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The ideal trial: defining causal estimands that balance relevance and feasibility in target trial emulations and actual randomized trials

理想试验:在目标试验模拟与随机试验中平衡因果目标的相关性和可行性

Causal inference is the goal of randomized trials and many observational studies. The first step in a formal causal inference framework is to define the causal estimand, and in both types of study this can be intuitively defined as the effect in an ideal trial: a hypothetical perfect randomized experiment (with representative sample, perfect adherence, etc.). The target trial framework is increasingly used for causal inference in observational studies, but clarity is lacking…

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Predicting Market Troughs: A Machine Learning Approach with Causal Interpretation

市场低谷预测:具有因果解释的机器学习方法

This paper provides robust, new evidence on the causal drivers of market troughs. We demonstrate that conclusions about these triggers are critically sensitive to model specification, moving beyond restrictive linear models with a flexible DML average partial effect causal machine learning framework. Our robust estimates identify the volatility of options-implied risk appetite and market liquidity as key causal drivers, relationships misrepresented or obscured by simpler…

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Design-Based and Network Sampling-Based Uncertainties in Network Experiments

网络实验中的设计不确定性与网络抽样不确定性

Ordinary least squares (OLS) estimators are widely used in network experiments to estimate spillover effects. We study the causal interpretation of, and inference for the OLS estimator under both design-based uncertainty from random treatment assignment and sampling-based uncertainty in network links. We show that correlations among regressors that capture the exposure to neighbors' treatments can induce contamination bias, preventing OLS from aggregating heterogeneous…

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Heterogeneous Treatment Effects via Linear Dynamic Panel Data Models

线性动态面板模型中的异质性处理效应

We study the identification of heterogeneous, intertemporal treatment effects (TE) when potential outcomes depend on past treatments. First, applying a dynamic panel data model to observed outcomes, we show that an instrumental variable (IV) version of the estimand in Arellano and Bond (1991) recovers a non-convex (negatively weighted) aggregate of TE plus non-vanishing trends. We then provide conditions on sequential exchangeability (SE) of treatment and on TE heterogeneity…

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Bayesian Inference for Confounding Variables and Limited Information

混杂变量与有限信息下的贝叶斯推断

A central challenge in statistical inference is the presence of confounding variables that may distort observed associations between treatment and outcome. Conventional "causal" methods, grounded in assumptions such as ignorability, exclude the possibility of unobserved confounders, leading to posterior inferences that overstate certainty. We develop a Bayesian framework that relaxes these assumptions by introducing entropy-favoring priors over hypothesis spaces that…

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Nonparametric estimation of the Patient Weighted While-Alive Estimand

患者加权存活期间估计目标的非参数估计

In clinical trials with recurrent events, such as repeated hospitalizations terminating with death, it is important to consider the patient events overall history for a thorough assessment of treatment effects. The occurrence of fewer events due to early deaths can lead to misinterpretation, emphasizing the importance of a while-alive strategy as suggested in Schmidli et al. (2023). In this study, we focus on the patient weighted while-alive estimand, represented as the…

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Treatment Effects of Multi-Valued Treatments in Hyper-Rectangle Model

超矩形模型中多值处理的处理效应

This study investigates the identification of marginal treatment responses within multi-valued treatment models. Extending the hyper-rectangle model introduced by Lee and Salanie (2018), this paper relaxes restrictive assumptions, including the requirement of known treatment selection thresholds and the dependence of treatments on all unobserved heterogeneity. By incorporating an additional ranked treatment assumption, this study demonstrates that the marginal treatment…

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Semi-supervised inference for treatment heterogeneity

处理效应异质性的半监督推断

In causal inference, measuring treatment heterogeneity is crucial as it provides scientific insights into how treatments influence outcomes and guides personalized decision-making. In this work, we study semi-supervised settings where a labeled dataset is accompanied by a large unlabeled dataset, and develop semi-supervised estimators for two measures of treatment heterogeneity: the total treatment heterogeneity (TTH) and the explained treatment heterogeneity (ETH) of a…

