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

Area of Science:

• Regulatory Science
• Translational Medicine
• Pharmacology

Background:

• The Accelerating Rare Disease Cures (ARC) program aims to develop regulatory and scientific tools for rare disease drug development.
• Tools include drug/disease modeling, dose selection, and translational medicine.

Purpose of the Study:

• To discuss the utility of quantitative systems pharmacology (QSP) in rare disease drug development.
• To identify challenges and solutions for QSP implementation.
• To propose a roadmap for QSP-informed rare disease drug development.

Main Methods:

• A virtual public workshop was hosted by the FDA and University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI).
• Participants included scientists from pharmaceutical companies, academia, and the FDA.
• Discussions focused on QSP's potential and challenges in rare disease drug development.

Main Results:

• The workshop identified the potential utility of QSP in rare disease drug development.
• Key challenges and potential solutions for QSP integration were discussed.
• Main findings and key takeaways from the workshop were reported.

Conclusions:

• QSP holds significant potential to aid rare disease drug development.
• A roadmap is proposed to facilitate the use of QSP in this field.
• Collaboration between regulatory bodies, industry, and academia is crucial.