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RWE On The Runway: Trio Of Upcoming Approval Decisions Will Test US FDA’s Thinking On RWE For Efficacy

Executive Summary

Real-world evidence provides external controls for bluebird bio’s eli-cel and Ipsen’s palovarotene, while Reata used RWE to address FDA concerns about the pivotal single-arm study of omaveloxolone. In the post-market, Intercept is gearing up to seek full approval of Ocaliva relying in part on claims database.

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