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随机对照试验中存在竞争性伴发事件的处理效应估计AI 解读

The analysis of randomized controlled trials is often complicated by intercurrent events (IEs) -- events that occur after treatment initiation and affect either the interpretation or existence of outcome measurements. Examples include treatment discontinuation or the use of additional medications. In two recent clinical trials for systemic lupus erythematosus with complications of IEs, we classify the IEs into two broad categories: effect-informative (e.g., treatment…

临床试验常用模式混合模型的直接估计AI 解读

Pattern-mixture models have received increasing attention as they are commonly used to assess treatment effects in primary or sensitivity analyses for clinical trials with nonignorable missing data. Pattern-mixture models have traditionally been implemented using multiple imputation, where the variance estimation may be a challenge because the Rubin's approach of combining between- and within-imputation variance may not provide consistent variance estimation while bootstrap…

主分层处理效应中的选择偏差AI 解读

Estimation of treatment effect for principal strata has been studied for more than two decades. Existing research exclusively focuses on the estimation, but there is little research on forming and testing hypotheses for principal stratification-based estimands. In this brief report, we discuss a phenomenon in which the true treatment effect for a principal stratum may not equal zero even if the two treatments have the same effect at patient level which implies an equal…

利用多重插补估计依从者的治疗效应AI 解读

Randomized controlled trials are considered the gold standard to evaluate the treatment effect (estimand) for efficacy and safety. According to the recent International Council on Harmonisation (ICH)-E9 addendum (R1), intercurrent events (ICEs) need to be considered when defining an estimand, and principal stratum is one of the five strategies to handle ICEs. Qu et al. (2020, Statistics in Biopharmaceutical Research 12:1-18) proposed estimators for the adherer average causal…