因果推理
推论
估计员
结果(博弈论)
临床试验
随机对照试验
计量经济学
心理学
医学
计算机科学
统计
内科学
数学
人工智能
数理经济学
作者
Yongming Qu,Junxiang Luo,Stephen J. Ruberg
摘要
Summary Intercurrent events (ICEs) and missing values are inevitable in clinical trials of any size and duration, making it difficult to assess the treatment effect for all patients in randomized clinical trials. Defining the appropriate estimand that is relevant to the clinical research question is the first step in analyzing data. The tripartite estimands, which evaluate the treatment differences in the proportion of patients with ICEs due to adverse events, the proportion of patients with ICEs due to lack of efficacy, and the primary efficacy outcome for those who can adhere to study treatment under the causal inference framework, are of interest to many stakeholders in understanding the totality of treatment effects. In this manuscript, we discuss the details of how to estimate tripartite estimands based on a causal inference framework and how to interpret tripartite estimates through a phase 3 clinical study evaluating a basal insulin treatment for patients with type 1 diabetes.
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