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Continuous time asymptotic representations for adaptive experiments

自适应实验的连续时间渐近表示

This article develops a continuous-time asymptotic framework for analyzing adaptive experiments -- settings in which data collection and treatment assignment evolve dynamically in response to incoming information. A key challenge in analyzing fully adaptive experiments, where the assignment policy is updated after each observation, is that the sequence of policy rules often lack a well-defined asymptotic limit. To address this, we focus instead on the empirical allocation…

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On inferential equivalence classes of causal models

因果模型的推断等价类

Causal models in the same Markov equivalence class are, in the absence of strong assumptions, statistically indistinguishable at any sample size. For modest sample sizes there is also the possibility that several such classes are compatible with the data, pointing to a confidence set of causal models as the appropriate presentation of evidence. Non-identifiability of Gaussian causal models from the same Markov equivalence class corresponds to a plurality of…

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Rerandomization for covariate balance mitigates $p$-hacking caused by strategically selecting covariates in regression adjustment

协变量平衡的再随机化如何缓解回归调整中的显著性操纵

Rerandomization enforces covariate balance across treatment groups in the design stage of experiments. Despite its intuitive appeal, its theoretical justification remains unsatisfying because its benefits of improving efficiency for estimating the average treatment effect diminish if we use regression adjustment in the analysis stage. To strengthen the theory of rerandomization, we show that it mitigates false discoveries resulting from $p$-hacking caused by strategically…

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Partial Homogeneity in Staggered Difference-in-Differences

交错双重差分中的部分同质性

In staggered difference-in-differences (DiD) designs, units enter treatment at different calendar times, so the treatment effect is not a single number but a set of Cohort-Average Treatment effects on the Treated (CATTs), one per cohort-time cell. Estimating every CATT as its own parameter, as the standard fully flexible estimator does, is unbiased but inefficient when some of these effects are in fact equal, whereas pooling them all into a single two-way fixed effects…

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Causal Falsification of Digital Twins

数字孪生的因果证伪

Digital twins are simulation-based models designed to predict how a real-world process will evolve in response to interventions. This modelling paradigm holds substantial promise in many applications, but rigorous procedures for assessing their accuracy are essential for safety-critical settings. We consider how to assess the accuracy of a digital twin using real-world data. We formulate this as a causal inference problem, which leads to a precise definition of what it means…

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Three Routes to One Answer: Reconciling AIPW, TMLE, and Double Machine Learning for Applied Researchers

通向同一答案的三条路径:协调 AIPW、TMLE 与双重机器学习

Augmented inverse-probability weighting (AIPW), targeted maximum likelihood estimation (TMLE), and double/debiased machine learning (DML) are three routes to the same efficient influence function for the average treatment effect --- settled theory we treat as background. This tutorial's contribution is its worked, shared-nuisance reconciliation on real data: what a practitioner must actually match for the routes, and the software packages, to agree. Working the effect of…

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Proximal Individualized Functional Treatment Regimes

近端因果框架下的个体化函数型处理规则

Estimating individualized treatment regimes (ITRs) is fundamental in data-driven personalized decision-making problems, such as precision medicine. Most of the ITR literature either focuses on categorical/continuous treatments or assumes no unmeasured confounding. In this paper, we make the first attempt to estimate the optimal individualized functional treatment regime (IFTR) for observational data where the treatment is a function and unmeasured confounding is present. We…

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A primer on optimal transport for causal inference with observational data

观察数据因果推断中的最优传输入门

The theory of optimal transportation has developed into a powerful and elegant framework for comparing probability distributions, with wide-ranging applications in all areas of science. The fundamental idea of analyzing probabilities by comparing their underlying state space naturally aligns with the core idea of causal inference, where understanding and quantifying counterfactual states is paramount. Despite this intuitive connection, explicit research at the intersection…

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A Continuous-Weighted Win Ratio for Hierarchical Composite Endpoints

