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

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

Epizyme Inc. had hoped its real-world natural history study would convince the agency that the benefit clinical trial participants experienced with Tazverik (tazemetostat) was greater than what epithelioid sarcoma patients see with standard of care.

However, the real-world study, which was based on retrospective chart review, did not factor into the FDA’s efficacy assessment of...

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