Real-World Evidence At US FDA: Bavencio, Blincyto Approvals Point Way Toward Broader Use
Breakthrough-designated oncologics used historical comparator data to determine efficacy threshold for pivotal Phase II studies supporting accelerated approval; Blincyto’s subsequent full approval offers validation with conventional controlled study.
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Covis seeks to discuss study proposals and other accelerated approval precedents at still-unscheduled withdrawal hearing; CDER says company’s current RWE proposal is inadequate given its design and the failure of the PROLONG confirmatory clinical trial.
Methodological problems with RWE submissions for approved applications may give sponsors ‘a little bit of a pause’ as they work through data selection and analytical issues, Aetion’s Jeremy Rassen says. CDER’s Janet Woodcock says it is easier to use RWE when treatment effects are large.
Biopharma companies are reluctant to rely on real-world evidence trials, but while uncertainty remains about how these novel studies will be accepted, US and other regulators are encouraging drug developers to take a chance – and some have taken on the challenge.