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A comparative study of dose escalation designs in oncology phase 1 clinical trials
Journal of the Korean Data & Information Science Society 2023;34:587-606
Published online July 31, 2023;
© 2023 Korean Data and Information Science Society.

SuJin Heo1 · Jae Won Lee2

12Department of Statistics, Korea University
Correspondence to: This work was supported by the National Research Foundation of Korea (NRF) grant funded by the Korea government (MSIT) (No. RS-2023-00208882). This research was also supported and funded by the Korean National Police Agency. [Project Name: Advancing the Appraisal Techniques of Forensic Entomology / Project Number: PR10-04-000-22].
1 Graduate student, Department of Statistics, Korea University, Seoul 02841, Korea.
2 Professor, Department of Statistics, Korea University, E-mail:
Received May 20, 2023; Revised June 4, 2023; Accepted June 11, 2023.
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License ( which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
Oncology phase I clinical trials are typically aim to evaluate the safety and tolerability of dose escalation in a small number of patients and to determine the recommended dose for phase 2 clinical trials. Traditionally, the design of phase 1 clinical trials in oncology has been primarily rule-based design. Recently, however, model-based and model-assisted designs have been increasingly used. In this study, two of each of the three designs were selected and simulated to compare their performance differences in terms of safety and accuracy. The study also examined how the performance of each design varied with the target dose-limiting toxicity, number of subjects, and cohort size under different dose-toxicity scenarios. In comparison, model-based designs performed better in accuracy and model-assisted designs performed better in safety. The performance ranking of each design changed depending on the situation. When designing an oncology phase 1 clinical trial, an appropriate design should be selected based on the dose-response relationship, the target dose-limiting toxicity, and the number of subjects.
Keywords : Dose escalation designs, dose-limiting toxicities, maximum tolerated dose, oncology phase 1 clinical trial