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About this Virtual Workshop:

The U.S. Food and Drug Administration (FDA) in collaboration with the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) will host a virtual public workshop entitled “Creating a Roadmap to Quantitative Systems Pharmacology-Informed Rare Disease Drug Development” on May 11, 2023. The purpose of this workshop is to discuss the potential utility of quantitative systems pharmacology (QSP) in rare disease drug development and brainstorm the potential path to address the challenges and facilitate its use.

There are approximately 7,000 rare diseases. There is a public heath need to develop safe and efficacious drugs to treat these conditions. However, there are many challenges in developing drugs for rare diseases, including the small number of patients available for clinical trials to optimize the dosing regimen and demonstrate safety and efficacy of the drug, inter-individual variation in the course of disease, and a lack of well-characterized biomarkers to inform drug effects. QSP modeling can mechanistically and quantitatively link a drug candidate’s target via the pharmacological network to model and simulate drug responses and the extent of response variability to inform drug development decisions.

Workshop Objectives:

• Discuss rare drug development needs that may be addressed via QSP approaches
• Provide an overview of QSP and successful uses of QSP in drug development
• Discuss how to develop useful QSP models in rare diseases

Meeting Information and Registration: 

This workshop is open to the public with no cost to attend, but registration is required. 

For more information, visit the MCERSI Webpage. 

To register, visit the MCERSI Workshop Registration page.

Contact:

For questions regarding this event, please contact:

Tracey Lee
Office of Clinical Pharmacology
Center for Drug Evaluation and Research
U.S. Food and Drug Administration
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