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Two commonly used methods for improving precision and power in clinical trials are stratified randomization and covariate adjustment. However, many trials do not fully capitalize on the combined precision gains from these two methods, which…

统计方法学 · 统计学 2020-09-04 Bingkai Wang , Ryoko Susukida , Ramin Mojtabai , Masoumeh Amin-Esmaeili , Michael Rosenblum

In recent years, reinforcement learning (RL) has acquired a prominent position in health-related sequential decision-making problems, gaining traction as a valuable tool for delivering adaptive interventions (AIs). However, in part due to a…

机器学习 · 统计学 2024-07-16 Nina Deliu , Joseph Jay Williams , Bibhas Chakraborty

Personalized medicine has gained much popularity recently as a way of providing better healthcare by tailoring treatments to suit individuals. Our research, motivated by the UK INTERVAL blood donation trial, focuses on estimating the…

统计方法学 · 统计学 2023-02-24 Yuejia Xu , Angela M. Wood , David J. Roberts , Brian D. M. Tom

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…

统计方法学 · 统计学 2020-10-02 Brian D. Segal , W. Katherine Tan

The most common objective for response adaptive clinical trials is to seek to ensure that patients within a trial have a high chance of receiving the best treatment available by altering the chance of allocation on the basis of accumulating…

统计方法学 · 统计学 2020-11-09 Helen Yvette Barnett , Sofia S Villar , Helena Geys , Thomas Jaki

Testing whether a variable of interest affects the outcome is one of the most fundamental problem in statistics and is often the main scientific question of interest. To tackle this problem, the conditional randomization test (CRT) is…

统计方法学 · 统计学 2023-05-26 Dae Woong Ham , Jiaze Qiu

One of the challenges in the design of confirmatory trials is to deal with uncertainties regarding the optimal target population for a novel drug. Adaptive enrichment designs (AED) which allow for a data-driven selection of one or more…

应用统计 · 统计学 2020-09-15 Anh Nguyen Duc , Dominik Heinzmann , Claude Berge , Marcel Wolbers

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…

统计方法学 · 统计学 2026-01-16 Gianmarco Caruso , Pavel Mozgunov

It is common to conduct causal inference in matched observational studies by proceeding as though treatment assignments within matched sets are assigned uniformly at random and using this distribution as the basis for inference. This…

统计方法学 · 统计学 2023-11-14 Samuel D. Pimentel , Yaxuan Huang

External information borrowing is often considered in order to improve a clinical trial's efficiency. The Bayesian approach borrows such external information by specifying an informative prior distribution. A potential issue with this…

统计方法学 · 统计学 2025-08-25 Silvia Calderazzo , Manuel Wiesenfarth , Vivienn Weru , Annette Kopp-Schneider

This work bridges the gap between staggered adoption designs and survival analysis to estimate causal effects in settings with time-varying treatments, addressing a fundamental challenge in medical research exemplified by the Stanford Heart…

统计方法学 · 统计学 2025-03-04 Xiang Meng , Iavor Bojinov

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of…

统计方法学 · 统计学 2023-06-12 Kelly Van Lancker , Frank Bretz , Oliver Dukes

An individualized treatment rule (ITR) is a decision rule that aims to improve individual patients health outcomes by recommending optimal treatments according to patients specific information. In observational studies, collected data may…

统计方法学 · 统计学 2023-10-03 Zeyu Bian , Erica EM Moodie , Susan M Shortreed , Sylvie D Lambert , Sahir Bhatnagar

The field of precision medicine aims to tailor treatment based on patient-specific factors in a reproducible way. To this end, estimating an optimal individualized treatment regime (ITR) that recommends treatment decisions based on patient…

Fault detection is essential in complex industrial systems to prevent failures and optimize performance by distinguishing abnormal from normal operating conditions. With the growing availability of condition monitoring data, data-driven…

应用统计 · 统计学 2025-10-14 Han Sun , Olga Fink

Response-adaptive randomization (RAR) methods can be used to adapt randomization probabilities based on accumulating data, aiming to increase the probability of allocating patients to effective treatments. A popular RAR method is Thompson…

统计方法学 · 统计学 2026-03-10 Samuel Pawel , Leonhard Held

Individualized treatment recommendation (ITR) is an important analytic framework for precision medicine. The goal is to assign proper treatments to patients based on their individual characteristics. From the machine learning perspective,…

机器学习 · 统计学 2020-04-07 Haomiao Meng , Ying-Qi Zhao , Haoda Fu , Xingye Qiao

We introduce a new multiple type I error criterion for clinical trials with multiple populations. Such trials are of interest in precision medicine where the goal is to develop treatments that are targeted to specific sub-populations…

统计方法学 · 统计学 2021-02-05 Werner Brannath , Charlie Hillner , Kornelius Rohmeyer

Item Response Theory (IRT) is a well known method for assessing responses from humans in education and psychology. In education, IRT is used to infer student abilities and characteristics of test items from student responses. Interactions…

人工智能 · 计算机科学 2023-07-20 Antti Keurulainen , Isak Westerlund , Oskar Keurulainen , Andrew Howes

Individualized treatment effect lies at the heart of precision medicine. Interpretable individualized treatment rules (ITRs) are desirable for clinicians or policymakers due to their intuitive appeal and transparency. The gold-standard…

统计方法学 · 统计学 2021-08-20 Lili Wu , Shu Yang