Karin Hoelzer: I was honored to represent the National Organization for Rare Disorders and the rare community in this important discussion

“Drug development is hard, and doubly so for rare diseases. Great new article summarizing an enlightening discussion about case studies, best practices, and lessons learned during the December 14th FDA and Duke-Margolis Center for Health Policy workshop on incorporating patient experience data into rare disease drug development. Some of the highlights: better statistical tools (and more emphasis on innovative methods and approaches) specific to small sample sizes, and more and better data sources to make the most of real world data and real world evidence for rare diseases. I was honored to represent the National Organization for Rare Disorders and the rare community in this important discussion. Read the full article here, and the workshop recording is available now as well.”