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Bridging Local and Population Causal Effects: A Proximal Instrumental Variable Approach

连接局部与总体因果效应:近端工具变量方法

Instrumental variable (IV) methods address treatment endogeneity, but with non-compliance and heterogeneous treatment effects a binary instrument generally identifies the local average treatment effect (LATE) among compliers rather than the population average treatment effect (ATE). When treatment effects and compliance probabilities are heterogeneous and dependent through latent factors, the ATE need not be identified by IV variation alone. We develop a proximal…

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One-sample survival tests in the presence of non-proportional hazards in oncology clinical trials

肿瘤临床试验中非比例风险下的单样本生存检验

In oncology, conduct well-powered time-to-event randomized clinical trials may be challenging due to limited patietns number. Many designs for single-arm trials (SATs) have recently emerged as an alternative to overcome this issue. They rely on the (modified) one-sample log-rank test (OSLRT) under the proportional hazards to compare the survival curves of an experimental and an external control group. We extend Finkelstein's formulation of OSLRT as a score test by using a…

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Causal Effect of Functional Treatment

函数型处理的因果效应

We study the causal effect with a functional treatment variable, where practical applications often arise in neuroscience, biomedical sciences, etc. Previous research concerning the effect of a functional variable on an outcome is typically restricted to exploring correlation rather than causality. The generalized propensity score, which is often used to calibrate the selection bias, is not directly applicable to a functional treatment variable due to a lack of definition of…

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Bayesian analysis of restricted mean survival time adjusted for covariates using pseudo-observations

利用伪观测进行协变量调整的限制平均生存时间贝叶斯分析

The difference in restricted mean survival time (RMST) is a clinically meaningful measure to quantify treatment effect in randomized controlled trials, especially when the proportional hazards assumption does not hold. Several frequentist methods exist to estimate RMST adjusted for covariates based on modeling and integrating the survival function. A more natural approach may be a regression model on RMST using pseudo-observations, which allows for a direct estimation…

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PCA Rerandomization

基于主成分分析的再随机化

Mahalanobis distance between treatment group and control group covariate means is often adopted as a balance criterion when implementing a rerandomization strategy. However, this criterion may not work well for high-dimensional cases because it balances all orthogonalized covariates equally. Here, we propose leveraging principal component analysis (PCA) to identify proper subspaces in which Mahalanobis distance should be calculated. Not only can PCA effectively reduce the…

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