What We Do
As leaders in the rare disease space, the RARE Foundation sets the standard in policy engagement by connecting our community with legislators, and the biotech and pharmaceutical industries to advance policies that break barriers and provide science-based guidance. Our efforts are changing public policy and ensuring access to safe, effective treatments for all.
Events Hosted
We host meaningful events that activate the rare disease community, amplify our voices, and inform scientific advancements.
- RARE Disease Week: Brings nearly a thousand advocates from around the country to Capitol Hill in Washington, DC annually to meet directly with lawmakers and champion transformative rare disease policy priorities.
- RARE Across America: Connects advocates with their Members of Congress in local districts, providing a platform to build ongoing relationships with lawmakers via in-person and virtual meetings.
- State Policy Program: Connects our community members with their state legislators at state houses throughout the country. Many policies that impact rare diseases are made at the state level, including changes to Medicaid funding, recipient guidelines, and waiver programs. Our state policy program provides a powerful opportunity for people living with rare diseases to meet with their representatives, share their stories, and influence policies.
- Newborn Screening Bootcamp: Provides the rare disease community with the opportunity to learn about and discuss developments in newborn screening from experts and patient advocates currently navigating the process.
- RARE Scientific Workshop: Creates momentum across the landscape by bringing together rare disease patients, industry partners, governmental agency partners, and those in academia to discuss urgent issues and identify actionable recommendations for advancing regulatory and therapeutic development pathways.
- Our Scientific Workshop was the impetus for the FDA’s Rare Disease Innovation Hub. During a workshop, the RARE Foundation proposed the development of a Center of Excellence to enhance collaboration between the scientific community and government agencies to accelerate treatments. This idea gave way to the Rare Disease Innovation Hub.
Help the RARE Foundation continue to make an impact on the rare disease landscape.
What We’ve Accomplished
In the last 16 years, powered by patient advocates and patient advocacy organizations, we have significantly pushed the needle to achieve progress on behalf of our community.
How did we do this? We:
- Advanced policies at state and federal levels
- Published the National Economic Burden of Rare Disease – the first study of its kind in the world
- Advocated for the passage of the 21st Century Cures Act
- Led the Newborn Screening Modernization effort, including advocating for the National Academy of Medicine and Sciences (NASEM) report
- Led the passage of RUSP Alignment Legislation in 13 US states, meaning 52% of babies born in the US are born in RUSP-aligned states
- Facilitated the creation of the FDA’s Rare Disease Innovation Hub
- Created the ICD code roadmap
- Published the Guide to Patient Involvement in Rare Disease Therapy Involvement
- Conducted a study on the cost of delayed diagnosis in rare diseases.
To learn more about the impact of the RARE Foundation on the policy landscape, read more about our Issues. To learn how you can become an advocate, visit our every voice matters page.
