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The Bayesian expected power (BEP) has become increasingly popular in sample size determination and assessment of the probability of success (POS) for a future trial. The BEP takes into consideration the uncertainty around the parameters…

Methodology · Statistics 2020-07-01 Fang Liu

Adaptive enrichment trials aim to identify and recruit participants most likely to benefit from treatment based on evolving biomarker evidence, with the goal of informing individualized treatment recommendations. Bayesian methods are well…

Methodology · Statistics 2026-03-11 Lara Maleyeff , Shirin Golchi , Erica E. M. Moodie

Power and sample size analysis comprises a critical component of clinical trial study design. There is an extensive collection of methods addressing this problem from diverse perspectives. The Bayesian paradigm, in particular, has attracted…

Methodology · Statistics 2021-12-08 Jane Pan , Sudipto Banerjee

Targeted therapies on the basis of genomic aberrations analysis of the tumor have shown promising results in cancer prognosis and treatment. Regardless of tumor type, trials that match patients to targeted therapies for their particular…

Applications · Statistics 2018-04-18 Yanxun Xu , Peter Mueller , Apostolia M Tsimberidou , Donald Berry

The BayesPPDSurv (Bayesian Power Prior Design for Survival Data) R package supports Bayesian power and type I error calculations and model fitting using the power and normalized power priors incorporating historical data with for the…

Methodology · Statistics 2024-04-09 Yueqi Shen , Matthew A. Psioda , Joseph G. Ibrahim

External controls from historical trials or observational data can augment randomized controlled trials when large-scale randomization is impractical or unethical, such as in drug evaluation for rare diseases. However, non-randomized…

Methodology · Statistics 2025-05-08 Ke Zhu , Shu Yang , Xiaofei Wang

Basket trial designs are a type of master protocol in which the same therapy is tested in several strata of the patient cohort. Many basket trial designs implement borrowing mechanisms. These allow sharing information between similar strata…

Methodology · Statistics 2024-05-20 Lukas D Sauer , Alexander Ritz , Meinhard Kieser

We propose to use Bayesian optimization (BO) to improve the efficiency of the design selection process in clinical trials. BO is a method to optimize expensive black-box functions, by using a regression as a surrogate to guide the search.…

Methodology · Statistics 2021-05-20 Jakob Richter , Tim Friede , Jörg Rahnenführer

Recent substantial advances of molecular targeted oncology drug development is requiring new paradigms for early-phase clinical trial methodologies to enable us to evaluate efficacy of several subtypes simultaneously and efficiently. The…

Methodology · Statistics 2023-02-17 Satoshi Hattori , Satoshi Morita

Heterogeneity in efficacy is sometimes observed across baskets in basket trials. In this study, we propose a model-free clustering framework that groups baskets based on transition probabilities derived from the trajectories of treatment…

Methodology · Statistics 2026-01-05 Masahiro Kojima , Keisuke Hanada , Atsuya Sato

There is currently a focus on statistical methods which can use historical trial information to help accelerate the discovery, development and delivery of medicine. Bayesian methods can be constructed so that the borrowing is "dynamic" in…

Methodology · Statistics 2024-09-13 Darren A. V. Scott , Alex Lewin

Physical experiments in the national security domain are often expensive and time-consuming. Test engineers must certify the compatibility of aircraft and their weapon systems before they can be deployed in the field, but the testing…

Methodology · Statistics 2023-10-02 Daniel Ries , Victoria R. C. Sieck , Philip Jones , Julie Shaffer

Incorporating historical information into the design and analysis of a new clinical trial has been the subject of much recent discussion. For example, in the context of clinical trials of antibiotics for drug resistant infections, where…

Methodology · Statistics 2018-06-08 Isaac Gravestock , Leonhard Held

An early phase clinical trial is the first step in evaluating the effects in humans of a potential new anti-disease agent or combination of agents. Usually called "phase I" or "phase I/II" trials, these experiments typically have the…

Methodology · Statistics 2010-12-01 Peter F. Thall

We propose a restricted win probability estimand for comparing treatments in a randomized trial with a time-to-event outcome. We also propose Bayesian estimators for this summary measure as well as the unrestricted win probability. Bayesian…

Methodology · Statistics 2024-11-06 Michelle Leeberg , Xianghua Luo , Thomas A. Murray

In current clinical trial development, historical information is receiving more attention as it provides utility beyond sample size calculation. Meta-analytic-predictive (MAP) priors and robust MAP priors have been proposed for…

Methodology · Statistics 2022-08-03 Tianyu Zhan , Yiwang Zhou , Ziqian Geng , Yihua Gu , Jian Kang , Li Wang , Xiaohong Huang , Elizabeth H. Slate

Optimal design of a Phase I cancer trial can be formulated as a stochastic optimization problem. By making use of recent advances in approximate dynamic programming to tackle the problem, we develop an approximation of the Bayesian optimal…

Methodology · Statistics 2010-12-01 Jay Bartroff , Tze Leung Lai

We propose a multi-metric flexible Bayesian framework to support efficient interim decision-making in multi-arm multi-stage phase II clinical trials. Multi-arm multi-stage phase II studies increase the efficiency of drug development, but…

Applications · Statistics 2023-12-15 Suzanne M. Dufault , Angela M. Crook , Katie Rolfe , Patrick P. J. Phillips

We develop an empirical Bayes framework for experimental design that leverages information from prior related studies. When a researcher has access to estimates from previous studies on similar parameters, they can use empirical Bayes to…

Econometrics · Economics 2026-02-25 Zhiheng You

The optimization of atomic structures plays a pivotal role in understanding and designing materials with desired properties. However, conventional computational methods often struggle with the formidable task of navigating the vast…

Materials Science · Physics 2024-11-19 Peder Lyngby , Casper Larsen , Karsten Wedel Jacobsen