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Are Instrumental Variables Really That Instrumental? Endogeneity Resolution in Regression Models for Comparative Studies

工具变量究竟有多必要?比较研究回归模型中的内生性处理

We provide a justification for why, and when, endogeneity will not cause bias in the interpretation of the coefficients in a regression model. This technique can be a viable alternative to, or even used alongside, the instrumental variable method. We show that when performing any comparative study, it is possible to measure the true change in the coefficients under a broad set of conditions. Our results hold, as long as the product of the covariance structure between the…

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Calibration Error for Heterogeneous Treatment Effects

异质性处理效应的校准误差

Recently, many researchers have advanced data-driven methods for modeling heterogeneous treatment effects (HTEs). Even still, estimation of HTEs is a difficult task -- these methods frequently over- or under-estimate the treatment effects, leading to poor calibration of the resulting models. However, while many methods exist for evaluating the calibration of prediction and classification models, formal approaches to assess the calibration of HTE models are limited to the…

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Modelling volume-outcome relationships in health care

医疗服务量与结局关系的建模

Despite the ongoing strong interest in associations between quality of care and the volume of health care providers, a unified statistical framework for analyzing them is missing, and many studies suffer from poor statistical modelling choices. We propose a flexible, additive mixed model for studying volume-outcome associations in health care that takes into account individual patient characteristics as well as provider-specific effects through a multi-level approach. More…

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Bounds for Bias-Adjusted Treatment Effect in Linear Econometric Models

线性计量模型中偏误调整处理效应的界集

In linear econometric models with proportional selection on unobservables, omitted variable bias in estimated treatment effects are real roots of a cubic equation involving estimated parameters from a short and intermediate regression. The roots of the cubic are functions of $δ$, the degree of selection on unobservables, and $R_{max}$, the R-squared in a hypothetical long regression that includes the unobservable confounder and all observable controls. In this paper I…

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Bayesian Nonparametric Adjustment of Confounding

混杂调整的贝叶斯非参数方法

Analysis of observational studies increasingly confronts the challenge of determining which of a possibly high-dimensional set of available covariates are required to satisfy the assumption of ignorable treatment assignment for estimation of causal effects. We propose a Bayesian nonparametric approach that simultaneously 1) prioritizes inclusion of adjustment variables in accordance with existing principles of confounder selection; 2) estimates causal effects in a manner…

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Identifying Principal Stratum Causal Effects Conditional on a Post-treatment Intermediate Response

给定处理后中间响应的主分层因果效应识别

In neoadjuvant trials on early-stage breast cancer, patients are usually randomized into a control group and a treatment group with an additional target therapy. Early efficacy of the new regimen is assessed via the binary pathological complete response (pCR) and the eventual efficacy is assessed via long-term clinical outcomes such as survival. Although pCR is strongly associated with survival, it has not been confirmed as a surrogate endpoint. To fully understand its…

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Robust analyses for longitudinal clinical trials with missing and non-normal continuous outcomes

缺失且非正态连续结局的纵向临床试验稳健分析

Missing data is unavoidable in longitudinal clinical trials, and outcomes are not always normally distributed. In the presence of outliers or heavy-tailed distributions, the conventional multiple imputation with the mixed model with repeated measures analysis of the average treatment effect (ATE) based on the multivariate normal assumption may produce bias and power loss. Control-based imputation (CBI) is an approach for evaluating the treatment effect under the assumption…

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Improving Randomization Tests under Interference Based on Power Analysis

基于功效分析改进存在干扰时的随机化检验

In causal inference, we can consider a situation in which treatment on one unit affects others, i.e., interference exists. In the presence of interference, we cannot perform a classical randomization test directly because a null hypothesis is not sharp. Instead, we need to perform the randomization test restricted to a subset of units and assignments that makes the null hypothesis sharp. A previous study constructed a useful testing method, a biclique test, by reducing the…

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TreatmentEstimatoR: a Dashboard for Estimating Treatment Effects from Observational Health Data

TreatmentEstimatoR:观测健康数据处理效应估计仪表板

Observational health data can be leveraged to measure the real-world use and potential benefits or risks of existing medical interventions. However, lack of programming proficiency and advanced knowledge of causal inference methods excludes some clinicians and non-computational researchers from performing such analyses. Code-free dashboard tools provide accessible means to estimate and visualize treatment effects from observational health data. We present TreatmentEstimatoR,…

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A Fast Non-parametric Approach for Local Causal Structure Learning

局部因果结构学习的快速非参数方法

We study the problem of causal structure learning with essentially no assumptions on the functional relationships and noise. We develop DAG-FOCI, a computationally fast algorithm for this setting that is based on the FOCI variable selection algorithm in~\cite{azadkia2021simple}. DAG-FOCI outputs the set of parents of a response variable of interest. We provide theoretical guarantees of our procedure when the underlying graph does not contain any (undirected) cycle containing…

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Standard and reference-based conditional mean imputation

标准与参照组条件均值插补

Clinical trials with longitudinal outcomes typically include missing data due to missed assessments or structural missingness of outcomes after intercurrent events handled with a hypothetical strategy. Approaches based on Bayesian random multiple imputation and Rubin's rule for pooling results across multiple imputed datasets are increasingly used in order to align the analysis of these trials with the targeted estimand. We propose and justify deterministic conditional mean…

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Sensitivity analysis in longitudinal clinical trials via distributional imputation

通过分布插补开展纵向临床试验敏感性分析

Missing data is inevitable in longitudinal clinical trials. Conventionally, the missing at random assumption is assumed to handle missingness, which however is unverifiable empirically. Thus, sensitivity analysis is critically important to assess the robustness of the study conclusions against untestable assumptions. Toward this end, regulatory agencies often request using imputation models such as return-to-baseline, control-based, and washout imputation. Multiple…

