Journal of Market Access & Health Policy

17 pages, 465 KB

Open AccessArticle

Mapping the Use of Real-World Evidence Across the EU Health Technology Assessment Regulation: Methodological Considerations, Challenges, and Opportunities for Harmonization

by Grammati Sarri, Bengt Liljas, Keith R. Abrams, Stephen J. Duffield and Murtuza Bharmal

J. Mark. Access Health Policy 2026, 14(2), 20; https://doi.org/10.3390/jmahp14020020 - 8 Apr 2026

Abstract

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 [...] Read more.

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. Full article

(This article belongs to the Collection European Health Technology Assessment (EU HTA))

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9 pages, 641 KB

Open AccessPerspective

When Activism Becomes Survival: The Mental Health Costs of Constant Resistance in the Digital Era in the Balkans—A Health Policy Perspective

by Aleksandar Sič, Svetozar Mijuskovic and Nebojsa Brezic

J. Mark. Access Health Policy 2026, 14(2), 19; https://doi.org/10.3390/jmahp14020019 - 2 Apr 2026

Abstract

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 [...] Read more.

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. Full article

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18 pages, 1191 KB

Open AccessArticle

Cost-Effectiveness of 3D-Printed Patient-Specific Versus Off-the-Shelf Interbody Cages in Lumbar Spinal Fusion: A Markov Model Cost-Utility Analysis

by Jackson C. Hill, Ralph J. Mobbs, Marc Coughlan, Kevin A. Seex, Chloe A. Amaro, William R. Walsh and William C. H. Parr

J. Mark. Access Health Policy 2026, 14(2), 18; https://doi.org/10.3390/jmahp14020018 - 25 Mar 2026

Abstract

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 [...] Read more.

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. Full article

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17 pages, 274 KB

Open AccessArticle

Comparative Cost Evaluation of Managed Entry Agreement Techniques Using Real-World Data from High-Cost Anticancer Drugs in Thailand

by Piyapat Owat, Chaoncin Sooksriwong, Hataiwan Ratanabunjerdkul and Tuangrat Phodha

J. Mark. Access Health Policy 2026, 14(1), 17; https://doi.org/10.3390/jmahp14010017 - 20 Mar 2026

Abstract

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 [...] Read more.

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. Full article

14 pages, 502 KB

Open AccessArticle

Validation of a De Novo Health Economic Model for Finerenone in Heart Failure with Left Ventricular Ejection Fraction ≥40%

by Tobiasz Lemański, Kerstin Folkerts, Phil McEwan, Paul Mernagh, Mateusz Robert Żemojdzin and Michał Pochopień

J. Mark. Access Health Policy 2026, 14(1), 16; https://doi.org/10.3390/jmahp14010016 - 11 Mar 2026

Abstract

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 + [...] Read more.

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. Full article

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16 pages, 761 KB

Open AccessArticle

Addressing Unmet Medical Needs in Drug Development: Assessment and Implications for Regulatory and Clinical Development Strategies

by Carla Domingo-Esteban, Inka Heikkinen and Nanco Hefting

J. Mark. Access Health Policy 2026, 14(1), 15; https://doi.org/10.3390/jmahp14010015 - 9 Mar 2026

Abstract

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 [...] Read more.

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. Full article

(This article belongs to the Collection European Health Technology Assessment (EU HTA))

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16 pages, 293 KB

Open AccessPerspective

Can We Trust PAICs in Rare Diseases? Methodological Challenges and Limitations

by Mikolaj Parkitny, Samuel Aballéa, Piotr Wojciechowski and Mondher Toumi

J. Mark. Access Health Policy 2026, 14(1), 14; https://doi.org/10.3390/jmahp14010014 - 6 Mar 2026

Abstract

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 [...] Read more.

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. Full article

22 pages, 1075 KB

Open AccessArticle

Structural Enablers of Rare Disease Treatment Coverage in Latin America and the Caribbean: Lessons from Emicizumab

by Daniela Sugg Herrera, Dino Sepúlveda Viveros, Moisés Russo Namias and Natalia Garrido

J. Mark. Access Health Policy 2026, 14(1), 13; https://doi.org/10.3390/jmahp14010013 - 25 Feb 2026

Abstract

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 [...] Read more.

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. Full article

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9 pages, 404 KB

Open AccessArticle

Updated Conversion Table for the Multiple Criteria Qualitative Value-Based Pricing Framework “MARIE”

by Akina Takami and Ataru Igarashi

J. Mark. Access Health Policy 2026, 14(1), 12; https://doi.org/10.3390/jmahp14010012 - 25 Feb 2026

Abstract

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 [...] Read more.

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. Full article

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15 pages, 750 KB

Open AccessOpinion

Optimising Investment in Health Innovations in Europe

by Tosin Adeyemo, Tim Wilsdon, Christina Vandorou, Fiona Davies and Annabelle Godet

J. Mark. Access Health Policy 2026, 14(1), 11; https://doi.org/10.3390/jmahp14010011 - 13 Feb 2026

Abstract

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 [...] Read more.

