Food and Drug Administration Requirements for Clinical Studies in Pediatric Patients

Many drugs approved by the US Food and Drug Administration (FDA) for use in adults lack adequate data on safety and efficacy in pediatric patients, a potential source of unintended harm to pediatric patients. Through a series of laws, regulations, and guidance documents, the US Congress and FDA have created a program both to encourage and mandate clinical studies in pediatric patients to develop evidence-based dosing, safety, and efficacy information. A "Pediatric Study Plan" (PSP) is required for every new drug. FDA provides incentives for the voluntary conduct of clinical trials in pediatric patients, including opportunities for added marketing exclusivity and for obtaining a "priority review voucher." FDA also mandates that clinical studies for new drugs be conducted in each pediatric age group (newborns, infants, children, and adolescents), except in circumstances where a waiver or a deferral of studies can be justified. Sometimes this mandate can be met by extrapolation from studies in adults, or from patients in one pediatric age group to another, for evidence of efficacy. However, separate studies of safety and dosing are usually required for each pediatric age group. The package insert for each new drug now must address the use in pediatric patients. In addition, the FDA website displays all changes in drug labeling related to pediatric patients (excerpted from the labels for easy access), summaries of all pediatric studies that have led to labeling changes, links to FDA medical reviews of pediatric studies, summaries of all pediatric safety issues presented to the FDA Pediatric Advisory Committee (with links to the meeting materials and transcripts), and details of deferred pediatric studies with their timelines and progress. These measures reflect the increasing attention by FDA and the medical community to the importance of clinical studies in pediatric patients.