Challenge #2: Rare or Ultra-Rare Study Design Considerations and Use of Existing Data
There are more than 7,000 unique, rare diseases recognized in the United States, and 300 million people worldwide living with a rare disease; patients often experience a long journey to receive their diagnosis. Our expert panelists, Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO and Adora Ndu, PharmD, JD, Chief Regulatory Affairs Officer at BridgeBio, examine the regulatory requirements for safety and efficacy demonstration and identify the gaps in regulatory oversight that require clearer guidance.
VIEW THE VIDEO! Log In
Get unlimited access to:
Trend and Thought Leadership Articles
Case Studies & White Papers
Extensive Product Database
Members-Only Premium Content
Welcome Back! Please Log In to Continue. X
Enter your credentials below to log in. Not yet a member of Cell & Gene? Subscribe today.
Subscribe to Cell & Gene X
Subscribe to Cell & Gene