ArticleJournal of market access & health policy2015
Reimbursement of licensed cell and gene therapies across the major European healthcare markets.
Article in Journal of market access & health policy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
25 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Financing and Reimbursement Models for Personalised Medicine: A Systematic Review to Identify Current Models and Future Options.Applied health economics and health policy · 2022Pooled it
- Defining and Measuring the Affordability of New Medicines: A Systematic Review.PharmacoEconomics · 2017Pooled it
- Preferences for Advanced Therapy Medicinal Products: Understanding the Published Literature on the Value of Innovative Health Interventions.Inquiry : a journal of medical care organization, provision and financingPooled it
- Reimbursement routes and past practices for advanced therapy medicinal products in the Netherlands.Regenerative medicine · 2025Article
- Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union.Therapeutic innovation & regulatory science · 2025Article
- Managed Entry Agreements for High-Cost, One-Off Potentially Curative Therapies: A Framework and Calculation Tool to Determine Their Suitability.PharmacoEconomics · 2025Article
- Development of an multicriteria decision analysis framework for rare disease reimbursement prioritization in Malaysia.International journal of technology assessment in health care · 2024Article
- Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec.Molecular therapy. Methods & clinical development · 2023Review
- "I just wanted to speak to someone- and there was no one…": using Burden of Treatment Theory to understand the impact of a novel ATMP on early recipients.Orphanet journal of rare diseases · 2023Article
- When Reality Does Not Meet Expectations-Experiences and Perceived Attitudes of Dutch Stakeholders Regarding Payment and Reimbursement Models for High-Priced Hospital Drugs.International journal of environmental research and public health · 2022Article
- Value-based pricing for advanced therapy medicinal products: emerging affordability solutions.The European journal of health economics : HEPAC : health economics in prevention and care · 2022Article
- Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A.HemaSphere · 2022Article
- Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision.PharmacoEconomics · 2021Review
- Do Advanced Therapies Have a Future in the Low- and Middle-Income Countries - The Case of Bulgaria, Romania, and Poland.Frontiers in public health · 2021Review
- Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products.Frontiers in medicine · 2021Article
- Mitigating Deficiencies in Evidence during Regulatory Assessments of Advanced Therapies: A Comparative Study with Other Biologicals.Molecular therapy. Methods & clinical development · 2020Article
- Advanced therapy medicinal products: value judgement and ethical evaluation in health technology assessment.The European journal of health economics : HEPAC : health economics in prevention and care · 2020Article
- Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries.Journal of market access & health policy · 2020Article
- Assessing the Preferences for Criteria in Multi-Criteria Decision Analysis in Treatments for Rare Diseases.Frontiers in public health · 2020Article
- Barriers and Opportunities for Implementation of Outcome-Based Spread Payments for High-Cost, One-Shot Curative Therapies.Frontiers in pharmacology · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveThe aim of this research is to identify the pricing, reimbursement, and market access (P&R&MA) considerations most relevant to advanced therapy medicinal products (ATMPs) in the Big5EU, and to inform their manufacturers about the key drivers for securing adoption at a commercially viable reimbursed price. METHODOLOGY: The research was structured following three main steps: 1) Identifying the market access pathways relevant to ATMPs through secondary research; 2) Validating the secondary research findings and addressing any data gaps in primary research, by qualitative interviews with national, regional, and local-level payers and their clinical and economic advisors; 3) Collating of primary and secondary findings to compare results across countries.
resultsThe incremental clinical benefit forms the basis for all P&R&MA processes. Budget impact is a key consideration, regardless of geography. Cost-effectiveness analyses are increasingly applied; however, only the United Kingdom has a defined threshold that links the cost per quality-adjusted life year (QALY) specifically and methodologically to the reimbursed price. Funding mechanisms to enable adoption of new and more expensive therapies exist in all countries, albeit to varying extents. Willingness to pay is typically higher in smaller patient populations, especially in populations with high disease burden. Outcomes modelling and risk-sharing agreements (RSAs) provide strategies to address the data gap and uncertainties often associated with trials in niche populations.
conclusionsThe high cost of ATMPs, coupled with the uncertainty at launch around their long-term claims, present challenges for their adoption at a commercially viable reimbursed price. Targeting populations of high disease burden and unmet needs may be advantageous, as the potential for improvement in clinical benefit is greater, as well as the potential for capitalising on healthcare cost offsets. Also, targeting small populations can also help reduce both payers' budget impact concerns and the risk of reimbursement restrictions being imposed.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.