There are currently no available treatment options for more than 90% of the approximately 7000 rare diseases identified to date. However, if these conditions result from correctable cellular or genetic defects, cell and gene therapies (CGTs) can significantly improve patients' quality of life and often represent their only hope for betterment of their condition. The catch is that CGTs come at a substantial cost to both payers and patients.
In the United States, 46 CGTs were FDA-approved by May 2025. CGTs are expected to generate $24 billion in US annual list price revenues by 2030.
In a viewpoint article published in JAMA Internal Medicine today (Oct. 20, 2025), researchers Audun Brendbekken, MD (University of Bergen, Norway) and Stacie Dusetzina, PhD (Vanderbilt University, USA) discuss the current status of regulatory approval and reimbursement for CGTs in the US and Europe.
Given the US's fragmented health insurance payer system, these therapies cause extremely high up-front costs per patient. The US urgently needs a new strategy for insurance coverage and access to these new CGT treatments as more and more of them are being developed, with very promising early results. In this process, the authors suggest that the US can learn from Europe.
In Europe, 19 CGTs are currently approved by the European Medicines Agency and in most western European countries, the majority of CGTs are reimbursed within two years of approval. In most nations, coverage and reimbursement decisions are based on the demonstrated added therapeutic value of a new drug compared to the existing standard of care. Contracts are then renegotiated based on uniform pricing, lowering per-patient prices.
In the US, on the other hand, CGTs with high up-front costs may not be covered because many patients frequently change insurers in the private insurance market and states face budget constraints for those covered by Medicaid.
The authors advocate for a nationwide single-payer system in the US, especially for high-cost therapies of short duration, which could reduce inequality in terms of access and strengthen price negotiation opportunities. For CGT manufacturers, a single-payer system could pay off in terms of greater predictability and a more robust market. Of course, important questions regarding funding and eligibility in such a system would remain and more research is needed. However, the authors emphasize that action is urgently needed to ensure equitable access to live-saving treatments to rare disease patients at a reasonable cost.
