DIA: Driving Insights to Action

MID-NET Among PMDA’s First Steps Into RWE

Dr. Yoshiaki Uyama, Director of the Office of Medical Informatics & Epidemiology for the Pharmaceuticals and Medical Devices Agency (PMDA), Japan, and DIA Global Forum Regulatory Science co-editor, shares his vision for utilizing real world evidence in regulatory science. “The challenge is, we still don’t understand completely the characteristics and the limitations of those data. We are very familiar with assessing or evaluating the data of a clinical trial. But real world data is not completely of the same character as that of a clinical trial because those real world data are not primarily collected for such purposes.”

Regulatory Science 2019: Leading or Holding Back Innovation?

In 2018, the US FDA approved a record high number–59–of new, novel drugs. “We are at a point now where there is so much innovation in this space and the regulatory aspects are catching up, so we now have three approved gene therapy products in the US and over seven to eight hundred INDs for cell and gene therapy filed with FDA,” explains Dr. Adora Ndu, Executive Director of Global Regulatory Policy, Research & Engagement, BioMarin Pharmaceutical, and Regulatory Science Co-Editor for DIA Global Forum. “With each interaction with each product that’s filed and with each product that’s approved, the regulatory experience is broadened.”

23andMe: Information Empowering Patient Action

“Genetics is about everybody, and this information is really fundamental to you and your health,” explains Dr. Emily Drabant Conley, Vice President of Business Development for 23andMe, the first company to receive US regulatory approval for their over the counter genetic ancestry and health tests. “If individuals are empowered with information about their genetic predisposition, it puts them in a position to take action.”