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Separable effects for adherence

治疗依从性的可分离效应

Comparing different medications is complicated when adherence to these medications differs. We can overcome the adherence issue by assessing effectiveness under sustained use, as in the usual causal `per-protocol' estimand. However, when sustained use is challenging to satisfy in practice, the usefulness of this estimand can be limited. Here we propose a different class of estimands: separable effects for adherence. These estimands compare modified medications, holding fixed…

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OpportunityFinder: A Framework for Automated Causal Inference

OpportunityFinder:自动化因果推断框架

We introduce OpportunityFinder, a code-less framework for performing a variety of causal inference studies with panel data for non-expert users. In its current state, OpportunityFinder only requires users to provide raw observational data and a configuration file. A pipeline is then triggered that inspects/processes data, chooses the suitable algorithm(s) to execute the causal study. It returns the causal impact of the treatment on the configured outcome, together with…

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Mediation analysis with the mediator and outcome missing not at random

中介与结果非随机缺失时的中介分析

Mediation analysis is widely used for investigating direct and indirect causal pathways through which an effect arises. However, many mediation analysis studies are challenged by missingness in the mediator and outcome. In general, when the mediator and outcome are missing not at random, the direct and indirect effects are not identifiable without further assumptions. In this work, we study the identifiability of the direct and indirect effects under some interpretable…

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Estimating hypothetical estimands with causal inference and missing data estimators in a diabetes trial

糖尿病试验中假设性目标量的因果推断与缺失数据估计

The recently published ICH E9 addendum on estimands in clinical trials provides a framework for precisely defining the treatment effect that is to be estimated, but says little about estimation methods. Here we report analyses of a clinical trial in type 2 diabetes, targeting the effects of randomised treatment, handling rescue treatment and discontinuation of randomised treatment using the so-called hypothetical strategy. We show how this can be estimated using mixed models…

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Multiply Robust Federated Estimation of Targeted Average Treatment Effects

目标总体平均处理效应的多重稳健联邦估计

Federated or multi-site studies have distinct advantages over single-site studies, including increased generalizability, the ability to study underrepresented populations, and the opportunity to study rare exposures and outcomes. However, these studies are challenging due to the need to preserve the privacy of each individual's data and the heterogeneity in their covariate distributions. We propose a novel federated approach to derive valid causal inferences for a target…

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Effects of Adolescent Victimization on Offending: Flexible Methods for Missing Data & Unmeasured Confounding

青少年受害经历对违法行为的影响:缺失数据与未测混杂的灵活方法

The causal link between victimization and violence later in life is largely accepted but has been understudied for victimized adolescents. In this work we use the Add Health dataset, the largest nationally representative longitudinal survey of adolescents, to estimate the relationship between victimization and future offending in this population. To accomplish this, we derive a new doubly robust estimator for the average treatment effect on the treated (ATT) when the…

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Testing a Large Number of Composite Null Hypotheses Using Conditionally Symmetric Multidimensional Gaussian Mixtures in Genome-Wide Studies

全基因组研究中基于条件对称多维高斯混合的大规模复合零假设检验

Causal mediation analysis, pleiotropy analysis, and replication analysis are three highly popular genetic study designs. Although these analyses address different scientific questions, the underlying inference problems all involve large-scale testing of composite null hypotheses. The goal is to determine whether all null hypotheses - as opposed to at least one - in a set of individual tests should simultaneously be rejected. Various recent methodology has been proposed for…

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Survival causal rule ensemble method considering the main effect for estimating heterogeneous treatment effects

考虑主效应的生存因果规则集成方法:异质性处理效应估计

With an increasing focus on precision medicine in medical research, numerous studies have been conducted in recent years to clarify the relationship between treatment effects and patient characteristics. The treatment effects for patients with different characteristics are always heterogeneous, and various heterogeneous treatment effect machine learning estimation methods have been proposed owing to their flexibility and high prediction accuracy. However, most machine…

