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Genomics, especially multi-omics, has made precision medicine feasible. The completion and publicly accessible multi-omics resource with clinical outcome, such as The Cancer Genome Atlas (TCGA) is a great test bed for developing…

Genomics · Quantitative Biology 2020-08-31 Lana X Garmire

It is increasingly common in digital environments to use A/B tests to compare the performance of recommendation algorithms. However, such experiments often violate the stable unit treatment value assumption (SUTVA), particularly SUTVA's "no…

Clinical trials often involve the assessment of multiple endpoints to comprehensively evaluate the efficacy and safety of interventions. In the work, we consider a global nonparametric testing procedure based on multivariate rank for the…

Methodology · Statistics 2023-06-29 Kexuan Li , Lingli Yang , Shaofei Zhao , Susie Sinks , Luan Lin , Peng Sun

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…

Methodology · Statistics 2018-05-14 Kirsty Rhodes , Rebecca Turner , Rupert Payne , Ian White

Random-effects meta-analyses are very commonly used in medical statistics. Recent methodological developments include multivariate (multiple outcomes) and network (multiple treatments) meta-analysis. Here we provide a new model and…

Methodology · Statistics 2017-08-16 Dan Jackson , Sylwia Bujkiewicz , Martin Law , Richard D Riley , Ian White

Evaluating and tracking patients' functional status through the post-acute care continuum requires a common instrument. However, different post-acute service providers such as nursing homes, inpatient rehabilitation facilities and home…

Methodology · Statistics 2020-01-22 Chenyang Gu , Roee Gutman

The Stable Unit Treatment Value Assumption (SUTVA) includes the condition that there are no multiple versions of treatment in causal inference. Though we could not control the implementation of treatment in observational studies, multiple…

Methodology · Statistics 2026-01-05 Kohei Yoshikawa , Shuichi Kawano

Multiple technologies that measure expression levels of protein mixtures in the human body offer a potential for detection and understanding the disease. The recent increase of these technologies prompts researchers to evaluate the…

Machine Learning · Computer Science 2026-05-12 Michal Valko , Richard Pelikan , Miloš Hauskrecht

Survival analysis plays a crucial role in understanding time-to-event (survival) outcomes such as disease progression. Despite recent advancements in causal mediation frameworks for survival analysis, existing methods are typically based on…

Methodology · Statistics 2026-05-07 Seungjun Ahn , Weijia Fu , Maaike van Gerwen , Lei Liu , Zhigang Li

The proliferation of early diagnostic technologies, including self-monitoring systems and wearables, coupled with the application of these technologies on large segments of healthy populations may significantly aggravate the problem of…

Machine Learning · Computer Science 2021-07-23 Anna Fedyukova , Douglas Pires , Daniel Capurro

Several recently developed methods have the potential to harness machine learning in the pursuit of target quantities inspired by causal inference, including inverse weighting, doubly robust estimating equations and substitution estimators…

Balanced representation learning methods have been applied successfully to counterfactual inference from observational data. However, approaches that account for survival outcomes are relatively limited. Survival data are frequently…

Machine Learning · Statistics 2021-03-04 Paidamoyo Chapfuwa , Serge Assaad , Shuxi Zeng , Michael J. Pencina , Lawrence Carin , Ricardo Henao

Estimating heterogeneous treatment effect (HTE) for survival outcomes has gained increasing attention, as it captures the variation in treatment efficacy across patients or subgroups in delaying disease progression. However, most existing…

Methodology · Statistics 2025-11-27 Na Bo , Ying Ding

Randomized clinical trials are the gold standard when estimating the average treatment effect. However, they are usually not a random sample from the real-world population because of the inclusion/exclusion rules. Meanwhile, observational…

Methodology · Statistics 2024-12-11 Kuan Jiang , Wenjie Hu , Shu Yang , Xinxing Lai , Xiaohua Zhou

Machine learning methods in healthcare have traditionally focused on using data from a single modality, limiting their ability to effectively replicate the clinical practice of integrating multiple sources of information for improved…

Machine Learning · Computer Science 2024-02-13 Felix Krones , Umar Marikkar , Guy Parsons , Adam Szmul , Adam Mahdi

We consider a randomized controlled trial between two groups. The objective is to identify a population with characteristics such that the test therapy is more effective than the control therapy. Such a population is called a subgroup. This…

Methodology · Statistics 2021-12-06 Shintaro Yuki , Kensuke Tanioka , Hiroshi Yadohisa

Studies often estimate associations between an outcome and multiple variates. For example, studies of diagnostic test accuracy estimate sensitivity and specificity, and studies of predictive and prognostic factors typically estimate…

Surrogate endpoint (SE) for overall survival in cancer patients is essential to improving the efficiency of oncology drug development. In practice, we may discover a new patient level association with survival, based on one or more clinical…

Applications · Statistics 2022-11-08 Wei Zou

Various approaches to gene selection for cancer classification based on microarray data can be found in the literature and they may be grouped into two categories: univariate methods and multivariate methods. Univariate methods look at each…

Quantitative Methods · Quantitative Biology 2015-06-18 Min Xu , Rudy Setiono

Federated causal inference enables multi-site treatment effect estimation without sharing individual-level data, offering a privacy-preserving solution for real-world evidence generation. However, data heterogeneity across sites, manifested…

Machine Learning · Computer Science 2025-05-06 Haoyang Li , Jie Xu , Kyra Gan , Fei Wang , Chengxi Zang