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