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In large observational studies, the case-cohort design is commonly used to reduce the cost associated with covariate measurement. For survival outcomes, literature has suggested that the restricted mean survival time (RMST) be a more…

Methodology · Statistics 2026-05-08 Andy Ni , Wei-En Lu , Bo Lu

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…

Randomized controlled trials are not only the golden standard in medicine and vaccine trials but have spread to many other disciplines like behavioral economics, making it an important interdisciplinary tool for scientists. When designing…

Methodology · Statistics 2021-11-30 Tassilo Schwarz

Adapting the final sample size of a trial to the evidence accruing during the trial is a natural way to address planning uncertainty. Designs with adaptive sample size need to account for their optional stopping to guarantee strict type-I…

Experimental comparisons of performance represent an important aspect of research on optimization algorithms. In this work we present a methodology for defining the required sample sizes for designing experiments with desired statistical…

Neural and Evolutionary Computing · Computer Science 2018-10-16 Felipe Campelo , Fernanda Takahashi

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

The micro-randomized trial (MRT) is a sequential randomized experimental design to empirically evaluate the effectiveness of mobile health (mHealth) intervention components that may be delivered at hundreds or thousands of decision points.…

Methodology · Statistics 2021-12-14 Jieru Shi , Zhenke Wu , Walter Dempsey

Restricting randomization in the design of experiments (e.g., using blocking/stratification, pair-wise matching, or rerandomization) can improve the treatment-control balance on important covariates and therefore improve the estimation of…

Econometrics · Economics 2020-11-02 Brian Quistorff , Gentry Johnson

This paper studies non-separable models with a continuous treatment when the dimension of the control variables is high and potentially larger than the effective sample size. We propose a three-step estimation procedure to estimate the…

Methodology · Statistics 2019-03-07 Liangjun Su , Takuya Ura , Yichong Zhang

Simulation studies are computer experiments that involve creating data by pseudorandom sampling. The key strength of simulation studies is the ability to understand the behaviour of statistical methods because some 'truth' (usually some…

Methodology · Statistics 2019-01-18 Tim P Morris , Ian R White , Michael J Crowther

We address the personalized policy learning problem using longitudinal mobile health application usage data. Personalized policy represents a paradigm shift from developing a single policy that may prescribe personalized decisions by…

Methodology · Statistics 2020-01-13 Xinyu Hu , Min Qian , Bin Cheng , Ying Kuen Cheung

Clustering and dependence are common in trials. For example, in some cluster randomized trials (CRTs), pre-existing clusters are enrolled, randomized, and serve as the basis of intervention delivery. Such CRTs are "fully clustered":…

This paper considers some designs for sampling and interventions in dynamic networks and spatial temporal settings. The sample spreads through the population largely by tracing network links, although random sampling or spatial designs may…

Methodology · Statistics 2013-06-05 Steven K. Thompson

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

We study nonasymptotic (finite-sample) confidence intervals for treatment effects in randomized experiments. In the existing literature, the effective sample sizes of nonasymptotic confidence intervals tend to be looser than the…

In this article, we propose novel structural nested models to estimate causal effects of continuous treatments based on mobile health data. To find the treatment regime that optimizes the expected short-term outcomes for patients, we define…

Methodology · Statistics 2020-07-24 Liangyu Zhu , Wenbin Lu , Rui Song

Randomized clinical trials are the gold standard when estimating the average treatment effect. However, they are usually not a random sample from the real-world population because of the inclusion/exclusion rules. Meanwhile, observational…

Methodology · Statistics 2024-12-11 Kuan Jiang , Wenjie Hu , Shu Yang , Xinxing Lai , Xiaohua Zhou

We propose and create an incentive based recommendation algorithm aimed at improving the lifestyle of diabetic patients. This algorithm is integrated into a real world mobile application to provide personalized health recommendations.…

Human-Computer Interaction · Computer Science 2025-04-22 Wasim Abbas , Hafiz Syed Muhammad Bilal , Asim Abbas , Muhammad Afzal , Je-Hoon Lee

Recently, there as been an increasing interest in the use of heavily restricted randomization designs which enforces balance on observed covariates in randomized controlled trials. However, when restrictions are strict, there is a risk that…

Methodology · Statistics 2021-10-15 Mattias Nordin , Mårten Schultzberg

This article discusses the application of stochastic intervention to find the optimal treatment distribution yielding a high value of expected potential outcome under the setting where the number of treatments is allowed to vary with $n$.…

Statistics Theory · Mathematics 2026-04-22 Rohit Chaudhuri