层级复合终点的连续加权胜率比

Hierarchical composite endpoints are commonly used in clinical trials when component out- comes differ in clinical importance. The win ratio compares patients across treatment groups according to a pre-specified order of clinical priority and has an intuitive interpretation. A strict hierarchical rule, however, uses a lower-priority endpoint only when all higher-priority endpoints are tied or non-informative. This may reduce power when the treatment effect is mainly ex-…

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Bridging Balancing Weights and Augmentation in Covariate-adjusted Analyses with Time-to-Event Endpoints: Theory and Practical Recommendations

生存结局协变量调整中平衡加权与增广方法的联系

Covariate adjustment improves the efficiency of treatment-effect analyses in randomized clinical trials, provided the adjustment targets the correct quantity. For time-to-event endpoints, two marginal targets are of primary interest: the log-rank test for the presence of a treatment effect and the marginal hazard ratio for its magnitude. Existing covariate adjustment approaches reach these targets by different ways. Augmentation adjusts the log-rank score by regressing…

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An Entropy-based Coefficient of Determination with Adjustment of Optimization Bias

校正优化偏差的熵型决定系数

Classical likelihood-ratio tests and $Δ$AIC exacerbate the statistical significance crisis by scaling with sample size, often flagging negligible improvements as highly significant. While causal estimands like the average treatment effect (ATE) quantify practical magnitude, their reliance on the expectation operator ties them to the data's original coordinate scale. Furthermore, existing pseudo-$R^2$ metrics are inadequate: variance-based measures ignore higher-order…

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Target-Weighted Neyman Allocation: Experimental Design for Heterogeneous Treatment Effects under Population Shift

目标加权 Neyman 分配:总体迁移下异质性处理效应的实验设计

Randomized experiments are often run in one population to guide decisions in another. Allocating by experimental proportions wastes budget on groups that rarely appear in deployment, whereas allocating by deployment proportions under-samples groups that are hard to measure precisely. We propose \textbf{TWNA} (Target-Weighted Neyman Allocation), a two-stage stratified design that uses pilot estimates of group--arm outcome variances to allocate final-stage sample sizes and…

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Asymptotics for estimating a diverging number of parameters -- with and without sparsity

参数数量发散时的估计渐近理论:稀疏与非稀疏情形

We develop a general asymptotic theory for estimating equations whose dimension diverges with the sample size. For both unpenalized and sparse penalized problems, we establish population-level conditions for existence, consistency, uniqueness, and asymptotic normality; in the penalized setting, we also establish selection consistency under a generalized version of the mutual incoherence condition. Our results cover stepwise procedures with a diverging number of steps,…

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Heterogeneous Treatment Effects under Network Interference: A Nonparametric Approach Based on Node Connectivity

网络干扰下异质性处理效应:基于节点连通性的非参数方法

In network settings, interference between units makes causal inference more challenging as outcomes may depend on the treatments received by others in the network. Typical estimands in network settings focus on treatment effects aggregated across individuals in the population. We propose a framework for estimating node-wise counterfactual means, allowing for more granular insights into the impact of network structure on treatment effect heterogeneity. We develop a doubly…

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On the uncertainty from the first-stage estimation of prognostic covariate adjustment in randomized controlled trials

随机对照试验中预后协变量调整的第一阶段估计不确定性

Prognostic covariate adjustment (PROCOVA) is a two-sample two-stage estimation method for covariate adjustment in randomized controlled trials. In the first stage, a prognostic score, defined as the conditional expectation of an outcome given covariates under the control treatment, is estimated using historical data. In the second stage, analysis of covariance with the estimated prognostic score and treatment assignment as explanatory variables is performed, and the average…

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Adversarial Causal Intervention Falsification

对抗式因果干预证伪

Generative models can reproduce an observational distribution while encoding an incorrect causal structure. We study a sequential game in which a structural causal generator proposes observational and interventional distributions, while an adversarial experimentalist selects interventions intended to maximally falsify the generator. The discriminator is therefore not merely a real-versus-synthetic classifier: it is indexed by an intervention and tests whether the generator…

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Micro-randomized Trials with Categorical Treatments and Binary Proximal Outcome: Causal Effect Estimation and Sample Size Calculation

