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In observational studies, weighting methods that directly optimize the balance between treatment and covariates have received much attention lately; however these have mainly focused on binary treatments. Inspired by domain adaptation, we…

Statistics Theory · Mathematics 2020-02-27 Guillaume Martinet

To make informed health policy decisions regarding a treatment, we must consider both its cost and its clinical effectiveness. In past work, we introduced the net benefit separation (NBS) as a novel measure of cost-effectiveness. The NBS is…

Methodology · Statistics 2021-01-27 Nicholas Illenberger , Nandita Mitra , Andrew J. Spieker

We study the interpretability of conditional probability estimates for binary classification under the agnostic setting or scenario. Under the agnostic setting, conditional probability estimates do not necessarily reflect the true…

Machine Learning · Computer Science 2017-03-01 Yihan Gao , Aditya Parameswaran , Jian Peng

Stochastic volatility often implies increasing risks that are difficult to capture given the dynamic nature of real-world applications. We propose using arc length, a mathematical concept, to quantify cumulative variations (the total…

Methodology · Statistics 2022-12-21 Yan Gao , Rodney A. Sparapani , Sanjib Basu

Accelerated failure time (AFT) models are frequently used to model survival data, providing a direct quantification of the relationship between event times and covariates. These models allow for the acceleration or deceleration of failure…

Methodology · Statistics 2024-12-23 Aishwarya Bhaskaran , Ding Ma , Benoit Liquet , Angela Hong , Stephane Heritier , Serigne N Lo , Jun Ma

In observational studies, the observed association between an exposure and outcome of interest may be distorted by unobserved confounding. Causal sensitivity analysis can be used to assess the robustness of observed associations to…

Methodology · Statistics 2025-11-04 Rui Hu , Ted Westling

Survival analysis models the distribution of time until an event of interest, such as discharge from the hospital or admission to the ICU. When a model's predicted number of events within any time interval is similar to the observed number,…

Machine Learning · Computer Science 2021-01-15 Mark Goldstein , Xintian Han , Aahlad Puli , Adler J. Perotte , Rajesh Ranganath

In observational studies of survival time featuring a binary time-dependent treatment, the hazard ratio (an instantaneous measure) is often used to represent the treatment effect. However, investigators are often more interested in the…

Methodology · Statistics 2020-05-21 Yun Li , Douglas E. Schaubel , Kevin He

In large observational studies, the case-cohort design is commonly used to reduce the cost associated with covariate measurement. For survival outcomes, literature has suggested that the restricted mean survival time (RMST) be a more…

Methodology · Statistics 2026-05-08 Andy Ni , Wei-En Lu , Bo Lu

Identifying reliable biomarkers for predicting clinical events in longitudinal studies is important for accurate disease prognosis and for guiding development of new treatments. However, prognostic studies are often observational, making it…

Methodology · Statistics 2025-08-12 Ainesh Sewak , Vanda Inacio , Joanne Wuu , Michael Benatar , Torsten Hothorn

There has been a split in the statistics community about the need for taking covariates into account in the design phase of a clinical trial. There are many advocates of using stratification and covariate-adaptive randomization to promote…

Methodology · Statistics 2011-02-21 William F. Rosenberger , Oleksandr Sverdlov

Covariate-adaptive randomization (CAR) procedures are frequently used in comparative studies to increase the covariate balance across treatment groups. However, because randomization inevitably uses the covariate information when forming…

Statistics Theory · Mathematics 2022-07-08 Wei Ma , Yichen Qin , Yang Li , Feifang Hu

Despite recent progress in predicting biomarker trajectories from real clinical data, uncertainty in the predictions poses high-stakes risks (e.g., misdiagnosis) that limit their clinical deployment. To enable safe and reliable use of such…

Machine Learning · Statistics 2025-11-19 Vasiliki Tassopoulou , Charis Stamouli , Haochang Shou , George J. Pappas , Christos Davatzikos

The use of instrumental variables for estimating the effect of an exposure on an outcome is popular in econometrics, and increasingly so in epidemiology. This increasing popularity may be attributed to the natural occurrence of instrumental…

Methodology · Statistics 2016-08-03 T. Martinussen , S. Vansteelandt , E. J. Tchetgen Tchetgen , D. M. Zucker

Composite binary endpoints are increasingly used as primary endpoints in clinical trials. When designing a trial, it is crucial to determine the appropriate sample size for testing the statistical differences between treatment groups for…

Applications · Statistics 2019-01-15 Marta Bofill Roig , Guadalupe Gómez Melis

In randomized clinical trials, adjusting for baseline covariates can improve credibility and efficiency for demonstrating and quantifying treatment effects. This article studies the augmented inverse propensity weighted (AIPW) estimator,…

Methodology · Statistics 2024-03-27 Marlena S. Bannick , Jun Shao , Jingyi Liu , Yu Du , Yanyao Yi , Ting Ye

Active surveillance (AS) using repeated biopsies to monitor disease progression has been a popular alternative to immediate surgical intervention in cancer care. However, a biopsy procedure is invasive and sometimes leads to severe side…

Methodology · Statistics 2024-05-21 Muxuan Liang , Yingqi Zhao , Daniel W. Lin , Matthew Cooperberg , Yingye Zheng

Covariate adaptive randomization (CAR) procedures are extensively used to reduce the likelihood of covariate imbalances occurring in clinical trials. In literatures, a lot of CAR procedures have been proposed so that the specified…

Statistics Theory · Mathematics 2026-03-10 Zhang Li-Xin

A set of probabilistic predictions is well calibrated if the events that are predicted to occur with probability p do in fact occur about p fraction of the time. Well calibrated predictions are particularly important when machine learning…

Machine Learning · Statistics 2014-01-14 Mahdi Pakdaman Naeini , Gregory F. Cooper , Milos Hauskrecht

Relative survival methodology deals with a competing risks survival model where the cause of death is unknown. This lack of information occurs regularly in population-based cancer studies. Non-parametric estimation of the net survival is…

Statistics Theory · Mathematics 2025-02-14 Oskar Laverny , Nathalie Grafféo , Roch Giorgi