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Assurance Methods for designing a clinical trial with a delayed treatment effect

延迟治疗效应下临床试验设计的保证概率方法

An assurance calculation is a Bayesian alternative to a power calculation. One may be performed to aid the planning of a clinical trial, specifically setting the sample size or to support decisions about whether or not to perform a study. Immuno-oncology is a rapidly evolving area in the development of anticancer drugs. A common phenomenon that arises in trials of such drugs is one of delayed treatment effects, that is, there is a delay in the separation of the survival…

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Toward a Theory of Causation for Interpreting Neural Code Models

面向神经代码模型解释的因果理论

Neural Language Models of Code, or Neural Code Models (NCMs), are rapidly progressing from research prototypes to commercial developer tools. As such, understanding the capabilities and limitations of such models is becoming critical. However, the abilities of these models are typically measured using automated metrics that often only reveal a portion of their real-world performance. While, in general, the performance of NCMs appears promising, currently much is unknown…

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Direct and Indirect Treatment Effects in the Presence of Semi-Competing Risks

半竞争风险下的直接与间接处理效应

Semi-competing risks refer to the phenomenon that the terminal event (such as death) can censor the non-terminal event (such as disease progression) but not vice versa. The treatment effect on the terminal event can be delivered either directly following the treatment or indirectly through the non-terminal event. We consider two strategies to decompose the total effect into a direct effect and an indirect effect under the framework of mediation analysis in completely…

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Correcting Attrition Bias using Changes-in-Changes

利用变化中的变化方法纠正样本流失偏差

Attrition is a common and potentially important threat to internal validity in treatment effect studies. We extend the changes-in-changes approach to identify the average treatment effect for respondents and the entire study population in the presence of attrition. Our method, which exploits baseline outcome data, can be applied to randomized experiments as well as quasi-experimental difference-in-difference designs. A formal comparison highlights that while widely used…

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Assessing the overall and partial causal well-specification of nonlinear additive noise models

评估非线性加性噪声模型的整体与局部因果设定正确性

We propose a method to detect model misspecifications in nonlinear causal additive and potentially heteroscedastic noise models. We aim to identify predictor variables for which we can infer the causal effect even in cases of such misspecification. We develop a general framework based on knowledge of the multivariate observational data distribution. We then propose an algorithm for finite sample data, discuss its asymptotic properties, and illustrate its performance on…

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A Correction of Pseudo Log-Likelihood Method

伪对数似然方法的一项修正

Pseudo log-likelihood is a type of maximum likelihood estimation (MLE) method used in various fields including contextual bandits, influence maximization of social networks, and causal bandits. However, in previous literature \citep{li2017provably, zhang2022online, xiong2022combinatorial, feng2023combinatorial1, feng2023combinatorial2}, the log-likelihood function may not be bounded, which may result in the algorithm they proposed not well-defined. In this paper, we give a…

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Doubly robust causal inference through penalized bias-reduced estimation: combining non-probability samples with designed surveys

惩罚化偏差缩减的双重稳健因果推断:融合非概率样本与设计调查

Causal inference on the average treatment effect (ATE) using non-probability samples, such as electronic health records (EHR), faces challenges from sample selection bias and high-dimensional covariates. This requires considering a selection model alongside treatment and outcome models that are typical ingredients in causal inference. This paper considers integrating large non-probability samples with external probability samples from a design survey, addressing moderately…

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Bayesian multivariate logistic regression for superiority and inferiority decision-making under observable treatment heterogeneity

可观测治疗异质性下优效与劣效决策的贝叶斯多元逻辑回归

The effects of treatments may differ between persons with different characteristics. Addressing such treatment heterogeneity is crucial to investigate whether patients with specific characteristics are likely to benefit from a new treatment. The current paper presents a novel Bayesian method for superiority decision-making in the context of randomized controlled trials with multivariate binary responses and heterogeneous treatment effects. The framework is based on three…

