The FDA has issued draft guidance creating a "Plausible Mechanism Framework" to accelerate approval of individualized therapies for ultra-rare diseases where traditional clinical trials are not feasible. The guidance focuses on gene editing and RNA-based treatments targeting specific genetic abnormalities.
The FDA released draft guidance establishing a new approval framework for individualized gene-editing and RNA-based therapies targeting ultra-rare diseases, allowing approvals based on small studies when traditional trials are not feasible.
Federal regulators unveiled a new regulatory approach to accelerate approval of personalized genetic treatments for rare, life-threatening conditions, allowing smaller studies when traditional large-scale trials aren't practical while maintaining post-market safety oversight.
