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Causal inference under transportability assumptions for conditional relative effect measures

条件相对效应可迁移假设下的因果推断

When extending inferences from a randomized trial to a new target population, the transportability condition for conditional difference effect measures is invoked to identify the marginal causal mean difference in the target population. However, many clinical investigators believe that conditional relative effect measures are more likely to be "transportable" between populations. Here, we examine the identification and estimation of the marginal counterfactual mean…

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Transportability of Principal Causal Effects

主层因果效应的可迁移性

Recent research in causal inference has made important progress in addressing challenges to the external validity of trial findings. Such methods weight trial participant data to more closely resemble the distribution of effect-modifying covariates in a well-defined target population. In the presence of participant non-adherence to study medication, these methods effectively transport an intention-to-treat effect that averages over heterogeneous compliance behaviors. In this…

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Using shrinkage methods to estimate treatment effects in overlapping subgroups in randomized clinical trials with a time-to-event endpoint

用收缩方法估计生存终点随机试验中重叠亚组的治疗效应

In randomized controlled trials, forest plots are frequently used to investigate the homogeneity of treatment effect estimates in subgroups. However, the interpretation of subgroup-specific treatment effect estimates requires great care due to the smaller sample size of subgroups and the large number of investigated subgroups. Bayesian shrinkage methods have been proposed to address these issues, but they often focus on disjoint subgroups while subgroups displayed in forest…

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Discovery and inference of possibly bi-directional causal relationships with invalid instrumental variables

利用可能无效的工具变量发现双向因果关系并进行推断

Learning causal relationships between pairs of complex traits from observational studies is of great interest across various scientific domains. However, most existing methods assume the absence of unmeasured confounding and restrict causal relationships between two traits to be uni-directional, which may be violated in real-world systems. In this paper, we address the challenge of causal discovery and effect inference for two traits while accounting for unmeasured…

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Restricted mean survival times for comparing grouped survival data: a Bayesian nonparametric approach

比较分组生存数据的限制平均生存时间:贝叶斯非参数方法

Comparing survival experiences of different groups of data is an important issue in several applied problems. A typical example is where one wishes to investigate treatment effects. Here we propose a new Bayesian approach based on restricted mean survival times (RMST). A nonparametric prior is specified for the underlying survival functions: this extends the standard univariate neutral to the right processes to a multivariate setting and induces a prior for the RMST's. We…

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Semiparametric Efficient Inference for the Probability of Necessary and Sufficient Causation

必要与充分因果概率的半参数有效推断

Causal attribution, which aims to explain why events or behaviors occur, is crucial in causal inference and enhances our understanding of cause-and-effect relationships in scientific research. The probabilities of necessary causation (PN) and sufficient causation (PS) are two of the most common quantities for attribution in causal inference. While many works have explored the identification or bounds of PN and PS, efficient estimation remains unaddressed. To fill this gap,…

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Optimal Kernel Choice for Score Function-based Causal Discovery

基于评分函数的因果发现中的最优核选择

Score-based methods have demonstrated their effectiveness in discovering causal relationships by scoring different causal structures based on their goodness of fit to the data. Recently, Huang et al. proposed a generalized score function that can handle general data distributions and causal relationships by modeling the relations in reproducing kernel Hilbert space (RKHS). The selection of an appropriate kernel within this score function is crucial for accurately…

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Identification of Average Causal Effects in Confounded Additive Noise Models

存在混杂的加性噪声模型中平均因果效应的识别

Additive noise models (ANMs) are an important setting studied in causal inference. Most of the existing works on ANMs assume causal sufficiency, i.e., there are no unobserved confounders. This paper focuses on confounded ANMs, where a set of treatment variables and a target variable are affected by an unobserved confounder that follows a multivariate Gaussian distribution. We introduce a novel approach for estimating the average causal effects (ACEs) of any subset of the…

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Counterfactual Generative Models for Time-Varying Treatments

时变处理的反事实生成模型

Estimating the counterfactual outcome of treatment is essential for decision-making in public health and clinical science, among others. Often, treatments are administered in a sequential, time-varying manner, leading to an exponentially increased number of possible counterfactual outcomes. Furthermore, in modern applications, the outcomes are high-dimensional and conventional average treatment effect estimation fails to capture disparities in individuals. To tackle these…

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Meta-Analysis with Untrusted Data

结合不可信数据的元分析

[See paper for full abstract] Meta-analysis is a crucial tool for answering scientific questions. It is usually conducted on a relatively small amount of ``trusted'' data -- ideally from randomized, controlled trials -- which allow causal effects to be reliably estimated with minimal assumptions. We show how to answer causal questions much more precisely by making two changes. First, we incorporate untrusted data drawn from large observational databases, related scientific…

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Identification and Estimation of the Bi-Directional MR with Some Invalid Instruments

存在部分无效工具变量时双向孟德尔随机化的识别与估计

We consider the challenging problem of estimating causal effects from purely observational data in the bi-directional Mendelian randomization (MR), where some invalid instruments, as well as unmeasured confounding, usually exist. To address this problem, most existing methods attempt to find proper valid instrumental variables (IVs) for the target causal effect by expert knowledge or by assuming that the causal model is a one-directional MR model. As such, in this paper, we…

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Inference procedures in sequential trial emulation with survival outcomes: comparing confidence intervals based on the sandwich variance estimator, bootstrap and jackknife

生存结局序贯试验模拟的推断:夹心方差、Bootstrap 与 Jackknife 比较

Sequential trial emulation (STE) is an approach to estimating causal treatment effects by emulating a sequence of target trials from observational data. In STE, inverse probability weighting is commonly utilised to address time-varying confounding and/or dependent censoring. Then structural models for potential outcomes are applied to the weighted data to estimate treatment effects. For inference, the simple sandwich variance estimator is popular but conservative, while…

