given the available budget. Suboptimal coverage leads to suboptimal benefit if herd protection is not fully achieved. This may disappoint health authorities and may result in program discontinuation. Conventional cost-effectiveness analysis is therefore not ideally suited to vaccine price setting, as it is based on the concept of ‘more for more’, i.e., higher health gain achieved at a higher reimbursement cost that does not account for limited budgets. Constrained optimization (CO) combines value assessment with constrained budget allocation into one analysis method and may therefore be the better option for vaccine pricing. 2025-10-02 JMAHP, Vol. 13, Pages 50: Vaccine Development, Its Implementation and Price Setting: A Historical Perspective with Proposed Ways to Move Forward

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040050

Authors: Baudouin Standaert Oleksandr Topachevskyi Olivier Ethgen

Vaccination has resulted in substantial public health benefits for human populations worldwide since it was first introduced more than a century ago. This article presents an overview of the history of vaccine development, its implementation, and price setting, the latter mainly from a developed world perspective. It considers potential issues and challenges. Over time, vaccine development and production has evolved to a market-driven approach, conducted largely by private commercial entities. The complex processes of identifying potential vaccine targets and developing and producing vaccines at scale have now become more efficient. However, vaccine pricing is an emerging concern. The elements that maximize the overall health benefit of vaccination include high volume, high coverage, and rapid initial implementation to achieve the high coverage with the vaccine as quickly as possible. It therefore requires substantial initial investment. Consequently, the price set for the vaccine should be reasonable to avoid limiting the coverage given the available budget. Suboptimal coverage leads to suboptimal benefit if herd protection is not fully achieved. This may disappoint health authorities and may result in program discontinuation. Conventional cost-effectiveness analysis is therefore not ideally suited to vaccine price setting, as it is based on the concept of ‘more for more’, i.e., higher health gain achieved at a higher reimbursement cost that does not account for limited budgets. Constrained optimization (CO) combines value assessment with constrained budget allocation into one analysis method and may therefore be the better option for vaccine pricing.

]]> Vaccine Development, Its Implementation and Price Setting: A Historical Perspective with Proposed Ways to Move Forward Baudouin Standaert Oleksandr Topachevskyi Olivier Ethgen doi: 10.3390/jmahp13040050 Journal of Market Access & Health Policy 2025-10-02 Journal of Market Access & Health Policy 2025-10-02 13 4 Opinion 50 10.3390/jmahp13040050 https://www.mdpi.com/2001-6689/13/4/50 JMAHP, Vol. 13, Pages 49: Balancing Costs and Clinical Outcomes: A Cost-Effectiveness Study of PICC Types Across Chinese Healthcare Settings https://www.mdpi.com/2001-6689/13/4/49 Long-term intravenous therapies often necessitate the use of peripherally inserted central catheters (PICCs). Antimicrobial-coated PICCs have been introduced to minimize central line-associated bloodstream infections (CLABSIs). A decision-analytic cost-effectiveness model was developed from a societal perspective, utilizing real-world data concerning PICC-related complications and costs from Class 3A hospitals and community hospital settings in China. The analysis compared the quality-adjusted life years (QALYs) for patients receiving antimicrobial-coated PICCs versus standard PICCs, with catheter-associated costs included. Incremental cost-effectiveness ratios (ICERs) were calculated in Chinese Yuan (CNY) per QALY gained. Patients with antimicrobial-coated PICCs experienced slightly fewer complication-related events, leading to significantly lower costs for managing complications. In the Class 3A hospital setting, the average total cost per patient was lower with antimicrobial-coated PICCs (CNY 62,800) compared to standard PICCs (CNY 102,900), primarily due to the reduced expenses for treating CLABSIs and related unknown fever. The ICER demonstrated that the coated PICC was the strongest option, showing a negative ICER (cost-saving of approximately CNY 4 million per QALY gained in the base-case Class 3A hospital scenario). In community hospital care scenarios, the cost advantage of antimicrobial-coated PICCs remained. Thus, the antimicrobial-coated PICC strategy was cost-saving, providing equal or improved health outcomes at lower costs in China’s medical center and community hospitals, making it a more efficient choice for long-term vascular access. 2025-09-29 JMAHP, Vol. 13, Pages 49: Balancing Costs and Clinical Outcomes: A Cost-Effectiveness Study of PICC Types Across Chinese Healthcare Settings

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040049

Authors: Jia Xu Hossein Zare Xia Yan Herng-Chia Chiu Renan Castillo

Long-term intravenous therapies often necessitate the use of peripherally inserted central catheters (PICCs). Antimicrobial-coated PICCs have been introduced to minimize central line-associated bloodstream infections (CLABSIs). A decision-analytic cost-effectiveness model was developed from a societal perspective, utilizing real-world data concerning PICC-related complications and costs from Class 3A hospitals and community hospital settings in China. The analysis compared the quality-adjusted life years (QALYs) for patients receiving antimicrobial-coated PICCs versus standard PICCs, with catheter-associated costs included. Incremental cost-effectiveness ratios (ICERs) were calculated in Chinese Yuan (CNY) per QALY gained. Patients with antimicrobial-coated PICCs experienced slightly fewer complication-related events, leading to significantly lower costs for managing complications. In the Class 3A hospital setting, the average total cost per patient was lower with antimicrobial-coated PICCs (CNY 62,800) compared to standard PICCs (CNY 102,900), primarily due to the reduced expenses for treating CLABSIs and related unknown fever. The ICER demonstrated that the coated PICC was the strongest option, showing a negative ICER (cost-saving of approximately CNY 4 million per QALY gained in the base-case Class 3A hospital scenario). In community hospital care scenarios, the cost advantage of antimicrobial-coated PICCs remained. Thus, the antimicrobial-coated PICC strategy was cost-saving, providing equal or improved health outcomes at lower costs in China’s medical center and community hospitals, making it a more efficient choice for long-term vascular access.

]]> Balancing Costs and Clinical Outcomes: A Cost-Effectiveness Study of PICC Types Across Chinese Healthcare Settings Jia Xu Hossein Zare Xia Yan Herng-Chia Chiu Renan Castillo doi: 10.3390/jmahp13040049 Journal of Market Access & Health Policy 2025-09-29 Journal of Market Access & Health Policy 2025-09-29 13 4 Article 49 10.3390/jmahp13040049 https://www.mdpi.com/2001-6689/13/4/49 JMAHP, Vol. 13, Pages 48: Clinical and Economic Evidence Supporting the Value of Fluorescence Imaging of Bacteria in Wound Care https://www.mdpi.com/2001-6689/13/4/48 Wound infection significantly hinders the healing process. Clinical signs and symptoms (CSS) of infection are used to assess the presence of infection and guide whether to intervene. However, CSS may not be dependable, lacking sensitivity and specificity, and may not accurately reflect bacterial load. The interpretation of CSS can be subjective and can vary between clinicians since they depend on patient characteristics, type of wound, and stage of infection. In addition, conditions such as peripheral vascular disease or diabetes can mask the signs and symptoms of infection. Inaccurate or late diagnosis of infected wounds can be costly to the patient and to healthcare systems. Fluorescence imaging (FLI) provides a safe, objective, highly sensitive approach to detect clinically significant bacterial levels in wounds. This information allows individualized treatment plans and a way to monitor bacterial burden and wound healing longitudinally. This publication reviews the evidence for point-of-care FLI as a means of improving wound identification with a high bacterial burden and the clinical and healthcare economic benefits of earlier and more accurate detection of bacteria. 2025-09-26 JMAHP, Vol. 13, Pages 48: Clinical and Economic Evidence Supporting the Value of Fluorescence Imaging of Bacteria in Wound Care

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040048

Authors: Jonathan Johnson Gregory Bohn

Wound infection significantly hinders the healing process. Clinical signs and symptoms (CSS) of infection are used to assess the presence of infection and guide whether to intervene. However, CSS may not be dependable, lacking sensitivity and specificity, and may not accurately reflect bacterial load. The interpretation of CSS can be subjective and can vary between clinicians since they depend on patient characteristics, type of wound, and stage of infection. In addition, conditions such as peripheral vascular disease or diabetes can mask the signs and symptoms of infection. Inaccurate or late diagnosis of infected wounds can be costly to the patient and to healthcare systems. Fluorescence imaging (FLI) provides a safe, objective, highly sensitive approach to detect clinically significant bacterial levels in wounds. This information allows individualized treatment plans and a way to monitor bacterial burden and wound healing longitudinally. This publication reviews the evidence for point-of-care FLI as a means of improving wound identification with a high bacterial burden and the clinical and healthcare economic benefits of earlier and more accurate detection of bacteria.

]]> Clinical and Economic Evidence Supporting the Value of Fluorescence Imaging of Bacteria in Wound Care Jonathan Johnson Gregory Bohn doi: 10.3390/jmahp13040048 Journal of Market Access & Health Policy 2025-09-26 Journal of Market Access & Health Policy 2025-09-26 13 4 Review 48 10.3390/jmahp13040048 https://www.mdpi.com/2001-6689/13/4/48 JMAHP, Vol. 13, Pages 47: Shaping the Future of HTA in Italy: Insights from the Italian Health Policy Forum https://www.mdpi.com/2001-6689/13/4/47 The implementation of the European (EU) Health Technology Assessment (HTA) Regulation 2021/2282 (EU HTAR) offers many opportunities, aimed at harmonizing HTA procedures and improving access to innovations; it also represents a significant challenge for the European healthcare system. Within the 2024 Health Policy Forum Italy meeting, different actors, stakeholders, and institutions had the opportunity to discuss major criticism and opportunities coming from the EU-HTA Regulation addressing future developments in the healthcare sector. Two groups, EU & Italy Pharmaceuticals and EU-Italy Medical Devices, worked distinctively on the EU HTAR by highlighting key issues that may pose challenges at both European and national levels, proposing potential solutions. The allocation of participants into two groups, according to their affiliation with either the pharmaceutical or the medical device sector, enhances the diversity of professional backgrounds and institutional perspectives, thereby fostering a more comprehensive and informed discussion. The recommendations highlighted by the two groups emphasize the need to promote cooperation among Member States, strengthen training for decision-makers, and develop a monitoring system to evaluate EU HTA’s impact. 2025-09-24 JMAHP, Vol. 13, Pages 47: Shaping the Future of HTA in Italy: Insights from the Italian Health Policy Forum

Journal of Market Access & Health Policy doi: 10.3390/jmahp13040047

Authors: Paolo Sciattella Roberta Laurita Chiara Bini Eugenio Di Brino Dario Sacchini Giandomenico Nollo

The implementation of the European (EU) Health Technology Assessment (HTA) Regulation 2021/2282 (EU HTAR) offers many opportunities, aimed at harmonizing HTA procedures and improving access to innovations; it also represents a significant challenge for the European healthcare system. Within the 2024 Health Policy Forum Italy meeting, different actors, stakeholders, and institutions had the opportunity to discuss major criticism and opportunities coming from the EU-HTA Regulation addressing future developments in the healthcare sector. Two groups, EU & Italy Pharmaceuticals and EU-Italy Medical Devices, worked distinctively on the EU HTAR by highlighting key issues that may pose challenges at both European and national levels, proposing potential solutions. The allocation of participants into two groups, according to their affiliation with either the pharmaceutical or the medical device sector, enhances the diversity of professional backgrounds and institutional perspectives, thereby fostering a more comprehensive and informed discussion. The recommendations highlighted by the two groups emphasize the need to promote cooperation among Member States, strengthen training for decision-makers, and develop a monitoring system to evaluate EU HTA’s impact.

]]> Shaping the Future of HTA in Italy: Insights from the Italian Health Policy Forum Paolo Sciattella Roberta Laurita Chiara Bini Eugenio Di Brino Dario Sacchini Giandomenico Nollo doi: 10.3390/jmahp13040047 Journal of Market Access & Health Policy 2025-09-24 Journal of Market Access & Health Policy 2025-09-24 13 4 Opinion 47 10.3390/jmahp13040047 https://www.mdpi.com/2001-6689/13/4/47 JMAHP, Vol. 13, Pages 46: Shaping the Future of DHT Assessment: Insights on Industry Challenges, Developer Needs, and a Harmonized, European HTA Framework https://www.mdpi.com/2001-6689/13/3/46 Introduction: Market access, pricing, and reimbursement of digital health technologies (DHTs) in Europe are significantly challenged by regulatory fragmentation and various assessment methodologies. Understanding the challenges and priorities of technology developers is essential for developing effective and relevant policy responses. This study explores perceived barriers and developer-driven priorities to inform the development of a harmonized health technology assessment (HTA) framework under the EDiHTA project. Methods: A mixed-methods approach was adopted, including a scoping review to identify key challenges, a survey of 20 DHT developers, and interviews and focus groups with 29 industry representatives from startups to multinational companies across 10 European countries during 2024. Results: Key challenges included a lack of transparency in reimbursement processes, fragmented HTA requirements, and misalignment between traditional evidence models and the agile development of DHTs. Developers highlighted the need to integrate real-world evidence, consider usability and implementation factors, and provide structured, lifecycle-based guidance. Financial barriers and procedural burdens were particularly significant for small and medium-sized enterprises. Conclusions: These findings highlight the need for an HTA framework that reflects the iterative nature of digital development, integrates real-world evidence, and reduces uncertainty for developers. The EDiHTA project aims to respond to these challenges by building a harmonized and flexible approach that aligns with the goals of the European HTA Regulation. 2025-09-04 JMAHP, Vol. 13, Pages 46: Shaping the Future of DHT Assessment: Insights on Industry Challenges, Developer Needs, and a Harmonized, European HTA Framework

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030046

Authors: Fruzsina Mezei Emmanouil Tsiasiotis Michele Basile Ilaria Sciomenta Elena Maria Calosci Debora Antonini Adam Lukacs Rossella Di Bidino Americo Cicchetti Dario Sacchini

Introduction: Market access, pricing, and reimbursement of digital health technologies (DHTs) in Europe are significantly challenged by regulatory fragmentation and various assessment methodologies. Understanding the challenges and priorities of technology developers is essential for developing effective and relevant policy responses. This study explores perceived barriers and developer-driven priorities to inform the development of a harmonized health technology assessment (HTA) framework under the EDiHTA project. Methods: A mixed-methods approach was adopted, including a scoping review to identify key challenges, a survey of 20 DHT developers, and interviews and focus groups with 29 industry representatives from startups to multinational companies across 10 European countries during 2024. Results: Key challenges included a lack of transparency in reimbursement processes, fragmented HTA requirements, and misalignment between traditional evidence models and the agile development of DHTs. Developers highlighted the need to integrate real-world evidence, consider usability and implementation factors, and provide structured, lifecycle-based guidance. Financial barriers and procedural burdens were particularly significant for small and medium-sized enterprises. Conclusions: These findings highlight the need for an HTA framework that reflects the iterative nature of digital development, integrates real-world evidence, and reduces uncertainty for developers. The EDiHTA project aims to respond to these challenges by building a harmonized and flexible approach that aligns with the goals of the European HTA Regulation.

]]> Shaping the Future of DHT Assessment: Insights on Industry Challenges, Developer Needs, and a Harmonized, European HTA Framework Fruzsina Mezei Emmanouil Tsiasiotis Michele Basile Ilaria Sciomenta Elena Maria Calosci Debora Antonini Adam Lukacs Rossella Di Bidino Americo Cicchetti Dario Sacchini doi: 10.3390/jmahp13030046 Journal of Market Access & Health Policy 2025-09-04 Journal of Market Access & Health Policy 2025-09-04 13 3 Article 46 10.3390/jmahp13030046 https://www.mdpi.com/2001-6689/13/3/46 JMAHP, Vol. 13, Pages 45: Safe Staffing Standards for Pharmacy Technicians in Hospital Settings https://www.mdpi.com/2001-6689/13/3/45 Pharmacy technicians (PT) are vital to the efficient and safe operation of hospital pharmacy services, fulfilling a range of technical and clinical responsibilities that directly impact patient care. However, increasing healthcare demands have underscored the importance of adequate staffing levels to sustain service quality and safeguard patient outcomes. This perspective paper explores how appropriate staffing levels for PT in hospital settings are essential and important to support safe, efficient care and a sustainable workforce. It compares evidence-informed staffing models, highlights real-world benchmarks, and proposes governance recommendations to guide policies that strengthen pharmacy services. Recommendations are made to inform clinical governance, suggesting that staffing policies, continuous training, and professional development programs are essential to supporting PT effectiveness and retention. The findings advocate for regulated staffing ratios and governance measures to foster an environment where PTs can deliver high-quality care and uphold safety standards within hospital pharmacies. 2025-09-04 JMAHP, Vol. 13, Pages 45: Safe Staffing Standards for Pharmacy Technicians in Hospital Settings

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030045

Authors: Vítor Silva João José Joaquim Shane Desselle Samantha Quaye Cristiano Matos

Pharmacy technicians (PT) are vital to the efficient and safe operation of hospital pharmacy services, fulfilling a range of technical and clinical responsibilities that directly impact patient care. However, increasing healthcare demands have underscored the importance of adequate staffing levels to sustain service quality and safeguard patient outcomes. This perspective paper explores how appropriate staffing levels for PT in hospital settings are essential and important to support safe, efficient care and a sustainable workforce. It compares evidence-informed staffing models, highlights real-world benchmarks, and proposes governance recommendations to guide policies that strengthen pharmacy services. Recommendations are made to inform clinical governance, suggesting that staffing policies, continuous training, and professional development programs are essential to supporting PT effectiveness and retention. The findings advocate for regulated staffing ratios and governance measures to foster an environment where PTs can deliver high-quality care and uphold safety standards within hospital pharmacies.

]]> Safe Staffing Standards for Pharmacy Technicians in Hospital Settings Vítor Silva João José Joaquim Shane Desselle Samantha Quaye Cristiano Matos doi: 10.3390/jmahp13030045 Journal of Market Access & Health Policy 2025-09-04 Journal of Market Access & Health Policy 2025-09-04 13 3 Perspective 45 10.3390/jmahp13030045 https://www.mdpi.com/2001-6689/13/3/45 JMAHP, Vol. 13, Pages 44: Value-Based Healthcare as a Competitive Strategy—A Multi-Stakeholder Perception Analysis in Portuguese Healthcare https://www.mdpi.com/2001-6689/13/3/44 Designing an accessible, financially viable healthcare system is a key challenge for society. The value-based healthcare (VBHC) strategic model aims to simultaneously improve the quality of healthcare and the efficiency of health systems. The aim of this research was to describe the perceptions of different stakeholders in the Portuguese health industry about the creation of value and the understanding of VBHC as a competitive advantage. A qualitative study was conducted using the inductive method of Braun and Clarke, designed according to the COREQ criteria. Based on the results of the literature review, a semi-structured script for an interview was created, consisting of eight questions. The initial interview script was based on a thorough narrative literature review and tested with two professionals with practical experience in VBHC. The final version of the semi-structured interview guide consisted of eight open-ended questions. The questions were designed to elicit in-depth, reflective responses, and their neutrality was reviewed to avoid leading language that might introduce bias. As the interviews progressed, minor iterative changes were made to include participant-suggested additions, always maintaining alignment with the research objectives. This iterative process was essential to capture the nuanced perspectives of stakeholders and conformed to COREQ standards for qualitative research. A total of 15 stakeholders in VBHC were interviewed. The interviews were transcribed and coded, and 605 codes were created, divided into subthemes and themes. VBHC implementation faces several challenges, requiring a collaborative effort by the stakeholders involved, to achieve a comprehensive vision of value and appropriate multi-stakeholder alignment. The implementation of VBHC can confer a sustainable competitive advantage, and its adoption as a strategic model will be inevitable in the future. 2025-09-02 JMAHP, Vol. 13, Pages 44: Value-Based Healthcare as a Competitive Strategy—A Multi-Stakeholder Perception Analysis in Portuguese Healthcare

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030044

Authors: Filipe Santiago Filipe Costa Eduardo Redondo Cristiano Matos

Designing an accessible, financially viable healthcare system is a key challenge for society. The value-based healthcare (VBHC) strategic model aims to simultaneously improve the quality of healthcare and the efficiency of health systems. The aim of this research was to describe the perceptions of different stakeholders in the Portuguese health industry about the creation of value and the understanding of VBHC as a competitive advantage. A qualitative study was conducted using the inductive method of Braun and Clarke, designed according to the COREQ criteria. Based on the results of the literature review, a semi-structured script for an interview was created, consisting of eight questions. The initial interview script was based on a thorough narrative literature review and tested with two professionals with practical experience in VBHC. The final version of the semi-structured interview guide consisted of eight open-ended questions. The questions were designed to elicit in-depth, reflective responses, and their neutrality was reviewed to avoid leading language that might introduce bias. As the interviews progressed, minor iterative changes were made to include participant-suggested additions, always maintaining alignment with the research objectives. This iterative process was essential to capture the nuanced perspectives of stakeholders and conformed to COREQ standards for qualitative research. A total of 15 stakeholders in VBHC were interviewed. The interviews were transcribed and coded, and 605 codes were created, divided into subthemes and themes. VBHC implementation faces several challenges, requiring a collaborative effort by the stakeholders involved, to achieve a comprehensive vision of value and appropriate multi-stakeholder alignment. The implementation of VBHC can confer a sustainable competitive advantage, and its adoption as a strategic model will be inevitable in the future.

