Comment from PORTAL (Program on Regulation, Therapeutics, and Law)

Summary: The Program On Regulation, Therapeutics, And Law (PORTAL) at Brigham and Women’s Hospital and Harvard Medical School supports the draft guidance but recommends specific refinements. They argue for limiting the framework to ultra-rare diseases, enhancing post-marketing safety monitoring, prioritizing the accelerated approval pathway, and increasing transparency regarding surrogate endpoints and platform-based approvals.