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Causal exposure-response curve estimation with surrogate confounders: a study of air pollution and children's health in Medicaid claims data

利用混杂因素替代变量估计因果暴露反应曲线:空气污染与儿童健康

In this paper, we undertake a case study to estimate a causal exposure-response function (ERF) for long-term exposure to fine particulate matter (PM$_{2.5}$) and respiratory hospitalizations in socioeconomically disadvantaged children using nationwide Medicaid claims data. These data present specific challenges. First, family income-based Medicaid eligibility criteria for children differ by state, creating socioeconomically distinct populations and leading to clustered data.…

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Maximum Entropy Estimation of Heterogeneous Causal Effects

异质性因果效应的最大熵估计

For the purpose of causal inference we employ a stochastic model of the data generating process, utilizing individual propensity probabilities for the treatment, and also individual and counterfactual prognosis probabilities for the outcome. We assume a generalized version of the stable unit treatment value assumption, but we do not assume any version of strongly ignorable treatment assignment. Instead of conducting a sensitivity analysis, we utilize the principle of maximum…

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An Introduction to Causal Discovery

因果发现导论

In social sciences and economics, causal inference traditionally focuses on assessing the impact of predefined treatments (or interventions) on predefined outcomes, such as the effect of education programs on earnings. Causal discovery, in contrast, aims to uncover causal relationships among multiple variables in a data-driven manner, by investigating statistical associations rather than relying on predefined causal structures. This approach, more common in computer science,…

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Causal inference through multi-stage learning and doubly robust deep neural networks

多阶段学习与双重稳健深度神经网络的因果推断

Deep neural networks (DNNs) have demonstrated remarkable empirical performance in large-scale supervised learning problems, particularly in scenarios where both the sample size $n$ and the dimension of covariates $p$ are large. This study delves into the application of DNNs across a wide spectrum of intricate causal inference tasks, where direct estimation falls short and necessitates multi-stage learning. Examples include estimating the conditional average treatment effect…

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Adjustment Identification Distance: A gadjid for Causal Structure Learning

调整识别距离:用于因果结构学习的gadjid工具

Evaluating graphs learned by causal discovery algorithms is difficult: The number of edges that differ between two graphs does not reflect how the graphs differ with respect to the identifying formulas they suggest for causal effects. We introduce a framework for developing causal distances between graphs which includes the structural intervention distance for directed acyclic graphs as a special case. We use this framework to develop improved adjustment-based distances as…

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Robust CATE Estimation Using Novel Ensemble Methods

使用新型集成方法稳健估计条件平均处理效应

The estimation of Conditional Average Treatment Effects (CATE) is crucial for understanding the heterogeneity of treatment effects in clinical trials. We evaluate the performance of common methods, including causal forests and various meta-learners, across a diverse set of scenarios, revealing that each of the methods struggles in one or more of the tested scenarios. Given the inherent uncertainty of the data-generating process in real-life scenarios, the robustness of a…

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Adaptive Experiments Toward Learning Treatment Effect Heterogeneity

面向处理效应异质性学习的自适应实验

Understanding treatment effect heterogeneity has become an increasingly popular task in various fields, as it helps design personalized advertisements in e-commerce or targeted treatment in biomedical studies. However, most of the existing work in this research area focused on either analyzing observational data based on strong causal assumptions or conducting post hoc analyses of randomized controlled trial data, and there has been limited effort dedicated to the design of…

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Two Stage Least Squares with Time-Varying Instruments: An Application to an Evaluation of Treatment Intensification for Type-2 Diabetes

使用时变工具变量的两阶段最小二乘:二型糖尿病治疗强化评估

As longitudinal data becomes more available in many settings, policy makers are increasingly interested in the effect of time-varying treatments (e.g. sustained treatment strategies). In settings such as this, the preferred analysis techniques are the g-methods, however these require the untestable assumption of no unmeasured confounding. Instrumental variable analyses can minimise bias through unmeasured confounding. Of these methods, the Two Stage Least Squares technique…

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Aggregate Bayesian Causal Forests: The ABCs of Flexible Causal Inference for Hierarchically Structured Data

聚合贝叶斯因果森林:分层数据的灵活因果推断

This paper introduces aggregate Bayesian Causal Forests (aBCF), a new Bayesian model for causal inference using aggregated data. Aggregated data are common in policy evaluations where we observe individuals such as students, but participation in an intervention is determined at a higher level of aggregation, such as schools implementing a curriculum. Interventions often have millions of individuals but far fewer higher-level units, making aggregation computationally…

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Increased risk of type I errors for detecting heterogeneity of treatment effects in cluster-randomized trials using mixed-effect models

集群随机试验中用混合效应模型检验处理效应异质性的第一类错误风险

Evaluating heterogeneity of treatment effects (HTE) across subgroups is common in both randomized trials and observational studies. Although several statistical challenges of HTE analyses including low statistical power and multiple comparisons are widely acknowledged, issues arising for clustered data, including cluster randomized trials (CRTs), have received less attention. Notably, the potential for model misspecification is increased given the complex clustering…

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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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How to Add Baskets to an Ongoing Basket Trial with Information Borrowing

如何在信息借用下向进行中的篮式试验加入新篮

Basket trials test a single therapeutic treatment on several patient populations under one master protocol. A desirable adaptive design feature in these studies may be the incorporation of new baskets to an ongoing study. Limited basket sample sizes can cause issues in power and precision of treatment effect estimates which could be amplified in added baskets due to the shortened recruitment time. While various Bayesian information borrowing techniques have been introduced…

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