Under the US Food and Drug Administration (FDA) rare disease endpoint advancement pilot program (RDEA), preference will be given to applications with well-developed efficacy endpoints that can be applied to other rare diseases. That was the message from agency officials speaking at a workshop sponsored by the Duke-Margolis Center for Health Policy and FDA.
Rare disease drug development is challenging, said Kerry Jo Lee, associate director of rare diseases in the Center for Drug Evaluation and Research (CDER) Office of New Drugs (OND) and leader of the Rare Diseases Team. These diseases are progressive, serious, and often lack adequate approved therapies. Small patient populations often restrict options for study design.
Yet these challenges are not deterring drug developers from getting into this space. Julienne Vaillancourt, policy advisor and rare disease liaison in the Office of the Director in the Center for Biologics Evaluation and Research (CBER), noted the growing number of applications being submitted for rare disease treatments…
