Baby Steps To Real-World Evidence Of Efficacy: External Controls Gain Popularity In Rare Disease Trials
Executive Summary
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...
Finding The Sweet Spot For Real-World Evidence: Aetion Stresses Early Feasibility Assessment
Robust natural history data sets with well-chosen outcomes are fundamental in Aetion’s regulatory feasibility tool for real-world evidence, built through analysis of US FDA case studies and guidance.
External Controls: Sarepta’s DMD Gene Therapy Not In the Same Boat As Zolgensma, US FDA Says
Sarepta conducted study-level and integrated-level comparison analyses of SRP-9001-treated patients and external controls. However, heterogeneous nature of DMD and potentially moderate treatment effect of SRP-9001 distinguish it from Novaritis' spinal muscular atrophy treatment, where natural history data were used to support single-arm trial results, the FDA said.
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.