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Gene Therapy: US FDA Willing To Accept Single-Arm Trials For Approval, But Sponsors May Hesitate

 
• By Derrick Gingery

Data indicates a smaller percentage of studies intended for product registration or indication expansion are single-arm trials, compared to the proportion for all gene therapy trials.

gene therapy pills
Most registrational gene therapy clinical trials do not use single-arm designs. • Source: Shutterstock

Sponsors might not be confident that single-arm studies can support gene therapy approvals in the US despite Peter Marks’ statements to the contrary.

Marks, the director of the US Food and Drug Administration’s Center for Biologics Evaluation and Research, wants to grow gene therapy development, particularly in rare diseases, through the accelerated approval pathway

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