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Methods for extending -- generalizing or transporting -- inferences from a randomized trial to a target population involve conditioning on a large set of covariates that is sufficient for rendering the randomized and non-randomized groups…

统计方法学 · 统计学 2021-10-04 Sarah E Robertson , Jon A Steingrimsson , Issa J Dahabreh

When devising a course of treatment for a patient, doctors often have little quantitative evidence on which to base their decisions, beyond their medical education and published clinical trials. Stanford Health Care alone has millions of…

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…

统计方法学 · 统计学 2023-09-18 Shanshan Luo , Yechi Zhang , Wei Li

Researchers often run resource-intensive randomized controlled trials (RCTs) to estimate the causal effects of interventions on outcomes of interest. Yet these outcomes are often noisy, and estimated overall effects can be small or…

计量经济学 · 经济学 2023-12-21 Jann Spiess , Vasilis Syrgkanis , Victor Yaneng Wang

In modern drug development, the broader availability of high-dimensional observational data provides opportunities for scientist to explore subgroup heterogeneity, especially when randomized clinical trials are unavailable due to cost and…

统计方法学 · 统计学 2021-02-24 Xinzhou Guo , Linqing Wei , Chong Wu , Jingshen Wang

Subgroup analyses of randomized controlled trials (RCTs) constitute an important component of the drug development process in precision medicine. In particular, subgroup analyses of early-stage trials often influence the design and…

统计方法学 · 统计学 2025-02-12 Daniel Schwartz , Riddhiman Saha , Steffen Ventz , Lorenzo Trippa

Causal inference across multiple data sources offers a promising avenue to enhance the generalizability and replicability of scientific findings. However, data integration methods for time-to-event outcomes, common in biomedical research,…

统计方法学 · 统计学 2025-05-16 Yi Liu , Alexander W. Levis , Ke Zhu , Shu Yang , Peter B. Gilbert , Larry Han

With medical tests becoming increasingly available, concerns about over-testing and over-treatment dramatically increase. Hence, it is important to understand the influence of testing on treatment selection in general practice. Most…

统计方法学 · 统计学 2020-08-11 Yun Li , Irina Bondarenko , Michael R. Elliott , Timothy P. Hofer , Jeremy M. G. Taylor

The era of big data has witnessed an increasing availability of multiple data sources for statistical analyses. We consider estimation of causal effects combining big main data with unmeasured confounders and smaller validation data with…

统计方法学 · 统计学 2021-08-24 Shu Yang , Peng Ding

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

统计方法学 · 统计学 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

In high-stakes settings where machine learning models are used to automate decision-making about individuals, the presence of algorithmic bias can exacerbate systemic harm to certain subgroups of people. These biases often stem from the…

机器学习 · 计算机科学 2026-04-07 Erin Tan , Judy Hanwen Shen , Irene Y. Chen

Unmeasured confounding presents a significant challenge in causal inference from observational studies. Classical approaches often rely on collecting proxy variables, such as instrumental variables. However, in applications where the…

统计方法学 · 统计学 2025-01-16 Xiaochuan Shi , Dehan Kong , Linbo Wang

Dynamic prediction of causal effects under different treatment regimes conditional on an individual's characteristics and longitudinal history is an essential problem in precision medicine. This is challenging in practice because outcomes…

统计方法学 · 统计学 2023-03-07 Yizhen Xu , Jisoo Kim , Laura K. Hummers , Ami A. Shah , Scott Zeger

Motivated by two case studies using primary care records from the Clinical Practice Research Datalink, we describe statistical methods that facilitate the analysis of tall data, with very large numbers of observations. Our focus is on…

统计方法学 · 统计学 2018-05-14 Kirsty Rhodes , Rebecca Turner , Rupert Payne , Ian White

There is strong interest in estimating how the magnitude of treatment effects of an intervention vary across sub-groups of the population of interest. In our paper, we propose a two-study approach to first propose and then test…

统计方法学 · 统计学 2020-06-23 Rahul Ladhania , Amelia Haviland , Neeraj Sood , Edward Kennedy , Ateev Mehrotra

Estimating treatment effects conditional on observed covariates can improve the ability to tailor treatments to particular individuals. Doing so effectively requires dealing with potential confounding, and also enough data to adequately…

Estimation of heterogeneous treatment effects is an active area of research. Most of the existing methods, however, focus on estimating the conditional average treatment effects of a single, binary treatment given a set of pre-treatment…

统计方法学 · 统计学 2025-05-30 Max Goplerud , Kosuke Imai , Nicole E. Pashley

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

统计方法学 · 统计学 2026-01-22 Xinyuan Chen , Fan Li

Although randomized experiments are widely regarded as the gold standard for estimating causal effects, missing data of the pretreatment covariates makes it challenging to estimate the subgroup causal effects. When the missing data…

统计理论 · 数学 2014-01-08 Peng Ding , Zhi Geng

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,…

机器学习 · 统计学 2023-09-25 Larry Han , Zhu Shen , Jose Zubizarreta