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The Limits of Experimental Design: Covariate Balance Beyond Low Dimension

实验设计的极限:超越低维的协变量平衡

We study how fast experimental designs can approach the semiparametric efficiency bound in finite samples, as measured by the excess variance of unadjusted treatment effect estimation. We prove an impossibility theorem: under weak conditions, no design can approach the variance bound uniformly over smooth outcome models unless covariate dimension $d \ll \log n$. Even in experiments with thousands of units, this permits only a handful of covariates. Motivated by this, we…

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Transporting Trial Evidence Under Posterior Drift and Possible Hidden Confounding

后验漂移与潜在隐藏混杂下的试验证据迁移

Randomized trials provide internally valid treatment-effect evidence, but trial participants may not represent the target population. In contrast, observational studies are often closer to the target population, but their treatment assignment may be affected by possible hidden confounding. We develop a robust posterior-drift framework for estimating the average treatment effect in an observational target population when exact conditional-effect transportability may fail. The…

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Exact Inference in Fixed-Effect Regressions with Concentrated Identifying Variation

识别变异集中时固定效应回归的精确推断

In fixed-effect regressions with many groups, fixed effects can absorb most identifying variation, leaving a handful of observations to carry what remains. When variation is this concentrated, conventional $t$-tests can reject a true null more than half the time, and any fixed critical value is either invalid or so conservative it has essentially no power. This paper builds an exact test from the design alone. A \textit{nuisance-annihilating contrast} is a linear combination…

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Forest Kernel Balancing Weights: Outcome-Guided Features for Causal Inference

森林核平衡权重:由结局引导的因果推断特征

While balancing covariates between groups is central for observational causal inference, selecting which features to balance remains a challenging problem. Kernel balancing is a promising approach that first estimates a kernel that captures similarity across units and then balances a (possibly low-dimensional) summary of that kernel, indirectly learning important features to balance. In this paper, we propose forest kernel balancing, which leverages the underappreciated fact…

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adjustedCurves: Estimating Confounder-Adjusted Survival Curves in R

adjustedCurves:在 R 中估计混杂调整后的生存曲线

Kaplan-Meier curves stratified by treatment allocation are the most popular way to depict causal effects in studies with right-censored time-to-event endpoints. If the treatment is randomly assigned and the sample size of the study is adequate, this method produces unbiased estimates of the population-averaged counterfactual survival curves. However, in observational studies, this is no longer the case. Instead, specific methods that allow adjustment for confounding must be…

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Learning What to Learn: Experimental Design when Combining Experimental with Observational Evidence

学习该学什么:结合实验与观察证据的实验设计

Experiments deliver credible treatment-effect estimates but, because they are costly, are often restricted to specific sites, small populations, or particular mechanisms. A common practice across several fields is therefore to combine experimental estimates with reduced-form or structural external (observational) evidence to answer broader policy questions, such as those involving general equilibrium effects or external validity. We develop a unified framework for the design…

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Selective Inference for Time-Varying Moderated Effects

时变调节效应的选择后推断

Causal effect moderation investigates how the effect of interventions (or treatments) on outcome variables changes based on observed characteristics of individuals, known as potential effect moderators. With advances in data collection, datasets containing many observed features as potential moderators have become increasingly common. High-dimensional analyses often lack interpretability, with important moderators masked by noise, while low-dimensional, marginal analyses…

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Optimal Control Variates for Survey Sampling and Causal Inference

调查抽样与因果推断中的最优控制变量

We propose a family of control variate estimators for variance reduction in design-based survey sampling and causal inference, with and without interference. In these settings, inverse probability weighting (IPW) estimators are widely used, but may have large variance when sampling, treatment, or exposure probabilities are small. Building on the observation that several common estimators, including the Hajek estimator, the normalized estimator, the augmented inverse…

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Sensitivity of weighted least squares estimators to omitted variables

