European Regulatory Perspective on Pediatric Physiologically Based Pharmacokinetic Models

Abstract

Physiologically based pharmacokinetic (PBPK) models offer a mechanistic understanding of the disposition of the drug in the body. When well informed and conducted, this may lead to more efficient clinical studies in the vulnerable pediatric population. A review of pediatric submissions to European regulatory authorities has shown a limited number of recent examples. The use of PBPK models to inform pediatric drug development is encouraged. It is, however, important to consider the confidence in the predictions. A qualification of the PBPK platform for the intended purpose should be performed, and a learn-and-confirm approach is recommended. Further research in this area is encouraged to inform important parameters, and to increase availability of pediatric data for model qualification.