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诊断测试与人工智能医疗器械的因果评估：效应修饰与信息增强决策AI 解读

Diagnostic medical tests and devices provide useful information for evaluating the potential benefits and risks of therapeutic treatments. However, unlike treatments, their impact on health outcomes is generally indirect because measuring diagnostic information typically does not itself affect patient outcomes, which complicates evaluation of their effectiveness. In this work, we develop a causal inference approach for evaluating diagnostic tests by distinguishing…

监管试验协变量调整的良好实践：从固定到数据自适应方法AI 解读

While randomization justifies the use of unadjusted effect estimators in randomized trials, there is growing interest in covariate adjustment to improve precision. Adjusting for baseline variables that are prognostic of the outcome can reduce estimator variance, resulting in narrower confidence intervals and increased statistical power. Recent guidance by the U.S. Food and Drug Administration supports fixed adjustment for prognostic covariates using parametric regression…

半参数推断的 V 折刀切法：方差、置信区间与同时置信带AI 解读

For decades, the bootstrap has been a default tool for statistical inference because of its broad applicability and minimal analytic requirements. Although its validity is well understood for smooth parametric estimators, its theoretical properties for many modern semiparametric and machine-learning estimators remain largely unstudied. Nevertheless, bootstrap procedures are often used routinely in such settings, even when their validity is unknown and their computational…

以外部真实世界数据扩充随机试验的估计量稳健设计AI 解读

Augmenting randomized controlled trials (RCTs) with external real-world data (RWD) has the potential to improve the finite sample efficiency of treatment effect estimators. We describe using adaptive targeted maximum likelihood estimation (A-TMLE) for estimating the average treatment effect (ATE) by decomposing the ATE estimand into two components: a pooled-ATE estimand that combines data from both the RCT and external sources, and a bias estimand that captures the…

利用 TMLE 提高随机对照试验子组分析的效率AI 解读

Subgroup analyses within randomized controlled trials are often underpowered due to limited sample sizes. We address this challenge by leveraging trial participants outside the subgroup of interest to augment estimation within the subgroup. Specifically, we study two Targeted Maximum Likelihood Estimators (TMLEs) that borrow information from non-subgroup participants within the same trial: a TMLE with pooled regression (TMLE-PR) and an Adaptive Targeted Maximum Likelihood…

利用 CV-TMLE 全局检验提高罕见病多组分终点研究的功效AI 解读

Rare disease trials face unique statistical challenges due to limited patient populations and heterogeneous clinical manifestations among patients. Multiple endpoints are often necessary to comprehensively capture treatment benefits. A global test is an approach for evaluating whether a treatment has any beneficial effect across multiple endpoints. We propose a new global test based on a weighted composite endpoint. The proposed global test employs shrinkage-based…

结合目标学习推断的在线元层级自适应设计：代理结局的评价与利用AI 解读

Adaptive designs are increasingly used in clinical trials and online experiments to improve participant outcomes by dynamically updating treatment allocation as data accumulate. In practice, experimenters often consider multiple candidate designs, each with distinct trade-offs. However, typically only one design is implemented at a time, leaving benefits and costs of alternative designs unobserved and unquantified. To address this, we propose a novel meta-level adaptive…

结合数据驱动模型选择的自适应去偏机器学习AI 解读

Debiased machine learning estimators for smooth functionals in nonparametric models can exhibit substantial variability and instability, often leading practitioners to instead rely on parametric or semiparametric working models. Such models, however, may be misspecified and can therefore introduce bias. We study how data-driven model selection can be combined with debiased machine learning to construct estimators that adapt to structure in the data-generating distribution.…

异质性处理效应变量重要性的目标学习AI 解读

Quantifying the heterogeneity of treatment effect is important for understanding how a commercial product or medical treatment affects different population subgroups. While much of treatment effect heterogeneity analysis focuses on the conditional average treatment effect, an alternative parameter that captures treatment effect heterogeneity is the variance of treatment effect across different covariate groups. One can also derive variable importance parameters that measure…

事件时间过程中校准缩放干预的因果推断AI 解读

This work develops a flexible inferential framework for nonparametric causal inference in time-to-event settings, based on stochastic interventions defined through multiplicative scaling of the intensity governing an intermediate event process. These interventions induce a family of estimands indexed by a scalar parameter α, representing effects of modifying event rates while preserving the temporal and covariate-dependent structure of the observed data generating mechanism.…

用于随机试验与外部数据混合研究的实验选择交叉验证 TMLEAI 解读

Augmenting a randomized controlled trial (RCT) with external data may increase power at the risk of introducing bias. To select and analyze the experiment (RCT alone or combined with external data) with the optimal bias-variance tradeoff, we develop a novel experiment-selector cross-validated targeted maximum likelihood estimator for randomized-external data studies (ES-CVTMLE). This estimator utilizes two estimates of bias to determine whether to integrate external data…

利用高度自适应 Lasso 构造无限维函数参数的置信区间AI 解读

Estimating the conditional mean function is a central task in statistical learning. In this paper, we consider estimation and inference for a nonparametric class of real-valued cadlag functions with bounded sectional variation (Gill et al., 1995), using the Highly Adaptive Lasso (HAL) (van der Laan, 2015; Benkeser and van der Laan, 2016; van der Laan, 2023), a flexible empirical risk minimizer over linear combinations of tensor products of zero- or higher-order spline basis…

