Real-World Evidence: Epizyme’s Epithelioid Sarcoma Natural History Study Falls Flat At US FDA

 
• By Sue Sutter

Sponsor had hoped to use retrospective case review study as an external control arm to support regular approval of tazemetostat; US FDA not only rejected this idea, it found the study design so flawed that it did not consider the results in its efficacy assessment for accelerated approval.

Wrong way traffic sign and safety cones.
Epizyme's natural history study was not well designed for the intended purpose, the US FDA said. • Source: Shutterstock

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