04/21/2026
NORD CEO Pamela Gavin opened our 2nd Rare Disease Scientific Symposium today with a powerful guiding message: Rare disease is no longer a scientific challenge alone. It is a systems challenge.
We are generating scientific breakthroughs at an unprecedented pace, but as medicine evolves, so must the infrastructure that delivers treatment to patients.
Too many patients still face barriers to access, uneven clinical resources, and fragmented data collection. The opportunity before us is clear: innovation must be matched by the systems that deliver it — consistently, equitably, and at scale.
In this transformative moment for the field, everyone present at the has a role to play. The FDA are evolving new regulatory pathways for emerging treatments. Researchers across rare disease specialties are sharing data to accelerate evidence generation. And clinical leaders are consulting with payers to expand access to more patients in need.
NORD’s Symposium exists to build and strengthen these bridges, between advocates and researchers, companies and regulators, and across the clinical ecosystem. We’re so excited to facilitate this collaboration in action over the next two days.
We hope those of you not present in person will follow along here as we break it down for the patient and caregiver community!
