Baby Steps To Real-World Evidence Of Efficacy: External Controls Gain Popularity In Rare Disease Trials
The Pink Sheet takes a closer look at recent clinical announcements showing how rare disease therapy sponsors are already increasingly reliant on natural history studies to guide drug development.
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Advisory committee will discuss ways to encourage inclusion of the Pediatric PRO-CTCAE tool in cancer trials on 11 May, followed the next day by a discussion on real-world data sources that could inform the efficacy and long-term safety of pediatric cancer treatments.
The latest drug development news and highlights from our US FDA Performance Tracker.
CDER deputy director for clinical science reiterates his long-held skepticism about whether real-world evidence can be used to demonstrate efficacy claims.