The Real World Is Not Enough: Comparability Is Common Obstacle For RWE In US FDA Decisions
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.
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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.
Oncology sponsors would handle ex-US trial enrollment, but Pazdur feels tapping into the NCI network would improve trial speed and diversity. Biggest challenge will likely be generating sponsor enthusiasm for move that would involve giving up some control of development.
The disclosure requirement to participate includes an option for the US FDA to include the ideas on new regulatory uses for real-world evidence in a guidance or speak about them at public forums in order to ‘promote innovation.’