ArticleMolecular therapy. Methods & clinical development2025
Ensuring patient access to gene therapies for rare diseases: Navigating reimbursement and coverage challenges.
Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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.
- rAAV production cost analysis: Indication-specific cost per dose and reduction strategies.Gene therapy · 2026Article
- Principles and practices for successful gene therapy innovative contracting: Insights from a multistakeholder convening.Journal of managed care & specialty pharmacy · 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
- Review
- Innovative Contracting for Gene Therapies: Current Landscape and Perspectives on the Future of Gene Therapy Financing in the USA.PharmacoEconomics · 2026Review
- Assessing the Early Economic Feasibility of a Curative Gene Therapy for Multiple Sclerosis Using a Risk-Adjusted Valuation Framework.Healthcare (Basel, Switzerland) · 2026Article
- Gene modification: Exploring the potential in treating kidney diseases.Pharmacological research · 2026Review
- Emerging polymeric nanocarriers for mRNA and protein therapeutics: design, challenges, and clinical outlook.Nanomedicine (London, England) · 2025Review
- Erratum: Characteristics and Comorbidities Influencing Mortality Risk Among Hereditary Angioedema Patients.Journal of health economics and outcomes research · 2025Article
- Optimizing regulatory frameworks for gene therapies in rare diseases: Challenges and solutions.Molecular therapy. Methods & clinical development · 2024Article
- Institutional readiness for novel therapeutics: A framework for multidisciplinary integration.Molecular genetics and metabolismReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The rapid transformation in rare disease treatment, driven by advances in genetic medicine and diagnostics, underscores the urgent need for access to these innovative therapies. With over 10,000 identified rare diseases globally, 80% of which are genetic, the current therapeutic landscape indicates that only 5% of these conditions have FDA-approved treatments. This article examines the critical logistical challenges in commercializing and paying for gene therapies for rare diseases. It highlights the importance of considering innovative payment models, addressing patient portability issues, and aligning payer coverage policies with FDA-approved indications. It emphasizes the need to account for the broader value of gene therapies, incorporate input from disease-specific clinical experts in payer coverage decisions, and reduce administrative barriers to coverage. By adopting a multifaceted approach, we can foster a more supportive environment for the sustainable delivery of gene therapies, significantly improving the lives of patients with rare genetic disorders while rewarding and driving continued innovation.
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.