Overview of the Extrapolation of Pediatric Efficacy, or Children May Not Be Little Adults But They Are Not Space Aliens

ABSTRACT

Objectives: The objectives are to describe the history and science behind extrapolation of pediatric efficacy, and to provide examples of the successful and unsuccessful application of extrapolation of efficacy in different therapeutic areas outside of psychiatry. Method(s) A review will be conducted of FDA, European, and international regulatory documents and articles from the medical literature discussing extrapolation of pediatric efficacy. Examples of medications with pediatric labeling that is based on extrapolation of pediatric efficacy will be identified with the help of the FDA pediatric labeling database. Result(s) The concept of extrapolation of pediatric efficacy was first introduced with the pediatric rule in 1994. Whether or not extrapolation of pediatric efficacy is appropriate depends on our understanding of a disease's clinical characteristics, developmental time course, prognosis, and pathophysiology, as well as the developmental pharmacokinetics and pharmacology of the drug or biologic. Examples from several therapeutic areas outside of psychiatry will be discussed, including monoclonal antibodies and vaccines against SARS-CoV-2. Conclusion(s) Extrapolation of pediatric efficacy is an important tool in pediatric drug development. Not only does it potentially reduce the number of pediatric patients who must be subjected to clinical trials, and potentially to placebo, but it also is one of the fastest approaches to providing pediatric data to inform clinicians. PPC, R, SZ Copyright © 2022