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 tazemetostat. The agency viewed the study to be poorly designed...
The agency’s critical comments about the design and utility of Epizyme’s real-world study provide useful insights for sponsors interested in pursuing similar approaches, particularly in very rare diseases where randomized,...