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Causal Perspectives on Network Meta-Analysis

网络元分析的因果视角

Pairwise and network meta-analyses occupy the highest tier of evidence-based medicine and routinely inform clinical guidelines and healthcare decision-making. Current approaches typically aggregate study-level treatment effects to obtain an overall estimate. We argue that the causal estimand should come first, with the aggregation derived only afterwards: the target population and the relevant sources of between-study heterogeneity should be explicitly defined before…

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Federated Causal Inference from Multi-Site Observational Data via Propensity Score Aggregation

通过倾向得分聚合实现多站点观测数据的联邦因果推断

Causal inference typically assumes centralized access to individual-level data. Yet, in practice, data are often decentralized across multiple sites, making centralization infeasible due to privacy, logistical, or legal constraints. We address this problem by estimating the Average Treatment Effect (ATE) from decentralized observational data via a Federated Learning (FL) approach, allowing inference through the exchange of aggregate statistics rather than individual-level…

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Set-Valued Policy Learning

集合值政策学习

Conventional treatment policies map patient covariates to a single recommended intervention in order to maximize expected clinical outcomes. Although a rich body of causal inference methods has been developed to estimate such policies, point-valued recommendations can be highly sensitive to estimation uncertainty, model specification, and finite-sample variability, while typically providing little guidance about how confident one should be in the recommended action. In this…

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Causal Meta-Analysis: Rethinking the Foundations of Evidence-Based Medicine

因果元分析:重新审视循证医学的基础

Meta-analysis, by synthesizing effect estimates from multiple studies conducted in diverse settings, stands at the top of the evidence hierarchy in clinical research. Yet, conventional approaches based on fixed- or random-effects models lack a causal framework, which may limit their interpretability and utility for public policy. Incorporating causal inference reframes meta-analysis as the estimation of well-defined causal effects on clearly specified populations, enabling a…

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Rethinking the Win Ratio: A Causal Framework for Hierarchical Outcome Analysis

重新审视胜率比:层级结局分析的因果框架

Quantifying causal effects in the presence of complex and multivariate outcomes remains a key challenge in treatment evaluation. For hierarchical multivariate outcomes, the FDA recommends the Win Ratio and Generalized Pairwise Comparisons approaches \citep{Pocock2011winratio,Buyse2010}. However, commonly used estimators can yield treatment recommendations that target a population-level estimand (the probability that a randomly sampled patient under treatment fares better…

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Treatment Effect Estimation in Causal Survival Analysis: Practical Recommendations

因果生存分析中的处理效应估计:实践建议

The restricted mean survival time (RMST) difference offers an interpretable causal contrast to estimate the treatment effect for time-to-event outcomes, yet a wide range of available estimators leaves limited guidance for practice. We provide a unified review of RMST estimators for randomized trials and observational studies, establish identification and asymptotic properties, and supply new derivations where needed. Our extensive simulation study compares simple…

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Policy learning under constraint: Maximizing a primary outcome while controlling an adverse event

约束下的政策学习:最大化主要结果并控制不良事件

A medical policy aims to support decision-making by mapping patient characteristics to individualized treatment recommendations. Standard approaches typically optimize a single outcome criterion. For example, recommending treatment according to the sign of the Conditional Average Treatment Effect (CATE) maximizes the policy "value" by exploiting treatment effect heterogeneity. This point of view shifts policy learning towards the challenge of learning a reliable CATE…

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Risk ratio, odds ratio, risk difference... Which causal measure is easier to generalize?

风险比、优势比与风险差:哪种因果效应尺度更易推广?

There are many measures to report so-called treatment or causal effects: absolute difference, ratio, odds ratio, number needed to treat, and so on. The choice of a measure, e.g. absolute versus relative, is often debated because it leads to different impressions of the benefit or risk of a treatment. Besides, different causal measures may lead to various treatment effect heterogeneity: some input variables may have an influence on some causal measures and no effect at all on…

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Quantifying Treatment Effects: Estimating Risk Ratios in Causal Inference

量化处理效应:因果推断中的风险比估计

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is promising, as additional sources of evidence. In both RCT and observational data, the Risk Difference (RD) is often used to characterize the effect of a drug. Additionally, medical guidelines recommend to also report the Risk Ratio (RR),…

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A Unified Framework for the Transportability of Population-Level Causal Measures

