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

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 continue encouraging randomized clinical trials when they make sense, but acknowledged that in rare diseases