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Risk modeling with EHR data is challenging due to a lack of direct observations on the disease outcome, and the high dimensionality of the candidate predictors. In this paper, we develop a surrogate assisted semi-supervised-learning (SAS)…

Statistics Theory · Mathematics 2021-05-05 Jue Hou , Zijian Guo , Tianxi Cai

Adaptive subgroup enrichment design is an efficient design framework that allows accelerated development for investigational treatments while also having flexibility in population selection within the course of the trial. The adaptive…

Methodology · Statistics 2022-10-21 Liwen Wu , Qing Li , Mengya Liu , Jianchang Lin

The primary analysis in two-arm clinical trials usually involves inference on a scalar treatment effect parameter; e.g., depending on the outcome, the difference of treatment-specific means, risk difference, risk ratio, or odds ratio. Most…

Methodology · Statistics 2022-04-25 Anastasios A. Tsiatis , Marie Davidian

In clinical studies, the risk of the primary (terminal) event may be modified by intermediate events, resulting in semicompeting risks. To study the treatment effect on the terminal event mediated by the intermediate event, researchers wish…

Methodology · Statistics 2026-05-26 Yuhao Deng , Rui Wang , Tao Zhang , Xiang Zhan

When a new treatment is considered for use, whether a pharmaceutical drug or a search engine ranking algorithm, a typical question that arises is, will its performance exceed that of the current treatment? The conventional way to answer…

Machine Learning · Computer Science 2016-10-27 Nir Rosenfeld , Yishay Mansour , Elad Yom-Tov

The synthetic control method (SCM) has become a popular tool for estimating causal effects in policy evaluation, where a single treated unit is observed, and a heterogeneous set of untreated units with pre- and post-policy change data are…

Methodology · Statistics 2023-08-21 Jizhou Liu , Eric J. Tchetgen Tchetgen , Carlos Varjão

A surrogate endpoint S in a clinical trial is an outcome that may be measured earlier or more easily than the true outcome of interest T. In this work, we extend causal inference approaches to validate such a surrogate using potential…

Methodology · Statistics 2022-02-04 Emily Roberts , Michael Elliott , Jeremy M. G. Taylor

Studies involving both randomized experiments as well as observational data typically involve time-to-event outcomes such as time-to-failure, death or onset of an adverse condition. Such outcomes are typically subject to censoring due to…

Methodology · Statistics 2023-02-27 Chirag Nagpal , Vedant Sanil , Artur Dubrawski

Before we apply nonlinear techniques, for example those inspired by chaos theory, to dynamical phenomena occurring in nature, it is necessary to first ask if the use of such advanced techniques is justified "by the data". While many…

chao-dyn · Physics 2009-10-31 Thomas Schreiber , Andreas Schmitz

Treatment policy estimands are frequently favored by regulators, as they assess the effect of treatment assignment regardless of post-randomization events. Despite best efforts, missing data due to study discontinuation cannot be fully…

Methodology · Statistics 2026-05-13 Ajmal Oodally , Craig Wang , Zheng Li , Tim Morris , Tobias Mütze , Arunava Chakravartty

When the primary outcome is hard to collect, surrogate endpoint is typically used as a substitute. However, even when the treatment has a positive average causal effect (ACE) on the surrogate endpoint, which also has a positive ACE on the…

Statistics Theory · Mathematics 2016-07-20 Yunjian Yin , Lan Liu , Zhi Geng , Peng Luo

Surrogate models provide compact relations between user-defined input parameters and output quantities of interest, enabling the efficient evaluation of complex parametric systems in many-query settings. Such capabilities are essential in a…

Numerical Analysis · Mathematics 2026-03-16 Matteo Giacomini , Pedro Díez

Hypothesis testing based on surrogate data has emerged as a popular way to test the null hypothesis that a signal is a realization of a linear stochastic process. Typically, this is done by generating surrogates which are made to conform to…

Chaotic Dynamics · Physics 2010-08-12 Diego Guarin , Alvaro Orozco , Edilson Delgado

Deciding on an appropriate intervention requires a causal model of a treatment, the outcome, and potential mediators. Causal mediation analysis lets us distinguish between direct and indirect effects of the intervention, but has mostly been…

Machine Learning · Computer Science 2023-06-19 Çağlar Hızlı , ST John , Anne Juuti , Tuure Saarinen , Kirsi Pietiläinen , Pekka Marttinen

In many decision-making problems, the primary outcome is expensive, time-consuming, or difficult to observe, so individualized treatment rules (ITRs) may be instead learned from surrogate endpoints. However, a surrogate that is highly…

Methodology · Statistics 2026-04-13 Zeyu Xu , Xiaojie Mao , Hao Mei , Yue Liu

When the primary outcome is difficult to collect, surrogate endpoint is typically used as a substitute. It is possible that for every individual, treatment has a positive effect on surrogate, and surrogate has a positive effect on primary…

Methodology · Statistics 2017-12-27 Linquan Ma , Yunjian Yin , Lan Liu , Zhi Geng

We investigate the effectiveness of a simple solution to the common problem of deep learning in medical image analysis with limited quantities of labeled training data. The underlying idea is to assign artificial labels to abundantly…

Computer Vision and Pattern Recognition · Computer Science 2019-01-28 Nima Tajbakhsh , Yufei Hu , Junli Cao , Xingjian Yan , Yi Xiao , Yong Lu , Jianming Liang , Demetri Terzopoulos , Xiaowei Ding

Estimating the effects of long-term treatments through A/B testing is challenging. Treatments, such as updates to product functionalities, user interface designs, and recommendation algorithms, are intended to persist within the system for…

Econometrics · Economics 2025-12-30 Shan Huang , Chen Wang , Yuan Yuan , Jinglong Zhao , Brocco , Zhang

The vast repositories of Electronic Health Records (EHR) and medical claims hold untapped potential for studying rare but critical events, such as suicide attempt. Conventional setups often model suicide attempt as a univariate outcome and…

Methodology · Statistics 2025-01-28 Xiaohui Yin , Shane Sacco , Robert H. Aseltine , Fei Wang , Kun Chen

Surrogate variables in electronic health records (EHR) and biobank data play an important role in biomedical studies due to the scarcity or absence of chart-reviewed gold standard labels. We develop a novel approach named SASH for {\bf…

Methodology · Statistics 2023-09-04 Yue Liu , Molei Liu , Zijian Guo , Tianxi Cai