Drug Evaluation Committee Complex Innovative Trial Design: Current Status and Case Studies - FDA Guidance with Japanese Translation
April 2024
In recent years, Complex Innovative Trial Design (hereinafter referred to as “CID”) has been gaining attention in the field of drug development. It is expected to accelerate drug development by enabling the conduct of appropriately designed trials and data analysis even in situations where it is difficult to conduct standard randomized controlled trials—particularly in the development of treatments for pediatric patients and patients with rare diseases.On the other hand, because CIDs involve more complex trial designs than conventional ones, a comprehensive evaluation of the characteristics of these trial designs is required.This report summarizes the innovative elements and points that may be subject to discussion with regulatory authorities by providing a Japanese translation of the FDA guidance document “Interacting with the FDA on Complex Innovative Clinical Trial Designs for Drugs and Biological Products,” along with details on the FDA’s initiatives related to CID and case studies of clinical trials that have adopted CID.This report is intended for statistical analysts involved in the planning and conduct of clinical trials, clinical staff planning clinical trials, and regulatory affairs staff responsible for regulatory affairs. We hope this report will serve as a useful resource for considering more innovative trial designs in Japan as well.
Japan Pharmaceutical Manufacturers Association (JPMA) Drug Evaluation Committee
Data Science Subcommittee, FY2023 Task Force 3-2