ArticleThe European journal of health economics : HEPAC : health economics in prevention and care2017
Why do health technology assessment coverage recommendations for the same drugs differ across settings? Applying a mixed methods framework to systematically compare orphan drug decisions in four European countries.
Article in The European journal of health economics : HEPAC : health economics in prevention and care, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers, 2 of them syntheses that pooled it.
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Who cites it
31 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- HTA Barriers for Conditional Approval Drugs.PharmacoEconomics · 2023Pooled it
- Multi-criteria decision analysis for health technology assessment: addressing methodological challenges to improve the state of the art.The European journal of health economics : HEPAC : health economics in prevention and care · 2019Pooled it
- The Post-Authorisation Maze: Mapping of European Pharmaceutical Market Access Pathways in the Early JCA Era.Journal of market access & health policy · 2026Article
- Procedural Theatre: EU-HTA Between Ritual and Purpose-Why the Buyer's Audit Has Outgrown the Transaction.Journal of market access & health policy · 2026Article
- What Drives Public Preference for Rare Drugs Coverage in China? Insights From a Multi-Center Discrete Choice Experiment.Health expectations : an international journal of public participation in health care and health policy · 2026Article
- Using a lottery to resolve indeterminacy when allocating resources for drugs for rare diseases.Journal of medical ethics · 2026Article
- Access to Orphan Medicinal Products in Bulgaria: An Analysis of the Positive Drug List and Individual Access Schemes.Healthcare (Basel, Switzerland) · 2025Article
- Stakeholder survey about broad elements of value in health technology assessment in Australia: industry and academia more similar than different.International journal of technology assessment in health care · 2025Article
- Article
- How can health technology assessment be improved to optimise access to medicines? Results from a Delphi study in Europe : Better access to medicines through HTA.The European journal of health economics : HEPAC : health economics in prevention and care · 2024Article
- Navigating the path towards successful implementation of the EU HTA Regulation: key takeaways from the 2023 Spring Convention of the European Access Academy.Health research policy and systems · 2024Article
- Are We Capturing the Socioeconomic Burden of Rare Genetic Disease? A Scoping Review of Economic Evaluations and Cost-of-Illness Studies.PharmacoEconomics · 2023Article
- Developing a Framework of Cost Elements of Socioeconomic Burden of Rare Disease: A Scoping Review.PharmacoEconomics · 2023Article
- The Beneluxa Initiative domain task force health technology assessment: a comparison of member countries' past health technology assessments.International journal of technology assessment in health care · 2023Article
- The role of drug regulatory authorities and health technology assessment agencies in shaping incentives for antibiotic R&D: a qualitative study.Journal of pharmaceutical policy and practice · 2023Article
- The reimbursement process in three national healthcare systems: variation in time to reimbursement of pembrolizumab for metastatic non-small cell lung cancer.Journal of pharmaceutical policy and practice · 2023Review
- Challenges and Opportunities for Companies to Build HTA/Payer Perspectives Into Drug Development Through the Use of a Dynamic Target Product Profile.Frontiers in pharmacology · 2022Article
- Using 5 consecutive years of NICE guidance to describe the characteristics and influencing factors on the economic evaluation of orphan oncology drugs.Frontiers in public health · 2022Article
- Non-Price-Related Determinants of Value and Access for Novel Non-small Cell Lung Cancer Treatments: A Cross-Country Review of HTA Decision Making.PharmacoEconomics - open · 2021Article
- Associations between uncertainties identified by the European Medicines Agency and national decision making on reimbursement by HTA agencies.Clinical and translational science · 2021Article
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Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeHealth technology assessment (HTA) coverage recommendations differ across countries for the same drugs. Unlike previous studies, this study adopts a mixed methods research design to investigate, in a systematic manner, these differences.
methodsHTA recommendations for ten orphan drugs appraised in England (NICE), Scotland (SMC), Sweden (TLV) and France (HAS) (N = 35) were compared using a validated methodological framework that breaks down these complex decision processes into stages facilitating their understanding, analysis and comparison, namely: (1) the clinical/cost-effectiveness evidence, (2) its interpretation (e.g. part of the deliberative process) and (3) influence on the final decision. This allowed qualitative and quantitative identification of the criteria driving recommendations and highlighted cross-country differences.
resultsSix out of ten drugs received diverging HTA recommendations. Reasons for cross-country differences included heterogeneity in the evidence appraised, in the interpretation of the same evidence, and in the different ways of dealing with the same uncertainty. These may have been influenced by agency-specific evidentiary, risk and value preferences, or stakeholder input. "Other considerations" (e.g. severity, orphan status) and other decision modulators (e.g. patient access schemes, lower discount rates, restrictions, re-assessments) also rendered uncertainty and cost-effectiveness estimates more acceptable. The different HTA approaches (clinical versus cost-effectiveness) and ways identified of dealing with orphan drug particularities also had implications on the final decisions.
conclusionsThis research contributes to better understanding the drivers of these complex decisions and why countries make different decisions. It also contributed to identifying those factors beyond the standard clinical and cost-effectiveness tools used in HTA, and their role in shaping these decisions.
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