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本期 268 篇
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arXiv · 本期首次发布

Double/debiased machine learning of quantile treatment effects on long-term outcomes in clinical trials

临床试验长期结局分位数处理效应的双重/去偏机器学习

Long-term outcomes are often unavailable in randomized clinical trials, although short-term surrogate outcomes are commonly observed. External observational data may contain the long-term outcome, but causal comparisons based on such data alone are vulnerable to confounding. Existing surrogate-based data integration methods for long-term outcomes have focused primarily on average treatment effects. We study estimation of quantile treatment effects for long-term outcomes in…

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Partial Identification of the Valuation Distribution in Sequential English Auctions

序贯英式拍卖中估值分布的部分识别

This paper extends the incomplete model of Haile and Tamer (2003) from static English auctions to sequential English auctions. Because bidders may wait for future opportunities, the static condition that bidders do not let rivals win at beatable prices need not hold. We replace it with a dynamic opportunity-cost restriction, yielding nonparametric valuation bounds without solving a dynamic equilibrium. Sharp bounds are also characterized. We propose a novel moment-condition…

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Generalizing conditional average treatment effects from nested randomized trials to all trial-eligible individuals

将嵌套随机试验的条件平均处理效应推广至全部符合资格者

Randomized controlled trials often enroll participants whose characteristics differ from those of a target population, which can limit the generalizability of the estimated treatment effects when effect modifiers differ across populations. While existing generalizability methods primarily focus on estimating the average treatment effect (ATE) in the target population, such summaries may obscure important heterogeneity that is relevant for clinical and policy decision-making.…

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Nonparametric inference for sublevel-set probabilities of conditional average treatment effect functions

条件平均处理效应函数下水平集概率的非参数推断

The average treatment effect can obscure important heterogeneity when individuals respond differently to a treatment. While the conditional average treatment effect (CATE) function captures such heterogeneity, it is difficult to communicate when it depends on many covariates. Sublevels sets of a multivariate CATE function are equally complicated objects, but the probability of a sublevel set of a CATE function is a single number with a simple interpretation as the proportion…

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Improving the Efficiency of Subgroup Analysis in Randomized Controlled Trials with TMLE

利用 TMLE 提高随机对照试验子组分析的效率

Subgroup analyses within randomized controlled trials are often underpowered due to limited sample sizes. We address this challenge by leveraging trial participants outside the subgroup of interest to augment estimation within the subgroup. Specifically, we study two Targeted Maximum Likelihood Estimators (TMLEs) that borrow information from non-subgroup participants within the same trial: a TMLE with pooled regression (TMLE-PR) and an Adaptive Targeted Maximum Likelihood…

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Leveraging heterogeneity for identifiability: Bayesian order-based learning of multiple DAGs

利用异质性实现可识别性:多个有向无环图的贝叶斯排序学习

We propose a joint order-based scoring framework for causal structure learning of directed acyclic graph (DAG) models under heterogeneous data settings. We show that leveraging heterogeneity improves the accuracy of causal ordering estimation. In the most favorable case, the causal ordering is identifiable up to two permutations. Building on this framework, we propose an order-based Bayesian method for Gaussian DAG models and establish its theoretical properties in the…

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Re-examining and calibrating weighted survival analysis for causal inference

重新审视并校准用于因果推断的加权生存分析

Causal inference with time-to-event outcomes is fundamental in various scientific studies. In a static setup with fitted propensity scores, weighted Kaplan-Meier estimation for survival probabilities and weighted Breslow-Peto estimation for hazard ratios have been widely used, but their statistical properties have been overlooked or studied only to a limited extent. We re-examine the weighted Kaplan-Meier method by formally linking it with the general framework of augmented…

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Nearest-Neighbour Matching on Unbounded Supports and Covariate Shift Transfer

无界支撑上的最近邻匹配与协变量漂移迁移

Expectations of multivariate functions with missing labels occur in various fields such as transfer learning and average treatment effects. Although non-parametric estimators based on nearest-neighbour matching are frequently used in this context, the existing literature assumes that the covariates live in some well-shaped compact subset of $\R^d$, with densities that are bounded away from zero. In this paper, we show that the usual rates of convergence can be achieved with…

