Creating a Roadmap to Quantitative Systems Pharmacology–Informed Rare Disease Drug Development: A Workshop Report

One of the goals of the Accelerating Rare Disease Cures (ARC) program in the Center for Drug Evaluation and Research (CDER) at the US Food and Drug Administration (FDA) is the development and use of regulatory and scientific tools, including drug/disease modeling, dose selection, and translational m...

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Bibliographic Details
Published in:Clinical pharmacology and therapeutics 2024-02, Vol.115 (2), p.201-205
Main Authors: Bai, Jane P.F., Stinchcomb, Audra L., Wang, Jie, Earp, Justin, Stern, Sydney, Schuck, Robert N.
Format: Article
Language:English
Subjects:
Drug Design
Drug Development
Humans
Network Pharmacology
Pharmaceutical Preparations
Rare Diseases - drug therapy
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Summary:One of the goals of the Accelerating Rare Disease Cures (ARC) program in the Center for Drug Evaluation and Research (CDER) at the US Food and Drug Administration (FDA) is the development and use of regulatory and scientific tools, including drug/disease modeling, dose selection, and translational medicine tools. To facilitate achieving this goal, the FDA in collaboration with the University of Maryland Center of Excellence in Regulatory Science and Innovation (M‐CERSI) hosted a virtual public workshop on May 11, 2023, entitled “Creating a Roadmap to Quantitative Systems Pharmacology–Informed Rare Disease Drug Development.” This workshop engaged scientists from pharmaceutical companies, academic institutes, and the FDA to discuss the potential utility of quantitative systems pharmacology (QSP) in rare disease drug development and identify potential challenges and solutions to facilitate its use. Here, we report the main findings from this workshop, highlight the key takeaways, and propose a roadmap to facilitate the use of QSP in rare disease drug development.
ISSN:0009-9236
1532-6535
DOI:10.1002/cpt.3096