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.
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Ames Gross
President
Pacific Bridge Medical
Bethesda, MD
www.pacificbridgemedical.comcontact@pacificbridgemedical.com------------------------------