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A Baker’s Dozen Of US FDA Efficacy Approvals Using Real World Evidence

 
• By Bridget Silverman

Fresenius Kabi’s Omegaven is most recent of 13 approvals, including three with breakthrough therapy designations, where FDA has relied on real world evidence to make a regulatory decision about efficacy.

The exigencies of ultra-orphan drug development have provided the early testing ground for the US FDA’s limited use of real-world evidence (RWE) in approval decisions, resulting in 13 approvals identified by the Pink Sheet that relied on external or historical controls to place single-arm efficacy trials in context or derived data from case reports and expanded access protocols.

FDA’s most recent approval using RWE – the July 27, 2018 clearance of Fresenius Kabi AG’s I.V

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