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Choosing the Right Approach at the Right Time: A Comparative Analysis of Causal Effect Estimation using Confounder Adjustment and Instrumental Variables

何时选择混杂调整或工具变量:因果效应估计的比较

In observational studies, potential unobserved confounding is a major barrier in isolating the average causal effect (ACE). In these scenarios, two main approaches are often used: confounder adjustment for causality (CAC) and instrumental variable analysis for causation (IVAC). Nevertheless, both are subject to untestable assumptions and, therefore, it may be unclear which assumption violation scenarios one method is superior in terms of mitigating inconsistency for the ACE.…

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An Anatomy of Event Studies: Hypothetical Experiments, Exact Decomposition, and Weighting Diagnostics

事件研究剖析:假想实验、精确分解与权重诊断

In recent decades, event studies have emerged as a central methodology in health and social research for evaluating the causal effects of staggered interventions. In this paper, we analyze event studies from experimental design principles for observational studies, with a focus on information borrowing across measurements. We develop robust weighting estimators that increasingly use more information across units and time periods, justified by increasingly stronger…

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Stochastic interventions, sensitivity analysis, and optimal transport

随机干预、敏感性分析与最优传输

Recent methodological research in causal inference has focused on effects of stochastic interventions, which assign treatment randomly, often according to subject-specific covariates. In this work, we demonstrate that the usual notion of stochastic interventions have a surprising property: when there is unmeasured confounding, bounds on their effects do not collapse when the policy approaches the observational regime. As an alternative, we propose to study generalized…

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Sensitivity analysis methods for outcome missingness using substantive-model-compatible multiple imputation and their application in causal inference

基于实质模型相容多重插补的结果缺失敏感性分析及因果应用

When using multiple imputation (MI) for missing data, maintaining compatibility between the imputation model and substantive analysis is important for avoiding bias. For example, some causal inference methods incorporate an outcome model with exposure-confounder interactions that must be reflected in the imputation model. Two approaches for compatible imputation with multivariable missingness have been proposed: Substantive-Model-Compatible Fully Conditional Specification…

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Causal and Counterfactual Views of Missing Data Models

缺失数据模型的因果与反事实视角

It is often said that the fundamental problem of causal inference is a missing data problem -- the comparison of responses to two hypothetical treatment assignments is made difficult because for every experimental unit only one potential response is observed. In this paper, we consider the implications of the converse view: that missing data problems are a form of causal inference. We make explicit how the missing data problem of recovering the complete data law from the…

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Assumption Smuggling in Intermediate Outcome Tests of Causal Mechanisms

因果机制中间结果检验中的隐含假设

Political scientists are increasingly interested in assessing causal mechanisms, or determining not just if a causal effect exists but also why it occurs. Even so, many researchers avoid formal causal mediation analyses due to their stringent assumptions, instead opting to explore causal mechanisms through what we call intermediate outcome tests. These tests estimate the effect of the treatment on one or more mediators and view such effects as suggestive evidence of a causal…

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On the consistency of bootstrap for matching estimators

匹配估计量的 Bootstrap 一致性

In a landmark paper, Abadie and Imbens (2008) showed that the naive bootstrap is inconsistent when applied to nearest neighbor matching estimators of the average treatment effect with a fixed number of matches. Since then, this finding has inspired numerous efforts to address the inconsistency issue, typically by employing alternative bootstrap methods. In contrast, this paper shows that the naive bootstrap is provably consistent for the original matching estimator, provided…

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Off-policy estimation with adaptively collected data: the power of online learning

自适应采集数据的离策略估计:在线学习的作用

We consider estimation of a linear functional of the treatment effect using adaptively collected data. This task finds a variety of applications including the off-policy evaluation (\textsf{OPE}) in contextual bandits, and estimation of the average treatment effect (\textsf{ATE}) in causal inference. While a certain class of augmented inverse propensity weighting (\textsf{AIPW}) estimators enjoys desirable asymptotic properties including the semi-parametric efficiency, much…

