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Improving Precision of RCT-Based CATE Estimation using Data Borrowing with Double Calibration

以双重校准借用观察数据提升随机试验 CATE 估计精度

Understanding how treatment effects vary across patient characteristics is essential for personalized medicine, yet randomized controlled trials (RCTs) are often underpowered to detect heterogeneous treatment effects (HTEs). We propose a framework that improves the efficiency of conditional average treatment effect (CATE) estimation in RCTs by leveraging large observational studies (OS) while preserving RCT unbiasedness. Framing CATE estimation as a supervised learning…

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Causal Inference for Functional Treatments with Stochastic Policies

随机策略下函数型处理的因果推断

Wearable devices can accurately measure human behavior, providing a unique opportunity to understand how behavior impacts health. Recent studies leveraging functional regression methods have found a strong relationship between accelerometer-collected physical activity and mortality. However, to determine if physical activity patterns impact mortality it is necessary to understand the causal effects of policies for physical activity, i.e., a function-valued treatment.…

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Sharp Bounds for Treatment Effect Generalization under Outcome Distribution Shift

结果分布偏移下处理效应推广的锐界

Generalizing treatment effects from a randomized trial to a target population requires the assumption that potential outcome distributions are invariant across populations after conditioning on observed covariates. This assumption fails when unmeasured effect modifiers are distributed differently between trial participants and the target population. We develop a sensitivity analysis framework that bounds how much conclusions can change when this transportability assumption…

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Omitted-Variable Sensitivity Analysis for Generalizing Randomized Trials

随机试验结果推广的遗漏变量敏感性分析

Randomized controlled trials (RCTs) yield internally valid causal effect estimates, but generalizing these results to target populations with different characteristics requires an untestable selection ignorability assumption: conditional on observed covariates, trial participation must be independent of potential outcomes. This assumption fails when unobserved effect modifiers are distributed differently between trial and target populations. We develop a sensitivity analysis…

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Improving RCT-Based CATE Estimation Under Covariate Mismatch via Double Calibration

协变量不匹配下通过双重校准改善随机试验的条件平均治疗效应估计

We develop estimators that improve precision of heterogeneous treatment effect estimates that allow borrowing information from observational studies when the available covariates in each data source do not perfectly match. Standard data-borrowing methods often assume perfectly matched covariates. We propose MR-OSCAR, an RCT-calibrated, two-stage estimation approach that first predicts the trial-missing variables using the observational data via imputation and then calibrates…

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Estimation of heterogeneous principal effects under principal ignorability

主层可忽略性下异质主层效应的估计

We study estimation and inference for heterogeneous principal causal effects with binary treatments and binary intermediate variables. Principal causal effects are subgroup effects within strata defined by potential values of an intermediate variable, including effects among compliers. We propose a framework for estimating and forming pointwise confidence intervals for heterogeneous principal causal effects under the principal ignorability assumption. Several estimators are…

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Estimating causal effects of functional treatments with modified functional treatment policies

通过修正函数型处理策略估计函数型处理的因果效应

Functional data are increasingly prevalent in biomedical research. While functional data analysis has been established for decades, causal inference with functional treatments remains largely unexplored. Existing methods typically focus on estimating the causal average dose response functional (ADRF), which requires strong positivity assumptions and offers limited interpretability. In this work, we target a new causal estimand, the modified functional treatment policy…

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Data adaptive covariate balancing for causal effect estimation for high dimensional data

高维数据因果效应估计中的数据自适应协变量平衡

A key challenge in estimating causal effects from observational data is handling confounding and is commonly achieved through weighting methods that balance distribution of covariates between treatment and control groups. Weighting approaches can be classified by whether weights are estimated using parametric or nonparametric methods, and by whether the model relies on modeling and inverting the propensity score or directly estimates weights to achieve distributional balance…

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Partially Retargeted Balancing Weights for Causal Effect Estimation Under Positivity Violations

正值性违背下用于因果效应估计的部分重定向平衡权重

Positivity violations, which occur when some subgroups either always or never receive a treatment of interest, pose significant challenges for causal effect estimation with observational data. Recent balancing weight methods have proved to be highly effective in confounding control, however their utility is diminished in the presence of positivity violations, resulting in bias and excess variance. Approaches that deal with positivity violations, on the other hand, work by…

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Exploring the effects of mechanical ventilator settings with modified vector-valued treatment policies

利用修正向量值处理政策分析机械通气参数的效应

Mechanical ventilation is critical for managing respiratory failure, but inappropriate ventilator settings can lead to ventilator-induced lung injury (VILI), increasing patient morbidity and mortality. Evaluating the causal impact of ventilator settings is challenging due to the complex interplay of multiple treatment variables and strong confounding due to ventilator guidelines. In this paper, we propose a modified vector-valued treatment policy (MVTP) framework coupled…

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A Unified Framework for Causal Estimand Selection

因果目标量选择的统一框架

Estimating the causal effect of a treatment or health policy with observational data can be challenging due to an imbalance of and a lack of overlap between treated and control covariate distributions. In the presence of limited overlap, researchers choose between 1) methods (e.g., inverse probability weighting) that imply traditional estimands but whose estimators are at risk of considerable bias and variance; and 2) methods (e.g., overlap weighting) which imply a different…

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Modified treatment policy effect estimation with weighted energy distance

利用加权能量距离估计修正处理政策效应

The causal effects of continuous treatments are often characterized through the average dose response function, which is challenging to estimate from observational data due to confounding and positivity violations. Modified treatment policies (MTPs) are an alternative approach that aim to assess the effect of a modification to observed treatment values and work under relaxed assumptions. Estimators for MTPs generally focus on estimating the conditional density of treatment…

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Transportability of Principal Causal Effects

主层因果效应的可迁移性

Recent research in causal inference has made important progress in addressing challenges to the external validity of trial findings. Such methods weight trial participant data to more closely resemble the distribution of effect-modifying covariates in a well-defined target population. In the presence of participant non-adherence to study medication, these methods effectively transport an intention-to-treat effect that averages over heterogeneous compliance behaviors. In this…

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Independence weights for causal inference with continuous treatments

连续处理因果推断的独立性权重

Studying causal effects of continuous treatments is important for gaining a deeper understanding of many interventions, policies, or medications, yet researchers are often left with observational studies for doing so. In the observational setting, confounding is a barrier to the estimation of causal effects. Weighting approaches seek to control for confounding by reweighting samples so that confounders are comparable across different treatment values. Yet, for continuous…

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Causally-interpretable meta-analysis: clearly-defined causal effects and two case studies

具有因果解释的元分析:明确的因果效应与两个案例

Meta-analysis is commonly used to combine results from multiple clinical trials, but traditional meta-analysis methods do not refer explicitly to a population of individuals to whom the results apply and it is not clear how to use their results to assess a treatment's effect for a population of interest. We describe recently-introduced causally-interpretable meta-analysis methods and apply their treatment effect estimators to two individual-participant data sets. These…

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Causally-Interpretable Random-Effects Meta-Analysis

具有因果解释的随机效应元分析

Recent work has made important contributions in the development of causally-interpretable meta-analysis. These methods transport treatment effects estimated in a collection of randomized trials to a target population of interest. Ideally, estimates targeted toward a specific population are more interpretable and relevant to policy-makers and clinicians. However, between-study heterogeneity not arising from differences in the distribution of treatment effect modifiers can…

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