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Consider an experiment, where a new drug is tested for the first time on human subjects - healthy volunteers. Such experiments are often performed as dose-escalation studies: a set of increasing doses is pre-selected, individuals are…

Statistics Theory · Mathematics 2016-08-17 Samuel Rosa , Radoslav Harman

In early-phase cancer clinical trials, the limited availability of data presents significant challenges in developing a framework to efficiently quantify treatment effectiveness. To address this, we propose a novel utility-based Bayesian…

Methodology · Statistics 2025-06-10 Saurabh Bhandari , Michael J. Daniels , Chenguang Wang

High-dimensional compositional data arise naturally in many applications such as metagenomic data analysis. The observed data lie in a high-dimensional simplex, and conventional statistical methods often fail to produce sensible results due…

Methodology · Statistics 2016-01-19 Yuanpei Cao , Wei Lin , Hongzhe Li

Motivation: HIV is difficult to treat because its virus mutates at a high rate and mutated viruses easily develop resistance to existing drugs. If the relationships between mutations and drug resistances can be determined from historical…

Quantitative Methods · Quantitative Biology 2019-07-08 Li Xing , Mary Lesperance , Xuekui Zhang

Complex or co-existing diseases are commonly treated using drug combinations, which can lead to higher risk of adverse side effects. The detection of polypharmacy side effects is usually done in Phase IV clinical trials, but there are still…

Machine Learning · Statistics 2019-05-03 Andreea Deac , Yu-Hsiang Huang , Petar Veličković , Pietro Liò , Jian Tang

Clinical trials in specific indications require the administration of rescue medication in case a patient does not sufficiently respond to investigational treatment. The application of additional treatment on an as needed basis causes…

Methodology · Statistics 2016-08-30 Gerd K. Rosenkranz

In conventional randomized controlled trials, adjustment for baseline values of covariates known to be at least moderately associated with the outcome increases the power of the trial. Recent work has shown particular benefit for more…

Methodology · Statistics 2023-11-27 James Willard , Shirin Golchi , Erica EM Moodie

In a Phase II dose-finding study with a placebo control, a new drug with several dose levels is compared with a placebo to test for the effectiveness of the new drug. The main focus of such studies often lies in the characterization of the…

Methodology · Statistics 2020-07-14 Saswati Saha , Werner Brannath

We explore applying a tensor completion approach to complete the DrugMatrix toxicogenomics dataset. Our hypothesis is that by preserving the 3-dimensional structure of the data, which comprises tissue, treatment, and transcriptomic…

Machine Learning · Computer Science 2025-07-08 Tan Nguyen , Guojing Cong

The initiation of dose optimization has driven a paradigm shift in oncology clinical trials to determine the optimal biological dose (OBD). Early-phase trials with randomized doses can facilitate additional investigation of the identified…

Methodology · Statistics 2025-02-27 Gina DAngelo , Guannan Chen , Di Ran

In clinical trials, there is potential to improve precision and reduce the required sample size by appropriately adjusting for baseline variables in the statistical analysis. This is called covariate adjustment. Despite recommendations by…

Methodology · Statistics 2022-06-20 Kelly Van Lancker , Joshua Betz , Michael Rosenblum

In situations where it is difficult to enroll patients in randomized controlled trials, external data can improve efficiency and feasibility. In such cases, adaptive trial designs could be used to decrease enrollment in the control arm of…

Methodology · Statistics 2020-10-02 Brian D. Segal , W. Katherine Tan

As the COVID-19 pandemic progresses, researchers are reporting findings of randomized trials comparing standard care with care augmented by experimental drugs. The trials have small sample sizes, so estimates of treatment effects are…

Econometrics · Economics 2020-06-02 Charles F. Manski , Aleksey Tetenov

We propose a flexible design for the identification of optimal dose combinations in dual-agent dose-finding clinical trials. The design is called AAA, standing for three adaptations: adaptive model selection, adaptive dose insertion, and…

Methodology · Statistics 2017-06-13 Jiaying Lyu , Yuan Ji , Naiqing Zhao , Daniel V. T. Catenacci

The primary goal of dose allocation in phase I trials is to minimize patient exposure to subtherapeutic or excessively toxic doses, while accurately recommending a phase II dose that is as close as possible to the maximum tolerated dose…

Methodology · Statistics 2025-03-21 Jiapeng Xu , Dehua Bi , Shenghua Kelly Fan , Bee Leng Lee , Ying Lu

The Binary Emax model is widely employed in dose-response analysis during drug development, where missing data often pose significant challenges. Addressing nonignorable missing binary responses, where the likelihood of missing data is…

Methodology · Statistics 2026-01-01 Jiangshan Zhang , Vivek Pradhan , Yuxi Zhao

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…

Adherence can be defined as "the extent to which patients take their medications as prescribed by their healthcare providers"[Osterberg and Blaschke, 2005]. World Health Organization's reports point out that, in developed countries, only…

Machine Learning · Computer Science 2018-11-30 Thomas Janssoone , Clémence Bic , Dorra Kanoun , Pierre Hornus , Pierre Rinder

In drug discovery, highly automated high-throughput laboratories are used to screen a large number of compounds in search of effective drugs. These experiments are expensive, so one might hope to reduce their cost by only experimenting on a…

Machine Learning · Computer Science 2025-04-15 Ihor Neporozhnii , Julien Roy , Emmanuel Bengio , Jason Hartford

Assessing drug-target affinity is a critical step in the drug discovery and development process, but to obtain such data experimentally is both time consuming and expensive. For this reason, computational methods for predicting binding…

Machine Learning · Computer Science 2022-09-15 Elizaveta Vinogradova , Karina Pats , Ferdinand Molnár , Siamac Fazli
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