Review of tools to support Target Trial Emulation

IF 4.5 2区医学 Q2 COMPUTER SCIENCE, INTERDISCIPLINARY APPLICATIONS

Journal of Biomedical Informatics Pub Date : 2025-09-26 DOI:10.1016/j.jbi.2025.104897

Christina A. van Hal , Elmer V. Bernstam , Todd R. Johnson

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引用次数: 0

Abstract

Objective:

Randomized Controlled Trials (RCTs) are the gold standard for clinical evidence, but ethical and practical constraints sometimes necessitate or warrant the use of observational data. The aim of this study is to identify informatics tools that support the design and conduct of Target Trial Emulations (TTEs), a framework for designing observational studies that closely emulate RCTs so as to minimize biases that often arise when using real-world evidence (RWE) to estimate causal effects.

Methods:

We divided the process of conducting TTEs into three phases and seven steps. We then systematically reviewed the literature to identify currently available tools that support one or more of the seven steps required to conduct a TTE. For each tool, we noted which step or steps the tool supports.

Results:

7625 papers were included in the initial review, with 76 meeting our inclusion criteria. Our review identified 24 distinct tools applicable to the three phases of TTE. Specifically, 3 tools support the Design Phase, 5 support the Implementation Phase, and 19 support the Analysis Phase, with some tools applicable to multiple phases.

Conclusion:

This review revealed significant gaps in tool support for the Design Phase of TTEs, while support for the Implementation and Analysis phases was highly variable. No single tool currently supports all aspects of TTEs from start to finish and few tools are interoperable, meaning they cannot be easily integrated into a unified workflow. The results highlight the need for further development of informatics tools for supporting TTEs.

Abstract Image

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回顾支持目标试验仿真的工具。

目的：随机对照试验（RCTs）是临床证据的黄金标准，但伦理和实践限制有时需要或保证使用观察性数据。本研究的目的是确定支持目标试验模拟（TTEs）设计和实施的信息学工具，目标试验模拟是一种设计密切模仿随机对照试验的观察性研究的框架，以便最大限度地减少使用真实世界证据（RWE）估计因果效应时经常出现的偏差。方法：将其分为3个阶段和7个步骤。然后，我们系统地回顾了文献，以确定当前可用的工具，这些工具支持进行TTE所需的七个步骤中的一个或多个步骤。对于每个工具，我们记录了该工具支持的步骤。结果：初审共纳入7625篇论文，其中76篇符合我们的纳入标准。我们的审查确定了适用于TTE三个阶段的24种不同工具。具体来说，有3个工具支持设计阶段，5个工具支持实现阶段，19个工具支持分析阶段，其中一些工具适用于多个阶段。结论：这篇综述揭示了对设计阶段的工具支持的显著差距，而对实施和分析阶段的支持是高度可变的。目前还没有一个工具能够从头到尾支持tte的所有方面，而且很少有工具是可互操作的，这意味着它们不能很容易地集成到一个统一的工作流中。研究结果强调了进一步开发支持tts的信息学工具的必要性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Biomedical Informatics 医学-计算机：跨学科应用

CiteScore

8.90

自引率

6.70%

发文量

243

审稿时长

32 days

期刊介绍： The Journal of Biomedical Informatics reflects a commitment to high-quality original research papers, reviews, and commentaries in the area of biomedical informatics methodology. Although we publish articles motivated by applications in the biomedical sciences (for example, clinical medicine, health care, population health, and translational bioinformatics), the journal emphasizes reports of new methodologies and techniques that have general applicability and that form the basis for the evolving science of biomedical informatics. Articles on medical devices; evaluations of implemented systems (including clinical trials of information technologies); or papers that provide insight into a biological process, a specific disease, or treatment options would generally be more suitable for publication in other venues. Papers on applications of signal processing and image analysis are often more suitable for biomedical engineering journals or other informatics journals, although we do publish papers that emphasize the information management and knowledge representation/modeling issues that arise in the storage and use of biological signals and images. System descriptions are welcome if they illustrate and substantiate the underlying methodology that is the principal focus of the report and an effort is made to address the generalizability and/or range of application of that methodology. Note also that, given the international nature of JBI, papers that deal with specific languages other than English, or with country-specific health systems or approaches, are acceptable for JBI only if they offer generalizable lessons that are relevant to the broad JBI readership, regardless of their country, language, culture, or health system.