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Policy Learning with Observational Data: The Case of Hepatitis C Treatment for HIV/HCV Co-Infected Patients

基于观察数据的政策学习:HIV/HCV 共感染患者的丙肝治疗

Decision-makers frequently must choose a single action from a finite set of alternatives -- for example, physicians selecting a treatment, investors choosing a portfolio risk level, or judges determining sentences. To improve outcomes, policymakers often issue policy rules or guidelines to inform such choices. In this paper, I show how to generally derive policy rules from observational data in a multi-action framework under relatively weak assumptions about the underlying…

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Sample size and power calculations for causal inference with time-to-event outcomes

事件时间结局因果推断的样本量与功效计算

This paper develops power and sample size formulas for causal inference with time-to-event outcomes. The target estimand is the marginal hazard ratio: the coefficient of a marginal structural Cox proportional hazard model with treatment as the only predictor. We extend the robust sandwich variance theory and derive the analytical form of the asymptotic variance for the inverse probability weighted partial likelihood estimator. Building on this, we derive a new analytical…

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A Workflow for Evaluating Regional Treatment Effect Heterogeneity in Multi-Regional Clinical Trials

多区域临床试验中区域处理效应异质性的评估流程

Multi-regional clinical trials (MRCTs) enable efficient global drug development by assessing treatment effects across regions within a single protocol. While powered for overall efficacy, MRCTs are typically not designed to provide confirmatory evidence on regional differences, making an assessment of observed regional heterogeneity largely exploratory and susceptible to sampling variability. Despite this challenge, understanding regional heterogeneity remains important for…

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Single World Intervention Graphs as Distributions: A Framework for Causal Identification

将单世界干预图视为分布:因果识别框架

Causal inference seeks to estimate the effect of an intervention on an outcome using observed data, typically via Rubin's potential-outcome framework or Pearl's do-calculus. Following section 9 of Richardson and Robins (2013), this essay treats single-world intervention graphs (SWIGs) as representations of both the observed-data distribution and the interventional distribution, rather than as a bridge to potential outcomes. We demonstrate that this perspective provides a…

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Asymptotic Variance Theory for Trimmed Least Squares and Trimmed Least Absolute Deviations in Censored Panel Models with Fixed Effects

固定效应删失面板模型中修剪最小二乘与最小绝对偏差的渐近方差

We study inference using trimmed least squares (TLS) and trimmed least absolute deviations (TLAD) estimators of \citet{honore_trimmed_1992} in censored two-period panel-data models with fixed effects. We show that the published asymptotic variance formulas rely on additional regularity conditions that are not fully stated in the original analysis. For TLS, the published Hessian formula requires that the regressor-difference index vanish only when the regressor difference…

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Valuing Winners: When and How to Correct for Selection Bias in Randomized Experiments

评估胜出方案:何时及如何校正随机实验中的选择偏差

Decision-makers often deploy the best-performing treatment from a randomized experiment, creating a winner's curse: selection favors treatments whose observed outcomes are high partly because of statistical noise, so the naïve estimate of the winner is upward biased. We distinguish two forms of winner's curse, bias relative to the true best treatment (global) and bias relative to the selected treatment's true mean (selective), and link them to regret from deploying a…

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Transporting treatment effects by calibrating large-scale observational outcomes

通过校准大规模观察结局迁移处理效应

A high-quality experimental dataset is often much smaller than a corresponding observational dataset. When this holds with possibly biased measurements of the outcome of interest in the latter, we propose an estimation and inference procedure for a transported treatment effect. Our point estimator can be computed as follows. First, we estimate the conditional average treatment effect (CATE) by calibrating a treatment-control contrast estimated using the observational…

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Double/Debiased Machine Learning for Continuous Treatment Effects in Panel Data with Endogeneity