]]> Value-Based Healthcare as a Competitive Strategy—A Multi-Stakeholder Perception Analysis in Portuguese Healthcare Filipe Santiago Filipe Costa Eduardo Redondo Cristiano Matos doi: 10.3390/jmahp13030044 Journal of Market Access & Health Policy 2025-09-02 Journal of Market Access & Health Policy 2025-09-02 13 3 Article 44 10.3390/jmahp13030044 https://www.mdpi.com/2001-6689/13/3/44 JMAHP, Vol. 13, Pages 43: Deploying Experienced Utility in Health Economic Evaluation: A Quantitative Study https://www.mdpi.com/2001-6689/13/3/43 Background: Expected utility has been deployed in order to predict health behaviour in health economic evaluation. However, only limited variance in health behaviour is explained by this construct. This limited explained variance is often attributed to the dubious foundational postulates underlying the construct (e.g., absolute rationality, complete information, fixed preferences). Due to these limitations it has been hypothesized that substituting or complementing expected utility with experienced utility may enhance predictions of health behaviour. As this hypothesis has not yet been subjected to empirical scrutiny, this study examines if deployment of experienced utility or expected utility and experienced utility combined enhances predictions of health behaviour relative to expected utility separately. Methods: Online questionnaires were distributed across a panel of Dutch citizens (N = 2550). The questionnaire includes items and scales on sample characteristics, expected utility, experienced utility and health behaviour. Data analysis was conducted by employing descriptive, reliability, validity and model statistics. Results: Experienced utility has a significant direct effect on health behaviour that is stronger than expected utility. Experienced utility also explains more variance in health behaviour than expected utility. Expected utility and experienced utility combined have a significant direct and indirect effect on health behaviour that is stronger than each type of utility separately. Expected utility and experienced utility combined also explain more variance in health behaviour than each type of utility separately. Conclusions: Deploying experienced utility separately or in combination with expected utility in health economic evaluation seems pertinent as it has considerable impact on health behaviour and may provide health economists with an even sturdier foundation for conducting health economic evaluation. 2025-08-28 JMAHP, Vol. 13, Pages 43: Deploying Experienced Utility in Health Economic Evaluation: A Quantitative Study

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030043

Authors: Damien S. E. Broekharst Sjaak Bloem Robert J. Blomme Edward A. G. Groenland Patrick P. T. Jeurissen Michel van Agthoven

Background: Expected utility has been deployed in order to predict health behaviour in health economic evaluation. However, only limited variance in health behaviour is explained by this construct. This limited explained variance is often attributed to the dubious foundational postulates underlying the construct (e.g., absolute rationality, complete information, fixed preferences). Due to these limitations it has been hypothesized that substituting or complementing expected utility with experienced utility may enhance predictions of health behaviour. As this hypothesis has not yet been subjected to empirical scrutiny, this study examines if deployment of experienced utility or expected utility and experienced utility combined enhances predictions of health behaviour relative to expected utility separately. Methods: Online questionnaires were distributed across a panel of Dutch citizens (N = 2550). The questionnaire includes items and scales on sample characteristics, expected utility, experienced utility and health behaviour. Data analysis was conducted by employing descriptive, reliability, validity and model statistics. Results: Experienced utility has a significant direct effect on health behaviour that is stronger than expected utility. Experienced utility also explains more variance in health behaviour than expected utility. Expected utility and experienced utility combined have a significant direct and indirect effect on health behaviour that is stronger than each type of utility separately. Expected utility and experienced utility combined also explain more variance in health behaviour than each type of utility separately. Conclusions: Deploying experienced utility separately or in combination with expected utility in health economic evaluation seems pertinent as it has considerable impact on health behaviour and may provide health economists with an even sturdier foundation for conducting health economic evaluation.

]]> Deploying Experienced Utility in Health Economic Evaluation: A Quantitative Study Damien S. E. Broekharst Sjaak Bloem Robert J. Blomme Edward A. G. Groenland Patrick P. T. Jeurissen Michel van Agthoven doi: 10.3390/jmahp13030043 Journal of Market Access & Health Policy 2025-08-28 Journal of Market Access & Health Policy 2025-08-28 13 3 Article 43 10.3390/jmahp13030043 https://www.mdpi.com/2001-6689/13/3/43 JMAHP, Vol. 13, Pages 42: European Health Technology Assessment Considerations Related to Gene Therapies in Eyecare: The Neovascular Age-Related Macular Degeneration Example https://www.mdpi.com/2001-6689/13/3/42 Gene therapies that induce the body to produce therapeutic anti-vascular endothelial growth factor (anti-VEGF) proteins are an emerging topic related to neovascular age-related macular degeneration (nAMD). Continuous delivery of anti-VEGF protein directly to the target tissue offers the possibility of lifelong efficacy without the need for repeated and frequent eye injections. This novel approach could revolutionize patient management through optimizing clinical outcomes while simplifying service delivery. However, such gene therapies are anticipated to face unique challenges related to patients’ access and health technology assessment (HTA), and their integration into real-world eyecare practices. This article presents key elements raised at the European Access Academy (EAA) Fall convention (held in Rome in October 2024) regarding anticipated HTA challenges for gene therapies in nAMD. The important role of HTA and policymakers in ensuring that emerging gene therapies are accessible to all eligible patients is also highlighted. This article mainly focuses on the need for a fit-for-purpose EU HTA framework to address the widely varying utilization of standard of care in nAMD clinical practice, and to incorporate considerations about the long-term durability of gene therapies in nAMD. The importance of integrating real-world evidence (RWE) into the EU HTA framework is also discussed. 2025-08-27 JMAHP, Vol. 13, Pages 42: European Health Technology Assessment Considerations Related to Gene Therapies in Eyecare: The Neovascular Age-Related Macular Degeneration Example

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030042

Authors: Kevin Douglas Gianni Pardhanani Laetitia Mariani Maria Chaita

Gene therapies that induce the body to produce therapeutic anti-vascular endothelial growth factor (anti-VEGF) proteins are an emerging topic related to neovascular age-related macular degeneration (nAMD). Continuous delivery of anti-VEGF protein directly to the target tissue offers the possibility of lifelong efficacy without the need for repeated and frequent eye injections. This novel approach could revolutionize patient management through optimizing clinical outcomes while simplifying service delivery. However, such gene therapies are anticipated to face unique challenges related to patients’ access and health technology assessment (HTA), and their integration into real-world eyecare practices. This article presents key elements raised at the European Access Academy (EAA) Fall convention (held in Rome in October 2024) regarding anticipated HTA challenges for gene therapies in nAMD. The important role of HTA and policymakers in ensuring that emerging gene therapies are accessible to all eligible patients is also highlighted. This article mainly focuses on the need for a fit-for-purpose EU HTA framework to address the widely varying utilization of standard of care in nAMD clinical practice, and to incorporate considerations about the long-term durability of gene therapies in nAMD. The importance of integrating real-world evidence (RWE) into the EU HTA framework is also discussed.

]]> European Health Technology Assessment Considerations Related to Gene Therapies in Eyecare: The Neovascular Age-Related Macular Degeneration Example Kevin Douglas Gianni Pardhanani Laetitia Mariani Maria Chaita doi: 10.3390/jmahp13030042 Journal of Market Access & Health Policy 2025-08-27 Journal of Market Access & Health Policy 2025-08-27 13 3 Opinion 42 10.3390/jmahp13030042 https://www.mdpi.com/2001-6689/13/3/42 JMAHP, Vol. 13, Pages 41: Development of a Work-Related Quality of Life Questionnaire for Medical Doctors (WQMD-9) in Japan: Questionnaire Design and Quantitative Survey https://www.mdpi.com/2001-6689/13/3/41 Background: With the ongoing development of game-changing technologies, assessing healthcare provider burden is desirable. This requires developing and evaluating subjective outcome measures, but there is no single scale that measures this burden. We developed a measure of quality of life (QOL) to address this, focusing on medical doctors (MDs). Methods: Based on Japan’s national statistical distribution of MDs in Japan, we qualitatively interviewed twenty MDs to identify factors that influenced their QOL and another eight MDs to verify the appropriateness and interpretability of the questions. Validity and reliability were evaluated and verified in a quantitative survey of 374 MDs to finalize the questionnaire. Results: Based on our initial research and interviews, we derived nine dimensions and developed the work-related QOL questionnaire for MDs (WQMD-9) accordingly. Correlation coefficients between questionnaire items were 0.3–0.7 and Cronbach’s α was 0.897, confirming the validity and reliability of the questionnaire. Conclusions: The WQMD-9 is an original profile-type scale with nine dimensions and five levels. We expect that as new technologies develop, evaluations of the associated medical treatment will involve measuring the QOL of not only patients but also MDs, and the WQMD-9 will facilitate this process. 2025-08-19 JMAHP, Vol. 13, Pages 41: Development of a Work-Related Quality of Life Questionnaire for Medical Doctors (WQMD-9) in Japan: Questionnaire Design and Quantitative Survey

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030041

Authors: Miyuki Ezura Katsuhiko Sawada Yusuke Takushima Lida Teng Ataru Igarashi

Background: With the ongoing development of game-changing technologies, assessing healthcare provider burden is desirable. This requires developing and evaluating subjective outcome measures, but there is no single scale that measures this burden. We developed a measure of quality of life (QOL) to address this, focusing on medical doctors (MDs). Methods: Based on Japan’s national statistical distribution of MDs in Japan, we qualitatively interviewed twenty MDs to identify factors that influenced their QOL and another eight MDs to verify the appropriateness and interpretability of the questions. Validity and reliability were evaluated and verified in a quantitative survey of 374 MDs to finalize the questionnaire. Results: Based on our initial research and interviews, we derived nine dimensions and developed the work-related QOL questionnaire for MDs (WQMD-9) accordingly. Correlation coefficients between questionnaire items were 0.3–0.7 and Cronbach’s α was 0.897, confirming the validity and reliability of the questionnaire. Conclusions: The WQMD-9 is an original profile-type scale with nine dimensions and five levels. We expect that as new technologies develop, evaluations of the associated medical treatment will involve measuring the QOL of not only patients but also MDs, and the WQMD-9 will facilitate this process.

]]> Development of a Work-Related Quality of Life Questionnaire for Medical Doctors (WQMD-9) in Japan: Questionnaire Design and Quantitative Survey Miyuki Ezura Katsuhiko Sawada Yusuke Takushima Lida Teng Ataru Igarashi doi: 10.3390/jmahp13030041 Journal of Market Access & Health Policy 2025-08-19 Journal of Market Access & Health Policy 2025-08-19 13 3 Article 41 10.3390/jmahp13030041 https://www.mdpi.com/2001-6689/13/3/41 JMAHP, Vol. 13, Pages 40: Managed Entry Agreements for Pharmaceutical Products in Three Maghreb Countries: Payer and Supplier Perspectives https://www.mdpi.com/2001-6689/13/3/40 Our objective is to describe the experience and challenges of using Managed Entry Agreements (MEAs) in Algeria, Morocco, and Tunisia. We conducted online interviews with key decision-makers in Algeria, Morocco, and Tunisia between March 2021 and December 2023. The questionnaire captured experience with MEAs, types of agreements implemented, and challenges to implementing MEAs. Three, five, and seven participants working, respectively, in the Algerian, Moroccan, and Tunisian pharmaceutical sectors, participated in the interviews. Participants were from the public (8/15) and the private sector (7/15). Only Tunisian respondents reported having dealt with MEAs contracts, such as financial-based agreements (FBAs) related to standard discounts and volume-based price discounts. All respondents were aware of the potential need for structuring contracts differently for expensive medicines. Hurdles in implementing MEAs noted by respondents were mainly related to the absence of a legal framework and the lack of budget allocated for new medicines. Most respondents projected an increase in the use of MEAs to improve reimbursement and access to new, highly priced medicines. Recommendations include strengthening pricing, reimbursement processes, and HTA use. The adoption of FBAs is suggested as a practical initial approach. 2025-08-11 JMAHP, Vol. 13, Pages 40: Managed Entry Agreements for Pharmaceutical Products in Three Maghreb Countries: Payer and Supplier Perspectives

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030040

Authors: Hajer Dahmani Leila Achour Maghreb Research Group Maghreb Research Group Mondher Toumi Ines Fradi

Our objective is to describe the experience and challenges of using Managed Entry Agreements (MEAs) in Algeria, Morocco, and Tunisia. We conducted online interviews with key decision-makers in Algeria, Morocco, and Tunisia between March 2021 and December 2023. The questionnaire captured experience with MEAs, types of agreements implemented, and challenges to implementing MEAs. Three, five, and seven participants working, respectively, in the Algerian, Moroccan, and Tunisian pharmaceutical sectors, participated in the interviews. Participants were from the public (8/15) and the private sector (7/15). Only Tunisian respondents reported having dealt with MEAs contracts, such as financial-based agreements (FBAs) related to standard discounts and volume-based price discounts. All respondents were aware of the potential need for structuring contracts differently for expensive medicines. Hurdles in implementing MEAs noted by respondents were mainly related to the absence of a legal framework and the lack of budget allocated for new medicines. Most respondents projected an increase in the use of MEAs to improve reimbursement and access to new, highly priced medicines. Recommendations include strengthening pricing, reimbursement processes, and HTA use. The adoption of FBAs is suggested as a practical initial approach.

]]> Managed Entry Agreements for Pharmaceutical Products in Three Maghreb Countries: Payer and Supplier Perspectives Hajer Dahmani Leila Achour Maghreb Research Group Maghreb Research Group Mondher Toumi Ines Fradi doi: 10.3390/jmahp13030040 Journal of Market Access & Health Policy 2025-08-11 Journal of Market Access & Health Policy 2025-08-11 13 3 Article 40 10.3390/jmahp13030040 https://www.mdpi.com/2001-6689/13/3/40 JMAHP, Vol. 13, Pages 39: Innovative Payment Mechanisms for High-Cost Medical Devices in Latin America: Experience in Designing Outcome Protection Programs in the Region https://www.mdpi.com/2001-6689/13/3/39 Introduction and Objectives: Risk-sharing agreements (RSAs) have emerged as a key strategy for financing high-cost medical technologies while ensuring financial sustainability. These payment mechanisms mitigate clinical and financial uncertainties, optimizing pricing and reimbursement decisions. Despite their widespread adoption globally, Latin America has reported limited implementation, particularly for high-cost medical devices. This study aims to share insights from designing RSAs in the form of Outcome Protection Programs (OPPs) for medical devices in Latin America from the perspective of a medical devices company. Methods: The report follows a structured approach, defining key OPP dimensions: payment base, access criteria, pricing schemes, risk assessment, and performance incentives. Risks were categorized as financial, clinical, and operational. The framework applied principles from prior models, emphasizing negotiation, program design, implementation, and evaluation. A multidisciplinary task force analyzed patient needs, provider motivations, and payer constraints to ensure alignment with health system priorities. Results: Over two semesters, a panel of seven experts from the manufacturer designed n = 105 innovative payment programs implemented in Argentina (n = 7), Brazil (n = 7), Colombia (n = 75), Mexico (n = 9), Panama (n = 4), and Puerto Rico (n = 3). The programs targeted eight high-burden conditions, including Coronary Artery Disease, atrial fibrillation, Heart Failure, and post-implantation arrhythmias, among others. Private providers accounted for 80% of experiences. Challenges include clinical inertia and operational complexities, necessitating structured training and monitoring mechanisms. Conclusions: Outcome Protection Programs offer a viable and practical risk-sharing approach to financing high-cost medical devices in Latin America. Their implementation requires careful stakeholder alignment, clear eligibility criteria and endpoints, and robust monitoring frameworks. These findings contribute to the ongoing dialogue on sustainable healthcare financing, emphasizing the need for tailored approaches in resource-constrained settings. 2025-08-04 JMAHP, Vol. 13, Pages 39: Innovative Payment Mechanisms for High-Cost Medical Devices in Latin America: Experience in Designing Outcome Protection Programs in the Region

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030039

Authors: Daniela Paredes-Fernández Juan Valencia-Zapata

Introduction and Objectives: Risk-sharing agreements (RSAs) have emerged as a key strategy for financing high-cost medical technologies while ensuring financial sustainability. These payment mechanisms mitigate clinical and financial uncertainties, optimizing pricing and reimbursement decisions. Despite their widespread adoption globally, Latin America has reported limited implementation, particularly for high-cost medical devices. This study aims to share insights from designing RSAs in the form of Outcome Protection Programs (OPPs) for medical devices in Latin America from the perspective of a medical devices company. Methods: The report follows a structured approach, defining key OPP dimensions: payment base, access criteria, pricing schemes, risk assessment, and performance incentives. Risks were categorized as financial, clinical, and operational. The framework applied principles from prior models, emphasizing negotiation, program design, implementation, and evaluation. A multidisciplinary task force analyzed patient needs, provider motivations, and payer constraints to ensure alignment with health system priorities. Results: Over two semesters, a panel of seven experts from the manufacturer designed n = 105 innovative payment programs implemented in Argentina (n = 7), Brazil (n = 7), Colombia (n = 75), Mexico (n = 9), Panama (n = 4), and Puerto Rico (n = 3). The programs targeted eight high-burden conditions, including Coronary Artery Disease, atrial fibrillation, Heart Failure, and post-implantation arrhythmias, among others. Private providers accounted for 80% of experiences. Challenges include clinical inertia and operational complexities, necessitating structured training and monitoring mechanisms. Conclusions: Outcome Protection Programs offer a viable and practical risk-sharing approach to financing high-cost medical devices in Latin America. Their implementation requires careful stakeholder alignment, clear eligibility criteria and endpoints, and robust monitoring frameworks. These findings contribute to the ongoing dialogue on sustainable healthcare financing, emphasizing the need for tailored approaches in resource-constrained settings.

]]> Innovative Payment Mechanisms for High-Cost Medical Devices in Latin America: Experience in Designing Outcome Protection Programs in the Region Daniela Paredes-Fernández Juan Valencia-Zapata doi: 10.3390/jmahp13030039 Journal of Market Access & Health Policy 2025-08-04 Journal of Market Access & Health Policy 2025-08-04 13 3 Article 39 10.3390/jmahp13030039 https://www.mdpi.com/2001-6689/13/3/39 JMAHP, Vol. 13, Pages 38: Patient Involvement in Health Technology Assessments: Lessons for EU Joint Clinical Assessments https://www.mdpi.com/2001-6689/13/3/38 Patient involvement in health technology assessment (HTA) processes is increasingly recognized as pivotal for informed, equitable, and patient-relevant health care decision-making. With the implementation of Joint Scientific Consultations (JSCs) and Joint Clinical Assessments (JCAs) under Regulation (EU) 2021/2282, the European Union has a unique opportunity to design harmonized mechanisms that reflect best practices from established HTA systems. This article, drawing on the Acute Leukemia Advocates Network (ALAN)’s comparative analysis of HTA practices across seven countries (Canada, England, Scotland, France, Germany, Spain, and Italy), examines how current patient involvement processes can inform the JCA framework. It identifies opportunities to replicate effective practices and proposes strategies to embed patient voices meaningfully into the JCA process. By prioritizing robust and inclusive patient involvement, the EU can establish a global benchmark for impactful and consistent HTA processes. By leveraging lessons from international HTA systems and prioritizing clear frameworks, early involvement, and capacity building, the EU can set a global standard for meaningful patient participation in HTA processes. ALAN is an independent global network of patient organizations dedicated to improving outcomes for patients with acute leukemia. 2025-07-28 JMAHP, Vol. 13, Pages 38: Patient Involvement in Health Technology Assessments: Lessons for EU Joint Clinical Assessments

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030038

Authors: Anne-Pierre Pickaert

Patient involvement in health technology assessment (HTA) processes is increasingly recognized as pivotal for informed, equitable, and patient-relevant health care decision-making. With the implementation of Joint Scientific Consultations (JSCs) and Joint Clinical Assessments (JCAs) under Regulation (EU) 2021/2282, the European Union has a unique opportunity to design harmonized mechanisms that reflect best practices from established HTA systems. This article, drawing on the Acute Leukemia Advocates Network (ALAN)’s comparative analysis of HTA practices across seven countries (Canada, England, Scotland, France, Germany, Spain, and Italy), examines how current patient involvement processes can inform the JCA framework. It identifies opportunities to replicate effective practices and proposes strategies to embed patient voices meaningfully into the JCA process. By prioritizing robust and inclusive patient involvement, the EU can establish a global benchmark for impactful and consistent HTA processes. By leveraging lessons from international HTA systems and prioritizing clear frameworks, early involvement, and capacity building, the EU can set a global standard for meaningful patient participation in HTA processes. ALAN is an independent global network of patient organizations dedicated to improving outcomes for patients with acute leukemia.

]]> Patient Involvement in Health Technology Assessments: Lessons for EU Joint Clinical Assessments Anne-Pierre Pickaert doi: 10.3390/jmahp13030038 Journal of Market Access & Health Policy 2025-07-28 Journal of Market Access & Health Policy 2025-07-28 13 3 Review 38 10.3390/jmahp13030038 https://www.mdpi.com/2001-6689/13/3/38 JMAHP, Vol. 13, Pages 37: Pivotal Studies for Drugs About to Be Launched for Rare Diseases: Will They Better Support Health Technology Assessment and Market Access than in the Past? https://www.mdpi.com/2001-6689/13/3/37 The designs of clinical trials of drugs for rare diseases are challenged by health technology assessment organisations and payers. Phase II pivotal studies, single-arm or open-label designs, the extensive use of non-final endpoints, and the limited use of patient-reported outcomes (PROs) are the main points of contention. The evidence on the actual design of these trials is limited, but corroborates the concerns of the above. Our aim is to scrutinise whether the design of pivotal studies of drugs for rare diseases to be launched into the Italian market by 2026 present similar issues. The drugs and the relevant pivotal studies were retrieved from Biomedtracker and US and European clinical trial databases. We identified 154 new drugs for rare diseases. Single-arm designs account for 36% of trials. Almost 50% of randomised control trials (RCTs) are designed using an active comparator and 61% are double-blinded. Primary endpoints are mostly (82%) surrogate. A total of 59% of studies include PROs. Our findings were partially expected (e.g., extensive use of surrogate endpoints) and partially not (e.g., RCTs and an active comparator), considering previous studies on the same topic. Having more head-to-head studies may reduce uncertainty concerning evidence at market launch, but different issues persist, including the still limited role of PROs. 2025-07-25 JMAHP, Vol. 13, Pages 37: Pivotal Studies for Drugs About to Be Launched for Rare Diseases: Will They Better Support Health Technology Assessment and Market Access than in the Past?

