RWE’s Biggest Role In US FDA Approvals: External Controls For Breakthrough Rare Disease Therapies
Despite broad interest, real-world evidence of efficacy in FDA decisions remains largely limited to orphan diseases and natural history, a Pink Sheet analysis shows.
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Real-World Evidence: Amylyx’s Use Of External Controls To Frame Relyvrio’s Survival Benefit Rife With Problems
The post hoc comparison of an exploratory, long-term survival benefit in the CENTAUR trial to historical controls in two ALS patient databases lacked prespecification and a common treatment protocol, and potential differences in prognostic factors may have confounded the results.
Unsuccessful attempts to use real-world evidence for approvals highlight the need to demonstrate relationship between real-world data and clinical trial patients. The Pink Sheet examines what went wrong for Bluebird/Bristol’s Abecma, Ultragenyx’s Dojolvi, and Karyopharm’s Xpovio.
The Citeline Awards celebrate the efforts of biopharma research and development communities, with 12 categories ranging from use of technology to quality results. Nominations are open through 20 January 2023.