For Rare and Rapidly Progressing Disease US FDA Has Heard ‘Well and Clear’ Placebo Is Unacceptable

 
• By Sarah Karlin-Smith

Remarks at the NORD Breakthrough Summit from the FDA’s Lola Fashoyin-Aje may suggest the agency is becoming more comfortable with single-arm trials.

one single human arm raised up on an angle
The FDA's Lola Fashoyin-Aje said the agency understands people do not want to enroll in randomized studies for rapidly progressing fatal diseases with no available therapies. (Shutterstock)

Patients and caregivers are understandably unwilling to participate in clinical trials for rare and rapidly progressing fatal diseases that lack available therapies if they could be randomized to placebo, but the US Food and Drug Administration’s Lola Fashoyin-Aje assured them there are “lots of ways around this.”

Key Takeaways
  • If there are no available or effective therapies for a rapidly, progressive fatal disease, the FDA's current thinking is that there is not equipoise for...

Fashoyin-Aje, director of the Office of Clinical Evaluation (Cell and Gene Therapies) in the Center for Biologics Evaluation and Research’s Office of Therapeutic Products, responded 22 October to a comment...

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