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Randomization-based Z-estimation for evaluating average and individual treatment effects

用于评估平均与个体处理效应的随机化型 Z 估计

Randomized experiments have been the gold standard for drawing causal inference. The conventional model-based approach has been one of the most popular ways for analyzing treatment effects from randomized experiments, which is often carried through inference for certain model parameters. In this paper, we provide a systematic investigation of model-based analyses for treatment effects under the randomization-based inference framework. This framework does not impose any…

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A joint modeling approach to treatment effects estimation with unmeasured confounders

存在未测量混杂时处理效应估计的联合建模方法

Estimating treatment effects using observation data often relies on the assumption of no unmeasured confounders. However, unmeasured confounding variables may exist in many real-world problems. It can lead to a biased estimation without incorporating the unmeasured confounding effect. To address this problem, this paper proposes a new mixed-effects joint modeling approach to identifying and estimating the OR function and the PS function in the presence of unmeasured…

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Doubly Robust Estimation of Causal Excursion Effects in Micro-Randomized Trials with Missing Longitudinal Outcomes

纵向结果缺失的微随机试验中因果偏离效应的双重稳健估计

Micro-randomized trials (MRTs) are increasingly utilized for optimizing mobile health interventions, with the causal excursion effect (CEE) as a central quantity for evaluating interventions under policies that deviate from the experimental policy. However, MRT often contains missing data due to reasons such as missed self-reports or participants not wearing sensors, which can bias CEE estimation. In this paper, we propose a two-stage, doubly robust estimator for CEE in MRTs…

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Causal Inference under Network Interference Using a Mixture of Randomized Experiments

利用混合随机实验进行网络干扰下的因果推断

In randomized experiments, the classic Stable Unit Treatment Value Assumption (SUTVA) posits that the outcome for one experimental unit is unaffected by the treatment assignments of other units. However, this assumption is frequently violated in settings such as online marketplaces and social networks, where interference between units is common. We address the estimation of the total treatment effect in a network interference model by employing a mixed randomization design…

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G-computation for increasing performances of clinical trials with individual randomization and binary response

G计算提升个体随机化二分类响应临床试验的表现

In a clinical trial, the random allocation aims to balance prognostic factors between arms, preventing true confounders. However, residual differences due to chance may introduce near-confounders. Adjusting on prognostic factors is therefore recommended, especially because the related increase of the power. In this paper, we hypothesized that G-computation associated with machine learning could be a suitable method for randomized clinical trials even with small sample sizes.…

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Causal Discovery and Classification Using Lempel-Ziv Complexity

利用 Lempel–Ziv 复杂度开展因果发现与分类

Inferring causal relationships in the decision-making processes of machine learning algorithms is a crucial step toward achieving explainable Artificial Intelligence (AI). In this research, we introduce a novel causality measure and a distance metric derived from Lempel-Ziv (LZ) complexity. We explore how the proposed causality measure can be used in decision trees by enabling splits based on features that most strongly \textit{cause} the outcome. We further evaluate the…

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The inclusive Synthetic Control Method

包容式合成控制法

We introduce the inclusive synthetic control method (iSCM), a modification of synthetic control methods that includes units in the donor pool potentially affected, directly or indirectly, by an intervention. This method is ideal for situations where including treated units in the donor pool is essential or where donor units may experience spillover effects. The iSCM is straightforward to implement with most synthetic control estimators. As an empirical illustration, we…

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Debiased machine learning for counterfactual survival functionals based on left-truncated right-censored data

基于左截断右删失数据的反事实生存泛函去偏机器学习

Learning causal effects of a binary exposure on time-to-event endpoints can be challenging because survival times may be partially observed due to censoring and systematically biased due to truncation. In this work, we present debiased machine learning-based nonparametric estimators of the joint distribution of a counterfactual survival time and baseline covariates for use when the observed data are subject to covariate-dependent left truncation and right censoring and when…

