Rare on the Hill 2025 Report
Rare on the Hill 2025 Report from Cristina Brennan
Rare on the Hill 2025 was an amazing event, both personally and professionally!
Personally
First, and often most importantly, it is always wondrous and heart-filling to be in a room with other rare disease families. I met many individuals living with rare disorders and many families and caregivers advocating for their rare loved ones. My heart is very full. The only thing missing was other representatives from our STXBP1 community. So, my mission for next year is to invite as many people as possible from our tribe to join me in DC to help advocate for legislation to support our rare kiddos
Professionally
The purpose of the annual Rare on the Hill event is to teach rare advocates about important federal legislation that impacts the rare community and to meet with Senators and Congresspeople to advocate for the passage of the legislation.
This year, there were four items, aka “Legislative Asks” to learn about and discuss with our Senators and Congresspeople. Below is a brief summary of each. I am from New York, so I met with the offices of Senator Schumer, Senator Gillibrand and Congressman Suozzi to seek support for the 4 Legislative Asks.
2025 Legislative Asks
Support Steady and Robust Leadership, Federal Biomedical Research Funding, and Public Health Agencies This request simply reminds the legislators of the importance of funding the NIH, CDC and FDA to the rare community. See this link for additional information.
Support the Reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program This request asks to reintroduce legislation that was allowed to expire in December 2024, which provided significant financial incentives for pharmaceutical companies to bring treatments for rare diseases to market. See this link for additional information.
Include the “Accelerating Kids Access to Care Act” This legislation helps facilitate care for rare patients across state lines by expediting the process for doctors to enroll in the out-of-state patient’s state Medicare program. See this link for additional information.
Join the Rare Disease Congressional Caucus This requests asks the lawmakers to join the Rare Disease Caucus to bring public and Congressional awareness to the unique needs of the rare disease community (including patients, physicians, scientists, and industry), and creates opportunities to address barriers to the development of and access to life-altering treatments. See this link for additional information.
IMHO HIGHLIGHT
If you want to choose just one item to support, my personal opinion is that you urge your legislators to reintroduce the PRV program (#2 above). Here’s why.
As we all know, pharmaceutical companies do not have a lot of incentives to spend money on research for rare disorders because it may not be profitable to treat a small population like STXBP1 – as of January 2025 there are 348 known STXBP1 patients in the US. To help incentivize pharma, the PRV program offered valuable vouchers to companies seeking approval for rare disease treatments. These vouchers could be sold to larger pharmaceutical companies for many millions of dollars. This money could then be used to support the costs to bring the rare treatment to market. And, this program did not cost the taxpayer a penny.
Since this legislation expired in December, may rare treatments have been shelved or deprioritized due to the lack of funding. This is devastating to the rare community.
Who is Cristina Brennan?
I am an attorney from New York with 13 years of experience working in politics and government. I am also mom to a 12 year old STXer named Tristan. As a board member of the STXBP1 Foundation, I hope to use my legal background to help all of our community members become legal advocates for our STXers. Read more about me in my bio on our Team page here.
What can you do to help advocate?
Join me in DC next year!!
Don’t want to or can’t get to DC? Visit your Senators and Congresspeople in their local offices near your home.
You can also send letters and emails requesting their support of our rare legislation.Learn! Visit the RDLA website. The RDLA (Rare Disease Legislative Advocates) is a program offered by the EveryLife Foundation which provides information designed to support the advocacy of all rare disease patients and organizations.