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030037

Authors: Claudio Jommi Marzia Bonfanti Melissa Guardigni Andrea Aiello Andrea Marcellusi Pier Luigi Canonico Fulvio Luccini Chiara Lucchetti

The designs of clinical trials of drugs for rare diseases are challenged by health technology assessment organisations and payers. Phase II pivotal studies, single-arm or open-label designs, the extensive use of non-final endpoints, and the limited use of patient-reported outcomes (PROs) are the main points of contention. The evidence on the actual design of these trials is limited, but corroborates the concerns of the above. Our aim is to scrutinise whether the design of pivotal studies of drugs for rare diseases to be launched into the Italian market by 2026 present similar issues. The drugs and the relevant pivotal studies were retrieved from Biomedtracker and US and European clinical trial databases. We identified 154 new drugs for rare diseases. Single-arm designs account for 36% of trials. Almost 50% of randomised control trials (RCTs) are designed using an active comparator and 61% are double-blinded. Primary endpoints are mostly (82%) surrogate. A total of 59% of studies include PROs. Our findings were partially expected (e.g., extensive use of surrogate endpoints) and partially not (e.g., RCTs and an active comparator), considering previous studies on the same topic. Having more head-to-head studies may reduce uncertainty concerning evidence at market launch, but different issues persist, including the still limited role of PROs.

]]> Pivotal Studies for Drugs About to Be Launched for Rare Diseases: Will They Better Support Health Technology Assessment and Market Access than in the Past? Claudio Jommi Marzia Bonfanti Melissa Guardigni Andrea Aiello Andrea Marcellusi Pier Luigi Canonico Fulvio Luccini Chiara Lucchetti doi: 10.3390/jmahp13030037 Journal of Market Access & Health Policy 2025-07-25 Journal of Market Access & Health Policy 2025-07-25 13 3 Article 37 10.3390/jmahp13030037 https://www.mdpi.com/2001-6689/13/3/37 JMAHP, Vol. 13, Pages 36: Physician Practice Affiliation Drives Site of Care Cost Differentials: An Opportunity to Reduce Healthcare Expenditures https://www.mdpi.com/2001-6689/13/3/36 The continued migration of physicians from independent practice to affiliation with larger entities has garnered significant scrutiny. These affiliation models include hospitals and health systems, payers and corporate entities, and management services organizations, which may or may not be private equity (PE)-backed. Data on the impact of different physician affiliation models on cost of care is limited. We examined the relationship between provider affiliation model, site of care (SOC), and cost of care for certain high-volume procedures in procedure-intensive specialties for both Medicare and commercial insurance. We found that hospital-affiliated physicians are least likely—and PE-affiliated physicians are most likely—to provide care in lower-cost settings. For both Medicare and commercial insurance, SOC contributes meaningfully to procedure unit price, which is consistently greater in hospital-based settings. These findings suggest that the physician affiliation model and associated SOC cost differentials contribute materially to healthcare expenditures. As the Medicare cost differentials are set by statute and regulations, strategies such as site-neutral payments are needed to mitigate the monetary impact of historical and future physician practice migration. 2025-07-24 JMAHP, Vol. 13, Pages 36: Physician Practice Affiliation Drives Site of Care Cost Differentials: An Opportunity to Reduce Healthcare Expenditures

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030036

Authors: Deepak A. Kapoor Mark Camel David Eagle Lauren C. Makhoul Justin Maroney Zhou Yang Paul Berggreen

The continued migration of physicians from independent practice to affiliation with larger entities has garnered significant scrutiny. These affiliation models include hospitals and health systems, payers and corporate entities, and management services organizations, which may or may not be private equity (PE)-backed. Data on the impact of different physician affiliation models on cost of care is limited. We examined the relationship between provider affiliation model, site of care (SOC), and cost of care for certain high-volume procedures in procedure-intensive specialties for both Medicare and commercial insurance. We found that hospital-affiliated physicians are least likely—and PE-affiliated physicians are most likely—to provide care in lower-cost settings. For both Medicare and commercial insurance, SOC contributes meaningfully to procedure unit price, which is consistently greater in hospital-based settings. These findings suggest that the physician affiliation model and associated SOC cost differentials contribute materially to healthcare expenditures. As the Medicare cost differentials are set by statute and regulations, strategies such as site-neutral payments are needed to mitigate the monetary impact of historical and future physician practice migration.

]]> Physician Practice Affiliation Drives Site of Care Cost Differentials: An Opportunity to Reduce Healthcare Expenditures Deepak A. Kapoor Mark Camel David Eagle Lauren C. Makhoul Justin Maroney Zhou Yang Paul Berggreen doi: 10.3390/jmahp13030036 Journal of Market Access & Health Policy 2025-07-24 Journal of Market Access & Health Policy 2025-07-24 13 3 Article 36 10.3390/jmahp13030036 https://www.mdpi.com/2001-6689/13/3/36 JMAHP, Vol. 13, Pages 35: Preparing for the EU HTA Regulation: Insights from the Dutch Perspective https://www.mdpi.com/2001-6689/13/3/35 The European Health Technology Assessment (HTA) regulation (HTAR) came into effect in January 2025 and impacts the HTA process in all European Member States. Member States must give due consideration to the joint clinical assessment (JCA) report. This may require adaptations at the national level. This paper describes the anticipated changes to the Dutch national HTA process and how the Dutch National Health Care Institute (Zorginstituut Nederland, ZIN) prepared for this, because sharing experience between Member States can be of general interest for future expansion of the EU HTAR. ZIN’s implementation activities were facilitated by a project-governance structure and by a continuous gap analysis of the current national assessment and appraisal process of medicinal products, resulting in a concrete action plan. The implementation of the HTAR has two major implications for ZIN’s HTA process, namely that the scoping phase starts much earlier and that the JCA report is the starting point for the national assessment. Gaps, challenges and issues were identified in the categories: information and knowledge, IT and template, communication and stakeholder engagement, capacity and resources, and financial aspects. Based on a thorough and well-defined implementation plan, ZIN is ready to implement the HTAR in national HTA processes and to take on (co-)assessor roles for JCA of medicinal products in 2025. 2025-07-24 JMAHP, Vol. 13, Pages 35: Preparing for the EU HTA Regulation: Insights from the Dutch Perspective

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030035

Authors: Anne Willemsen Maureen Rutten-van Mölken Riam al Dulaimi Hedi Schelleman Wim Goettsch Lonneke Timmers

The European Health Technology Assessment (HTA) regulation (HTAR) came into effect in January 2025 and impacts the HTA process in all European Member States. Member States must give due consideration to the joint clinical assessment (JCA) report. This may require adaptations at the national level. This paper describes the anticipated changes to the Dutch national HTA process and how the Dutch National Health Care Institute (Zorginstituut Nederland, ZIN) prepared for this, because sharing experience between Member States can be of general interest for future expansion of the EU HTAR. ZIN’s implementation activities were facilitated by a project-governance structure and by a continuous gap analysis of the current national assessment and appraisal process of medicinal products, resulting in a concrete action plan. The implementation of the HTAR has two major implications for ZIN’s HTA process, namely that the scoping phase starts much earlier and that the JCA report is the starting point for the national assessment. Gaps, challenges and issues were identified in the categories: information and knowledge, IT and template, communication and stakeholder engagement, capacity and resources, and financial aspects. Based on a thorough and well-defined implementation plan, ZIN is ready to implement the HTAR in national HTA processes and to take on (co-)assessor roles for JCA of medicinal products in 2025.

]]> Preparing for the EU HTA Regulation: Insights from the Dutch Perspective Anne Willemsen Maureen Rutten-van Mölken Riam al Dulaimi Hedi Schelleman Wim Goettsch Lonneke Timmers doi: 10.3390/jmahp13030035 Journal of Market Access & Health Policy 2025-07-24 Journal of Market Access & Health Policy 2025-07-24 13 3 Article 35 10.3390/jmahp13030035 https://www.mdpi.com/2001-6689/13/3/35 JMAHP, Vol. 13, Pages 34: An Estimation of the Economic and Environmental Impact of Inhaler Devices Switch for Non-Clinical Reasons in COPD and Asthma: The Case for Spain https://www.mdpi.com/2001-6689/13/3/34 In respiratory patients, limited adherence to and misuse of devices hinder the effectiveness of inhalation therapy. Switching inhalers for non-clinical reasons poses a risk of deterioration of respiratory disease and/or promotes poor adherence to therapy. The objective of this work was to explore the impact of device changes for non-clinical reasons on clinical outcomes (primary) and costs (secondary), including carbon emissions in Spain. After a comprehensive literature search, the increased use of resources following worsening outcomes was apportioned using Spanish cost data and following the recommended pathways for care. We calculated the cost of re-training these patients and attributed carbon emissions in metric tons of CO2 equivalent (tCO2eq) to the excess resource use. In Spain, the impact of uncontrolled switching for non-clinical reasons in COPD has an annual estimated cost of EUR 923/patient, leading to an excess annual expenditure of more than EUR 216 million. For asthma patients, the annual impact is almost EUR 263/patient, representing an additional EUR 118 million excess annual expenditure. The environmental consequence of both conditions can be equated to almost 45 thousand tCO2eq. Training all these patients on the new device would cost around EUR 35 million and would generate an extra impact reduction of about 2.6 thousand tCO2eq in carbon emissions levy. 2025-07-17 JMAHP, Vol. 13, Pages 34: An Estimation of the Economic and Environmental Impact of Inhaler Devices Switch for Non-Clinical Reasons in COPD and Asthma: The Case for Spain

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030034

Authors: Oriol Solà-Morales Joan B Soriano Míriam Solozabal-Coll Jose Vicente Galindo

In respiratory patients, limited adherence to and misuse of devices hinder the effectiveness of inhalation therapy. Switching inhalers for non-clinical reasons poses a risk of deterioration of respiratory disease and/or promotes poor adherence to therapy. The objective of this work was to explore the impact of device changes for non-clinical reasons on clinical outcomes (primary) and costs (secondary), including carbon emissions in Spain. After a comprehensive literature search, the increased use of resources following worsening outcomes was apportioned using Spanish cost data and following the recommended pathways for care. We calculated the cost of re-training these patients and attributed carbon emissions in metric tons of CO2 equivalent (tCO2eq) to the excess resource use. In Spain, the impact of uncontrolled switching for non-clinical reasons in COPD has an annual estimated cost of EUR 923/patient, leading to an excess annual expenditure of more than EUR 216 million. For asthma patients, the annual impact is almost EUR 263/patient, representing an additional EUR 118 million excess annual expenditure. The environmental consequence of both conditions can be equated to almost 45 thousand tCO2eq. Training all these patients on the new device would cost around EUR 35 million and would generate an extra impact reduction of about 2.6 thousand tCO2eq in carbon emissions levy.

]]> An Estimation of the Economic and Environmental Impact of Inhaler Devices Switch for Non-Clinical Reasons in COPD and Asthma: The Case for Spain Oriol Solà-Morales Joan B Soriano Míriam Solozabal-Coll Jose Vicente Galindo doi: 10.3390/jmahp13030034 Journal of Market Access & Health Policy 2025-07-17 Journal of Market Access & Health Policy 2025-07-17 13 3 Article 34 10.3390/jmahp13030034 https://www.mdpi.com/2001-6689/13/3/34 JMAHP, Vol. 13, Pages 33: EU-HTA Guidance for Clinical Validity: Misconceptions and Flawed Processes https://www.mdpi.com/2001-6689/13/3/33 This review of the scope of the European Health Technology Assessment (EU HTA)’s guidance on clinical trial validity in its randomized controlled trials (RCTs) highlights several key issues that undermine its practical application and effectiveness, including misconceptions, errors, and inconsistencies [...] 2025-07-15 JMAHP, Vol. 13, Pages 33: EU-HTA Guidance for Clinical Validity: Misconceptions and Flawed Processes

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030033

Authors: Mondher Toumi Bruno Falissard Asma Jouini Samuel Aballéa Laurent Boyer Pascal Auquier

This review of the scope of the European Health Technology Assessment (EU HTA)’s guidance on clinical trial validity in its randomized controlled trials (RCTs) highlights several key issues that undermine its practical application and effectiveness, including misconceptions, errors, and inconsistencies [...]

]]> EU-HTA Guidance for Clinical Validity: Misconceptions and Flawed Processes Mondher Toumi Bruno Falissard Asma Jouini Samuel Aballéa Laurent Boyer Pascal Auquier doi: 10.3390/jmahp13030033 Journal of Market Access & Health Policy 2025-07-15 Journal of Market Access & Health Policy 2025-07-15 13 3 Editorial 33 10.3390/jmahp13030033 https://www.mdpi.com/2001-6689/13/3/33 JMAHP, Vol. 13, Pages 32: The PICO Puzzle: Can Public Data Predict EU HTA Expectations for All EU Countries? https://www.mdpi.com/2001-6689/13/3/32 With the European Union (EU) Health Technology Assessment (HTA) regulation, Joint Clinical Assessments (JCA) are now required for oncological and advanced therapy medicinal products. The JCA assessment scope is determined through the PICO framework (Population, Intervention, Comparator, Outcome). Given the tight JCA timelines, Health Technology Developers (HTD) must anticipate PICO elements early to prepare dossiers effectively. This study investigates whether PICO can be predicted across EU member states using publicly available information. A systematic literature review was conducted to identify relevant peer-reviewed articles. Additionally, an extensive search of publicly available HTA documents, including reports, methodological guidelines, submission templates, and market access information was performed across 29 European countries. Relevant information for PICO anticipation was extracted. For many member states, a wealth of relevant information is publicly accessible: 66% have HTA reports publicly available, 79% have HTA methodological guidelines, 69% have dossier templates, and 100% have market access status lists. Between countries, the requirements for population and outcomes are largely aligned, making comparator the central element in PICO anticipation. PICO can be anticipated reliably based on public information. HTDs must be prepared to adjust their strategies as national procedures adapt, ensuring alignment with both current and emerging EU and national requirements. 2025-06-26 JMAHP, Vol. 13, Pages 32: The PICO Puzzle: Can Public Data Predict EU HTA Expectations for All EU Countries?

Journal of Market Access & Health Policy doi: 10.3390/jmahp13030032

Authors: Karolin Eberle Lisa-Maria Hagemann Maria Katharina Schweitzer Martin Justl Jana Maurer Alexandra Carls Eva-Maria Reuter

With the European Union (EU) Health Technology Assessment (HTA) regulation, Joint Clinical Assessments (JCA) are now required for oncological and advanced therapy medicinal products. The JCA assessment scope is determined through the PICO framework (Population, Intervention, Comparator, Outcome). Given the tight JCA timelines, Health Technology Developers (HTD) must anticipate PICO elements early to prepare dossiers effectively. This study investigates whether PICO can be predicted across EU member states using publicly available information. A systematic literature review was conducted to identify relevant peer-reviewed articles. Additionally, an extensive search of publicly available HTA documents, including reports, methodological guidelines, submission templates, and market access information was performed across 29 European countries. Relevant information for PICO anticipation was extracted. For many member states, a wealth of relevant information is publicly accessible: 66% have HTA reports publicly available, 79% have HTA methodological guidelines, 69% have dossier templates, and 100% have market access status lists. Between countries, the requirements for population and outcomes are largely aligned, making comparator the central element in PICO anticipation. PICO can be anticipated reliably based on public information. HTDs must be prepared to adjust their strategies as national procedures adapt, ensuring alignment with both current and emerging EU and national requirements.

]]> The PICO Puzzle: Can Public Data Predict EU HTA Expectations for All EU Countries? Karolin Eberle Lisa-Maria Hagemann Maria Katharina Schweitzer Martin Justl Jana Maurer Alexandra Carls Eva-Maria Reuter doi: 10.3390/jmahp13030032 Journal of Market Access & Health Policy 2025-06-26 Journal of Market Access & Health Policy 2025-06-26 13 3 Article 32 10.3390/jmahp13030032 https://www.mdpi.com/2001-6689/13/3/32 JMAHP, Vol. 13, Pages 31: Opportunities for and Challenges of Conducting Indirect Treatment Comparisons and Meta-Analyses for Vaccines in Post-EU HTA Regulation Era https://www.mdpi.com/2001-6689/13/2/31 The dynamic nature of infectious diseases introduces inherent challenges to the design of vaccine clinical trials, which consequently makes vaccine indirect treatment comparisons (ITCs) and meta-analyses (MAs) more challenging compared with regular pharmaceuticals. However, comparisons of efficacy and safety between vaccines are being frequently required in vaccine decision making due to a low number of head-to-head clinical trials in the vaccine landscape. The introduction of the European Union Health Technology Assessment (HTA) Regulation (EU HTAR) aims to harmonize HTA efforts across Europe. However, the EU HTAR could also escalate existing challenges for conducting vaccine MAs and ITCs. Such challenges include generating efficacy evidence in time for Joint Clinical Assessment (JCA), incorporating high levels of heterogeneity due to infectious disease-specific characteristics, and tackling a high number of PICOs per submission—likely driven by heterogeneity in the available data and differences in national vaccine calendars. Opportunities to tackle these challenges include introducing a stepwise approach to vaccine assessment in JCA, best-practice recommendations for conducting/interpreting vaccine MAs and ITCs, and condensing the number of PICOs to create larger ‘catch-all’ ITC networks. This perspective article explores these challenges and opportunities further. 2025-06-11 JMAHP, Vol. 13, Pages 31: Opportunities for and Challenges of Conducting Indirect Treatment Comparisons and Meta-Analyses for Vaccines in Post-EU HTA Regulation Era

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020031

Authors: Charlotte Graham Erin Barker Joe Moss Emily Gregg Rachael McCool Nathalie Largeron Mélanie Trichard José Bartelt-Hofer Maribel Tribaldos

The dynamic nature of infectious diseases introduces inherent challenges to the design of vaccine clinical trials, which consequently makes vaccine indirect treatment comparisons (ITCs) and meta-analyses (MAs) more challenging compared with regular pharmaceuticals. However, comparisons of efficacy and safety between vaccines are being frequently required in vaccine decision making due to a low number of head-to-head clinical trials in the vaccine landscape. The introduction of the European Union Health Technology Assessment (HTA) Regulation (EU HTAR) aims to harmonize HTA efforts across Europe. However, the EU HTAR could also escalate existing challenges for conducting vaccine MAs and ITCs. Such challenges include generating efficacy evidence in time for Joint Clinical Assessment (JCA), incorporating high levels of heterogeneity due to infectious disease-specific characteristics, and tackling a high number of PICOs per submission—likely driven by heterogeneity in the available data and differences in national vaccine calendars. Opportunities to tackle these challenges include introducing a stepwise approach to vaccine assessment in JCA, best-practice recommendations for conducting/interpreting vaccine MAs and ITCs, and condensing the number of PICOs to create larger ‘catch-all’ ITC networks. This perspective article explores these challenges and opportunities further.

]]> Opportunities for and Challenges of Conducting Indirect Treatment Comparisons and Meta-Analyses for Vaccines in Post-EU HTA Regulation Era Charlotte Graham Erin Barker Joe Moss Emily Gregg Rachael McCool Nathalie Largeron Mélanie Trichard José Bartelt-Hofer Maribel Tribaldos doi: 10.3390/jmahp13020031 Journal of Market Access & Health Policy 2025-06-11 Journal of Market Access & Health Policy 2025-06-11 13 2 Perspective 31 10.3390/jmahp13020031 https://www.mdpi.com/2001-6689/13/2/31 JMAHP, Vol. 13, Pages 30: Health Technology Assessment and Cardiology: A Review of the Present and Future of Innovation https://www.mdpi.com/2001-6689/13/2/30 Background and Objective: Innovation is a key enabler of patient-centered care in cardiology, with new medical devices and digital health technologies offering the potential to improve outcomes and efficiency. However, the evaluation of these innovations poses challenges for clinicians, regulators, and procurement stakeholders, particularly within the complex European healthcare landscape. This review aims to explore the current state of health technology assessment (HTA) for cardiology-related medical devices in Europe, offering a clinical perspective. Material and Methods: Three independent scoping reviews were conducted following the PRISMA-ScR guidelines. Keywords included “innovation”, “health technology assessment”, and “cardiology”. The search was supplemented by the relevant literature on European HTA policies, regulatory directives, and emerging technologies. Results: The review identified three central themes: (1) the evolving role of clinicians in HTA processes, (2) the integration of innovative technologies such as digital tools and artificial intelligence within HTA frameworks, and (3) the considerable variation in HTA practices and policies across EU member states. Conclusions: HTA in Europe is undergoing a transformation, with increasing emphasis on interdisciplinary collaboration and frameworks that support innovation. While the goal of harmonization across the EU remains a work in progress, new regulatory efforts, such as the HTA Regulation (HTAR), offer promising avenues for aligning clinical practice with evidence-based assessment and reimbursement decisions. 2025-06-09 JMAHP, Vol. 13, Pages 30: Health Technology Assessment and Cardiology: A Review of the Present and Future of Innovation

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020030

Authors: Ruben Casado-Arroyo Lucia Osoro

Background and Objective: Innovation is a key enabler of patient-centered care in cardiology, with new medical devices and digital health technologies offering the potential to improve outcomes and efficiency. However, the evaluation of these innovations poses challenges for clinicians, regulators, and procurement stakeholders, particularly within the complex European healthcare landscape. This review aims to explore the current state of health technology assessment (HTA) for cardiology-related medical devices in Europe, offering a clinical perspective. Material and Methods: Three independent scoping reviews were conducted following the PRISMA-ScR guidelines. Keywords included “innovation”, “health technology assessment”, and “cardiology”. The search was supplemented by the relevant literature on European HTA policies, regulatory directives, and emerging technologies. Results: The review identified three central themes: (1) the evolving role of clinicians in HTA processes, (2) the integration of innovative technologies such as digital tools and artificial intelligence within HTA frameworks, and (3) the considerable variation in HTA practices and policies across EU member states. Conclusions: HTA in Europe is undergoing a transformation, with increasing emphasis on interdisciplinary collaboration and frameworks that support innovation. While the goal of harmonization across the EU remains a work in progress, new regulatory efforts, such as the HTA Regulation (HTAR), offer promising avenues for aligning clinical practice with evidence-based assessment and reimbursement decisions.

