Rare Disease Week at Capitol Hill 2025

I'm late in sharing this, but back in February I attended Rare Disease Week at Capitol Hill, hosted by the EveryLife Foundation for Rare Diseases. I was part of a team of 7 people from 4 states representing the Pheo Para Alliance. Nearly 1,000 rare disease advocates from all 50 states gathered for the legislative conference on February 25 to connect and learn about rare disease legislation and how it impacts patients. The legislative asks this year included:

1. Support Steady and Robust Leadership, Federal Biomedical Research Funding, and Public Health Agencies
2. Support the Reauthorization of the Rare Pediatric Disease Priority Review Voucher Program 
3. Ask Members of Congress to Join the Rare Disease Congressional Caucus 
4. Include the Accelerating Kids’ Access to Care Act (AKACA)
   

The following day, we met with the Congressional representatives or legislative staff for our respective states. Because the 119th Congress was sworn in only a month earlier, this was an excellent opportunity to bring awareness to new House Representatives and Senators about rare disease legislation and to share our stories. An estimated 10% of the U.S. population has a rare disease, and only 5% of rare diseases have an approved FDA treatment. 

This year, there was increased energy around saving funding for biomedical research. The research funded today will drive the treatment options that are available in the future, and I remembered the hope that the currently available treatments would buy Scott time to benefit from new medical advances. The same actions that benefit the rare disease community, such as accelerating the discovery and availability of new treatments, can benefit all patients and their families. It's critical to show our elected leaders through our voices and our votes that we want a robust system for advancing research toward new medical therapies. 

With the mighty Pheo Para Alliance team

A beautiful day for advocacy on Capitol Hill

Meeting with the office of Rep. Sarah Elfreth of Maryland

It was inspiring to hear stories from patients and families from all over the country. About half of the 30 million rare disease patients in the U.S. are children.

Ending the day on Capitol Hill at the National Organization for Rare Diseases (NORD) reception