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.
You may also be interested in...
Pediatric Cancer Trials: US FDA Panel To Weigh Use Of Real-World Data, Patient-Reported Outcomes
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.
Keeping Track: Industry Beefs Up 2020 Review Pipeline With Submissions Galore
The latest drug development news and highlights from our US FDA Performance Tracker.
US FDA's Temple On Real-World Evidence: 'I Find The Whole Thing Very Frustrating'
CDER deputy director for clinical science reiterates his long-held skepticism about whether real-world evidence can be used to demonstrate efficacy claims.