Food and Drug Administration Requirements for Clinical Studies in Pediatric Patients

Therapeutic Innovation & Regulatory Science
Edward Tabor

Abstract

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...Continue Reading

References

Sep 14, 2006·JAMA : the Journal of the American Medical Association·Daniel K BenjaminJennifer S Li
Feb 8, 2007·JAMA : the Journal of the American Medical Association·Jennifer S LiDaniel K Benjamin
Dec 23, 2011·Journal for Specialists in Pediatric Nursing : JSPN·Lauren Thorngate, Shuyuann Wang Foreman
Jun 6, 2012·JAMA : the Journal of the American Medical Association·Jeffrey D Sachs
Nov 28, 2012·The Journal of Pediatric Pharmacology and Therapeutics : JPPT : the Official Journal of PPAG·Michael L Christensen
Jan 16, 2013·Pediatrics·Marilyn J FieldThomas F Boat

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Citations

Apr 16, 2020·Biochemia Medica·Lara Milevoj KopcinovicJelena Culej
Dec 5, 2018·Therapeutic Innovation & Regulatory Science·G Caleb AlexanderChristopher D Breder

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