概述
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 现任 Certara 高级副总裁兼 QSP 负责人，同时也是莱顿大学系统药理学教授。Piet 是 2013 - 2016 年莱顿药物研究学术中心的研究主任。1999 - 2013 年，Piet 在辉瑞公司的研发生物学、药代动力学和药物代谢、以及临床药理学等部门担任过多种领导职务。Piet 于2012 - 2018 年担任 CPT: Pharmacometrics & Systems Pharmacology 的创始主编，随后成为 Clinical Pharmacology & Therapeutics 的主编。Piet 曾在伦敦国王学院师从诺贝尔奖获得者 Sir James Black，接受了临床医学的博士培训。他曾获得 2024 年美国临床药理学与治疗学会（ASCPT）颁发的 Gary Neil 药物开发创新奖，同时也是国际定量药理学会（ISoP）2021 年领袖奖的获得者。Piet 是英国药理学会的当选委员，在定量药理学和药物开发领域发表了超过 200 篇同行评议的论文。
Head of QSP US, Translational Disease Modeling (TDM), Translational Medicine Unit, Sanofi R&D
Director, Quantitative Systems Pharmacology, Sanofi
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