Methodological Challenges Related to Rare and Orphan Disease Drug Development

Chairs: Joan Busner, PhD; Judy Dunn, PhD; Ravi Anand, MD
Drug development for rare and orphan CNS diseases is associated with a unique set of methodological challenges, often underappreciated, that can delay trial completion, complicate interpretation, and ultimately threaten trial success.  This session will provide a high-level, candid, examination of challenges and potential strategies for overcoming them.  The session will cover the role of patient advocacy in meeting challenges, statistical considerations in small N trials, emerging trends in data submission requirements, challenges in identifying disease-specific and patient-centric outcomes, logistical challenges including far-spread patients, illness-related travel restriction, and lack of research-trained investigators, and the FDA perspective including public health considerations, pediatric regulations, and incentives to developers.  As the first ISCTM session to address orphan and rare disease drug development; we anticipate high levels of audience engagement and discussion.