Nested Randomized Controlled Trials in Large Databases: An Opportunity for Inflammatory Bowel Disease?

Maria Jose Temido, Sailish Honap, Silvio Danese, Vipul Jairath, Fernando Magro, Francisco Portela, Laurent Peyrin-Biroulet
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Abstract

Although randomized controlled trials (RCTs) are the gold standard for investigating the efficacy and safety of interventions, they present major operational challenges due to their complexity, time-consuming nature, and high costs. To address some of these difficulties, RCTs nested in cohorts (RCTsNC) have been developed to enable patient enrolment and randomization from existing databases. RCTsNC is an emerging trial design, which has been successfully utilized across several medical disciplines but not inflammatory bowel disease (IBD). This narrative review outlines the principles of RCTsNC and discusses the numerous advantages it affords for IBD, including harnessing longer-term longitudinal data for safety and efficacy assessment, and enhanced recruitment and follow up processes. Leveraging pre-existing cohorts and their organizational structures improves patient acceptance and is more economical compared to traditional randomized trials. Observational data for IBD, derived from research (cohort and case-control studies) and non-research sources (electronic health records and registries), provides access to comprehensive records for a large number of IBD patients. It permits researchers to address knowledge gaps in IBD where traditional RCTs have had a limited role, such as specific sub-populations typically excluded from pivotal trials, or assessing the effect of environmental exposures on disease course. This review also details caveats of this study design that include the risk of selection bias and constraints related to comparisons with placebo. In conclusion, RCTsNC offers a promising opportunity IBD research given the challenges of the current IBD RCT landscape.

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大型数据库中的嵌套随机对照试验:炎症性肠病的机会?
尽管随机对照试验(RCT)是研究干预措施有效性和安全性的黄金标准,但由于其复杂性、耗时性和高成本,在操作上面临着巨大挑战。为了解决其中一些困难,人们开发了嵌套在队列中的 RCTsNC(RCTsNC),以便从现有数据库中招募患者并进行随机化。RCTsNC 是一种新兴的试验设计,已成功应用于多个医学学科,但炎症性肠病 (IBD) 尚未应用。本综述概述了 RCTsNC 的原理,并讨论了它为 IBD 带来的诸多优势,包括利用长期纵向数据进行安全性和疗效评估,以及改进招募和随访流程。与传统的随机试验相比,利用已有的队列及其组织结构可提高患者的接受度,而且更经济。通过研究(队列和病例对照研究)和非研究来源(电子健康记录和登记处)获得的 IBD 观察数据,可以获取大量 IBD 患者的全面记录。它允许研究人员填补传统 RCT 作用有限的 IBD 知识空白,例如通常被排除在关键试验之外的特定亚人群,或评估环境暴露对疾病进程的影响。本综述还详细介绍了这种研究设计的注意事项,包括选择偏倚的风险以及与安慰剂进行比较的相关限制。总之,鉴于当前 IBD RCT 面临的挑战,RCTsNC 为 IBD 研究提供了一个大有可为的机会。
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