Welcome & Introduction
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Welcome & Introductory Remarks
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Robert Schuck, PharmD, PhD
Audra Stinchcomb, PhD
Deputy Director, DTPM, OCP, CDER, FDA, Professor, School of Pharmacy, University of Maryland
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Video | Presentation (PDF)
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Session 1: RARE DISEASE DRUG DEVELOPMENT NEEDS THAT MAY BE ADDRESSED VIA QSP APPROACHES
Session Chair: Robert Schuck, PharmD, PhD Deputy Director, DTPM, OCP, CDER, FDA
Moderator: Valeriu Damian, PhD Senior Director, System Modeling & Translational Biology, Computational Sciences, Medicine Design, R&D, GlaxoSmithKline-Upper Providence
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| Considerations in Rare Disease Drug Development and How CDER is Accelerating Rare Disease Treatments |
Kerry Jo Lee, MD Associate Director for Rare Diseases, DRDMG, OND, CDER, FDA |
Video | Presentation (PDF)
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| The Quantitative and the Mechanistic: Strategies to Advance Rare Disease Drug Development through Regulatory Science |
Issam Zineh, PharmD, MPH, FCP, FCCP Director, OCP, OTS, CDER, FDA |
Video | Presentation (PDF)
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| Supporting Orphan Drug Development with Retrospective Quantitative Natural History Modeling – Conceptual Framework, Opportunities and Limitations |
Markus Ries, MD, PhD, MHSc, FCP Professor, Pediatric Neurology and Metabolic Medicine, Center for Rare Diseases, Center for Pediatric and Adolescent Medicine, Heidelberg University Hospital, Heidelberg, Germany |
Video | Presentation (PDF) |
| Session 1 Panel Discussion |
Issam Zineh, PharmD, MPH, FCP, FCCP Director, OCP, OTS, CDER, FDA
Kerry Jo Lee, MD Associate Director for Rare Diseases, DRDMG, OND, CDER, FDA
Markus Ries, MD, PhD, MHSc, FCP Professor, Pediatric Neurology and Metabolic Medicine, Center for Rare Diseases, Center for Pediatric and Adolescent Medicine, Heidelberg University Hospital, Heidelberg, Germany
Steven Chang, BS EE, MS EE President & CEO, Immunetrics, Inc.
Hilary Vernon, MD, PhD Associate Professor of Genetic Medicine, Department of Genetic Medicine, Johns Hopkins University School of Medicine
Robert Schuck, PharmD, PhD Deputy Director, DTPM, OCP, CDER, FDA
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Video |
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Session 2: OVERVIEW OF QSP AND SUCCESSFUL USES OF QSP IN DRUG DEVELOPMENT
Session Chair: Jane Bai, PhD Expert Regulatory Scientist (Systems Pharmacology), DARS, OCP, CDER, FDA
Moderator: Justin Earp, PhD Lead Pharmacokineticist, DPM, OCP, OTS, CDER, FDA
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| Challenges and Opportunities for QSP in Drug Development and Regulatory Evaluation |
Stephan Schmidt, BPharm, PhD, FCP Endowed Professor, Department of Pharmaceutics, Director, Center for Pharmaceutics and Systems Pharmacology, University of Florida |
Video | Presentation (PDF)
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| Development and Application of a Quantitative Systems Pharmacology Model of Tumor Receptor Occupancy to Support New Dosing Regimens for Nivolumab Across Indications |
Brian J. Schmidt, PhD Executive Director, Head QSP & PBPK Department, Bristol Myers Squibb |
Video | Presentation (PDF)
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| QSP-Model Based Assessment of Mechanistic Similarity of Disease and Response to Olipudase alfa Between Pediatric and Adult Acid Sphingomyelinase Deficiency (ASMD) Patients |
Susana Zaph, PhD Senior Director, Head of Translational Disease Modeling-Rare & Neuro, Sanofi |
Video | Presentation (PDF)
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| Session 2 Panel Discussion |
Stephan Schmidt, BPharm, PhD, FCP Endowed Professor, Department of Pharmaceutics Director, Center for Pharmaceutics and Systems Pharmacology, University of Florida
Brian Schmidt, PhD Executive Director, Head QSP & PBPK Department, Bristol Myers Squibb
Susana Zaph, PhD Senior Director, Head of Translational Disease Modeling-Rare & Neuro, Sanofi
Rajanikanth (Raj) Madabushi, PhD Associate Director, Guidance and Scientific Policy, OCP, OTS, CDER, FDA
Christina Friedrich, PhD Chief Engineer, Rosa & Co
Jane Bai, PhD Expert Regulatory Scientist (Systems Pharmacology), DARS, OCP, CDER, FDA
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Video |
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Session 3: HOW CAN WE DEVELOP USEFUL QSP MODELS IN RARE DISEASES?
Session Chair: Audra Stinchcomb, PhD Professor, School of Pharmacy, University of Maryland
Moderator: Cynthia J Musante, PhD Vice President of Scientific Research & Global Head of QSP, Pfizer, Inc
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| Maximizing the Potential of Digital Twin Technology in Drug Development for Rare Diseases |
Tina Hernandez-Boussard, PhD, MPH, MS, FACMI Associate Dean for Research; Professor of Medicine, of Biomedical Data Science, of Surgery, and by courtesy Epidemiology and Population Health; Director, Health Informatics, Stanford Center for Clinical and Translational Research, and Education, Stanford School of Medicine |
Video | Presentation (PDF)
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| QSP Modeling Evaluates CNS Enzyme Delivery for Different Treatment Modalities for Hunter Syndrome |
Kapil Gadkar, PhD Staff Scientist, Senior Director, Denali Therapeutics |
Video | Presentation (PDF)
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| What it Will Take to Cross the Valley of Death: Dealing with Biological Heterogeneity and Epistemic Uncertainty with AgentBased Modeling Using an Adaptation of the Principle of Maximal Entropy/Ignorance |
Gary An, MD, FACS Professor of Surgery and Vice Chair of Surgical Research, Department of Surgery, University of Vermont Larner College of Medicine |
Video | Presentation (PDF)
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| Session 3 Panel Discussion |
Tina Hernandez-Boussard, PhD, MPH, MS, FACMI Associate Dean for Research; Professor of Medicine, of Biomedical Data Science, of Surgery, and by courtesy Epidemiology and Population Health; Director, Health Informatics, Stanford Center for Clinical and Translational Research, and Education, Stanford School of Medicine
Kapil Gadkar, PhD Staff Scientist, Senior Director, Denali Therapeutics
Gary An, MD, Professor of Surgery and Vice Chair of Surgical Research, Department of Surgery, Jie (Jack) Wang, PhD Hao Zhu, PhD, Mstat University of Vermont Larner College of Medicine
Jie (Jack) Wang, PhD Team Leader, Rare Diseases and Inborn Errors of Metabolism, DTPM, OCP, CDER, FDA
Hao Zhu, PhD, Mstat Division Director, Division of Pharmacometrics, OCP, OTS, CDER, FDA
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Video |
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Closing Remarks
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Jane Bai, PhD Expert Regulatory Scientist (Systems Pharmacology), DARS, OCP, CDER, FDA |
Video
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