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Causal inference with outcome dependent sampling and mismeasured outcome

结果依赖抽样与结果测量误差下的因果推断

Outcome-dependent sampling designs are extensively utilized in various scientific disciplines, including epidemiology, ecology, and economics, with retrospective case-control studies being specific examples of such designs. Additionally, if the outcome used for sample selection is also mismeasured, then it is even more challenging to estimate the average treatment effect (ATE) accurately. To our knowledge, no existing method can address these two issues simultaneously. In…

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More Robust Estimators for Instrumental-Variable Panel Designs, With An Application to the Effect of Imports from China on US Employment

工具变量面板设计的更稳健估计:以中国进口对美国就业的影响为例

We show that first-difference two-stages-least-squares regressions identify non-convex combinations of location-and-period-specific treatment effects. Thus, those regressions could be biased if effects are heterogeneous. We propose an alternative instrumental-variable correlated-random-coefficient (IV-CRC) estimator, that is more robust to heterogeneous effects. We revisit Autor et al. (2013), who use a first-difference two-stages-least-squares regression to estimate the…

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Optimal subgroup selection

最优子群选择

In clinical trials and other applications, we often see regions of the feature space that appear to exhibit interesting behaviour, but it is unclear whether these observed phenomena are reflected at the population level. Focusing on a regression setting, we consider the subgroup selection challenge of identifying a region of the feature space on which the regression function exceeds a pre-determined threshold. We formulate the problem as one of constrained optimisation,…

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Incorporating Participants' Welfare into Sequential Multiple Assignment Randomized Trials

将参与者福利纳入序贯多重分配随机试验

Dynamic treatment regimes (DTRs) are sequences of decision rules that recommend treatments based on patients' time-varying clinical conditions. The sequential multiple assignment randomized trial (SMART) is an experimental design that can provide high-quality evidence for constructing optimal DTRs. In a conventional SMART, participants are randomized to available treatments at multiple stages with balanced randomization probabilities. Despite its relative simplicity of…

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Causal Selection of Covariates in Regression Calibration for Mismeasured Continuous Exposure

连续暴露测量误差下回归校准的因果协变量选择

Regression calibration as developed by Rosner, Spiegelman and Willet is used to correct the bias in effect estimates due to measurement error in continuous exposures. The method involves two models: a measurement error model (MEM) relating the mismeasured exposure to the true exposure and an outcome model relating the mismeasured exposure to outcome. However, no comprehensive guidance exists for determining which covariates should be included in each model. In this paper, we…

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Causal Discovery and Counterfactual Explanations for Personalized Student Learning

面向个性化学生学习的因果发现与反事实解释

The paper focuses on identifying the causes of student performance to provide personalized recommendations for improving pass rates. We introduce the need to move beyond predictive models and instead identify causal relationships. We propose using causal discovery techniques to achieve this. The study's main contributions include using causal discovery to identify causal predictors of student performance and applying counterfactual analysis to provide personalized…

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Discovery and inference of a causal network with hidden confounding

存在隐藏混杂时的因果网络发现与推断

This article proposes a novel causal discovery and inference method called GrIVET for a Gaussian directed acyclic graph with unmeasured confounders. GrIVET consists of an order-based causal discovery method and a likelihood-based inferential procedure. For causal discovery, we generalize the existing peeling algorithm to estimate the ancestral relations and candidate instruments in the presence of hidden confounders. Based on this, we propose a new procedure for instrumental…

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Nonlinear Permuted Granger Causality

非线性置换 Granger 因果分析

Granger causal inference is a contentious but widespread method used in fields ranging from economics to neuroscience. The original definition addresses the notion of causality in time series by establishing functional dependence conditional on a specified model. Adaptation of Granger causality to nonlinear data remains challenging, and many methods apply in-sample tests that do not incorporate out-of-sample predictability, leading to concerns of model overfitting. To allow…

