Drug development for rare and orphan diseases poses complex challenges to the biopharmaceutical industry, including unique issues around clinical trial recruitment; identification and validation of biomarkers; development of clinical endpoints; and collection of natural history data. These complexities make drug development in this space inherently risky; yet in recent years we have seen tremendous growth fueled by the entrance of large pharmaceutical companies, an increased number of partnerships across diverse stakeholders, and positive incentives for both the research and development and commercialization of orphan drug products. This growth promises to improve the health of patients with debilitating and life-threatening rare disorders. The Orphan Disease Forum will provide a unique perspective on the international orphan drug marketplace; new and emerging funding models; the vital role of patient advocacy organizations; and scientific and regulatory considerations for orphan drug development.

See the complete session line-up below.


Sponsored by CSL Behring and Genzyme, a Sanofi Company

Session ID: 2644

Sub Sessions
myBIO Chatter
Educational Topics
None Identified