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

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

Few regulatory concepts have received the attention that real-world evidence (RWE) has over the past few years, but its use to support efficacy in approval decisions by the US FDA remains largely limited to supporting roles providing external control arms or contextual data in rare diseases.

In the years following the release of FDA’s RWE framework in December 2018, the agency has approved at least 14...

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

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