02/27/2026
Yesterday, the EveryLife Foundation for Rare Diseases was thrilled to host the Rare Disease Congressional Caucus briefing as part of Rare Disease Week 2026. We were joined in-person by Senator Amy Klobuchar and via video message by Representative Gus Bilirakis, two of the Co-Chairs of the Caucus. The panel included Ron Bartek, Co-Founder of the Friedrich's Ataxia Research Alliance; rare disease advocates, Michael and Allison Beacham and Brittany Clayborne; and Jamie Sullivan, Senior Vice President of Policy & Advocacy for the EveryLife Foundation.
The hearing was moderated by James Valentine, Director at Hyman, Phelps & McNamara and a member of our Board of Directors with welcoming remarks from Janelle Gillings, Director of Policy and Government Relations at Merck..
Senator Klobuchar outlined some of the major wins our rare disease community has seen recently: the creation of the Rare Disease Innovation Hub, and the passage of both the Give Kids a Chance Act and the Accelerating Kids Access to Care Act.
Senator Klobuchar also called out the need for consistent funding to the NIH, increased incentives for clinicians to ensure accessible rare disease treatment, and increased funding toward staffing in both hospitals and research institutions.
Representative Bilirakis emphasized the need for bipartisan leadership in Congress to address the needs of the rare disease community. He also spoke about the importance of targeted incentives and strong, patient-centered policies in order for life-saving innovation to exist, as well as what a critical tool newborn screening is to detect rare diseases early and begin treatment. Said the Congressman, "Awareness alone is not enough, action must follow."
Our incredible rare disease advocates shared their powerful stories about their own journeys, exemplifying the need for patient involvement in rare disease policy. The panel discussion revolved around priorities for our community, such as consistent and reliable Newborn Screening programs, federal support for biological research, and patient engagement in the therapy development process.
Jamie also shared how Congress can address the gaps represented in the speaker’s stories, including advancing timely diagnosis through newborn screening and access to genetic testing, continuing to fund biomedical research, and engaging with the FDA to ensure all the tools that Congress provided the FDA for accelerating rare disease therapy innovation are used consistently.
After this standing-room-only event, the community was back on the Hill for more meetings with their Members. Thank you to everyone who attended in person or joined the livestream!
ICYM watch the recorded livestream here: https://www.youtube.com/watch?v=ukFbSZYOY0o&t=3s
And check out the video message from Rep. Bilirakis:
https://youtu.be/kIHXUNLp99M
