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Nested Instrumental Variables Analysis: Switcher Average Treatment Effect, Identification, Efficient Estimation and Generalizability

嵌套工具变量分析:转换者平均处理效应及可推广性

Instrumental variables (IVs) are widely used to estimate causal effects from non-randomized data. A canonical example is a randomized trial with noncompliance, in which the randomized treatment assignment serves as an IV for the non-ignorable treatment received. Under a monotonicity assumption, a valid IV nonparametrically identifies the average treatment effect among a latent complier subgroup, whose generalizability is often under debate. In many studies, there exist…

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Robust Causal Inference for EHR-based Studies of Point Exposures with Missingness in Eligibility Criteria

Missingness in variables that define study eligibility criteria is a seldom addressed challenge in electronic health record (EHR)-based settings. Patients with incomplete eligibility information are typically excluded from analysis without consideration of the (implicit) assumptions being made, leaving study conclusions subject to potential selection bias. In an effort to ascertain eligibility for more patients, researchers may look back further in time prior to study…

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Generalized projection tests for function-valued parameters with applications to testing structural causal assumptions

函数值参数的广义投影检验及其在结构性因果假设检验中的应用

Structural assumptions are central to the causal inference literature. In practice, it is often crucial to assess their validity or to test implications that follow from them. In many settings, such tests can be framed as evaluating whether a function-valued parameter equals zero. In this paper, we propose a class of generalized projection tests based on series estimators for function-valued parameters. We establish conditions under which the proposed tests are valid and…

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Empirical stratification for predictive treatment effect heterogeneity with post-treatment variables

利用处理后变量研究预测性处理效应异质性的经验分层

Post-treatment variables (PVs), such as intercurrent events, treatment noncompliance, and behavioral responses to treatment, provide information about individuals' post-treatment responses and may help characterize heterogeneity in treatment effects on the primary outcome. This paper develops an empirical stratification framework to study the treatment effect heterogeneity across baseline-predicted PV response profiles. Specifically, we construct empirical scores from…

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Graph Neural Networks for Generalized Mundlak Estimator under Network Confounding

网络混杂下基于图神经网络的广义 Mundlak 估计量

This paper proposes a generalized Mundlak estimator based on graph neural networks (GME-GNN). The estimator is designed to mitigate bias arising from group-level heterogeneity and to accommodate within-group dependence among individuals. Traditional fixed-effects models handle group heterogeneity via group-specific intercepts, but require overly strict linear additivity and intra-group independence assumptions, and are confined to within-group comparisons. Rather than…

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A tutorial on conducting sample size and power calculations for detecting treatment effect heterogeneity in cluster randomized trials with linear mixed models

用线性混合模型检测整群随机试验处理效应异质性:样本量与功效教程

Cluster-randomized trials (CRTs) are a well-established class of designs for evaluating community-based interventions. An essential task in planning these trials is determining the number of clusters and cluster sizes needed to achieve sufficient statistical power for detecting a clinically relevant effect size. While methods for evaluating the average treatment effect (ATE) for the entire study population are well-established, sample size methods for testing heterogeneity…

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A Statistical Framework for Understanding Causal Effects that Vary by Treatment Initiation Time in EHR-based Studies

电子健康记录研究中随治疗启动时间变化的因果效应框架

Standard practice in electronic health record (EHR)-based studies evaluating the comparative effectiveness of bariatric surgery relative to no surgery is to estimate and report a constant treatment effect across calendar time. However, real-world treatment strategies can evolve, particularly when comparators include standard of care or surgical procedures where techniques may improve, making it clinically important to ascertain whether efficacy of bariatric surgery has…

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Identification in Nonlinear Dynamic Panel Models under Partial Stationarity

部分平稳性下非线性动态面板模型的识别

This paper provides a general identification approach for a wide range of nonlinear panel data models, including binary choice, ordered response, and other types of limited dependent variable models. Our approach accommodates dynamic models with any number of lagged dependent variables as well as other types of endogenous covariates. Our identification strategy relies on a partial stationarity condition, which allows for not only an unknown distribution of errors, but also…

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GMM with Many Weak Moment Conditions and Nuisance Parameters: General Theory and Applications to Causal Inference

大量弱矩条件与干扰参数下的 GMM:一般理论及因果推断应用

Weak identification arises in many statistical problems when key variables exhibit weak correlations-for example, when instrumental variables correlate weakly with treatment, or when proxy variables correlate weakly with unmeasured confounders. Under weak identification, standard estimation methods such as the generalized method of moments (GMM) can produce substantial bias, both in finite samples and asymptotically. This challenge is compounded in modern applications that…

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Handling incomplete outcomes and covariates in cluster-randomized trials: doubly-robust estimation, efficiency considerations, and sensitivity analysis

