Hydrocephalus Association Unites Advocates for 2024 Rare Disease Week

Members from the Hydrocephalus Association Action Network came to Washington, DC, to participate in the Everylife Foundation for Rare Disease’s Rare Disease Week, which took place from February 25-28, 2024. The annual event brings together advocates from across the country to lobby on Capitol Hill for the needs of the Rare Disease Community. HA first participated in the event in 2016, uniting our hydrocephalus voice with the larger rare disease community. 

Although hydrocephalus as a whole is not considered a rare disease due to the over 1 million individuals who have the condition, the various medical classifications (etiologies) of hydrocephalus are considered rare. For example, hydrocephalus caused by a brain bleed (post hemorrhagic hydrocephalus) or a genetic malformation like X-Link Hydrocephalus. This rare disease characterization allows hydrocephalus to be eligible for federal funding within the rare disease space and special pathways that provide special considerations or incentives that allow drugs and devices to reach patients faster. Hydrocephalus is under the Rare Disease Category in the Peer Reviewed Medical Research Program (PRMRP), a program within the Department of Defense’s Congressionally Directed Medical Research Program (CDMRP) that provides funding for high-impact scientific and medical research.

Hydrocephalus Association & Rare Disease Week on Capitol Hill

The 2024 Rare Disease Week included four days of events for advocates to take part in. The week opened with a rare disease documentary screening of the story of Chris Bombardier’s physical, psychological, and emotional journey to prepare to summit Mount Everest. Chris is a hemophilia patient. The screening was followed by a reception with Chris and the filmmaker. 

Energized from an evening of connecting and socializing, participants turned their attention to the political agenda ahead with the Legislative Conference on Monday, February 26th. This event brought in a variety of speakers from the disability and health policy space to talk about the current legislative issues affecting the rare disease community. Some of the topics included step therapy protocol, pediatric out of state medical licensing, and Rare Disease Priority Review Voucher Reauthorization. These educational lessons provided advocates with the resources and knowledge to effectively lobby their members of Congress the next day. Additionally, advocates were grouped by their state and congressional districts to meet other advocates and practice for their meetings. Following the Legislative Conference, the hydrocephalus advocates joined HA staff for a breakout training on the issues important to our community. 

The next morning, advocates headed to Capitol Hill for congressional meetings with their state Representatives. During the meetings, they were able to share the connection between the legislative issues and their healthcare journey. Most advocates met with over 5 congressional offices on both the Senate and House side.

Our hydrocephalus advocates came from Washington, Tennessee, New York, Maryland, and the District of Columbia. A special thank you to Jonathan Moe (Washington), Kevin Flynn (Tennessee), Elana Scwartz and Yehuda Gelman (New York), Amanda Garzon (Maryland), Lakisha Harris (representing Louisiana), and Davis Kaderli (representing California) for bringing our voice to Capitol Hill.

Kevin had the opportunity to meet directly with Senators Marsha Blackburn and Bill Hagerty from his home state He shared his story and the challenges faced by the hydrocephalus community. “Coming to Rare Disease Week with the Hydrocephalus Association was pivotal for me in embracing who I am as a hydrocephalus survivor. Advocating on Capitol Hill empowers me to remember that despite my challenges through 15 brain surgeries, I can help fight to make change, in order to make life easier for people with hydrocephalus and the community around them,” shared Kevin.  

Rare Disease Week concluded on Wednesday with two events on Capitol Hill. The Rare Disease Week 2024 DEIA discussion focused on prior authorization and other utilization management practice challenges in rural communities followed by a look at cell and gene therapy disparities. The Rare Disease Congressional Caucus Briefing, held in the Capitol, brought rare disease advocates to speak to Congressional staffers on the various policies advocates discussed in their individual meetings. Their powerful stories showed the need for legislative change within the healthcare system. 

These lobbying efforts had significant impact, resulting in the addition of two new members to the Pediatric and Adult Hydrocephalus Caucus. Congressman Jerry Nadler (NY-12) became the second member from New York to join the Caucus, while Congressman Van Orden (WI-03) made history as the first member from Wisconsin to join. The addition of these new members show the impact of continued advocacy from individuals within the hydrocephalus community.

For those who are interested in advocacy, there are many ways you can contribute on the local side. Please reach out to Davis@hydroassoc.org to ensure your voice is heard!