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结合内部与外部对照对临床试验处理效应进行双重检验AI 解读

Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial patients given access to novel treatments. However, due to lack of randomization, RCT patients and external controls may differ with respect to covariates that may or may not have been measured. Hence, after controlling for measured…

安慰剂样本在观察性研究中的作用AI 解读

In an observational study, it is common to leverage known null effect to detect bias. One such strategy is to set aside a placebo sample -- a subset of data immune from the hypothesized cause-and-effect relationship. Existence of an effect in the placebo sample raises concern of unmeasured confounding bias while absence of it corroborates the causal conclusion. This paper establishes a formal framework for using a placebo sample to detect and remove bias. We state…

差异性选择下聚类观察研究因果效应的非参数识别AI 解读

The clustered observational study (COS) design is the observational study counterpart to the clustered randomized trial. In a COS, a treatment is assigned to intact groups, and all units within the group are exposed to the treatment. However, the treatment is non-randomly assigned. COSs are common in both education and health services research. In education, treatments may be given to all students within some schools but withheld from all students in other schools. In health…

整合实验与观测数据的极小极大收敛率和自适应性AI 解读

Randomized controlled trials (RCTs) are the gold standard for evaluating the causal effect of a treatment; however, they often have limited sample sizes and sometimes poor generalizability. On the other hand, non-randomized, observational data derived from large administrative databases have massive sample sizes and better generalizability, but they are prone to unmeasured confounding bias. It is thus of considerable interest to reconcile effect estimates obtained from…