RWE’s Biggest Role In US FDA Approvals: External Controls For Breakthrough Rare Disease Therapies

 
• By Bridget Silverman

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.

RWE breakthrough
When the US FDA relies on real-world evidence, it is most commonly for rare disease applications with breakthrough designations. • Source: Nielsen Hobbs; the Pink Sheet | Shutterstock images

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