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Precision Mental Health: Predicting Heterogeneous Treatment Effects for Depression through Data Integration

精准精神健康:通过数据整合预测抑郁症的异质性处理效应

When treating depression, clinicians are interested in determining the optimal treatment for a given patient, which is challenging given the amount of treatments available. To advance individualized treatment allocation, integrating data across multiple randomized controlled trials (RCTs) can enhance our understanding of treatment effect heterogeneity by increasing available information. However, extending these inferences to individuals outside of the original RCTs remains…

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Sharp Bounds on the Variance of General Regression Adjustment in Randomized Experiments

随机实验中一般回归调整的方差锐界

A growing statistical literature focuses on causal inference in the context of experiments where the target of inference is the average treatment effect in a finite population and random assignment determines which subjects are allocated to one of the experimental conditions. In this framework, variances of average treatment effect estimators remain unidentified because they depend on the covariance between treated and untreated potential outcomes, which are never jointly…

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Bayesian Network Propensity Score to Evaluate Treatment Effects in Observational Studies

用于观察性研究处理效应评估的贝叶斯网络倾向得分

This paper focuses on the Bayesian Network Propensity Score (BNPS), a novel approach for estimating treatment effects in observational studies characterized by unknown (and likely unbalanced) designs and complex dependency structures among covariates. Traditional methods, such as logistic regression, often impose rigid parametric assumptions that may lead to misspecification errors, compromising causal inference. Recent classical and machine learning alternatives, such as…

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Distal Causal Excursion Effects: Modeling Long-Term Effects of Time-Varying Treatments in Micro-Randomized Trials

远期因果偏离效应:微随机试验中时变处理的长期效应建模

Micro-randomized trials (MRTs) play a crucial role in optimizing digital interventions. In an MRT, each participant is sequentially randomized among treatment options hundreds of times. While the interventions tested in MRTs target short-term behavioral responses (proximal outcomes), their ultimate goal is to drive long-term behavior change (distal outcomes). However, existing causal inference methods, such as the causal excursion effect, are limited to proximal outcomes,…

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Confounder selection via iterative graph expansion

通过迭代扩展因果图选择混杂变量

Confounder selection, namely choosing a set of covariates to control for confounding between a treatment and an outcome, is arguably the most important step in the design of an observational study. Previous methods, such as Pearl's back-door criterion, typically require pre-specifying a causal graph, which can often be difficult in practice. We propose an interactive procedure for confounder selection that does not require pre-specifying the graph or the set of observed…

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Calibrated sensitivity models

校准敏感性模型

In causal inference, sensitivity models assess how unmeasured confounders could alter causal analyses, but the sensitivity parameter -- which quantifies the degree of unmeasured confounding -- is often difficult to interpret. For this reason, researchers sometimes compare the sensitivity parameter to an estimate of measured confounding. This is known as calibration, or benchmarking. However, calibrated estimates are not always interpreted correctly, and uncertainty in the…

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Nonparametric Bounds in Causal Mediation Analysis in the Presence of Unmeasured Confounding and Imperfect Compliance

未测混杂与不完全依从下因果中介分析的非参数界

The average causal mediation effect (ACME) and the natural direct effect (NDE) are two parameters of primary interest in causal mediation analysis. However, the two causal parameters are not identifiable from randomized experimental data in the presence of outcome-mediator confounding and treatment-assignment noncompliance. Sjölander (2009) addressed the partial identification issue and derived nonparametric bounds of the NDE in randomized controlled trials under a set of…

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Statistical Inference for Heterogeneous Treatment Effect with Right-censored Data from Synthesizing Randomized Clinical Trials and Real-world Data

融合随机试验与真实世界右删失数据的异质处理效应推断

The heterogeneous treatment effect plays a crucial role in precision medicine.There is evidence that real-world data, even subject to biases, can be employed as supplementary evidence for randomized clinical trials to improve the statistical efficiency of the heterogeneous treatment effect estimation. In this paper, for survival data with right censoring, we consider estimating the heterogeneous treatment effect, defined as the difference of the treatment-specific…

