本邦の医薬品開発におけるビッグデータ・リアルワールドデータの利活用に関する実態調査　―日本製薬工業協会加盟各社に対するアンケート調査―

抄録

Background: The use of big data/real-world data (BD/RWD) is expected to be a new option for evidence generation in drug development. Pharmaceutical companies are considered to be a group of key drivers to promote widespread use of BD/RWD in Japan. However, little is reported about the current status of the use of BD/RWD for drug development in Japan.

Objective: This study was conducted to reveal the current usage of BD/RWD by pharmaceutical companies in Japan and their current organization structure, with the aim to gain insight on the challenges and future perspective of BD/RWD in Japan.

Methods: A questionnaire survey was conducted on 66 pharmaceutical companies affiliated with Japan Pharmaceutical Manufacturers Association from 16 to 31 October 2018. The survey consisted of two parts: Questionnaire 1 investigated the current usage of BD/RWD in drug development in Japan, and Questionnaire 2 investigated the current organization structure.

Results: Of 52 companies that responded to Questionnaire 1, 30 companies (57.7％) were currently using BD/RWD for drug development in Japan for various purposes, 29 of which (96.7％) planned to continue using BD/RWD. Of 28 companies that responded to Questionnaire 2 and currently using BD/RWD, 9 companies (32.1％) had established divisions or organizations for internal management of BD/RWD, and 14 companies (50.0％) had standard operation procedures or guidance for handling legal and regulatory aspects associated with the use of BD/RWD for drug development in Japan.

Conclusion: BD/RWD is currently used for various purposes in drug development in Japan. However, the proportion of companies currently using BD/RWD for drug development is limited to approximately 50％. Proactive involvement of pharmaceutical companies toward the use of BD/RWD for drug development in Japan together with further industry-government-academia harmonization for environment improvement are awaited.