The FDA’s new director for the Center for Biologics Evaluation and Research (CBER) has committed to rapidly making rare disease drugs available to patients. Vinay Prasad announced that the agency will use surrogate endpoints to get promising medicines to patients before traditional efficacy is met.
Prasad stated that his priority is to provide products that are “even small steps forward” for rare diseases, emphasizing patient empowerment. The new policy aims to give patients early access to treatments despite limited evidence. The FDA plans to assess these medicines using real-world data (RWD) and engage with patients to gather more information.
Under this approach, the FDA will grant conditional approval based on scientifically plausible data from single-arm trials when randomized studies are not feasible. This pathway allows for faster review processes, as Prasad mentioned that the agency will publish general guidelines on its timeline. The FDA has also established a surveillance system to monitor those who receive treatments approved via this pathway. Experts had raised questions about this policy but look forward to further details.
Source: https://www.biospace.com/fda/fdas-prasad-vows-to-make-rare-disease-drugs-available-at-first-sign-of-promise