Design and Implementation of Observational Studies Emulating a Target Trial

作者：Yan Ren, Yulong Jia, Lingyan Liu, Houchen Lyv, Liyuan Tao, Yuezhen Li, Peng Zhao, Yiquan Xiong, Guowei Li, Jiayue Xu, Chunrong Liu, YongMei Lu, Kang Zou, Jing Tan, Xin Sun · 发表于：JAMA Network Open · 年份：2026 · DOI：10.1001/jamanetworkopen.2025.58262 · 被引用次数：7 · 研究领域：Advanced Causal Inference Techniques、Meta-analysis and systematic reviews、Genetic Associations and Epidemiology

Importance: Target trial emulation (TTE) is increasingly used to estimate causal effects with observational data, yet its clinical applications and methodologic quality remain unclear. Objective: To characterize the clinical scenarios where TTE has been applied, assess methodologic limitations in design and implementation, and provide structured recommendations for strengthening future practice. Design, Setting, and Participants: This cross-sectional study searched PubMed for TTE studies published from January 1, 2017, to December 15, 2023, in clinical journals in the top Journal Impact Factor quartile, supplemented by studies from 3 prior scoping reviews. Eligible studies explicitly aimed to emulate a target trial using observational data. Data were extracted using a standardized, consensus-based tool. Main Outcomes and Measures: Study characteristics, application scenarios, specification of target trials, and adherence to 7 methodologic domains of TTE were evaluated. Recommendations were developed to address observed gaps. Results: Among 237 included studies, over half (128 [54.0%]) evaluated drug interventions, commonly in infectious diseases, cardiology, and oncology. TTE was applied in 8 recurring clinical scenarios, including randomized clinical trial replication and extending evidence to underrepresented populations or rare outcomes. TTE was most frequently applied to assess treatment effectiveness (165 studies [69.6%]), long-term outcomes (39 [16.5%]), emergency-use m...