Journal of Market Access & Health Policy

9 pages, 186 KiB

Open AccessPerspective

Towards a Unified European View of Clinical Evidence: What ‘Health Technology Assessment Organizations’ Can Learn from Regulatory Experience

by Karl Broich and Wiebke Löbker

J. Mark. Access Health Policy 2025, 13(2), 19; https://doi.org/10.3390/jmahp13020019 - 28 Apr 2025

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Abstract

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 [...] Read more.

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. Full article

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

5 pages, 182 KiB

Open AccessEditorial

A New Health Networking Infrastructure on Cancer Is Taking Shape in Europe: A Not-to-Miss Opportunity for the EU Regulatory System

by Paolo Giovanni Casali and Stefano Capri

J. Mark. Access Health Policy 2025, 13(2), 18; https://doi.org/10.3390/jmahp13020018 - 28 Apr 2025

Viewed by 91

Abstract

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 [...] Full article

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

16 pages, 1973 KiB

Open AccessArticle

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

by Ekkehard Beck, Keya Joshi, Darshan Mehta, Stephane Lorenc, Bishoy Rizkalla and Nicolas Van de Velde

J. Mark. Access Health Policy 2025, 13(2), 17; https://doi.org/10.3390/jmahp13020017 - 24 Apr 2025

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Abstract

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 [...] Read more.

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. Full article

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14 pages, 1840 KiB

Open AccessOpinion

Strengthening the EU Health Technology Assessment Regulation: Integrating National Immunization Technical Advisory Groups for Comprehensive Vaccine Assessments

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

J. Mark. Access Health Policy 2025, 13(2), 16; https://doi.org/10.3390/jmahp13020016 - 18 Apr 2025

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Abstract

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 [...] Read more.

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. Full article

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

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3 pages, 164 KiB

Open AccessEditorial

Clinical Trial Validity Guidance from the HTACG: Looking for Chicken Teeth

by Mondher Toumi, Bruno Falissard, Asma Jouini, Samuel Aballéa and Laurent Boyer

J. Mark. Access Health Policy 2025, 13(2), 15; https://doi.org/10.3390/jmahp13020015 - 16 Apr 2025

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Abstract

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

9 pages, 195 KiB

Open AccessArticle

ARM’s Perspective on the First Joint Clinical Assessments for ATMPs: Challenges and Opportunities on the Path Ahead

by Paolo Morgese, Stephen Majors and Dilip Patel

J. Mark. Access Health Policy 2025, 13(2), 14; https://doi.org/10.3390/jmahp13020014 - 3 Apr 2025

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Abstract

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 [...] Read more.

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. Full article

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

11 pages, 216 KiB

Open AccessArticle

Healthcare Professionals’ Perceptions About Medical Cannabis in Greece: A Qualitative Study

by Christos Ntais, Yioula Melanthiou and Michael A. Talias

J. Mark. Access Health Policy 2025, 13(2), 13; https://doi.org/10.3390/jmahp13020013 - 2 Apr 2025

Viewed by 385

Abstract

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 [...] Read more.

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. Full article

17 pages, 2599 KiB

Open AccessArticle

A Methodological Proposal for Health Technology Assessments: A Case Study on Biosimilar Drugs

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

J. Mark. Access Health Policy 2025, 13(2), 12; https://doi.org/10.3390/jmahp13020012 - 31 Mar 2025

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Abstract

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 [...] Read more.

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. Full article

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18 pages, 1484 KiB

Open AccessArticle

The Cost-Effectiveness of Avatrombopag Versus Eltrombopag and Romiplostim in the Treatment of Patients with Immune Thrombocytopenia in the UK

by Nichola Cooper, Sebastian Guterres, Michał Pochopień, Koo Wilson, Sam James, Mondher Toumi, Anna Tytuła, Carly Rich and Daniel Eriksson

J. Mark. Access Health Policy 2025, 13(2), 11; https://doi.org/10.3390/jmahp13020011 - 24 Mar 2025

Viewed by 412

Abstract

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 [...] Read more.

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. Full article

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J. Mark. Access Health Policy, Volume 13, Issue 2 (June 2025) – 9 articles

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