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The use of Bayesian adaptive designs for randomised controlled trials has been hindered by the lack of software readily available to statisticians. We have developed a new software package (Bayesian Adaptive Trials Simulator Software -…

Background: Clinical prediction models are increasingly used to inform healthcare decisions, but determining the minimum sample size for their development remains a critical and unresolved challenge. Inadequate sample sizes can lead to…

Machine Learning · Computer Science 2026-03-02 Diana Shamsutdinova , Felix Zimmer , Oyebayo Ridwan Olaniran , Sarah Markham , Daniel Stahl , Gordon Forbes , Ewan Carr

Background: Determining an adequate sample size is essential for developing reliable and generalisable clinical prediction models, yet practical guidance on selecting appropriate methods remains limited. Existing analytical and…

Adaptive seamless designs combine confirmatory testing, a domain of phase III trials, with features such as treatment or subgroup selection, typically associated with phase II trials. They promise to increase the efficiency of development…

Methodology · Statistics 2019-01-25 Tim Friede , Nigel Stallard , Nicholas Parsons

Effect size indices are useful parameters that quantify the strength of association and are unaffected by sample size. There are many available effect size parameters and estimators, but it is difficult to compare effect sizes across…

Computation · Statistics 2023-02-27 Megan Jones , Kaidi Kang , Simon Vandekar

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…

We present a bayesassurance R package that computes the Bayesian assurance under various settings characterized by different assumptions and objectives. The package offers a constructive set of simulation-based functions suitable for…

Methodology · Statistics 2022-03-30 Jane Pan , Sudipto Banerjee

Dose-finding trials are a key component of the drug development process and rely on a statistical design to help inform dosing decisions. Triallists wishing to choose a design require knowledge of operating characteristics of competing…

Computation · Statistics 2025-03-11 Michael Sweeting , Daniel Slade , Dan Jackson , Kristian Brock

It is shown how to set up, conduct, and analyze large simulation studies with the new R package simsalapar = simulations simplified and launched parallel. A simulation study typically starts with determining a collection of input variables…

Computation · Statistics 2013-09-18 Marius Hofert , Martin Mächler

An important issue for many economic experiments is how the experimenter can ensure sufficient power for rejecting one or more hypotheses. Here, we apply methods developed mainly within the area of clinical trials for testing multiple…

Methodology · Statistics 2021-08-06 Sebastian Jobjörnsson , Henning Schaak , Oliver Mußhoff , Tim Friede

Adaptive designs have been proposed for clinical trials in which the nuisance parameters or alternative of interest are unknown or likely to be misspecified before the trial. Whereas most previous works on adaptive designs and mid-course…

Methodology · Statistics 2011-05-18 Jay Bartroff , Tze Leung Lai

We propose BaySize, a sample size calculator for phase I clinical trials using Bayesian models. BaySize applies the concept of effect size in dose finding, assuming the MTD is defined based on an equivalence interval. Leveraging a decision…

Methodology · Statistics 2023-03-29 Xiaolei Lin , Jiaying Lyu , Shijie Yuan , Sue-Jane Wang , Yuan Ji

Composite endpoints are widely used as primary endpoints in clinical trials. Designing trials with time-to-event endpoints can be particularly challenging because the proportional hazard assumption usually does not hold when using a…

Methodology · Statistics 2022-11-07 Jordi Cortés Martinez , Marta Bofill Roig , Guadalupe Gómez Melis

Existing approaches to sample size calculations for developing clinical prediction models have focused on ensuring that the expected value of a chosen performance measure meets a pre-specified target. For example, to limit…

Methodology · Statistics 2025-09-18 Menelaos Pavlou , Rumana Z. Omar , Gareth Ambler

Background: Advanced adaptive randomised clinical trials are increasingly used. Compared to their conventional counterparts, their flexibility may make them more efficient, increase the probability of obtaining conclusive results without…

Adaptive sample size re-estimation, early stopping, and trial re-design at interim analyses can reduce expected sample sizes in randomised trials. Cluster randomised trials, in which groups of participants are randomly allocated to…

Methodology · Statistics 2026-03-09 Samuel I. Watson , James Martin

Phase-3 clinical trials provide the highest level of evidence on drug safety and effectiveness needed for market approval by implementing large randomized controlled trials (RCTs). However, 30-40% of these trials fail mainly because such…

Applications · Statistics 2024-01-09 Sayeri Lala , Niraj K. Jha

Compressed Sensing (CS) facilitates rapid image acquisition by selecting a small subset of measurements sufficient for high-fidelity reconstruction. Adaptive CS seeks to further enhance this process by dynamically choosing future…

Image and Video Processing · Electrical Eng. & Systems 2024-07-12 Noam Elata , Tomer Michaeli , Michael Elad

The interAdapt R package is designed to be used by statisticians and clinical investigators to plan randomized trials. It can be used to determine if certain adaptive designs offer tangible benefits compared to standard designs, in the…

Applications · Statistics 2014-06-19 Aaron Fisher , Harris Jaffee , Michael Rosenblum

Stochastic gradient decent~(SGD) and its variants, including some accelerated variants, have become popular for training in machine learning. However, in all existing SGD and its variants, the sample size in each iteration~(epoch) of…

Machine Learning · Statistics 2019-09-18 Shen-Yi Zhao , Hao Gao , Wu-Jun Li
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