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Partial Identification of Personalized Treatment Response with Trial-reported Analyses of Binary Subgroups

仅凭试验报告的二元亚组分析,部分识别个体化治疗反应

Medical journals have adhered to a reporting practice that seriously limits the usefulness of published trial findings. Medical decision makers commonly observe many patient covariates and seek to use this information to personalize treatment choices. Yet standard summaries of trial findings only partition subjects into broad subgroups, typically into binary categories. Given this reporting practice, we study the problem of inference on long mean treatment outcomes…

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Unbiased estimation for additive exposure models

加性暴露模型的无偏估计

Causal inference methods have been applied in various fields where researchers want to estimate treatment effects. In traditional causal inference settings, one assumes that the outcome of a unit does not depend on treatments of other units. However, as causal inference methods are extended to more applications, there is a greater need for estimators of general causal effects. We use an exposure mapping framework [Aronow and Samii, 2017] to map the relationship between the…

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A Survey of Causal Inference Frameworks

因果推断框架综述

Causal inference is a science with multi-disciplinary evolution and applications. On the one hand, it measures effects of treatments in observational data based on experimental designs and rigorous statistical inference to draw causal statements. One of the most influential framework in quantifying causal effects is the potential outcomes framework. On the other hand, causal graphical models utilizes directed edges to represent causalities and encodes conditional…

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Greedy Causal Discovery is Geometric

贪婪因果发现的几何结构

Finding a directed acyclic graph (DAG) that best encodes the conditional independence statements observable from data is a central question within causality. Algorithms that greedily transform one candidate DAG into another given a fixed set of moves have been particularly successful, for example the GES, GIES, and MMHC algorithms. In 2010, Studený, Hemmecke and Lindner introduced the characteristic imset polytope, $\operatorname{CIM}_p$, whose vertices correspond to Markov…

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Extreme Continuous Treatment Effects: Measures, Estimation and Inference

极端连续处理效应:度量、估计与推断

This paper concerns estimation and inference for treatment effects in deep tails of the counterfactual distribution of unobservable potential outcomes corresponding to a continuously valued treatment. We consider two measures for the deep tail characteristics: the extreme quantile function and the tail mean function defined as the conditional mean beyond a quantile level. Then we define the extreme quantile treatment effect (EQTE) and the extreme average treatment effect…

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Causal Inference under Network Interference with Noise

带噪声网络干扰下的因果推断

Increasingly, there is a marked interest in estimating causal effects under network interference due to the fact that interference manifests naturally in networked experiments. However, network information generally is available only up to some level of error. We study the propagation of such errors to estimators of average causal effects under network interference. Specifically, assuming a four-level exposure model and Bernoulli random assignment of treatment, we…

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Tree-based Subgroup Discovery In Electronic Health Records: Heterogeneity of Treatment Effects for DTG-containing Therapies

电子健康记录中的树式亚组发现:含 DTG 疗法的治疗效应异质性

The rich longitudinal individual level data available from electronic health records (EHRs) can be used to examine treatment effect heterogeneity. However, estimating treatment effects using EHR data poses several challenges, including time-varying confounding, repeated and temporally non-aligned measurements of covariates, treatment assignments and outcomes, and loss-to-follow-up due to dropout. Here, we develop the Subgroup Discovery for Longitudinal Data (SDLD) algorithm,…

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Assessing the causal effects of a stochastic intervention in time series data: Are heat alerts effective in preventing deaths and hospitalizations?

时间序列中随机干预的因果效应:高温预警能否减少死亡和住院?

