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Identifying variables associated with clinical endpoints is of much interest in clinical trials. With the rapid growth of cell and gene therapy (CGT) and therapeutics for ultra-rare diseases, there is an urgent need for statistical methods…

Methodology · Statistics 2025-11-18 Lingxuan Kong , Yumin Zhang , Chenkun Wang , Yaoyuan Vincent Tan

We develop an empirical Bayes procedure for estimating the cell means in an unbalanced, two-way additive model with fixed effects. We employ a hierarchical model, which reflects exchangeability of the effects within treatment and within…

Methodology · Statistics 2016-05-30 Lawrence D. Brown , Gourab Mukherjee , Asaf Weinstein

Platform trials are a more efficient way of testing multiple treatments compared to running separate trials. In this paper we consider platform trials where, if a treatment is found to be superior to the control, it will become the new…

Methodology · Statistics 2023-11-30 Peter Greenstreet , Thomas Jaki , Alun Bedding , Pavel Mozgunov

Determination of posterior probability for go-no-go decision and predictive power are becoming increasingly common for resource optimization in clinical investigation. There are vast published literature on these topics; however, the…

Methodology · Statistics 2023-02-09 Madan G. Kundu , Sandipan Samanta , Shoubhik Mondal

We develop the scale transformed power prior for settings where historical and current data involve different data types, such as binary and continuous data, respectively. This situation arises often in clinical trials, for example, when…

Methodology · Statistics 2021-05-12 Brady Nifong , Matthew A. Psioda , Joseph G. Ibrahim

We present a new approach to the problems of evaluating and learning personalized decision policies from observational data of past contexts, decisions, and outcomes. Only the outcome of the enacted decision is available and the historical…

Machine Learning · Statistics 2019-06-04 Nathan Kallus

To use historical controls for indirect comparison with single-arm trials, the population difference between data sources should be adjusted to reduce confounding bias. The adjustment is more difficult for time-to-event data with a cure…

Methodology · Statistics 2023-08-23 Jixian Wang , Hongtao Zhang , Ram Tiwari

We discuss Bayesian inference for parameters selected using the data. First, we provide a critical analysis of the existing positions in the literature regarding the correct Bayesian approach under selection. Second, we propose two types of…

Statistics Theory · Mathematics 2021-05-12 Daniel G. Rasines , G. Alastair Young

When a new treatment is considered for use, whether a pharmaceutical drug or a search engine ranking algorithm, a typical question that arises is, will its performance exceed that of the current treatment? The conventional way to answer…

Machine Learning · Computer Science 2016-10-27 Nir Rosenfeld , Yishay Mansour , Elad Yom-Tov

Prior information is often incorporated informally when planning a clinical trial. Here, we present an approach on how to incorporate prior information, such as data from historical clinical trials, into the nuisance parameter based sample…

Applications · Statistics 2019-03-08 Tobias Mütze , Heinz Schmidli , Tim Friede

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

A question of some interest is how to characterize the amount of information that a prior puts into a statistical analysis. Rather than a general characterization, we provide an approach to characterizing the amount of information a prior…

Methodology · Statistics 2012-01-10 Michael Evans , Gun Ho Jang

Research in oncology has changed the focus from histological properties of tumors in a specific organ to a specific genomic aberration potentially shared by multiple cancer types. This motivates the basket trial, which assesses the efficacy…

Applications · Statistics 2020-02-11 Jin Jin , Marie-Karelle Riviere , Xiaodong Luo , Yingwen Dong

Bayesian dynamic borrowing has become an increasingly important tool for evaluating the consistency of regional treatment effects which is a key requirement for local regulatory approval of a new drug. It helps increase the precision of…

Methodology · Statistics 2026-01-06 Xin Zhang , Hui Zhang , Satrajit Roychoudhury

Science consists on conceiving hypotheses, confronting them with empirical evidence, and keeping only hypotheses which have not yet been falsified. Under deductive reasoning they are conceived in view of a theory and confronted with…

Machine Learning · Computer Science 2022-11-04 Diego Marcondes , Adilson Simonis , Junior Barrera

Algorithms and technologies are essential tools that pervade all aspects of our daily lives. In the last decades, health care research benefited from new computer-based recruiting methods, the use of federated architectures for data…

Computers and Society · Computer Science 2023-01-26 Chiara Criscuolo , Tommaso Dolci , Mattia Salnitri

It is not always clear how to adjust for control data in causal inference, balancing the goals of reducing bias and variance. We show how, in a setting with repeated experiments, Bayesian hierarchical modeling yields an adaptive procedure…

Methodology · Statistics 2025-01-23 Andrew Gelman , Matthijs Vákár

Predicting the winner of an election is of importance to multiple stakeholders. To formulate the problem, we consider an independent sequence of categorical data with a finite number of possible outcomes in each. The data is assumed to be…

Applications · Statistics 2024-10-17 Soudeep Deb , Rishideep Roy , Shubhabrata Das

The use of patient-level information from previous studies, registries, and other external datasets can support the analysis of single-arm and randomized controlled trials to evaluate and test experimental treatments. However, the…

Methodology · Statistics 2025-10-23 Gopal Kotecha , Daniel E. Schwartz , Steffen Ventz , Lorenzo Trippa

We use the language of uninformative Bayesian prior choice to study the selection of appropriately simple effective models. We advocate for the prior which maximizes the mutual information between parameters and predictions, learning as…

Data Analysis, Statistics and Probability · Physics 2018-02-16 Henry H. Mattingly , Mark K. Transtrum , Michael C. Abbott , Benjamin B. Machta
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