This draft guidance is intended to assist those sponsors of new drug applications (NDAs), biologics license applications (BLAs) for therapeutic biologics, and supplements to such applications who are planning to conduct clinical studies in pediatric populations.
Effectiveness, safety, or dose-finding studies in pediatric patients involve gathering clinical pharmacology information, such as information regarding a product’s pharmacokinetics and pharmacodynamics pertaining to dose selection and individualization. This guidance addresses general clinical pharmacology considerations for conducting studies so that the dosing and safety information for drugs and biologic products in pediatric populations can be sufficiently characterized, leading to well-designed trials to evaluate effectiveness.
In general, this draft guidance focuses on the clinical pharmacology information (e.g., exposure-response, pharmacokinetics, and pharmacodynamics) that supports findings of effectiveness and safety and helps identify appropriate doses in pediatric populations. This guidance also describes the use of quantitative approaches (i.e., pharmacometrics) to employ disease and exposure-response knowledge from relevant prior clinical studies to design and evaluate future pediatric studies. The guidance does not describe: (1) standards for approval of drug and biological products in the pediatric population, (2) criteria to allow a determination that the course of a
disease and the effects of a drug or a biologic are the same in adults and pediatric populations, or (3) clinical pharmacology studies for vaccine therapy, blood products, or other products not regulated by the Center for Drug Evaluation and Research.