written by Mckenna Fulton, Community Program Coordinator for the Myasthenia Gravis Association
Over three days, I had the incredible opportunity to participate in meaningful conversations about policies affecting rare diseases across the United States. I was surrounded by fellow Missourians, many of whom either live with a rare disease themselves or care for someone who does. Together, we came with a shared mission: to push for changes that could make a real difference in the lives of those affected by rare diseases.
Conditions like hereditary transthyretin amyloidosis, sickle cell disease, Mitchell syndrome, lymphangioleiomyomatosis, ulcerative colitis, Crohn's disease, and myasthenia gravis were just a few of the diseases represented from Missouri. We also spoke up for those living with undiagnosed conditions, people whose stories often go unheard. Throughout this experience, I had moments where I questioned my place in the conversation. At times, I even felt like an imposter, advocating for a disease I don’t have, even though I engage with it every day by working closely with those living with myasthenia gravis.
But then I realized that mindset is not only wrong, it’s harmful. My role in this advocacy isn’t about having the disease. It’s about amplifying the voices of those who are impacted. I’m standing alongside individuals who are fighting daily for better care, better treatments, and more awareness. My job is to support them, raise awareness, and make sure their voices are heard, whether I personally live with the disease or not.
What truly stayed with me during Rare Disease Week was the resilience and strength I saw in everyone. Hearing their struggles and challenges was eye-opening, but it was their perseverance and hope that inspired me the most.
It reaffirmed my commitment to raising awareness, supporting research, and advocating for the changes that are so desperately needed. I don’t have to be living with a rare disease to make a difference. I’m part of a community that can advocate for change and bring real impact to those who need it most.
Legislative Priorities at the Conference
Legislative Ask #1: Sustaining Federal Biomedical Research Funding
and Public Health Agency Support
Federal investment in biomedical research and public health agencies is crucial for the rare disease community. Agencies like the National Institutes of Health (NIH), the Food and Drug Administration (FDA), and the Centers for Disease Control and Prevention (CDC) play a key role in ensuring timely diagnosis, advancing research, facilitating the development of new therapies, and securing insurance coverage.
Unfortunately, recent executive orders and the removal of key experts from these agencies are putting vital programs at risk. If these changes continue, they will not only impact the rare disease community but could also have a long-term, harmful effect on the overall healthcare system.
Legislative Ask #2: Reauthorizing the Rare Pediatric Disease Priority
Review Voucher Program
Introduced in February 2025, this bill seeks to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program for another five years. This program is essential for developing treatments for pediatric rare diseases, which often face unique challenges due to small patient populations, difficulty in conducting clinical trials, and delays in diagnosis.
While seventy percent of rare diseases have a pediatric onset, ninety-five percent still lack an FDA-approved treatment. The PRV program expired in December 2024, and after September 2026, the FDA will no longer be able to award these vouchers, making it even harder for companies to develop treatments for children with rare diseases. Advocates are pushing for a swift reauthorization to address this critical issue.
Legislative Ask #3: Include the Accelerating Kids’ Access to Care Act
(AKACA) in the First Available Legislation Package
Many children with rare diseases live in states where specialized care is not readily available. Even in states with leading medical centers, some rare conditions lack the expertise needed for treatment. Over half of U.S. children rely on Medicaid or the Children’s Health Insurance Program (CHIP), but state rules often limit their ability to access timely treatment outside their home state.
The Accelerating Kids’ Access to Care Act (AKACA) aims to reduce these barriers, allowing children to receive care from specialists even if they live in another state. While the House of Representatives passed this bill unanimously in September 2024, it was unfortunately dropped from the final healthcare package at the end of the year. Advocates are now working to ensure it is included in the next legislative session.
Legislative Ask #4: Encourage Members of Congress to Join the Rare
Disease Congressional Caucus
The Rare Disease Congressional Caucus is a bipartisan group that aims to raise awareness of the unique needs of the rare disease community. The caucus brings together patients, physicians, scientists, and industry leaders to address the challenges of developing and accessing life-altering treatments.
Advocates are encouraging members of Congress to join this caucus to ensure that rare diseases remain a priority in legislative discussions. By increasing awareness and support, the caucus can help improve policy decisions that will benefit patients, doctors, and researchers.
Looking Ahead
Rare Disease Week has shown that there’s still a lot of work to do. Even though we’ve made some progress, many important bills are still stuck, and funding for research is still at risk. It’s crucial that we keep advocating for these legislative changes to ensure better healthcare access, support for research, and the development of life-saving treatments for people living with rare diseases.
If you care about these issues, consider reaching out to your representatives. You can also help by spreading awareness and staying up to date on upcoming legislation. Every voice counts, and together we can make a real difference. To see where your House representatives and Senators stand on these issues, check out the Legislative Scorecard. It scores legislators based on their actions for the rare disease community in the 118th Congress. While it doesn’t cover every bill, it gives you a good look at some of the key issues. Also, feel free to watch the Legislation Conference to learn more about the ongoing efforts to improve policy for rare diseases.
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