Lani Knutson, Cure CMD’s Advocacy Lead, provides a recap of 2024 Rare Disease Week on Capitol Hill, February 25 — 29.
Rare Disease Day was February 29, leap day, which only happens every four years, making it especially rare, just like our CMD Community. While many consider it as any other day, the 300 million people living with rare disease from around the world joined the celebration, both through in-person events and online, to lend their rare voice to raising awareness and influencing change that empowers those living with CMD and all the 10,000+ rare diseases.
In the U.S., leading up to Rare Disease Day, Cure CMD joined 700 rare disease advocates and 207 patient organizations in Washington D.C. and virtually kicked off Rare Disease Week on Capitol Hill 2024, hosted by Rare Disease Legislative Advocates, a program of the EveryLife Foundation for Rare Diseases!
A documentary viewing and panel discussion kicked off the week, building connections and sparking the drive that the rare disease community is: Unified. Amplified. Stronger than ever.
On Monday, advocates participated in the Legislative Conference, learning about proposed, landscape-changing legislation that would improve the lives of those living with rare disease and then taking that information further to our Members of Congress the very next day.
Hill Day: the day that the rare disease community takes over Capitol Hill. On Tuesday, advocates met with their respective legislators. This event allows participants to take part in sharing their stories to enact change that benefits the rare community and garners their powerful support. My fellow Wisconsin advocates and I had the rare opportunity to discuss our needs with rare champion, Senator Tammy Baldwin. Our advocates advocated for a number of policies, including:
Creation of an Interagency Coordinating Committee for Rare Disease: This mechanism would foster coordination and collaboration across all federal government agencies to ensure that services, policies, and prioritization of resources are efficiently utilized.
Accelerating Kids’ Access to Care Act: This bill would improve children’s access to much-needed out-of-state healthcare by streamlining the burdensome and time-consuming Medicaid provider screening and enrollment process.
Reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program: The Creating Hope Reauthorization Act would reauthorize this program for four more years, ensuring this vital incentive, which has no cost to taxpayers, can continue to spur innovation in rare diseases that disproportionately affect children.
Safe Step Act: This bill would aim to ensure patients can access the best treatment available, safely and efficiently, instead of “failing first,” requiring patients to take one or more alternative medications chosen by their insurance provider before they can access the medication prescribed by their healthcare provider.
Rare Disease Congressional Caucus: The bipartisan and bicameral caucus promotes awareness of rare disease issues and gives an ever-present voice to the rare disease community on Capitol Hill.
Along with:
Federal Aviation Act (FAA) Reauthorization: This bill, which passed the Senate Commerce Committee unanimously, will improve training for airline personnel who handle and stow wheelchairs and mobility devices on airplanes, and will provide training for crew members assisting passengers with disabilities. In addition, the bill strengthens the consumer air travel complaint process, supports disability representation on passenger air travel advisory committees, and provides a pathway for the development of wheelchair seating on planes.
SSI Savings Penalty Elimination Act: This bill would make changes to the existing Supplemental Security Income (SSI) asset limits; it would increase asset limits from $2,000 for individuals and $4,000 for married couples to $10,000 for individuals and $20,000 for married couples while factoring in inflation with any changes to future limits.
Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act: This bill would build on other laws to ensure patient perspectives are fully considered as part of the U.S. Food and Drug Administration (FDA) benefit-risk framework, by requiring the FDA to disclose how they use or incorporate patient experience data in the review of new therapies.
This year, not only did Congress hold an impromptu congressional hearing on Rare Disease Day to review rare disease legislation, but the White House also held its first-ever Rare Disease Forum. Leaders in the community, such as Tania Simoncelli from the Chan Zuckerberg Initiative, Paul Melmeyer from the Muscular Dystrophy Association, and Julie Tierney from the U.S. Food and Drug Administration were part of discussions around raising awareness for and addressing challenges in rare disease at the highest level of government.
Rare Disease Day also marked history as the disability community witnessed the U.S. Department of Transportation release a groundbreaking proposed rule to improve air travel, reinforcing the safety, rights, and dignity of air travelers with disabilities, during a public event at the White House. After reviewing the proposed rule, we encourage you to share your comments and experiences with disabled-access air travel until April 28, 2024. Please join your community in raising your voice for progress and inclusion in this vital service.
The remainder of Rare Disease Week included a discussion on DEIA (Diversity, Equity, Inclusion, Accessibility), the Rare Disease Congressional Caucus Capitol Hill Briefing, and this year’s Rare Artist Reception. In addition, the National Institutes of Health (NIH) hosted a day-long conference to showcase developments in research for rare diseases, and the week culminated with Rare Disease Day at the Food and Drug Administration (FDA).
Progress may seem slow, but with the community’s advocacy efforts, we are laying the groundwork for new ways to approach rare disease regulation, research, and treatments. Cure CMD is committed to collaborating with our advocacy partners like the EveryLife Foundation to ensure the rare disease community has a voice in enacting change. We might be “rare,” but we will continue to demand change along with other rare disease communities.
Every year, Rare Disease Week advances legislation beneficial to the rare disease community, empowering and driving us in our commitment to improve the quality of life for those living with a rare condition. The heart of advocacy is storytelling. You have a powerful story to tell. Participating in legislative advocacy can take as little or as much time as you want – from as little as 5 minutes to submit a pre-filled form to your legislators, to becoming more involved in the ongoing activities of our team. Keep an eye out for more opportunities to advocate in the coming days, and consider attending next year’s Rare Disease Week on Capitol Hill. Your voice truly matters!
Join us for 2025 Rare Disease Week on Capitol Hill taking place in Washington D.C., February 24-26. Attend a life-changing week with hundreds of rare disease community members from around the world who gather to be educated on legislative issues, connect with other rare advocates, and share their unique stories to enact positive change.
Meeting with your congressional representatives outside of Rare Disease Week. Right now, your state and federal representatives are considering bills that would make a positive impact on people living with CMD. But they need to hear from YOU! You can still participate by completing this action alert to have an automated email sent to your representatives asking them to support the CMD and rare disease communities.
Rare Across America 2024 will take place August 5-16. Rare disease advocates will have the opportunity to meet with their representatives at the member’s in-state or in-district office. Mark your calendar with these dates and stay tuned for more information! Learn more about Rare Across America.
Become a CMD Legislative Advocate, tapping into opportunities to promote legislative initiatives that improve the quality of life for those living with CMD with your local and federal representatives. Questions? Email Lani Knutson at advocacy@curecmd.org.