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Estimating the Marginal Effect of a Continuous Exposure on an Ordinal Outcome using Data Subject to Covariate-Driven Treatment and Visit Processes

协变量驱动暴露与随访下连续暴露对有序结局的边际效应估计

In the statistical literature, a number of methods have been proposed to ensure valid inference about marginal effects of variables on a longitudinal outcome in settings with irregular monitoring times. However, the potential biases due to covariate-driven monitoring times and confounding have rarely been considered simultaneously, and never in a setting with an ordinal outcome and a continuous exposure. In this work, we propose and demonstrate a methodology for causal…

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Causal Mediation Analysis: Selection with Asymptotically Valid Inference

因果中介分析:具有渐近有效推断的变量选择

Researchers are often interested in learning not only the effect of treatments on outcomes, but also the pathways through which these effects operate. A mediator is a variable that is affected by treatment and subsequently affects outcome. Existing methods for penalized mediation analyses may lead to ignoring important mediators and either assume that finite-dimensional linear models are sufficient to remove confounding bias, or perform no confounding control at all. In…

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DoubleML -- An Object-Oriented Implementation of Double Machine Learning in Python

DoubleML:双重机器学习的 Python 面向对象实现

DoubleML is an open-source Python library implementing the double machine learning framework of Chernozhukov et al. (2018) for a variety of causal models. It contains functionalities for valid statistical inference on causal parameters when the estimation of nuisance parameters is based on machine learning methods. The object-oriented implementation of DoubleML provides a high flexibility in terms of model specifications and makes it easily extendable. The package is…

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The Predictive Individual Effect for Survival Data

生存数据的预测个体效应

The call for patient-focused drug development is loud and clear, as expressed in the 21st Century Cures Act and in recent guidelines and initiatives of regulatory agencies. Among the factors contributing to modernized drug development and improved health-care activities are easily interpretable measures of clinical benefit. In addition, special care is needed for cancer trials with time-to-event endpoints if the treatment effect is not constant over time. We propose the…

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Propensity Score Weighting Analysis of Survival Outcomes Using Pseudo-observations

利用伪观测进行生存结局的倾向得分加权分析

Survival outcomes are common in comparative effectiveness studies and require unique handling because they are usually incompletely observed due to right-censoring. A ``once for all'' approach for causal inference with survival outcomes constructs pseudo-observations and allows standard methods such as propensity score weighting to proceed as if the outcomes are completely observed. For a general class of model-free causal estimands with survival outcomes on user-specified…

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Selection bias in the treatment effect for a principal stratum

主分层处理效应中的选择偏差

Estimation of treatment effect for principal strata has been studied for more than two decades. Existing research exclusively focuses on the estimation, but there is little research on forming and testing hypotheses for principal stratification-based estimands. In this brief report, we discuss a phenomenon in which the true treatment effect for a principal stratum may not equal zero even if the two treatments have the same effect at patient level which implies an equal…

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Using Bayesian Evidence Synthesis Methods to Incorporate Real World Evidence in Surrogate Endpoint Evaluation

以贝叶斯证据综合纳入真实世界证据评估替代终点

Objective: Traditionally validation of surrogate endpoints has been carried out using RCT data. However, RCT data may be too limited to validate surrogate endpoints. In this paper, we sought to improve validation of surrogate endpoints with the inclusion of real world evidence (RWE). Study Design and Setting: We use data from comparative RWE (cRWE) and single arm RWE (sRWE), to supplement RCT evidence for evaluation of progression free survival (PFS) as a surrogate endpoint…

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A generalized definition of the average causal effect for both binary and continuous treatments

兼容二元与连续处理的平均因果效应广义定义

One of the main tasks of causal inference is estimating well-defined causal parameters. One of the main causal parameters is the average causal effect (ACE) - the expected value of the individual level causal effects in the target population. For binary treatments, the individual level causal effect is defined as contrast between potential outcomes. For continuous outcomes, however, there are many such contrasts in finite samples, thus hampering their use as a useful summary…

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A Targeted Approach to Confounder Selection for High-Dimensional Data

高维数据中混杂变量选择的目标化方法

We consider the problem of selecting confounders for adjustment from a potentially large set of covariates, when estimating a causal effect. Recently, the high-dimensional Propensity Score (hdPS) method was developed for this task; hdPS ranks potential confounders by estimating an importance score for each variable and selects the top few variables. However, this ranking procedure is limited: it requires all variables to be binary. We propose an extension of the hdPS to…

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On the Assumptions of Synthetic Control Methods

合成控制方法的假设

Synthetic control (SC) methods have been widely applied to estimate the causal effect of large-scale interventions, e.g., the state-wide effect of a change in policy. The idea of synthetic controls is to approximate one unit's counterfactual outcomes using a weighted combination of some other units' observed outcomes. The motivating question of this paper is: how does the SC strategy lead to valid causal inferences? We address this question by re-formulating the causal…

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Measuring Equity: Funnel Representation Measurement

公平性的测量:产品漏斗中的群体代表性

We present a methodology to measure the gender representation for online product funnels. It is a part of the overall equity framework to better understand our products through funnel analysis. By leveraging the coarsened exact matching method from causal inference literature, we show that the funnel survival ratio metric we design can detect the representation differences inherent in our products. Understanding how big the representation differences are between different…

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Identifying Marginal Treatment Effects in the Presence of Sample Selection

样本选择下边际处理效应的识别

This article presents identification results for the marginal treatment effect (MTE) when there is sample selection. We show that the MTE is partially identified for individuals who are always observed regardless of treatment, and derive uniformly sharp bounds on this parameter under three increasingly restrictive sets of assumptions. The first result imposes standard MTE assumptions with an unrestricted sample selection mechanism. The second set of conditions imposes…

