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When a novel treatment has successfully passed phase I, different options to design subsequent phase II trials are available. One approach is a single-arm trial, comparing the response rate in the intervention group against a fixed…

Methodology · Statistics 2020-08-03 Johannes Krisam , Dorothea Weber , Richard F. Schlenk , Meinhard Kieser

Dose-finding trials for oncology studies are traditionally designed to assess safety in the early stages of drug development. With the rise of molecularly targeted therapies and immuno-oncology compounds, biomarker-driven approaches have…

Methodology · Statistics 2025-09-15 Xijin Chen , Pavel Mozgunov , Richard D. Baird , Thomas Jaki

We identify three properties of the standard oncology phase I trial design or 3 + 3 design. We show that the standard design implicitly uses isotonic regression to estimate a maximum tolerated dose. We next illustrate the relationship…

Applications · Statistics 2009-06-08 Gregory J. Hather , Howard Mackey

We consider a Bayesian framework based on "probability of decision" for dose-finding trial designs. The proposed PoD-BIN design evaluates the posterior predictive probabilities of up-and-down decisions. In PoD-BIN, multiple grades of…

Methodology · Statistics 2021-03-12 Meizi Liu , Yuan Ji , Ji Lin

We propose a Bayesian optimal phase 2 design for jointly monitoring efficacy and toxicity, referred to as BOP2-TE, to improve the operating characteristics of the BOP2 design proposed by Zhou et al. (2017). BOP2-TE utilizes a…

Methodology · Statistics 2024-08-13 Kai Chen , Heng Zhou , J. Jack Lee , Ying Yuan

Phase I dose-escalation trials constitute the first step in investigating the safety of potentially promising drugs in humans. Conventional methods for phase I dose-escalation trials are based on a single treatment schedule only. More…

Applications · Statistics 2021-03-04 Burak Kürsad Günhan , Sebastian Weber , Abdelkader Seroutou , Tim Friede

Multi-cancer early detection (MCED) tests offer to screen for multiple types of cancer with a single blood sample. Despite their promising diagnostic performance, evidence regarding their population benefit is not yet available. Expecting…

Applications · Statistics 2023-07-04 Jane M. Lange , Kemal C. Gogebakan , Roman Gulati , Ruth Etzioni

New medical treatment development requires multiple phases of clinical trials. Despite the significant human and financial costs of bringing a drug to market, less than 20% of drugs in testing will make it from the first phase to final…

Computation and Language · Computer Science 2024-08-21 Michael Reinisch , Jianfeng He , Chenxi Liao , Sauleh Ahmad Siddiqui , Bei Xiao

In early-phase cancer clinical trials, the limited availability of data presents significant challenges in developing a framework to efficiently quantify treatment effectiveness. To address this, we propose a novel utility-based Bayesian…

Methodology · Statistics 2025-06-10 Saurabh Bhandari , Michael J. Daniels , Chenguang Wang

Level set estimation (LSE), the problem of identifying the set of input points where a function takes value above (or below) a given threshold, is important in practical applications. When the function is expensive-to-evaluate and…

Machine Learning · Statistics 2024-12-02 Yu Inatsu , Shion Takeno , Kentaro Kutsukake , Ichiro Takeuchi

The aim of this chapter is to provide a brief introduction into the basics of a top-down multilevel tumor dynamics modeling method primarily based on discrete entity consideration and manipulation. The method is clinically oriented, one of…

Medical Physics · Physics 2010-09-14 Georgios Stamatakos

We propose a frequentist adaptive phase 2 trial design to evaluate the safety and efficacy of three treatment regimens (doses) compared to placebo for four types of helminth (worm) infections. This trial will be carried out in four…

Objective: The objective of this study is to develop a machine learning (ML)-based framework for early risk stratification of clinical trials (CTs) according to their likelihood of exhibiting a high rate of dosing errors, using information…

Machine Learning · Computer Science 2026-02-27 Félicien Hêche , Sohrab Ferdowsi , Anthony Yazdani , Sara Sansaloni-Pastor , Douglas Teodoro

Although there is an extensive statistical literature showing the disadvantages of discretizing continuous variables, categorization is a common practice in clinical research which results in substantial loss of information. A large…

Methodology · Statistics 2017-08-17 Márcio Augusto Diniz , Mourad Tighiouart , André Rogatko

A multicompartment mathematical model is presented with the goal of studying the role of dose-dense protocols in the context of combination cancer chemotherapy. Dose-dense protocols aim at reducing the period between courses of chemotherapy…

Populations and Evolution · Quantitative Biology 2019-09-04 Álvaro G. López , Kelly C. Iarosz , Antonio M. Batista , Jesús M. Seoane , Ricardo L. Viana , Miguel A. F. Sanjuán

Despite significant research efforts and advancements, cancer remains a leading cause of mortality. Early cancer prediction has become a crucial focus in cancer research to streamline patient care and improve treatment outcomes. Manual…

Computer Vision and Pattern Recognition · Computer Science 2024-08-19 Samta Rani , Tanvir Ahmad , Sarfaraz Masood , Chandni Saxena

Rectal cancer is one of the most common diseases and a major cause of mortality. For deciding rectal cancer treatment plans, T-staging is important. However, evaluating the index from preoperative MRI images requires high radiologists'…

Image and Video Processing · Electrical Eng. & Systems 2023-12-11 Saeko Sasuga , Akira Kudo , Yoshiro Kitamura , Satoshi Iizuka , Edgar Simo-Serra , Atsushi Hamabe , Masayuki Ishii , Ichiro Takemasa

Background: Phase I dose-finding trials increasingly encounter delayed-onset toxicities, especially with immunotherapies and targeted agents. The time-to-event continual reassessment method (TITE-CRM) handles incomplete follow-up using…

Methodology · Statistics 2026-02-24 Robert Amevor , Emmanuel Kubuafor , Dennis Baidoo

Deep learning-based automated contouring and treatment planning has been proven to improve the efficiency and accuracy of radiotherapy. However, conventional radiotherapy treatment planning process has the automated contouring and treatment…

Medical Physics · Physics 2024-12-02 Sangwook Kim , Aly Khalifa , Thomas G. Purdie , Chris McIntosh

Deep learning (DL) has proven highly effective for ultrasound-based computer-aided diagnosis (CAD) of breast cancers. In an automaticCAD system, lesion detection is critical for the following diagnosis. However, existing DL-based methods…

Image and Video Processing · Electrical Eng. & Systems 2023-06-13 Jian Wang , Liang Qiao , Shichong Zhou , Jin Zhou , Jun Wang , Juncheng Li , Shihui Ying , Cai Chang , Jun Shi