RDDS 2024: The Critical Need for Patient-Led Data Initiatives: Does Size Matter?

This was a session during the 2024 Rare Drug Development Symposium, presented by Global Genes, in conjunction with The Orphan Disease Center of the University of Pennsylvania.

Researchers and regulators need data. But what kind? Who is the end user? What is “enough data?” Do you need certain types of data for specific research projects? Why? Most importantly how do you evaluate your data set to be certain it’s useful?

Moderator:
Zohreh Talebizadeh, Ph.D.
Senior Director, RARE-X Research Program, Global Genes

Panelists:
Ramona Walls, Ph.D.
Executive Director of Data Science, C-Path

Ben Forred
Director, The CoRDS Registry, Sanford Health

Dave Jacoby, M.D., Ph.D.
BioMarin Fellow in Clinical Science & Vice President, Head of Discovery Medicine, Brineura/BioMarin