Peer-Reviewed Publication
Int J Clin Oncol2026August 20, 2026Journal Article
Recommendations for generating real-world evidence with regulatory-grade relevance and reliability.
Yasutoshi Sakamoto1, Hideaki Bando2,3,4, Yoshihiro Aoyagi5, Hiroki Sokuoka1, Toshihiro Misumi6, Koji Oba7, Yukiko Mori8, Jun Okui9, Makoto Miyazaki10,11, Yasunari Sadatsuki10,12, Kazuma Koizumi10,13, Rie Otake10,14, Akinori Yuri10,15, Eri Tajima16, Taro Ueno17, Takayuki Nakamura18, Hiroki Yoshihara19, Keisuke Itoya20, Ryosuke Annaka21, Harumasa Nakamura22, Natsuko Okita23, Hideaki Takahashi24, Tomohiro Kuroda25, Kenichi Nakamura26, Takayuki Yoshino27, Atsushi Ohtsu28
1Translational Research Support Office, Division of Drug and Diagnostic Development Promotion, Department for the Promotion of Drug and Diagnostic Development, National Cancer Center Hospital East, 6-5-1 Kashiwanoha, Kashiwa, Chiba, 277-8577, Japan.
2Translational Research Support Office, Division of Drug and Diagnostic Development Promotion, Department for the Promotion of Drug and Diagnostic Development, National Cancer Center Hospital East, 6-5-1 Kashiwanoha, Kashiwa, Chiba, 277-8577, Japan. hbando@east.ncc.go.jp.
3Department of Gastroenterology and Gastrointestinal Oncology, National Cancer Center Hospital East, 6-5-1 Kashiwanoha, Kashiwa, Chiba, 277-8577, Japan. hbando@east.ncc.go.jp.
4Department of Data Science, National Cancer Center Hospital East, 6-5-1 Kashiwanoha, Kashiwa, Chiba, 277-8577, Japan. hbando@east.ncc.go.jp.
5Department of Medical Information, National Cancer Center Hospital East, 6-5-1 Kashiwanoha, Kashiwa, Chiba, 277-8577, Japan.
6Department of Data Science, National Cancer Center Hospital East, 6-5-1 Kashiwanoha, Kashiwa, Chiba, 277-8577, Japan.
7Interfaculty Initiative in Information Studies, The University of Tokyo, Tokyo, 113-0033, Japan.
8Center for Digital Transfomation of Healthcare, Kyoto University, Kyoto, 606-8501, Japan.
9Department of Biostatistics, Keio University School of Medicine, Tokyo, 160-8582, Japan.
10Pharmacovigilance Subcommittee, Drug Evaluation Committee, Japan Pharmaceutical Manufacturers Association, Tokyo, 103-0023, Japan.
11Patient Safety Strategy BioPharma Department, Japan Patient Safety Division, R&D Japan, AstraZeneca K.K., Tokyo, 108-0023, Japan.
12Integrated Evidence Generation Post-Marketing Surveillance, Bayer Yakuhin Ltd., Osaka, 530-0001, Japan.
13Safety Program Operation, MSD.K.K, Tokyo, 102-8667, Japan.
14Japan PMS & Re-Examination, Takeda Pharmaceutical Co., Ltd., Tokyo, 103-8668, Japan.
15Safety Science 2 Department, Chugai Pharmaceutical Co., Ltd., Tokyo, 103-8324, Japan.
16Flatiron Health, K.K., Tokyo, 108-0075, Japan.
17SUSMED, Inc., Tokyo, 103-0023, Japan.
18Nuclear Liability Office, Research and Development Bureau, Ministry of Education, Culture, Sports, Science and Technology (MEXT), Government of Japan, Tokyo, 100-8959, Japan.
19Nishimura & Asahi (Gaikokuho Kyodo Jigyo), Tokyo, 100-8124, Japan.
20Health Policy Bureau, Ministry of Health, Labour and Welfare, Tokyo, 100-8916, Japan.
21Innovation Promotion Subcommittee, Pharmaceutical Industrial Policy Committee, Japan Pharmaceutical Manufacturers Association, Tokyo, 103-0023, Japan.
22Department of Clinical Research Support, National Center of Neurology and Psychiatry, 4-1-1 Ogawahigashi-cho, Kodaira, Tokyo, 187-8551, Japan.
23Clinical Research Support Office, Research Management Division, National Cancer Center Hospital, Tokyo, 104-0045, Japan.
24Regulatory Affairs Management Section, Clinical Research Planning Division, Clinical Research Support Office, National Cancer Center Hospital East, Chiba, 277-8577, Japan.
25Division of Medical Information Technology and Administration Planning, Kyoto University Hospital, Kyoto, 606-8501, Japan.
26Department of International Clinical Development, National Cancer Center Hospital, Tokyo, 104-0045, Japan.
27Department of Gastroenterology and Gastrointestinal Oncology, National Cancer Center Hospital East, 6-5-1 Kashiwanoha, Kashiwa, Chiba, 277-8577, Japan.
28Research Unit, Japanese Foundation For Cancer Research, Tokyo, 135-8550, Japan.
Abstract
Real-world data (RWD) and real-world evidence (RWE) are increasingly incorporated into regulatory decision-making to complement randomized controlled trials (RCTs), particularly in settings where conventional trial designs are infeasible, such as rare diseases, rare molecular subtypes, and post-marketing evaluation. Across regulatory authorities, the use of RWD/RWE is consistently framed by the fi…
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