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Testing Instrument Validity with Covariates

包含协变量时的工具变量有效性检验

We develop a novel test of the instrumental variable identifying assumptions for heterogeneous treatment effect models with conditioning covariates. We assume semiparametric dependence between potential outcomes and conditioning covariates. This allows us to obtain testable equality and inequality restrictions among the subdensities of estimable partial residuals. We propose jointly testing these restrictions. To improve power, we introduce distillation, where a trimmed…

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Generalizing Trimming Bounds for Endogenously Missing Outcome Data Using Random Forests

利用随机森林推广内生缺失结果数据的修剪界限

In many experimental or quasi-experimental studies, outcomes of interest are only observed for subjects who select (or are selected) to engage in the activity generating the outcome. Outcome data is thus endogenously missing for units who do not engage, in which case random or conditionally random treatment assignment prior to such choices is insufficient to point identify treatment effects. Non-parametric partial identification bounds are a way to address endogenous…

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Bandwidth Selection for Treatment Choice with Binary Outcomes

二元结果处理选择中的带宽选择

This study considers the treatment choice problem when outcome variables are binary. We focus on statistical treatment rules that plug in fitted values based on nonparametric kernel regression and show that optimizing two parameters enables the calculation of the maximum regret. Using this result, we propose a novel bandwidth selection method based on the minimax regret criterion. Finally, we perform a numerical analysis to compare the optimal bandwidth choices for the…

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Multiply robust estimation of causal effects using linked data

利用关联数据进行因果效应的多重稳健估计

Unmeasured confounding presents a common challenge in observational studies, potentially making standard causal parameters unidentifiable without additional assumptions. Given the increasing availability of diverse data sources, exploiting data linkage offers a potential solution to mitigate unmeasured confounding within a primary study of interest. However, this approach often introduces selection bias, as data linkage is feasible only for a subset of the study population.…

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To Predict or to Reject: Causal Effect Estimation with Uncertainty on Networked Data

预测还是拒绝:网络数据中具有不确定性评估的因果效应估计

Due to the imbalanced nature of networked observational data, the causal effect predictions for some individuals can severely violate the positivity/overlap assumption, rendering unreliable estimations. Nevertheless, this potential risk of individual-level treatment effect estimation on networked data has been largely under-explored. To create a more trustworthy causal effect estimator, we propose the uncertainty-aware graph deep kernel learning (GraphDKL) framework with…

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Sharp hypotheses and organic fiducial inference

尖锐假设与有机置信推断

A fundamental class of inferential problems are those characterised by there having been a substantial degree of pre-data (or prior) belief that the value of a model parameter $θ_j$ was equal or lay close to a specified value $θ^{*}_j$, which may, for example, be the value that indicates the absence of a treatment effect or the lack of correlation between two variables. This paper puts forward a generally applicable 'push-button' solution to problems of this type that…

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Anytime-valid inference in N-of-1 trials

单人试验中任意时点有效的统计推断

App-based N-of-1 trials offer a scalable experimental design for assessing the effects of health interventions at an individual level. Their practical success depends on the strong motivation of participants, which, in turn, translates into high adherence and reduced loss to follow-up. One way to maintain participant engagement is by sharing their interim results. Continuously testing hypotheses during a trial, known as "peeking", can also lead to shorter, lower-risk trials…

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Real Effect or Bias? Best Practices for Evaluating the Robustness of Real-World Evidence through Quantitative Sensitivity Analysis for Unmeasured Confounding

真实效应还是偏差?用未测混杂定量敏感性分析评估真实世界证据

The assumption of no unmeasured confounders is a critical but unverifiable assumption required for causal inference yet quantitative sensitivity analyses to assess robustness of real-world evidence remains underutilized. The lack of use is likely in part due to complexity of implementation and often specific and restrictive data requirements required for application of each method. With the advent of sensitivity analyses methods that are broadly applicable in that they do…

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