Yang Kyunghee, Gonzalez Daniel, Woodhead Jeffrey L, Bhargava Pallavi, Ramanathan Murali
Quantitative Systems Pharmacology Solutions, Simulations Plus Inc, North Carolina, USA.
Division of Clinical Pharmacology, Department of Medicine, Duke University School of Medicine, Durham, North Carolina, USA.
Clin Transl Sci. 2025 Jun;18(6):e70272. doi: 10.1111/cts.70272.
Incorporating inter-individual differences in drug disposition and responses is essential for ensuring the safe and effective use of drugs in real-world patients. Despite ongoing efforts, lower participation of children, older individuals, pregnant and breastfeeding women, postmenopausal women, and people with disease states and disabilities in drug clinical trials is frequent, and it requires multifaceted strategies and tools to evaluate drug exposure and responses in broad populations. The availability of modeling and simulation tools, such as physiologically based pharmacokinetic (PBPK) and quantitative systems pharmacology/toxicology (QSP/QST) modeling, enables the application of virtual populations that reflect the differences in drug disposition and responses for disease states and different stages of the lifespan. These models integrate clinical trial and real-world data (RWD) to predict drug exposure, efficacy, and safety. Additionally, machine learning (ML) and artificial intelligence (AI) offer powerful tools for analyzing large datasets and identifying key physiological determinants of drug response across the lifespan. This review discusses the application of in silico and AI models to advance the prediction of drug exposure and responses across the lifespan, including examples of virtual populations in PBPK and QSP/QST models. A case study on QST modeling for drug-induced liver injury (DILI) in postmenopausal women is presented, along with opportunities and challenges in applying AI for modeling physiological determinants of drug dosing in individuals ranging in age from 12 to > 80 years old in drug development.
纳入个体间药物处置和反应的差异对于确保在现实世界患者中安全有效地使用药物至关重要。尽管一直在努力,但儿童、老年人、孕妇和哺乳期妇女、绝经后妇女以及患有疾病和残疾的人在药物临床试验中的参与率仍然较低,这需要多方面的策略和工具来评估广泛人群中的药物暴露和反应。基于生理的药代动力学(PBPK)和定量系统药理学/毒理学(QSP/QST)建模等建模和模拟工具的可用性,使得能够应用反映疾病状态和生命周期不同阶段药物处置和反应差异的虚拟人群。这些模型整合临床试验和真实世界数据(RWD)以预测药物暴露、疗效和安全性。此外,机器学习(ML)和人工智能(AI)为分析大型数据集和识别整个生命周期药物反应的关键生理决定因素提供了强大工具。本综述讨论了计算机模拟和AI模型在推进整个生命周期药物暴露和反应预测方面的应用,包括PBPK和QSP/QST模型中虚拟人群的示例。还介绍了一项针对绝经后妇女药物性肝损伤(DILI)的QST建模案例研究,以及在药物开发中应用AI对年龄在12岁至80岁以上个体的药物剂量生理决定因素进行建模的机遇和挑战。