Final Working Group Report


Regulatory Science to Advance Precision Medicine

Translational Science Impact Statement/Description: Identify and formulate specific regulatory science topics and opportunities to accelerate precision medicine. These topics and background papers will inform a subsequent Regulatory Science to Advance Precision Medicine Forum that will further evaluate these key topics and provide recommendations to inform regulatory approaches and decision tools (and develop educational materials).

Project Timeline/Data Range: table below

Project Timeline

February 2020 Update, Closure Report and Recent Product/Deliverables: The third annual Regulatory Science to Advance Precision Medicine Forum was held on 25 September 2019 at the PhRMA Foundation in Washington, D.C. With over 40 attendees from all areas of research and regulatory arenas in attendance, the Forum included participants from across academia, industry, government agencies, and foundations. The working group decided to continue on the theme of Digital Health, which was the focus for the second annual forum in 2018. In the context of digital health, the 2019 Forum particularly focused on the opportunities and challenges to transform Real World Data (RWD) into Real World Evidence (RWE). The Forum identified some of the regulatory science opportunities and challenges for improving and utilizing digital measures as RWE to advance precision medicine. The breakout sessions focused on regulatory science needs and priorities for: 1) contributed data from biosensors and, 2) routinely collected data from the EMR and related administrative data.

The manuscript from the 2018 Forum has now been published in Digital Biomarkers (Translation of Digital Health Technologies to Advance Precision Medicine: Informing Regulatory Science). The writing group from the 2019 Forum has met and begun to draft the manuscript for the 2019 Forum, with necessary coordination support being provided via the University of Rochester CTSA Hub.

Future Plans: We have transitioned to an independent workgroup which will work through our individual institution to develop a process to identify and inform future Forum topics and plan to utilize a “Discussion Forum” in 2020 (and other support, where possible) to continue the annual Forum. However, the WG leadership recommends that Regulatory Science and Precision Medicine remain an area of continued focus by Methods and Processes. We suggest “Regulatory Science and Precision Medicine” be added to the list of priority areas the Methods and Processes Enterprise Committee (EC) will continue to focus on and discuss. - Translation of Digital Health Technologies to Advance Precision Medicine: Informing Regulatory Science

Publication Date
Adamo J.E., Bienvenu II R.V., Dolz F., Liebman M., Nilsen W., Steele S.J.