目标试验模拟（TTE）是一种基于真实世界数据（RWD）进行因果推断的方法学框架，其核心思想是将观察性研究的设计模拟成假定的随机对照试验（RCT），以提高研究内部效度，为利用RWD生成高质量证据提供严谨的方法学支撑。系统总结TTE的关键设计要点（合格标准、治疗策略、治疗分配、随访计划、结局指标、因果对比、分析计划）、实施步骤、关键偏倚（永恒时间偏倚、现用药者偏倚、时变混杂、选择偏倚）控制策略，并详细分析TTE在传染病、慢性病、急重症、肿瘤等不同临床场景下的应用，为临床研究者利用RWD开展高质量因果推断提供参考。TTE在混杂因素控制、数据质量、报告一致性等方面面临的挑战仍需持续关注和解决。未来，TTE研究的发展仍需要多学科紧密合作，共同推动数据标准化和应用场景拓展，为临床实践提供更高质量的证据。

Target Trial Emulation (TTE) is a methodological framework for causal inference based on Real World Data (RWD). Its core idea is to simulate the design of observational research into a hypothetical Randomized Controlled Trial (RCT) to improve the internal validity of the research and provide rigorous methodological support for generating high-quality evidence by using RWD. This paper systematically summarizes the key design points (eligibility criteria, treatment strategies, treatment distribution, follow-up plan, outcome index, causal contrasts and analysis plan), implementation steps and key bias (including immortal time bias, prevalent user bias, time-varying confounding, and selection bias) control strategies of TTE, and analyzes in detail the application of TTE in different clinical scenarios such as infectious diseases, chronic diseases, acute and severe diseases, tumors, etc., for the use of clinical researchers. In addition, this study further discusses the challenges TTE faces in confounding factor control, data quality, report consistency and so on, which still need continuous attention and solution. In the future, the development of TTE research still needs the close cooperation of multiple disciplines to jointly promote data standardization and application scenario expansion, and provide higher quality evidence for clinical practice.

R969.4

国家自然科学基金资助项目（82374519）； 2024年岐黄学者培养项目（中医药领军人才培养项目，FCZZX043）；河南省中医药科研专项课题（2025ZY3008； LHGJ20250419）；河南中医药大学第一附属医院博士科研启动基金项目（2024BSII008）