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Pulling back the curtain: the road from statistical estimand to machine-learning based estimator for epidemiologists (no wizard required)

从统计目标量到机器学习估计量:面向流行病学研究者的推导指南

Epidemiologists increasingly use causal inference methods that rely on machine learning, as these approaches can relax unnecessary model specification assumptions. While deriving and studying asymptotic properties of such estimators is a task usually associated with statisticians, it is useful for epidemiologists to understand the steps involved, as epidemiologists are often at the forefront of defining important new research questions and translating them into new…

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Online Generalized Method of Moments for Time Series

时间序列的在线广义矩估计

Online learning has gained popularity in recent years due to the urgent need to analyse large-scale streaming data, which can be collected in perpetuity and serially dependent. This motivates us to develop the online generalized method of moments (OGMM), an explicitly updated estimation and inference framework in the time series setting. The OGMM inherits many properties of offline GMM, such as its broad applicability to many problems in econometrics and statistics, natural…

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Zero-Inflated Bandits

零膨胀多臂老虎机

Many real-world bandit applications are characterized by sparse rewards, which can significantly hinder learning efficiency. Leveraging problem-specific structures for careful distribution modeling is recognized as essential for improving estimation efficiency in statistics. However, this approach remains under-explored in the context of bandits. To address this gap, we initiate the study of zero-inflated bandits, where the reward is modeled using a classic semi-parametric…

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Causal effect on the number of life years lost due to a specific event: Average treatment effect and variable importance

特定事件导致寿命损失年数的因果效应:平均处理效应与变量重要性

Competing risk is a common phenomenon when dealing with time-to-event outcomes in biostatistical applications. An attractive estimand in this setting is the "number of life-years lost due to a specific cause of death", Andersen et al. (2013). It provides a direct interpretation on the time-scale on which the data is observed. In this paper, we introduce the causal effect on the number of life years lost due to a specific event, and we give assumptions under which the average…

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Efficient Estimation Under Data Fusion

数据融合下的有效估计

We aim to make inferences about a smooth, finite-dimensional parameter by fusing data from multiple sources together. Previous works have studied the estimation of a variety of parameters in similar data fusion settings, including in the estimation of the average treatment effect and average reward under a policy, with the majority of them merging one historical data source with covariates, actions, and rewards and one data source of the same covariates. In this work, we…

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Semiparametric Localized Principal Stratification Analysis with Continuous Strata

连续主分层的半参数局部化分析

Principal stratification is essential for revealing causal mechanisms involving post-treatment intermediate variables, in real-world applications like surrogate marker evaluation. Principal stratification analysis with continuous intermediate variables is increasingly common but challenging due to the infinite principal strata and the nonidentifiability and nonregularity of principal causal effects. Inspired by recent research, we resolve these challenges by first using a…

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A Semiparametric Bayesian Method for Instrumental Variable Analysis with Partly Interval-Censored Time-to-Event Outcome

部分区间删失事件时间结局的半参数贝叶斯工具变量分析

This paper develops a semiparametric Bayesian instrumental variable analysis method for estimating the causal effect of an endogenous variable when dealing with unobserved confounders and measurement errors with partly interval-censored time-to-event data, where event times are observed exactly for some subjects but left-censored, right-censored, or interval-censored for others. Our method is based on a two-stage Dirichlet process mixture instrumental variable (DPMIV) model…

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Using negative controls to identify causal effects with invalid instrumental variables

利用负对照识别无效工具变量下的因果效应

Many proposals for the identification of causal effects require an instrumental variable that satisfies strong, untestable unconfoundedness and exclusion restriction assumptions. In this paper, we show how one can potentially identify causal effects under violations of these assumptions by harnessing a negative control population or outcome. This strategy allows one to leverage sub-populations for whom the exposure is degenerate, and requires that the instrument-outcome…

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On the Role of Surrogates in Conformal Inference of Individual Causal Effects

替代指标在个体因果效应共形推断中的作用

Learning the Individual Treatment Effect (ITE) is essential for personalized decision-making, yet causal inference has traditionally focused on aggregated treatment effects. While integrating conformal prediction with causal inference can provide valid uncertainty quantification for ITEs, the resulting prediction intervals are often excessively wide, limiting their practical utility. To address this limitation, we introduce \underline{S}urrogate-assisted…

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Treatment Effect Estimation with Observational Network Data using Machine Learning

利用机器学习估计观测网络数据中的处理效应

Causal inference methods for treatment effect estimation usually assume independent units. However, this assumption is often questionable because units may interact, resulting in spillover effects between them. We develop augmented inverse probability weighting (AIPW) for estimation and inference of the expected average treatment effect (EATE) with observational data from a single (social) network with spillover effects. In contrast to overall effects such as the global…

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Semiparametric Single-Index Estimation for Average Treatment Effects

平均处理效应的半参数单指标估计

We propose a semiparametric method to estimate the average treatment effect under the assumption of unconfoundedness given observational data. Our estimation method alleviates misspecification issues of the propensity score function by estimating the single-index link function involved through Hermite polynomials. Our approach is computationally tractable and allows for moderately large dimension covariates. We provide the large sample properties of the estimator and show…

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Causal inference targeting a concentration index for studies of health inequalities

以集中指数为目标的健康不平等因果推断

A concentration index, a standardized covariance between a health variable and relative income ranks, is often used to quantify income-related health inequalities. There is a lack of formal approach to study the effect of an exposure, e.g., education, on such measures of inequality. In this paper we contribute by filling this gap and developing the necessary theory and method. Thus, we define a counterfactual concentration index for different levels of an exposure. We give…

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On Doubly Robust Inference for Double Machine Learning in Semiparametric Regression

