Quantitative Systems Pharmacology for Rare Disease Drug Development |
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| Institution: | 1. Department of Pharmacology & Toxicology, University of Texas Medical Branch, Galveston, TX, USA;2. Department of Neurosurgery, The University of Texas MD Anderson Cancer Center, Houston, TX, USA;3. Department of Gynecology & Obstetrics, Johns Hopkins University, Baltimore, MD, USA;4. Center for Biomedical Engineering, University of Texas Medical Branch, Galveston, TX, USA;5. Department of Obstetrics and Gynecology, University of Texas Medical Branch, Galveston, TX, USA;6. Department of Anesthesiology, University of Texas Medical Branch, Galveston, TX, USA;7. Department of Biochemistry and Molecular Biology, University of Texas Medical Branch, Galveston, TX, USA;8. Department of Pharmacology & Toxicology, Faculty of Pharmacy, Al-Azhar University, Cairo, Egypt;1. Research Institute for Biomaterials, Tech Institute for Advanced Materials, College of Materials Science and Engineering, Suqian Advanced Materials Industry Technology Innovation Center, NJTech-BARTY Joint Research Center for Innovative Medical Technology, Nanjing Tech University, Nanjing, China;2. State Key Laboratory of Mechanics and Control of Mechanical Structures, Nanjing University of Aeronautics and Astronautics, Nanjing, China;1. Laboratory for Medicinal Chemistry Research, Shionogi & Co., Ltd., Osaka, 561-0825, Japan;2. Sustainability Management Department, Shionogi & Co., Ltd., Osaka, 541-0045, Japan;3. Intellectual Property Department, Shionogi & Co., Ltd., Osaka, 541-0045, Japan;4. Analysis and Evaluation Laboratory, Shionogi & Co., Ltd., Osaka, 561-0825, Japan;1. Biologics Drug Product Development, Novartis Pharma, Langkampfen, Austria;2. Biologics Analytical Development, Novartis Pharma, Langkampfen, Austria;1. Department of Pharmacy, National and Kapodistrian University of Athens, Zografou, Greece;2. Janssen Pharmaceutica NV, Beerse, Belgium;3. Chemical & Pharmaceutical Development, Merck Healthcare KGaA, The healthcare business of Merck KGaA, Darmstadt, Germany;4. Dissolution & Biopharmaceutics, Analytical Research and Development, Technical Research and Development, Novartis AG, CH-4056, Basel, Switzerland;5. Pharmaceutical Development, Technical Research and Development, Novartis AG, CH-4056, Basel, Switzerland;6. Pharmaceutical Development, Technical Research and Development, Novartis Pharmaceuticals Corporation, Fort Worth TX 76134, United States of America;1. Institute of Pharmacy and Molecular Biotechnology, Ruprecht-Karls-University, Im Neuenheimer Feld 329, 69120 Heidelberg, Germany;2. School of Pharmacy, University of Eastern Finland, P.O. Box 1627, 70211 Kuopio, Finland;3. Drug Research Program, Division of Pharmaceutical Biosciences, Faculty of Pharmacy, University of Helsinki, Viikinkaari 5 E, P.O. Box 56, Helsinki, 00014, Finland |
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| Abstract: | Though hundreds of drugs have been approved by the US Food and Drug Administration (FDA) for treating various rare diseases, most rare diseases still lack FDA-approved therapeutics. To identify the opportunities for developing therapies for these diseases, the challenges of demonstrating the efficacy and safety of a drug for treating a rare disease are highlighted herein. Quantitative systems pharmacology (QSP) has increasingly been used to inform drug development; our analysis of QSP submissions received by FDA showed that there were 121 submissions as of 2022, for informing rare disease drug development across development phases and therapeutic areas. Examples of published models for inborn errors of metabolism, non-malignant hematological disorders, and hematological malignancies were briefly reviewed to shed light on use of QSP in drug discovery and development for rare diseases. Advances in biomedical research and computational technologies can potentially enable QSP simulation of the natural history of a rare disease in the context of its clinical presentation and genetic heterogeneity. With this function, QSP may be used to conduct in-silico trials to overcome some of the challenges in rare disease drug development. QSP may play an increasingly important role in facilitating development of safe and effective drugs for treating rare diseases with unmet medical needs. |
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