A working group established in April by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) is tackling the knotty issues of how best to employ real-world data (RWD) in formulating regulations of orphan drugs and other novel treatments under development for patient populations so small that evaluating them is difficult using existing methodologies.
For example, if no clinical data is available, registry data for a certain disease may be helpful.
The group is made up of agency experts in new drug review, clinical compliance, epidemiology, and other regulatory fields. On the table in the discussions are the risks and benefits of using real-world data to clinically evaluate novel drug therapies without increasing safety risks for patients. No timeline has been set for the working group to report on findings or issue recommendations.
Written by: Ames Gross – President and Founder, Pacific Bridge Medical (PBM)
Mr. Gross founded PBM in 1988 and has helped hundreds of medical companies with regulatory and business development issues in Asia. He is recognized nationally and internationally as a leader in the Asian medical markets. Mr. Gross has a BA degree, Phi Beta Kappa, from the University of Pennsylvania and an MBA from Columbia University.