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

 
• By Bridget Silverman

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.

Real-world evidence is ready for takeoff
Several applications relying on RWE could soon be ready for take off. • Source: Nielsen Hobbs; the Pink Sheet | Shutterstock images

Three candidates for US FDA approval are using real-world evidence as part of the efficacy argument, illustrating some of the most common ways to RWE in applications.

In keeping with FDA’s history of approvals incorporating RWE of efficacy, all of the applications target rare diseases: Friedreich’s ataxia for Reata Pharmaceuticals, Inc.’s omaveloxolone; cerebral adrenoleukodystrophy (CALD) for...

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