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Using Principal Progression Rate to Quantify and Compare Disease Progression in Comparative Studies

利用主要进展速率量化和比较研究中的疾病进展

In comparative studies of progressive diseases, such as randomized controlled trials (RCTs), the mean Change From Baseline (CFB) of a continuous outcome at a pre-specified follow-up time across subjects in the target population is a standard estimand used to summarize the overall disease progression. Despite its simplicity in interpretation, the mean CFB may not efficiently capture important features of the trajectory of the mean outcome relevant to the evaluation of the…

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Interaction Testing in Variation Analysis

变异分析中的交互作用检验

Relationships of cause and effect are of prime importance for explaining scientific phenomena. Often, rather than just understanding the effects of causes, researchers also wish to understand how a cause $X$ affects an outcome $Y$ mechanistically -- i.e., what are the causal pathways that are activated between $X$ and $Y$. For analyzing such questions, a range of methods has been developed over decades under the rubric of causal mediation analysis. Traditional mediation…

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Confidence intervals for adaptive trial designs II: Case study and practical guidance

自适应试验设计的置信区间之二:案例研究与实践指南

In adaptive clinical trials, the conventional confidence interval (CI) for a treatment effect is prone to undesirable properties such as undercoverage and potential inconsistency with the final hypothesis testing decision. Accordingly, as is stated in recent regulatory guidance on adaptive designs, there is the need for caution in the interpretation of CIs constructed during and after an adaptive clinical trial. However, it may be unclear which of the available CIs in the…

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Confidence intervals for adaptive trial designs I: A methodological review

自适应试验设计的置信区间之一:方法综述

Regulatory guidance notes the need for caution in the interpretation of confidence intervals (CIs) constructed during and after an adaptive clinical trial. Conventional CIs of the treatment effects are prone to undercoverage (as well as other undesirable properties) in many adaptive designs, because they do not take into account the potential and realised trial adaptations. This paper is the first in a two-part series that explores CIs for adaptive trials. It provides a…

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Improving randomized controlled trial analysis via data-adaptive borrowing

通过数据自适应借用外部对照改进随机试验分析

In recent years, real-world external controls have grown in popularity as a tool to empower randomized placebo-controlled trials, particularly in rare diseases or cases where balanced randomization is unethical or impractical. However, as external controls are not always comparable to the trials, direct borrowing without scrutiny may heavily bias the treatment effect estimator. Our paper proposes a data-adaptive integrative framework capable of preventing unknown biases of…

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Probably approximately correct high-dimensional causal effect estimation given a valid adjustment set

已知有效调整集下高维因果效应估计的 PAC 保证

Accurate estimates of causal effects play a key role in decision-making across applications such as healthcare, economics, and operations. In the absence of randomized experiments, a common approach to estimating causal effects uses \textit{covariate adjustment}. In this paper, we study covariate adjustment for discrete distributions from the PAC learning perspective, assuming knowledge of a valid adjustment set $\bZ$, which might be high-dimensional. Our first main result…

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Flexible Functional Treatment Effect Estimation

灵活的函数型处理效应估计

We study treatment effect estimation with functional treatments where the average potential outcome functional is a function of functions, in contrast to continuous treatment effect estimation where the target is a function of real numbers. By considering a flexible scalar-on-function marginal structural model, a weight-modified kernel ridge regression (WMKRR) is adopted for estimation. The weights are constructed by directly minimizing the uniform balancing error resulting…

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Causal Inference in Randomized Trials with Partial Clustering

部分聚类随机试验中的因果推断

Clustering and dependence are common in trials. For example, in some cluster randomized trials (CRTs), pre-existing clusters are enrolled, randomized, and serve as the basis of intervention delivery. Such CRTs are "fully clustered": participants are dependent within clusters. In contrast, "partially clustered" trials contain a mix of participants that are dependent within clusters and participants that are completely independent. One example of this design is a trial where…

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