Quantitative system pharmacology as a legitimate approach to examine extrapolation strategies used to support pediatric drug development

CPT Pharmacometrics Syst Pharmacol. 2022 Jul;11(7):797-804. doi: 10.1002/psp4.12801. Epub 2022 May 24.


Extrapolation strategies from adult data for designing pediatric drug development programs are explored using the quantitative systems pharmacology (QSP) modeling approach, a mechanistic drug and disease modeling framework that can predict clinical response and guide pediatric drug development in general. This innovative model-informed drug discovery and development approach can leverage adult-pediatric pharmacology and disease similarity metrics to validate extrapolation assumptions. We describe the QSP model strategy and framework for extrapolation to design pediatric drug development programs by leveraging adult data across a wide range of therapeutic areas and illustrating stage-gate decisions informed by such an approach.

Publication types

  • Review

MeSH terms

  • Adult
  • Benchmarking
  • Child
  • Drug Development*
  • Drug Discovery
  • Humans
  • Models, Biological
  • Network Pharmacology
  • Pharmacology*
  • Research Design