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Causal Spatial Quantile Regression

因果空间分位数回归

Treatment effects in a wide range of economic, environmental, and epidemiological applications often vary across space, and understanding the heterogeneity of causal effects across space and outcome quantiles is a critical challenge in spatial causal inference. To effectively capture spatial heterogeneity in distributional treatment effects, we propose a novel semiparametric neural network-based causal framework leveraging deep spatial quantile regression and then construct…

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Interpretational errors with instrumental variables

工具变量研究中的解释错误

Instrumental variables (IV) are often used to identify causal effects in observational settings and experiments subject to non-compliance. Under canonical assumptions, IVs allow us to identify a so-called local average treatment effect (LATE). The use of IVs is often accompanied by a pragmatic decision to abandon the identification of the causal parameter that corresponds to the original research question and target the LATE instead. This pragmatic decision presents a…

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Mediation Analysis in the Presence of Sample Selection Bias with an Application to Disparities in Liver Transplantation Listing

样本选择偏差下的中介分析:肝移植候选名单差异的应用

The study of disparities in the liver transplantation process may focus on quantifying causal effects, particularly the average, direct, or indirect effects of various social determinants of health on being listed as a candidate for transplant. Selection bias arises when the data sample does not represent the target population, defined here as all individuals referred to the transplant clinic. Listing decisions are made for the subset of patients who complete the evaluation…

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On the role of the design phase in a linear regression

线性回归中研究设计阶段的作用

The "design phase" refers to a stage in observational studies, during which a researcher constructs a subsample that achieves a better balance in covariate distributions between the treated and untreated units. In this paper, we study the role of this preliminary phase in the context of linear regression, offering a justification for its utility. To that end, we first formalize the design phase as a process of estimand adjustment via selecting a subsample. Then, we show that…

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Distance and Kernel-Based Measures for Global and Local Two-Sample Conditional Distribution Testing

基于距离与核的全局及局部两样本条件分布检验

Testing the equality of two conditional distributions is crucial in various modern applications, including transfer learning and causal inference. Despite its importance, this fundamental problem has received surprisingly little attention in the literature, with existing works focusing exclusively on global two-sample conditional distribution testing. Based on distance and kernel methods, this paper presents the first unified framework for both global and local two-sample…

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Causal SHAP: Feature Attribution with Dependency Awareness through Causal Discovery

因果 SHAP:通过因果发现实现依赖关系感知的特征归因

Explaining machine learning (ML) predictions has become crucial as ML models are increasingly deployed in high-stakes domains such as healthcare. While SHapley Additive exPlanations (SHAP) is widely used for model interpretability, it fails to differentiate between causality and correlation, often misattributing feature importance when features are highly correlated. We propose Causal SHAP, a novel framework that integrates causal relationships into feature attribution while…

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ProCause: Generating Counterfactual Outcomes to Evaluate Prescriptive Process Monitoring Methods

ProCause:生成反事实结果以评估处方式流程监控方法

Prescriptive Process Monitoring (PresPM) is the subfield of Process Mining that focuses on optimizing processes through real-time interventions based on event log data. Evaluating PresPM methods is challenging due to the lack of ground-truth outcomes for all intervention actions in datasets. A generative deep learning approach from the field of Causal Inference (CI), RealCause, has been commonly used to estimate the outcomes for proposed intervention actions to evaluate a…

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Causal inference and racial bias in policing: New estimands and the importance of mobility data

警务种族偏见的因果推断:新目标参数与流动数据的重要性

Studying racial bias in policing is a critically important problem, but one that comes with a number of inherent difficulties due to the nature of the available data. In this manuscript we tackle multiple key issues in the causal analysis of racial bias in policing. First, we formalize race and place policing, the idea that individuals of one race are policed differently when they are in neighborhoods primarily made up of individuals of other races. We develop an estimand to…

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