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Updated: Sep 27, 2025

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Quantitative system pharmacology as a legitimate approach to examine extrapolation strategies used to support
Karim Azer1, Jeffrey S Barrett2
1Axcella Therapeutics, Cambridge, Massachusetts, USA.
Abstract:
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.
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