PMDA Perspective on Use of Real-World Data and Real-World Evidence as an External Control: Recent Examples and Considerations.

Recent discussions about the utilization of real-world data (RWD) and real-world evidence (RWE) have been more focused on drug development for regulatory approval rather than during the post-marketing stage. In Japan, RWD/RWE have been practically utilized as an external control for drug approval. Most cases were related to orphan diseases where the feasibility of conducting randomized controlled clinical trials was generally low.

Risk of artery dissection during systemic exposure to vascular endothelial growth factor pathway inhibitors.

Whether or not vascular endothelial growth factor pathway inhibitors (VPIs) increase the risk of artery dissection is still unknown. This study aimed to quantitatively evaluate the possibility of artery dissection as a class effect of VPIs using nationwide real-world data. This cohort study was conducted based on the National Database of Health Insurance Claims and Specific Health Checkups of Japan (NDB), which spans nearly the entire Japanese population of over 100 million individuals.