Gottlieb calls for end to abuse of orphan drug designation

FDA Commissioner Scott Gottlieb will take steps to ensure that the orphan drug designation is used appropriately, with new guidance to be issued on its application.

Dr Gottlieb said that the US Food and Drug Administration will hold a public meeting to get input on: “Scientific and regulatory issues such as those raised by molecularly targeted drugs and biologics, and the appropriate application of orphan incentives.”

This raises the possibility that the designation and its advantages, such as extended exclusivity, may be less generously conferred in future.