Cancer registry as external control data for regulatory submission in Japan

Area of Science:

• Regulatory Science
• Real-World Data (RWD) Utilization
• Oncology Registries

Background:

• Japan's regulatory authorities, via the Clinical Innovation Network, have improved real-world data (RWD) registry development.
• Guidelines and consultations enhance RWD registry integrity and effective use.
• Cancer registries are increasingly used as external control groups in regulatory and academic settings.

Purpose of the Study:

• To review Japanese regulatory authorities' perspectives and guidelines on cancer registries.
• To address the limited adoption of RWD/real-world evidence (RWE) for drug approval due to unclear standards.
• To inform the development of new guidelines for broader RWD/RWE application in Japan.

Main Methods:

• Review of guidelines and consultations from regulatory bodies (Ministry of Health, Labour and Welfare, Pharmaceuticals and Medical Devices Agency).
• Analysis of established cancer registries (e.g., SCRUM-Japan Registry, MASTER KEY, GALAXY).
• Initiation of the 'REALISE study' to define data relevancy and reliability for drug approval.

Main Results:

• Several high-quality cancer registries have been established, with SCRUM-Japan achieving a notable regulatory approval.
• Regulatory bodies and academic institutions promote the use of cancer registry data as external controls.
• Uncertainty regarding RWD/RWE standards for drug approval hinders broader regulatory use in Japan.

Conclusions:

• Clearer standards are needed to increase the regulatory use of RWD/RWE in Japan.
• The 'REALISE study' aims to define data requirements and methodologies for ensuring reliability.
• Findings will guide the creation of draft guidelines to expand RWD/RWE application in Japan.