Impact of Dose-Escalation Design on the Safety and Development of Anticancer Drugs in Clinical Trials.

ABSTRACT: The operational characteristics of dose-escalation design in phase I studies have been studied using simulations; however, there is limited analysis regarding its effects on the results of clinical trials. We collected the data of 394 clinical trials involving dose-escalation studies for anticancer drugs submitted to the Pharmaceuticals and Medical Devices Agency between 2013 and 2022. We used the internal data of the PMDA and published papers and analyzed outcomes such as enrollment and drug development. We identified model-based designs and rule-based designs as the two primary designs. The median number of dose-limiting toxicity (DLT)-evaluated patients was higher for model-based designs than for rule-based designs. The proportion of rule-based designs was higher in Japanese trials and

SUBMITTER: Nonami A 

PROVIDER: S-EPMC12483344 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature