Rare diseases: FDA proposes new system to therapy approvals
AP News • • business
Key Points:
- The FDA has proposed new guidelines to facilitate the development of customized treatments for rare and hard-to-treat diseases, including gene editing therapies, by creating a pathway for therapies tested in only a few patients.
- This new "plausible mechanism" pathway aims to overcome traditional barriers in drug approval processes, especially for rare genetic conditions that lack large patient populations and are often unprofitable for pharmaceutical companies.
- The proposal allows companies to commercialize experimental treatments that target well-understood conditions with a scientifically plausible basis, contrasting with the current "compassionate use" system that prohibits profit and is cumbersome.
- The FDA will accept public comments on the draft guidance for 60 days before finalizing it, emphasizing regulatory flexibility to encourage scientific advances and deliver
