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The sample size of a clinical trial relies on information about nuisance parameters such as the outcome variance. When no or only limited information is available, it has been proposed to include an internal pilot study in the design of the…

Applications · Statistics 2017-07-18 Tobias Mütze , Tim Friede

Accurate power and sample size estimation are crucial to the design and analysis of genetic association studies. When analyzing a binary trait via logistic regression, important covariates such as age and sex are typically included in the…

Methodology · Statistics 2022-10-05 Ziang Zhang , Lei Sun

Platform trials gained popularity during the last few years as they increase flexibility compared to multi-arm trials by allowing new experimental arms entering when the trial already started. Using a shared control group in platform trials…

Basket trials can efficiently evaluate a single treatment across multiple diseases with a common shared target. Prior methods for randomized basket trials required baskets to have the same sample and effect sizes. To that end, we developed…

Methodology · Statistics 2024-11-22 Sahil S. Patel , Desmond Zeya Chen , David Castle , Clement Ma

Time-to-event endpoints show an increasing popularity in phase II cancer trials. The standard statistical tool for such one-armed survival trials is the one-sample log-rank test. Its distributional properties are commonly derived in the…

Methodology · Statistics 2026-03-02 Moritz Fabian Danzer , Andreas Faldum , Rene Schmidt

The purpose of this paper is to investigate and develop methods for analysis of multi-center randomized clinical trials which only rely on the randomization process as a basis of inference. Our motivation is prompted by the fact that most…

Applications · Statistics 2008-07-28 Lu Zheng , Marvin Zelen

We automate soft robotic hand design iteration by co-optimizing design and control policy for dexterous manipulation skills in simulation. Our design iteration pipeline combines genetic algorithms and policy transfer to learn control…

Robotics · Computer Science 2024-06-27 Pragna Mannam , Xingyu Liu , Ding Zhao , Jean Oh , Nancy Pollard

Experimental design is an approach for selecting samples among a given set so as to obtain the best estimator for a given criterion. In the context of linear regression, several optimal designs have been derived, each associated with a…

Statistics Theory · Mathematics 2021-01-01 Geovani Rizk , Igor Colin , Albert Thomas , Moez Draief

One-shot decision making is required in situations in which we can evaluate a fixed number of solution candidates but do not have any possibility for further, adaptive sampling. Such settings are frequently encountered in neural network…

Neural and Evolutionary Computing · Computer Science 2019-12-23 Jakob Bossek , Pascal Kerschke , Aneta Neumann , Frank Neumann , Carola Doerr

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

Exploration of treatment effect heterogeneity (TEH) is an increasingly important aspect of modern statistical analysis for stratified medicine in randomised controlled trials (RCTs) as we start to gather more information on trial…

Methodology · Statistics 2019-01-14 Chieh-Hsi Wu , Chris C. Holmes

Platform trials are multi-arm designs that simultaneously evaluate multiple treatments for a single disease within the same overall trial structure. Unlike traditional randomized controlled trials, they allow treatment arms to enter and…

Methodology · Statistics 2024-12-16 Michele Santacatterina , Federico Macchiavelli Giron , Xinyi Zhang , Ivan Diaz

This article studies the benefits of using spatially randomized experimental designs which partition the experimental area into distinct, non-overlapping units with treatments assigned randomly. Such designs offer improved policy evaluation…

Statistics Theory · Mathematics 2025-11-18 Ying Yang , Chengchun Shi , Fang Yao , Shouyang Wang , Hongtu Zhu

We consider the problem of designing a randomized experiment on a source population to estimate the Average Treatment Effect (ATE) on a target population. We propose a novel approach which explicitly considers the target when designing the…

Methodology · Statistics 2021-09-07 My Phan , David Arbour , Drew Dimmery , Anup B. Rao

Clinical prediction models are estimated using a sample of limited size from the target population, leading to uncertainty in predictions, even when the model is correctly specified. Generally, not all patient profiles are observed…

Methodology · Statistics 2024-02-09 Doranne Thomassen , Saskia le Cessie , Hans van Houwelingen , Ewout Steyerberg

This paper considers the estimation of treatment effects in randomized experiments with complex experimental designs, including cases with interference between units. We develop a design-based estimation theory for arbitrary experimental…

Econometrics · Economics 2025-05-27 Haoge Chang

In this article, we develop methods for sample size and power calculations in four-level intervention studies when intervention assignment is carried out at any level, with a particular focus on cluster randomized trials (CRTs). CRTs…

Methodology · Statistics 2022-09-07 Xueqi Wang , Elizabeth L. Turner , John S. Preisser , Fan Li

The effective sample size (ESS) is widely used in sample-based simulation methods for assessing the quality of a Monte Carlo approximation of a given distribution and of related integrals. In this paper, we revisit the approximation of the…

Computation · Statistics 2022-04-14 Víctor Elvira , Luca Martino , Christian P. Robert

There is growing interest in a hybrid control design in which a randomized controlled trial is augmented with an external control arm from a previous trial or real world data. Existing methods for analyzing hybrid control studies include…

Methodology · Statistics 2025-01-30 Zhiwei Zhang , Jialuo Liu , Wei Liu

Sample size calculations play a central role in study design because sample size affects study interpretability, costs, hospital resources, and staff time. For most veterinary orthopaedic risk-factor studies, either the sample size…

Applications · Statistics 2023-10-10 Richard Evans , Antonio Pozzi
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