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A General Form of Covariate Adjustment in Randomized Clinical Trials

随机临床试验中协变量调整的一般形式

In randomized clinical trials, adjusting for baseline covariates can improve credibility and efficiency for demonstrating and quantifying treatment effects. This article studies the augmented inverse propensity weighted (AIPW) estimator, which is a general form of covariate adjustment that uses linear, generalized linear, and non-parametric or machine learning models for the conditional mean of the response given covariates. Under covariate-adaptive randomization, we…

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A Practical Introduction to Regression Discontinuity Designs: Extensions

断点回归设计实用导论:扩展篇

This monograph, together with its accompanying first part Cattaneo, Idrobo and Titiunik (2020), collects and expands the instructional materials we prepared for more than $50$ short courses and workshops on Regression Discontinuity (RD) methodology that we taught between 2014 and 2023. In this second monograph, we discuss several topics in RD methodology that build on and extend the analysis of RD designs introduced in Cattaneo, Idrobo and Titiunik (2020). Our first goal is…

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Covariate-adjusted Group Sequential Comparisons of Survival Probabilities

协变量调整的生存概率组序贯比较

In confirmatory clinical trials, survival outcomes are frequently studied and interim analyses for efficacy and/or futility are often desirable. Methods such as the log rank test and Cox regression model are commonly used to compare treatments in this setting. They rely on a proportional hazards (PH) assumption and are subject to type I error rate inflation and loss of power when PH are violated. Such violations may be expected a priori, particularly when the mechanisms of…

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Resistant Inference in Instrumental Variable Models

工具变量模型中的抗污染推断

The classical tests in the instrumental variable model can behave arbitrarily if the data is contaminated. For instance, one outlying observation can be enough to change the outcome of a test. We develop a framework to construct testing procedures that are robust to weak instruments, outliers and heavy-tailed errors in the instrumental variable model. The framework is constructed upon M-estimators. By deriving the influence functions of the classical weak instrument robust…

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Sample Empirical Likelihood Methods for Causal Inference

用于因果推断的样本经验似然方法

Causal inference is crucial for understanding the true impact of interventions, policies, or actions, enabling informed decision-making and providing insights into the underlying mechanisms that shape our world. In this paper, we establish a framework for the estimation and inference of average treatment effects using a two-sample empirical likelihood function. Two different approaches to incorporating propensity scores are developed. The first approach introduces propensity…

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The Informativeness of Combined Experimental and Observational Data under Dynamic Selection

动态选择下实验与观察数据结合的信息含量

This paper addresses the challenge of estimating the Average Treatment Effect on the Treated Survivors (ATETS; Vikstrom et al., 2018) in the absence of long-term experimental data, utilizing available long-term observational data instead. We establish two theoretical results. First, it is impossible to obtain informative bounds for the ATETS with no model restriction and no auxiliary data. Second, to overturn this negative result, we explore as a promising avenue the recent…

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Learning Directed Acyclic Graphs from Partial Orderings

利用部分因果排序学习有向无环图

Directed acyclic graphs (DAGs) are commonly used to model causal relationships among random variables. In general, learning the DAG structure is both computationally and statistically challenging. Moreover, without additional information, the direction of edges may not be estimable from observational data. In contrast, given a complete causal ordering of the variables, the problem can be solved efficiently, even in high dimensions. In this paper, we consider the intermediate…

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Optimized Model Selection for Estimating Treatment Effects from Costly Simulations of the US Opioid Epidemic

美国阿片类药物流行高成本模拟中的处理效应估计模型选择

Agent-based simulation with a synthetic population can help us compare different treatment conditions while keeping everything else constant within the same population (i.e., as digital twins). Such population-scale simulations require large computational power (i.e., CPU resources) to get accurate estimates for treatment effects. We can use meta models of the simulation results to circumvent the need to simulate every treatment condition. Selecting the best estimating model…

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Distribution-consistency Structural Causal Models