整群随机试验中不完整结果与协变量的处理

In cluster-randomized trials (CRTs), missing data can occur in various ways, including missing values in outcomes and baseline covariates at the individual or cluster level, or completely missing information for non-participants. Among the various types of missing data in CRTs, missing outcomes have attracted the most attention. However, no existing methods simultaneously address all aforementioned types of missing data in CRTs. To fill in this gap, we propose a…

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Adjusted Nelson--Aalen estimators by inverse treatment probability weighting with an estimated propensity score

使用估计倾向得分进行逆处理概率加权的调整 Nelson–Aalen 估计

Inverse probability of treatment weighting (IPW) has been well applied in causal inference to estimate population-level estimands from observational studies. For time-to-event outcomes, the failure time distribution can be estimated by estimating the cumulative hazard in the presence of random right censoring. IPW can be performed by weighting the event counting process and at-risk process by the inverse treatment probability, resulting in an adjusted Nelson--Aalen estimator…

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Partial Identification of Binary Choice Models with Misreported Outcomes

结果误报下二元选择模型的部分识别

This paper provides partial identification of various binary choice models with misreported dependent variables. We propose two distinct approaches by exploiting different instrumental variables respectively. In the first approach, the instrument is assumed to only affect the true dependent variable but not misreporting probabilities. The second approach uses an instrument that influences misreporting probabilities monotonically while having no effect on the true dependent…

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IV Regressions without Exclusion Restrictions

无需排除限制的工具变量回归

We study identification and estimation of endogenous linear and nonlinear regression models without excluded instrumental variables, based on the standard mean independence condition and a nonlinear relevance condition. Based on the identification results, we propose two semiparametric estimators as well as a discretization-based estimator that does not require any nonparametric regressions. We establish their asymptotic normality and demonstrate via simulations their robust…

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Double sampling for informatively missing data in electronic health record-based comparative effectiveness research

电子健康记录比较效果研究中信息性缺失数据的双重抽样

Missing data arise in most applied settings and are ubiquitous in electronic health records (EHR). When data are missing not at random (MNAR) with respect to measured covariates, sensitivity analyses are often considered. These post-hoc solutions, however, are often unsatisfying in that they are not guaranteed to yield concrete conclusions. Motivated by an EHR-based study of long-term outcomes following bariatric surgery, we consider the use of double sampling as a means to…

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Covariate Adjustment in Randomized Clinical Trials with Missing Covariate and Outcome Data

协变量和结果缺失时随机临床试验的协变量调整

When analyzing data from randomized clinical trials, covariate adjustment can be used to account for chance imbalance in baseline covariates and to increase precision of the treatment effect estimate. A practical barrier to covariate adjustment is the presence of missing data. In this paper, in the light of recent theoretical advancement, we first review several covariate adjustment methods with incomplete covariate data. We investigate the implications of the missing data…

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Estimating Marginal Treatment Effect in Cluster Randomized Trials with Multi-level Missing Outcomes

多层结局缺失下整群随机试验的边际处理效应估计

Analyses of cluster randomized trials (CRTs) can be complicated by informative missing outcome data. Methods such as inverse probability weighted generalized estimating equations have been proposed to account for informative missingness by weighting the observed individual outcome data in each cluster. These existing methods have focused on settings where missingness occurs at the individual level and each cluster has partially or fully observed individual outcomes. In the…

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Testing and Identifying Substitution and Complementarity Patterns

替代与互补关系模式的检验和识别

This paper studies semiparametric identification of substitution and complementarity patterns between two goods using a panel multinomial choice model with bundles. The model allows the two goods to be either substitutes or complements and admits heterogeneous complementarity through observed characteristics. I first provide testable implications for the complementarity relationship between goods. I then characterize the sharp identified set for the model parameters and…

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Point Identification of LATE with Two Imperfect Instruments

利用两个不完美工具变量点识别局部平均处理效应

This paper characterizes point identification results of the local average treatment effect (LATE) using two imperfect instruments. The classical approach (Imbens and Angrist (1994)) establishes the identification of LATE via an instrument that satisfies exclusion, monotonicity, and independence. However, it may be challenging to find a single instrument that satisfies all these assumptions simultaneously. My paper uses two instruments but imposes weaker assumptions on both…

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Determining the number of factors in a large-dimensional generalised factor model

大维广义因子模型中的因子个数确定

This paper proposes new estimators of the number of factors for a generalised factor model with more relaxed assumptions than the strict factor model. Under the framework of large cross-sections $N$ and large time dimensions $T$, we first derive the bias-corrected estimator $\hat σ^2_*$ of the noise variance in a generalised factor model by random matrix theory. Then we construct three information criteria based on $\hat σ^2_*$, further propose the consistent estimators of…

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