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Gene Therapies Without Randomized Clinical Trials: Marks Outlines Rare Disease Development Path

 
• By Derrick Gingery

A single-arm trial using a biomarker and animal model could be enough for an accelerated approval, especially in a pediatric rare disease where randomization and placebo controls may not be feasible, the CBER director said.

clinical trial and laboratory
Gene therapies in pediatric rare diseases may not need a randomized clinical trial to gain accelerated approval. • Source: Shutterstock

Peter Marks continues to sketch a pathway to market for pediatric gene therapies in rare diseases that could be faster than traditional product development and not require a randomized clinical trial.

Marks, who heads the US Food and Drug Administration’s Center for Biologics Evaluation and Research, said that the center will...

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