Drug Evaluation Committee Recent Cancer Phase I Trial Designs and Selection
Data Science Expert Committee
June 2023
The purpose of this report is to provide guidance for the appropriate selection of dose-escalation designs for cancer Phase I trials in an era of increasing diversity in cancer treatment modalities. Specifically, the report outlines the main trial designs for each of the three classes (Rule-based/Model-based/Model-assisted) and gives practical examples and case studies as an aid to understanding. In addition, the characteristics of the three classes of designs are organized in a tabular format, the background and objectives of the drug under development are broadly categorized in a flowchart, and recommendations for selecting a dose-escalation design are provided for each of the categorized situations. Other issues related to design selection are also organized from several perspectives. We hope that this report will contribute to the cross-functional consideration of dose escalation design in cancer Phase I trials, including for statisticians, and help in the development of cancer treatments in Japan and around the world.
Japan Pharmaceutical Manufacturers Association Drug Evaluation Committee
Data Science Subcommittee 2022 Task Force 3