Designing a Turnkey Orphan Drug Program
Summary: The orphan drug designation is a vital regulatory pathway, responsible for a substantial portion of all new approvals at the FDA. Just how are today’s drug developers navigating the incentives and challenges that come with following this route? This Endpoints News webinar brings together four experts who represent some of the key constituencies when developing a modern orphan program – from early research through approval.
What are the latest approaches in achieving proof-of-concept in orphan disease populations? How will your program evolve between IND through NDA? What are regulators looking for? And, how do we do all of this while keeping a laser focus on the patient?