Rare Disease Week was held from February 25th-March 1st, 2024, and Pheo Para Alliance was on the ground in the US capitol to raise awareness about pheochromocytoma and paraganglioma, as well as the challenges faced by those affected by these rare tumors.
Phearless at the Capitol
From February 25th-February 28th, we participated in Rare Disease Week on Capitol Hill. This event, organized by the EveryLife Foundation, gave 700 patients, caregivers, allies and advocates from every US state, the District of Columbia (DC) and Puerto Rico a special opportunity to speak to legislators and their staff about rare diseases like pheo para, and about some bills that, if passed, would help all rare disease patients. On February 27th, Congressional offices took 332 meeting with attendees! Our Community Engagement Specialist, Aimee Powell, was joined on the Hill by Jenny and Henry Rowen and new PPA Board member Bessie Lewis. They met with staff from the offices of five members of the US House of Representatives and five members of the US Senate, including
- From California
- Rep. David Valadao
- Rep. Juan Vargas
- Sen. Alex Padilla
- From Maryland
- Rep. Andy Harris
- Rep. David Sarbanes
- Sen. Ben Cardin
- Sen. Chris Van Hollen
- From North Carolina
- Rep. Deborah Ross
- Sen. Ted Budd
- Sen. Thom Tillis
All PPA members took the opportunity to talk about pheo para and the loss of our only FDA-approved therapy for metastatic disease, and left information on our legislative priorities with each legislator. We’d like to give special thanks to Sen. Van Hollen for showing up to his meeting personally and affirming his support for the rare disease community.
But Wait, There’s More!
After activities on the Hill wrapped up, Aimee ventured to the National Institutes of Health in Bethesda, Maryland to attend Rare Disease Day at the NIH on Leap Day (February 29th). The day was full of presentations on the latest progress in rare disease treatments and promising advancements on the horizon, with artificial intelligence and gene therapy taking center stage. We also took a little time to meet with some phriends in the neuroendocrine cancer space, PPA Medical Advisory Board member Dr. Jaydira Del Rivero and Healing NET Foundation Executive Director Mia Tepper! You can watch the recorded videocast here!
We wrapped up our week inside the Beltway by watching the US Food and Drug Administration’s (FDA) Rare Disease Day 2024 presentations via the watch party organized by the National Organization for Rare Disorders (NORD). In addition to discussing how medical products are reviewed and approved, and the importance of the patient perspective, the FDA presented on its strong desire for decentralized clinical trials, which would allow some trial-related activities to occur at locations other than clinical trial sites. This is the first year that the FDA has offered CME credits to healthcare professionals who attended the virtual event, and we hope to see it carried forward! Watch the recording of the day’s events here!
We’re grateful to all who participated and engaged with us, whether in-person or virtually. These events all take place every year at the end of February – we hope to see YOU there next year!
Photos
Rare Disease Week on Capitol Hill Days 1 and 2
Rare Disease Day at the NIH
NORD FDA Watch Party
