Prospective benchmarking of an observational analysis against a randomized trial increases confidence in the benchmarking process as it relies exclusively on aligning the protocol of the trial and the observational analysis, while the trials findings are unavailable. The Randomized Evaluation of Decreased Usage of Betablockers After Myocardial Infarction (REDUCE-AMI, ClinicalTrials.gov ID: NCT03278509) trial started recruitment in September 2017 and results are expected in 2024. REDUCE-AMI aimed to estimate the effect of long-term use of beta blockers on the risk of death and myocardial following a myocardial infarction with preserved left ventricular systolic ejection fraction. We specified the protocol of a target trial as similar as possible to that of REDUCE-AMI, then emulated the target trial using observational data from Swedish healthcare registries. Had everyone followed the treatment strategy as specified in the target trial protocol, the observational analysis estimated a reduction in the 5-year risk of death or myocardial infarction of 0.8 percentage points for beta blockers compared with no beta blockers; effects ranging from an absolute reduction of 4.5 percentage points to an increase of 2.8 percentage points in the risk of death or myocardial infarction were compatible with our data under conventional statistical criteria. Once results of REDUCE-AMI are published, we will compare the results of our observational analysis against those from the trial. If this prospective benchmarking is successful, it supports the credibility of additional analyses using these observational data, which can rapidly deliver answers to questions that could not be answered by the initial trial. If benchmarking proves unsuccessful, we will conduct a “postmortem” analysis to identify the reasons for the discrepancy. Prospective benchmarking shifts the investigator focus away from an endeavour to use observational data to obtain similar results as a completed randomized trial, to a systematic attempt to align the design and analysis of the trial and the observational analysis.

针对随机试验的观察性分析的前瞻性基准测试增加了对基准测试过程的信心，因为它完全依赖于调整试验方案和观察分析，而试验结果却无法获得。心肌梗死后减少使用β受体阻滞剂的随机评估（REDUCE-AMI，ClinicalTrials.gov ID：NCT03278509）试验于 2017 年 9 月开始招募，预计将于 2024 年得出结果。REDUCE-AMI 旨在评估长期使用β受体阻滞剂的效果β受体阻滞剂对左心室收缩射血分数保留的心肌梗塞后死亡和心肌风险的影响。我们指定的目标试验方案尽可能与 REDUCE-AMI 相似，然后使用瑞典医疗保健登记处的观察数据模拟目标试验。如果每个人都遵循目标试验方案中指定的治疗策略，观察分析估计，与不使用 β 受体阻滞剂相比，使用 β 受体阻滞剂的 5 年死亡或心肌梗塞风险可降低 0.8 个百分点；死亡或心肌梗塞风险绝对降低 4.5 个百分点到增加 2.8 个百分点的影响与我们在传统统计标准下的数据一致。 REDUCE-AMI 结果公布后，我们将把观察分析结果与试验结果进行比较。如果这种前瞻性基准测试成功，它将支持使用这些观察数据进行额外分析的可信度，这些分析可以快速提供初始试验无法回答的问题的答案。如果基准测试被证明不成功，我们将进行“事后”分析，以确定差异的原因。前瞻性基准测试将研究者的注意力从努力使用观察数据获得与完成的随机试验类似的结果转移到系统地尝试使试验的设计和分析与观察分析保持一致。