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The Surrogate Index: Combining Short-Term Proxies to Estimate Long-Term Treatment Effects More Rapidly and Precisely

替代指标指数:结合短期代理更快、更精确地估计长期处理效应

Abstract A common challenge in estimating the impact of interventions (e.g. job training programmes, educational programmes) is that many outcomes of interest (e.g. lifetime earnings or other labour market outcomes) are observed with a long delay. In biomedical settings, this is often addressed by using short-term outcomes as so-called “surrogates” for the outcome of interest, e.g. tumour size as a surrogate for mortality in cancer studies. We build on this literature by…

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A more robust approach to multivariable Mendelian randomization

更稳健的多变量孟德尔随机化方法

Summary Multivariable Mendelian randomization uses genetic variants as instrumental variables to infer the direct effects of multiple exposures on an outcome. However, unlike univariable Mendelian randomization, multivariable Mendelian randomization often faces greater challenges with many weak instruments, which can lead to bias not necessarily toward zero and inflation of Type-I errors. In this work, we introduce a new asymptotic regime that allows exposures to have…

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Minimum Resource Threshold Policy Under Partial Interference

部分干扰下的最低资源阈值政策

When developing policies for prevention of infectious diseases, policymakers often set specific, outcome-oriented targets to achieve. For example, when developing a vaccine allocation policy, policymakers may want to distribute them so that at least a certain fraction of individuals in a census block are disease-free and spillover effects due to interference within blocks are accounted for. The article proposes methods to estimate a block-level treatment policy that achieves…

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Assumption-Lean Analysis of Cluster Randomized Trials in Infectious Diseases for Intent-to-Treat Effects and Network Effects

传染病簇随机试验中意向治疗效应与网络效应的弱假设分析

Cluster randomized trials (CRTs) are a popular design to study the effect of interventions in infectious disease settings. However, standard analysis of CRTs primarily relies on strong parametric methods, usually mixed-effect models to account for the clustering structure, and focuses on the overall intent-to-treat (ITT) effect to evaluate effectiveness. The article presents two assumption-lean methods to analyze two types of effects in CRTs, ITT effects and network effects…

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Debiased inverse-variance weighted estimator in two-sample summary-data Mendelian randomization

两样本汇总数据孟德尔随机化中的去偏逆方差加权估计

Mendelian randomization (MR) has become a popular approach to study the effect of a modifiable exposure on an outcome by using genetic variants as instrumental variables. A challenge in MR is that each genetic variant explains a relatively small proportion of variance in the exposure and there are many such variants, a setting known as many weak instruments. To this end, we provide a theoretical characterization of the statistical properties of two popular estimators in MR:…

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Increasing Power for Observational Studies of Aberrant Response: An Adaptive Approach

提高观察性研究中异常结局检验功效的自适应方法

Abstract In many observational studies, the interest is in the effect of treatment on bad, aberrant outcomes rather than the average outcome. For such settings, the traditional approach is to define a dichotomous outcome indicating aberration from a continuous score and use the Mantel–Haenszel test with matched data. For example, studies of determinants of poor child growth use the World Health Organization’s definition of child stunting being height-for-age z-score ≤ − 2.…

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Testing endogeneity with high dimensional covariates

高维协变量下的内生性检验

Modern, high dimensional data has renewed investigation on instrumental variables (IV) analysis, primarily focusing on estimation of effects of endogenous variables and putting little attention towards specification tests. This paper studies in high dimensions the Durbin–Wu–Hausman (DWH) test, a popular specification test for endogeneity in IV regression. We show, surprisingly, that the DWH test maintains its size in high dimensions, but at an expense of power. We propose a…

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Confidence Intervals for Causal Effects with Invalid Instruments by Using Two-Stage Hard Thresholding with Voting

存在无效工具变量时的因果效应置信区间:两阶段硬阈值与投票

Summary A major challenge in instrumental variable (IV) analysis is to find instruments that are valid, or have no direct effect on the outcome and are ignorable. Typically one is unsure whether all of the putative IVs are in fact valid. We propose a general inference procedure in the presence of invalid IVs, called two-stage hard thresholding with voting. The procedure uses two hard thresholding steps to select strong instruments and to generate candidate sets of valid IVs.…

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Instrumental Variables Estimation With Some Invalid Instruments and its Application to Mendelian Randomization

部分工具变量无效时的工具变量估计及孟德尔随机化应用

Instrumental variables have been widely used for estimating the causal effect between exposure and outcome. Conventional estimation methods require complete knowledge about all the instruments’ validity; a valid instrument must not have a direct effect on the outcome and not be related to unmeasured confounders. Often, this is impractical as highlighted by Mendelian randomization studies where genetic markers are used as instruments and complete knowledge about instruments’…

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