Making our voices heard for Rare Disease Week and beyond

Rare Disease Week on Capitol Hill is now behind us, and I had the honor of participating this year with the U.S. Hereditary Angioedema Association (HAEA) and EveryLife Foundation’s Rare Disease Legislative Advocates program.

The week, which started on Feb. 24, was nothing short of incredible; it was an educational and uplifting experience that brought together rare disease warriors from across the nation. The energy in that space, the shared passion, and the sense of unity among advocates, patients, and policymakers created a feeling that words can hardly capture.

Rare Disease Week is often referred to as our community’s Super Bowl, and rightfully so. It’s a time when the voices of those living with rare diseases echo throughout the halls of Capitol Hill, urging policymakers to take action for a more equitable and accessible healthcare system.

But beyond policy, it was something deeper: a true community. The bonds formed during this week will last a lifetime, extending beyond the confines of Washington, D.C., and into the everyday battles we fight for better care, treatments, and support. I’ve mentioned before the importance of community in the rare disease space, and that week of advocacy reinforced the idea more than ever.

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Channeling uncertainty into action

Participants in the U.S. Hereditary Angioedema Association and EveryLife Foundation’s Rare Disease Legislative Advocates program lobby lawmakers in Washington, D.C., for Rare Disease Week 2025. (Courtesy of Hollie Amadio)

The week was empowering, showing us that even when we don’t think we have the courage to speak up, we do. We must, because our voices matter. Rare Disease Week gives people the platform and the confidence to tell their stories, to advocate for themselves and others, and to remind the world that we are here, and we will not be ignored.

As hereditary angioedema (HAE) patient Nikita Lebedz so perfectly put it, “Humans are fascinating creatures that have the ability to change our environment, rather than to simply adapt to whatever comes past.” We aren’t just adapting to the broken systems that have failed rare disease patients for too long; we’re changing them.

During the week, the HAEA urged Congress to pass bipartisan legislation such as the Safe Step Act, which aims to protect patients from step therapy protocols that delay access to physician-directed treatments. We also pushed for the HELP Copays Act, which ensures that copay assistance counts toward a patient’s out-of-pocket costs, making lifesaving treatments more affordable. These bills are critical for patients who rely on specialized care and cannot afford unnecessary delays or financial barriers.

A major focus was also placed on securing increased funding for the National Institutes of Health and the U.S. Centers for Disease Control and Prevention to expand research into rare diseases, including HAE with normal C1 inhibitors. Funding public health initiatives and rare disease research isn’t optional; it’s essential for saving lives and improving health outcomes. Furthermore, we continue to stand against the use of quality-adjusted life years and other discriminatory value assessment frameworks that undermine the worth of chronically ill and disabled individuals.

Advocates pose for a group photo before attending a legislative conference during Rare Disease Week 2025 in Washington, D.C. (Courtesy of Hollie Amadio)

Given today’s uncertain political climate, our work is far from over. The future of healthcare policy, especially for rare disease patients, is unclear, and that uncertainty can be unsettling. But rather than feeling powerless, we must channel that uncertainty into action. Advocacy doesn’t end when Rare Disease Week is over. It extends into our daily lives, into our communities, and into every conversation we have with our legislators.

We need every single person affected by rare diseases to take action — not just those who made the trip to Capitol Hill. Contacting legislators from home is just as impactful. Write emails, make phone calls, and engage with your representatives on social media. Tell them your story. Urge them to support the legislation that directly affects our lives. If we don’t fight for ourselves, who will?

Rare disease is bipartisan. It’s not about politics; it’s about human lives. Our voices are loud, and they’ll be heard. We proved that in Washington, and we must continue proving it every single day. We’re our own biggest advocates, and together, we’re unstoppable.

The momentum we built during Rare Disease Week must carry us forward, and I’m proud to be part of a movement that refuses to be ignored. Together, we’re shaping a future where rare disease patients have the access, affordability, and respect we deserve.

Note: Angioedema News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Angioedema News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to angioedema.