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Longitudinal causal inference under informative missingness using the self-censoring model

自删失模型下信息性缺失的纵向因果推断

Longitudinal observational data are often subject to informative missingness. For example, in healthcare research using electronic health records, covariates and outcomes are measured only when patients interact with the healthcare system. Whether these data are observed usually depends on the unobserved variable values themselves, introducing a common form of data missing not at random. We develop identification and estimation methods for the effects of longitudinal…

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Efficient transport and generalization of survival treatment effects

生存治疗效应的有效迁移与推广

Randomized controlled trials provide internally valid estimates of treatment effects, but their results may not directly apply to broader target populations due to differences in baseline covariate distributions, adherence to treatment or variations in outcome mechanisms. Under standard transport and time-to-event identifiability assumptions, we develop nonparametric, debiased machine learning estimators for transporting and generalizing causal survival treatment effect…

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Automated, efficient and model-free inference for randomized clinical trials via data-driven covariate adjustment

通过数据驱动协变量调整实现随机临床试验的自动化高效无模型推断

In 2023, the U.S. Food and Drug Administration issued guidance for adjustment of covariates in randomized clinical trials, emphasizing its role in enhancing precision and power through prognostic baseline variables. Despite its potential, many trials underutilize this method partly due to challenges in pre-specifying optimal baseline covariates and their functional forms. We explore the potential of automated, data-adaptive methods-including stepwise regression, Lasso and…

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Modified treatment policies that depend on the natural history of treatment

依赖处理自然历史的修正处理政策

Longitudinal modified treatment policies (LMTP) are a class of interventions that allow the definition, identification, and estimation of causal effects in general settings, such as with continuous or multivariate exposures, treatment regimens that require grace periods. Targeted machine learning estimators (i.e., double/debiased) have been formulated for LMTPs that assign the exposure at time $t$ as a function of the natural value of treatment at time $t$. However,…

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Non-overlap Average Treatment Effect Bounds

无重叠情况下的平均处理效应界

The average treatment effect (ATE), the mean difference in potential outcomes under treatment and control, is a canonical causal effect. Overlap, which says that all subjects have non-zero probability of either treatment status, is necessary to identify and estimate the ATE. When overlap fails, the standard solution is to change the estimand, and target a trimmed effect in a subpopulation satisfying overlap. When the outcome is bounded, we demonstrate that this compromise is…

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Orthogonal machine learning for conditional odds and risk ratios

条件优势比与风险比的正交机器学习

Conditional effects are commonly used measures for understanding how treatment effects vary across different groups, and are often used to target treatments/interventions to groups who benefit most. In this work we review existing methods and propose novel ones, focusing on the odds ratio (OR) and the risk ratio (RR). While estimation of the conditional average treatment effect (ATE) has been widely studied, estimators for the OR and RR lag behind, and cutting edge…

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Comparing causal parameters with many treatments and positivity violations

多处理与正值性违背下的因果参数比较

Comparing outcomes across treatments is essential in medicine and public policy. To do so, researchers typically estimate a set of parameters, possibly counterfactual, with each targeting a different treatment. Treatment-specific means are commonly used, but their identification requires a positivity assumption, that every subject has a non-zero probability of receiving each treatment. This is often implausible, especially when treatment can take many values. Causal…

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Computationally and statistically efficient estimation of time-smoothed counterfactual curves

时间平滑反事实曲线的计算与统计高效估计

Longitudinal causal inference is concerned with defining, identifying, and estimating the effect of a time-varying intervention on a time-varying outcome that is indexed by a follow-up time. In an observational study, Robins's generalized g-formula can identify causal effects induced by a broad class of time-varying interventions. Various methods for estimating the generalized g-formula have been posed for different outcome types, such as a failure event indicator by a…

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Identification and estimation of mediational effects of longitudinal modified treatment policies

纵向修正处理政策的中介效应识别与估计

We demonstrate a comprehensive semiparametric approach to causal mediation analysis, addressing the complexities inherent in settings with longitudinal and continuous treatments, confounders, and mediators. Our methodology utilizes a nonparametric structural equation model and a cross-fitted sequential regression technique based on doubly robust pseudo-outcomes, yielding an efficient, asymptotically normal estimator without relying on restrictive parametric modeling…

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Propensity score weighting across counterfactual worlds: longitudinal effects under positivity violations

跨反事实世界的倾向得分加权:正值性违背下的纵向效应

When examining a contrast between two interventions, longitudinal causal inference studies frequently encounter positivity violations when one or both regimes are impossible to observe for some subjects. Existing weighting methods either assume positivity holds or produce effects that conflate interventions' impacts on ultimate outcomes with their effects on intermediate treatments and covariates. We propose a novel class of estimands -- cumulative cross-world weighted…

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Nonparametric estimation of an optimal treatment rule with fused randomized trials and missing effect modifiers

融合随机试验且效应修饰变量缺失时最优治疗规则的非参数估计

A fundamental principle of clinical medicine is that a treatment should only be administered to those patients who would benefit from it. Treatment strategies that assign treatment to patients as a function of their individual characteristics are known as dynamic treatment rules. The dynamic treatment rule that optimizes the outcome in the population is called the optimal dynamic treatment rule. Randomized clinical trials are considered the gold standard for estimating the…

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Asymptotically Efficient Data-adaptive Penalized Shrinkage Estimation with Application to Causal Inference

