《生命科学》 2026, 38(2): 268-280

虚拟临床试验技术与应用态势分析

靳晨琦¹ , 孟 晶² , 施慧琳¹ , 许 丽^1,* , 李积宗^3,4,*

¹中国科学院上海生命科学信息中心，中国科学院上海营养与健康研究所，上海 200031 ²复旦大学附属妇产科医院，上海 200011 ³上海市生物医药技术研究院，上海 200237 ⁴长三角国家技术创新中心，上海长三角技术创新研究院，上海 201210

摘　要：

虚拟临床试验作为一种以计算模型为核心的工具，正逐步改变传统的药物与医疗器械研发模式。随着建模与仿真技术、人工智能技术以及数字孪生技术的迭代与融合应用，虚拟临床试验技术体系逐步丰富与完善。多元化技术持续赋能药物与医疗器械全生命周期研发，药物发现、临床前以及临床试验等各个阶段的应用持续拓展与深化，大幅提升了研发效率并降低了成本。相关监管框架进一步完善，模型可信度评估等标准也逐步建立，计算机模型与人工智能生成的虚拟证据在产品审评审批中的应用也日益受到重视。尽管还面临数据基础、模型解释、监管标准及技术转化等问题，未来依赖跨尺度模型整合验证、标准化数据生态建设与全球监管的协同推进，虚拟临床试验有望深度赋能生物医药研发的转型升级。

通讯作者：许 丽 , Email:xuli@sinh.ac.cn 李积宗 , Email:jizongli@stcsm.sh.gov.cn

Advances and trends in technology and application of virtual clinical trials

JIN Chen-Qi¹ , MENG Jing² , SHI Hui-Lin¹ , XU Li^1,* , LI Ji-Zong^3,4,*

¹Shanghai Information Center for Life Sciences, Shanghai Institute of Nutrition and Health, Chinese Academy of Sciences, Shanghai 200031, China ²Obstetrics & Gynecology Hospital of Fudan University, Shanghai 200011, China ³Shanghai Institute for Biomedical and Pharmaceutical Technologies, Shanghai 200237, China ⁴National Innovation Center par Excellence, Shanghai Yangtze Delta Innovation Institute, Shanghai 201210, China

Abstract:

Virtual clinical trials (VCTs), as a computational model-centered tool, are progressively transforming the traditional research and development (R&D) paradigms for drugs and medical devices. With the iterative advancement and integrated application of computer modeling and simulation technologies, artificial intelligence (AI), and digital twin (DT), the VCT technology framework is continuously being enriched and refined. These diversified technologies continuously empower the entire lifecycle R&D of drugs and medical devices, with applications expanding and deepening across stages including drug discovery, preclinical research, and clinical trials, significantly enhancing R&D efficiency and reducing costs. The regulatory framework has been further refined, standards such as model credibility assessment are being gradually established, and the application of computer models and AI-generated virtual evidence in product evaluation and approval is receiving increasing attention. Despite existing challenges related to data foundations, model interpretability, regulatory standards, and technology translation, future advancements will rely on the integrated verification of multi-scale models, the construction of standardized data ecosystems, and the coordinated promotion of global regulatory harmonization. VCTs are expected to profoundly empower the transformation and upgrading of biomedical R&D.

Communication Author：XU Li , Email:xuli@sinh.ac.cn LI Ji-Zong , Email:jizongli@stcsm.sh.gov.cn