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Validity of tests for time-to-event endpoints in studies with the Pocock and Simon covariate-adaptive randomization

Pocock–Simon 协变量自适应随机化下生存终点检验的有效性

In the presence of prognostic covariates, inference about the treatment effect with time-to-event endpoints is mostly conducted via the stratified log-rank test or the score test based on the Cox proportional hazards model. In their ground-breaking work Ye and Shao (2020) have demonstrated theoretically that when the model is misspecified, the robust score test (Wei and Lin, 1989) as well as the unstratified log-rank test are conservative in trials with stratified…

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Causal inference from treatment-control studies having an additional factor with unknown assignment mechanism

附加因素分配机制未知时的处理与对照研究因果推断

Consider a situation with two treatments, the first of which is randomized but the second is not, and the multifactor version of this. Interest is in treatment effects, defined using standard factorial notation. We define estimators for the treatment effects and explore their properties when there is information about the nonrandomized treatment assignment and when there is no information on the assignment of the nonrandomized treatment. We show when and how hidden…

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Estimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions

受新冠疫情影响的临床试验:目标估计量及其估计方法

The COVID-19 pandemic continues to affect the conduct of clinical trials globally. Complications may arise from pandemic-related operational challenges such as site closures, travel limitations and interruptions to the supply chain for the investigational product, or from health-related challenges such as COVID-19 infections. Some of these complications lead to unforeseen intercurrent events in the sense that they affect either the interpretation or the existence of the…

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Sensitivity Analysis in the Generalization of Experimental Results

实验结果推广中的敏感性分析

Randomized controlled trials (RCT's) allow researchers to estimate causal effects in an experimental sample with minimal identifying assumptions. However, to generalize or transport a causal effect from an RCT to a target population, researchers must adjust for a set of treatment effect moderators. In practice, it is impossible to know whether the set of moderators has been properly accounted for. In the following paper, I propose a three parameter sensitivity analysis for…

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Evaluation Methods and Measures for Causal Learning Algorithms

因果学习算法的评价方法与指标

The convenient access to copious multi-faceted data has encouraged machine learning researchers to reconsider correlation-based learning and embrace the opportunity of causality-based learning, i.e., causal machine learning (causal learning). Recent years have therefore witnessed great effort in developing causal learning algorithms aiming to help AI achieve human-level intelligence. Due to the lack-of ground-truth data, one of the biggest challenges in current causal…

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Causal Inference Using Tractable Circuits

利用可处理电路进行因果推断

The aim of this paper is to discuss a recent result which shows that probabilistic inference in the presence of (unknown) causal mechanisms can be tractable for models that have traditionally been viewed as intractable. This result was reported recently to facilitate model-based supervised learning but it can be interpreted in a causality context as follows. One can compile a non-parametric causal graph into an arithmetic circuit that supports inference in time linear in the…

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Simple yet Sharp Sensitivity Analysis for Unmeasured Confounding

未测混杂的简明且锐利的敏感性分析

We present a method for assessing the sensitivity of the true causal effect to unmeasured confounding. The method requires the analyst to set two intuitive parameters. Otherwise, the method is assumption-free. The method returns an interval that contains the true causal effect, and whose bounds are arbitrarily sharp, i.e. practically attainable. We show experimentally that our bounds can be tighter than those obtained by the method of Ding and VanderWeele (2016a) which,…

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Incorporating baseline covariates to validate surrogate endpoints with a constant biomarker under control arm

纳入基线协变量验证对照组生物标志物恒定的替代终点

A surrogate endpoint S in a clinical trial is an outcome that may be measured earlier or more easily than the true outcome of interest T. In this work, we extend causal inference approaches to validate such a surrogate using potential outcomes. The causal association paradigm assesses the relationship of the treatment effect on the surrogate with the treatment effect on the true endpoint. Using the principal surrogacy criteria, we utilize the joint conditional distribution…

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Ergodic descriptors of nonergodic stochastic processes

非遍历随机过程的遍历性描述指标

The stochastic processes underlying the growth and stability of biological and psychological systems reveal themselves when far from equilibrium. Far from equilibrium, nonergodicity reigns. Nonergodicity implies that the average outcome for a group/ensemble (i.e., of representative organisms/minds) is not necessarily a reliable estimate of the average outcome for an individual over time. However, the scientific interest in causal inference suggests that we somehow aim at…

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Some asymptotic results for time series model selection

时间序列模型选择的若干渐近结果

We consider the model selection problem for a large class of time series models, including, multivariate count processes, causal processes with exogenous covariates. A procedure based on a general penalized contrast is proposed. Some asymptotic results for weak and strong consistency are established. The non consistency issue is addressed, and a class of penalty term, that does not ensure consistency is provided. Examples of continuous valued and multivariate count…

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Causal Bias Quantification for Continuous Treatments

连续处理的因果偏差量化

We extend the definition of the marginal causal effect to the continuous treatment setting and develop a novel characterization of causal bias in the framework of structural causal models. We prove that our derived bias expression is zero if, and only if, the causal effect is identifiable via covariate adjustment. We show that under some restrictions on the structural equations, the causal bias can be estimated efficiently and allows for causal regularization of predictive…

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Causal inference under mis-specification: adjustment based on the propensity score

模型误设下的因果推断:基于倾向得分的调整

We study Bayesian approaches to causal inference via propensity score regression. Much of the Bayesian literature on propensity score methods have relied on approaches that cannot be viewed as fully Bayesian in the context of conventional `likelihood times prior' posterior inference; in addition, most methods rely on parametric and distributional assumptions, and presumed correct specification. We emphasize that causal inference is typically carried out in settings of…

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On the Use of Instrumental Variables in Mediation Analysis