]]> Health Technology Assessment and Cardiology: A Review of the Present and Future of Innovation Ruben Casado-Arroyo Lucia Osoro doi: 10.3390/jmahp13020030 Journal of Market Access & Health Policy 2025-06-09 Journal of Market Access & Health Policy 2025-06-09 13 2 Review 30 10.3390/jmahp13020030 https://www.mdpi.com/2001-6689/13/2/30 JMAHP, Vol. 13, Pages 29: Exploring Trust in Health Insurers: Insights from Enrollees’ Perceptions and Experiences https://www.mdpi.com/2001-6689/13/2/29 Managed competition is a key driver in healthcare systems in countries like Germany, Switzerland, and The Netherlands. Trust in health insurers is vital but currently low in The Netherlands. This may be due to perceptions regarding profit motives, negative experiences, media coverage, and a lack of understanding of insurers’ roles. This study explores how enrollees perceive health insurers and how the aforementioned factors contribute to these perceptions. Semi-structured interviews were conducted with 17 participants from the Nivel Dutch Health Care Consumer Panel in March and April 2023. Data were analysed using Braun and Clarke’s six-step method for inductive thematic analysis. Participants generally view health insurers positively in terms of managing finances and ensuring care accessibility. However, some perceive insurers as profit-driven and prioritising cost reduction over individual needs, leading to dissatisfaction. Negative experiences and media coverage also shape these perceptions. Participants believe that insurers should ensure care accessibility and quality, distribute costs fairly, provide guidance, and prioritise preventive measures. To foster trust, insurers should communicate their non-profit status and use of benefits, increase transparency in purchasing decisions, and maintain clear communication about payment obligations. Enhancing communication about their contributions to healthcare and raising awareness of their broader roles may also help build trust. 2025-06-09 JMAHP, Vol. 13, Pages 29: Exploring Trust in Health Insurers: Insights from Enrollees’ Perceptions and Experiences

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020029

Authors: Frank J. P. van der Hulst Sanne Huijgen Anne E. M. Brabers Judith D. de Jong

Managed competition is a key driver in healthcare systems in countries like Germany, Switzerland, and The Netherlands. Trust in health insurers is vital but currently low in The Netherlands. This may be due to perceptions regarding profit motives, negative experiences, media coverage, and a lack of understanding of insurers’ roles. This study explores how enrollees perceive health insurers and how the aforementioned factors contribute to these perceptions. Semi-structured interviews were conducted with 17 participants from the Nivel Dutch Health Care Consumer Panel in March and April 2023. Data were analysed using Braun and Clarke’s six-step method for inductive thematic analysis. Participants generally view health insurers positively in terms of managing finances and ensuring care accessibility. However, some perceive insurers as profit-driven and prioritising cost reduction over individual needs, leading to dissatisfaction. Negative experiences and media coverage also shape these perceptions. Participants believe that insurers should ensure care accessibility and quality, distribute costs fairly, provide guidance, and prioritise preventive measures. To foster trust, insurers should communicate their non-profit status and use of benefits, increase transparency in purchasing decisions, and maintain clear communication about payment obligations. Enhancing communication about their contributions to healthcare and raising awareness of their broader roles may also help build trust.

]]> Exploring Trust in Health Insurers: Insights from Enrollees’ Perceptions and Experiences Frank J. P. van der Hulst Sanne Huijgen Anne E. M. Brabers Judith D. de Jong doi: 10.3390/jmahp13020029 Journal of Market Access & Health Policy 2025-06-09 Journal of Market Access & Health Policy 2025-06-09 13 2 Article 29 10.3390/jmahp13020029 https://www.mdpi.com/2001-6689/13/2/29 JMAHP, Vol. 13, Pages 28: Health and Social Care Integration: Insights from International Implementation Cases https://www.mdpi.com/2001-6689/13/2/28 The integration of health and social care is increasingly recognized as essential to address population ageing, the rise in chronic diseases, and persistent health inequities. Across Europe, diverse models have been developed to improve service coordination, resource efficiency, and person-centered care. This paper aims to explore international experiences in integrating health and social care, identify common strategies and challenges, and provide insights to inform policy development in countries where integration remains incipient, with a focus on Portugal. A qualitative comparative approach was employed. A systematic literature review was conducted across PUBMED, MEDLINE, and Google Scholar, including peer-reviewed articles, policy reports, and government documents. Thematic analysis was used to identify integration models, enablers, and barriers across different countries. Different models reveal that joint governance, pooled funding, strong community involvement, and digital innovation are key enablers of integration. However, common challenges persist, including fragmented governance, inconsistent implementation, and financial sustainability. In Portugal, structural separation between the health and social sectors continues to limit strategic alignment. Successful integration depends on political commitment, shared vision, and active stakeholder collaboration. European models offer adaptable lessons for Portugal and similar systems, especially regarding intersectoral coordination and preventive care. Integrating health and social care is vital for building resilient, equitable systems. Portugal must adopt a cohesive national strategy; strengthen local implementation; and embrace person-centered, sustainable solutions to ensure long-term impact. Integrating the health and social sectors is indispensable in navigating the ever-evolving healthcare landscape and promoting holistic well-being. 2025-06-05 JMAHP, Vol. 13, Pages 28: Health and Social Care Integration: Insights from International Implementation Cases

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020028

Authors: Ricardo Correia de Matos Generosa do Nascimento Adalberto Campos Fernandes Cristiano Matos

The integration of health and social care is increasingly recognized as essential to address population ageing, the rise in chronic diseases, and persistent health inequities. Across Europe, diverse models have been developed to improve service coordination, resource efficiency, and person-centered care. This paper aims to explore international experiences in integrating health and social care, identify common strategies and challenges, and provide insights to inform policy development in countries where integration remains incipient, with a focus on Portugal. A qualitative comparative approach was employed. A systematic literature review was conducted across PUBMED, MEDLINE, and Google Scholar, including peer-reviewed articles, policy reports, and government documents. Thematic analysis was used to identify integration models, enablers, and barriers across different countries. Different models reveal that joint governance, pooled funding, strong community involvement, and digital innovation are key enablers of integration. However, common challenges persist, including fragmented governance, inconsistent implementation, and financial sustainability. In Portugal, structural separation between the health and social sectors continues to limit strategic alignment. Successful integration depends on political commitment, shared vision, and active stakeholder collaboration. European models offer adaptable lessons for Portugal and similar systems, especially regarding intersectoral coordination and preventive care. Integrating health and social care is vital for building resilient, equitable systems. Portugal must adopt a cohesive national strategy; strengthen local implementation; and embrace person-centered, sustainable solutions to ensure long-term impact. Integrating the health and social sectors is indispensable in navigating the ever-evolving healthcare landscape and promoting holistic well-being.

]]> Health and Social Care Integration: Insights from International Implementation Cases Ricardo Correia de Matos Generosa do Nascimento Adalberto Campos Fernandes Cristiano Matos doi: 10.3390/jmahp13020028 Journal of Market Access & Health Policy 2025-06-05 Journal of Market Access & Health Policy 2025-06-05 13 2 Perspective 28 10.3390/jmahp13020028 https://www.mdpi.com/2001-6689/13/2/28 JMAHP, Vol. 13, Pages 27: Enhancing Patient Engagement in HTA: Using Consensus Research to Overcome PICO Scoping Challenges Under the EU HTAR https://www.mdpi.com/2001-6689/13/2/27 The evolving landscape of Health Technology Assessment (HTA) in Europe, shaped by the implementation of the new EU HTA Regulation (HTAR), places an emphasis on engaging all stakeholders, including patients, in collaborative evidence generation. Yet integrating patients’ perspectives into critical processes like PICO scoping remains a challenge, with concerns around subjectivity, representativeness, and methodological robustness. This opinion paper examines the complexities of patient engagement in HTA, highlighting both the opportunities for patients to make meaningful contributions and the barriers that stand in the way. We propose a framework that employes the Delphi panel methodology to (1) foster scientific validity and increase transparency in patient contributions, (2) establish a structured and consistent patient engagement framework, and (3) and understand European patients’ perspectives while promoting collaboration among EU countries. By facilitating iterative feedback and fostering agreement among diverse groups of patients and caregivers contributing with their expertise, consensus methods like Delphi panels can help refine PICO criteria, align diverse stakeholders’ expectations, and increase the relevance of HTA outcomes. A study is now underway to evaluate the feasibility and value of using the modified Delphi panel methodology for patient engagement in PICO scoping. The authors propose that embracing patient engagement through carefully designed consensus frameworks could enhance the legitimacy and completeness of HTA processes, driving more patient-centered decision making across Europe. 2025-06-02 JMAHP, Vol. 13, Pages 27: Enhancing Patient Engagement in HTA: Using Consensus Research to Overcome PICO Scoping Challenges Under the EU HTAR

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020027

Authors: Emanuele Arcà Adele Barlassina Adaeze Eze Valentina Strammiello

The evolving landscape of Health Technology Assessment (HTA) in Europe, shaped by the implementation of the new EU HTA Regulation (HTAR), places an emphasis on engaging all stakeholders, including patients, in collaborative evidence generation. Yet integrating patients’ perspectives into critical processes like PICO scoping remains a challenge, with concerns around subjectivity, representativeness, and methodological robustness. This opinion paper examines the complexities of patient engagement in HTA, highlighting both the opportunities for patients to make meaningful contributions and the barriers that stand in the way. We propose a framework that employes the Delphi panel methodology to (1) foster scientific validity and increase transparency in patient contributions, (2) establish a structured and consistent patient engagement framework, and (3) and understand European patients’ perspectives while promoting collaboration among EU countries. By facilitating iterative feedback and fostering agreement among diverse groups of patients and caregivers contributing with their expertise, consensus methods like Delphi panels can help refine PICO criteria, align diverse stakeholders’ expectations, and increase the relevance of HTA outcomes. A study is now underway to evaluate the feasibility and value of using the modified Delphi panel methodology for patient engagement in PICO scoping. The authors propose that embracing patient engagement through carefully designed consensus frameworks could enhance the legitimacy and completeness of HTA processes, driving more patient-centered decision making across Europe.

]]> Enhancing Patient Engagement in HTA: Using Consensus Research to Overcome PICO Scoping Challenges Under the EU HTAR Emanuele Arcà Adele Barlassina Adaeze Eze Valentina Strammiello doi: 10.3390/jmahp13020027 Journal of Market Access & Health Policy 2025-06-02 Journal of Market Access & Health Policy 2025-06-02 13 2 Opinion 27 10.3390/jmahp13020027 https://www.mdpi.com/2001-6689/13/2/27 JMAHP, Vol. 13, Pages 26: The Art of Bridge Building: A Look at the European-Level Cooperation in HTA (EU-HTA) https://www.mdpi.com/2001-6689/13/2/26 Health technology assessment (HTA) is a methodological and a scientific evidence-based process that allows competent authorities to determine the relative effectiveness of new or existing health technologies and to inform real-world decisions [...] 2025-05-28 JMAHP, Vol. 13, Pages 26: The Art of Bridge Building: A Look at the European-Level Cooperation in HTA (EU-HTA)

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020026

Authors: Rui Santos Ivo Tiago Rodrigues Sara Couto Mariane Cossito

Health technology assessment (HTA) is a methodological and a scientific evidence-based process that allows competent authorities to determine the relative effectiveness of new or existing health technologies and to inform real-world decisions [...]

]]> The Art of Bridge Building: A Look at the European-Level Cooperation in HTA (EU-HTA) Rui Santos Ivo Tiago Rodrigues Sara Couto Mariane Cossito doi: 10.3390/jmahp13020026 Journal of Market Access & Health Policy 2025-05-28 Journal of Market Access & Health Policy 2025-05-28 13 2 Editorial 26 10.3390/jmahp13020026 https://www.mdpi.com/2001-6689/13/2/26 JMAHP, Vol. 13, Pages 25: Price Analysis of Systemic Therapies and Transarterial Radioembolization for Treatment of Unresectable Hepatocellular Carcinoma https://www.mdpi.com/2001-6689/13/2/25 Systemic therapy (ST) and transarterial radioembolization (TARE) are widely used treatments for advanced-stage hepatocellular carcinoma (HCC). This study quantified the significant variability in treatment costs for unresectable HCC from payer and provider perspectives. An Excel-based price analysis model was developed to estimate the prices of ST and TARE over a 21-month time horizon using 2015–2021 data. Median prices were calculated from Medicare Average Sales Price (ASP), provider Wholesale Acquisition Cost (WAC), and Average Wholesale Price (AWP). Sensitivity analyses evaluated price fluctuations associated with a ±10% variation in treatment duration. ST prices demonstrated marked variability across perspectives, with the median ASP at $175,625, WAC at $198,719, and AWP at $262,892. However, TARE prices were stable, ranging from $21,594 to $24,052. Sensitivity analyses revealed that treatment duration variation resulted in price changes of $35,000–$50,000 for ST, compared with ~$5000 for TARE. The variability in ST pricing was driven by treatment duration and drug-specific pricing mechanisms, particularly immunotherapy-based regimens, which accounted for the higher cost range. Conversely, TARE’s consistent pricing is attributed to standardized procedural costs. Substantial variability exists in ST prices compared with the consistent costs of TARE, underscoring the economic advantage of TARE in appropriate clinical contexts. 2025-05-27 JMAHP, Vol. 13, Pages 25: Price Analysis of Systemic Therapies and Transarterial Radioembolization for Treatment of Unresectable Hepatocellular Carcinoma

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020025

Authors: Abimbola O. Williams Nicholas Anderson Young-Gwan Gwon Wendy Wifler

Systemic therapy (ST) and transarterial radioembolization (TARE) are widely used treatments for advanced-stage hepatocellular carcinoma (HCC). This study quantified the significant variability in treatment costs for unresectable HCC from payer and provider perspectives. An Excel-based price analysis model was developed to estimate the prices of ST and TARE over a 21-month time horizon using 2015–2021 data. Median prices were calculated from Medicare Average Sales Price (ASP), provider Wholesale Acquisition Cost (WAC), and Average Wholesale Price (AWP). Sensitivity analyses evaluated price fluctuations associated with a ±10% variation in treatment duration. ST prices demonstrated marked variability across perspectives, with the median ASP at $175,625, WAC at $198,719, and AWP at $262,892. However, TARE prices were stable, ranging from $21,594 to $24,052. Sensitivity analyses revealed that treatment duration variation resulted in price changes of $35,000–$50,000 for ST, compared with ~$5000 for TARE. The variability in ST pricing was driven by treatment duration and drug-specific pricing mechanisms, particularly immunotherapy-based regimens, which accounted for the higher cost range. Conversely, TARE’s consistent pricing is attributed to standardized procedural costs. Substantial variability exists in ST prices compared with the consistent costs of TARE, underscoring the economic advantage of TARE in appropriate clinical contexts.

]]> Price Analysis of Systemic Therapies and Transarterial Radioembolization for Treatment of Unresectable Hepatocellular Carcinoma Abimbola O. Williams Nicholas Anderson Young-Gwan Gwon Wendy Wifler doi: 10.3390/jmahp13020025 Journal of Market Access & Health Policy 2025-05-27 Journal of Market Access & Health Policy 2025-05-27 13 2 Article 25 10.3390/jmahp13020025 https://www.mdpi.com/2001-6689/13/2/25 JMAHP, Vol. 13, Pages 24: Optimizing Patient Access to Orphan Medicinal Products: Lessons from Central and Eastern Europe https://www.mdpi.com/2001-6689/13/2/24 This study examines patient access to orphan medicinal products (OMPs) in Central and Eastern Europe (CEE) over the past five years, focusing on seven countries: Bulgaria, Czechia, Hungary, Poland, Romania, Slovakia, and Slovenia. While these jurisdictions have undergone rapid healthcare transformations, significant disparities in OMP access persist compared to Western Europe. This study aimed to address this gap by identifying barriers and enablers to optimize patient access to OMPs in a sustainable and equitable manner. A mixed-methodology approach was utilized, combining systematic literature reviews, in-depth interviews, and advisory board insights. Perspectives were gathered from a wide range of stakeholders, including policymakers, payers, academia, industry associations, and patient advocacy groups. Additionally, the study incorporated data from CEE-specific initiatives to triangulate findings and evaluate barriers, enablers, and best practices in OMP access. The analysis identified sub-optimal OMP access across most CEE countries, marked by prolonged delays and lower reimbursement rates compared to Western Europe, with Slovenia and Czechia as notable exceptions. Key barriers include limited awareness, inadequate health technology assessment (HTA) frameworks, insufficient financing mechanisms, underutilization of novel access schemes, and fragmented patient engagement. Conversely, enablers include the presence of rare disease policies, OMP-specific HTA frameworks, and patient-inclusive decision-making processes. 2025-05-26 JMAHP, Vol. 13, Pages 24: Optimizing Patient Access to Orphan Medicinal Products: Lessons from Central and Eastern Europe

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020024

Authors: Tomasz Kluszczynski Bertalan Nemeth Magdalena Władysiuk Marcin Czech Maria Kamusheva Nicolae Fotin Sandra Rose Tomáš Doležal Rok Hren

This study examines patient access to orphan medicinal products (OMPs) in Central and Eastern Europe (CEE) over the past five years, focusing on seven countries: Bulgaria, Czechia, Hungary, Poland, Romania, Slovakia, and Slovenia. While these jurisdictions have undergone rapid healthcare transformations, significant disparities in OMP access persist compared to Western Europe. This study aimed to address this gap by identifying barriers and enablers to optimize patient access to OMPs in a sustainable and equitable manner. A mixed-methodology approach was utilized, combining systematic literature reviews, in-depth interviews, and advisory board insights. Perspectives were gathered from a wide range of stakeholders, including policymakers, payers, academia, industry associations, and patient advocacy groups. Additionally, the study incorporated data from CEE-specific initiatives to triangulate findings and evaluate barriers, enablers, and best practices in OMP access. The analysis identified sub-optimal OMP access across most CEE countries, marked by prolonged delays and lower reimbursement rates compared to Western Europe, with Slovenia and Czechia as notable exceptions. Key barriers include limited awareness, inadequate health technology assessment (HTA) frameworks, insufficient financing mechanisms, underutilization of novel access schemes, and fragmented patient engagement. Conversely, enablers include the presence of rare disease policies, OMP-specific HTA frameworks, and patient-inclusive decision-making processes.

]]> Optimizing Patient Access to Orphan Medicinal Products: Lessons from Central and Eastern Europe Tomasz Kluszczynski Bertalan Nemeth Magdalena Władysiuk Marcin Czech Maria Kamusheva Nicolae Fotin Sandra Rose Tomáš Doležal Rok Hren doi: 10.3390/jmahp13020024 Journal of Market Access & Health Policy 2025-05-26 Journal of Market Access & Health Policy 2025-05-26 13 2 Article 24 10.3390/jmahp13020024 https://www.mdpi.com/2001-6689/13/2/24 JMAHP, Vol. 13, Pages 23: Streamlining Endoscopy Cleaning: The Impact of a New Detergent on Time and Water Use https://www.mdpi.com/2001-6689/13/2/23 Reprocessing reusable flexible endoscopes is resource-intensive and involves high water consumption. This study evaluated the impact of replacing a standard detergent with EndoPreZyme™, a novel detergent, at Blackpool Teaching Hospitals NHS Foundation Trust. We assessed manual cleaning times, water usage, costs, and technician experiences. A direct observational time system analysis was conducted over two one-week periods to record technician tasks before and after implementing EndoPreZyme™, allowing for the omission of the final rinse after manual cleaning. Technician surveys captured user experiences during the transition. The results showed that removing the final rinse after manual cleaning reduced water consumption by 25 litres per endoscope, resulting in an estimated saving of 725,000 L annually. The average manual cleaning time decreased from 13 min 10.2 s to 11 min 10.7 s—a reduction of 1 min 59.5 s per endoscope (15%). This efficiency gain translated to approximately 962.9 fewer technician hours being required annually for manual cleaning. Cost analysis revealed a slight per-endoscope cost reduction (GBP 4.88 vs. GBP 4.90). Technicians reported improved productivity, reduced workload, and an awareness of water conservation. These findings demonstrate that EndoPreZyme™ supports NHS sustainability goals by decreasing water usage and enhancing operational efficiency in healthcare delivery. 2025-05-16 JMAHP, Vol. 13, Pages 23: Streamlining Endoscopy Cleaning: The Impact of a New Detergent on Time and Water Use

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020023

Authors: Joshua Hicks Mutsa Mutowo

Reprocessing reusable flexible endoscopes is resource-intensive and involves high water consumption. This study evaluated the impact of replacing a standard detergent with EndoPreZyme™, a novel detergent, at Blackpool Teaching Hospitals NHS Foundation Trust. We assessed manual cleaning times, water usage, costs, and technician experiences. A direct observational time system analysis was conducted over two one-week periods to record technician tasks before and after implementing EndoPreZyme™, allowing for the omission of the final rinse after manual cleaning. Technician surveys captured user experiences during the transition. The results showed that removing the final rinse after manual cleaning reduced water consumption by 25 litres per endoscope, resulting in an estimated saving of 725,000 L annually. The average manual cleaning time decreased from 13 min 10.2 s to 11 min 10.7 s—a reduction of 1 min 59.5 s per endoscope (15%). This efficiency gain translated to approximately 962.9 fewer technician hours being required annually for manual cleaning. Cost analysis revealed a slight per-endoscope cost reduction (GBP 4.88 vs. GBP 4.90). Technicians reported improved productivity, reduced workload, and an awareness of water conservation. These findings demonstrate that EndoPreZyme™ supports NHS sustainability goals by decreasing water usage and enhancing operational efficiency in healthcare delivery.

]]> Streamlining Endoscopy Cleaning: The Impact of a New Detergent on Time and Water Use Joshua Hicks Mutsa Mutowo doi: 10.3390/jmahp13020023 Journal of Market Access & Health Policy 2025-05-16 Journal of Market Access & Health Policy 2025-05-16 13 2 Article 23 10.3390/jmahp13020023 https://www.mdpi.com/2001-6689/13/2/23 JMAHP, Vol. 13, Pages 22: Joint Scientific Consultation Eligibility Criterion: Hubris or Naïveté https://www.mdpi.com/2001-6689/13/2/22 The eligibility criteria for Joint Scientific Consultation (JSC) raise important questions about the approach taken by the Member State Coordination Group on Health Technology Assessment (HTACG) [...] 2025-05-15 JMAHP, Vol. 13, Pages 22: Joint Scientific Consultation Eligibility Criterion: Hubris or Naïveté

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020022

Authors: Mondher Toumi Bruno Falissard Laurent Boyer Pascal Auquier

The eligibility criteria for Joint Scientific Consultation (JSC) raise important questions about the approach taken by the Member State Coordination Group on Health Technology Assessment (HTACG) [...]

