Rider Jennifer R, Wasserman Asher, Slipski Lukas, Carrigan Gillis, Harvey Raymond, Jiao Xiaolong, McRoy Lynn, Pace Nelson D, Becnel Lauren, Bruno Amanda, Eckert Joy C, Hodgkins Priscilla, Jain Purva, Merola David, Ovbiosa Osayi E, Natanzon Yanina, Pinheiro Simone, Quinn Jameson, Rodriguez-Watson Carla, Campbell Ulka
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Am J Epidemiol. 2025 Jun 3;194(6):1783-1793. doi: 10.1093/aje/kwae346.
By evaluating published emulations of oncology randomized control trial (RCT) studies in which both the active and comparator groups are sourced from real-world data (RWD) and target trial results are available for benchmarking, this systematic review aims to gain insight into factors related to emulation performance. Thirteen oncology emulation studies using various types of RWD were identified through an online database search of PubMed through 2022. Based on the ROBINS-I tool, most studies (n = 8) had a serious risk of overall bias driven by risk of bias from confounding. Approximately half of the studies (n = 6) fully proxied the RCT entry criteria. Of 11 RWD studies that provided sufficient detail to quantify emulation performance, the emulation hazard ratio (HR) estimate fell within the 95% confidence interval (CI) of the trial estimate in 9 of the studies. There were no clear trends between risk of bias or degree to which the entry criteria were proxied and emulation performance. Findings may have been influenced by publication bias and researcher degrees of freedom, as only one emulation study preregistered its protocol. Tools for comprehensively characterizing factors that affect emulation performance, including the real-world clinical context as it relates to the RCT research question, are needed to evaluate the feasibility of a RCT emulation. This article is part of a Special Collection on Pharmacoepidemiology.
通过评估已发表的肿瘤学随机对照试验（RCT）模拟研究，其中活性组和对照比较组均来源于真实世界数据（RWD）且有目标试验结果可用于基准比较，本系统评价旨在深入了解与模拟性能相关的因素。通过对PubMed在线数据库进行检索，截至2022年，共识别出13项使用各种类型RWD的肿瘤学模拟研究。基于ROBINS - I工具，大多数研究（n = 8）因混杂因素导致的偏倚风险而存在严重的总体偏倚风险。约一半的研究（n = 6）完全模拟了RCT纳入标准。在11项提供了足够详细信息以量化模拟性能的RWD研究中，9项研究的模拟风险比（HR）估计值落在试验估计值的95%置信区间（CI）内。偏倚风险或纳入标准被模拟的程度与模拟性能之间没有明显趋势。研究结果可能受到发表偏倚和研究者自由度的影响，因为只有一项模拟研究预先注册了其方案。需要有工具来全面表征影响模拟性能的因素，包括与RCT研究问题相关的真实世界临床背景，以评估RCT模拟的可行性。本文是药物流行病学特刊的一部分。
