Upcoming FDA-ARM Workshop to Focus on Pediatric CGT Clinical Trials

The Food and Drug Administration and the Alliance for Regenerative Medicine (ARM) will host a joint public workshop on April 9 to examine scientific, ethical, regulatory, and practical considerations for pediatric cell and gene therapy (CGT) clinical trials.

The workshop will explore challenges and potential solutions related to enrolling children in clinical trials, including the timing of pediatric enrollment, the interpretation of potential direct benefit and considerations for studying therapies in presymptomatic or early-stage disease. Discussions will also address diseases that affect both pediatric and adult populations, as well as conditions that occur primarily in children.

The event will take place at FDA’s White Oak Campus in Silver Spring, Md., with options for both in-person and virtual participation. Registration is required.