Drug Evaluation Committee Complex Innovative Trial Design: Current Status and Case Studies - FDA Guidance with Japanese Translation
Data Science Subcommittee
April 2024
In recent years, Complex Innovative Trial Design (hereinafter referred to as CID) has been attracting attention in the field of drug development, especially in the development of treatments for pediatric patients and patients with rare diseases. CID is expected to accelerate drug development by making it possible to conduct appropriately designed trials and analyze data even in situations where it is difficult to conduct conventional randomized controlled trials, such as in the development of treatments for pediatric patients and patients with rare diseases. On the other hand, CID requires a comprehensive evaluation of study design characteristics because the study design is more complex than in the past. This report provides a Japanese translation of the FDA guidance "Interacting with the FDA on Complex Innovative Clinical Trial Designs For Drugs and Biological Products," as well as an overview of the FDA's efforts related to CID and case studies of clinical trials employing CIDs, summarizing innovative elements and points of potential discussion with regulators. The intended audience for this report is statistical analysts involved in planning and conducting clinical trials, clinical members planning clinical trials, and regulatory affairs members responsible for regulatory affairs. We hope that this report will help to consider more innovative study designs in Japan.
Japan Pharmaceutical Manufacturers Association Drug Evaluation Committee
Data Science Subcommittee FY2023 Task Force 3-2