Wu Yue-E, Zheng Yuan-Yuan, Li Qiu-Yue, Yao Bu-Fan, Cao Jing, Liu Hui-Xin, Hao Guo-Xiang, van den Anker John, Zheng Yi, Zhao Wei
Department of Clinical Pharmacy, Institute of Clinical Pharmacology, Key Laboratory of Chemical Biology (Ministry of Education), NMPA Key Laboratory for Clinical Research and Evaluation of Innovative Drug, School of Pharmaceutical Sciences, Cheeloo College of Medicine, Shandong University, Jinan, China.
Division of Clinical Pharmacology, Children's National Medical Center, Washington, DC, USA; Departments of Pediatrics, Pharmacology & Physiology, George Washington University, School of Medicine and Health Sciences, Washington, DC, USA; Department of Paediatric Pharmacology and Pharmacometrics, University Children's Hospital Basel, Basel, Switzerland.
Adv Drug Deliv Rev. 2024 Aug;211:115364. doi: 10.1016/j.addr.2024.115364. Epub 2024 Jun 25.
The challenges of drug development in pediatric, pregnant and geriatric populations are a worldwide concern shared by regulatory authorities, pharmaceutical companies, and healthcare professionals. Model-informed drug development (MIDD) can integrate and quantify real-world data of physiology, pharmacology, and disease processes by using modeling and simulation techniques to facilitate decision-making in drug development. In this article, we reviewed current MIDD policy updates, reflected on the integrity of physiological data used for MIDD and the effects of physiological changes on the drug PK, as well as summarized current MIDD strategies and applications, so as to present the state of the art of MIDD in pediatric, pregnant and geriatric populations. Some considerations are put forth for the future improvements of MIDD including refining regulatory considerations, improving the integrity of physiological data, applying the emerging technologies, and exploring the application of MIDD in new therapies like gene therapies for special populations.
儿科、孕妇和老年人群体的药物研发挑战是监管机构、制药公司和医疗保健专业人员共同面临的全球性问题。模型引导药物研发（MIDD）可以通过使用建模和模拟技术，整合和量化生理学、药理学和疾病过程的真实世界数据，以促进药物研发中的决策制定。在本文中，我们回顾了当前MIDD政策更新，反思了用于MIDD的生理学数据的完整性以及生理变化对药物药代动力学的影响，并总结了当前MIDD策略和应用，从而呈现MIDD在儿科、孕妇和老年人群体中的最新进展。针对MIDD未来的改进提出了一些考虑因素，包括完善监管考量、提高生理数据的完整性、应用新兴技术以及探索MIDD在特殊人群基因治疗等新疗法中的应用。