分类处理与二元近端结局微随机试验的因果效应估计及样本量计算

Micro-randomized trials (MRTs) provide a framework for evaluating the marginal and moderated effects of mobile health (mHealth) interventions. In many applications, treatments take the form of categorical variables with multiple levels, such as different message contents or delivery strategies. Many scientifically meaningful longitudinal outcomes in mHealth studies are binary, such as whether a participant opens an app, engages with content, or completes a target behavior…

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Improving the efficiency of infectious disease prevention trials using negative control outcome event times

利用负对照结局事件时间提高传染病预防试验效率

Baseline covariate adjustment can enhance the efficiency of randomized trials by improving precision of treatment effect estimates. However, the precision gain depends on how strongly the baseline covariates are prognostic for the primary outcome. In randomized trials of infectious disease prevention interventions (e.g., vaccines or passively administered antibodies), an individual's exposure to the pathogen is a leading prognostic factor but is rarely measurable at…

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Partial Identification of Causal Effects Using Proxy Variables

利用代理变量对因果效应进行部分识别

Proximal causal inference is a framework for evaluating the causal effects in the presence of unmeasured confounding. For point identification, it leverages a pair of proxy variables to identify a bridge function that matches the dependence of potential outcomes or treatment variables on the hidden factors to corresponding functions of observed proxies. Unique identification requires that proxies are sufficiently relevant for hidden factors, a requirement that has previously…

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A Unified Causal Inference Framework for the Desirability of Outcome Ranking Paradigm in Benefit-Risk Evaluation

获益—风险评价中结局期望排序概率的统一因果推断框架

We developed a unified covariate-adjusted causal inference framework for estimating the desirability of outcome ranking (DOOR) probability for benefit-risk evaluation in randomized trials and observational studies. The framework expresses the DOOR probability as a bilinear functional of the marginal ordinal outcome distributions under the two treatment strategies, estimates conditional ordinal distributions through sequential risk-set hazards, and derives the efficient…

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A Design-Based Minimax Theory for Network Experiments

网络实验的设计型极小极大理论

Network experiments are used throughout the social and medical sciences to investigate causal effects under the presence of interference. While a large body of work has developed improved statistical procedures, the fundamental limits of statistical estimation in these settings is less well understood. In this paper, we develop and investigate a design-based theory of minimax risk for network experiments under an arbitrary neighborhood interference model. Our notion of…

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Differentially Private Tests of Fisher's Sharp Null Hypothesis for Binary Outcomes

二元结局下 Fisher 尖锐零假设的差分隐私检验

Across many disciplines, causal inference often relies on randomized experiments with binary outcomes. In such experiments, often analysts are interested in testing Fisher's sharp null hypothesis, i.e., the treatment has zero effect on the outcome for every study subject. Sometimes the outcomes are sensitive and must be kept confidential, for example, when they comprise physical or mental health measurements. Releasing test statistics or p-values computed with the…

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Structured Semiparametric Estimation of Average Treatment Effects with Treatment-Specific Non-Gaussian Error Distributions

处理组特异非高斯误差下平均处理效应的结构化半参数估计

This paper studies average treatment effect (ATE) estimation for continuous outcomes when the treatment-covariate mean is structured but the error distributions are unknown and may differ across treatment arms in scale, skewness, or tail behavior. We introduce a semiparametric model with a finite-dimensional mean, or a prespecified basis expansion, and separate unrestricted additive error laws for treated and control outcomes. The central theoretical contribution is the…

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The Categorical Instrumental Variable Model: Characterization, Partial Identification, and Statistical Inference

类别型工具变量模型:刻画、部分识别与统计推断

We study categorical instrumental variable (IV) models with instrument, treatment and outcome taking finitely many values. We derive a simple closed-form characterization of the set of joint distributions of potential outcomes that are compatible with a given observed data distribution in terms of a minimal set of inequalities. These inequalities unify several different IV models defined by versions of the independence and exclusion restriction assumptions. They lead to…

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