ArticleNature biotechnology2014
The special case of gene therapy pricing.
Article in Nature biotechnology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 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
32 citing papers in PubMed, 2 syntheses or guidelines 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
- Application of Managed Entry Agreements for Innovative Therapies in Different Settings and Combinations: A Feasibility Analysis.International journal of environmental research and public health · 2020Pooled it
- Innovative Insurance to Improve US Patient Access to Cell and Gene Therapy.The Milbank quarterly · 2025Article
- Comparative policy analysis of national rare disease funding policies in Australia, Singapore, South Korea, the United Kingdom and the United States: a scoping review.Health economics review · 2024Article
- An Ethical and Financial Obligation for Sickle Cell Disease Gene Therapy in the United States.Annals of internal medicine · 2024Article
- The estimated annual financial impact of gene therapy in the United States.Gene therapy · 2023Article
- Individualized interventions for rare genetic conditions and the research-treatment spectrum: Stakeholder perspectives.Genetics in medicine : official journal of the American College of Medical Genetics · 2023Article
- Adeno-associated virus vector system controlling capsid expression improves viral quantity and quality.iScience · 2023Article
- Scaffold-free cell-based tissue engineering therapies: advances, shortfalls and forecast.NPJ Regenerative medicine · 2021Review
- Barriers and Opportunities for Implementation of Outcome-Based Spread Payments for High-Cost, One-Shot Curative Therapies.Frontiers in pharmacology · 2020Review
- The Evolving Landscape of Gene Therapy in Plastic Surgery.Seminars in plastic surgery · 2019Review
- Assessing the Joint Value of Genomic-Based Diagnostic Tests and Gene Therapies.Journal of personalized medicine · 2019Article
- Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Review
- Observational
- Beating the organ clock.Nature biotechnology · 2018Article
- Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned.Cell stem cell · 2017Review
- Challenges in the gene therapy commercial ecosystem.Nature biotechnology · 2017Article
- The state of gene therapy research in Africa, its significance and implications for the future.Gene therapy · 2017Review
- Adaptive Biomedical Innovation: Evolving Our Global System to Sustainably and Safely Bring New Medicines to Patients in Need.Clinical pharmacology and therapeutics · 2016Article
- Genome editing and the next generation of antiviral therapy.Human genetics · 2016Review
Corrections and comments
- Commented on by
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy companies that pursue high, one-time payments for their products risk a backlash from payors. A better solution may lie in a pay-for-performance model.
Indexed as
Identifiers
25203033What 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.