The methodological development of this paper is motivated by the need to address the following scientific question: does the issuance of heat alerts prevent adverse health effects? Our goal is to address this question within a causal inference framework in the context of time series data. A key challenge is that causal inference methods require the overlap assumption to hold: each unit (i.e., a day) must have a positive probability of receiving the treatment (i.e., issuing a…

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A Bayesian nonparametric approach for causal inference with multiple mediators

多重中介因果推断的贝叶斯非参数方法

Mediation analysis with contemporaneously observed multiple mediators is an important area of causal inference. Recent approaches for multiple mediators are often based on parametric models and thus may suffer from model misspecification. Also, much of the existing literature either only allow estimation of the joint mediation effect, or, estimate the joint mediation effect as the sum of individual mediator effects, which often is not a reasonable assumption. In this paper,…

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Progression models for repeated measures: Estimating novel treatment effects in progressive diseases

重复测量进展模型:估计进行性疾病的新型治疗效应

Mixed Models for Repeated Measures (MMRMs) are ubiquitous when analyzing outcomes of clinical trials. However, the linearity of the fixed-effect structure in these models largely restrict their use to estimating treatment effects that are defined as linear combinations of effects on the outcome scale. In some situations, alternative quantifications of treatment effects may be more appropriate. In progressive diseases, for example, one may want to estimate if a drug has…

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How estimating nuisance parameters can reduce the variance (with consistent variance estimation)

估计干扰参数如何降低方差并实现一致方差估计

We often estimate a parameter of interest psi when the identifying conditions involve a nuisance parameter theta. Examples from causal inference are Inverse Probability Weighting, Marginal Structural Models and Structural Nested Models. To estimate treatment effects from observational data, these methods posit a (pooled) logistic regression model for the treatment and/or censoring probabilities and estimate these first. These methods are all based on unbiased estimating…

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Covariate Balancing Methods for Randomized Controlled Trials Are Not Adversarially Robust

随机对照试验协变量平衡方法的对抗鲁棒性局限

The first step towards investigating the effectiveness of a treatment via a randomized trial is to split the population into control and treatment groups then compare the average response of the treatment group receiving the treatment to the control group receiving the placebo. In order to ensure that the difference between the two groups is caused only by the treatment, it is crucial that the control and the treatment groups have similar statistics. Indeed, the validity and…

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Testing Monotonicity of Mean Potential Outcomes in a Continuous Treatment with High-Dimensional Data

高维数据下连续处理平均潜在结果的单调性检验

While most treatment evaluations focus on binary interventions, a growing literature also considers continuously distributed treatments. We propose a Cramér-von Mises-type test for testing whether the mean potential outcome given a specific treatment has a weakly monotonic relationship with the treatment dose under a weak unconfoundedness assumption. In a nonseparable structural model, applying our method amounts to testing monotonicity of the average structural function in…

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Bayesian design and analysis of two-arm cluster randomised trials using assurance

基于 assurance 的两组整群随机试验贝叶斯设计与分析

We consider the design of a two-arm superiority cluster randomised controlled trial (RCT) with a continuous outcome. We detail Bayesian inference for the analysis of the trial using a linear mixed-effects model. The treatment is compared to control using the posterior distribution for the treatment effect. We develop the form of the assurance to choose the sample size based on this analysis, and its evaluation using a two loop Monte Carlo sampling scheme. We assess the…

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Neyman allocation is minimax optimal for best arm identification with two arms

两处理最优臂识别中 Neyman 分配的极小极大最优性

This note describes the optimal policy rule, according to the local asymptotic minimax regret criterion, for best arm identification when there are only two treatments. It is shown that the optimal sampling rule is the Neyman allocation, which allocates a constant fraction of units to each treatment in a manner that is proportional to the standard deviation of the treatment outcomes. When the variances are equal, the optimal ratio is one-half. This policy is independent of…

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Covariate Adjustment in Regression Discontinuity Designs

断点回归设计中的协变量调整

The Regression Discontinuity (RD) design is a widely used non-experimental method for causal inference and program evaluation. While its canonical formulation only requires a score and an outcome variable, it is common in empirical work to encounter RD analyses where additional variables are used for adjustment. This practice has led to misconceptions about the role of covariate adjustment in RD analysis, from both methodological and empirical perspectives. In this chapter,…

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Regression discontinuity design in perinatal epidemiology and birth cohort research

