ArticleJournal of market access & health policy2020
Health economic evaluation of gene replacement therapies: methodological issues and recommendations.
Article in Journal of market access & health policy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers, 2 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
11 citing papers in PubMed, 2 syntheses or guidelines pooled 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
- Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy.Advances in therapy · 2022Pooled it
- Surrogate endpoints in cost-effectiveness analysis for use in health technology assessment - a qualitative analysis.International journal of technology assessment in health care · 2026Article
- 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
- Market Access Challenges and Solutions in Cell and Gene Therapy in The Netherlands.Journal of market access & health policy · 2024Article
- Using automated text classification to explore uncertainty in NICE appraisals for drugs for rare diseases.International journal of technology assessment in health care · 2024Article
- 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
- 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
- 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
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
PubMed holds no abstract for this paper.
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.