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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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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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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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Testing for Causal Influence using a Partial Coherence Statistic

利用偏相干统计量检验因果影响

In this paper we explore partial coherence as a tool for evaluating causal influence of one signal sequence on another. In some cases the signal sequence is sampled from a time- or space-series. The key idea is to establish a connection between questions of causality and questions of partial coherence. Once this connection is established, then a scale-invariant partial coherence statistic is used to resolve the question of causality. This coherence statistic is shown to be 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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Demystifying statistical learning based on efficient influence functions

理解基于有效影响函数的统计学习

Evaluation of treatment effects and more general estimands is typically achieved via parametric modelling, which is unsatisfactory since model misspecification is likely. Data-adaptive model building (e.g. statistical/machine learning) is commonly employed to reduce the risk of misspecification. Naive use of such methods, however, delivers estimators whose bias may shrink too slowly with sample size for inferential methods to perform well, including those based on the…

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Design and Analysis of Bipartite Experiments under a Linear Exposure-Response Model

线性暴露—响应模型下双部图实验的设计与分析

A bipartite experiment consists of one set of units being assigned treatments and another set of units for which we measure outcomes. The two sets of units are connected by a bipartite graph, governing how the treated units can affect the outcome units. In this paper, we consider estimation of the average total treatment effect in the bipartite experimental framework under a linear exposure-response model. We introduce the Exposure Reweighted Linear (ERL) estimator, and show…

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Synthetic Design: An Optimization Approach to Experimental Design with Synthetic Controls

合成设计:利用合成控制优化实验设计

We investigate the optimal design of experimental studies that have pre-treatment outcome data available. The average treatment effect is estimated as the difference between the weighted average outcomes of the treated and control units. A number of commonly used approaches fit this formulation, including the difference-in-means estimator and a variety of synthetic-control techniques. We propose several methods for choosing the set of treated units in conjunction with the…

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Estimation of a score-explained non-randomized treatment effect in fixed and high dimensions

固定维与高维下由评分解释的非随机处理效应估计

Non-randomized treatment effect models are widely used for the assessment of treatment effects in various fields and in particular social science disciplines like political science, psychometry, psychology. More specifically, these are situations where treatment is assigned to an individual based on some of their characteristics (e.g. scholarship is allocated based on merit or antihypertensive treatments are allocated based on blood pressure level) instead of being allocated…

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Ensuring valid inference for hazard ratios after variable selection

变量选择后危险比推断的有效性

The problem of how to best select variables for confounding adjustment forms one of the key challenges in the evaluation of exposure effects in observational studies, and has been the subject of vigorous recent activity in causal inference. A major drawback of routine procedures is that there is no finite sample size at which they are guaranteed to deliver exposure effect estimators and associated confidence intervals with adequate performance. In this work, we will consider…

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Causal Analysis and Classification of Traffic Crash Injury Severity Using Machine Learning Algorithms

利用机器学习分析和分类交通事故伤害严重程度

Causal analysis and classification of injury severity applying non-parametric methods for traffic crashes has received limited attention. This study presents a methodological framework for causal inference, using Granger causality analysis, and injury severity classification of traffic crashes, occurring on interstates, with different machine learning techniques including decision trees (DT), random forest (RF), extreme gradient boosting (XGBoost), and deep neural network…

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Adaptive Combination of Randomized and Observational Data

随机试验与观察数据的自适应组合

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but may be limited in sample size and patient heterogeneity for estimating conditional average treatment effects for a broad range of patients. Estimates from the observational study can potentially compensate for these limitations, but there…

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Identification of Subgroups With Similar Benefits in Off-Policy Policy Evaluation

离策略政策评估中收益相近子群的识别

Off-policy policy evaluation methods for sequential decision making can be used to help identify if a proposed decision policy is better than a current baseline policy. However, a new decision policy may be better than a baseline policy for some individuals but not others. This has motivated a push towards personalization and accurate per-state estimates of heterogeneous treatment effects (HTEs). Given the limited data present in many important applications, individual…

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Online Causal Inference with Application to Near Real-Time Post-Market Vaccine Safety Surveillance

在线因果推断及其在近实时疫苗上市后安全监测中的应用

Streaming data routinely generated by mobile phones, social networks, e-commerce, and electronic health records present new opportunities for near real-time surveillance of the impact of an intervention on an outcome of interest via causal inference methods. However, as data grow rapidly in volume and velocity, storing and combing data become increasingly challenging. The amount of time and effort spent to update analyses can grow exponentially, which defeats the purpose of…

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