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Bayesian adaptive designs have gained popularity in all phases of clinical trials with numerous new developments in the past few decades. During the COVID-19 pandemic, the need to establish evidence for the effectiveness of vaccines,…

Methodology · Statistics 2022-03-08 Shirin Golchi

Randomized clinical trials are considered the gold standard for estimating causal effects. Nevertheless, in studies that are aimed at examining adverse effects of interventions, such trials are often impractical because of ethical and…

Methodology · Statistics 2020-01-20 Anthony D. Scotina , Andrew R. Zullo , Robert J. Smith , Roee Gutman

We propose to use Bayesian optimization (BO) to improve the efficiency of the design selection process in clinical trials. BO is a method to optimize expensive black-box functions, by using a regression as a surrogate to guide the search.…

Methodology · Statistics 2021-05-20 Jakob Richter , Tim Friede , Jörg Rahnenführer

Basket trials in oncology enroll multiple patients with cancer harboring identical gene alterations and evaluate their response to targeted therapies across cancer types. Several existing methods have extended a Bayesian hierarchical model…

Methodology · Statistics 2024-12-17 Ryo Kitabayashi , Hiroyuki Sato , Akihiro Hirakawa

The power prior is a popular class of informative priors for incorporating information from historical data. It involves raising the likelihood for the historical data to a power, which acts as a discounting parameter. When the discounting…

Methodology · Statistics 2025-05-26 Yueqi Shen , Matthew A. Psioda , Luiz M. Carvalho , Joseph G. Ibrahim

Commonly, clinical trials report effects not only for the full study population but also for patient subgroups. Meta-analyses of subgroup-specific effects and treatment-by-subgroup interactions may be inconsistent, especially when trials…

Methodology · Statistics 2025-12-23 Renato Panaro , Christian Röver , Tim Friede

When incorporating historical control data into the analysis of current randomized controlled trial data, it is critical to account for differences between the datasets. When the cause of the difference is an unmeasured factor and…

Methodology · Statistics 2025-09-10 Tomohiro Ohigashi , Kazushi Maruo , Takashi Sozu , Masahiko Gosho

Efficient design of genomic perturbation experiments is crucial for accelerating drug discovery and therapeutic target identification, yet exhaustive perturbation of the human genome remains infeasible due to the vast search space of…

Machine Learning · Statistics 2026-03-24 Yanke Li , Tianyu Cui , Tommaso Mansi , Mangal Prakash , Rui Liao

In recent years, basket trials, which allow the evaluation of an experimental therapy across multiple tumor types within a single protocol, have gained prominence in early-phase oncology development. Unlike traditional trials, which…

Applications · Statistics 2025-07-18 Haiming Zhou , Rex Shen , Sutan Wu , Philip He

Incorporating historical data or real-world evidence has a great potential to improve the efficiency of phase I clinical trials and to accelerate drug development. For model-based designs, such as the continuous reassessment method (CRM),…

Methodology · Statistics 2020-06-11 Yanhong Zhou , J. Jack Lee , Shunguang Wang , Stuart Bailey , Ying Yuan

Optimal sampling strategies are critical for surveys of deeper coral reef and shoal systems, due to the significant cost of accessing and field sampling these remote and poorly understood ecosystems. Additionally, well-established standard…

Methodology · Statistics 2022-08-31 Dilishiya De Silva , Rebecca Fisher , Ben Radford , Helen Thompson , James McGree

Group therapy is a central treatment modality for behavioral health disorders such as alcohol and other drug use (AOD) and depression. Group therapy is often delivered under a rolling (or open) admissions policy, where new clients are…

Applications · Statistics 2011-08-02 Susan M. Paddock , Sarah B. Hunter , Katherine E. Watkins , Daniel F. McCaffrey

Gene therapies aim to address the root causes of diseases, particularly those stemming from rare genetic defects that can be life-threatening or severely debilitating. Although an increasing number of gene therapies have received regulatory…

Methodology · Statistics 2026-01-19 Tianyu Pan , Yiyao Shi , Xiang Zhang , Weining Shen , Ting Ye

Meta-analysis is widely used to integrate results from multiple experiments to obtain generalized insights. Since meta-analysis datasets are often heteroscedastic due to varying subgroups and temporal heterogeneity arising from experiments…

Methodology · Statistics 2026-01-19 Kohsuke Kubota , Shonosuke Sugasawa , Keiichi Ochiai , Takahiro Hoshino

Active learning is usually applied to acquire labels of informative data points in supervised learning, to maximize accuracy in a sample-efficient way. However, maximizing the accuracy is not the end goal when the results are used for…

Machine Learning · Statistics 2021-10-22 Louis Filstroff , Iiris Sundin , Petrus Mikkola , Aleksei Tiulpin , Juuso Kylmäoja , Samuel Kaski

Adaptive experimentation under unknown network interference requires solving two coupled problems: (i) learning the underlying dynamics of interference among units and (ii) using these dynamics to inform treatment allocation in order to…

Machine Learning · Statistics 2026-05-13 Aidan Gleich , Eric Laber , Alexander Volfovsky

We consider problems of parameter estimation where design variables can be actively optimized to maximize information gain. To this end, we introduce JADAI, a framework that jointly amortizes Bayesian adaptive design and inference by…

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 use of external data in clinical trials offers numerous advantages, such as reducing the number of patients, increasing study power, and shortening trial durations. In Bayesian inference, information in external data can be transferred…

Methodology · Statistics 2025-09-17 Xuetao Lu , J. Jack Lee

Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as…

Methodology · Statistics 2026-04-16 Qinwei Yang , Jingyi Li , Peng Wu , Shu Yang
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