This draft guidance is intended to assist those sponsors of new drug applications (NDAs), biologics license applications (BLAs) for therapeutic biologics, and supplements to such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 that 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 In general, this draft guidance focuses on the clinical pharmacology information response, pharmacokinetics, and pharmacodynamics that support findings of effectiveness and safety and helps identify appropriate doses in pediatric populations. This guidance also describes the use of quantitative approaches 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: standards for approval of drug and biological products in the pediatric population, 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 clinical pharmacology studies for vaccine therapy, blood products, or other products not regulated by the Center for Drug Evaluation and Research.  FDA's guidance documents, including this guidance, do not establish legally enforceable responsibilities. Instead, guidance’s describe the Agency's current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidance’s means that something is suggested or recommended, but not required.

There are several recognized approaches to providing substantial evidence to support the safe and effective use of drugs in pediatric populations, including evidence from adequate and  well-controlled investigations of a specific pediatric indication different from the indication  approved for adults; evidence from adequate and well-controlled investigations in pediatric  populations to support the same indication(s) approved for adults; or evidence from adequate  and well-controlled studies in adults and additional information in the specific pediatric population. The first approach generally requires a full pediatric development program. The second approach above generally involves the use of prior disease and exposure-response knowledge from studies in adults and relevant pediatric information to design and, in some cases, analyze new pediatric studies. For the third approach, the assumption is that the course of the disease and the effects of the drug are sufficiently similar in the pediatric and adult populations to permit extrapolation of the adult efficacy data to pediatric patients. If the third approach is taken, there would ordinarily be a pediatric study to determine a in the pediatric population that provides a drug exposure similar to the exposure that is effective in adults. If there is a concern that exposure-response relationships might be different  in pediatric patients, studies relating blood levels of drug to pertinent pharmacodynamic effects other than the desired clinical outcomes.

Best Regards,
Nancy Ella
Associate Managing Editor
Drug Designing: Open Access