]]> Joint Scientific Consultation Eligibility Criterion: Hubris or Naïveté Mondher Toumi Bruno Falissard Laurent Boyer Pascal Auquier doi: 10.3390/jmahp13020022 Journal of Market Access & Health Policy 2025-05-15 Journal of Market Access & Health Policy 2025-05-15 13 2 Editorial 22 10.3390/jmahp13020022 https://www.mdpi.com/2001-6689/13/2/22 JMAHP, Vol. 13, Pages 21: Effects of Primary Healthcare Quality and Effectiveness on Hospitalization Indicators in Brazil https://www.mdpi.com/2001-6689/13/2/21 Advances in primary healthcare coverage for the improvement in health outcomes at the population level comprise a major goal of public policies of health, particularly considering increases in hospitalization costs linked to chronic diseases in recent decades. Previous evidence shows the positive effects of access to primary healthcare on hospitalization indicators in high-income countries; however, there is a lack of literature on the subject in Latin American countries. Thus, the present study proposes a quantitative investigation on connections between primary healthcare quality and effectiveness in relation to hospitalization indicators, in addition to the identification of its effects on inequalities in hospitalizations in Brazil. The study was based on an empirical analysis of data from five cross-sectional surveys representative at the population level conducted by the Brazilian Institute for Geography and Statistics (IBGE) in 1998, 2003, 2008, 2013, and 2019. Information on the demographic, socioeconomic, and health characteristics of individuals compatible across surveys were included in the analyses, in addition to data on household and survey characteristics. The statistical analyses were based on the estimation of logistic regression models for the exploration of effects of primary healthcare quality and effectiveness on hospitalizations, inpatient days, and perception of quality of hospital care. Furthermore, the estimation of concentration indexes and their disaggregation allowed to verify trends and determinants of inequalities in hospitalization indicators in Brazil throughout the period. The results indicate that primary healthcare effectiveness is associated with the lower occurrence and frequency of hospitalizations, and a lower length of stay in hospitals. Primary healthcare quality was associated with the perception of higher quality of hospital care. Trends in hospitalization indicators showed reduction in inequalities towards low-income individuals from 1998 to 2013, and primary healthcare quality presented minor influence on inequalities in hospitalizations, inpatient days, and perception of quality of hospital care. 2025-05-09 JMAHP, Vol. 13, Pages 21: Effects of Primary Healthcare Quality and Effectiveness on Hospitalization Indicators in Brazil

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020021

Authors: Bruna Leão Freitas Maria Luisa de Oliveira Collino Antiga Flavia Mori Sarti

Advances in primary healthcare coverage for the improvement in health outcomes at the population level comprise a major goal of public policies of health, particularly considering increases in hospitalization costs linked to chronic diseases in recent decades. Previous evidence shows the positive effects of access to primary healthcare on hospitalization indicators in high-income countries; however, there is a lack of literature on the subject in Latin American countries. Thus, the present study proposes a quantitative investigation on connections between primary healthcare quality and effectiveness in relation to hospitalization indicators, in addition to the identification of its effects on inequalities in hospitalizations in Brazil. The study was based on an empirical analysis of data from five cross-sectional surveys representative at the population level conducted by the Brazilian Institute for Geography and Statistics (IBGE) in 1998, 2003, 2008, 2013, and 2019. Information on the demographic, socioeconomic, and health characteristics of individuals compatible across surveys were included in the analyses, in addition to data on household and survey characteristics. The statistical analyses were based on the estimation of logistic regression models for the exploration of effects of primary healthcare quality and effectiveness on hospitalizations, inpatient days, and perception of quality of hospital care. Furthermore, the estimation of concentration indexes and their disaggregation allowed to verify trends and determinants of inequalities in hospitalization indicators in Brazil throughout the period. The results indicate that primary healthcare effectiveness is associated with the lower occurrence and frequency of hospitalizations, and a lower length of stay in hospitals. Primary healthcare quality was associated with the perception of higher quality of hospital care. Trends in hospitalization indicators showed reduction in inequalities towards low-income individuals from 1998 to 2013, and primary healthcare quality presented minor influence on inequalities in hospitalizations, inpatient days, and perception of quality of hospital care.

]]> Effects of Primary Healthcare Quality and Effectiveness on Hospitalization Indicators in Brazil Bruna Leão Freitas Maria Luisa de Oliveira Collino Antiga Flavia Mori Sarti doi: 10.3390/jmahp13020021 Journal of Market Access & Health Policy 2025-05-09 Journal of Market Access & Health Policy 2025-05-09 13 2 Article 21 10.3390/jmahp13020021 https://www.mdpi.com/2001-6689/13/2/21 JMAHP, Vol. 13, Pages 20: Validating the Predictions of a Dynamic Transmission Model Using Real-World Data from a Universal Varicella Vaccination Program in Germany https://www.mdpi.com/2001-6689/13/2/20 Dynamic transmission models (DTMs) have been used to estimate various aspects of the public health impact of varicella vaccination programs. The aim of this study was to validate the predictions of a DTM—developed using the typical approach to varicella modeling—using real-world data from a country with a long-term universal varicella vaccination (UVV) program and to assess the sensitivity of the predictions to changes in model input parameters. A compartmental, age-stratified DTM was developed using the settings corresponding to the existing UVV program in Germany. The model-predicted total number of varicella cases followed the same trend as observed in the reported data. The agreement between the simulations’ results and the data was the highest for the age group most exposed to varicella (0–5 years old), while for other age groups, a decline in accuracy was observed. Sensitivity analyses identified the input parameters having a crucial impact on the model’s long-term predictions. The results supported the reliability of the DTM for assessing the impact of varicella vaccination programs over the first decades after their introduction and provided an insight into how certain parameters and assumptions influence the model output and thus require careful evaluation in the studies of future varicella vaccination programs. 2025-05-06 JMAHP, Vol. 13, Pages 20: Validating the Predictions of a Dynamic Transmission Model Using Real-World Data from a Universal Varicella Vaccination Program in Germany

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020020

Authors: Iwona Żerda Tomasz Stanisz Tomasz Fundament Filip Chełmikowski Wioletta Kłębczyk Michał Pochopień Emilie Clay Samuel Aballéa Mondher Toumi

Dynamic transmission models (DTMs) have been used to estimate various aspects of the public health impact of varicella vaccination programs. The aim of this study was to validate the predictions of a DTM—developed using the typical approach to varicella modeling—using real-world data from a country with a long-term universal varicella vaccination (UVV) program and to assess the sensitivity of the predictions to changes in model input parameters. A compartmental, age-stratified DTM was developed using the settings corresponding to the existing UVV program in Germany. The model-predicted total number of varicella cases followed the same trend as observed in the reported data. The agreement between the simulations’ results and the data was the highest for the age group most exposed to varicella (0–5 years old), while for other age groups, a decline in accuracy was observed. Sensitivity analyses identified the input parameters having a crucial impact on the model’s long-term predictions. The results supported the reliability of the DTM for assessing the impact of varicella vaccination programs over the first decades after their introduction and provided an insight into how certain parameters and assumptions influence the model output and thus require careful evaluation in the studies of future varicella vaccination programs.

]]> Validating the Predictions of a Dynamic Transmission Model Using Real-World Data from a Universal Varicella Vaccination Program in Germany Iwona Żerda Tomasz Stanisz Tomasz Fundament Filip Chełmikowski Wioletta Kłębczyk Michał Pochopień Emilie Clay Samuel Aballéa Mondher Toumi doi: 10.3390/jmahp13020020 Journal of Market Access & Health Policy 2025-05-06 Journal of Market Access & Health Policy 2025-05-06 13 2 Article 20 10.3390/jmahp13020020 https://www.mdpi.com/2001-6689/13/2/20 JMAHP, Vol. 13, Pages 19: Towards a Unified European View of Clinical Evidence: What ‘Health Technology Assessment Organizations’ Can Learn from Regulatory Experience https://www.mdpi.com/2001-6689/13/2/19 The harmonization of pharmaceutical regulations within the European Union has been a crucial step towards ensuring high safety standards and efficient access to innovative medicines. The evolution from fragmented national regulations to a unified legal framework has streamlined the marketing authorization process, fostered scientific collaboration, and reduced administrative burdens. The establishment of the centralized marketing authorization procedure and the European Medicines Agency (EMA) has played a pivotal role in coordinating regulatory efforts across member states. This article examines the historical developments, regulatory milestones, and the impact of harmonization on pharmaceutical assessments. Furthermore, it explores key lessons learned—including the value of centralized coordination, standardization, capacity and knowledge-sharing, transparency and trust—from the regulatory landscape that could inform the evolving Health Technology Assessment (HTA) framework in the EU. 2025-04-28 JMAHP, Vol. 13, Pages 19: Towards a Unified European View of Clinical Evidence: What ‘Health Technology Assessment Organizations’ Can Learn from Regulatory Experience

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020019

Authors: Karl Broich Wiebke Löbker

The harmonization of pharmaceutical regulations within the European Union has been a crucial step towards ensuring high safety standards and efficient access to innovative medicines. The evolution from fragmented national regulations to a unified legal framework has streamlined the marketing authorization process, fostered scientific collaboration, and reduced administrative burdens. The establishment of the centralized marketing authorization procedure and the European Medicines Agency (EMA) has played a pivotal role in coordinating regulatory efforts across member states. This article examines the historical developments, regulatory milestones, and the impact of harmonization on pharmaceutical assessments. Furthermore, it explores key lessons learned—including the value of centralized coordination, standardization, capacity and knowledge-sharing, transparency and trust—from the regulatory landscape that could inform the evolving Health Technology Assessment (HTA) framework in the EU.

]]> Towards a Unified European View of Clinical Evidence: What ‘Health Technology Assessment Organizations’ Can Learn from Regulatory Experience Karl Broich Wiebke Löbker doi: 10.3390/jmahp13020019 Journal of Market Access & Health Policy 2025-04-28 Journal of Market Access & Health Policy 2025-04-28 13 2 Perspective 19 10.3390/jmahp13020019 https://www.mdpi.com/2001-6689/13/2/19 JMAHP, Vol. 13, Pages 18: A New Health Networking Infrastructure on Cancer Is Taking Shape in Europe: A Not-to-Miss Opportunity for the EU Regulatory System https://www.mdpi.com/2001-6689/13/2/18 At the inaugural EAA convention in Copenhagen (Europe’s Evolving HTA Regulation and Its Relevance for ‘Beating Cancer’, in May 2022), it was suggested that, in the future, the pillars necessary to fully address the problem of cancer should be communicated (i [...] 2025-04-28 JMAHP, Vol. 13, Pages 18: A New Health Networking Infrastructure on Cancer Is Taking Shape in Europe: A Not-to-Miss Opportunity for the EU Regulatory System

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020018

Authors: Paolo Giovanni Casali Stefano Capri

At the inaugural EAA convention in Copenhagen (Europe’s Evolving HTA Regulation and Its Relevance for ‘Beating Cancer’, in May 2022), it was suggested that, in the future, the pillars necessary to fully address the problem of cancer should be communicated (i [...]

]]> A New Health Networking Infrastructure on Cancer Is Taking Shape in Europe: A Not-to-Miss Opportunity for the EU Regulatory System Paolo Giovanni Casali Stefano Capri doi: 10.3390/jmahp13020018 Journal of Market Access & Health Policy 2025-04-28 Journal of Market Access & Health Policy 2025-04-28 13 2 Editorial 18 10.3390/jmahp13020018 https://www.mdpi.com/2001-6689/13/2/18 JMAHP, Vol. 13, Pages 17: Workplace Vaccination Against COVID-19 and Seasonal Influenza in the United States: A Modeling-Based Estimation of the Health and Economic Benefits for Employers and Employees https://www.mdpi.com/2001-6689/13/2/17 The objectives were to assess the economic burden of COVID-19 and impact of workplace COVID-19 vaccination in the United States (US). An economic model estimated COVID-19 workplace burden (infections, long COVID, inpatient/outpatient care, absent days) with and without vaccination, compared with seasonal influenza vaccination for context, using Optum’s de-identified Clinformatics® Data Mart Database. Without workplace vaccination, an average US business (with 10,000 employees), had 18,175 absent days from COVID-19 and lost productivity costs of USD 5.08 million. Implementing COVID-19 workplace vaccination (at 70% coverage) prevented approximately 3132 absent days, saving employers USD 876,453 (lost productivity) and USD 240,633 (medical costs); and saving employees USD 182,196 (medical costs) and USD 198,250 (lost wages) versus no COVID-19 workplace vaccination. The burden and vaccination impact were greater for COVID-19 versus seasonal influenza. Workplace vaccination for COVID-19 and seasonal influenza can have a significant impact for both the employer and employees through averted disease. 2025-04-24 JMAHP, Vol. 13, Pages 17: Workplace Vaccination Against COVID-19 and Seasonal Influenza in the United States: A Modeling-Based Estimation of the Health and Economic Benefits for Employers and Employees

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020017

Authors: Ekkehard Beck Keya Joshi Darshan Mehta Stephane Lorenc Bishoy Rizkalla Nicolas Van de Velde

The objectives were to assess the economic burden of COVID-19 and impact of workplace COVID-19 vaccination in the United States (US). An economic model estimated COVID-19 workplace burden (infections, long COVID, inpatient/outpatient care, absent days) with and without vaccination, compared with seasonal influenza vaccination for context, using Optum’s de-identified Clinformatics® Data Mart Database. Without workplace vaccination, an average US business (with 10,000 employees), had 18,175 absent days from COVID-19 and lost productivity costs of USD 5.08 million. Implementing COVID-19 workplace vaccination (at 70% coverage) prevented approximately 3132 absent days, saving employers USD 876,453 (lost productivity) and USD 240,633 (medical costs); and saving employees USD 182,196 (medical costs) and USD 198,250 (lost wages) versus no COVID-19 workplace vaccination. The burden and vaccination impact were greater for COVID-19 versus seasonal influenza. Workplace vaccination for COVID-19 and seasonal influenza can have a significant impact for both the employer and employees through averted disease.

]]> Workplace Vaccination Against COVID-19 and Seasonal Influenza in the United States: A Modeling-Based Estimation of the Health and Economic Benefits for Employers and Employees Ekkehard Beck Keya Joshi Darshan Mehta Stephane Lorenc Bishoy Rizkalla Nicolas Van de Velde doi: 10.3390/jmahp13020017 Journal of Market Access & Health Policy 2025-04-24 Journal of Market Access & Health Policy 2025-04-24 13 2 Article 17 10.3390/jmahp13020017 https://www.mdpi.com/2001-6689/13/2/17 JMAHP, Vol. 13, Pages 16: Strengthening the EU Health Technology Assessment Regulation: Integrating National Immunization Technical Advisory Groups for Comprehensive Vaccine Assessments https://www.mdpi.com/2001-6689/13/2/16 Background: Given their crucial role in vaccine assessment, National Immunization Technical Advisory Groups (NITAGs) should be considered in the Regulation on Health Technology Assessment (EU HTAR) to maximize the benefits of the EU HTAR for vaccines. This review and perspective piece identifies the gaps arising from NITAGs potential lack of involvement and proposes strategies for involving them. Methods: A targeted literature and guideline review was conducted to evaluate NITAGs’ current and future role in relation to the EU HTAR. The impact of the EU HTAR on diverse national HTA frameworks was explored in a three-country case study. Recommendations were developed to leverage strengths and address weaknesses to ensure consistent and cohesive vaccine assessments. Results: The case study revealed potential overlaps between NITAGs and the EU HTAR, particularly regarding horizon scanning and joint scientific consultations. The involvement of NITAGs in national assessments varies, influencing how well joint clinical assessment reports will ultimately align with and be applicable to individual Member States. Conclusions: Stronger consideration of vaccines within the EU HTAR and NITAG involvement can streamline assessments, reduce duplication, and improve alignment between European and national processes. Strategic actions, including capacity building and collaborations between NITAGs, are key in facilitating this process. 2025-04-18 JMAHP, Vol. 13, Pages 16: Strengthening the EU Health Technology Assessment Regulation: Integrating National Immunization Technical Advisory Groups for Comprehensive Vaccine Assessments

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020016

Authors: Jasmijn Beekman Adrianne de Roo Sharon Wolters Ramesh Marapin Gabriel Gurgel do Amaral Evgeni Dvortsin Sibilia Quilici Chiara de Waure Elena Petelos Maarten Postma Anna Viceré

Background: Given their crucial role in vaccine assessment, National Immunization Technical Advisory Groups (NITAGs) should be considered in the Regulation on Health Technology Assessment (EU HTAR) to maximize the benefits of the EU HTAR for vaccines. This review and perspective piece identifies the gaps arising from NITAGs potential lack of involvement and proposes strategies for involving them. Methods: A targeted literature and guideline review was conducted to evaluate NITAGs’ current and future role in relation to the EU HTAR. The impact of the EU HTAR on diverse national HTA frameworks was explored in a three-country case study. Recommendations were developed to leverage strengths and address weaknesses to ensure consistent and cohesive vaccine assessments. Results: The case study revealed potential overlaps between NITAGs and the EU HTAR, particularly regarding horizon scanning and joint scientific consultations. The involvement of NITAGs in national assessments varies, influencing how well joint clinical assessment reports will ultimately align with and be applicable to individual Member States. Conclusions: Stronger consideration of vaccines within the EU HTAR and NITAG involvement can streamline assessments, reduce duplication, and improve alignment between European and national processes. Strategic actions, including capacity building and collaborations between NITAGs, are key in facilitating this process.

]]> Strengthening the EU Health Technology Assessment Regulation: Integrating National Immunization Technical Advisory Groups for Comprehensive Vaccine Assessments Jasmijn Beekman Adrianne de Roo Sharon Wolters Ramesh Marapin Gabriel Gurgel do Amaral Evgeni Dvortsin Sibilia Quilici Chiara de Waure Elena Petelos Maarten Postma Anna Viceré doi: 10.3390/jmahp13020016 Journal of Market Access & Health Policy 2025-04-18 Journal of Market Access & Health Policy 2025-04-18 13 2 Opinion 16 10.3390/jmahp13020016 https://www.mdpi.com/2001-6689/13/2/16 JMAHP, Vol. 13, Pages 15: Clinical Trial Validity Guidance from the HTACG: Looking for Chicken Teeth https://www.mdpi.com/2001-6689/13/2/15 The Member State Coordination Group on Health Technology Assessment (HTACG) guidance on the validity of clinical studies [...] 2025-04-16 JMAHP, Vol. 13, Pages 15: Clinical Trial Validity Guidance from the HTACG: Looking for Chicken Teeth

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020015

Authors: Mondher Toumi Bruno Falissard Asma Jouini Samuel Aballéa Laurent Boyer

The Member State Coordination Group on Health Technology Assessment (HTACG) guidance on the validity of clinical studies [...]

]]> Clinical Trial Validity Guidance from the HTACG: Looking for Chicken Teeth Mondher Toumi Bruno Falissard Asma Jouini Samuel Aballéa Laurent Boyer doi: 10.3390/jmahp13020015 Journal of Market Access & Health Policy 2025-04-16 Journal of Market Access & Health Policy 2025-04-16 13 2 Editorial 15 10.3390/jmahp13020015 https://www.mdpi.com/2001-6689/13/2/15 JMAHP, Vol. 13, Pages 14: ARM’s Perspective on the First Joint Clinical Assessments for ATMPs: Challenges and Opportunities on the Path Ahead https://www.mdpi.com/2001-6689/13/2/14 Advanced Therapy Medicinal Products (ATMPs) are revolutionising modern medicine. By addressing the root cause rather than the symptoms of disease, ATMPs hold the promise of long-lasting benefits or even cures for severe, genetic, and rare diseases—including rare cancers—for patients with few or no viable treatment options. At the same time, the inherent complexities of ATMPs pose challenges to traditional HTA frameworks. Unlike conventional treatments, ATMPs are often one-time therapies with a high magnitude of effect. However, their long-term durability remains uncertain at launch. The Joint Clinical Assessment (JCA), under the EU’s Health Technology Assessment (HTA) Regulation, represents a once-in-a-generation opportunity to consolidate the strengths of national HTA processes into a unified framework that accounts for the specificities of ATMPs and streamlines decision-making, cementing Europe’s position as a pioneer in innovative HTA approaches. While concerns remain regarding the suitability of current JCA methodologies for ATMPs, the HTA Regulation continues to bring the HTA ecosystem closer together, with numerous benefits already emerging from EU-wide collaboration on JCAs. This article outlines the HTA challenges posed for and by ATMPs, and ARM’s perspective on the JCA’s implementation. A ‘fit for purpose’ JCA holds the promise to unlocking these therapies’ benefits for individuals across Europe. 2025-04-03 JMAHP, Vol. 13, Pages 14: ARM’s Perspective on the First Joint Clinical Assessments for ATMPs: Challenges and Opportunities on the Path Ahead

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020014

Authors: Paolo Morgese Stephen Majors Dilip Patel

Advanced Therapy Medicinal Products (ATMPs) are revolutionising modern medicine. By addressing the root cause rather than the symptoms of disease, ATMPs hold the promise of long-lasting benefits or even cures for severe, genetic, and rare diseases—including rare cancers—for patients with few or no viable treatment options. At the same time, the inherent complexities of ATMPs pose challenges to traditional HTA frameworks. Unlike conventional treatments, ATMPs are often one-time therapies with a high magnitude of effect. However, their long-term durability remains uncertain at launch. The Joint Clinical Assessment (JCA), under the EU’s Health Technology Assessment (HTA) Regulation, represents a once-in-a-generation opportunity to consolidate the strengths of national HTA processes into a unified framework that accounts for the specificities of ATMPs and streamlines decision-making, cementing Europe’s position as a pioneer in innovative HTA approaches. While concerns remain regarding the suitability of current JCA methodologies for ATMPs, the HTA Regulation continues to bring the HTA ecosystem closer together, with numerous benefits already emerging from EU-wide collaboration on JCAs. This article outlines the HTA challenges posed for and by ATMPs, and ARM’s perspective on the JCA’s implementation. A ‘fit for purpose’ JCA holds the promise to unlocking these therapies’ benefits for individuals across Europe.

