Journal of Market Access & Health Policy

11 pages, 208 KiB

Open AccessReview

Patient Involvement in Health Technology Assessments: Lessons for EU Joint Clinical Assessments

by Anne-Pierre Pickaert

J. Mark. Access Health Policy 2025, 13(3), 38; https://doi.org/10.3390/jmahp13030038 - 28 Jul 2025

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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 [...] Read more.

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. Full article

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

11 pages, 235 KiB

Open AccessArticle

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?

by Claudio Jommi, Marzia Bonfanti, Melissa Guardigni, Andrea Aiello, Andrea Marcellusi, Pier Luigi Canonico, Fulvio Luccini and Chiara Lucchetti

J. Mark. Access Health Policy 2025, 13(3), 37; https://doi.org/10.3390/jmahp13030037 - 25 Jul 2025

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Abstract

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 [...] Read more.

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. Full article

15 pages, 1045 KiB

Open AccessArticle

Physician Practice Affiliation Drives Site of Care Cost Differentials: An Opportunity to Reduce Healthcare Expenditures

by Deepak A. Kapoor, Mark Camel, David Eagle, Lauren C. Makhoul, Justin Maroney, Zhou Yang and Paul Berggreen

J. Mark. Access Health Policy 2025, 13(3), 36; https://doi.org/10.3390/jmahp13030036 - 24 Jul 2025

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Abstract

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 [...] Read more.

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. Full article

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19 pages, 424 KiB

Open AccessArticle

Preparing for the EU HTA Regulation: Insights from the Dutch Perspective

by Anne Willemsen, Maureen Rutten-van Mölken, Riam al Dulaimi, Hedi Schelleman, Wim Goettsch and Lonneke Timmers

J. Mark. Access Health Policy 2025, 13(3), 35; https://doi.org/10.3390/jmahp13030035 - 24 Jul 2025

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Abstract

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 [...] Read more.

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. Full article

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

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11 pages, 496 KiB

Open AccessArticle

An Estimation of the Economic and Environmental Impact of Inhaler Devices Switch for Non-Clinical Reasons in COPD and Asthma: The Case for Spain

by Oriol Solà-Morales, Joan B Soriano, Míriam Solozabal-Coll and Jose Vicente Galindo

J. Mark. Access Health Policy 2025, 13(3), 34; https://doi.org/10.3390/jmahp13030034 - 17 Jul 2025

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Abstract

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 [...] Read more.

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. Full article

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4 pages, 168 KiB

Open AccessEditorial

EU-HTA Guidance for Clinical Validity: Misconceptions and Flawed Processes

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

J. Mark. Access Health Policy 2025, 13(3), 33; https://doi.org/10.3390/jmahp13030033 - 15 Jul 2025

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Abstract

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 [...] Full article

21 pages, 615 KiB

Open AccessArticle

The PICO Puzzle: Can Public Data Predict EU HTA Expectations for All EU Countries?

by Karolin Eberle, Lisa-Maria Hagemann, Maria Katharina Schweitzer, Martin Justl, Jana Maurer, Alexandra Carls and Eva-Maria Reuter

J. Mark. Access Health Policy 2025, 13(3), 32; https://doi.org/10.3390/jmahp13030032 - 26 Jun 2025

Viewed by 507

Abstract

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, [...] Read more.

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. Full article

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

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J. Mark. Access Health Policy, Volume 13, Issue 3 (September 2025) – 7 articles

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