ReviewBMC health services research2023
How are health technology assessment bodies responding to the assessment challenges posed by cell and gene therapy?
Review in BMC health services research, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 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.
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, 1 synthesis or guideline pooled it.
- Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix.Orphanet journal of rare diseases · 2025Pooled it
- Estimating the Potential Benefits of Confirmatory Trials for Drugs with Accelerated Approval: A Comprehensive Value of Information Framework.PharmacoEconomics · 2023Trial
- 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
- Value of information analyses for advanced cell therapies: a systematic review.Health economics review · 2026Review
- CAR-T cell therapies: patient access and affordability solutions.Future science OA · 2025Review
- A scoping review of challenges in pediatric health technology assessments with a focus on pharmaceutical interventions.International journal of technology assessment in health care · 2025Article
- Constraints on delivering cell and gene therapies identified during technology appraisal by the National Institute for Health and Care Excellence.International journal of technology assessment in health care · 2025Article
- Connecting minds and catalyzing collaboration: the interest groups of health technology assessment international.International journal of technology assessment in health care · 2025Review
- Alternative Payment Models for Innovative Medicines: A Framework for Effective Implementation.Applied health economics and health policy · 2025Article
- Clinical trials to gene therapy development and production in Brazil: a review.Lancet regional health. Americas · 2025Review
- Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis.Applied health economics and health policy · 2025Article
- How can actuarial science contribute to the field of health technology assessment? An interdisciplinary perspective.International journal of technology assessment in health care · 2025Review
- Design and Features of Pricing and Payment Schemes for Health Technologies: A Scoping Review and a Proposal for a Flexible Need-Driven Classification.PharmacoEconomics · 2025Article
- A hierarchical and configurational analysis of Health Technology Assessment outcomes for cell and gene therapies.Frontiers in pharmacology · 2025Article
- Advanced Therapy Medicinal Products: Availability, Access and Expenditure in Italy.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Article
- Mixture and Non-mixture Cure Models for Health Technology Assessment: What You Need to Know.PharmacoEconomics · 2024Article
- Mapping Payment and Pricing Schemes for Health Innovation: Protocol of a Scoping Literature Review.PharmacoEconomics - open · 2024Article
- Market Access Challenges and Solutions in Cell and Gene Therapy in The Netherlands.Journal of market access & health policy · 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
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe aims of this research were to provide a better understanding of the specific evidence needs for assessment of clinical and cost-effectiveness of cell and gene therapies, and to explore the extent that the relevant categories of evidence are considered in health technology assessment (HTA) processes.
methodsA targeted literature review was conducted to identify the specific categories of evidence relevant to the assessment of these therapies. Forty-six HTA reports for 9 products in 10 cell and gene therapy indications across 8 jurisdictions were analysed to determine the extent to which various items of evidence were considered.
resultsThe items to which the HTA bodies reacted positively were: treatment was for a rare disease or serious condition, lack of alternative therapies, evidence indicating substantial health gains, and when alternative payment models could be agreed. The items to which they reacted negatively were: use of unvalidated surrogate endpoints, single arm trials without an adequately matched alternative therapy, inadequate reporting of adverse consequences and risks, short length of follow-up in clinical trials, extrapolating to long-term outcomes, and uncertainty around the economic estimates.
conclusionsThe consideration by HTA bodies of evidence relating to the particular features of cell and gene therapies is variable. Several suggestions are made for addressing the assessment challenges posed by these therapies. Jurisdictions conducting HTAs of these therapies can consider whether these suggestions could be incorporated within their existing approach through strengthening deliberative decision-making or performing additional analyses.
Indexed as
Identifiers
What 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.