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We present a new experimental design procedure that divides a set of experimental units into two groups in order to minimize error in estimating an additive treatment effect. One concern is minimizing error at the experimental design stage…

Methodology · Statistics 2021-02-02 Abba M. Krieger , David Azriel , Adam Kapelner

Traditionally, the major objective in phase I trials is to identify a working-dose for subsequent studies, whereas the major endpoint in phase II and III trials is treatment efficacy. The dose sought is typically referred to as the maximum…

Methodology · Statistics 2016-08-14 Mourad Tighiouart , André Rogatko

Large Language Models (LLMs) are increasingly applied to automate software engineering tasks, including the generation of UML class diagrams from natural language descriptions. While prior work demonstrates that LLMs can produce…

Software Engineering · Computer Science 2026-04-07 Rabia Iftikhar , Andreas Rausch

Trials enroll a large number of subjects in order to attain power, making them expensive and time-consuming. Sample size calculations are often performed with the assumption of an unadjusted analysis, even if the trial analysis plan…

Methodology · Statistics 2021-07-06 Alejandro Schuler

The Bayesian Optimal Phase II (BOP2) framework is a flexible trial design that can naturally facilitate complex adaptations due to its Bayesian setting. BOP2 uses equal randomisation and equally placed interim analyses in its design, but it…

Applications · Statistics 2025-11-19 Connor Fitchett , Ayon Mukherjee , Sofía S. Villar , David S. Robertson

Combination drug therapies hold significant promise for enhancing treatment efficacy, particularly in fields such as oncology, immunotherapy, and infectious diseases. However, designing clinical trials for these regimens poses unique…

Methodology · Statistics 2025-06-04 Nan Miles Xi , Man Mandy Jin , Lin Wang , Xin Huang

Technological advancements in the field of mobile devices and wearable sensors have helped overcome obstacles in the delivery of care, making it possible to deliver behavioral treatments anytime and anywhere. Increasingly the delivery of…

Applications · Statistics 2017-11-13 Walter Dempsey , Peng Liao , Santosh Kumar , Susan A. Murphy

In this article, universally optimal multivariate crossover designs are studied. The multiple response crossover design is motivated by a $3 \times 3$ crossover setup, where the effect of $3$ doses of an oral drug are studied on gene…

Methodology · Statistics 2024-07-16 Shubham Niphadkar , Siuli Mukhopadhyay

Longitudinal cluster randomized trials (L-CRTs) are increasingly used to evaluate the cost-effectiveness of healthcare interventions across multiple assessment periods, yet design methods for powering these trials remain underdeveloped.…

Methodology · Statistics 2026-03-20 Hao Wang , Jingxia Liu , Drew B. Cameron , Jiaqi Tong , Donna Spiegelman , Daniella Meeker , Fan Li

From clinical development of cancer therapies to investigations into partisan bias, adaptive sequential designs have become increasingly popular method for causal inference, as they offer the possibility of improved precision over their…

Methodology · Statistics 2023-10-17 Jessica Dai , Paula Gradu , Christopher Harshaw

Simulation offers a simple and flexible way to estimate the power of a clinical trial when analytic formulae are not available. The computational burden of using simulation has, however, restricted its application to only the simplest of…

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

Clinical trials are typically run in order to understand the effects of a new treatment on a given population of patients. However, patients in large populations rarely respond the same way to the same treatment. This heterogeneity in…

Machine Learning · Statistics 2024-02-12 Alihan Hüyük , Zhaozhi Qian , Mihaela van der Schaar

Nonlinear regression models addressing both efficacy and toxicity outcomes are increasingly used in dose-finding trials, such as in pharmaceutical drug development. However, research on related experimental design problems for corresponding…

Methodology · Statistics 2016-01-06 Holger Dette , Katrin Kettelhake , Kirsten Schorning , Weng Kee Wong , Frank Bretz

Group sequential designs drive innovation in clinical, industrial, and corporate settings. Early stopping for failure in sequential designs conserves experimental resources, whereas early stopping for success accelerates access to improved…

Methodology · Statistics 2025-11-27 Luke Hagar , Shirin Golchi , Marina B. Klein

Two-dimensional, resonant scanners have been utilized in a large variety of imaging modules due to their compact form, low power consumption, large angular range, and high speed. However, resonant scanners have problems with non-optimal and…

Image and Video Processing · Electrical Eng. & Systems 2023-05-05 Zhanghao Sun , Ronald Quan , Olav Solgaard

Feature screening for ultrahigh-dimension, in general, proceeds with two essential steps. The first step is measuring and ranking the marginal dependence between response and covariates, and the second is determining the threshold. We…

Methodology · Statistics 2022-07-28 Linsui Deng , Yilin Zhang

Clinicians and researchers alike are increasingly interested in how best to personalize interventions. A dynamic treatment regimen (DTR) is a sequence of pre-specified decision rules which can be used to guide the delivery of a sequence of…

Adaptive experiments are becoming increasingly popular in real-world applications for effectively maximizing in-sample welfare and efficiency by data-driven sampling. Despite their growing prevalence, however, the statistical foundations…

Statistics Theory · Mathematics 2026-04-15 Ziang Niu , Zhimei Ren

Traditional health authority approval for oncology drugs is based on a clinical benefit endpoint, or a valid surrogate. In 1992 the FDA created the Accelerated Approval pathway to allow for earlier approval of therapies in serious…

Applications · Statistics 2026-02-26 Jane She , Xiaofei Chen , Malini Iyengar , Judy Li