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Nested Instrumental Variables Analysis: Switcher Average Treatment Effect, Identification, Efficient Estimation and Generalizability

嵌套工具变量分析:转换者平均处理效应及可推广性

Instrumental variables (IVs) are widely used to estimate causal effects from non-randomized data. A canonical example is a randomized trial with noncompliance, in which the randomized treatment assignment serves as an IV for the non-ignorable treatment received. Under a monotonicity assumption, a valid IV nonparametrically identifies the average treatment effect among a latent complier subgroup, whose generalizability is often under debate. In many studies, there exist…

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Structural Nested Mean Models Under Parallel Trends Assumptions

平行趋势假设下的结构嵌套均值模型

We link and extend two approaches to estimating time-varying treatment effects on repeated continuous outcomes--time-varying Difference in Differences (DiD; see Roth et al. (2023) and Chaisemartin et al. (2023) for reviews) and Structural Nested Mean Models (SNMMs; see Vansteelandt and Joffe (2014) for a review). In particular, we show that SNMMs, previously known to be nonparametrically identified under a no unobserved confounding assumption, are also identified under a…

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Nonparametric tests of treatment effect homogeneity for policy-makers

面向政策制定者的处理效应同质性非参数检验

Recent work has focused on nonparametric estimation of conditional treatment effects, but inference has remained relatively unexplored. We propose a class of nonparametric tests for both quantitative and qualitative treatment effect heterogeneity. The tests can incorporate a variety of structured assumptions on the conditional average treatment effect, allow for both continuous and discrete covariates, and do not require sample splitting to obtain a tractable asymptotic null…

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On estimands in target trial emulation

目标试验模拟中的目标估计量

The target trial framework enables causal inference from longitudinal observational data by emulating randomized trials initiated at multiple time points. Precision is often improved by pooling information across trials, with standard models typically assuming - among other things - a time-constant treatment effect. However, this obscures interpretation when the true treatment effect varies, which we argue to be likely as a result of relying on noncollapsible estimands. To…

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Using causal diagrams to assess parallel trends in difference-in-differences studies

利用因果图评估双重差分研究的平行趋势

Difference-in-differences (DID) is popular because it can allow for unmeasured confounding when the key assumption of parallel trends holds. However, there exists little guidance on how to decide a priori whether this assumption is reasonable. We attempt to develop such guidance by considering the relationship between a causal diagram and the parallel trends assumption. This is challenging because parallel trends is scale-dependent and causal diagrams are generally…

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Using negative controls to identify causal effects with invalid instrumental variables

利用负对照识别无效工具变量下的因果效应

Many proposals for the identification of causal effects require an instrumental variable that satisfies strong, untestable unconfoundedness and exclusion restriction assumptions. In this paper, we show how one can potentially identify causal effects under violations of these assumptions by harnessing a negative control population or outcome. This strategy allows one to leverage sub-populations for whom the exposure is degenerate, and requires that the instrument-outcome…

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On Doubly Robust Inference for Double Machine Learning in Semiparametric Regression

半参数回归中双重机器学习的双重稳健推断

Due to concerns about parametric model misspecification, there is interest in using machine learning to adjust for confounding when evaluating the causal effect of an exposure on an outcome. Unfortunately, exposure effect estimators that rely on machine learning predictions are generally subject to so-called plug-in bias, which can render naive $p$-values and confidence intervals invalid. Progress has been made via proposals like targeted minimum loss estimation and more…

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Doubly-robust inference and optimality in structure-agnostic models with smoothness

带平滑性约束的结构无关模型中的双重稳健推断与最优性

We study the problem of constructing an estimator of the average treatment effect (ATE) with observational data. The celebrated doubly-robust, augmented-IPW (AIPW) estimator generally requires consistent estimation of both nuisance functions for standard root-n inference, and moreover that the product of the errors of the nuisances should shrink at a rate faster than $n^{-1/2}$. A recent strand of research has aimed to understand the extent to which the AIPW estimator can be…

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Augmented balancing weights as linear regression

将增广平衡权重表示为线性回归

We provide a novel characterization of augmented balancing weights, also known as automatic debiased machine learning (AutoDML). These popular doubly robust or de-biased machine learning estimators combine outcome modeling with balancing weights - weights that achieve covariate balance directly in lieu of estimating and inverting the propensity score. When the outcome and weighting models are both linear in some (possibly infinite) basis, we show that the augmented estimator…

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Proximal mediation analysis

近端中介分析

A common concern when trying to draw causal inferences from observational data is that the measured covariates are insufficiently rich to account for all sources of confounding. In practice, many of the covariates may only be proxies of the latent confounding mechanism. Recent work has shown that in certain settings where the standard 'no unmeasured confounding' assumption fails, proxy variables can be leveraged to identify causal effects. Results currently exist for the…

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The Use of Covariate Adjustment in Randomized Controlled Trials: An Overview

随机对照试验中协变量调整的使用:综述

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of distinguishing between conditional and marginal treatment effects. Although these effects coincide in linear models, this is not typically the case in other settings, and this distinction is often overlooked in clinical trial practice. Considering…

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Alternative approaches for analysing repeated measures data that are missing not at random

非随机缺失重复测量数据的替代分析方法

We consider studies where multiple measures on an outcome variable are collected over time, but some subjects drop out before the end of follow up. Analyses of such data often proceed under either a 'last observation carried forward' or 'missing at random' assumption. We consider two alternative strategies for identification; the first is closely related to the difference-in-differences methodology in the causal inference literature. The second enables correction for…

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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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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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Translating questions to estimands in randomized clinical trials with intercurrent events

将伴发事件下的随机临床试验问题转化为目标估计量

Intercurrent (post-treatment) events occur frequently in randomized trials, and investigators often express interest in treatment effects that suitably take account of these events. A naive conditioning on intercurrent events does not have a straight-forward causal interpretation, and the practical relevance of other commonly used approaches is debated. In this work, we discuss how to formulate and choose an estimand, beyond the marginal intention to treat effect, from the…

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