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随机化后伴随用药差异下的生存结局处理效应估计AI 解读

In placebo-controlled randomized trials, the post-randomization use of concomitant medications may be higher in the placebo arm than in the treatment arm. This may dilute the full benefits of the randomized drug as estimated by the intention-to-treat analysis. We focus on cardiovascular outcomes trials in type-2 diabetes patients of glucose-lowering treatments where patients in the placebo arm are more likely to add other glucose-lowering agents with established…

利用外部对照提高随机试验累积发生率估计的精度AI 解读

Augmenting the control arm in clinical trials with external data can improve statistical power for demonstrating treatment effects. In many time-to-event outcome trials, participants are subject to truncation by death. Direct application of methods for competing risks analysis on the joint data may introduce bias, for example, due to covariate shifts between the populations. In this work, we consider transportability of the conditional cause-specific hazard of the event of…

近端间接比较AI 解读

We consider the problem of indirect comparison, where a treatment arm of interest is absent by design in one randomized controlled trial but available in the other. The former is the target trial, and the latter is the source trial. The identifiability of the target population average treatment effect often relies on conditional transportability assumptions. However, it is a common concern whether all relevant effect modifiers are measured and controlled for. We give a new…

利用多来源数据高效估计目标总体平均处理效应AI 解读

We consider estimation of the target population average treatment effect (TATE) when outcome information is unavailable. Instead, we observe the outcome in multiple source populations and wish to combine the treatment effects therein to make inference on the TATE. In contrast to existing works that assume transportability on the conditional distribution of potential outcomes or conditional treatment-specific means, we work under a weaker form of effect transportability.…