中介分析中工具变量的使用

Empirical researchers are often interested in not only whether a treatment affects an outcome of interest, but also how the treatment effect arises. Causal mediation analysis provides a formal framework to identify causal mechanisms through which a treatment affects an outcome. The most popular identification strategy relies on so-called sequential ignorability (SI) assumption which requires that there is no unobserved confounder that lies in the causal paths between the…

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Meta-Learners for Estimation of Causal Effects: Finite Sample Cross-Fit Performance

因果效应元学习器:交叉拟合的有限样本表现

Estimation of causal effects using machine learning methods has become an active research field in econometrics. In this paper, we study the finite sample performance of meta-learners for estimation of heterogeneous treatment effects under the usage of sample-splitting and cross-fitting to reduce the overfitting bias. In both synthetic and semi-synthetic simulations we find that the performance of the meta-learners in finite samples greatly depends on the estimation…

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Causal Discovery with Heterogeneous Observational Data

异质性观测数据下的因果发现

We consider the problem of causal discovery (structure learning) from heterogeneous observational data. Most existing methods assume a homogeneous sampling scheme, which leads to misleading conclusions when violated in many applications. To this end, we propose a novel approach that exploits data heterogeneity to infer possibly cyclic causal structures from causally insufficient systems. The core idea is to model the direct causal effects as functions of exogenous covariates…

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Heterogeneous Treatment Effect Estimation based on a Partially Linear Nonparametric Bayes Model

基于部分线性非参数贝叶斯模型的异质性处理效应估计

Recently, conditional average treatment effect (CATE) estimation has been attracting much attention due to its importance in various fields such as statistics, social and biomedical sciences. This study proposes a partially linear nonparametric Bayes model for the heterogeneous treatment effect estimation. A partially linear model is a semiparametric model that consists of linear and nonparametric components in an additive form. A nonparametric Bayes model that uses a…

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BCDAG: An R package for Bayesian structure and Causal learning of Gaussian DAGs

BCDAG:高斯有向无环图的贝叶斯结构与因果学习R包

Directed Acyclic Graphs (DAGs) provide a powerful framework to model causal relationships among variables in multivariate settings; in addition, through the do-calculus theory, they allow for the identification and estimation of causal effects between variables also from pure observational data. In this setting, the process of inferring the DAG structure from the data is referred to as causal structure learning or causal discovery. We introduce BCDAG, an R package for…

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Estimating Heterogeneous Treatment Effects for General Responses

一般响应变量的异质性处理效应估计

Heterogeneous treatment effect models allow us to compare treatments at subgroup and individual levels, and are of increasing popularity in applications like personalized medicine, advertising, and education. In this talk, we first survey different causal estimands used in practice, which focus on estimating the difference in conditional means. We then propose DINA, the difference in natural parameters, to quantify heterogeneous treatment effect in exponential families and…

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Bayesian Covariance Structure Modeling of Multi-Way Nested Data

多重嵌套数据的贝叶斯协方差结构建模

A Bayesian multivariate model with a structured covariance matrix for multi-way nested data is proposed. This flexible modeling framework allows for positive and for negative associations among clustered observations, and generalizes the well-known dependence structure implied by random effects. A conjugate shifted-inverse gamma prior is proposed for the covariance parameters which ensures that the covariance matrix remains positive definite under posterior analysis. A…

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Distributed Learning of Generalized Linear Causal Networks

广义线性因果网络的分布式学习

We consider the task of learning causal structures from data stored on multiple machines, and propose a novel structure learning method called distributed annealing on regularized likelihood score (DARLS) to solve this problem. We model causal structures by a directed acyclic graph that is parameterized with generalized linear models, so that our method is applicable to various types of data. To obtain a high-scoring causal graph, DARLS simulates an annealing process to…

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High-dimensional model-assisted inference for treatment effects with multi-valued treatments

多值处理效应的高维模型辅助推断

Consider estimation of average treatment effects with multi-valued treatments using augmented inverse probability weighted (IPW) estimators, depending on outcome regression and propensity score models in high-dimensional settings. These regression models are often fitted by regularized likelihood-based estimation, while ignoring how the fitted functions are used in the subsequent inference about the treatment parameters. Such separate estimation can be associated with known…

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Treatment effect estimation with Multilevel Regression and Poststratification

利用多层回归与后分层估计处理效应

Multilevel regression and poststratification (MRP) is a flexible modeling technique that has been used in a broad range of small-area estimation problems. Traditionally, MRP studies have been focused on non-causal settings, where estimating a single population value using a nonrepresentative sample was of primary interest. In this manuscript, MRP-style estimators will be evaluated in an experimental causal inference setting. We simulate a large-scale randomized control trial…

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Estimation of Conditional Random Coefficient Models using Machine Learning Techniques

利用机器学习估计条件随机系数模型

Nonparametric random coefficient (RC)-density estimation has mostly been considered in the marginal density case under strict independence of RCs and covariates. This paper deals with the estimation of RC-densities conditional on a (large-dimensional) set of control variables using machine learning techniques. The conditional RC-density allows to disentangle observable from unobservable heterogeneity in partial effects of continuous treatments adding to a growing literature…

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Automated causal inference in application to randomized controlled clinical trials

应用于随机对照临床试验的自动因果推断

Randomized controlled trials (RCTs) are considered as the gold standard for testing causal hypotheses in the clinical domain. However, the investigation of prognostic variables of patient outcome in a hypothesized cause-effect route is not feasible using standard statistical methods. Here, we propose a new automated causal inference method (AutoCI) built upon the invariant causal prediction (ICP) framework for the causal re-interpretation of clinical trial data. Compared to…

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