围产期流行病学与出生队列研究中的断点回归设计

Regression discontinuity design (RDD) is a quasi-experimental approach to study the causal effects of an intervention/treatment on later health outcomes. It exploits a continuously measured assignment variable with a clearly defined cut-off above or below which the population is at least partially assigned to the intervention/treatment. We describe the RDD and outline the applications of RDD in the context of perinatal epidemiology and birth cohort research. There is an…

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Strategic Decision-Making in the Presence of Information Asymmetry: Provably Efficient RL with Algorithmic Instruments

信息不对称下的策略决策:利用算法工具变量的高效强化学习

We study offline reinforcement learning under a novel model called strategic MDP, which characterizes the strategic interactions between a principal and a sequence of myopic agents with private types. Due to the bilevel structure and private types, strategic MDP involves information asymmetry between the principal and the agents. We focus on the offline RL problem, where the goal is to learn the optimal policy of the principal concerning a target population of agents based…

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Truncated Rank-Based Tests for Two-Part Models with Excessive Zeros and Applications to Microbiome Data

含大量零值的两部分模型截断秩检验及微生物组应用

High-throughput sequencing technology allows us to test the compositional difference of bacteria in different populations. One important feature of human microbiome data is that it often includes a large number of zeros. Such data can be treated as being generated from a two-part model that includes a zero point-mass. Motivated by analysis of such non-negative data with excessive zeros, we introduce several truncated rank-based two-group and multi-group tests for such data,…

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Flexible evaluation of surrogacy in Bayesian adaptive platform studies

贝叶斯自适应平台研究中替代终点的灵活评价

Trial level surrogates are useful tools for improving the speed and cost effectiveness of trials, but surrogates that have not been properly evaluated can cause misleading results. The evaluation procedure is often contextual and depends on the type of trial setting. There have been many proposed methods for trial level surrogate evaluation, but none, to our knowledge, for the specific setting of Bayesian adaptive platform studies. As adaptive studies are becoming more…

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Application of Causal Inference to Analytical Customer Relationship Management in Banking and Insurance

因果推断在银行与保险分析型客户关系管理中的应用

Of late, in order to have better acceptability among various domain, researchers have argued that machine intelligence algorithms must be able to provide explanations that humans can understand causally. This aspect, also known as causability, achieves a specific level of human-level explainability. A specific class of algorithms known as counterfactuals may be able to provide causability. In statistics, causality has been studied and applied for many years, but not in great…

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Valid Instrumental Variables Selection Methods using Negative Control Outcomes and Constructing Efficient Estimator

利用负对照结局筛选有效工具变量并构造高效估计量

In observational studies, instrumental variable (IV) methods are commonly applied when there exists some unmeasured covariates. In Mendelian Randomization (MR), constructing an allele score by using many single nucleotide polymorphisms (SNPs) is often implemented; however, there are risks estimating biased causal effects by including some invalid IVs. Invalid IVs are candidates of IVs associated with some unobserved variables. To solve this problem, we propose a novel…

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Using Machine Learning to Test Causal Hypotheses in Conjoint Analysis

利用机器学习检验联合分析中的因果假设

Conjoint analysis is a popular experimental design used to measure multidimensional preferences. Researchers examine how varying a factor of interest, while controlling for other relevant factors, influences decision-making. Currently, there exist two methodological approaches to analyzing data from a conjoint experiment. The first focuses on estimating the average marginal effects of each factor while averaging over the other factors. Although this allows for…

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A Practical Guide to Counterfactual Estimators for Causal Inference with Time-Series Cross-Sectional Data

时间序列截面数据因果推断的反事实估计实用指南

This paper introduces a simple framework of counterfactual estimation for causal inference with time-series cross-sectional data, in which we estimate the average treatment effect on the treated by directly imputing counterfactual outcomes for treated observations. We discuss several novel estimators under this framework, including the fixed effects counterfactual estimator, interactive fixed effects counterfactual estimator, and matrix completion estimator. They provide…

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