A batch of new FDA guidance documents for neurology drugs released Thursday February 15 support Commissioner Scott Gottlieb’s oft-stated goal to offer manufacturers more flexibility in how they can win the agency’s approval.
For drugs intended for children age 4 and older with partial onset seizures, the FDA will no longer require that efficacy trials be conducted in children. The agency will now consider efficacy data from adult patients to be sufficient for pediatric approval.
FDA’s reasoning in this instance is that previous trials of seizure drugs conducted in children have shown that their dose-response patterns do not differ from those seen in adult patients. However, formulations of drugs for pediatric patients must still take into consideration the special needs of children, and it’s conceivable that trials will be required to support approval of particular products. “FDA encourages sponsors to explore innovative approaches to pediatric formulation development and testing,” the guidance noted. And the agency will continue to require that clinical trials be conducted in children for safety endpoints.