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A Causal Inference Approach for Evaluating Diagnostic Tests and AI-Enabled Medical Devices: From Effect Modification to Information-Augmented Decision-Making

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

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…

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An Online Meta-Level Adaptive Design Framework with Targeted Learning Inference: Applications to Evaluating and Utilizing Surrogate Outcomes in Adaptive Designs

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

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…

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Data reuse enables cost-efficient randomized trials of medical AI models

通过数据复用降低医疗 AI 模型随机试验成本

Randomized controlled trials (RCTs) are indispensable for establishing the clinical value of medical artificial-intelligence (AI) tools, yet their high cost and long timelines hinder timely validation as new models emerge rapidly. Here, we propose BRIDGE, a data-reuse RCT design for AI-based risk models. AI risk models support a broad range of interventions, including screening, treatment selection, and clinical alerts. BRIDGE trials recycle participant-level data from…

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Constructing Confidence Intervals for Infinite-Dimensional Functional Parameters by Highly Adaptive Lasso

利用高度自适应 Lasso 构造无限维函数参数的置信区间

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…

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HAL-Based Plug-in Estimation with Pointwise Asymptotic Normality of the Causal Dose-Response Curve

基于 HAL 的因果剂量反应曲线插件估计与逐点渐近正态性

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…

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Efficient Statistical Estimation for Sequential Adaptive Experiments with Implications for Adaptive Designs

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

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'…

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Design-based causal inference in bipartite experiments

二部实验中基于设计的因果推断

Bipartite experiments arise in various fields, in which the treatments are randomized over one set of units, while the outcomes are measured over another separate set of units. However, existing methods often rely on strong model assumptions about the data-generating process. Under the potential outcomes formulation, we explore design-based causal inference in bipartite experiments under weak assumptions by leveraging the sparsity structure of the bipartite graph that…

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Data-Adaptive Identification of Effect Modifiers through Stochastic Shift Interventions and Cross-Validated Targeted Learning

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

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…

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