分布一致性结构因果模型

In the field of causal modeling, potential outcomes (PO) and structural causal models (SCMs) stand as the predominant frameworks. However, these frameworks face notable challenges in practically modeling counterfactuals, formalized as parameters of the joint distribution of potential outcomes. Counterfactual reasoning holds paramount importance in contemporary decision-making processes, especially in scenarios that demand personalized incentives based on the joint values of $(Y(0), Y(1))$…

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Identifying Linearly-Mixed Causal Representations from Multi-Node Interventions

从多节点干预识别线性混合的因果表示

The task of inferring high-level causal variables from low-level observations, commonly referred to as causal representation learning, is fundamentally underconstrained. As such, recent works to address this problem focus on various assumptions that lead to identifiability of the underlying latent causal variables. A large corpus of these preceding approaches consider multi-environment data collected under different interventions on the causal model. What is common to…

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Single Proxy Control

单代理混杂控制

Negative control variables are sometimes used in non-experimental studies to detect the presence of confounding by hidden factors. A negative control outcome (NCO) is an outcome that is influenced by unobserved confounders of the exposure effects on the outcome in view, but is not causally impacted by the exposure. Tchetgen Tchetgen (2013) introduced the Control Outcome Calibration Approach (COCA) as a formal NCO counterfactual method to detect and correct for residual…

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A Causal Analysis of CO2 Reduction Strategies in Electricity Markets Through Machine Learning-Driven Metalearners

基于机器学习元学习器的电力市场减排策略因果分析

This study employs the Causal Machine Learning (CausalML) statistical method to analyze the influence of electricity pricing policies on carbon dioxide (CO2) levels in the household sector. Investigating the causality between potential outcomes and treatment effects, where changes in pricing policies are the treatment, our analysis challenges the conventional wisdom surrounding incentive-based electricity pricing. The study's findings suggest that adopting such policies may…

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A Transfer Learning Causal Approach to Evaluate Racial/Ethnic and Geographic Variation in Outcomes Following Congenital Heart Surgery

迁移学习因果方法:先天性心脏手术结局的族裔与地区差异

Congenital heart defects (CHD) are the most prevalent birth defects in the United States and surgical outcomes vary considerably across the country. The outcomes of treatment for CHD differ for specific patient subgroups, with non-Hispanic Black and Hispanic populations experiencing higher rates of mortality and morbidity. A valid comparison of outcomes within racial/ethnic subgroups is difficult given large differences in case-mix and small subgroup sizes. We propose a…

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Interpretable Causal Inference for Analyzing Wearable, Sensor, and Distributional Data

可穿戴设备、传感器与分布型数据的可解释因果推断

Many modern causal questions ask how treatments affect complex outcomes that are measured using wearable devices and sensors. Current analysis approaches require summarizing these data into scalar statistics (e.g., the mean), but these summaries can be misleading. For example, disparate distributions can have the same means, variances, and other statistics. Researchers can overcome the loss of information by instead representing the data as distributions. We develop an…

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Forster-Warmuth Counterfactual Regression: A Unified Learning Approach

Forster–Warmuth 反事实回归:统一学习方法

Series or orthogonal basis regression is one of the most popular non-parametric regression techniques in practice, obtained by regressing the response on features generated by evaluating the basis functions at observed covariate values. The most routinely used series estimator is based on ordinary least squares fitting, which is known to be minimax rate optimal in various settings, albeit under stringent restrictions on the basis functions and the distribution of covariates.…

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Data integration methods for micro-randomized trials

微随机试验的数据整合方法

Existing statistical methods for the analysis of micro-randomized trials (MRTs) are designed to estimate causal excursion effects using data from a single MRT. In practice, however, researchers can often find previous MRTs that employ similar interventions. In this paper, we develop data integration methods that capitalize on this additional information, leading to statistical efficiency gains. To further increase efficiency, we demonstrate how to combine these approaches…

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