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Personalized Subsidy Rules

个性化补贴规则

Subsidies are commonly used to encourage behaviors that can lead to short- or long-term benefits. Typical examples include subsidized job training programs and provisions of preventive health products, in which both behavioral responses and associated gains can exhibit heterogeneity. This study uses the marginal treatment effect (MTE) framework to study personalized assignments of subsidies based on individual characteristics. First, we derive the optimality condition for a…

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Random Graph Asymptotics for Treatment Effect Estimation under Network Interference

网络干扰下处理效应估计的随机图渐近理论

The network interference model for causal inference places all experimental units at the vertices of an undirected exposure graph, such that treatment assigned to one unit may affect the outcome of another unit if and only if these two units are connected by an edge. This model has recently gained popularity as means of incorporating interference effects into the Neyman--Rubin potential outcomes framework; and several authors have considered estimation of various causal…

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Bias in multivariable Mendelian randomization studies due to measurement error on exposures

暴露测量误差导致的多变量孟德尔随机化偏倚

Multivariable Mendelian randomization estimates the causal effect of multiple exposures on an outcome, typically using summary statistics of genetic variant associations. However, exposures of interest in Mendelian randomization applications will often be measured with error. The summary statistics will therefore not be of the genetic associations with the exposure, but with the exposure measured with error. Classical measurement error will not bias genetic association…

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Undersmoothing Causal Estimators with Generative Trees

利用生成树对因果估计器进行欠平滑处理

Inferring individualised treatment effects from observational data can unlock the potential for targeted interventions. It is, however, hard to infer these effects from observational data. One major problem that can arise is covariate shift where the data (outcome) conditional distribution remains the same but the covariate (input) distribution changes between the training and test set. In an observational data setting, this problem is materialised in control and treated…

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Distributed Design for Causal Inferences on Big Observational Data

大型观察数据因果推断的分布式研究设计

A fundamental issue in causal inference for Big Observational Data is confounding due to covariate imbalances between treatment groups. This can be addressed by designing the data prior to analysis. Existing design methods, developed for traditional observational studies with single designers, can yield unsatisfactory designs with suboptimum covariate balance for Big Observational Data due to their inability to accommodate the massive dimensionality, heterogeneity, and…

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Modelling hetegeneous treatment effects by quantitle local polynomial decision tree and forest

利用分位点局部多项式决策树与森林建模异质性处理效应

To further develop the statistical inference problem for heterogeneous treatment effects, this paper builds on Breiman's (2001) random forest tree (RFT)and Wager et al.'s (2018) causal tree to parameterize the nonparametric problem using the excellent statistical properties of classical OLS and the division of local linear intervals based on covariate quantile points, while preserving the random forest trees with the advantages of constructible confidence intervals and…

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Synthesizing cross-design evidence and cross-format data using network meta-regression

利用网络元回归综合跨研究设计证据与跨格式数据

In network meta-analysis (NMA), we synthesize all relevant evidence about health outcomes with competing treatments. The evidence may come from randomized controlled trials (RCT) or non-randomized studies (NRS) as individual participant data (IPD) or as aggregate data (AD). We present a suite of Bayesian NMA and network meta-regression (NMR) models allowing for cross-design and cross-format synthesis. The models integrate a three-level hierarchical model for synthesizing IPD…

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Synthetic Controls in Action

合成控制的实践应用

In this article we propose a set of simple principles to guide empirical practice in synthetic control studies. The proposed principles follow from formal properties of synthetic control estimators, and pertain to the nature, implications, and prevention of over-fitting biases within a synthetic control framework, to the interpretability of the results, and to the availability of validation exercises. We discuss and visually demonstrate the relevance of the proposed…

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Bayesian Copula Directional Dependence for causal inference on gene expression data

用于基因表达数据因果推断的贝叶斯 Copula 方向依赖

Modelling and understanding directional gene networks is a major challenge in biology as they play an important role in the architecture and function of genetic systems. Copula Directional Dependence (CDD) can measure the directed connectivity among variables without any strict requirements of distributional and linearity assumptions. Furthermore, copulas can achieve that by isolating the dependence structure of a joint distribution. In this work, a novel extension of the…

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Multi-Task Adversarial Learning for Treatment Effect Estimation in Basket Trials

篮式试验中处理效应估计的多任务对抗学习

Estimating treatment effects from observational data provides insights about causality guiding many real-world applications such as different clinical study designs, which are the formulations of trials, experiments, and observational studies in medical, clinical, and other types of research. In this paper, we describe causal inference for application in a novel clinical design called basket trial that tests how well a new drug works in patients who have different types of…

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Minimax Kernel Machine Learning for a Class of Doubly Robust Functionals with Application to Proximal Causal Inference

双重稳健泛函的极小极大核学习及近端因果推断应用

Robins et al. (2008) introduced a class of influence functions (IFs) which could be used to obtain doubly robust moment functions for the corresponding parameters. However, that class does not include the IF of parameters for which the nuisance functions are solutions to integral equations. Such parameters are particularly important in the field of causal inference, specifically in the recently proposed proximal causal inference framework of Tchetgen Tchetgen et al. (2020),…

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A sensitivity analysis approach for the causal hazard ratio in randomized and observational studies

随机与观测研究中因果风险率比的敏感性分析

The Hazard Ratio (HR) is often reported as the main causal effect when studying survival data. Despite its popularity, the HR suffers from an unclear causal interpretation. As already pointed out in the literature, there is a built-in selection bias in the HR, because similarly to the truncation by death problem, the HR conditions on post-treatment survival. A recently proposed alternative, inspired by the Survivor Average Causal Effect (SACE), is the causal HR, defined as…

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