While the US FDA grapples with its emerging regulatory framework for real-world evidence, a small wave of recent clinical announcements illustrates how rare disease therapy sponsors are already increasingly reliant on natural history studies to guide drug development.
The FDA’s January 2019 draft guidance on rare disease drug development emphasizes the role of natural history data. "The natural history of rare diseases is often poorly understood, and...
Read the full article – start your free trial today!
Join thousands of industry professionals who rely on Pink Sheet for daily insights
- Start your 7-day free trial
- Explore trusted news, analysis, and insights
- Access comprehensive global coverage
- Enjoy instant access – no credit card required
Already a subscriber?