Population Physiologically-Based Pharmacokinetic Modeling to Determine Ontogeny: A Quantitative Clinical Pharmacology Example in Pediatric Rare Disease.

ABSTRACT: Pediatric physiologically-based pharmacokinetic (PBPK) modelling plays an increasing role in selecting doses in children and addressing clinical pharmacology questions. Ethical concerns often limit clinical pharmacology studies that have no direct therapeutic benefit in children, highlighting the value of PBPK model predictions. However, regulatory acceptance of pediatric PBPK models remains limited because of uncertainties in system-specific information and inadequate model qualification. Ambiguous ontogeny data of drug metabolizing enzymes (DME) and transporters are recognized as significant obstacles to the accurate pharmacokinetics (PK) prediction in children and the leading cause of insufficient pediatric PBPK model qualification. To address this challenge, a population PBPK modeling

SUBMITTER: Cleary Y 

PROVIDER: S-EPMC12853142 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature