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Testing Prioritized Composite Endpoint with Multiple Follow-up Time Examinations

结合多个随访时点检验优先级复合终点

Composite endpoints are widely used in cardiovascular clinical trials. In recent years, hierarchical composite endpoints-particularly the win ratio approach and its predecessor, the Finkelstein-Schoenfeld (FS) test, also known as the unmatched win ratio test-have gained popularity. These methods involve comparing individuals across multiple endpoints, ranked by priority, with mortality typically assigned the highest priority in many applications. However, these methods have…

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Subgroup comparisons within and across studies in meta-analysis

元分析中研究内与跨研究的亚组比较

Subgroup-specific meta-analysis synthesizes treatment effects for patient subgroups across randomized trials. Methods include joint or separate modeling of subgroup effects and treatment-by-subgroup interactions, but inconsistencies arise when subgroup prevalence differs between studies (e.g., proportion of non-smokers). A key distinction is between study-generated evidence within trials and synthesis-generated evidence obtained by contrasting results across trials. This…

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Non-Existent Outcomes in Research on Inequality: A Causal Approach

不平等研究中不存在的结果:一种因果方法

Scholars of social stratification often study exposures that shape life outcomes. But some outcomes (such as wage) only exist for some people (such as those who are employed). We show how a common practice -- dropping cases with non-existent outcomes -- can obscure causal effects when a treatment affects both outcome existence and outcome values. The effects of both beneficial and harmful treatments can be underestimated. Drawing on existing approaches for principal…

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Nonparametric Bounds for Evaluating the Clinical Utility of Treatment Rules

评估临床治疗规则效用的非参数界限

Evaluating the value of new clinical treatment rules based on patient characteristics is important but often complicated by hidden confounding factors in observational studies. Standard methods for estimating the average patient outcome if a new rule were universally adopted typically rely on strong, untestable assumptions about these hidden factors. This paper tackles this challenge by developing nonparametric bounds - a range of plausible values - for the expected outcome…

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Gender disparities in rehospitalisations after coronary artery bypass grafting: evidence from a sparse functional causal mediation analysis of the MIMIC-IV data

冠状动脉旁路移植术后再住院的性别差异:MIMIC-IV 数据的稀疏函数因果中介分析

Hospital readmissions following coronary artery bypass grafting (CABG) not only impose a substantial cost burden on healthcare systems but also serve as a potential indicator of the quality of medical care. Previous studies of gender effects on complications after CABG surgery have consistently revealed that women tend to suffer worse outcomes. To better understand the causal pathway from gender to the number of rehospitalisations, we study the postoperative central venous…

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Partial Identification of Causal Effects for Endogenous Continuous Treatments

内生连续处理因果效应的部分识别

No unmeasured confounding is a common assumption when reasoning about counterfactual outcomes, but such an assumption may not be plausible in observational studies. Sensitivity analysis is often employed to assess the robustness of causal conclusions to unmeasured confounding, but existing methods are predominantly designed for binary treatments. In this paper, we provide natural extensions of two extensively used sensitivity frameworks -- the Rosenbaum and Marginal…

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Bounding Causal Effects and Counterfactuals

因果效应与反事实的界限估计

Causal inference often hinges on strong assumptions - such as no unmeasured confounding or perfect compliance - that are rarely satisfied in practice. Partial identification offers a principled alternative: instead of relying on unverifiable assumptions to estimate causal effects precisely, it derives bounds that reflect the uncertainty inherent in the data. Despite its theoretical appeal, partial identification remains underutilized in applied work, in part due to the…

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On a Debiased and Semiparametric Efficient Changes-in-Changes Estimator

去偏且具有半参数效率的变化中变化估计量

We present a novel extension of the influential changes-in-changes (CiC) framework of Athey and Imbens (2006) for estimating the average treatment effect on the treated (ATT) and distributional causal effects in panel data with unmeasured confounding. While CiC relaxes the parallel trends assumption in difference-in-differences (DiD), existing methods typically assume a scalar unobserved confounder and monotonic outcome relationships, and lack inference tools that…

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Reasonable uncertainty: Confidence intervals in empirical Bayes discrimination detection

合理量化不确定性:经验贝叶斯歧视检测中的置信区间

We revisit empirical Bayes discrimination detection, focusing on uncertainty arising from both partial identification and sampling variability. While prior work has mostly focused on partial identification, we find that some empirical findings are not robust to sampling uncertainty. To better connect statistical evidence to the magnitude of real-world discriminatory behavior, we propose a counterfactual odds-ratio estimand with a attractive properties and interpretation. Our…

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Bayesian Double Machine Learning for Causal Inference

用于因果推断的贝叶斯双重机器学习

This paper proposes a simple, novel, and fully-Bayesian approach for causal inference in partially linear models with high-dimensional control variables. Off-the-shelf machine learning methods can introduce biases in the causal parameter known as regularization-induced confounding. To address this, we propose a Bayesian Double Machine Learning (BDML) method, which modifies a standard Bayesian multivariate regression model and recovers the causal effect of interest from the…

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A More Robust Approach to Multivariable Mendelian Randomization

多变量孟德尔随机化的一种更稳健方法

Multivariable Mendelian randomization (MVMR) uses genetic variants as instrumental variables to infer the direct effects of multiple exposures on an outcome. However, unlike univariable Mendelian randomization, MVMR often faces greater challenges with many weak instruments, which can lead to bias not necessarily toward zero and inflation of type I errors. In this work, we introduce a new asymptotic regime that allows exposures to have varying degrees of instrument strength,…

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A statistician's guide to weak-instrument-robust inference in instrumental variables regression with illustrations in Python

工具变量回归中弱工具稳健推断的统计学指南及 Python 示例

We provide an overview of results relating to estimation and weak-instrument-robust inference in instrumental variables regression. Methods are implemented in the ivmodels software package for Python, which we use to illustrate results.

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Efficient Statistical Estimation for Sequential Adaptive Experiments with Implications for Adaptive Designs

序贯自适应实验的有效统计估计及其对自适应设计的启示

Adaptive experimental designs have gained popularity in clinical trials and online experiments. Unlike traditional, fixed experimental designs, adaptive designs can dynamically adjust treatment randomization probabilities and other design features in response to data accumulated sequentially during the experiment. These adaptations are useful to achieve diverse objectives, including reducing uncertainty in the estimation of causal estimands or increasing participants'…

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PanelMatch: Matching Methods for Causal Inference with Time-Series Cross-Section Data

PanelMatch:时间序列截面数据因果推断的匹配方法

Analyzing time-series cross-sectional (also known as longitudinal or panel) data is an important process across a number of fields, including the social sciences, economics, finance, and medicine. PanelMatch is an R package that implements a set of tools enabling researchers to apply matching methods for causal inference with time-series cross-sectional data. Relative to other commonly used methods for longitudinal analyses, like regression with fixed effects, the…

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Differentially Private Covariate Balancing Causal Inference

差分隐私下的协变量平衡因果推断

Differential privacy is the leading mathematical framework for privacy protection, providing a probabilistic guarantee that safeguards individuals' private information when publishing statistics from a dataset. This guarantee is achieved by applying a randomized algorithm to the original data, which introduces unique challenges in data analysis by distorting inherent patterns. In particular, causal inference using observational data in privacy-sensitive contexts is…

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Multiply robust estimation for causal survival analysis with treatment noncompliance

治疗不依从下因果生存分析的多重稳健估计

Comparative effectiveness research frequently addresses a time-to-event outcome and can require unique considerations in the presence of treatment noncompliance. Motivated by the challenges in addressing noncompliance in the ADAPTABLE pragmatic clinical trial, we develop a multiply robust estimator to estimate the principal survival causal effects under the principal ignorability and monotonicity. The multiply robust estimator is consistent even if one, and sometimes two, of…

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Adaptive sparsening and smoothing of the treatment model for longitudinal causal inference using outcome-adaptive LASSO and marginal fused LASSO

纵向因果推断中治疗模型的自适应稀疏化与平滑

Causal variable selection in time-varying treatment settings is challenging due to evolving confounding effects. Existing methods mainly focus on time-fixed exposures and are not directly applicable to time-varying scenarios. We propose a novel two-step procedure for variable selection when modeling the treatment probability at each time point. We first introduce a novel approach to longitudinal confounder selection using a Longitudinal Outcome Adaptive LASSO (LOAL) that…

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How Much Does Home Field Advantage Matter in Soccer Games? A Causal Inference Approach for English Premier League Analysis

足球主场优势有多重要:英超联赛的因果推断分析

In many sports, it is commonly believed that the home team has an advantage over the visiting team, known as the home field advantage. Yet its causal effect on team performance is largely unknown. In this paper, we propose a novel causal inference approach to study the causal effect of home field advantage in English Premier League. We develop a hierarchical causal model and show that both league level and team level causal effects are identifiable and can be conveniently…

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Functional Analysis of Variance for Association Studies

关联研究中的函数型方差分析

While progress has been made in identifying common genetic variants associated with human diseases, for most of common complex diseases, the identified genetic variants only account for a small proportion of heritability. Challenges remain in finding additional unknown genetic variants predisposing to complex diseases. With the advance in next-generation sequencing technologies, sequencing studies have become commonplace in genetic research. The ongoing exome-sequencing and…

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A Two-Step Test to Identify Zero-Inflated Biomarkers in Early-Phase Clinical Trials

早期临床试验中零膨胀生物标志物的两步检验

In early-phase clinical trials, a predictive biomarker may identify subgroups that benefit from an experimental therapy, even when the overall average treatment effect is negligible. Recently proposed nonparametric interaction tests such as the Average Kolmogorov-Smirnov Approach (AKSA) avoid prespecified biomarker cutting points and model assumptions, but their power degrades when the biomarker distribution is zero-inflated. We propose a two-step test that partitions the…

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Partial identification via conditional linear programs: estimation and policy learning

基于条件线性规划的部分识别:估计与政策学习

Many important quantities of interest are only partially identified from observable data: the data can limit them to a set of plausible values, but not uniquely determine them. This paper develops a unified framework for covariate-assisted estimation, inference, and decision making in partial identification problems where the parameter of interest satisfies a series of linear constraints, conditional on covariates. In such settings, bounds on the parameter can be written as…

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Causal Identification for Complex Functional Longitudinal Studies

复杂函数型纵向研究的因果识别

Real-time monitoring in modern medical research introduces functional longitudinal data, characterized by continuous-time measurements of outcomes, treatments, and confounders. This complexity leads to uncountably infinite treatment-confounder feedbacks, which traditional causal inference methodologies cannot handle. Inspired by the coarsened data framework, we adopt stochastic process theory, measure theory, and net convergence to propose a nonparametric causal…

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Incremental Causal Effect for Time to Treatment Initialization

治疗启动时间的增量因果效应

We consider time to treatment initialization. This can commonly occur in preventive medicine, such as disease screening and vaccination; it can also occur with non-fatal health conditions such as HIV infection without the onset of AIDS; or in tech industry where items wait to be reviewed manually as abusive or not, etc. While traditional causal inference focused on `when to treat' and its effects, including their possible dependence on subject characteristics, we consider…

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Inference under Staggered Adoption: Case Study of the Affordable Care Act

交错实施下的推断:以《平价医疗法案》为例

Panel data consists of a collection of $N$ units that are observed over $T$ units of time. A policy or treatment is subject to staggered adoption if different units take on treatment at different times and remains treated (or never at all). Assessing the effectiveness of such a policy requires estimating the treatment effect, corresponding to the difference between outcomes for treated versus untreated units. We develop inference procedures that build upon a computationally…

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