Institute for Research in Operative Medicine, Faculty of Health, School of Medicine, Witten/Herdecke University, 51067 Cologne, Germany.
Institute for Research in Operative Medicine, Faculty of Health, School of Medicine, Witten/Herdecke University, 51067 Cologne, Germany.
J Clin Epidemiol. 2021 May;133:1-13. doi: 10.1016/j.jclinepi.2020.12.019. Epub 2021 Jan 13.
We assessed disagreements between nonrandomized controlled studies based on real-world data (NRCS-RWDs) and randomized controlled trials (RCTs).
We systematically searched for studies that compared treatment effect estimates from NRCS-RWDs and RCTs on the same clinical question. We assessed the potential difference between NRCS-RWDs and RCTs related to internal and external validity. We calculated various meta-epidemiological measures to assess agreement. In case of disagreements, we tried to identify the probable causes of disagreements.
We included 12 studies comparing 15 treatment effect estimates of NRCS-RWDs and RCTs. There were many potential causes of disagreement. Ninety-five percent confidence intervals overlapped for 12 of 15 treatment effect estimates. Our analysis on predicted vs. observed overlap showed that there were no more disagreements than expected by chance. We observed only two substantial differences between the 15 treatment effect estimates. In both cases, we identified risk of bias in the NRCS-RWDs as the most probable cause of disagreement.
Our findings suggest that there are clinical questions where the difference in risk of bias between a well-conducted NRCS-RWD and an RCT is negligible. In our analysis, threats to external validity appeared to have no or only a weak impact on the disagreements of treatment effect estimates.
我们评估了基于真实世界数据（NRCS-RWD）的非随机对照研究（NRCS-RWD）与随机对照试验（RCT）之间的差异。
我们系统地搜索了比较 NRCS-RWD 和 RCT 对同一临床问题的治疗效果估计的研究。我们评估了与内部和外部有效性相关的 NRCS-RWD 和 RCT 之间的潜在差异。我们计算了各种元流行病学指标来评估一致性。在存在分歧的情况下，我们试图找出分歧的可能原因。
我们纳入了 12 项研究，比较了 15 项 NRCS-RWD 和 RCT 的治疗效果估计。存在许多潜在的分歧原因。15 项治疗效果估计中有 12 项的 95%置信区间重叠。我们对预测与观察到的重叠的分析表明，分歧并不比偶然多。我们仅观察到 15 项治疗效果估计中的两个实质性差异。在这两种情况下，我们都将 NRCS-RWD 中的偏倚风险确定为分歧的最可能原因。
我们的研究结果表明，在某些临床问题上，经过良好设计的 NRCS-RWD 和 RCT 之间的偏倚风险差异可以忽略不计。在我们的分析中，外部有效性的威胁似乎对治疗效果估计的分歧没有或只有微弱的影响。
