Drug Evaluation Committee Recent Cancer Phase I Trial Design and Selection
June 2023
The purpose of this report is to provide guidelines for the appropriate selection of dose-escalation designs for Phase I cancer trials in an era of diversifying cancer treatment modalities.Specifically, this report outlines the main trial designs for each of the three classes—rule-based, model-based, and model-assisted—and provides practical examples and case studies to aid understanding.In addition, the characteristics of the designs in the three classes are summarized in tabular form, and the background and objectives of the drug under development are broadly categorized using flowcharts, with recommendations provided for selecting dose-escalation designs for each categorized scenario. Furthermore, other issues related to design selection are organized from several perspectives.We hope this report will contribute to cross-functional discussions on dose-escalation designs for Phase I cancer trials—including those involving statisticians—and serve as a resource for the development of cancer treatments in Japan and around the world.
Japan Pharmaceutical Manufacturers Association, Drug Evaluation Committee, Task Force 3, Fiscal Year 2022
Recent Phase I Cancer Trial Designs and Their Selection (2.6 MB)