加权最小二乘估计量对遗漏变量的敏感性

We introduce tools for assessing the sensitivity, to unobserved confounding, of a common estimator of causal effects that employs weights: the weighted linear regression of the outcome on the treatment and observed covariates. This estimator's bias is a function of two intuitive weighted partial $R^2$ values: (i) the proportion of weighted variance in the treatment that unobserved confounding explains given the covariates and (ii) the proportion of weighted variance in the…

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Pattern-Based Sequential Multiple Imputation for Missing Data in Clinical Trials: An Extension for Baseline-Only Early Dropout Subjects

临床试验序贯多重插补:仅有基线数据的早期退出者扩展

Under the ICH E9 (R1) addendum, treatment policy strategies for intercurrent events target the treatment effect regardless of treatment discontinuation. Sequential multiple imputation (MI) models that condition each visit's imputation on discontinuation status or pattern reduce bias relative to mixed models and standard MI, but require every subject to contribute at least one post-baseline observation, an assumption violated by subjects who withdraw before any post-baseline…

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NP-LEAP: Nonparametric Latent Exchangeability Prior for Model-Lean Borrowing from Historical Data

NP-LEAP:借用历史数据的非参数潜在可交换性先验

Bayesian dynamic borrowing (BDB) methods leverage historical data to reduce treatment effect uncertainty, yet existing approaches rely on parametric outcome models susceptible to misspecification. We propose the nonparametric latent exchangeability prior (NP-LEAP), an outcome-agnostic, assumption-lean framework to borrow information from historical data. The NP-LEAP performs individual-level exchangeability assessment, inducing Bayesian model averaging over all possible…

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From Estimands to Robust Inference of Treatment Effects in Master Protocol Trials

主方案试验:从目标量定义到治疗效应的稳健推断

Master protocol trials use a single overarching protocol to evaluate multiple interventions, diseases, or disease subtypes, where individuals are often randomized to different subsets of intervention arms based on individual characteristics, enrollment timing, and intervention availability. While offering increased flexibility, this constrained and non-uniform intervention assignment poses two fundamental inferential challenges: the precise definition of treatment effects…

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Randomization inference for treatment effects on survival outcomes

生存结局治疗效应的随机化推断

The log-rank test and Kaplan--Meier plot are standard tools for analyzing time-to-event data in randomized clinical trials, yet neither provides a summary of the magnitude of the treatment effect. Practitioners typically fill this gap by reporting a hazard ratio from a Cox proportional-hazards model or an acceleration factor from an accelerated failure time (AFT) model, but both require assumptions beyond those needed for the log-rank test or Kaplan--Meier estimator. We…

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Estimation and Inference for Peer Effects under Conditional Random Assignment

条件随机分组下同伴效应的估计与推断

Empirical studies of peer effects often exploit conditional random assignment to peer groups within urns. We develop a GMM framework for estimation and inference in this setting. The framework separately identifies endogenous and contextual peer effects and nests tests of random peer-group assignment as a special case. It permits unknown heteroskedasticity and corrects finite-urn bias in variance estimation. Its asymptotic theory allows the number of peer groups to grow…

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Mixed-effects Outcome-Adaptive Lasso for Propensity Score Estimation under Partial Interference

部分干扰下用于倾向得分估计的混合效应结果自适应 Lasso

Interference occurs when one individual's treatment or exposure affects another individual's outcome. In particular, we assume partial interference, where individuals are divided into groups such that there is no interference between individuals in different groups. In observational studies, inverse probability weighting (IPW) based on propensity scores is often used for causal effect estimation. However, under partial interference, the group-level propensity score must be…

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Doubly robust estimation of while-alive estimands in individually-randomized and cluster-randomized trials

个体与整群随机试验中存活期间目标量的双重稳健估计

Randomized trials in chronic disease settings often measure treatment benefit through recurrent non-fatal events that are truncated by death, where conventional summaries either discard recurrences, conflate the treatment effect with survival, or treat death as censoring and forfeit a causal interpretation. While-alive estimands measure event burden per unit time alive, but doubly robust estimation for the exposure-weighted while-alive rate remains undeveloped, particularly…

