The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
The FDA issued draft guidance establishing a plausible mechanism framework for approving individualized therapies targeting ultra-rare diseases when randomized controlled trials are not feasible due to small patient populations.
The FDA reversed its decision to reject Moderna's mRNA influenza vaccine application, accepting it for review one week after an initial refusal-to-file that reportedly involved senior leaders overruling staff advice.
The FDA released draft guidance establishing a new approval framework for individualized gene-editing and RNA-based therapies targeting ultra-rare diseases, allowing approvals based on small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.
The FDA proposed a new framework to accelerate approvals of personalized treatments for rare genetic diseases, allowing drugmakers to rely on small studies when traditional trials are not feasible while requiring post-approval evidence collection.
