Objective: Randomized controlled trial (RCT) results often inform clinical decision-making, but the highly curated populations of trials and the care provided during the trial are often not reflective of real-world practice. The objective of this scoping review is to identify the ability of methods to transport findings from RCTs to target populations. Study design: A scoping review was conducted on the literature focusing on the transportability of the results from RCTs to observational cohorts. Each study was assessed based on the methodology used for transportability and the extent to which the treatment effect from the RCT was estimated in the target population in observational data. Results: A total of 15 published papers were included. The research topics include cardiovascular diseases, infectious diseases, psychiatry, oncology, orthopedics, anesthesiology, and hematology. These studies show that the findings from RCTs could be translated to real-world settings, with varying degrees of effect size and precision. In some cases, the estimated treatment effect for the target population were statistically significantly different from those in RCTs. Conclusion: Despite variations in the magnitude of effects between RCTs and real-world studies, transportability methods play an important role in effectively bridging the RCTs and real-world care delivery, offering valuable insights for evidence-based medicine.
翻译:目的:随机对照试验(RCT)的结果常为临床决策提供依据,但试验中高度筛选的人群及试验期间提供的护理往往无法反映真实世界实践。本项范围综述旨在识别将RCT结果移植至目标人群的方法能力。研究设计:通过范围综述,聚焦将RCT结果可移植至观察性队列的相关文献。根据可移植性所采用的方法学,以及RCT的治疗效应在观察数据的目标人群中估算程度,对每项研究进行评估。结果:共纳入15篇已发表论文。研究主题涵盖心血管疾病、感染性疾病、精神医学、肿瘤学、骨科学、麻醉学及血液病学。这些研究表明,RCT的发现可转化至真实世界场景,但效应量与精确性存在差异。部分案例中,目标人群的治疗效应估计值与RCT结果存在统计学显著差异。结论:尽管RCT与真实世界研究之间的效应量存在差异,可移植性方法在有效衔接RCT与真实世界医疗实践方面发挥重要作用,为循证医学提供宝贵见解。