Public Meeting on Patient-Focused Drug Development for Neurologic Manifestations of Inborn Errors of Metabolism

摘要

The Food and Drug Administration (FDA or Agency) is announcing a public meeting and an opportunity for public comment on Patient- Focused Drug Development for neurologic manifestations of inborn errors of metabolism. Patient-Focused Drug Development is part of FDA's performance commitments made as part of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The public meeting is intended to allow FDA to obtain patient perspectives on the impact of neurologic manifestations of inborn errors of metabolism on daily life as well as patient views on treatment approaches for neurologic manifestations of inborn errors of metabolism.