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临床试验长期结局分位数处理效应的双重／去偏机器学习AI 解读

Long-term outcomes are often unavailable in randomized clinical trials, although short-term surrogate outcomes are commonly observed. External observational data may contain the long-term outcome, but causal comparisons based on such data alone are vulnerable to confounding. Existing surrogate-based data integration methods for long-term outcomes have focused primarily on average treatment effects. We study estimation of quantile treatment effects for long-term outcomes in…

多个实验的潜力：识别潜在结果的联合分布AI 解读

Typical causal effects are defined based on the marginal distribution of potential outcomes. However, many real-world applications require causal estimands involving the joint distribution of potential outcomes to enable more nuanced treatment evaluation and selection. In this article, we propose a novel framework for identifying and estimating the joint distribution of potential outcomes using multiple experimental datasets. We introduce the assumption of transportability…

通过自适应借用外部对照改善试验中的处理效应估计AI 解读

Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as an effective way to augment small RCTs and thereby enhance estimation efficiency. However, ECs are not always comparable to RCTs, and direct borrowing without careful evaluation can introduce substantial bias and, paradoxically, undermine…

二元结果下的个体风险量化AI 解读

Understanding treatment effect heterogeneity is crucial for reliable decision-making in treatment evaluation and selection. The conditional average treatment effect (CATE) is widely used to capture treatment effect heterogeneity induced by observed covariates and to design individualized treatment policies. However, it is an average metric within subpopulations, which prevents it from revealing individual risk, potentially leading to misleading results. This article fills…

可控制伤害率的安全个体化处理规则AI 解读

Estimating individualized treatment rules (ITRs) is crucial for tailoring interventions in precision medicine. Typical ITR estimation methods rely on conditional average treatment effects (CATEs) to guide treatment assignments. However, such methods overlook individual-level harm within covariate-specific subpopulations, potentially leading many individuals to experience worse outcomes under CATE-based ITRs. In this article, we aim to estimate ITRs that maximize the reward…

利用外部对照改进处理效应估计的自适应数据借用AI 解读

Randomized controlled trials (RCTs) often exhibit limited inferential efficiency in estimating treatment effects due to small sample sizes. In recent years, the combination of external controls has gained increasing attention as a means of improving the efficiency of RCTs. However, external controls are not always comparable to RCTs, and direct borrowing without careful evaluation can introduce substantial bias and reduce the efficiency of treatment effect estimation. In…

基于匹配的组平均处理效应非参数估计AI 解读

Heterogeneous treatment effects, which vary according to individual covariates, are crucial in fields such as personalized medicine and tailored treatment strategies. In many applications, rather than considering the heterogeneity induced by all covariates, practitioners focus on a few key covariates to develop tailored treatment decisions. Based on this, we aim to estimate the group average treatment effects (GATEs), which represent heterogeneous treatment effects across…

数据组合下平均处理效应估计的比较分析AI 解读

There is growing interest in exploring causal effects in target populations via data combination. However, most approaches are tailored to specific settings and lack comprehensive comparative analyses. In this article, we focus on a typical scenario involving a source dataset and a target dataset. We first design six settings under covariate shift and conduct a comparative analysis by deriving the semiparametric efficiency bounds for the ATE in the target population. We then…

必要与充分因果概率的半参数有效推断AI 解读

Causal attribution, which aims to explain why events or behaviors occur, is crucial in causal inference and enhances our understanding of cause-and-effect relationships in scientific research. The probabilities of necessary causation (PN) and sufficient causation (PS) are two of the most common quantities for attribution in causal inference. While many works have explored the identification or bounds of PN and PS, efficient estimation remains unaddressed. To fill this gap,…

结合外部观测数据识别与估计临床试验长期处理效应AI 解读

In biomedical studies, estimating drug effects on chronic diseases requires a long follow-up period, which is difficult to meet in randomized clinical trials (RCTs). The use of a short-term surrogate to replace the long-term outcome for assessing the drug effect relies on stringent assumptions that empirical studies often fail to satisfy. Motivated by a kidney disease study, we investigate the drug effects on long-term outcomes by combining an RCT without observation of…

用于异质性治疗效应因果推断的倾向得分回归AI 解读

Understanding how treatment effects vary on individual characteristics is critical in the contexts of personalized medicine, personalized advertising and policy design. When the characteristics are of practical interest are only a subset of full covariate, non-parametric estimation is often desirable; but few methods are available due to the computational difficult. Existing non-parametric methods such as the inverse probability weighting methods have limitations that hinder…

高维数据下协变量特定处理效应的模型辅助推断AI 解读

Covariate-specific treatment effects (CSTEs) represent heterogeneous treatment effects across subpopulations defined by certain selected covariates. In this article, we consider marginal structural models where CSTEs are linearly represented using a set of basis functions of the selected covariates. We develop a new approach in high-dimensional settings to obtain not only doubly robust point estimators of CSTEs, but also model-assisted confidence intervals, which are valid…