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The partial effectiveness of drugs is of importance to the pharmaceutical industry. Randomized controlled trials (RCTs) assuming the existence of a subgroup sensitive to the treatment are already used. These designs, however, are available…

Applications · Statistics 2018-12-07 Daria Rukina

There is a growing interest in the implementation of platform trials, which provide the flexibility to incorporate new treatment arms during the trial and the ability to halt treatments early based on lack of benefit or observed…

Methodology · Statistics 2023-08-25 Peter Greenstreet , Thomas Jaki , Alun Bedding , Pavel Mozgunov

A multi-arm multi-stage trial is a multi-arm trial which includes interim analyses - analysing the data at certain specified points, generally discontinuing treatments which are concluded to not work and proceeding with the remainder. It is…

Methodology · Statistics 2022-11-14 Martin Law

The treatment assignment mechanism in a randomized clinical trial can be optimized for statistical efficiency within a specified class of randomization mechanisms. Optimal designs of this type have been characterized in terms of the…

Methodology · Statistics 2025-09-03 Wei Zhang , Zhiwei Zhang , Aiyi Liu

Finding an effective medical treatment often requires a search by trial and error. Making this search more efficient by minimizing the number of unnecessary trials could lower both costs and patient suffering. We formalize this problem as…

Machine Learning · Computer Science 2021-02-18 Samuel Håkansson , Viktor Lindblom , Omer Gottesman , Fredrik D. Johansson

Treatment effect heterogeneity refers to the systematic variation in treatment effects across subgroups. There is an increasing need for clinical trials that aim to investigate treatment effect heterogeneity and estimate subgroup-specific…

Methodology · Statistics 2026-03-06 Xianglin Zhao , Shirin Golchi , Jean-Philippe Gouin , Kaberi Dasgupta

A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments, where patients are often assigned to different subsets of treatment arms based on individual characteristics, enrollment…

Clinical trials (CTs) often fail due to inadequate patient recruitment. This paper tackles the challenges of CT retrieval by presenting an approach that addresses the patient-to-trials paradigm. Our approach involves two key components in a…

Information Retrieval · Computer Science 2023-07-04 Wojciech Kusa , Óscar E. Mendoza , Petr Knoth , Gabriella Pasi , Allan Hanbury

We propose a multiple cohort expansion (MUCE) approach as a design or analysis method for phase 1b multiple expansion cohort trials, which are novel first-in-human studies conducted following phase 1a dose escalation. The MUCE design is…

Methodology · Statistics 2025-05-21 Jiaying Lyu , Tianjian Zhou , Shijie Yuan , Wentian Guo , Yuan Ji

In intensity-modulated radiation therapy, optimal intensity distributions of incoming beams are decomposed into linear combinations of leaf openings of a multileaf collimator (segments). In order to avoid inefficient dose delivery, the…

Medical Physics · Physics 2010-09-29 Antje Kiesel , Tobias Gauer

Identifying relationships between molecular variations and their clinical presentations has been challenged by the heterogeneous causes of a disease. It is imperative to unveil the relationship between the high dimensional molecular…

Methodology · Statistics 2021-09-02 Wennan Chang , Changlin Wan , Yong Zang , Chi Zhang , Sha Cao

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

Methodology · Statistics 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

We present two approaches to system identification, i.e. the identification of partial differential equations (PDEs) from measurement data. The first is a regression-based Variational System Identification procedure that is advantageous in…

Computational Physics · Physics 2024-03-28 Zhenlin Wang , Bowei Wu , Krishna Garikipati , Xun Huan

For many kinds of interventions, such as a new advertisement, marketing intervention, or feature recommendation, it is important to target a specific subset of people for maximizing its benefits at minimum cost or potential harm. However, a…

Methodology · Statistics 2020-11-12 Yanbo Xu , Divyat Mahajan , Liz Manrao , Amit Sharma , Emre Kiciman

Online system identification algorithms are widely used for monitoring, diagnostics and control by continuously adapting to time-varying dynamics. Typically, these algorithms consider a model structure that lacks parsimony and offers…

Systems and Control · Electrical Eng. & Systems 2025-04-28 Koen Classens , Rodrigo A. González , Tom Oomen

Distribution shifts remain a fundamental problem for the safe application of machine learning systems. If undetected, they may impact the real-world performance of such systems or will at least render original performance claims invalid. In…

Machine Learning · Computer Science 2023-03-10 Lisa M. Koch , Christian M. Schürch , Christian F. Baumgartner , Arthur Gretton , Philipp Berens

In this study, we propose an innovative dynamic classification algorithm aimed at achieving zero missed detections and minimal false positives,acritical in safety-critical domains (e.g., medical diagnostics) where undetected cases risk…

Machine Learning · Computer Science 2025-06-02 Ziyuan Zhong , Junyang Zhou

When developing clinical prediction models, it can be challenging to balance between global models that are valid for all patients and personalized models tailored to individuals or potentially unknown subgroups. To aid such decisions, we…

The problem of subgroups is ubiquitous in scientific research (ex. disease heterogeneity, spatial distributions in ecology...), and piecewise regression is one way to deal with this phenomenon. Morse-Smale regression offers a way to…

Machine Learning · Statistics 2017-08-22 Colleen M. Farrelly

Targeted therapies based on biomarker profiling are becoming a mainstream direction of cancer research and treatment. Depending on the expression of specific prognostic biomarkers, targeted therapies assign different cancer drugs to…

Applications · Statistics 2015-03-24 Yanxun Xu , Lorenzo Trippa , Peter Müller , Yuan Ji