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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…

统计方法学 · 统计学 2024-03-15 Bénédicte Colnet , Julie Josse , Gaël Varoquaux , Erwan Scornet

To draw real-world evidence about the comparative effectiveness of multiple time-varying treatments on patient survival, we develop a joint marginal structural survival model and a novel weighting strategy to account for time-varying…

统计方法学 · 统计学 2023-08-08 Liangyuan Hu , Jiayi Ji , Himanshu Joshi , Erick Scott , Fan Li

Estimating causal effects from observational data is challenging due to selection bias, which leads to imbalanced covariate distributions across treatment groups. Propensity score-based weighting methods are widely used to address this…

机器学习 · 计算机科学 2025-08-08 Ahmad Saeed Khan , Erik Schaffernicht , Johannes Andreas Stork

In this work, we are interested in studying the causal effect of an endogenous binary treatment on a dependently censored duration outcome. By dependent censoring, it is meant that the duration time ($T$) and right censoring time ($C$) are…

计量经济学 · 经济学 2025-04-04 Gilles Crommen , Jad Beyhum , Ingrid Van Keilegom

Estimating individualized treatment rules is a central task for personalized medicine. [zhao2012estimating] and [zhang2012robust] proposed outcome weighted learning to estimate individualized treatment rules directly through maximizing the…

统计方法学 · 统计学 2017-10-02 Yifan Cui , Ruoqing Zhu , Michael Kosorok

Studying causal effects of continuous treatments is important for gaining a deeper understanding of many interventions, policies, or medications, yet researchers are often left with observational studies for doing so. In the observational…

统计方法学 · 统计学 2023-06-16 Jared D. Huling , Noah Greifer , Guanhua Chen

Observational data have been actively used to estimate treatment effect, driven by the growing availability of electronic health records (EHRs). However, EHRs typically consist of longitudinal records, often introducing time-dependent…

机器学习 · 计算机科学 2024-06-14 Junghwan Lee , Simin Ma , Nicoleta Serban , Shihao Yang

Marginal structural models (MSMs) with inverse probability weighting offer an approach to estimating causal effects of treatment sequences on repeated outcome measures in the presence of time-varying confounding and dependent censoring.…

统计方法学 · 统计学 2018-07-02 Sean Yiu , Li Su

Propensity score methods are increasingly being used to reduce estimation bias of treatment effects for observational studies. Previous research has shown that propensity score methods consistently estimate the marginal hazard ratio for…

统计方法学 · 统计学 2019-11-19 Haodi Liang , Cecilia Cotton

Long-term behavioral and health risk factors constitute a primary focus of research on the etiology of chronic diseases. Yet, identifying critical time-windows during which risk factors have the strongest impact on disease risk is…

统计方法学 · 统计学 2022-01-14 Maude Wagner , Francine Grodstein , Karen Leffondre , Cécilia Samieri , Cécile Proust-Lima

Optimizing survival outcomes, such as patient survival or customer retention, is a critical objective in data-driven decision-making. Off-Policy Evaluation~(OPE) provides a powerful framework for assessing such decision-making policies…

统计方法学 · 统计学 2026-03-25 Kohsuke Kubota , Mitsuhiro Takahashi , Yuta Saito

In studies based on electronic health records (EHR), the frequency of covariate monitoring can vary by covariate type, across patients, and over time. This can lead to major challenges: first, the difference in monitoring protocols may…

Propensity score (PS) weighting methods are often used in non-randomized studies to adjust for confounding and assess treatment effects. The most popular among them, the inverse probability weighting (IPW), assigns weights that are…

统计方法学 · 统计学 2020-11-04 Yunji Zhou , Roland A. Matsouaka , Laine Thomas

Individual treatment effect (ITE) estimation is to evaluate the causal effects of treatment strategies on some important outcomes, which is a crucial problem in healthcare. Most existing ITE estimation methods are designed for centralized…

机器学习 · 计算机科学 2025-03-10 Changchang Yin , Hong-You Chen , Wei-Lun Chao , Ping Zhang

In attempt to advance the current practice for assessing and predicting the primary ovarian insufficiency (POI) risk in female childhood cancer survivors, we propose two estimating function based approaches for age-specific logistic…

统计方法学 · 统计学 2026-03-31 Haoxuan , Zhou , X. Joan Hu , Yi Xiong , Yan Yuan

Marginal structural models (MSMs) estimate the causal effect of a time-varying treatment in the presence of time-dependent confounding via weighted regression. The standard approach of using inverse probability of treatment weighting (IPTW)…

统计方法学 · 统计学 2019-08-13 Nathan Kallus , Michele Santacatterina

Nested case-control (NCC) is a sampling method widely used for developing and evaluating risk models with expensive biomarkers on large prospective cohort studies. The biomarker values are typically obtained on a sub-cohort, consisting of…

统计方法学 · 统计学 2021-04-07 Qian M. Zhou , Xuan Wang , Yingye Zheng , Tianxi Cai

Randomized controlled trials are the gold standard for measuring causal effects. However, they are often not always feasible, and causal treatment effects must be estimated from observational data. Observational studies do not allow robust…

In stepped wedge cluster randomized trials (SW-CRTs), the intervention is rolled out to clusters over multiple periods. A standard approach for analyzing SW-CRTs utilizes the linear mixed model, where the treatment effect is only present…

Researchers are often interested in estimating effects of generalized time-varying treatment strategies on the mean of an outcome at one or more selected follow-up times of interest. For example, the Medications and Weight Gain in PCORnet…