ArticleThe European journal of health economics : HEPAC : health economics in prevention and care2020
HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies.
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 37 papers, 4 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
37 citing papers in PubMed, 4 syntheses or guidelines pooled it.
- Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review.PharmacoEconomics · 2024Pooled it
- A systematic review on the appropriate discounting rates for the economic evaluation of gene therapies: whether a specific approach is justified to tackle the challenges?International journal of technology assessment in health care · 2024Pooled it
- A systematic review of economic evaluations forInternational journal of technology assessment in health care · 2023Pooled it
- Capturing the Impact of Constraints on the Cost-Effectiveness of Cell and Gene Therapies: A Systematic Review.PharmacoEconomics · 2023Pooled it
- Patient, consumer and societal values, perceptions and preferences on high-cost gene therapies.Gene therapy · 2026Article
- Surrogate endpoints in cost-effectiveness analysis for use in health technology assessment - a qualitative analysis.International journal of technology assessment in health 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
- Harnessing health economic evaluation for policy and practice.Communications medicine · 2026Review
- Value of information analyses for advanced cell therapies: a systematic review.Health economics review · 2026Review
- The impacts of pricing and reimbursement policies on access to cell and gene therapies across Europe.Journal of community genetics · 2026Review
- The economic impact of retinal diseases for which gene therapy is emerging: a systematic literature review.Health economics review · 2025Review
- Market Access Challenges and Solutions in Cell and Gene Therapy in The Netherlands.Journal of market access & health policy · 2024Article
- Opportunities and Challenges in Cross-Country Collaboration: Insights from the Beneluxa Initiative.Journal of market access & health policy · 2024Article
- Challenges for gene therapy in the financial sustainability of health systems: a scoping review.Orphanet journal of rare diseases · 2024Article
- A situational and stakeholder analysis of health technology assessment in Zimbabwe.International journal of technology assessment in health care · 2024Article
- Navigating Gene Therapy Access: The Case of Bulgaria in the Context of the EU Regulatory Landscape.Healthcare (Basel, Switzerland) · 2024Article
- Real-world evidence for coverage determination of treatments for rare diseases.Orphanet journal of rare diseases · 2024Article
- Cost of implementing CAR-T activity and managing CAR-T patients: an exploratory study.BMC health services research · 2024Article
- Unraveling elements of value-based pricing from a pharmaceutical industry's perspective: a scoping review.Frontiers in pharmacology · 2024Article
- Global Trends on β-Thalassemia Research Over 10 Years: A Bibliometric Analysis.International journal of general medicine · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
16 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
This last decade has been marked by significant advances in the development of cell and gene (C&G) therapies, such as gene targeting or stem cell-based therapies. C&G therapies offer transformative benefits to patients but present a challenge to current health technology decision-making systems because they are typically reviewed when clinical efficacy data are very limited and when there is uncertainty about the long-term durability of outcomes. These challenges are not unique to C&G therapies, but they face more of these barriers, reflecting the need for adapting existing value assessment frameworks. Still, C&G therapies have the potential to be cost-effective even at very high price points. The impact on healthcare budgets will depend on the success rate of pipeline assets and on the extent to which C&G therapies will expand to wider pathologies beyond rare or ultra-rare diseases. Getting pricing and reimbursement models right is important for incentivising research and development investment while not jeopardising the sustainability of healthcare systems. Payers and manufacturers therefore need to acknowledge each other's constraints-limitations in the evidence generation on the manufacturer side, budget considerations on the payer side-and embrace innovative thinking and approaches to ensure timely delivery of therapies to patients. Several experts in health technology assessment and clinical experts have worked together to produce this publication and identify methodological and policy options to improve the assessment of C&G therapies, and make it happen better, faster and sustainably in the coming years.
Indexed as
Identifiers
32794011What OpenQuestion holds
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.