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Leveraging Generative Artificial Intelligence for Causal Inference with Unstructured Data

利用生成式人工智能对非结构化数据开展因果推断

We introduce GenAI-Powered Inference (GPI), a statistical framework for both causal and predictive inference using unstructured data, including text and images. GPI leverages open-source Generative Artificial Intelligence (GenAI) models---such as large language models and diffusion models---not only to generate unstructured data at scale but also to extract low-dimensional representations that are guaranteed to capture their underlying structure. Applying machine learning to…

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Synthetic Control with Weight Uncertainty: Robust Identification and Statistical Inference

权重不确定性下的合成控制:稳健识别与统计推断

The synthetic control method estimates causal effects by comparing a treated unit with weighted controls matched on its pre-treatment trajectory. However, validity can be compromised when highly correlated controls leave the synthetic weights weakly determined or when treated-control relationships shift after treatment. We propose a new estimand, the weight-robust treatment effect, defined as the optimizer of a worst-case optimization problem over an uncertainty class of…

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How Many Samples Are Needed to Determine Causal Direction? Sharp Minimax Bounds for Bivariate LiNGAM

确定因果方向需要多少样本?双变量 LiNGAM 的尖锐极小极大界

We study how many observations are needed to determine the causal direction between two linearly related variables. Classical LiNGAM theory shows that independent non-Gaussian disturbances identify the direction, but does not quantify the difficulty when the causal effect is weak or the disturbances are nearly Gaussian. Let $β$ bound the absolute structural coefficient from below, let $ν$ measure each standardized disturbance's distance from Gaussianity, and let the…

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Causal mediation analysis for zero-inflated longitudinal data in the presence of treatment non-compliance and multiple mediators

存在处理不依从与多个中介时零膨胀纵向数据的因果中介分析

Understanding whether a digital marketing campaign is effective is central to designing effective customer engagement strategies. We analyze a large-scale, longitudinal promotional email campaign conducted by a U.S.\ retailer to evaluate how value-added incentives, such as free shipping, compare with traditional price discounts in influencing customer purchasing behavior. The analysis is complicated by non-compliance, due to not opening emails, multiple longitudinal…

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Rectified Linear Unit Regression

修正线性单元回归

This paper develops a regression framework for analyzing integrated conditional distribution and quantile functions. The proposed method, termed rectified linear unit (ReLU) regression, projects the ReLU-transformed outcome onto covariates and admits a closed-form estimator. Its population regression function is the best linear approximation to the integrated conditional distribution function of the outcome, and the corresponding convex conjugate, obtained via the…

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Energy Balancing Weights for Mediation Analysis

中介分析的能量平衡权重

Causal mediation analysis requires reconstruction of counterfactual distributions to estimate natural direct and indirect effects. Inverse probability weighting estimators rely on models for treatment assignment and mediator density ratios, whereas moment balancing approaches require researchers to specify in advance which functions of the covariates and mediators should be balanced. We propose Energy Balancing Weights for Mediation Analysis (EBWMA), which targets the joint…

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On the Graphical Rules for Recovering the Average Treatment Effect Under Selection Bias

选择偏差下恢复平均处理效应的图形规则

Selection bias is a major obstacle toward valid causal inference in epidemiology. Over the past decade, several graphical rules based on causal diagrams have been proposed as sufficient identification conditions for addressing selection bias and recovering causal effects. However, these simple graphical rules are typically coupled with specific identification strategies and estimators. In this article, we show two important cases of selection bias that fall outside the scope…

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Toward Efficient Estimation of Regional Treatment Effects in Multi-Regional Clinical Trials

多区域临床试验中区域处理效应的有效估计

A multi-regional clinical trial (MRCT) is a single clinical trial conducted in multiple regions simultaneously under a common protocol, which may be used to support parallel submissions to multiple regulatory authorities. For a regional regulatory authority, treatment effects defined specifically for its own region are more relevant to consider than overall treatment effects based on all regions included in an MRCT. A regional treatment effect can be estimated consistently…

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