Objective: Randomized clinical trial (RCT) results often define clinical decision making, but the highly curated populations of trails, and the rigorous methodology utilized, are often not reflective of real-world practice. The objective of this scoping review is to identify the ability of practical methods to transport findings from randomized clinical trials (RCTs) to target populations. Study design: A scoping review was conducted on the literature regarding transportability from RCTs to observational data cohorts. Each study was assessed for the transportability methodology used, and to what degree the RCT treatment effect (estimated target parameter) was seen in the target population (intended patients) 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 outcomes from RCTs could be extended to real-world settings, with varying degrees of effect size and precision. However, in some cases, the estimated target parameters for the target population were statistically significantly different from those in clinical trials. Conclusion: Despite variations in the magnitude of effects between RCTs and real-world studies, transportability methods can play an important role in effectively bridging the gap between clinical research and practical healthcare applications, offering valuable insights for evidence-based medicine.
翻译:摘要:目的:随机临床试验的结果常决定临床决策,但试验中高度筛选的人群及严谨的方法学往往无法反映真实世界实践。本范围综述旨在评估实用方法将随机临床试验结论迁移至目标人群的能力。研究设计:针对随机临床试验向观察性数据队列的可迁移性文献开展范围综述。每项研究均评估其使用的可迁移性方法,以及观察性数据中随机临床试验的治疗效应(目标参数估计值)在目标人群(目标患者群体)中的体现程度。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、精神病学、肿瘤学、骨科学、麻醉学与血液学。这些研究表明,随机临床试验的结局可扩展至真实世界环境,但效应量及精确度存在差异。然而,部分案例中目标人群的目标参数估计值与临床试验结果存在统计学显著差异。结论:尽管随机临床试验与真实世界研究之间的效应量存在变异,可迁移性方法在有效弥合临床研究与实际医疗应用之间的鸿沟方面发挥重要作用,为循证医学提供有价值的见解。