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Bounds on the conditional and average treatment effect with unobserved confounding factors

存在未观测混杂时条件与平均处理效应的界

For observational studies, we study the sensitivity of causal inference when treatment assignments may depend on unobserved confounders. We develop a loss minimization approach for estimating bounds on the conditional average treatment effect (CATE) when unobserved confounders have a bounded effect on the odds ratio of treatment selection. Our approach is scalable and allows flexible use of model classes in estimation, including nonparametric and black-box machine learning…

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Estimation and Validation of Ratio-based Conditional Average Treatment Effects Using Observational Data

利用观察数据估计与验证比值型条件平均处理效应

While sample sizes in randomized clinical trials are large enough to estimate the average treatment effect well, they are often insufficient for estimation of treatment-covariate interactions critical to studying data-driven precision medicine. Observational data from real world practice may play an important role in alleviating this problem. One common approach in trials is to predict the outcome of interest with separate regression models in each treatment arm, and…

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Model-free approach to quantifying the proportion of treatment effect explained by a surrogate marker

无模型方法量化替代标志物解释的治疗效应比例

Summary In randomized clinical trials, the primary outcome, $Y$, often requires long-term follow-up and/or is costly to measure. For such settings, it is desirable to use a surrogate marker, $S$, to infer the treatment effect on $Y$, $\Delta$. Identifying such an $S$ and quantifying the proportion of treatment effect on $Y$ explained by the effect on $S$ are thus of great importance. Most existing methods for quantifying the proportion of treatment effect are model based and…

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Robust Alternatives to ANCOVA for Estimating the Treatment Effect via a Randomized Comparative Study

随机比较研究中估计处理效应的稳健 ANCOVA 替代方法

In comparing two treatments via a randomized clinical trial, the analysis of covariance (ANCOVA) technique is often utilized to estimate an overall treatment effect. The ANCOVA is generally perceived as a more efficient procedure than its simple two sample estimation counterpart. Unfortunately, when the ANCOVA model is nonlinear, the resulting estimator is generally not consistent. Recently, various nonparametric alternatives to the ANCOVA, such as the augmentation methods,…

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A Simple Method for Estimating Interactions Between a Treatment and a Large Number of Covariates

估计处理与大量协变量交互作用的简便方法

We consider a setting in which we have a treatment and a potentially large number of covariates for a set of observations, and wish to model their relationship with an outcome of interest. We propose a simple method for modeling interactions between the treatment and covariates. The idea is to modify the covariate in a simple way, and then fit a standard model using the modified covariates and no main effects. We show that coupled with an efficiency augmentation procedure,…

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Landmark Estimation of Survival and Treatment Effect in a Randomized Clinical Trial

随机临床试验中生存率与治疗效应的界标估计

In many studies with a survival outcome, it is often not feasible to fully observe the primary event of interest. This often leads to heavy censoring and thus, difficulty in efficiently estimating survival or comparing survival rates between two groups. In certain diseases, baseline covariates and the event time of nonfatal intermediate events may be associated with overall survival. In these settings, incorporating such additional information may lead to gains in efficiency…

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Effectively Selecting a Target Population for a Future Comparative Study

为未来比较研究有效选择目标人群

When comparing a new treatment with a control in a randomized clinical study, the treatment effect is generally assessed by evaluating a summary measure over a specific study population. The success of the trial heavily depends on the choice of such a population. In this paper, we show a systematic, effective way to identify a promising population, for which the new treatment is expected to have a desired benefit, utilizing the data from a current study involving similar…

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