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. Full article

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19 pages, 1676 KB

Open AccessArticle

Adopting a Societal Perspective in Health-Economic Evaluation: Analysis of Nine HTA Methodological Guidelines on How to Integrate Societal Costs

by Chloé Gervès-Pinquié, Hortense Nanoux, Sarah Akarkoub, Rémi Brazeilles, Henri Bonnabau and Katell Le Lay

J. Mark. Access Health Policy 2026, 14(1), 10; https://doi.org/10.3390/jmahp14010010 - 10 Feb 2026

Abstract

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 [...] Read more.

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. Full article

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17 pages, 923 KB

Open AccessArticle

Pulmonary Arterial Hypertension and Cancer: Unveiling Parallels in Epidemiology, Clinical Pathways, and Therapeutic Strategies

by Karim El-Kersh, Nadine Zawadzki, Catelyn Coyle, Shurui Zhang, Dhruv Dalal, Anna Watzker, Dominik Lautsch and Jason Shafrin

J. Mark. Access Health Policy 2026, 14(1), 9; https://doi.org/10.3390/jmahp14010009 - 6 Feb 2026

Abstract

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 [...] Read more.

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. Full article

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24 pages, 848 KB

Open AccessArticle

A Cost-Effectiveness Analysis of the Sentio Bone Conduction Hearing Implant System in the Australian Healthcare Setting

by Magnus Värendh, Ida Haggren, Helén Lagerkvist, Maria Åberg Håkansson and Jonas Hjelmgren

J. Mark. Access Health Policy 2026, 14(1), 8; https://doi.org/10.3390/jmahp14010008 - 27 Jan 2026

Abstract

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 [...] Read more.

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. Full article

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13 pages, 949 KB

Open AccessArticle

Evaluating [¹⁸F]-DCFPyL for Detecting Prostate Cancer Recurrence: A Cost–Consequence Comparison with Alternative PET Radiotracers in Spain

by Tiago Matos, Mrunmayee Godbole, Rithvik Badinedi, Madhusubramanian Muthukumar, Marina Hodolic, Nicolas Tchouen and Anthony Berthon

J. Mark. Access Health Policy 2026, 14(1), 7; https://doi.org/10.3390/jmahp14010007 - 23 Jan 2026

Abstract

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, [...] Read more.

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. Full article

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12 pages, 2457 KB

Open AccessArticle

Enhancing Patient-Centered Health Technology Assessment: A Modified Delphi Panel for PICOS Scoping in Spinal Muscular Atrophy

by Emanuele Arcà, Adele Barlassina, Adaeze Eze and Valentina Strammiello

J. Mark. Access Health Policy 2026, 14(1), 6; https://doi.org/10.3390/jmahp14010006 - 19 Jan 2026

Abstract

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 [...] Read more.

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. Full article

(This article belongs to the Collection European Health Technology Assessment (EU HTA))

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15 pages, 1087 KB

Open AccessArticle

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

by 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 and Jörg Ruof Show full author list Hide full author list

J. Mark. Access Health Policy 2026, 14(1), 5; https://doi.org/10.3390/jmahp14010005 - 15 Jan 2026

Abstract

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 [...] Read more.

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. Full article

(This article belongs to the Collection European Health Technology Assessment (EU HTA))

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8 pages, 1288 KB

Open AccessCommunication

Conformity Assessment of Medical Devices: An Overview from a Notified Body

by Andreas F. Stange and Elaine Julian

J. Mark. Access Health Policy 2026, 14(1), 4; https://doi.org/10.3390/jmahp14010004 - 8 Jan 2026

Abstract

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 [...] Read more.

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. Full article

(This article belongs to the Collection European Health Technology Assessment (EU HTA))

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15 pages, 2221 KB

Open AccessArticle

European Joint Clinical Assessment PICO Scoping Process: Analysis of Current Approaches and Recommendations

by Kalpana D’Oca, Eline Darquennes, Chloé Garrigues, Aristeidis Draganigos and Natalie Steck

J. Mark. Access Health Policy 2026, 14(1), 3; https://doi.org/10.3390/jmahp14010003 - 29 Dec 2025

Abstract

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 [...] Read more.

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. Full article

(This article belongs to the Collection European Health Technology Assessment (EU HTA))

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21 pages, 2070 KB

Open AccessArticle

Overcoming Patient Access Barriers in Complex Conditions: Lessons from Schizophrenia for Broader Healthcare Applications

by Saartje Burgmans, Anne Rieper Hald, Sayagi Tina Markandaier, Nicolas Hall, Rafael Loiseau, Xandra Lie and Bregt Kappelhoff

J. Mark. Access Health Policy 2026, 14(1), 2; https://doi.org/10.3390/jmahp14010002 - 23 Dec 2025

Abstract

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, [...] Read more.

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. Full article

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