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Guide on Methodological Standards in Pharmacoepidemiology (2nd edition) and their series interpretation (14): research designs and case studies of target trial emulation

Published on Feb. 27, 2026Total Views: 38 times Total Downloads: 10 times Download Mobile

Author: ZHAO Houyu 1, 2 CHENG Yinchu 3 SUN Feng 2, 4, 5, 6, 7 ZHAN Siyan 1, 2, 4, 8

Affiliation: 1. Research Center of Clinical Epidemiology, Peking University Third Hospital, Beijing 100191, China 2. Key Laboratory of Epidemiology of Major Diseases (Peking University), Ministry of Education, Beijing 100191, China 3. Department of Pharmacy, Peking University Third Hospital, Beijing 100191, China 4. Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing 100191, China 5. Department of Ophthalmology, Peking University Third Hospital, Beijing 100191, China 6. School of Traditional Chinese Medicine, Xinjiang Medical Univerisity, Urumqi 830017, China 7. School of Public Health, Shihezi Univerisity, Shihezi 832000, Xinjiang Uygur Autonomous Region, China 8. Center for Intelligent Public Health, Institute for Artificial Intelligence, Peking University, Beijing 100191, China

Keywords: Target trial emulation Pharmacoepidemiology Methodology Guidelines Active comparator new-user design Sequential trial Clone-censor-weight

DOI: 10.12173/j.issn.1005-0698.202601123

Reference: ZHAO Houyu, CHENG Yinchu, SUN Feng, ZHAN Siyan. Guide on Methodological Standards in Pharmacoepidemiology (2nd edition) and their series interpretation (14): research designs and cases studies of target trial emulation[J]. Yaowu Liuxingbingxue Zazhi, 2026, 35(2): 121-132. DOI: 10.12173/j.issn.1005-0698.202601123.[Article in Chinese]

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Abstract

Target trial emulation (TTE) has gradually developed into a mainstream framework for causal inference in pharmacoepidemiological research. By emulating the design elements of randomized controlled trials (RCTs), TTE significantly enhances the quality of observational studies. Based on the Guide on Methodological Standards in Pharmacoepidemiology (2nd edition), this article interprets the fundamental principles of commonly used TTE designs. It focuses on introducing the basic principles, applicable scenarios, and implementation steps of the three commonly used TTE designs: the active comparator new-user (ACNU) design, sequential trials, and the clone-censor-weight (CCW) approach. Typical case studies are presented to illustrate the specific applications of each design in pharmacoepidemiology. Finally, this article compares the fundamental characteristics of these three designs and offers guidance on selecting an appropriate design type based on research objectives.

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References

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