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Quantile Regression under Limited Dependent Variable

受限因变量下的分位数回归

A new Stata command, ldvqreg, is developed to estimate quantile regression models for the cases of censored (with lower and/or upper censoring) and binary dependent variables. The estimators are implemented using a smoothed version of the quantile regression objective function. Simulation exercises show that it correctly estimates the parameters and it should be implemented instead of the available quantile regression methods when censoring is present. An empirical…

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Assessing Time-Varying Causal Effect Moderation in the Presence of Cluster-Level Treatment Effect Heterogeneity

群组层面处理效应异质性下的时变因果效应调节评估

The micro-randomized trial (MRT) is a sequential randomized experimental design to empirically evaluate the effectiveness of mobile health (mHealth) intervention components that may be delivered at hundreds or thousands of decision points. MRTs have motivated a new class of causal estimands, termed "causal excursion effects", for which semiparametric inference can be conducted via a weighted, centered least squares criterion (Boruvka et al., 2018). Existing methods assume…

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Covariate Balancing Sensitivity Analysis for Extrapolating Randomized Trials across Locations

跨地区外推随机试验的协变量平衡敏感性分析

The ability to generalize experimental results from randomized control trials (RCTs) across locations is crucial for informing policy decisions in targeted regions. Such generalization is often hindered by the lack of identifiability due to unmeasured effect modifiers that compromise direct transport of treatment effect estimates from one location to another. We build upon sensitivity analysis in observational studies and propose an optimization procedure that allows us to…

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Efficient counterfactual estimation in semiparametric discrete choice models: a note on Chiong, Hsieh, and Shum (2017)

半参数离散选择模型的高效反事实估计:一则方法补充

I suggest an enhancement of the procedure of Chiong, Hsieh, and Shum (2017) for calculating bounds on counterfactual demand in semiparametric discrete choice models. Their algorithm relies on a system of inequalities indexed by cycles of a large number $M$ of observed markets and hence seems to require computationally infeasible enumeration of all such cycles. I show that such enumeration is unnecessary because solving the "fully efficient" inequality system exploiting…

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Planning a method for covariate adjustment in individually-randomised trials: a practical guide

个体随机试验中协变量调整方法的规划指南

Background: It has long been advised to account for baseline covariates in the analysis of confirmatory randomised trials, with the main statistical justifications being that this increases power and, when a randomisation scheme balanced covariates, permits a valid estimate of experimental error. There are various methods available to account for covariates but it is not clear how to choose among them. Methods: Taking the perspective of writing a statistical analysis plan,…

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Non parametric estimation of causal populations in a counterfactual scenario

反事实情景中因果人群分布的非参数估计

In causality, estimating the effect of a treatment without confounding inference remains a major issue because requires to assess the outcome in both case with and without treatment. Not being able to observe simultaneously both of them, the estimation of potential outcome remains a challenging task. We propose an innovative approach where the problem is reformulated as a missing data model. The aim is to estimate the hidden distribution of \emph{causal populations}, defined…

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Regression-based causal inference with factorial experiments: estimands, model specifications, and design-based properties

析因实验的回归因果推断:目标量、模型设定与设计性质

Factorial designs are widely used due to their ability to accommodate multiple factors simultaneously. The factor-based regression with main effects and some interactions is the dominant strategy for downstream data analysis, delivering point estimators and standard errors via one single regression. Justification of these convenient estimators from the design-based perspective requires quantifying their sampling properties under the assignment mechanism conditioning on the…

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A decomposition method to evaluate the `paradox of progress' with evidence for Argentina

评估“进步悖论”的分解方法:阿根廷证据

The `paradox of progress' is an empirical regularity that associates more education with larger income inequality. Two driving and competing factors behind this phenomenon are the convexity of the `Mincer equation' (that links wages and education) and the heterogeneity in its returns, as captured by quantile regressions. We propose a joint least-squares and quantile regression statistical framework to derive a decomposition in order to evaluate the relative contribution of…

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Deep Quantile and Deep Composite Model Regression

深度分位数回归与深度拼接模型回归

A main difficulty in actuarial claim size modeling is that there is no simple off-the-shelf distribution that simultaneously provides a good distributional model for the main body and the tail of the data. In particular, covariates may have different effects for small and for large claim sizes. To cope with this problem, we introduce a deep composite regression model whose splicing point is given in terms of a quantile of the conditional claim size distribution rather than a…

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A Robust, Differentially Private Randomized Experiment for Evaluating Online Educational Programs With Sensitive Student Data

面向敏感学生数据的稳健差分隐私随机实验

Randomized control trials (RCTs) have been the gold standard to evaluate the effectiveness of a program, policy, or treatment on an outcome of interest. However, many RCTs assume that study participants are willing to share their (potentially sensitive) data, specifically their response to treatment. This assumption, while trivial at first, is becoming difficult to satisfy in the modern era, especially in online settings where there are more regulations to protect…

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Estimation and visualization of treatment effects for multiple outcomes

多结果变量处理效应的估计与可视化

We consider a randomized controlled trial between two groups. The objective is to identify a population with characteristics such that the test therapy is more effective than the control therapy. Such a population is called a subgroup. This identification can be made by estimating the treatment effect and identifying interactions between treatments and covariates. To date, many methods have been proposed to identify subgroups for a single outcome. There are also multiple…

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Dimension-Free Average Treatment Effect Inference with Deep Neural Networks

利用深度神经网络进行不依赖维数的平均处理效应推断

This paper investigates the estimation and inference of the average treatment effect (ATE) using deep neural networks (DNNs) in the potential outcomes framework. Under some regularity conditions, the observed response can be formulated as the response of a mean regression problem with both the confounding variables and the treatment indicator as the independent variables. Using such formulation, we investigate two methods for ATE estimation and inference based on the…

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