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Related papers: On Bayesian Sequential Clinical Trial Designs

200 papers

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…

This paper offers a comprehensive introduction to Bayesian inference, combining historical context, theoretical foundations, and core analytical examples. Beginning with Bayes' theorem and the philosophical distinctions between Bayesian and…

Methodology · Statistics 2025-12-08 Juan Sosa , Carlos A. Martínez , Danna Cruz

The landscape of dose-finding designs for phase I clinical trials is rapidly shifting in the recent years, noticeably marked by the emergence of interval-based designs. We categorize them as the iDesigns and the IB-Designs. The iDesigns are…

Methodology · Statistics 2017-06-15 Yuan Ji , Shengjie Yang

Combining data has become an indispensable tool for managing the current diversity and abundance of data. But, as data complexity and data volume swell, the computational demands of previously proposed models for combining data escalate…

Methodology · Statistics 2024-06-13 Mario Figueira , David Conesa , Antonio López-Quílez , Iosu Paradinas

The ICH E9(R1) addendum (2019) proposed principal stratification (PS) as one of five strategies for dealing with intercurrent events. Therefore, understanding the strengths, limitations, and assumptions of PS is important for the broad…

In oncology, phase II or multiple expansion cohort trials are crucial for clinical development plans. This is because they aid in identifying potent agents with sufficient activity to continue development and confirm the proof of concept.…

Methodology · Statistics 2024-05-24 Takuya Yoshimoto , Satoru Shinoda , Kouji Yamamoto , Kouji Tahata

There is growing interest in Bayesian clinical trial designs with informative prior distributions, e.g. for extrapolation of adult data to pediatrics, or use of external controls. While the classical type I error is commonly used to…

Methodology · Statistics 2023-09-06 Nicky Best , Maxine Ajimi , Beat Neuenschwander , Gaelle Saint-Hilary , Simon Wandel

The literature for count modeling provides useful tools to conduct causal inference when outcomes take non-negative integer values. Applied to the potential outcomes framework, we link the Bayesian causal inference literature to statistical…

Methodology · Statistics 2020-08-10 Young Lee , Wicher P. Bergsma , Marie-Abele C. Bind

We consider after-study statistical inference for sequentially designed experiments wherein multiple units are assigned treatments for multiple time points using treatment policies that adapt over time. Our goal is to provide inference…

Machine Learning · Statistics 2025-06-10 Raaz Dwivedi , Katherine Tian , Sabina Tomkins , Predrag Klasnja , Susan Murphy , Devavrat Shah

In this paper we apply a two-stage sequential design to item calibration problems under a three-parameter logistic model assumption. The measurement errors of the estimates of the latent trait levels of examinees are considered in our…

Applications · Statistics 2013-05-23 Yuan-chin Ivan Chang

We propose a Bayesian Sequential procedure to test hypotheses concerning the Relative Risk between two specific treatments based on the binary data obtained from the two-arm clinical trial. Our development is based on the optimal sequential…

Methodology · Statistics 2025-04-07 Jiayue Wang , Ben Boukai

Epidemics are inherently stochastic, and stochastic models provide an appropriate way to describe and analyse such phenomena. Given temporal incidence data consisting of, for example, the number of new infections or removals in a given time…

Methodology · Statistics 2024-05-24 Sam A. Whitaker , Andrew Golightly , Colin S. Gillespie , Theodore Kypraios

Closed-loop performance of sequential decision making algorithms, such as model predictive control, depends strongly on the choice of controller parameters. Bayesian optimization allows learning of parameters from closed-loop experiments,…

Systems and Control · Electrical Eng. & Systems 2025-11-18 Sebastian Hirt , Lukas Theiner , Rolf Findeisen

Bayesian persuasion and its derived information design problem has been one of the main research agendas in the economics and computation literature over the past decade. However, when attempting to apply its model and theory, one is often…

Computer Science and Game Theory · Computer Science 2023-03-21 Bonan Ni , Weiran Shen , Pingzhong Tang

The Bayesian Mallows model is a flexible tool for analyzing data in the form of complete or partial rankings, and transitive or intransitive pairwise preferences. In many potential applications of preference learning, data arrive…

Computation · Statistics 2025-11-26 Øystein Sørensen , Anja Stein , Waldir Leoncio Netto , David S. Leslie

In this paper, we propose a general method for testing composite hypotheses. Our idea is to use confidence limits to define stopping and decision rules. The requirements of operating characteristic function can be satisfied by adjusting the…

Statistics Theory · Mathematics 2012-02-10 Xinjia Chen

We consider planning longitudinal covariate measurements in follow-up studies where covariates are time-varying. We assume that the entire cohort cannot be selected for longitudinal measurements due to financial limitations and study how a…

Methodology · Statistics 2024-07-03 Jaakko Reinikainen , Juha Karvanen

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

In many clinical trials, individuals in different subgroups have experience differential treatment effects. This leads to individualized differences in treatment benefit. In this article, we introduce the general concept of predictive…

Methodology · Statistics 2018-07-11 Debashis Ghosh , Youngjoo Cho

Background: Well-designed phase II trials must have acceptable error rates relative to a pre-specified success criterion, usually a statistically significant p-value. Such standard designs may not always suffice from a clinical perspective…

Applications · Statistics 2020-02-10 Satrajit Roychoudhury , Nicolas Scheuer , Beat Neuenschwander