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We study the design of experiments with multiple treatment levels, a setting common in clinical trials and online A/B/n testing. Unlike single-treatment studies, practical analyses of multi-treatment experiments typically first select a…

Methodology · Statistics 2025-10-07 Jiachen Xu , Jian Qian , Zijun Gao

Novel dose-finding designs, using estimation to assign the best estimated maximum- tolerated-dose (MTD) at each point in the experiment, most commonly via Bayesian techniques, have recently entered large-scale implementation in Phase I…

Methodology · Statistics 2017-01-24 Assaf P. Oron , Peter D. Hoff

A two-stage adaptive optimal design is an attractive option for increasing the efficiency of clinical trials. In these designs, based on interim data, the locally optimal dose is chosen for further exploration, which induces dependencies…

Methodology · Statistics 2019-05-24 Zhantao Lin , Nancy Flournoy , William F. Rosenberger

The paper develops methods to construct a one-stage optimal design of dilution experiments under the total available volume constraint typical for bio-medical applications. We consider various design criteria based on the Fisher information…

Statistics Theory · Mathematics 2013-05-08 Maryam Zolghadr , Sergei Zuyev

Phase I dose-escalation trials must be guided by a safety model in order to avoid exposing patients to unacceptably high risk of toxicities. Traditionally, these trials are based on one type of schedule. In more recent practice, however,…

Methodology · Statistics 2020-08-18 Burak Kürsad Günhan , Sebastian Weber , Tim Friede

In randomized dose-finding trials, although drug exposure data form a part of key information for dose selection, the evaluation of the dose-response (DR) relationship often mainly uses DR data. We examine the benefit of…

Methodology · Statistics 2025-08-07 Jixian Wang , Zhiwei Zhang , Ram Tiwari

There are multiple cluster randomised trial designs that vary in when the clusters cross between control and intervention states, when observations are made within clusters, and how many observations are made at that time point. Identifying…

Methodology · Statistics 2023-07-20 Samuel I. Watson , Alan Girling , Karla Hemming

We improve the existing results of optimal partial profile paired choice designs and provide new designs for situations where the choice set sizes are greater than two. The optimal designs are obtained under the main effects models and the…

Methodology · Statistics 2015-10-28 Soumen Manna , Ashish Das

Broadening eligibility criteria in cancer trials has been advocated to represent the true patient population more accurately. While the advantages are clear in terms of generalizability and recruitment, novel dose-finding designs are needed…

Applications · Statistics 2023-01-12 Rebecca B. Silva , Bin Cheng , Richard D. Carvajal , Shing M. Lee

In this article, we propose a phase I-II design in two stages for the combination of molecularly targeted therapies. The design is motivated by a published case study that combines a MEK and a PIK3CA inhibitors; a setting in which higher…

Methodology · Statistics 2025-05-21 José L. Jiménez , Mourad Tighiouart

The use of `backfilling', assigning additional patients to doses deemed safe, in phase I dose-escalation studies has been used in practice to collect additional information on the safety profile, pharmacokinetics and activity of a drug.…

Applications · Statistics 2022-04-22 Helen Barnett , Oliver Boix , Dimitris Kontos , Thomas Jaki

An important objective in the development of targeted therapies is to identify the populations where the treatment under consideration has positive benefit risk balance. We consider pivotal clinical trials, where the efficacy of a treatment…

This study designs an adaptive experiment for efficiently estimating average treatment effects (ATEs). In each round of our adaptive experiment, an experimenter sequentially samples an experimental unit, assigns a treatment, and observes…

Methodology · Statistics 2024-06-21 Masahiro Kato , Akihiro Oga , Wataru Komatsubara , Ryo Inokuchi

In this paper we consider two-stage adaptive dose-response study designs, where the study design is changed at an interim analysis based on the information collected so far. In a simulation study, two approaches will be compared for these…

Methodology · Statistics 2016-02-08 Emma McCallum , Björn Bornkamp

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

In an order-of-addition experiment, each treatment is a permutation of m components. It is often unaffordable to test all the m! treatments, and the design problem arises. We consider a model that incorporates the order of each pair of…

Statistics Theory · Mathematics 2018-05-15 Jiayu Peng , Rahul Mukerjee , Dennis K. J. Lin

Oncology drug development starts with a dose escalation phase to find the maximal tolerable dose (MTD). Dose limiting toxicity (DLT) is the primary endpoint for dose escalation phase. Traditionally, model-based dose escalation trial designs…

Applications · Statistics 2024-04-18 Arnab Kumar Maity , Satrajit Roy Chowdhury , Ray Li , Lada Markovtsova , Roberto Bugarini

The design of experiments in psychology can often be summarized to participants reacting to stimuli. For such an experiment, the mixed effects model with crossed random effects is usually the appropriate tool to analyse the data because it…

Methodology · Statistics 2020-10-19 Jaromil Frossard , Olivier Renaud

This paper presents a quasi-sequential optimal design framework for toxicology experiments, specifically applied to sea urchin embryos. The authors propose a novel approach combining robust optimal design with adaptive, stage-based testing…

Methodology · Statistics 2025-03-04 Elvis Han Cui , Michael Collins , Jessica Munson , Weng Kee Wong

Dose-escalation trials in oncology drug development still today typically aim to identify 1-size-fits-all dose recommendations, as arbitrary quantiles of the toxicity thresholds evident in patient samples. In the late 1990s efforts to…

Category Theory · Mathematics 2025-07-03 David C. Norris
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