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The statistical efficiency of randomized clinical trials can be improved by incorporating information from baseline covariates (i.e., pre-treatment patient characteristics). This can be done in the design stage using stratified (permutated…

Methodology · Statistics 2025-02-04 Zhiwei Zhang

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

Background: trials to identify the minimal effective treatment duration are needed in different therapeutic areas, including bacterial infections, TB and Hepatitis--C. However, standard non-inferiority designs have several limitations,…

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

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

We propose a flexible design for the identification of optimal dose combinations in dual-agent dose-finding clinical trials. The design is called AAA, standing for three adaptations: adaptive model selection, adaptive dose insertion, and…

Methodology · Statistics 2017-06-13 Jiaying Lyu , Yuan Ji , Naiqing Zhao , Daniel V. T. Catenacci

Randomized controlled trials (RCTs) can be used to generate guarantees on treatment effects. However, RCTs often spend unnecessary resources exploring sub-optimal treatments, which can reduce the power of treatment guarantees. To address…

Computers and Society · Computer Science 2024-10-16 Santiago Cortes-Gomez , Naveen Raman , Aarti Singh , Bryan Wilder

Optimal design of a Phase I cancer trial can be formulated as a stochastic optimization problem. By making use of recent advances in approximate dynamic programming to tackle the problem, we develop an approximation of the Bayesian optimal…

Methodology · Statistics 2010-12-01 Jay Bartroff , Tze Leung Lai

Externally controlled trials (ECTs) are increasingly used when randomized controls are infeasible, unethical, or insufficient, including applications in rare diseases, oncology, pediatrics, and post-approval effectiveness research. Although…

Individualized treatment rules (ITRs) tailor treatments according to individual patient characteristics. They can significantly improve patient care and are thus becoming increasingly popular. The data collected during randomized clinical…

Methodology · Statistics 2015-06-30 Stanislav Minsker , Ying-Qi Zhao , Guang Cheng

There is growing interest in using machine learning (ML) to support clinical diagnosis, but most approaches rely on static, fully observed datasets and fail to reflect the sequential, resource-aware reasoning clinicians use in practice.…

Artificial Intelligence · Computer Science 2025-11-18 Silas Ruhrberg Estévez , Nicolás Astorga , Mihaela van der Schaar

Significant evidence has become available that emphasizes the importance of personalization in medicine. In fact, it has become a common belief that personalized medicine is the future of medicine. The core of personalized medicine is the…

Methodology · Statistics 2020-04-30 Qiong Zhang , Amin Khademi , Yongjia Song

This paper presents adaptive conformal selection (ACS), an interactive framework for model-free selection with guaranteed error control. Building on conformal selection (Jin and Cand\`es, 2023b), ACS generalizes the approach to support…

Methodology · Statistics 2025-07-22 Yu Gui , Ying Jin , Yash Nair , Zhimei Ren

We propose an adaptive sequential framework for testing two simple hypotheses that analytically ensures finite exposure to the less effective treatment. Our proposed procedure employs a likelihood ratio-driven adaptive allocation rule,…

Statistics Theory · Mathematics 2025-11-26 Sampurna Kundu , Jayant Jha , Subir Kumar Bhandari

With the increase of research in self-adaptive systems, there is a need to better understand the way research contributions are evaluated. Such insights will support researchers to better compare new findings when developing new knowledge…

Software Engineering · Computer Science 2021-03-23 Ilias Gerostathopoulos , Thomas Vogel , Danny Weyns , Patricia Lago

Covariate adjustment aims to improve the statistical efficiency of randomized trials by incorporating information from baseline covariates. Popular methods for covariate adjustment include analysis of covariance for continuous endpoints and…

Methodology · Statistics 2025-05-09 Zhiwei Zhang , Ya Wang , Dong Xi

Clinical trials often collect data on multiple outcomes, such as overall survival (OS), progression-free survival (PFS), and response to treatment (RT). In most cases, however, study designs only use primary outcome data for interim and…

Applications · Statistics 2026-04-28 Massimiliano Russo , Steffen Ventz , Lorenzo Trippa

In the domain of assistive robotics, the significance of effective modeling is well acknowledged. Prior research has primarily focused on enhancing model accuracy or involved the collection of extensive, often impractical amounts of data.…

Robotics · Computer Science 2024-06-07 Hamid Osooli , Christopher Coco , Johnathan Spanos , Amin Majdi , Reza Azadeh

In this paper, a reinforced soft robot prototype with a custom-designed actuator-space string encoder are created to investigate dynamic soft robotic trajectory tracking. The soft robot prototype embedded with the proposed adaptive…

Robotics · Computer Science 2022-09-26 Milad Azizkhani , Anthony L. Gunderman , Isuru S. Godage , Yue Chen

Background: Clinical prediction models are increasingly used to inform healthcare decisions, but determining the minimum sample size for their development remains a critical and unresolved challenge. Inadequate sample sizes can lead to…

Machine Learning · Computer Science 2026-03-02 Diana Shamsutdinova , Felix Zimmer , Oyebayo Ridwan Olaniran , Sarah Markham , Daniel Stahl , Gordon Forbes , Ewan Carr