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Introduction: Increasing interest in real-world evidence has fueled the development of study designs incorporating real-world data (RWD). Using the Causal Roadmap, we specify three designs to evaluate the difference in risk of major adverse…

Contextual sensing and delivery of digital interventions to improve health outcomes have gained significant traction in behavioral and psychiatric studies. Micro-randomized trials (MRTs) are a common experimental design for obtaining…

Methodology · Statistics 2025-04-01 Jieru Shi , Zhenke Wu , Walter Dempsey

The analysis of platform trials can be enhanced by utilizing non-concurrent controls. Since including this data might also introduce bias in the treatment effect estimators if time trends are present, methods for incorporating…

Methodology · Statistics 2026-05-21 Pavla Krotka , Martin Posch , Marta Bofill Roig

Real-time control systems often require dedicated hardware and software, including real-time operating systems, while many systems are available for off-line computing, mainly based on standard system units (PCs), standard network…

Instrumentation and Detectors · Physics 2007-05-23 F. Acernese , F. Barone , R. De Rosa , R. Esposito , P. Mastroserio , L. Milano , S. Pardi , K. Qipiani , F. Silvestri , G. Spadaccini

We aim to generalize the results of a randomized controlled trial (RCT) to a target population with the help of some observational data. This is a problem of causal effect identification with multiple data sources. Challenges arise when the…

Methodology · Statistics 2022-06-15 Juha Karvanen

Uncertainty quantification is necessary for developers, physicians, and regulatory agencies to build trust in machine learning predictors and improve patient care. Beyond measuring uncertainty, it is crucial to express it in clinically…

Computer Vision and Pattern Recognition · Computer Science 2025-03-04 Jacopo Teneggi , J Webster Stayman , Jeremias Sulam

Generation of realistic synthetic data has garnered considerable attention in recent years, particularly in the health research domain due to its utility in, for instance, sharing data while protecting patient privacy or determining optimal…

Other Statistics · Statistics 2025-01-30 Niki Z. Petrakos , Erica E. M. Moodie , Nicolas Savy

Randomized discontinuation design (RDD) is an enrichment strategy commonly used to address limitations of traditional placebo-controlled trials, particularly the ethical concern of prolonged placebo exposure. RDD consists of two phases: an…

Methodology · Statistics 2025-06-03 Ayon Mukherjee , Oleksandr Sverdlov , Ngoc-Thuy Ha , Yu Deng

In cluster-randomized trials, generalized linear mixed models and generalized estimating equations have conventionally been the default analytic methods for estimating the average treatment effect as routine practice. However, recent…

Methodology · Statistics 2025-09-19 Fan Li , Jiaqi Tong , Xi Fang , Chao Cheng , Brennan C. Kahan , Bingkai Wang

Clinical risk prediction is a valuable tool for guiding healthcare interventions toward those most likely to benefit. Yet, evaluating the pairing of a risk prediction model with an intervention using randomized controlled trials presents…

Methodology · Statistics 2025-10-31 Valerie Odeh-Couvertier , Gabriel Zayas-Caban , Brian Patterson , Amy Cochran

The identification of similar patient pathways is a crucial task in healthcare analytics. A flexible tool to address this issue are parametric competing risks models, where transition intensities may be specified by a variety of parametric…

Methodology · Statistics 2024-01-10 Kathrin Möllenhoff , Nadine Binder , Holger Dette

The Linked Clinical Trials (LinkedCT) project aims at publishing the first open semantic web data source for clinical trials data. The database exposed by LinkedCT is generated by (1) transforming existing data sources of clinical trials…

Databases · Computer Science 2009-08-06 Oktie Hassanzadeh , Anastasios Kementsietsidis , Lipyeow Lim , Renee J. Miller , Min Wang

The process of matching patients with suitable clinical trials is essential for advancing medical research and providing optimal care. However, current approaches face challenges such as data standardization, ethical considerations, and a…

Computation and Language · Computer Science 2023-08-08 Jiayi Yuan , Ruixiang Tang , Xiaoqian Jiang , Xia Hu

As data shift or new data become available, updating clinical machine learning models may be necessary to maintain or improve performance over time. However, updating a model can introduce compatibility issues when the behavior of the…

Machine Learning · Statistics 2023-08-11 Erkin Ötleş , Brian T. Denton , Jenna Wiens

Although randomized controlled trials (RCTs) are a cornerstone of comparative effectiveness, they typically have much smaller sample size than observational studies because of financial and ethical considerations. Therefore there is…

Methodology · Statistics 2023-11-16 Lauren D. Liao , Emilie Højbjerre-Frandsen , Alan E. Hubbard , Alejandro Schuler

Randomized controlled trials (RCTs) frequently utilize covariate-adaptive randomization (CAR) (e.g., stratified block randomization) and commonly suffer from imperfect compliance. This paper studies the identification and inference for the…

Econometrics · Economics 2025-05-02 Federico A. Bugni , Mengsi Gao , Filip Obradovic , Amilcar Velez

Random testing (RT) is a well-studied testing method that has been widely applied to the testing of many applications, including embedded software systems, SQL database systems, and Android applications. Adaptive random testing (ART) aims…

Software Engineering · Computer Science 2020-07-15 Rubing Huang , Weifeng Sun , Yinyin Xu , Haibo Chen , Dave Towey , Xin Xia

Platform trials can evaluate the efficacy of several treatments compared to a control. The number of treatments is not fixed, as arms may be added or removed as the trial progresses. Platform trials are more efficient than independent…

There is growing interest in a hybrid control design for treatment evaluation, where a randomized controlled trial is augmented with external control data from a previous trial or a real world data source. The hybrid control design has the…

Methodology · Statistics 2026-05-06 Zhiwei Zhang , Peisong Han , Wei Zhang

Generalizability and transportability methods have been proposed to address the external validity bias of randomized clinical trials that results from differences in the distribution of treatment effect modifiers between trial and target…