基于 HAL 的因果剂量反应曲线插件估计与逐点渐近正态性AI 解读

Estimating and obtaining reliable inference for the marginally adjusted causal dose-response curve for continuous treatments without relying on parametric assumptions is a well-known statistical challenge. Parametric models risk introducing significant bias through model misspecification, compromising the accurate representation of the underlying data and dose-response relationship. On the other hand, nonparametric models face difficulties as the dose-response curve is not…

序贯自适应实验的有效统计估计及其对自适应设计的启示AI 解读

Adaptive experimental designs have gained popularity in clinical trials and online experiments. Unlike traditional, fixed experimental designs, adaptive designs can dynamically adjust treatment randomization probabilities and other design features in response to data accumulated sequentially during the experiment. These adaptations are useful to achieve diverse objectives, including reducing uncertainty in the estimation of causal estimands or increasing participants'…

以目标学习估计抽样方差以改进推断AI 解读

For robust statistical inference it is crucial to obtain a good estimator of the variance of the proposed estimator of the statistical estimand. A commonly used estimator of the variance for an asymptotically linear estimator is the sample variance of the estimated influence function. This estimator has been shown to be anti-conservative in limited samples or in the presence of near-positivity violations, leading to elevated Type-I error rates and poor coverage. In this…

结合随机试验与真实世界数据的平均处理效应自适应 TMLEAI 解读

We consider the problem of estimating the average treatment effect (ATE) when both randomized control trial (RCT) data and external real-world data (RWD) are available. We decompose the ATE estimand as the difference between a pooled-ATE estimand that integrates RCT and RWD and a bias estimand that captures the conditional effect of RCT enrollment on the outcome. We introduce an adaptive targeted maximum likelihood estimation (A-TMLE) framework to estimate them. We prove…

技术说明：针对新目标总体标准化平均处理效应的靶向最大似然估计AI 解读

In this technical note we present a targeted maximum likelihood estimator (TMLE) for a previously studied target parameter that aims to transport an average treatment effect (ATE) on a clinical outcome in a source population to what the ATE would have been in another target population. It is assumed that one only observes baseline covariates in the target population, while we assume that one can learn the conditional treatment effect on the outcome of interest in the source…

通过随机位移干预与交叉验证靶向学习自适应识别效应修饰因素AI 解读

In epidemiology, identifying subpopulations that are particularly vulnerable to exposures and those who may benefit differently from exposure-reducing interventions is essential. Factors such as age, gender-specific vulnerabilities, and physiological states such as pregnancy are critical for policymakers when setting regulatory guidelines. However, current semi-parametric methods for estimating heterogeneous treatment effects are often limited to binary exposures and can…

以因果路线图和模拟提高真实数据应用的严谨性与可复现性AI 解读

The Causal Roadmap outlines a systematic approach to asking and answering questions of cause-and-effect: define the quantity of interest, evaluate needed assumptions, conduct statistical estimation, and carefully interpret results. To protect research integrity, it is essential that the algorithm for statistical estimation and inference be pre-specified prior to conducting any effectiveness analyses. However, it is often unclear which algorithm will perform optimally for the…

高维混合暴露的因果中介路径识别：数据自适应目标参数策略AI 解读

Mediation analysis in causal inference typically concentrates on one binary exposure, using deterministic interventions to split the average treatment effect into direct and indirect effects through a single mediator. Yet, real-world exposure scenarios often involve multiple continuous exposures impacting health outcomes through varied mediation pathways, which remain unknown a priori. Addressing this complexity, we introduce NOVAPathways, a methodological framework that…

纵向中介分析的目标最大似然估计AI 解读

Causal mediation analysis with random interventions has become an area of significant interest for understanding time-varying effects with longitudinal and survival outcomes. To tackle causal and statistical challenges due to the complex longitudinal data structure with time-varying confounders, competing risks, and informative censoring, there exists a general desire to combine machine learning techniques and semiparametric theory. In this manuscript, we focus on targeted…

最优动态治疗规则价值估计量的表现与应用AI 解读

Given an (optimal) dynamic treatment rule, it may be of interest to evaluate that rule -- that is, to ask the causal question: what is the expected outcome had every subject received treatment according to that rule? In this paper, we study the performance of estimators that approximate the true value of: 1) an $a$ $priori$ known dynamic treatment rule 2) the true, unknown optimal dynamic treatment rule (ODTR); 3) an estimated ODTR, a so-called "data-adaptive parameter,"…

连续事件时间结局中特定时点干预效应的目标最大似然估计AI 解读

Targeted maximum likelihood estimation is a general methodology combining flexible ensemble learning and semiparametric efficiency theory in a two-step procedure for estimation of causal parameters. Proposed targeted maximum likelihood procedures for survival and competing risks analysis have so far focused on events taken values in discrete time. We here present a targeted maximum likelihood estimation procedure for event times that take values in R+. We focuson the…

情境多臂老虎机实验后的统计推断AI 解读

Contextual bandit algorithms are increasingly replacing non-adaptive A/B tests in e-commerce, healthcare, and policymaking because they can both improve outcomes for study participants and increase the chance of identifying good or even best policies. To support credible inference on novel interventions at the end of the study, nonetheless, we still want to construct valid confidence intervals on average treatment effects, subgroup effects, or value of new policies. The…