2-C-KJS-6 Reo Tanoshima ^1,2、Naoko Inagaki ^1,3、Manabu Nitta ^1,4、Soichiro Sue ^1,3、Sayuri Shimizu ²、Tatsuya Haze ^1,5、Kotaro Senuki ¹、Chihiro Sano ¹、Hajime Takase ^1,6、Makoto Kaneko ²、Akito Nozaki ^1,7、Kozo Okada ^1,8、Kohei Ohyama ⁹、Atsushi Kawaguchi ^1,10,11,12、Yusuke Kobayashi ¹、Hideki Oi ¹³、Shin Maeda ³、Yuichiro Yano ¹⁴、Yuji Kumagai ¹⁵、Etsuko Miyagi ^{1,16  1,2 1}YCU Center for Novel and Exploratory Clinical Trials (Y-NEXT), Yokohama City University Hospital, Yokohama, Japan、²Department of Health Data Science, Graduate School of Data Science, Yokohama City University, Yokohama, Japan、³Department of Gastroenterology Yokohama City University Graduate School of Medicine Yokohama Japan.、⁴Department of Cardiology, Yokohama City University Graduate School of Medicine, Yokohama, Japan.、⁵Department of Medical Science and Cardiorenal Medicine, Yokohama City University Graduate School of Medicine, Yokohama, Japan.、⁶Department of Neurosurgery, Graduate School of Medicine, Yokohama City University, Yokohama, Japan.、⁷Gastroenterological Center, Yokohama City University Medical Center, Yokohama, Japan.、⁸Division of Cardiology, Yokohama City University Medical Center, Yokohama, Japan、⁹Medical Care Bureau, Yokohama City、¹⁰Department of Pediatrics, St. Marianna University, Kawasaki, Japan、¹¹Department of Anesthesia, Yokohama City University, Yokohama, Japan、¹²CHU Sainte Justine Research Centre, University of Montreal, Montreal, Canada、¹³Department of Clinical Data Science, Clinical Research and Education Promotion Division, National Center of Neurology and Psychiatry, Tokyo, Japan.、¹⁴NCD Epidemiology Research Center (NERC), Shiga University of Medical Science, Shiga, Japan.、¹⁵Kitasato Clinical Research Center, School of Medicine, Kitasato University, Sagamihara, Japan. ¹ Background
Real-world data (RWD) studies have played an important role in clinical pharmacology. In addition to its conventional objectives such as epidemiology and post-market drug evaluation, several examples have been identified where RWD is used in drug development and decision-making by policymakers. To effectively incorporate RWD studies into these areas, stringent quality controls are crucial.
Risk based approach (RBA) is an activity of quality management where the researchers systematically assess, control, communicate, and review the risks associated with clinical trials. While RBA is originally introduced and mainly applied to interventional studies with a focus on patients’ safety, extending its application to all types of clinical trials becomes imperative to ensure high quality.
Methods
We implemented RBA in a RWD study using a designated intractable disease. Our team comprising of diverse background assessed the risks of the RWD study comprehensively and systematically.
Results
In RWD studies, patients are not subjected to interventions for research purposes, and risks to patient safety are uncommon. The researchers can only use the existing data in the database, and obtaining additional data is a challenge. Hence, we concluded that selecting optimal database is vital for the successful completion of RWD studies. Implementing RBA to database selection, alongside research planning and execution is crucial. We quantitatively evaluated the risk of two candidate databases, and chose the National Database of designated intractable diseases of Japan as the optimal database. We also assessed and ranked risks associated with research planning, and prioritized them for consideration during the research execution.
Conclusion
Implementing RBA to both database selection and research planning/conducting is important to RWD studies.
Acknowledgement
This work is supported by the Japan Agency for Medical Research and Development (JP22yk0126017 and JP23yk0126027).