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Multi-arm multi-stage (MAMS) trials have gained popularity to enhance the efficiency of clinical trials, potentially reducing both duration and costs. This paper focuses on designing MAMS trials where no control treatment exists. This can…

Methodology · Statistics 2025-02-12 Peter Greenstreet , Thomas Jaki , Alun Bedding , Pavel Mozgunov

Cancer is a complex disease that is characterized by uncontrolled growth and division of cells. It involves a complex interplay between genetic and environmental factors that lead to the initiation and progression of tumors. Recent advances…

Biomolecules · Quantitative Biology 2025-02-07 Reza Bozorgpour

Generative models for structure-based molecular design hold significant promise for drug discovery, with the potential to speed up the hit-to-lead development cycle, while improving the quality of drug candidates and reducing costs. Data…

Machine Learning · Statistics 2022-04-25 Lucian Chan , Rajendra Kumar , Marcel Verdonk , Carl Poelking

The integration of artificial intelligence [AI] into clinical trials has revolutionized the process of drug development and personalized medicine. Among these advancements, deep learning and predictive modelling have emerged as…

Machine Learning · Computer Science 2024-12-11 Sydney Anuyah , Mallika K Singh , Hope Nyavor

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

An important objective in the development of targeted therapies is to identify the populations where the treatment under consideration has positive benefit risk balance. We consider pivotal clinical trials, where the efficacy of a treatment…

In a sequential multiple-assignment randomized trial (SMART), a sequence of treatments is given to a patient over multiple stages. In each stage, randomization may be done to allocate patients to different treatment groups. Even though…

Methodology · Statistics 2024-01-09 Rik Ghosh , Bibhas Chakraborty , Inbal Nahum-Shani , Megan E. Patrick , Palash Ghosh

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

Purpose. Patients with advanced cancer may undergo multiple lines of treatment, switching therapies as their disease progresses. Motivated by a study of metastatic prostate cancer, we develop a microsimulation framework to study therapy…

Discovering new medicines is the hallmark of human endeavor to live a better and longer life. Yet the pace of discovery has slowed down as we need to venture into more wildly unexplored biomedical space to find one that matches today's high…

Artificial Intelligence · Computer Science 2022-02-16 Tri Minh Nguyen , Thin Nguyen , Truyen Tran

It is crucial to design Phase II cancer clinical trials that balance the efficiency of treatment selection with clinical practicality. Sargent and Goldberg proposed a frequentist design that allow decision-making even when the primary…

Methodology · Statistics 2025-05-15 Moka Komaki , Satoru Shinoda , Haiyan Zheng , Kouji Yamamoto

Recently, there has been a resurgence of interest in rigorous algorithms for the inference of cancer progression from genomic data. The motivations are manifold: (i) growing NGS and single cell data from cancer patients, (ii) need for novel…

Machine Learning · Computer Science 2016-02-25 Daniele Ramazzotti

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

The US Food and Drug Administration launched Project Optimus with the aim of shifting the paradigm of dose-finding and selection towards identifying the optimal biological dose that offers the best balance between benefit and risk, rather…

Methodology · Statistics 2023-09-13 Ying Yuan , Heng Zhou , Suyu Liu

Multimodal deep learning (MDL) has emerged as a transformative approach in computational pathology. By integrating complementary information from multiple data sources, MDL models have demonstrated superior predictive performance across…

Quantitative Methods · Quantitative Biology 2025-11-17 Seth Alain Chang , Muhammad Mueez Amjad , Noorul Wahab , Ethar Alzaid , Nasir Rajpoot , Adam Shephard

Recent advances in transformer architectures have revolutionised natural language processing, but their application to healthcare domains presents unique challenges. Patient timelines are characterised by irregular sampling, variable…

Computation and Language · Computer Science 2025-05-26 Linglong Qian , Zina Ibrahim

Cancer is one of the leading causes of death worldwide. Fast and safe early-stage, pre- and intra-operative diagnostics can significantly contribute to successful cancer identification and treatment. Artificial intelligence has played an…

Tissues and Organs · Quantitative Biology 2022-08-10 Marina Zajnulina

Broadening eligibility criteria in cancer trials has been advocated to represent the true patient population more accurately. While the advantages are clear in terms of generalizability and recruitment, novel dose-finding designs are needed…

Applications · Statistics 2023-01-12 Rebecca B. Silva , Bin Cheng , Richard D. Carvajal , Shing M. Lee

Clinical trials usually involve sequential patient entry. When designing a clinical trial, it is often desirable to include a provision for interim analyses of accumulating data with the potential for stopping the trial early. We review…

Methodology · Statistics 2023-03-13 Tianjian Zhou , Yuan Ji

Medical coding translates free-text clinical documentation into standardized codes drawn from classification systems that contain tens of thousands of entries and are updated annually. It is central to billing, clinical research, and…

Artificial Intelligence · Computer Science 2026-04-01 Joakim Edin , Andreas Motzfeldt , Simon Flachs , Lars Maaløe