New CBER Director Pledges Swift Action, Flexibility for Rare Disease Treatments

Endpoints News -June 3rd    Vinay Prasad, the new head of the FDA’s Center for Biologics Evaluation and Research (CBER), recently outlined a flexible and patient-focused approach to rare disease treatment approvals. Speaking at the National Organization for Rare Disorders’ conference, Prasad stated his intention to “rapidly make available” even small advances in rare disease therapies, signaling a more adaptable stance than some of his previous criticisms of the FDA suggested.

Prasad pledged close collaboration with industry and emphasized the agency’s flexibility regarding the type of data needed to support rare disease treatments. This marks a notable shift from past comments where he criticized the FDA as a “rubber stamp” for certain rare disease therapies and expressed concerns about the overuse of accelerated approvals.

In one of his initial public outlines of his regulatory philosophy, Prasad indicated that the FDA would expedite approvals for rare disease drugs, sometimes based on limited evidence, to ensure quick patient access. This approach includes utilizing surrogate endpoints, a practice he had previously scrutinized, but with a firm commitment to rigorous post-market surveillance using real-world data to confirm actual efficacy. He clarified that the agency would consider both surrogate endpoints and overall survival, aiming to “empower patients to make choices right for them.”

Prasad stressed that the FDA would “take action at the first sign of promise for rare diseases” and would not delay approvals. Simultaneously, he committed to closely tracking approved products in the post-market space. He also mentioned plans to publish general guidelines detailing accelerated review processes for some rare disease drugs, emphasizing the importance of engaging with patients and their families.