Comparative Analysis of Real-World Data Utilization in Regulatory Decision-Making: Current Status in South Korea compared to FDA and EMA

Randomized Controlled Trials are the gold standard for clinical evidence but have limitations in reflecting realworld settings. To address this, regulatory agencies are increasingly utilizing Real-World Data (RWD) and Real-World Evidence (RWE). This study compared the purpose of RWD/RWE utilization in regulatory decision-making based on drugs approved by the FDA/EMA and their corresponding approval reports from MFDS to assess the current status of RWD/ RWE utilization in South Korea over the past five years. Approval reports were reviewed using predefined RWD/RWErelated keywords. A review of FDA/EMA and MFDS approval reports revealed that while FDA and EMA predominantly utilized RWD/RWE for post-marketing evaluations (55%), the MFDS applied RWD/RWE less frequently and without a strong concentration in specific regulatory contexts. However, in South Korea drug approval reports from 2019-2024, of 242 distinct approvals, only 22 cases used RWD/RWE. Additionally, only 1 case (4.5%) showed a significant influence of RWD/RWE indicated by its explicit mention in the reviewer’s evaluation. This suggests that the use of RWD/RWE differed significantly between the FDA/EMA and MFDS, even for the same drug. Moreover, the use of RWD/RWE in South Korea was relatively limited, highlighting that the need for further research to explore the underlying reasons.