]]> ARM’s Perspective on the First Joint Clinical Assessments for ATMPs: Challenges and Opportunities on the Path Ahead Paolo Morgese Stephen Majors Dilip Patel doi: 10.3390/jmahp13020014 Journal of Market Access & Health Policy 2025-04-03 Journal of Market Access & Health Policy 2025-04-03 13 2 Article 14 10.3390/jmahp13020014 https://www.mdpi.com/2001-6689/13/2/14 JMAHP, Vol. 13, Pages 13: Healthcare Professionals’ Perceptions About Medical Cannabis in Greece: A Qualitative Study https://www.mdpi.com/2001-6689/13/2/13 Background: Medical cannabis continues to generate interest as a potential therapeutic option, yet its acceptance in clinical practice faces challenges, including regulatory barriers, social stigma, and gaps in scientific evidence. Methods: This study explores the perspectives of Greek medical doctors and pharmacists on medical cannabis—key stakeholders in its clinical application—through semi-structured interviews with 12 participants from each profession. Results: Medical doctors and pharmacists expressed a range of views on medical cannabis, with many acknowledging its potential while emphasizing the need for rigorous, disease-specific research. Medical doctors highlighted the lack of consistent clinical trials, concerns about drug interactions, and the fine line between medical use and misuse. Pharmacists echoed these concerns, citing regulatory inconsistencies and the need for standardized dosing. Both groups agreed that social stigma and misinformation hinder cannabis adoption, advocating for targeted education and transparent research communication. Participants indicated that regulatory barriers also pose challenges, with calls for harmonized policies and phased market entry approaches. Effective communication strategies, including digital outreach and clear messaging, were suggested to differentiate medical cannabis from recreational use and improve trust among healthcare providers and patients. Participants also highlighted the urgent need for collaboration between policymakers, researchers, and healthcare professionals to establish medical cannabis as a credible therapeutic option. Conclusion: The insights gained provide actionable recommendations to bridge existing gaps and emphasize the need for a responsible, evidence-based approach to the acceptance of medical cannabis as a therapeutic option. 2025-04-02 JMAHP, Vol. 13, Pages 13: Healthcare Professionals’ Perceptions About Medical Cannabis in Greece: A Qualitative Study

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020013

Authors: Christos Ntais Yioula Melanthiou Michael A. Talias

Background: Medical cannabis continues to generate interest as a potential therapeutic option, yet its acceptance in clinical practice faces challenges, including regulatory barriers, social stigma, and gaps in scientific evidence. Methods: This study explores the perspectives of Greek medical doctors and pharmacists on medical cannabis—key stakeholders in its clinical application—through semi-structured interviews with 12 participants from each profession. Results: Medical doctors and pharmacists expressed a range of views on medical cannabis, with many acknowledging its potential while emphasizing the need for rigorous, disease-specific research. Medical doctors highlighted the lack of consistent clinical trials, concerns about drug interactions, and the fine line between medical use and misuse. Pharmacists echoed these concerns, citing regulatory inconsistencies and the need for standardized dosing. Both groups agreed that social stigma and misinformation hinder cannabis adoption, advocating for targeted education and transparent research communication. Participants indicated that regulatory barriers also pose challenges, with calls for harmonized policies and phased market entry approaches. Effective communication strategies, including digital outreach and clear messaging, were suggested to differentiate medical cannabis from recreational use and improve trust among healthcare providers and patients. Participants also highlighted the urgent need for collaboration between policymakers, researchers, and healthcare professionals to establish medical cannabis as a credible therapeutic option. Conclusion: The insights gained provide actionable recommendations to bridge existing gaps and emphasize the need for a responsible, evidence-based approach to the acceptance of medical cannabis as a therapeutic option.

]]> Healthcare Professionals’ Perceptions About Medical Cannabis in Greece: A Qualitative Study Christos Ntais Yioula Melanthiou Michael A. Talias doi: 10.3390/jmahp13020013 Journal of Market Access & Health Policy 2025-04-02 Journal of Market Access & Health Policy 2025-04-02 13 2 Article 13 10.3390/jmahp13020013 https://www.mdpi.com/2001-6689/13/2/13 JMAHP, Vol. 13, Pages 12: A Methodological Proposal for Health Technology Assessments: A Case Study on Biosimilar Drugs https://www.mdpi.com/2001-6689/13/2/12 This work proposes a methodological approach that could be useful in multidisciplinary health technology assessments (HTAs). Mathematical models based on real data were used to make predictions for the initial price and actual cost of three classes of biological drugs. Through a comparison of real data, with the data derived through this approach, degree coefficients were formulated to rank the negotiating capabilities of Italian regions. The proposed method could represent a valid means of support for healthcare decisionmakers in planning and reducing pharmaceutical spending, evaluating data, and finding uses for particular medical technologies. This study could be a useful tool for achieving the objectives of HTAs, providing a means of analysis that can be adapted to any data, which may be useful for rationalizing the use of health technologies, reducing waste, and optimally reallocating resources. 2025-03-31 JMAHP, Vol. 13, Pages 12: A Methodological Proposal for Health Technology Assessments: A Case Study on Biosimilar Drugs

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020012

Authors: Marilisa Pia Dimmito Lisa Marinelli Eleonora Chiara Toto Giuseppe Di Biase Ivana Cacciatore Pierpaolo Toto Michele Ciulla Benedetta Monti Fiorenzo Santoleri Alberto Costantini Antonio Di Stefano

This work proposes a methodological approach that could be useful in multidisciplinary health technology assessments (HTAs). Mathematical models based on real data were used to make predictions for the initial price and actual cost of three classes of biological drugs. Through a comparison of real data, with the data derived through this approach, degree coefficients were formulated to rank the negotiating capabilities of Italian regions. The proposed method could represent a valid means of support for healthcare decisionmakers in planning and reducing pharmaceutical spending, evaluating data, and finding uses for particular medical technologies. This study could be a useful tool for achieving the objectives of HTAs, providing a means of analysis that can be adapted to any data, which may be useful for rationalizing the use of health technologies, reducing waste, and optimally reallocating resources.

]]> A Methodological Proposal for Health Technology Assessments: A Case Study on Biosimilar Drugs Marilisa Pia Dimmito Lisa Marinelli Eleonora Chiara Toto Giuseppe Di Biase Ivana Cacciatore Pierpaolo Toto Michele Ciulla Benedetta Monti Fiorenzo Santoleri Alberto Costantini Antonio Di Stefano doi: 10.3390/jmahp13020012 Journal of Market Access & Health Policy 2025-03-31 Journal of Market Access & Health Policy 2025-03-31 13 2 Article 12 10.3390/jmahp13020012 https://www.mdpi.com/2001-6689/13/2/12 JMAHP, Vol. 13, Pages 11: The Cost-Effectiveness of Avatrombopag Versus Eltrombopag and Romiplostim in the Treatment of Patients with Immune Thrombocytopenia in the UK https://www.mdpi.com/2001-6689/13/2/11 Background: Thrombopoietin receptor agonists—romiplostim, eltrombopag and avatrombopag—are commonly used as second-line treatments for immune thrombocytopenia (ITP). Methods: A Markov model was developed to estimate the cost effectiveness of the three TPO-RAs in adults with insufficient response to previous treatment from the perspective of the UK National Health Service (NHS). The model considered the effects of bleeding events, concomitant ITP medications, rescue therapies and treatment related adverse events over a lifetime horizon. Model inputs for effectiveness were based on a network meta-analysis and other published literature on ITP management. Other model inputs included costs (e.g., drug acquisition and administration) and healthcare resource utilisation. Results: Avatrombopag was associated with higher quality-adjusted life-years (QALYs) (10.979) than romiplostim (10.628) and eltrombopag (10.085), producing incremental QALYs of −0.351 and −0.894, respectively. Avatrombopag was associated with lower total costs (GBP £319,334) compared with romiplostim (GBP 406,361 [cost saving of GBP 87,027]) and higher total costs compared with eltrombopag (GBP 313,987 [incremental cost of GBP 5347]). Avatrombopag therefore dominated romiplostim (more effective and less expensive) and was cost-effective versus eltrombopag (incremental cost-effectiveness ratio of GBP 5982 per QALY). Conclusions: Avatrombopag is a cost-effective treatment compared with romiplostim and eltrombopag for the second-line treatment of adults with ITP from the perspective of the UK NHS. 2025-03-24 JMAHP, Vol. 13, Pages 11: The Cost-Effectiveness of Avatrombopag Versus Eltrombopag and Romiplostim in the Treatment of Patients with Immune Thrombocytopenia in the UK

Journal of Market Access & Health Policy doi: 10.3390/jmahp13020011

Authors: Nichola Cooper Sebastian Guterres Michał Pochopień Koo Wilson Sam James Mondher Toumi Anna Tytuła Carly Rich Daniel Eriksson

Background: Thrombopoietin receptor agonists—romiplostim, eltrombopag and avatrombopag—are commonly used as second-line treatments for immune thrombocytopenia (ITP). Methods: A Markov model was developed to estimate the cost effectiveness of the three TPO-RAs in adults with insufficient response to previous treatment from the perspective of the UK National Health Service (NHS). The model considered the effects of bleeding events, concomitant ITP medications, rescue therapies and treatment related adverse events over a lifetime horizon. Model inputs for effectiveness were based on a network meta-analysis and other published literature on ITP management. Other model inputs included costs (e.g., drug acquisition and administration) and healthcare resource utilisation. Results: Avatrombopag was associated with higher quality-adjusted life-years (QALYs) (10.979) than romiplostim (10.628) and eltrombopag (10.085), producing incremental QALYs of −0.351 and −0.894, respectively. Avatrombopag was associated with lower total costs (GBP £319,334) compared with romiplostim (GBP 406,361 [cost saving of GBP 87,027]) and higher total costs compared with eltrombopag (GBP 313,987 [incremental cost of GBP 5347]). Avatrombopag therefore dominated romiplostim (more effective and less expensive) and was cost-effective versus eltrombopag (incremental cost-effectiveness ratio of GBP 5982 per QALY). Conclusions: Avatrombopag is a cost-effective treatment compared with romiplostim and eltrombopag for the second-line treatment of adults with ITP from the perspective of the UK NHS.

]]> The Cost-Effectiveness of Avatrombopag Versus Eltrombopag and Romiplostim in the Treatment of Patients with Immune Thrombocytopenia in the UK Nichola Cooper Sebastian Guterres Michał Pochopień Koo Wilson Sam James Mondher Toumi Anna Tytuła Carly Rich Daniel Eriksson doi: 10.3390/jmahp13020011 Journal of Market Access & Health Policy 2025-03-24 Journal of Market Access & Health Policy 2025-03-24 13 2 Article 11 10.3390/jmahp13020011 https://www.mdpi.com/2001-6689/13/2/11 JMAHP, Vol. 13, Pages 10: Public Health Decision-Maker Perspective on Joint Clinical Assessments in Central European EU Member States https://www.mdpi.com/2001-6689/13/1/10 The HTA R introduces provisions that may benefit member states, in particular the opportunity to share national or regional assessment reports, cooperate outside of clinical domains, or use the methodological guidelines on a local level for technologies that are not subject to joint assessment. Challenges related to the timelines, differences between assessment scopes, and diverging guidance may jeopardize the full potential of the HTA R in Central European EU member states. However, these are more likely to be related to the commitment and vigilance of local competent authorities. We attempt to address these opportunities and mark some challenges imposed by the application of the HTA R by taking the perspective of public health decision-makers in Central European EU member states. We conclude that the foundations for capitalizing on the opportunities offered by the HTA R are already laid in the region, and we foresee policymakers and payers sharing the responsibility of acting as drivers of change in health policy to reduce the duplication (or multiplication) of efforts by HTDs, as well as to increase the efficient use of HTA bodies’ resources. 2025-03-04 JMAHP, Vol. 13, Pages 10: Public Health Decision-Maker Perspective on Joint Clinical Assessments in Central European EU Member States

Journal of Market Access & Health Policy doi: 10.3390/jmahp13010010

Authors: Gergő Merész Dávid Dankó Márk Péter Molnár

The HTA R introduces provisions that may benefit member states, in particular the opportunity to share national or regional assessment reports, cooperate outside of clinical domains, or use the methodological guidelines on a local level for technologies that are not subject to joint assessment. Challenges related to the timelines, differences between assessment scopes, and diverging guidance may jeopardize the full potential of the HTA R in Central European EU member states. However, these are more likely to be related to the commitment and vigilance of local competent authorities. We attempt to address these opportunities and mark some challenges imposed by the application of the HTA R by taking the perspective of public health decision-makers in Central European EU member states. We conclude that the foundations for capitalizing on the opportunities offered by the HTA R are already laid in the region, and we foresee policymakers and payers sharing the responsibility of acting as drivers of change in health policy to reduce the duplication (or multiplication) of efforts by HTDs, as well as to increase the efficient use of HTA bodies’ resources.

]]> Public Health Decision-Maker Perspective on Joint Clinical Assessments in Central European EU Member States Gergő Merész Dávid Dankó Márk Péter Molnár doi: 10.3390/jmahp13010010 Journal of Market Access & Health Policy 2025-03-04 Journal of Market Access & Health Policy 2025-03-04 13 1 Opinion 10 10.3390/jmahp13010010 https://www.mdpi.com/2001-6689/13/1/10 JMAHP, Vol. 13, Pages 9: Ensuring the Efficiency and Effectiveness of Joint Clinical Assessment in National HTA Decision-Making: Insights from the 2024 CIRS Multi-Stakeholder Workshop https://www.mdpi.com/2001-6689/13/1/9 Background: This study explored the readiness and strategic considerations of companies and key stakeholders for the implementation of the Joint Clinical Assessment (JCA) under the European Health Technology Assessment Regulation (HTAR). It examined the implications of the JCA process for jurisdictional submission strategies, and decision-making across Europe. The study aimed at identifying key measures for an efficient and effective JCA process to enable national rollout. Methods: A survey was conducted with international pharmaceutical companies, followed by a multi-stakeholder workshop that expanded on the findings. The survey and workshop focused on key areas such as time to market, submission strategies, and the role of JCA in national decision-making processes. Descriptive and qualitative analyses were performed to identify recommendations for measuring and improving the JCA process. Results: 13 companies responded to the survey, respondents were generally prepared for the JCA process (readiness rated 6–7/10), but concerns persist about timeline uncertainties and timely JCA report delivery. In the short term, success for the HTAR from the company perspective is measured by positive recommendations across EU jurisdictions. Long term, the focus shifts to aligning HTA methodologies and evidence requirements across the EU. Establishing metrics to assess the efficiency and effectiveness of the JCA is a key step in the HTAR’s ongoing learning journey. To enhance the efficiency of the JCA process, a list of metrics is recommended for continuous improvement, as well as establishing training programs to strengthen member states’ capabilities, fostering open dialog for sharing technology-specific insights, and creating open-source tools to support companies. Additionally, research should be conducted to understand agencies’ expectations of the JCA and how they will use its reports, grouping agencies by archetype to identify trends. A key recommendation is the development of a product-based scorecard to evaluate JCA submissions and reviews from various perspectives, ensuring the process meets stakeholders’ needs and can be effectively utilized in national decision-making. Conclusions: The JCA process offers a significant opportunity to streamline HTA decision-making across Europe. This study highlights several key measures and consideration for its successful rollout, including the need for clearer communication about the role of JCA in national decisions, measurement of rollout time components, and the development of quality evaluation frameworks. A collaborative, iterative approach, where stakeholders continually refine the system, will be essential for its effectiveness. Addressing these challenges will enable the JCA to enhance efficiency, consistency, and ultimately improve access to treatments for patients. 2025-03-03 JMAHP, Vol. 13, Pages 9: Ensuring the Efficiency and Effectiveness of Joint Clinical Assessment in National HTA Decision-Making: Insights from the 2024 CIRS Multi-Stakeholder Workshop

Journal of Market Access & Health Policy doi: 10.3390/jmahp13010009

Authors: Ting Wang Neil McAuslane

Background: This study explored the readiness and strategic considerations of companies and key stakeholders for the implementation of the Joint Clinical Assessment (JCA) under the European Health Technology Assessment Regulation (HTAR). It examined the implications of the JCA process for jurisdictional submission strategies, and decision-making across Europe. The study aimed at identifying key measures for an efficient and effective JCA process to enable national rollout. Methods: A survey was conducted with international pharmaceutical companies, followed by a multi-stakeholder workshop that expanded on the findings. The survey and workshop focused on key areas such as time to market, submission strategies, and the role of JCA in national decision-making processes. Descriptive and qualitative analyses were performed to identify recommendations for measuring and improving the JCA process. Results: 13 companies responded to the survey, respondents were generally prepared for the JCA process (readiness rated 6–7/10), but concerns persist about timeline uncertainties and timely JCA report delivery. In the short term, success for the HTAR from the company perspective is measured by positive recommendations across EU jurisdictions. Long term, the focus shifts to aligning HTA methodologies and evidence requirements across the EU. Establishing metrics to assess the efficiency and effectiveness of the JCA is a key step in the HTAR’s ongoing learning journey. To enhance the efficiency of the JCA process, a list of metrics is recommended for continuous improvement, as well as establishing training programs to strengthen member states’ capabilities, fostering open dialog for sharing technology-specific insights, and creating open-source tools to support companies. Additionally, research should be conducted to understand agencies’ expectations of the JCA and how they will use its reports, grouping agencies by archetype to identify trends. A key recommendation is the development of a product-based scorecard to evaluate JCA submissions and reviews from various perspectives, ensuring the process meets stakeholders’ needs and can be effectively utilized in national decision-making. Conclusions: The JCA process offers a significant opportunity to streamline HTA decision-making across Europe. This study highlights several key measures and consideration for its successful rollout, including the need for clearer communication about the role of JCA in national decisions, measurement of rollout time components, and the development of quality evaluation frameworks. A collaborative, iterative approach, where stakeholders continually refine the system, will be essential for its effectiveness. Addressing these challenges will enable the JCA to enhance efficiency, consistency, and ultimately improve access to treatments for patients.

]]> Ensuring the Efficiency and Effectiveness of Joint Clinical Assessment in National HTA Decision-Making: Insights from the 2024 CIRS Multi-Stakeholder Workshop Ting Wang Neil McAuslane doi: 10.3390/jmahp13010009 Journal of Market Access & Health Policy 2025-03-03 Journal of Market Access & Health Policy 2025-03-03 13 1 Article 9 10.3390/jmahp13010009 https://www.mdpi.com/2001-6689/13/1/9 JMAHP, Vol. 13, Pages 8: The Integration of Social and Health Sectors in Scotland: An Analysis from the Prism of Different Public Policy Models https://www.mdpi.com/2001-6689/13/1/8 The integration of health and social care has been a key focus in Scotland, driven by demographic changes, rising healthcare costs, and the need for more efficient service delivery. The Public Bodies (Joint Working) (Scotland) Act 2014 sought to formalise this integration by restructuring governance and service provision to improve coordination between health and social care sectors. Despite these efforts, challenges remain in fully achieving the intended outcomes of the integration. This study analysed Scotland’s integrated health and social care through the theoretical frameworks of public choice, institutionalism, and functionalism. The objective was to examine policy drivers, structural mechanisms, and governance implications, providing insights into the broader impact of integrated care reforms. A qualitative research approach was employed, synthesising data from peer-reviewed literature, government publications, and policy documents. The findings on integration were systematically examined through the lens of each public policy model, allowing for a nuanced analysis of how Scotland’s approach to integration aligns with and diverges from these frameworks. A literature search was performed on PUBMED, Google Scholar, and Scottish government portals. While integration improved coordination and service delivery in some areas, limitations in funding allocation, workforce distribution, and governance autonomy limited its overall success. Scotland’s integrated care model demonstrates potential benefits in reducing service fragmentation and improving patient-centred care; however, persistent challenges such as funding constraints, workforce shortages, and governance conflicts indicate that integration alone is not sufficient to resolve systemic healthcare inefficiencies. This study provides a perspective on Scotland’s health and social care integration, offering valuable lessons for other European countries facing similar demographic and healthcare challenges. 2025-02-27 JMAHP, Vol. 13, Pages 8: The Integration of Social and Health Sectors in Scotland: An Analysis from the Prism of Different Public Policy Models

Journal of Market Access & Health Policy doi: 10.3390/jmahp13010008

Authors: Ricardo Correia de Matos Generosa do Nascimento Adalberto Campos Fernandes Cristiano Matos

The integration of health and social care has been a key focus in Scotland, driven by demographic changes, rising healthcare costs, and the need for more efficient service delivery. The Public Bodies (Joint Working) (Scotland) Act 2014 sought to formalise this integration by restructuring governance and service provision to improve coordination between health and social care sectors. Despite these efforts, challenges remain in fully achieving the intended outcomes of the integration. This study analysed Scotland’s integrated health and social care through the theoretical frameworks of public choice, institutionalism, and functionalism. The objective was to examine policy drivers, structural mechanisms, and governance implications, providing insights into the broader impact of integrated care reforms. A qualitative research approach was employed, synthesising data from peer-reviewed literature, government publications, and policy documents. The findings on integration were systematically examined through the lens of each public policy model, allowing for a nuanced analysis of how Scotland’s approach to integration aligns with and diverges from these frameworks. A literature search was performed on PUBMED, Google Scholar, and Scottish government portals. While integration improved coordination and service delivery in some areas, limitations in funding allocation, workforce distribution, and governance autonomy limited its overall success. Scotland’s integrated care model demonstrates potential benefits in reducing service fragmentation and improving patient-centred care; however, persistent challenges such as funding constraints, workforce shortages, and governance conflicts indicate that integration alone is not sufficient to resolve systemic healthcare inefficiencies. This study provides a perspective on Scotland’s health and social care integration, offering valuable lessons for other European countries facing similar demographic and healthcare challenges.

