IQ Consortium Clinical Pharmacology Pediatric WG Extrapolation Case Study
April 24, 2020
Pediatric extrapolation utilizes and integrates available knowledge in adult populations (“priors”), and identifies critical gaps and uncertainties in that knowledge, to define a targeted set of required clinical data to fill the knowledge gaps for application to pediatric patients. Although the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) and National Medical Products Administration (NMPA) have adopted the International Council for Harmonization (ICH) Guideline: E11 (R1) Addendum thereby encouraging extrapolation, good use cases were lacking and the full potential and application opportunities were not fully realized. In particular, questions of general applicability of pediatric extrapolation to a certain disease, disease similarity, and whether or not these are pertinent to the use of a new molecular entity in pediatric patients were sometimes the reason for controversial perceptions of industry, academia and even between regulatory bodies.