半参数回归中双重机器学习的双重稳健推断

Due to concerns about parametric model misspecification, there is interest in using machine learning to adjust for confounding when evaluating the causal effect of an exposure on an outcome. Unfortunately, exposure effect estimators that rely on machine learning predictions are generally subject to so-called plug-in bias, which can render naive $p$-values and confidence intervals invalid. Progress has been made via proposals like targeted minimum loss estimation and more…

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Efficient Generalization and Transportation

因果效应推广与迁移的高效方法

When estimating causal effects, it is important to assess external validity, i.e., determine how useful a given study is to inform a practical question for a specific target population. One challenge is that the covariate distribution in the population underlying a study may be different from that in the target population. If some covariates are effect modifiers, the average treatment effect (ATE) may not generalize to the target population. To tackle this problem, we…

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Trustworthy assessment of heterogeneous treatment effect estimator

异质性处理效应估计器的可信评估

Accurate heterogeneous treatment effect (HTE) estimation is essential for personalized recommendations, making it important to evaluate and compare HTE estimators. Traditional assessment methods are inapplicable due to missing counterfactuals. Current HTE evaluation methods rely on additional estimation or matching on test data, often ignoring the uncertainty introduced and potentially leading to incorrect conclusions. We propose incorporating uncertainty quantification into…

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Semiparametric Inference for Regression-Discontinuity Designs

回归不连续设计的半参数推断

Treatment effects in regression discontinuity designs (RDDs) are often estimated using local regression methods. \cite{Hahn:01} demonstrated that the identification of the average treatment effect at the cutoff in RDDs relies on the unconfoundedness assumption and that, without this assumption, only the local average treatment effect at the cutoff can be identified. In this paper, we propose a semiparametric framework tailored for identifying the average treatment effect in…

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Locally Regular and Efficient Tests in Non-Regular Semiparametric Models

非正则半参数模型中的局部正则与有效检验

This paper considers hypothesis testing in semiparametric models which may be non-regular. I show that C($α$) style tests are locally regular under mild conditions, including in cases where locally regular estimators do not exist, such as models which are (semiparametrically) weakly identified. I characterise the appropriate limit experiment in which to study local (asymptotic) optimality of tests in the non-regular case and generalise classical power bounds to this case. I…

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Semiparametric Joint Modeling to Estimate the Treatment Effect on a Longitudinal Surrogate with Application to Chronic Kidney Disease Trials

估计纵向替代结局治疗效应的半参数联合模型:慢性肾病试验应用

In clinical trials where long follow-up is required to measure the primary outcome of interest, there is substantial interest in using an accepted surrogate outcome that can be measured earlier in time or with less cost to estimate a treatment effect. For example, in clinical trials of chronic kidney disease (CKD), the effect of a treatment is often demonstrated on a surrogate outcome, the longitudinal trajectory of glomerular filtration rate (GFR). However, estimating the…

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Variable importance measures for heterogeneous treatment effects with survival outcome

生存结局下异质性处理效应的变量重要性度量

Treatment effect heterogeneity plays an important role in many areas of causal inference and within recent years, estimation of the conditional average treatment effect (CATE) has received much attention in the statistical community. While accurate estimation of the CATE-function through flexible machine learning procedures provides a tool for prediction of the individual treatment effect, it does not provide further insight into the driving features of potential treatment…

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Semiparametric Discrete Choice Models for Bundles

组合商品选择的半参数离散选择模型

We propose two approaches to estimate semiparametric discrete choice models for bundles. Our first approach is a kernel-weighted rank estimator based on a matching-based identification strategy. We establish its complete asymptotic properties and prove the validity of the nonparametric bootstrap for inference. We then introduce a new multi-index least absolute deviations (LAD) estimator as an alternative, of which the main advantage is its capacity to estimate preference…

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A Double Machine Learning Approach for the Evaluation of COVID-19 Vaccine Effectiveness under the Test-Negative Design: Analysis of Québec Administrative Data

检测阴性设计下评估新冠疫苗有效性的双重机器学习方法

The test-negative design (TND), which is routinely used for monitoring seasonal flu vaccine effectiveness (VE), has recently become integral to COVID-19 vaccine surveillance, notably in Québec, Canada. Some studies have addressed the identifiability and estimation of causal parameters under the TND, but efficiency bounds for nonparametric estimators of the target parameter under the unconfoundedness assumption have not yet been investigated. Motivated by the goal of…

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Data-Adaptive Identification of Effect Modifiers through Stochastic Shift Interventions and Cross-Validated Targeted Learning

通过随机位移干预与交叉验证靶向学习自适应识别效应修饰因素

In epidemiology, identifying subpopulations that are particularly vulnerable to exposures and those who may benefit differently from exposure-reducing interventions is essential. Factors such as age, gender-specific vulnerabilities, and physiological states such as pregnancy are critical for policymakers when setting regulatory guidelines. However, current semi-parametric methods for estimating heterogeneous treatment effects are often limited to binary exposures and can…

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Convolution Mode Regression

卷积众数回归

For highly skewed or fat-tailed distributions, mean or median-based methods often fail to capture the central tendencies in the data. Despite being a viable alternative, estimating the conditional mode given certain covariates (or mode regression) presents significant challenges. Nonparametric approaches suffer from the "curse of dimensionality", while semiparametric strategies often lead to non-convex optimization problems. In order to avoid these issues, we propose a novel…

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Locally robust semiparametric estimation of sample selection models without exclusion restrictions

无排除限制的样本选择模型局部稳健半参数估计

Existing identification and estimation methods for semiparametric sample selection models rely heavily on exclusion restrictions. However, it is difficult in practice to find a credible excluded variable that has a correlation with selection but no correlation with the outcome. In this paper, we establish a new identification result for a semiparametric sample selection model without the exclusion restriction. The key identifying assumptions are nonlinearity on the selection…

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