]]> The Integration of Social and Health Sectors in Scotland: An Analysis from the Prism of Different Public Policy Models Ricardo Correia de Matos Generosa do Nascimento Adalberto Campos Fernandes Cristiano Matos doi: 10.3390/jmahp13010008 Journal of Market Access & Health Policy 2025-02-27 Journal of Market Access & Health Policy 2025-02-27 13 1 Perspective 8 10.3390/jmahp13010008 https://www.mdpi.com/2001-6689/13/1/8 JMAHP, Vol. 13, Pages 7: Healthcare Resource Utilization (HCRU) and Direct Medical Costs Associated with Long COVID or Post-COVID-19 Conditions: Findings from a Literature Review https://www.mdpi.com/2001-6689/13/1/7 Approximately 10–20% of individuals suffering from COVID-19 develop prolonged symptoms known as long COVID or post-COVID condition (LC). This review aimed to assess healthcare resource use (HCRU) and healthcare costs associated with LC. Because LC is not clearly defined and often remains undiagnosed, studies reporting on long-term follow-up of individuals with a COVID-19 diagnosis were also included. Among the 41 publications included, 36 reported on HCRU and 16 on costs. Individuals with LC had significantly elevated HCRU and healthcare costs vs. controls without a COVID-19 diagnosis over ≥15 months, with a 7.6–13.1% increase in total healthcare costs per person per month as assessed by difference-in-difference analysis. Among studies that did not specifically refer to LC, having a COVID-19 diagnosis was associated with a significant 4–10% increase in long-term total HCRU over 6–8 months and a 1.3- to 2.9-fold relative increase in total healthcare costs over 6 months. Due to the heterogeneity of the included studies, high-quality evidence is needed to better understand the economic burden of LC. In the absence of effective treatments, prioritizing the prevention of acute COVID-19, e.g., through vaccination, may be crucial for preventing LC and the associated long-term HCRU and medical spending. 2025-02-12 JMAHP, Vol. 13, Pages 7: Healthcare Resource Utilization (HCRU) and Direct Medical Costs Associated with Long COVID or Post-COVID-19 Conditions: Findings from a Literature Review

Journal of Market Access & Health Policy doi: 10.3390/jmahp13010007

Authors: Elżbieta Łukomska Krzysztof Kloc Malwina Kowalska Aleksandra Matjaszek Keya Joshi Stefan Scholz Nicolas Van de Velde Ekkehard Beck

Approximately 10–20% of individuals suffering from COVID-19 develop prolonged symptoms known as long COVID or post-COVID condition (LC). This review aimed to assess healthcare resource use (HCRU) and healthcare costs associated with LC. Because LC is not clearly defined and often remains undiagnosed, studies reporting on long-term follow-up of individuals with a COVID-19 diagnosis were also included. Among the 41 publications included, 36 reported on HCRU and 16 on costs. Individuals with LC had significantly elevated HCRU and healthcare costs vs. controls without a COVID-19 diagnosis over ≥15 months, with a 7.6–13.1% increase in total healthcare costs per person per month as assessed by difference-in-difference analysis. Among studies that did not specifically refer to LC, having a COVID-19 diagnosis was associated with a significant 4–10% increase in long-term total HCRU over 6–8 months and a 1.3- to 2.9-fold relative increase in total healthcare costs over 6 months. Due to the heterogeneity of the included studies, high-quality evidence is needed to better understand the economic burden of LC. In the absence of effective treatments, prioritizing the prevention of acute COVID-19, e.g., through vaccination, may be crucial for preventing LC and the associated long-term HCRU and medical spending.

]]> Healthcare Resource Utilization (HCRU) and Direct Medical Costs Associated with Long COVID or Post-COVID-19 Conditions: Findings from a Literature Review Elżbieta Łukomska Krzysztof Kloc Malwina Kowalska Aleksandra Matjaszek Keya Joshi Stefan Scholz Nicolas Van de Velde Ekkehard Beck doi: 10.3390/jmahp13010007 Journal of Market Access & Health Policy 2025-02-12 Journal of Market Access & Health Policy 2025-02-12 13 1 Review 7 10.3390/jmahp13010007 https://www.mdpi.com/2001-6689/13/1/7 JMAHP, Vol. 13, Pages 6: Avoiding Error and Finding the Right Balance in European Health Technology Assessments: Insights Generated by the European Access Academy https://www.mdpi.com/2001-6689/13/1/6 Background: We examined four potential challenges for the implementation of the European Union (EU) Regulation 2021/2282 on Health Technology Assessment (EU HTAR): interaction with the European Medicines Agency (EMA), expert input, the interface of European health technology assessment (EU HTA) joint procedures with those within Member States, and the management of conflict of interest. This research aims to explore how to address these challenges in a balanced manner and prioritise key actions for effective collaboration in the context of the EU HTA. Methods: The methodology included a pre-convention survey among relevant stakeholders as well as working groups and the plenary ranking of discussion outcomes at the European Access Academy (EAA) Spring Convention 2024. Results: In the survey, 65.5% of respondents indicated that experts are currently not sufficiently included in the upcoming joint scientific consultations and clinical assessments; only 37.9% suggested that the EU HTA joint procedures would accelerate national appraisal decision-making, and 58.6% believed that the principles of ‘transparency’ and ‘competency’ are balanced in the EU HTA position on conflict of interest. The top priority action points identified in the working groups were the involvement of the best available expertise, the early and inclusive involvement of experts, strengthened early scientific dialogue, and the fostering of the political willingness/financial support of EU Member States to increase capacities. Conclusions: The key topics identified were an approach to conflict of interest that balances transparency obligations and the need for expertise, strengthens the involvement of clinical and patient experts, intensifies early interaction between the EMA and EU HTA, and increases the involvement of the EU Member States. 2025-02-10 JMAHP, Vol. 13, Pages 6: Avoiding Error and Finding the Right Balance in European Health Technology Assessments: Insights Generated by the European Access Academy

Journal of Market Access & Health Policy doi: 10.3390/jmahp13010006

Authors: Elaine Julian Tom Belleman Maria João Garcia Maureen Rutten-van Mölken Robin Doeswijk Rosa Giuliani Bernhard J. Wörmann Daniel Widmer Patrick Tilleul Ruben Casado Arroyo Valentina Strammiello Kate Morgan Marcus Guardian Michael Ermisch Renato Bernardini Fabrizio Gianfrate Stefano Capri Carin A. Uyl-de Groot Mira Pavlovic Jörg Ruof

Background: We examined four potential challenges for the implementation of the European Union (EU) Regulation 2021/2282 on Health Technology Assessment (EU HTAR): interaction with the European Medicines Agency (EMA), expert input, the interface of European health technology assessment (EU HTA) joint procedures with those within Member States, and the management of conflict of interest. This research aims to explore how to address these challenges in a balanced manner and prioritise key actions for effective collaboration in the context of the EU HTA. Methods: The methodology included a pre-convention survey among relevant stakeholders as well as working groups and the plenary ranking of discussion outcomes at the European Access Academy (EAA) Spring Convention 2024. Results: In the survey, 65.5% of respondents indicated that experts are currently not sufficiently included in the upcoming joint scientific consultations and clinical assessments; only 37.9% suggested that the EU HTA joint procedures would accelerate national appraisal decision-making, and 58.6% believed that the principles of ‘transparency’ and ‘competency’ are balanced in the EU HTA position on conflict of interest. The top priority action points identified in the working groups were the involvement of the best available expertise, the early and inclusive involvement of experts, strengthened early scientific dialogue, and the fostering of the political willingness/financial support of EU Member States to increase capacities. Conclusions: The key topics identified were an approach to conflict of interest that balances transparency obligations and the need for expertise, strengthens the involvement of clinical and patient experts, intensifies early interaction between the EMA and EU HTA, and increases the involvement of the EU Member States.

]]> Avoiding Error and Finding the Right Balance in European Health Technology Assessments: Insights Generated by the European Access Academy Elaine Julian Tom Belleman Maria João Garcia Maureen Rutten-van Mölken Robin Doeswijk Rosa Giuliani Bernhard J. Wörmann Daniel Widmer Patrick Tilleul Ruben Casado Arroyo Valentina Strammiello Kate Morgan Marcus Guardian Michael Ermisch Renato Bernardini Fabrizio Gianfrate Stefano Capri Carin A. Uyl-de Groot Mira Pavlovic Jörg Ruof doi: 10.3390/jmahp13010006 Journal of Market Access & Health Policy 2025-02-10 Journal of Market Access & Health Policy 2025-02-10 13 1 Article 6 10.3390/jmahp13010006 https://www.mdpi.com/2001-6689/13/1/6 JMAHP, Vol. 13, Pages 5: Agenzia Italiana del Farmaco (AIFA): Developments and Strategy in a Transitioning European HTA Landscape https://www.mdpi.com/2001-6689/13/1/5 The Agenzia Italiana del Farmaco (AIFA) is the national public body that regulates medicines for human use in Italy [...] 2025-02-05 JMAHP, Vol. 13, Pages 5: Agenzia Italiana del Farmaco (AIFA): Developments and Strategy in a Transitioning European HTA Landscape

Journal of Market Access & Health Policy doi: 10.3390/jmahp13010005

Authors: Robert Nisticò

The Agenzia Italiana del Farmaco (AIFA) is the national public body that regulates medicines for human use in Italy [...]

]]> Agenzia Italiana del Farmaco (AIFA): Developments and Strategy in a Transitioning European HTA Landscape Robert Nisticò doi: 10.3390/jmahp13010005 Journal of Market Access & Health Policy 2025-02-05 Journal of Market Access & Health Policy 2025-02-05 13 1 Editorial 5 10.3390/jmahp13010005 https://www.mdpi.com/2001-6689/13/1/5 JMAHP, Vol. 13, Pages 4: Musculoskeletal Magazine Advertising Focuses on White Individuals and Overlooks Minority Consumers https://www.mdpi.com/2001-6689/13/1/4 Introduction: Demographic disparities in musculoskeletal (MSK) health exist in the US. Racial representation in advertising has been shown to influence consumer behavior and buying patterns. Direct-to-consumer advertising that does not target a racially diverse audience may exacerbate MSK disparities by failing to reach minorities. We explore the hypothesis that minorities are underrepresented in direct-to-consumer MSK advertisements in this cross-sectional analysis. Methods: Using magazines from four databases, eight health-related magazine types were selected and advertisement categories were established. Racial distribution was analyzed using Pearson’s Chi-squared and Chi-squared tests. Fisher’s Exact test was used when >20% of cells had expected frequencies <5. Significance was set at α = 0.05. Results: Of the advertisements featuring at least one model, 68.5% featured a white-presenting model, followed by 17.6% with a black model. Further, 92.7% of advertisements were monoethnic or monoracial with an overrepresentation of white models (p < 0.001). Black models were overrepresented as athletes (p < 0.001) and underrepresented in advertisements for pain relief (p < 0.001). Hispanic/Latinx and Asian models were underrepresented across all advertisement categories (p < 0.001). Discussion: The causes of musculoskeletal health disparities are multifactorial. One potential influence is adjacent industries such as MSK health-related advertisements. When controlling for US population demographics, white models were overrepresented and minority race models were underrepresented, demonstrating racioethnic disparities in MSK advertising. Improving the racial and ethnic diversity of models within MSK advertisements may serve to improve patient perceptions of orthopaedic products and services and improve MSK disparities. 2025-02-04 JMAHP, Vol. 13, Pages 4: Musculoskeletal Magazine Advertising Focuses on White Individuals and Overlooks Minority Consumers

Journal of Market Access & Health Policy doi: 10.3390/jmahp13010004

Authors: Wei Shao Tung Kelsey A. Rankin Robert John Oris Adithi Wijesekera Daniel H. Wiznia

Introduction: Demographic disparities in musculoskeletal (MSK) health exist in the US. Racial representation in advertising has been shown to influence consumer behavior and buying patterns. Direct-to-consumer advertising that does not target a racially diverse audience may exacerbate MSK disparities by failing to reach minorities. We explore the hypothesis that minorities are underrepresented in direct-to-consumer MSK advertisements in this cross-sectional analysis. Methods: Using magazines from four databases, eight health-related magazine types were selected and advertisement categories were established. Racial distribution was analyzed using Pearson’s Chi-squared and Chi-squared tests. Fisher’s Exact test was used when >20% of cells had expected frequencies <5. Significance was set at α = 0.05. Results: Of the advertisements featuring at least one model, 68.5% featured a white-presenting model, followed by 17.6% with a black model. Further, 92.7% of advertisements were monoethnic or monoracial with an overrepresentation of white models (p < 0.001). Black models were overrepresented as athletes (p < 0.001) and underrepresented in advertisements for pain relief (p < 0.001). Hispanic/Latinx and Asian models were underrepresented across all advertisement categories (p < 0.001). Discussion: The causes of musculoskeletal health disparities are multifactorial. One potential influence is adjacent industries such as MSK health-related advertisements. When controlling for US population demographics, white models were overrepresented and minority race models were underrepresented, demonstrating racioethnic disparities in MSK advertising. Improving the racial and ethnic diversity of models within MSK advertisements may serve to improve patient perceptions of orthopaedic products and services and improve MSK disparities.

]]> Musculoskeletal Magazine Advertising Focuses on White Individuals and Overlooks Minority Consumers Wei Shao Tung Kelsey A. Rankin Robert John Oris Adithi Wijesekera Daniel H. Wiznia doi: 10.3390/jmahp13010004 Journal of Market Access & Health Policy 2025-02-04 Journal of Market Access & Health Policy 2025-02-04 13 1 Communication 4 10.3390/jmahp13010004 https://www.mdpi.com/2001-6689/13/1/4 JMAHP, Vol. 13, Pages 3: The EU Health Technology Assessment Regulation Halo Effect: Are Cross-Functional Teams Ready? https://www.mdpi.com/2001-6689/13/1/3 The focus of manufacturers preparing for implementation of the EU HTA Regulation (HTAR) in 2025 has understandably been on their market access teams, and how they can be best equipped to adapt to this significant change. Considering the critical nature of market access in ensuring innovation reaches patients, it should be no surprise that the EU HTAR will have impacts far beyond this function. Here, we utilize published EU HTAR guidance, a pragmatic literature review, internal analysis, and insights from engagements with manufacturers, to outline some of the key cross-functional considerations arising from JSC and JCA, and how manufacturers should account for these in their EU HTAR readiness plans. 2025-01-30 JMAHP, Vol. 13, Pages 3: The EU Health Technology Assessment Regulation Halo Effect: Are Cross-Functional Teams Ready?

Journal of Market Access & Health Policy doi: 10.3390/jmahp13010003

Authors: Sian Tanner Rebecca Coady Ana Lisica Edel Falla Anke van Engen

The focus of manufacturers preparing for implementation of the EU HTA Regulation (HTAR) in 2025 has understandably been on their market access teams, and how they can be best equipped to adapt to this significant change. Considering the critical nature of market access in ensuring innovation reaches patients, it should be no surprise that the EU HTAR will have impacts far beyond this function. Here, we utilize published EU HTAR guidance, a pragmatic literature review, internal analysis, and insights from engagements with manufacturers, to outline some of the key cross-functional considerations arising from JSC and JCA, and how manufacturers should account for these in their EU HTAR readiness plans.

]]> The EU Health Technology Assessment Regulation Halo Effect: Are Cross-Functional Teams Ready? Sian Tanner Rebecca Coady Ana Lisica Edel Falla Anke van Engen doi: 10.3390/jmahp13010003 Journal of Market Access & Health Policy 2025-01-30 Journal of Market Access & Health Policy 2025-01-30 13 1 Opinion 3 10.3390/jmahp13010003 https://www.mdpi.com/2001-6689/13/1/3 JMAHP, Vol. 13, Pages 2: Multi-Year Multi-Indication Agreements for Supporting Patient Access to Oncology Medicines with Multiple Indications: An Experimental Approach or Here to Stay? https://www.mdpi.com/2001-6689/13/1/2 Over the past decade, an increasing number of oncology medicines with indications for multiple cancer types have been delivering benefits to patients. To ensure these products reach patients, pricing and reimbursement systems have had to adapt to address the value assessment, time-to-access, affordability, and budget uncertainty challenges this creates. Multi-year multi-indication (MYMI) agreements are made between payers and manufacturers and aim to ensure that patients have access to effective treatments for multiple conditions over time; this includes future indications of the treatment. MYMI agreements were first introduced as a solution in several European countries in 2017, offering a range of potential benefits. MYMI agreements have since demonstrated evidence of success in mitigating many of the challenges associated with assessing and reimbursing multi-indication products, time-to-patient access, and budget impact. The purpose of this article is to discuss the recent progress made with MYMI implementation across countries and provide a view on whether it is delivering for patients, healthcare systems, and innovators. We find that MYMI is not a one-size-fits-all solution but a model that needs to be adapted to the unique needs and characteristics of different healthcare systems. The intended benefits of MYMI to patients (speed and breadth of access to new indications) appear to have been realised in practice in some countries but not all. However, the administrative burden associated with MYMI implementation in some countries risks jeopardising the intended efficiency benefits. Payers and policymakers can also benefit from improved budget predictability and sustainability. 2025-01-24 JMAHP, Vol. 13, Pages 2: Multi-Year Multi-Indication Agreements for Supporting Patient Access to Oncology Medicines with Multiple Indications: An Experimental Approach or Here to Stay?

Journal of Market Access & Health Policy doi: 10.3390/jmahp13010002

Authors: Hannah Armstrong Angelina Petrova Tim Wilsdon Henriette Homoki Alexander Roediger

Over the past decade, an increasing number of oncology medicines with indications for multiple cancer types have been delivering benefits to patients. To ensure these products reach patients, pricing and reimbursement systems have had to adapt to address the value assessment, time-to-access, affordability, and budget uncertainty challenges this creates. Multi-year multi-indication (MYMI) agreements are made between payers and manufacturers and aim to ensure that patients have access to effective treatments for multiple conditions over time; this includes future indications of the treatment. MYMI agreements were first introduced as a solution in several European countries in 2017, offering a range of potential benefits. MYMI agreements have since demonstrated evidence of success in mitigating many of the challenges associated with assessing and reimbursing multi-indication products, time-to-patient access, and budget impact. The purpose of this article is to discuss the recent progress made with MYMI implementation across countries and provide a view on whether it is delivering for patients, healthcare systems, and innovators. We find that MYMI is not a one-size-fits-all solution but a model that needs to be adapted to the unique needs and characteristics of different healthcare systems. The intended benefits of MYMI to patients (speed and breadth of access to new indications) appear to have been realised in practice in some countries but not all. However, the administrative burden associated with MYMI implementation in some countries risks jeopardising the intended efficiency benefits. Payers and policymakers can also benefit from improved budget predictability and sustainability.

]]> Multi-Year Multi-Indication Agreements for Supporting Patient Access to Oncology Medicines with Multiple Indications: An Experimental Approach or Here to Stay? Hannah Armstrong Angelina Petrova Tim Wilsdon Henriette Homoki Alexander Roediger doi: 10.3390/jmahp13010002 Journal of Market Access & Health Policy 2025-01-24 Journal of Market Access & Health Policy 2025-01-24 13 1 Opinion 2 10.3390/jmahp13010002 https://www.mdpi.com/2001-6689/13/1/2 JMAHP, Vol. 13, Pages 1: Assessing the Value of Further Investment in R&D Using Mixed Methods: A Case Study of Biosensor-Integrated Arteriovenous Grafts https://www.mdpi.com/2001-6689/13/1/1 This study illustrates the utility of a mixed-methods approach in assessing the value of an example novel technology—biosensor-integrated self-reporting arteriovenous grafts (smart AVGs). Currently in preclinical development, the device will detect arteriovenous graft stenosis (surveillance-only use case) and treat stenosis (interventional use case). The approach to value assessment adopted in this study was multifaceted, with one stage informing the next and comprised a stakeholder engagement with clinical experts to explore the device’s clinical value, a cost–utility analysis (CUA) from a US Medicare perspective to estimate pricing headroom, and an investment model estimating risk-adjusted net present value analysis (rNPVs) to determine commercial viability. The stakeholder engagement suggested that it would currently be difficult to establish the current value of the surveillance-only use case due to the lack of well-established interventions for preclinical stenosis. Based on this, the CUA focused on the interventional use case and estimated economically justifiable prices at assumed effectiveness levels. Using these prices, rNPVs were estimated over a range of scenarios. This value assessment informs early decision-making on health technology R&D by identifying the conditions (including clinical study success, potential market size and penetration, market access strategies, and assumptions associated with CUA) under which investment may be considered attractive. 2025-01-15 JMAHP, Vol. 13, Pages 1: Assessing the Value of Further Investment in R&D Using Mixed Methods: A Case Study of Biosensor-Integrated Arteriovenous Grafts

Journal of Market Access & Health Policy doi: 10.3390/jmahp13010001

Authors: Samuel Owusu Achiaw Neil Hawkins Olivia Wu John Mercer

This study illustrates the utility of a mixed-methods approach in assessing the value of an example novel technology—biosensor-integrated self-reporting arteriovenous grafts (smart AVGs). Currently in preclinical development, the device will detect arteriovenous graft stenosis (surveillance-only use case) and treat stenosis (interventional use case). The approach to value assessment adopted in this study was multifaceted, with one stage informing the next and comprised a stakeholder engagement with clinical experts to explore the device’s clinical value, a cost–utility analysis (CUA) from a US Medicare perspective to estimate pricing headroom, and an investment model estimating risk-adjusted net present value analysis (rNPVs) to determine commercial viability. The stakeholder engagement suggested that it would currently be difficult to establish the current value of the surveillance-only use case due to the lack of well-established interventions for preclinical stenosis. Based on this, the CUA focused on the interventional use case and estimated economically justifiable prices at assumed effectiveness levels. Using these prices, rNPVs were estimated over a range of scenarios. This value assessment informs early decision-making on health technology R&D by identifying the conditions (including clinical study success, potential market size and penetration, market access strategies, and assumptions associated with CUA) under which investment may be considered attractive.

