Abstract:
The past decade has seen a substantial increase in the number of randomized controlled trials (RCTs) conducted in inflammatory bowel disease (IBD). Randomized controlled trials are the gold standard method for generating robust evidence of drug safety and efficacy but are expensive, time-consuming, and may have ethical implications. Observational studies in IBD are often used to fill the gaps in evidence but are typically hindered by significant bias. There are several approaches for making statistical inferences from observational data with some that focus on study design and others on statistical techniques. Target trial emulation is an emerging methodological process that aims to bridge this gap and improve the quality of observational studies by applying the principles of an ideal, or \"target,\" randomized trial to routinely collected clinical data. There has been a rapid expansion of observational studies that have emulated trials over the past 5 years in other medical fields, but this has yet to be adopted in gastroenterology and IBD. The wealth of nonrandomized clinical data available through electronic health records, patient registries, and administrative health databases afford innumerable hypothesis-generating opportunities for IBD research. This review outlines the principles of target trial emulation, discusses the merits to IBD observational studies in reducing the most common biases and improving confidence in causality, and details the caveats of using this approach.
Target trial emulation uses observational data to mimic the principles of an ideal or “target” randomized trial. This framework offers several opportunities to strengthen the quality of observational research in inflammatory bowel disease by reducing common sources of bias.
摘要:
在过去的十年中,在炎症性肠病(IBD)中进行的随机对照试验(RCT)的数量大大增加。随机对照试验是产生药物安全性和有效性的有力证据的黄金标准方法,但价格昂贵,耗时,可能会产生伦理影响。IBD的观察性研究通常用于填补证据空白,但通常受到明显偏见的阻碍。有几种方法可以从观察数据中得出统计推断,其中一些方法侧重于研究设计,另一些方法侧重于统计技术。目标试验仿真是一个新兴的方法学过程,旨在通过应用理想的原则来弥合这一差距并提高观察研究的质量,或\"目标,“随机试验常规收集临床数据。在过去的5年中,其他医学领域的观察性研究迅速扩展,但这尚未在胃肠病学和IBD中采用。通过电子健康记录获得的大量非随机临床数据,病人登记处,和行政健康数据库为IBD研究提供了无数的假设生成机会。这篇综述概述了目标试验仿真的原则,讨论了IBD观察性研究在减少最常见偏差和提高因果关系信心方面的优点,并详细说明了使用这种方法的注意事项。
目标试验仿真使用观察数据来模拟理想或“目标”随机试验的原理。该框架通过减少常见的偏倚来源,为增强炎症性肠病的观察性研究质量提供了几个机会。