目标试验模拟在卫生政策评估中的应用与挑战:从个体干预到群体效应的框架扩展

Target Trial Emulation in Health Policy Evaluation: Translation and Challenges from Individual Interventions to Population Effects

  • 摘要: 随机对照试验(randomized controlled trial,RCT)是评估干预措施因果效应的“金标准” ,然而在卫生政策评估领域,RCT因可行性与伦理限制而鲜少应用。目标试验模拟(target trial emulation,TTE)作为一个从临床流行病学兴起的研究框架,通过在观察性数据中明确模拟一个理想化的“目标试验”,为进行严谨的因果推断提供了可能。近年来,TTE的应用已从个体化临床干预(如药物或手术)逐渐扩展至群体水平的卫生政策评估。这种跨领域的“转化”并非简单的概念平移,而是伴随一系列方法学的调整与挑战。本文系统性梳理TTE框架从个体临床研究向卫生政策评估领域扩展的核心逻辑,深入剖析其在研究单位、干预定义、时间零点、因果估量及分析策略等关键要素上的转化与重构,探讨其面临的独特挑战(如政策异质性、交错采纳、同期政策干扰及数据局限性),同时评述工具变量、双重差分法等分析策略在该领域的应用,以期为应用真实世界数据进行高质量政策评估提供方法学参考与前瞻性思考。

     

    Abstract: Randomized controlled trials (RCTs) represent the "gold standard" for estimating the causal effects of interventions; however, their implementation in the field of health policy evaluation is frequently hindered by logistical feasibility and ethical constraints. Target Trial Emulation (TTE), a framework originating in clinical epidemiology, facilitates rigorous causal inference from observational data by explicitly emulating the design of an idealized "target trial." Recently, the application of TTE has transitioned from individual-level clinical interventions-such as pharmacological or surgical treatments-to population-level health policy evaluations. This interdisciplinary translation is not a localized conceptual shift but necessitates a series of comprehensive methodological adaptations. This paper systematically delineates the core logic of extending the TTE framework into the realm of health policy, providing a profound analysis of the transformation and reconstruction of critical elements, including study units, intervention definitions, time zero, causal estimands, and analytical strategies. Furthermore, it examines the unique challenges inherent in policy contexts, such as policy heterogeneity, staggered adoption, concurrent policy interference, and data granularity limitations. The paper also evaluates the integration of analytical methods, such as instrumental variables (IV) and difference-in-differences (DID), within the TTE framework. This synthesis aims to provide methodological guidance and prospective insights for conducting high-quality policy evaluations using real-world data (RWD).

     

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