Achieving Meaningful Regulatory and Clinical Outcomes for Patients: Strategies in Rare Disease Therapy Development

This session will focus on addressing the unique regulatory complexities and challenges specific to orphan drug development. Experts will examine the industry’s perspective on many important issues, including, heterogeneity, natural histories and progression, and endpoints. FDA representatives will provide key information about programs available to expedite the development of orphan products. This session will close with an interactive roundtable discussion that will demonstrate the importance of cooperation and collaboration of all key stakeholders.