ReviewJournal of comparative effectiveness research2024
Improving access to gene therapies: a holistic view of current challenges and future policy solutions in the United States.
Review in Journal of comparative effectiveness research, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 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
6 citing papers in PubMed.
- Transplantation as disease modifying therapy in the era of gene therapy medicinal products - health policy considerations.Orphanet journal of rare diseases · 2026Review
- Innovative Contracting for Gene Therapies: Current Landscape and Perspectives on the Future of Gene Therapy Financing in the USA.PharmacoEconomics · 2026Review
- When Do Annuity-Based Payments Help to Address the Affordability Challenge of Funding Advanced Therapies? Insights from a Budget Impact Simulation.Journal of market access & health policy · 2026Article
- Expert assessment of the attributes of high-cost medications: a case study of CAR T-cell therapy in the gulf cooperation council.Frontiers in pharmacology · 2026Article
- Emerging Therapies for Sickle Cell Disease: From Symptom Management to Curative Gene Therapy.Cureus · 2025Review
- A hierarchical and configurational analysis of Health Technology Assessment outcomes for cell and gene therapies.Frontiers in pharmacology · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapies hold the promise of delivering groundbreaking improvements in health outcomes for previously intractable diseases. These therapies possess unique characteristics that differ from traditional small molecule or biologic treatments, posing new challenges for healthcare stakeholders. While previous analyses have predominantly focused on the payment challenges associated with emerging gene therapies, a more holistic examination reveals numerous obstacles that currently hinder optimal patient access. In the United States (US), these include production and distribution issues, logistical and treatment administration challenges and coverage and reimbursement limitations imposed by US healthcare payers. Several opportunities exist to address these challenges and improve patient access, including the use of appropriate value assessment methods, the development of innovative payment solutions, the removal of inappropriate access barriers imposed by payers and improving education and awareness regarding treatment availability, benefits and risks. Moving forward, we must collectively strive for comprehensive policy and market solutions that are sensitive to the uniqueness of gene therapies to ensure their full potential is realized and these treatments are made available to those in need.
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.