Estimands in published protocols of randomised trials: urgent improvement needed.
Estimands in published protocols of randomised trials: urgent improvement needed.
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DOI：
10.1186/s13063-021-05644-4
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发表时间：
2021-10-09
期刊：
影响因子：
2.5
通讯作者：
Cro S
中科院分区：
文献类型：
--
作者：
Kahan BC;Morris TP;White IR;Carpenter J;Cro S
被估量是对试验（问题）中待估计的治疗效果的精确描述，与统计分析方法（如何回答问题）不同。最近发表的关于2019年临床试验中被估量使用的ICH E9（R1）附录支持了被估量用于改善试验研究和报告的潜在用途。我们着手评估被估量在已发表的试验方案中的描述情况。我们回顾了2020年10月发表在Trials和BMJ Open上的50项试验方案。对于每个方案，我们确定主要结局的被估量是否明确说明，未说明但可推断（即可以根据给定的信息构建）或不可推断。50项试验中没有一项明确描述了主要结局的被估量，在74%的试验中，无法从方案中包含的信息推断被估量。在36%的试验中无法推断被估量的群体属性，在20%的试验中无法推断治疗条件属性，在34%的试验中无法推断群体水平的汇总测量，在60%的试验中无法推断并发事件的处理（在32%的方案中，处理不依从性的策略是不可推断的，并且在80%的适用方案中无法推断处理死亡率的策略）。相反，所有试验均声明了结局属性。在28%的试验中，五个被估量属性中的三个或更多个无法推断。在已发表的试验方案中，被估量的描述很差，在大多数试验中，不可能准确地理解所估计的治疗效果。考虑到被估量对改善临床研究和报告的效用，这迫切需要改变。在线版本包含补充材料，可通过10.1186/s13063-021-05644-4获得。
An estimand is a precise description of the treatment effect to be estimated from a trial (the question) and is distinct from the methods of statistical analysis (how the question is to be answered). The potential use of estimands to improve trial research and reporting has been underpinned by the recent publication of the ICH E9(R1) Addendum on the use of estimands in clinical trials in 2019. We set out to assess how well estimands are described in published trial protocols. We reviewed 50 trial protocols published in October 2020 in Trials and BMJ Open. For each protocol, we determined whether the estimand for the primary outcome was explicitly stated, not stated but inferable (i.e. could be constructed from the information given), or not inferable. None of the 50 trials explicitly described the estimand for the primary outcome, and in 74% of trials, it was impossible to infer the estimand from the information included in the protocol. The population attribute of the estimand could not be inferred in 36% of trials, the treatment condition attribute in 20%, the population-level summary measure in 34%, and the handling of intercurrent events in 60% (the strategy for handling non-adherence was not inferable in 32% of protocols, and the strategy for handling mortality was not inferable in 80% of the protocols for which it was applicable). Conversely, the outcome attribute was stated for all trials. In 28% of trials, three or more of the five estimand attributes could not be inferred. The description of estimands in published trial protocols is poor, and in most trials, it is impossible to understand exactly what treatment effect is being estimated. Given the utility of estimands to improve clinical research and reporting, this urgently needs to change. The online version contains supplementary material available at 10.1186/s13063-021-05644-4.
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影响因子：
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