Summary
Join us for an insightful webinar exploring how Quantitative Systems Pharmacology (QSP) and digital twins are reshaping clinical development strategies in rare diseases. With patient populations often too small for traditional trial designs, digital twin modeling presents a transformative approach to improve therapeutic development and regulatory interactions. Our partners from Sanofi will showcase real-world applications, including case studies in ASMD and Pompe disease, highlighting how QSP-based digital twins can address unique challenges in rare disease trials and gene therapies. Whether you’re a translational scientist, clinical developer, or regulatory strategist, this session will equip you with practical insights into cutting-edge modeling approaches for rare disease drug development.
Learning Objectives
By attending this webinar, participants will:
- Understand the role and value of QSP in clinical development for rare diseases.
- Learn how digital twin models are developed and used to support therapeutic decisions.
- Explore real-world case studies demonstrating regulatory applications of QSP-based digital twins.
- Identify challenges and opportunities in applying these models to rare disease and gene therapy trials.
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Senior Vice President and Head of Quantitative Systems Pharmacology, Certara
Piet van der Graaf is Senior Vice President and Head of Quantitative Systems Pharmacology at Certara and Professor of Systems Pharmacology at Leiden University. From 2013-2016 he was the Director of Research of the Leiden Academic Centre for Drug Research. From 1999-2013 he held various leadership positions at Pfizer in Discovery Biology, Pharmacokinetics and Drug Metabolism and Clinical Pharmacology. He was the founding Editor-in-Chief of CPT: Pharmacometrics & Systems Pharmacology from 2012-2018 before becoming Editor-in-Chief of Clinical Pharmacology & Therapeutics. Piet received his doctorate training in clinical medicine with Nobel prize laureate Sir James Black at King’s College London. He has been awarded the 2024 Gary Neil Prize for Innovation in Drug Development from the American Society of Clinical Pharmacology and Therapeutics (ASCPT) and was the recipient of the 2021 Leadership Award from the International Society of Pharmacometrics (ISoP). Piet is an elected Fellow of the British Pharmacological Society and has published >200 articles in the area of quantitative pharmacology and drug development.
Head of QSP US, Translational Disease Modeling (TDM), Translational Medicine Unit, Sanofi R&D
Director, Quantitative Systems Pharmacology, Sanofi
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