US Food and Drug Administration Facilitated Pediatric Approval Programs: Application to Pediatric Neurological Disorders.

Abstract

Crucial time is often lost while waiting for approval of therapies for pediatric neurological disorders, many of which have aggressive manifestations with devastating effects. There are logistical, ethical, and financial impediments that face the studies needed to determine efficacy and safety of therapies in children. In this article, the authors present the Food and Drug Administration's programs aimed at facilitating the development of pediatric drugs, focusing on their application to pediatric neurological disorders. They also provide examples of drugs that received, or are currently enrolled in, these programs. Reflecting upon the commonalities of drugs receiving these designations, the authors highlight underlying ethical issues related to pediatric drug development and emphasize the need for structured incentives to stimulate approval and production of drug therapies for pediatric neurology patients. By consolidating information that applies to drug approval of pediatric neurological disorders, stakeholders in drug development can enhance treatment development for these disorders.