Appropriate Relevancy and Reliability of Real-World Data for the Utilization of Regulatory Submission.

The extraction of data that contribute to regulatory approval from real-world data (RWD) is difficult because of the lack of a standardized data format and extraction methodology. Additionally, when real-world evidence (RWE) is used as an external control group, the similarity between internal and external control data is not evaluated. To investigate the data extraction methodology for the external control data of rare molecular subtypes, we have initiated the "REALISE" study. In this study, we aim to elucidate the "relevance" and "reliability" of RWD/RWE necessary for regulatory approval. As most databases are not designed for regulatory use in the creation phase, we will investigate retrospective methodologies to ensure RWD/RWE reliability. This study will compare the "relevance" and "reliability" of the ARCAD global database, SCRUM-Japan Registry, SCRUM-Japan observational study, and Flatiron Health RWD, and statistically analyze the differences and similarities among the four databases. We will also examine the methodology for extracting sufficiently relevant data from the SCRUM-Japan observational study. Additionally, if the reliability of the RWD/RWE does not reach the required level for regulatory approval, we will examine the methodologies to ensure the "reliability" of the SCRUM-Japan observational study for regulatory approval. The obtained results will be submitted to the "Consultation for Development of Registry" in the Pharmaceuticals and Medical Devices Agency, and we will discuss the standard methodology. The procedures and findings identified in the REALISE study will be organized from the perspectives of "database construction," "data analysis," and "outcome evaluation" and will be issued as "the draft guidelines."

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Disclosure Hideaki Bando reports research funding from Ono Pharmaceutical and honoraria from Ono Pharmaceutical, Taiho Pharmaceutical, and Eli Lilly in Japan. Yasutoshi Sakamoto reports no conflict of interests. Toshihiro Misumi reports honoraria from Chugai, AstraZeneca, and Miyarisan. Yuriko Takeda reports no conflict of interests. Yoshiaki Nakamura reports honoraria from Chugai Pharmaceutical, Merck Biopharma, and Guardant Health AMEA and research grants from Taiho Pharmaceutical, Chugai Pharmaceutical, Guardant Health, Genomedia, Daiichi Sankyo, Seagen, and Roche Diagnostics. Kazuya Mizuguchi and Yoshihiro Aoyagi reports no conflict of interests. Takayuki Yoshino reports honoraria from Taiho Pharmaceutical, Chugai Pharmaceutical, Eli Lilly, Merck Biopharma, Bayer Yakuhin, Ono Pharmaceutical, and MSD and research funding from Taiho Pharmaceutical, Ono Pharmaceutical, Chugai Pharmaceutical, Amgen, Parexel International, MSD, Daiichi Sankyo, Pfizer, Genomedia, Sysmex, Nihon Boehringer Ingelheim, and Sanofi. Atsushi Ohtsu reports research funding from BMS and honoraria from Chugai and Ono Pharmaceutical.