总体层面因果效应度量可迁移性的统一框架

Generalization methods offer a powerful solution to one of the key drawbacks of randomized controlled trials (RCTs): their limited representativeness. By enabling the transport of treatment effect estimates to target populations subject to distributional shifts, these methods are increasingly recognized as the future of meta-analysis, the current gold standard in evidence-based medicine. Yet most existing approaches focus on the risk difference, overlooking the diverse range…

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Federated Causal Inference: Multi-Study ATE Estimation beyond Meta-Analysis

联邦因果推断:超越元分析的多研究平均处理效应估计

We study Federated Causal Inference, an approach to estimate treatment effects from decentralized data across centers. We compare three classes of Average Treatment Effect (ATE) estimators derived from the Plug-in G-Formula, ranging from simple meta-analysis to one-shot and multi-shot federated learning, the latter leveraging the full data to learn the outcome model (albeit requiring more communication). Focusing on Randomized Controlled Trials (RCTs), we derive the…

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Reweighting the RCT for generalization: finite sample error and variable selection

随机试验重加权推广:有限样本误差与变量选择

Randomized Controlled Trials (RCTs) may suffer from limited scope. In particular, samples may be unrepresentative: some RCTs over- or under- sample individuals with certain characteristics compared to the target population, for which one wants conclusions on treatment effectiveness. Re-weighting trial individuals to match the target population can improve the treatment effect estimation. In this work, we establish the exact expressions of the bias and variance of such…

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Positivity-free Policy Learning with Observational Data

无需正值性假设的观察数据政策学习

Policy learning utilizing observational data is pivotal across various domains, with the objective of learning the optimal treatment assignment policy while adhering to specific constraints such as fairness, budget, and simplicity. This study introduces a novel positivity-free (stochastic) policy learning framework designed to address the challenges posed by the impracticality of the positivity assumption in real-world scenarios. This framework leverages incremental…

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Generalizing treatment effects with incomplete covariates: identifying assumptions and multiple imputation algorithms

协变量不完整时的处理效应推广:识别假设与多重插补算法

We focus on the problem of generalizing a causal effect estimated on a randomized controlled trial (RCT) to a target population described by a set of covariates from observational data. Available methods such as inverse propensity sampling weighting are not designed to handle missing values, which are however common in both data sources. In addition to coupling the assumptions for causal effect identifiability and for the missing values mechanism and to defining appropriate…

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Causal effect on a target population: a sensitivity analysis to handle missing covariates

目标总体的因果效应:处理协变量缺失的敏感性分析

Randomized Controlled Trials (RCTs) are often considered the gold standard for estimating causal effect, but they may lack external validity when the population eligible to the RCT is substantially different from the target population. Having at hand a sample of the target population of interest allows us to generalize the causal effect. Identifying the treatment effect in the target population requires covariates to capture all treatment effect modifiers that are shifted…

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Doubly robust treatment effect estimation with missing attributes

属性缺失下的双重稳健处理效应估计

Missing attributes are ubiquitous in causal inference, as they are in most applied statistical work. In this paper, we consider various sets of assumptions under which causal inference is possible despite missing attributes and discuss corresponding approaches to average treatment effect estimation, including generalized propensity score methods and multiple imputation. Across an extensive simulation study, we show that no single method systematically out-performs others. We…

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denoiseR: A Package for Low Rank Matrix Estimation

denoiseR:低秩矩阵估计的 R 软件包

We introduce denoiseR, an R package that provides a unified implementation of several state-of-the-art proposals for regularized low rank matrix estimation, along with automatic selection of the regularization parameters. We also extend these methods to allow for missing values. The regularization schemes discussed in this paper are built around singular-value shrinkage and bootstrap-based stability arguments. We illustrate how to use out package by applying it to several…

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Bootstrap-Based Regularization for Low-Rank Matrix Estimation

低秩矩阵估计的自助法正则化

We develop a flexible framework for low-rank matrix estimation that allows us to transform noise models into regularization schemes via a simple bootstrap algorithm. Effectively, our procedure seeks an autoencoding basis for the observed matrix that is stable with respect to the specified noise model; we call the resulting procedure a stable autoencoder. In the simplest case, with an isotropic noise model, our method is equivalent to a classical singular value shrinkage…

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Confidence Areas for Fixed-Effects PCA

固定效应主成分分析的置信区域

PCA is often used to visualize data when the rows and the columns are both of interest. In such a setting there is a lack of inferential methods on the PCA output. We study the asymptotic variance of a fixed-effects model for PCA, and propose several approaches to assessing the variability of PCA estimates: a method based on a parametric bootstrap, a new cell-wise jackknife, as well as a computationally cheaper approximation to the jackknife. We visualize the confidence…

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