ArticleThe European journal of health economics : HEPAC : health economics in prevention and care2020
Advanced therapy medicinal products: value judgement and ethical evaluation in health technology assessment.
Article in The European journal of health economics : HEPAC : health economics in prevention and care, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 1 of them a synthesis 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.
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Who cites it
17 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Real-world data: a comprehensive literature review on the barriers, challenges, and opportunities associated with their inclusion in the health technology assessment process.Journal of pharmacy & pharmaceutical sciences : a publication of the Canadian Society for Pharmaceutical Sciences, Societe canadienne des sciences pharmaceutiques · 2024Pooled it
- Pricing of innovative medicines and advanced therapies worldwide: an scoping review.The European journal of health economics : HEPAC : health economics in prevention and care · 2026Article
- Pathing the way from regulatory approval to market access for gene therapy products (GTPs): An integrative review in the US, EU5, Japan and China.Regenerative therapy · 2026Review
- Connecting minds and catalyzing collaboration: the interest groups of health technology assessment international.International journal of technology assessment in health care · 2025Review
- Cartilage repair: unleashing PRP's potential in organoid models.Cytotechnology · 2025Review
- Validation of the FROM-16 in family members of patients receiving advanced therapy medicinal product (ATMP).Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025Article
- Advanced Therapy Medicinal Products as Potential Therapeutic Strategy against COVID-19 and Immune-Related Disorders.International journal of molecular sciences · 2024Article
- Cost of implementing CAR-T activity and managing CAR-T patients: an exploratory study.BMC health services research · 2024Article
- How are health technology assessment bodies responding to the assessment challenges posed by cell and gene therapy?BMC health services research · 2023Review
- Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.Pharmaceutics · 2022Review
- Evaluation of Advanced Therapy Medicinal Products by the National Institute for Health and Care Excellence (NICE): An Updated Review.PharmacoEconomics - open · 2022Review
- Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives.Journal of neuromuscular diseases · 2022Review
- Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment.Frontiers in public health · 2022Review
- Ethical Challenges in Organoid Use.Biotech (Basel (Switzerland)) · 2021Article
- Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision.PharmacoEconomics · 2021Review
- Regenerative medicine clinical readiness.Regenerative medicine · 2021Review
- Development and Use of Gene Therapy Orphan Drugs-Ethical Needs for a Broader Cooperation Between the Pharmaceutical Industry and Society.Frontiers in medicine · 2020Article
Corrections and comments
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Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Advanced therapy medicinal products (ATMPs) are a heterogeneous class of medicinal products that by offering the potential of cure represent a paradigm shift in the approach of many life-threatening diseases. Although a common regulatory framework for ATMPs has been established in the EU, the health technology assessment (HTA) and financing decisions remain local. The aim of this article is to present an integrated analysis of the current status of the value judgment of ATMPs and the integration of ethical evaluation in the HTA process. It has been identified that approaching the specificities of ATMPs in terms of market access will require a broadening of the definition of value to be able to systematically capture elements of value not traditionally considered. Outcomes modelling will play an important role in the pricing and reimbursement of ATMPs, providing a way to bridge the gap caused by the absence of data from clinical studies or real-world data. Given the nature and disruptive consequences of ATMPs the assessment and adoption of these medicinal products raises important ethical questions, both at a policy and at society level that should be properly addressed. HTA can be made more transparent and reliable, and simultaneously promote robust and accountable decision making, by turning explicit the value judgments implicit in HTA. Ultimately, there should be no core conflict between ethical requirements and HTA in a scenario where the goal is to promote equity and access of patients to truly innovative therapies such as ATMPs, while assuring the sustainability of healthcare systems.
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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.