]]> Assessing the Value of Further Investment in R&D Using Mixed Methods: A Case Study of Biosensor-Integrated Arteriovenous Grafts Samuel Owusu Achiaw Neil Hawkins Olivia Wu John Mercer doi: 10.3390/jmahp13010001 Journal of Market Access & Health Policy 2025-01-15 Journal of Market Access & Health Policy 2025-01-15 13 1 Article 1 10.3390/jmahp13010001 https://www.mdpi.com/2001-6689/13/1/1 JMAHP, Vol. 12, Pages 403-414: Managing Pharmaceutical Costs in Health Systems: A Review of Affordability, Accessibility and Sustainability Strategies https://www.mdpi.com/2001-6689/12/4/31 Background: This paper reviews cost containment policies to control pharmaceutical expenditure either by regulating the pharmaceutical industry or targeting the demand side. Methods: The method used was the narrative literature review of studies which assessed the effect of pharmaceutical cost containment policies. Results: Governments worldwide have implemented a great variety of policy measures to manage pharmaceutical expenditure while ensuring fair access to essential medicines. Cost-sharing schemes, value-based pricing, reimbursement, reference pricing, payback mechanisms and the substitution of original drugs with generics and biosimilars are pivotal in these efforts, albeit with differing effectiveness across healthcare systems. Overall, it appears that any gains may be outweighed by the unfavorable effects of policies impacting patients. Although interventions have been created to improve physicians’ prescribing practice, they often achieve very minor benefits and at considerable cost. Policy measures pertaining to the regulation of the supply side must be supported by thorough evaluation in order to ascertain costs and effects and guarantee that unintended consequences are minimized. Conclusions: Policymakers frequently enact numerous laws and regulations to control pharmaceutical expenditure, even if there is limited evidence that they are cost-effective. The most crucial component of any policy’s success, regardless of the one selected, is its evaluation. Further research is needed to develop context-specific guidance that balances cost containment, equity and sustainability. 2024-12-10 JMAHP, Vol. 12, Pages 403-414: Managing Pharmaceutical Costs in Health Systems: A Review of Affordability, Accessibility and Sustainability Strategies

Journal of Market Access & Health Policy doi: 10.3390/jmahp12040031

Authors: Christos Ntais Michael A. Talias John Fanourgiakis Nikolaos Kontodimopoulos

Background: This paper reviews cost containment policies to control pharmaceutical expenditure either by regulating the pharmaceutical industry or targeting the demand side. Methods: The method used was the narrative literature review of studies which assessed the effect of pharmaceutical cost containment policies. Results: Governments worldwide have implemented a great variety of policy measures to manage pharmaceutical expenditure while ensuring fair access to essential medicines. Cost-sharing schemes, value-based pricing, reimbursement, reference pricing, payback mechanisms and the substitution of original drugs with generics and biosimilars are pivotal in these efforts, albeit with differing effectiveness across healthcare systems. Overall, it appears that any gains may be outweighed by the unfavorable effects of policies impacting patients. Although interventions have been created to improve physicians’ prescribing practice, they often achieve very minor benefits and at considerable cost. Policy measures pertaining to the regulation of the supply side must be supported by thorough evaluation in order to ascertain costs and effects and guarantee that unintended consequences are minimized. Conclusions: Policymakers frequently enact numerous laws and regulations to control pharmaceutical expenditure, even if there is limited evidence that they are cost-effective. The most crucial component of any policy’s success, regardless of the one selected, is its evaluation. Further research is needed to develop context-specific guidance that balances cost containment, equity and sustainability.

]]> Managing Pharmaceutical Costs in Health Systems: A Review of Affordability, Accessibility and Sustainability Strategies Christos Ntais Michael A. Talias John Fanourgiakis Nikolaos Kontodimopoulos doi: 10.3390/jmahp12040031 Journal of Market Access & Health Policy 2024-12-10 Journal of Market Access & Health Policy 2024-12-10 12 4 Review 403 10.3390/jmahp12040031 https://www.mdpi.com/2001-6689/12/4/31 JMAHP, Vol. 12, Pages 388-402: Operational Efficiency of Public Hospitals in Greece During the COVID-19 Pandemic: A Comparative Analysis Using DEA and AHP Models https://www.mdpi.com/2001-6689/12/4/30 This study evaluates the efficiency of public hospitals in Greece during the COVID-19 epidemic in 2020, using Data Envelopment Analysis (DEA) and the Analytical Hierarchy Process (AHP). Faced with unprecedented pressure from increased demand for medical services, these hospitals had to adapt quickly while playing a crucial role in supporting local economies, similar to the effect of tourism on rural economies. This study reveals that, despite average efficiency scores of 83% for result-oriented models (BCC) and 65% for constant return models (CCR), inefficiencies of scale emerged under the pressures of the pandemic. The AHP, by incorporating qualitative criteria and decision-makers’ preferences, offers a valuable perspective but shows little correlation with DEA’s quantitative results. This research emphasizes the importance of utilizing integrated methods to formulate a more comprehensive assessment, adapted to the complex challenges of the healthcare sector during crisis periods. 2024-12-10 JMAHP, Vol. 12, Pages 388-402: Operational Efficiency of Public Hospitals in Greece During the COVID-19 Pandemic: A Comparative Analysis Using DEA and AHP Models

Journal of Market Access & Health Policy doi: 10.3390/jmahp12040030

Authors: Athanasios Mitakos Panagiotis Mpogiatzidis

This study evaluates the efficiency of public hospitals in Greece during the COVID-19 epidemic in 2020, using Data Envelopment Analysis (DEA) and the Analytical Hierarchy Process (AHP). Faced with unprecedented pressure from increased demand for medical services, these hospitals had to adapt quickly while playing a crucial role in supporting local economies, similar to the effect of tourism on rural economies. This study reveals that, despite average efficiency scores of 83% for result-oriented models (BCC) and 65% for constant return models (CCR), inefficiencies of scale emerged under the pressures of the pandemic. The AHP, by incorporating qualitative criteria and decision-makers’ preferences, offers a valuable perspective but shows little correlation with DEA’s quantitative results. This research emphasizes the importance of utilizing integrated methods to formulate a more comprehensive assessment, adapted to the complex challenges of the healthcare sector during crisis periods.

]]> Operational Efficiency of Public Hospitals in Greece During the COVID-19 Pandemic: A Comparative Analysis Using DEA and AHP Models Athanasios Mitakos Panagiotis Mpogiatzidis doi: 10.3390/jmahp12040030 Journal of Market Access & Health Policy 2024-12-10 Journal of Market Access & Health Policy 2024-12-10 12 4 Article 388 10.3390/jmahp12040030 https://www.mdpi.com/2001-6689/12/4/30 JMAHP, Vol. 12, Pages 378-387: Gatekeeping or Provider Choice for Sustainable Health Systems? A Literature Review on Their Impact on Efficiency, Access, and Quality of Services https://www.mdpi.com/2001-6689/12/4/29 As early as 1978, the World Health Organization set primary healthcare as the basis on which health systems should be built worldwide. However, the health systems of the different countries show considerable variations in terms of the implementation of gatekeeping from primary to secondary healthcare and direct access to specialists and hospital care. This literature review attempts to present the gatekeeping system with references to the UK, Sweden, the Netherlands, and Germany compared to the situation in Greece, where no gatekeeping system exists. Particular emphasis is placed on the impact of gatekeeping on the healthcare system’s efficiency, equity of access, and the quality of the services provided. Evidence on the effects of gatekeeping is conflicting or limited by the low internal validity. Making the right gatekeeping implementation decisions is difficult in the absence of data. High-quality research studies on health outcomes, clinical efficacy, cost-effectiveness, quality of life, healthcare quality, utilisation of healthcare services, the burden in the healthcare system, and the opinions of patients, physicians, and policymakers are all necessary for developing policy. 2024-12-06 JMAHP, Vol. 12, Pages 378-387: Gatekeeping or Provider Choice for Sustainable Health Systems? A Literature Review on Their Impact on Efficiency, Access, and Quality of Services

Journal of Market Access & Health Policy doi: 10.3390/jmahp12040029

Authors: Christos Ntais Nikolaos Kontodimopoulos Michael A. Talias

As early as 1978, the World Health Organization set primary healthcare as the basis on which health systems should be built worldwide. However, the health systems of the different countries show considerable variations in terms of the implementation of gatekeeping from primary to secondary healthcare and direct access to specialists and hospital care. This literature review attempts to present the gatekeeping system with references to the UK, Sweden, the Netherlands, and Germany compared to the situation in Greece, where no gatekeeping system exists. Particular emphasis is placed on the impact of gatekeeping on the healthcare system’s efficiency, equity of access, and the quality of the services provided. Evidence on the effects of gatekeeping is conflicting or limited by the low internal validity. Making the right gatekeeping implementation decisions is difficult in the absence of data. High-quality research studies on health outcomes, clinical efficacy, cost-effectiveness, quality of life, healthcare quality, utilisation of healthcare services, the burden in the healthcare system, and the opinions of patients, physicians, and policymakers are all necessary for developing policy.

]]> Gatekeeping or Provider Choice for Sustainable Health Systems? A Literature Review on Their Impact on Efficiency, Access, and Quality of Services Christos Ntais Nikolaos Kontodimopoulos Michael A. Talias doi: 10.3390/jmahp12040029 Journal of Market Access & Health Policy 2024-12-06 Journal of Market Access & Health Policy 2024-12-06 12 4 Review 378 10.3390/jmahp12040029 https://www.mdpi.com/2001-6689/12/4/29 JMAHP, Vol. 12, Pages 369-377: Technology Assessment vs. Technology Appraisal—How to Strengthen the Science/Value Dichotomy with EU HTA? https://www.mdpi.com/2001-6689/12/4/28 Many countries around the world use health technology assessment (HTA) to inform reimbursement and pricing decisions. HTA is often split into two steps, called assessment and appraisal. While the term HTA itself has been defined by international consortia, there is heterogeneity in the way different stakeholders use the terms assessment and appraisal. This creates ambiguity regarding which activities are included in technology assessment. With the new EU HTA Regulation, the HTA community should urgently seek to clarify the distinction between assessment and appraisal, as the regulation aims to centralize the clinical part of technology assessment at the European level. Failure to clarify this terminology will put the ambition of the regulation such as increased efficiency and reduction in duplication at risk. In this article, we argue that the distinction between assessment and appraisal should be seen as a science/value dichotomy. We discuss the transition from centralized assessment activities to country-level appraisal, which should culminate in a categorization of the overall added benefit in a local context. Finally, we touch on the important dimension of uncertainty always present in medical decision making. 2024-11-18 JMAHP, Vol. 12, Pages 369-377: Technology Assessment vs. Technology Appraisal—How to Strengthen the Science/Value Dichotomy with EU HTA?

Journal of Market Access & Health Policy doi: 10.3390/jmahp12040028

Authors: Sandro Gsteiger Heiner C. Bucher James Ryan Jörg Ruof

Many countries around the world use health technology assessment (HTA) to inform reimbursement and pricing decisions. HTA is often split into two steps, called assessment and appraisal. While the term HTA itself has been defined by international consortia, there is heterogeneity in the way different stakeholders use the terms assessment and appraisal. This creates ambiguity regarding which activities are included in technology assessment. With the new EU HTA Regulation, the HTA community should urgently seek to clarify the distinction between assessment and appraisal, as the regulation aims to centralize the clinical part of technology assessment at the European level. Failure to clarify this terminology will put the ambition of the regulation such as increased efficiency and reduction in duplication at risk. In this article, we argue that the distinction between assessment and appraisal should be seen as a science/value dichotomy. We discuss the transition from centralized assessment activities to country-level appraisal, which should culminate in a categorization of the overall added benefit in a local context. Finally, we touch on the important dimension of uncertainty always present in medical decision making.

]]> Technology Assessment vs. Technology Appraisal—How to Strengthen the Science/Value Dichotomy with EU HTA? Sandro Gsteiger Heiner C. Bucher James Ryan Jörg Ruof doi: 10.3390/jmahp12040028 Journal of Market Access & Health Policy 2024-11-18 Journal of Market Access & Health Policy 2024-11-18 12 4 Opinion 369 10.3390/jmahp12040028 https://www.mdpi.com/2001-6689/12/4/28 JMAHP, Vol. 12, Pages 342-368: Economic Evidence on Cost Sharing and Alternative Insurance Designs to Address Moral and Behavioral Hazards in High-Income Health Care Systems: A Systematic Review https://www.mdpi.com/2001-6689/12/4/27 In health insurance, “moral hazard” describes the concept that coverage without an out-of-pocket cost to consumers could result in health care utilization beyond economically efficient levels. In response, payers in the United States (US) have designed pharmaceutical benefit plans with significant cost exposure (e.g., co-pays, co-insurance, or deductibles). While substantial evidence links patient cost exposure to reduced drug spending, it remains unclear to what degree this translates into greater efficiency or an indiscriminate drop in overall consumption also reducing needed utilization. We conducted a systematic literature review to understand whether commonly implemented utilization management (UM) strategies and insurance designs with a behavioral or value-based (BID/VBID) component have been explored as tools to mitigate moral hazard and to assess how cost-sharing policies and innovative insurance designs impact consumer spending. Eligible studies compared conventional cost-exposure policies to BID/VBID, including tiered cost-sharing and other UM strategies. We found that broad implementation of patient cost exposure is not well supported by empirical evidence assessing efficiency—defined as the use of clinically appropriate services with value at or above the marginal cost of health care utilization in the contemporary US setting. As a result, payers and policy makers alike ought to explore insurance alternatives that more closely align health care consumption incentives to value of care. 2024-11-14 JMAHP, Vol. 12, Pages 342-368: Economic Evidence on Cost Sharing and Alternative Insurance Designs to Address Moral and Behavioral Hazards in High-Income Health Care Systems: A Systematic Review

Journal of Market Access & Health Policy doi: 10.3390/jmahp12040027

Authors: Marlon Graf James R. Baumgardner Ulrich Neumann Iris P. Brewer Jacquelyn W. Chou A. Mark Fendrick

In health insurance, “moral hazard” describes the concept that coverage without an out-of-pocket cost to consumers could result in health care utilization beyond economically efficient levels. In response, payers in the United States (US) have designed pharmaceutical benefit plans with significant cost exposure (e.g., co-pays, co-insurance, or deductibles). While substantial evidence links patient cost exposure to reduced drug spending, it remains unclear to what degree this translates into greater efficiency or an indiscriminate drop in overall consumption also reducing needed utilization. We conducted a systematic literature review to understand whether commonly implemented utilization management (UM) strategies and insurance designs with a behavioral or value-based (BID/VBID) component have been explored as tools to mitigate moral hazard and to assess how cost-sharing policies and innovative insurance designs impact consumer spending. Eligible studies compared conventional cost-exposure policies to BID/VBID, including tiered cost-sharing and other UM strategies. We found that broad implementation of patient cost exposure is not well supported by empirical evidence assessing efficiency—defined as the use of clinically appropriate services with value at or above the marginal cost of health care utilization in the contemporary US setting. As a result, payers and policy makers alike ought to explore insurance alternatives that more closely align health care consumption incentives to value of care.

]]> Economic Evidence on Cost Sharing and Alternative Insurance Designs to Address Moral and Behavioral Hazards in High-Income Health Care Systems: A Systematic Review Marlon Graf James R. Baumgardner Ulrich Neumann Iris P. Brewer Jacquelyn W. Chou A. Mark Fendrick doi: 10.3390/jmahp12040027 Journal of Market Access & Health Policy 2024-11-14 Journal of Market Access & Health Policy 2024-11-14 12 4 Systematic Review 342 10.3390/jmahp12040027 https://www.mdpi.com/2001-6689/12/4/27 JMAHP, Vol. 12, Pages 326-341: Clinical Benefits of Sustained Oral Nirmatrelvir/Ritonavir Use for the Outpatient Treatment of COVID-19: Findings from the Taiwanese Health Authority Perspective Using a Decision Tree Modeling Approach https://www.mdpi.com/2001-6689/12/4/26 Despite the observed clinical benefits of nirmatrelvir/ritonavir (NMV/r), it is uncertain whether Taiwan will continue covering NMV/r for high-risk individuals with mild-to-moderate coronavirus disease 2019 (COVID-19). This analysis assessed the impact of sustained utilization of NMV/r on COVID-19-associated healthcare resource utilization (HCRU) and mortality from the Taiwanese health authority perspective (THAP). A decision tree model estimated the incremental number of clinical events associated with NMV/r utilization over a 30-day period. Model results compared (1) a base case using current rates of NMV/r from the THAP, and (2) a hypothetical scenario assuming the current supply of NMV/r is not extended in Taiwan. NMV/r utilization rates included 80% and 0% in the base case and hypothetical scenario, respectively. Outcomes included the number of hospitalizations involving a general ward (GW) stay, intensive care unit (ICU) stay, and mechanical ventilation (MV) use, as well as the number of bed days, symptom days, and hospitalization deaths. Based on epidemiologic data, 150,255 patients with COVID-19 were eligible for treatment from the THAP. In the hypothetical scenario, HCRU increased by 175% compared to the base case, including increases in hospitalizations involving GW, ICU, and MV use (differences: 2067; 623; 591, respectively), bed days (difference: 51,521), symptom days (difference: 51,714), and deaths (difference: 480). Findings indicate that sustained utilization of NMV/r from the THAP reduces the clinical burden of mild-to-moderate COVID-19 through the reduced incidence of COVID-19-related HCRU and deaths. 2024-11-12 JMAHP, Vol. 12, Pages 326-341: Clinical Benefits of Sustained Oral Nirmatrelvir/Ritonavir Use for the Outpatient Treatment of COVID-19: Findings from the Taiwanese Health Authority Perspective Using a Decision Tree Modeling Approach

Journal of Market Access & Health Policy doi: 10.3390/jmahp12040026

Authors: Matthew Sussman Jennifer Benner Tendai Mugwagwa Jackie Lee Sheng-Tzu Hung Ya-Min Yang Yixi Chen

Despite the observed clinical benefits of nirmatrelvir/ritonavir (NMV/r), it is uncertain whether Taiwan will continue covering NMV/r for high-risk individuals with mild-to-moderate coronavirus disease 2019 (COVID-19). This analysis assessed the impact of sustained utilization of NMV/r on COVID-19-associated healthcare resource utilization (HCRU) and mortality from the Taiwanese health authority perspective (THAP). A decision tree model estimated the incremental number of clinical events associated with NMV/r utilization over a 30-day period. Model results compared (1) a base case using current rates of NMV/r from the THAP, and (2) a hypothetical scenario assuming the current supply of NMV/r is not extended in Taiwan. NMV/r utilization rates included 80% and 0% in the base case and hypothetical scenario, respectively. Outcomes included the number of hospitalizations involving a general ward (GW) stay, intensive care unit (ICU) stay, and mechanical ventilation (MV) use, as well as the number of bed days, symptom days, and hospitalization deaths. Based on epidemiologic data, 150,255 patients with COVID-19 were eligible for treatment from the THAP. In the hypothetical scenario, HCRU increased by 175% compared to the base case, including increases in hospitalizations involving GW, ICU, and MV use (differences: 2067; 623; 591, respectively), bed days (difference: 51,521), symptom days (difference: 51,714), and deaths (difference: 480). Findings indicate that sustained utilization of NMV/r from the THAP reduces the clinical burden of mild-to-moderate COVID-19 through the reduced incidence of COVID-19-related HCRU and deaths.

]]> Clinical Benefits of Sustained Oral Nirmatrelvir/Ritonavir Use for the Outpatient Treatment of COVID-19: Findings from the Taiwanese Health Authority Perspective Using a Decision Tree Modeling Approach Matthew Sussman Jennifer Benner Tendai Mugwagwa Jackie Lee Sheng-Tzu Hung Ya-Min Yang Yixi Chen doi: 10.3390/jmahp12040026 Journal of Market Access & Health Policy 2024-11-12 Journal of Market Access & Health Policy 2024-11-12 12 4 Article 326 10.3390/jmahp12040026 https://www.mdpi.com/2001-6689/12/4/26 JMAHP, Vol. 12, Pages 317-325: Developer and Partnership Differences in COVID-19 and Other Infections: Insights from DNA Vaccines https://www.mdpi.com/2001-6689/12/4/25 Historically, vaccine development has been heavily supported by government and public institutions. On the other hand, private biopharmaceutical companies have played a significant role in the development of innovative new therapies using novel pharmaceutical technologies. COVID-19 vaccines using new vaccine technologies, such as mRNA and adenoviral vectors, were rapidly developed by emerging biopharmaceutical companies in collaboration with large corporations and public organizations. This underscores the crucial role of emerging biopharma and public–private partnerships in advancing new vaccine technologies. While these innovations have been suggested as models for future vaccines, their applicability to other infectious diseases requires careful assessment. This study investigated the characteristics of the developers and partnerships in the development of DNA vaccines as a next-generation vaccine platform. The analysis revealed that while emerging biopharmaceutical companies and private–private and private–public partnerships were crucial during the COVID-19 pandemic, public organizations and public–public collaborations primarily led to the clinical development of vaccines for other diseases. Strategies for vaccine development using new vaccine technologies should be tailored to the specific characteristics of each disease. 2024-10-29 JMAHP, Vol. 12, Pages 317-325: Developer and Partnership Differences in COVID-19 and Other Infections: Insights from DNA Vaccines

Journal of Market Access & Health Policy doi: 10.3390/jmahp12040025

Authors: Ryo Okuyama

Historically, vaccine development has been heavily supported by government and public institutions. On the other hand, private biopharmaceutical companies have played a significant role in the development of innovative new therapies using novel pharmaceutical technologies. COVID-19 vaccines using new vaccine technologies, such as mRNA and adenoviral vectors, were rapidly developed by emerging biopharmaceutical companies in collaboration with large corporations and public organizations. This underscores the crucial role of emerging biopharma and public–private partnerships in advancing new vaccine technologies. While these innovations have been suggested as models for future vaccines, their applicability to other infectious diseases requires careful assessment. This study investigated the characteristics of the developers and partnerships in the development of DNA vaccines as a next-generation vaccine platform. The analysis revealed that while emerging biopharmaceutical companies and private–private and private–public partnerships were crucial during the COVID-19 pandemic, public organizations and public–public collaborations primarily led to the clinical development of vaccines for other diseases. Strategies for vaccine development using new vaccine technologies should be tailored to the specific characteristics of each disease.

]]> Developer and Partnership Differences in COVID-19 and Other Infections: Insights from DNA Vaccines Ryo Okuyama doi: 10.3390/jmahp12040025 Journal of Market Access & Health Policy 2024-10-29 Journal of Market Access & Health Policy 2024-10-29 12 4 Communication 317 10.3390/jmahp12040025 https://www.mdpi.com/2001-6689/12/4/25