渐近有效的数据自适应惩罚收缩估计及其因果推断应用

A rich literature exists on constructing non-parametric estimators with optimal asymptotic properties. In addition to asymptotic guarantees, it is often of interest to design estimators with desirable finite-sample properties; such as reduced mean-squared error of a large set of parameters. We provide examples drawn from causal inference where this may be the case, such as estimating a large number of group-specific treatment effects. We show how finite-sample properties of…

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Pulling back the curtain: the road from statistical estimand to machine-learning based estimator for epidemiologists (no wizard required)

从统计目标量到机器学习估计量:面向流行病学研究者的推导指南

Epidemiologists increasingly use causal inference methods that rely on machine learning, as these approaches can relax unnecessary model specification assumptions. While deriving and studying asymptotic properties of such estimators is a task usually associated with statisticians, it is useful for epidemiologists to understand the steps involved, as epidemiologists are often at the forefront of defining important new research questions and translating them into new…

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Causal survival analysis under competing risks using longitudinal modified treatment policies

利用纵向修正治疗策略开展竞争风险下的因果生存分析

Longitudinal modified treatment policies (LMTP) have been recently developed as a novel method to define and estimate causal parameters that depend on the natural value of treatment. LMTPs represent an important advancement in causal inference for longitudinal studies as they allow the non-parametric definition and estimation of the joint effect of multiple categorical, numerical, or continuous exposures measured at several time points. We extend the LMTP methodology to…

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Longitudinal Generalizations of the Average Treatment Effect on the Treated for Multi-valued and Continuous Treatments

多值与连续处理下已处理者平均处理效应的纵向推广

The Average Treatment Effect on the Treated (ATT) is a common causal parameter defined as the average effect of a binary treatment among the subset of the population receiving treatment. We propose a novel family of parameters, Generalized ATTs (GATTs), that generalize the concept of the ATT to longitudinal data structures, multi-valued or continuous treatments, and conditioning on arbitrary treatment subsets. We provide a formal causal identification result that expresses…

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Causal Inference for Continuous Multiple Time Point Interventions

连续多时点干预的因果推断

There are limited options to estimate the treatment effects of variables which are continuous and measured at multiple time points, particularly if the true dose-response curve should be estimated as closely as possible. However, these situations may be of relevance: in pharmacology, one may be interested in how outcomes of people living with -- and treated for -- HIV, such as viral failure, would vary for time-varying interventions such as different drug concentration…

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Non-parametric efficient estimation of marginal structural models with multi-valued time-varying treatments

多值时变处理下边际结构模型的非参数有效估计

Marginal structural models are a popular method for estimating causal effects in the presence of time-varying exposures. In spite of their popularity, no scalable non-parametric estimator exist for marginal structural models with multi-valued and time-varying treatments. In this paper, we use machine learning together with recent developments in semiparametric efficiency theory for longitudinal studies to propose such an estimator. The proposed estimator is based on a study…

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A Surrogate Endpoint Based Provisional Approval Causal Roadmap

基于替代终点的临时批准因果路线图

For many rare diseases with no approved preventive interventions, promising interventions exist, yet it has been difficult to conduct a pivotal phase 3 trial that could provide direct evidence demonstrating a beneficial effect on the target disease outcome. When a promising putative surrogate endpoint(s) for the target outcome is available, surrogate-based provisional approval of an intervention may be pursued. We apply the Causal Roadmap rubric to define a surrogate…

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Efficient estimation of longitudinal treatment effects using difference-in-differences and machine learning

利用双重差分与机器学习高效估计纵向处理效应

Difference-in-differences is based on a parallel trends assumption, which states that changes over time in average potential outcomes are independent of treatment assignment, possibly conditional on covariates. With time-varying treatments, parallel trends assumptions can identify many types of parameters, but most work has focused on group-time average treatment effects and similar parameters conditional on the treatment trajectory. This paper focuses instead on…

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Two-Step Targeted Minimum-Loss Based Estimation for Non-Negative Two-Part Outcomes

非负两部分结局的两步目标最小损失估计

Non-negative two-part outcomes are defined as outcomes with a density function that have a zero point mass but are otherwise positive. Examples, such as healthcare expenditure and hospital length of stay, are common in healthcare utilization research. Despite the practical relevance of non-negative two-part outcomes, very few methods exist to leverage knowledge of their semicontinuity to achieve improved performance in estimating causal effects. In this paper, we develop a…

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Non-agency interventions for causal mediation in the presence of intermediate confounding

存在中间混杂时用于因果中介分析的非行动主体干预

Recent approaches to causal inference have focused on causal effects defined as contrasts between the distribution of counterfactual outcomes under hypothetical interventions on the nodes of a graphical model. In this article we develop theory for causal effects defined with respect to a different type of intervention, one which alters the information propagated through the edges of the graph. These information transfer interventions may be more useful than node…

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Efficient estimation of modified treatment policy effects based on the generalized propensity score

基于广义倾向得分的修正处理政策效应有效估计

Continuous treatments have posed a significant challenge for causal inference, both in the formulation and identification of scientifically meaningful effects and in their robust estimation. Traditionally, focus has been placed on techniques applicable to binary or categorical treatments with few levels, allowing for the application of propensity score-based methodology with relative ease. Efforts to accommodate continuous treatments introduced the generalized propensity…

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Efficient and flexible causal mediation with time-varying mediators, treatments, and confounders

时变中介、处理与混杂下的高效灵活因果中介分析

Interventional effects have been proposed as a solution to the unidentifiability of natural (in)direct effects under mediator-outcome confounders affected by the exposure. Such confounders are an intrinsic characteristic of studies with time-varying exposures and mediators, yet the generalization of the interventional effect framework to the time-varying case has received little attention in the literature. We present an identification result for interventional effects in a…

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