WASHINGTON, Feb 23 (Reuters) –The U.S. Food and Drug Administration (FDA) has proposed a new regulatory framework aimed at accelerating approvals for personalized treatments targeting rare and life-threatening genetic diseases. Announced on February 23, the draft guidance would allow drugmakers to rely on small, well-controlled studies when traditional large-scale randomized trials are not feasible due to extremely limited patient populations.
The proposal updates approval standards for individualized genetic medicines, creating a clearer pathway for genome-editing and RNA-based therapies developed for ultra-rare conditions. Under the framework, companies could seek approval based on early efficacy signals and strong biological rationale rather than full-scale randomized trials.
To qualify, sponsors must justify why randomized studies are impractical and commit to collecting real-world evidence after approval. Confirmatory trials must already be underway if accelerated approval is granted, and the FDA retains the authority to withdraw products if post-marketing studies fail or are not completed.
The agency emphasized the importance of early baseline and natural-history data collection. For therapies targeting multiple mutations within the same gene, the FDA recommends observational studies and “master protocol” designs that allow shared evidence generation across related products.
Patient protections will remain central to the framework, including informed consent requirements and oversight by institutional review boards. Manufacturing standards will not change, although companies may leverage validated platforms and prior development experience to streamline production.
FDA officials anticipate a significant increase in applications under the new pathway, reflecting growing innovation in genome editing, RNA therapeutics, and other precision genetic technologies.