内生性面板数据中连续处理效应的双重/去偏机器学习

We propose a double/debiased machine learning framework to estimate average derivative effects in nonparametric panel models with two-way fixed effects. It extends instrumental variable methods to panel settings, handles continuous treatments and various forms of endogeneity, and introduces a cross-fitting scheme to restore independence after eliminating time fixed effects. A penalized GMM debiasing term enables automatic debiased machine learning with endogeneity. Our…

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Stable Causal Discovery via Directed Acyclic Graph Aggregation

通过有向无环图聚合实现稳定因果发现

Directed Acyclic Graphs (DAGs) are central to uncovering causal structure in complex systems, yet learning a single DAG from data is often challenging: model uncertainty, finite samples, and a combinatorially large search space frequently yield unstable estimates. We propose DAGgr, a model averaging framework that aggregates multiple candidate DAGs into a single stable representation. Candidate graphs are weighted by their out-of-sample predictive likelihood across repeated…

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SAGA: A Sequence-Adaptive Generative Architecture for Multi-Horizon Probabilistic Forecasting with Adaptive Temporal Conformal Prediction

SAGA:结合自适应时间共形预测的多期限概率预测架构

Microsimulation models used by ministries of finance and central banks rely on parametric processes for lifetime earnings that capture only first and second moments of the conditional distribution and miss long-range nonlinear structure. We propose SAGA, a decoder-only transformer for irregular tabular panel sequences, paired with a split conformal calibration wrapper that delivers individual-level prediction intervals with finite-sample marginal coverage guarantees. Trained…

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Causal treatment effect decompositions with time-to-event outcomes under competing events

竞争事件下生存时间结局的因果治疗效应分解

Inference about treatment effects for time-to-event outcomes is often obscured by the presence of competing events. A particularly complex situation arises when the treatment influences the occurrence of the competing event. A comprehensive assessment should then account for different mechanisms by which the treatment and the competing event together produce the apparent treatment effect. Here, we propose a decomposition of the treatment's effect on the event of interest…

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Set-Valued Policy Learning

集合值政策学习

Conventional treatment policies map patient covariates to a single recommended intervention in order to maximize expected clinical outcomes. Although a rich body of causal inference methods has been developed to estimate such policies, point-valued recommendations can be highly sensitive to estimation uncertainty, model specification, and finite-sample variability, while typically providing little guidance about how confident one should be in the recommended action. In this…

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Identifying Interventional Joint Distributions via Extended Bridge Functions

利用扩展桥函数识别干预联合分布

Existing identification results in proximal causal inference often focus on marginal interventional distributions using standard outcome or treatment bridge functions. These methods do not generally identify joint interventional distributions that contain all proxy variables that were used to define the corresponding bridge functions. In many applications, however, these joint interventional distributions are a natural target of interest. We introduce extended bridge…

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The Harmonic Synthetic Control Method

谐波合成控制方法

Synthetic control methods can produce misleading counterfactual predictions when outcome series contain unit-specific stochastic trends, a common feature of nonstationary macroeconomic data. Existing remedies, such as pre-filtering or differencing, reduce spurious matching but may discard shared nonstationary variation that helps estimate donor weights. We propose Harmonic Synthetic Control (HSC), which replaces this binary choice with a soft allocation mechanism. HSC…

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Endogenous Quantile Regression with Measurement Error in Dependent Variable

因变量存在测量误差时的内生分位数回归

This paper studies quantile regression with an endogenous regressor and measurement error in the dependent variable. Standard quantile regression estimators ignoring these two elements can induce substantial bias. We adopt a control-function approach in a triangular system and show that the conditional quantile coefficient functions, together with all other distributional parameters, are nonparametrically identifiable. Building on this constructive identification result, we…

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Application of Propensity Score Models and Causal Estimators in Observational Studies under Model Misspecification

模型误设下观测研究中倾向得分模型与因果估计量的应用

Propensity score (PS) methods are widely used in observational studies to reduce confounding and estimate causal treatment effects. However, the validity of PS-based causal estimators depends heavily on correct model specification, and model misspecification may lead to substantial bias and instability. In this study, we systematically evaluate the performance of commonly used causal estimators, including response surface modeling (RSM), inverse probability weighting (IPW),…

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