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This paper investigates an interesting weakly supervised regression setting called regression with interval targets (RIT). Although some of the previous methods on relevant regression settings can be adapted to RIT, they are not…

Machine Learning · Computer Science 2023-06-21 Xin Cheng , Yuzhou Cao , Ximing Li , Bo An , Lei Feng

Background: Phase I dose-finding trials increasingly encounter delayed-onset toxicities, especially with immunotherapies and targeted agents. The time-to-event continual reassessment method (TITE-CRM) handles incomplete follow-up using…

Methodology · Statistics 2026-02-24 Robert Amevor , Emmanuel Kubuafor , Dennis Baidoo

Effective medication management in Parkinson's Disease (PD) is challenging due to heterogeneous disease progression, variable patient response, and medication side effects. While AI models can forecast levodopa equivalent daily dose (LEDD)…

Machine Learning · Computer Science 2026-05-21 Ricardo Diaz-Rincon , Muxuan Liang , Adolfo Ramirez-Zamora , Benjamin Shickel

A common problem in health research is that we have a large database with many variables measured on a large number of individuals. We are interested in measuring additional variables on a subsample; these measurements may be newly…

Methodology · Statistics 2022-03-22 Thomas Lumley , Tong Chen

Regression discontinuity designs are frequently used to estimate the causal effect of election outcomes and policy interventions. In these contexts, treatment effects are typically estimated with covariates included to improve efficiency.…

Applications · Statistics 2020-05-06 L. Jason Anastasopoulos

Benchmark dose analysis aims to estimate the level of exposure to a toxin that results in a clinically-significant adverse outcome and quantifies uncertainty using the lower limit of a confidence interval for this level. We develop a novel…

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

An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…

Methodology · Statistics 2018-11-27 Marius Thomas , Björn Bornkamp , Katja Ickstadt

Estimating causal effects of continuous treatments is a common problem in practice, for example, in studying average dose-response functions. Classical analyses typically assume that all confounders are fully observed, whereas in real-world…

Statistics Theory · Mathematics 2026-04-14 Shuyuan Chen , Peng Zhang , Yifan Cui

Sparse additive modeling is a class of effective methods for performing high-dimensional nonparametric regression. This paper develops a sparse additive model focused on estimation of treatment effect-modification with simultaneous…

Methodology · Statistics 2020-06-02 Hyung Park , Eva Petkova , Thaddeus Tarpey , R. Todd Ogden

Precision medicine aims to tailor treatment decisions according to patients' characteristics. G-estimation and dynamic weighted ordinary least squares (dWOLS) are double robust statistical methods that can be used to identify optimal…

Methodology · Statistics 2021-11-10 Denis Talbot , Erica EM Moodie , Caroline Diorio

Combining information from multiple samples is often needed in biomedical and economic studies, but the differences between these samples must be appropriately taken into account in the analysis of the combined data. We study estimation for…

Methodology · Statistics 2018-08-14 Heng Shu , Zhiqiang Tan

Multi-arm trials are gaining interest in practice given the statistical and logistical advantages they can offer. The standard approach uses a fixed allocation ratio, but there is a call for making it adaptive and skewing the allocation of…

Methodology · Statistics 2026-01-16 Gianmarco Caruso , Pavel Mozgunov

The primary objective of phase I cancer clinical trials is to evaluate the safety of a new experimental treatment and to find the maximum tolerated dose (MTD). We show that the MTD estimation problem can be regarded as a level set…

Machine Learning · Statistics 2025-04-15 Keiichiro Seno , Kota Matsui , Shogo Iwazaki , Yu Inatsu , Shion Takeno , Shigeyuki Matsui

Estimating causal effects from observational data informs us about which factors are important in an autonomous system, and enables us to take better decisions. This is important because it has applications in selecting a treatment in…

Machine Learning · Computer Science 2021-10-29 Plabon Shaha , Talha Islam Zadid , Ismat Rahman , Md. Mosaddek Khan

Evaluating the influence of continuous covariates, like exposure time or dose, on a response variable is a pivotal objective in the assessment of a compound's effect, particularly when determining toxicity in pre-clinical research or…

Methodology · Statistics 2026-04-16 Lucia Ameis , Niklas Hagemann , Kathrin Möllenhoff

Adaptive radiation therapy (ART) seeks to maintain accurate dose delivery by monitoring anatomical changes during treatment and modifying plans accordingly, yet commonly used approaches for estimating cumulative dose rely on heuristic,…

Medical Physics · Physics 2026-03-03 Robert Boyd , Wolfgang A. Tomé

We study how to efficiently estimate average treatment effects (ATEs) using adaptive experiments. In adaptive experiments, experimenters sequentially assign treatments to experimental units while updating treatment assignment probabilities…

Machine Learning · Statistics 2025-02-21 Masahiro Kato , Takuya Ishihara , Junya Honda , Yusuke Narita

Pattern-mixture models have received increasing attention as they are commonly used to assess treatment effects in primary or sensitivity analyses for clinical trials with nonignorable missing data. Pattern-mixture models have traditionally…

Methodology · Statistics 2024-10-10 Jitong Lou , Mallikarjuna Rettiganti , Yongming Qu

Complex biological processes are usually experimented along time among a collection of individuals. Longitudinal data are then available and the statistical challenge is to better understand the underlying biological mechanisms. The…

Statistics Theory · Mathematics 2015-06-11 Pierre Barbillon , Célia Barthélémy , Adeline Samson