Investigation of Medical Information Databases Utilization for Drug Effectiveness Assessment in FDA-Approved Products

Abstract

Background : There is a globally growing movement to promote the utilization of real-world data (RWD) in pharmaceutical regulatory approval. In Japan, there has been little progress in discussions regarding their utilization in new drug approvals. Objectives : This study aimed to clarify the current situation regarding the utilization of medical information databases in new drug approvals by the FDA, to promote discussions regarding their utilization in Japan. Method : We identified FDA-approved products that may have utilized RWD, reviewed the review reports for these products, and identified the background factors of RWD utilization, such as RWD type, role in submission packages, data format type, and whether or not confounding adjustment was applied. We also categorized the contribution level of RWD to the approval decision and evaluated the contribution for each background factor. Results : We identified 27 products with RWD utilization in FDA submissions in 2019-2021. There were many patterns of RWD utilization for effectiveness, and the degree of contribution to approval varied among background factors. Medical information databases were utilized in 5 products, with contributions to approval found in 1 product. Conclusion : The low contribution of medical information databases might be derived from the insufficient preliminary investigation at the planning stage due to the lack of experience in RWD utilization. To create real world evidence from medical information databases, it is essential to fully understand the information generated and accumulated in daily medical practice, select an appropriate database that meets the objectives, and apply an appropriate design and statistical method.