Chevret Sylvie, Timsit Jean-François, Biard Lucie
Department of Biostatistics, Hôpital Saint-Louis, Paris, France.
ECSTRRA Team, INSERM U1153,Université de Paris, 75010, Paris, France.
BMC Med Res Methodol. 2022 Dec 15;22(1):321. doi: 10.1186/s12874-022-01769-5.
To improve the efficiency of clinical trials, leveraging external data on control and/or treatment effects, which is almost always available, appears to be a promising approach.
We used data from the experimental arm of the Covidicus trial evaluating high-dose dexamethasone in severely ill and mechanically ventilated COVID-19 patients, using published data from the Recovery trial as external data, to estimate the 28-day mortality rate. Primary approaches to deal with external data were applied.
Estimates ranged from 0.241 ignoring the external data up to 0.294 using hierarchical Bayesian models. Some evidence of differences in mortality rates between the Covidicus and Recovery trials were observed, with an matched adjusted odds ratio of death in the Covidicus arm of 0.41 compared to the Recovery arm.
These indirect comparisons appear sensitive to the method used. None of those approaches appear robust enough to overcome randomized clinical trial data.
Covidicus Trial: NCT04344730, First Posted: 14/04/2020; Recovery trial: NCT04381936.
为提高临床试验效率，利用几乎总是可获取的关于对照和/或治疗效果的外部数据似乎是一种很有前景的方法。
我们使用了Covidicus试验实验组的数据，该试验评估高剂量地塞米松对重症且接受机械通气的新冠肺炎患者的疗效，将恢复试验的已发表数据用作外部数据来估计28天死亡率。应用了处理外部数据的主要方法。
估计值范围从忽略外部数据时的0.241到使用分层贝叶斯模型时的0.294。观察到Covidicus试验和恢复试验之间死亡率存在一些差异证据，Covidicus组与恢复组相比，死亡的匹配调整比值比为0.41。
这些间接比较似乎对所使用的方法敏感。这些方法中没有一种看起来强大到足以克服随机临床试验数据。
Covidicus试验：NCT04344730，首次发布：2020年4月14日；恢复试验：NCT04381936。
