ArticlePharmacoEconomics2020
Recalibrating Health Technology Assessment Methods for Cell and Gene Therapies.
Article in PharmacoEconomics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 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
23 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review.PharmacoEconomics · 2024Pooled it
- Capturing the Impact of Constraints on the Cost-Effectiveness of Cell and Gene Therapies: A Systematic Review.PharmacoEconomics · 2023Pooled it
- Economic Evidence on Potentially Curative Gene Therapy Products: A Systematic Literature Review.PharmacoEconomics · 2021Pooled it
- 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
- The impacts of pricing and reimbursement policies on access to cell and gene therapies across Europe.Journal of community genetics · 2026Review
- 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
- Article
- A narrative review of precision and ethical considerations in cardiovascular health: CRISPR-Cas9, telemedicine, and lifestyle interventions.Frontiers in public health · 2025Review
- A hierarchical and configurational analysis of Health Technology Assessment outcomes for cell and gene therapies.Frontiers in pharmacology · 2025Article
- Market Access Challenges and Solutions in Cell and Gene Therapy in The Netherlands.Journal of market access & health policy · 2024Article
- Cost of implementing CAR-T activity and managing CAR-T patients: an exploratory study.BMC health services research · 2024Article
- Deliberative processes in health technology assessment of medicines: the case of Spain.International journal of technology assessment in health care · 2023Review
- Article
- Pricing and reimbursement mechanisms for advanced therapy medicinal products in 20 countries.Frontiers in pharmacology · 2023Article
- Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment.Frontiers in public health · 2022Review
- Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives.Journal of neuromuscular diseases · 2022Review
- Critical Reflections on Reimbursement and Access of Advanced Therapies.Frontiers in pharmacology · 2022Article
- The impact of COVID-19 on the cell and gene therapies industry: Disruptions, opportunities, and future prospects.Drug discovery today · 2021Review
- Evidence generation and reproducibility in cell and gene therapy research: A call to action.Molecular therapy. Methods & clinical development · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Recently licensed cell and gene therapies have promising but highly uncertain clinical benefits. They are entering the market at very high prices, with the latest entrants costing hundreds of thousands of dollars. The significant long-term uncertainty posed by these therapies has already complicated the use of conventional economic evaluation approaches such as cost-effectiveness and cost-utility analyses, which are widely used for assessing the value of new health interventions. Cell and gene therapies also risk jeopardising healthcare systems' financial sustainability. As a result, there is a need to recalibrate the current health technology assessment methods used to measure and compensate their value. In this paper, we outline a set of technical adaptations and methodological refinements to address key challenges in the appraisal of cell and gene therapies' value, including the assessment of efficiency and affordability. We also discuss the potential role of alternative financing mechanisms. Ultimately, uncertainties associated with cell and gene therapies can only be meaningfully addressed by improving the evidence base supporting their approval and adoption in healthcare systems.
Indexed as
Identifiers
32960434What 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.