ABSTRACT
Limited pharmacokinetic (PK) and pharmacodynamic (PD) data exist in pediatric populations for the majority of medicines approved for adult indications. Despite the lack of dosing and PK/PD information available, physicians treating children are often left with the difficult task of adjusting adult dosages or simply avoid prescribing the available medications. Seeking to improve on this situation, the
U.S. Food and Drug Administration has requested that drug companies study drugs likely to be administered to pediatric populations in prospective dose-finding trials. While actual efficacy trials in pediatric populations are seldom conducted, these guidelines have increased the number of studies available to define pediatric dosing regimens. The FDA Modernization Act of 1997 [21 U.S.C. 355a(b)] required the FDA to develop, prioritize, and publish an initial list (Docket No. 98N-0056) of approved drugs for which pediatric information may produce benefits in the pediatric population. While the list does not constitute a written request under 21 U.S.C. 355(a) or infer that the drug is entitled to pediatric exclusivity, it does reveal the indications and compound classes for which additional experience in the pediatric population is warranted. The regulatory appreciation for the necessity of studying pediatric populations has been further codified by 21 CFR Parts 201, 312, 314, and 601 which require drug manufacturers to provide sufficient data and information to support directions for pediatric use for claimed indications. These regulations have been reauthorized with the “Best Pharmaceuticals for Children Act,” which was signed into law in January 2002.
