A Call to Action for the Rare Disease Community
Despite scientific progress, fewer than 5% of rare diseases have an FDA-approved treatment. Families continue to face delayed diagnoses, limited treatment options, and overwhelming financial hardships. By uniting patients, caregivers, healthcare professionals, policymakers, researchers, and innovators, we can create a more equitable healthcare system—one that serves every family, regardless of their rare condition.
Our Policy Recommendations
We call on national leaders to:
✔️ Preserve and strengthen incentives for therapeutic development
✔️ Ensure predictable, science-driven evaluation of rare disease therapies
✔️ Modernize the newborn screening system to enable early intervention
✔️ Support affordable access to approved therapies through Medicaid and CHIP
Key Policy Focus Areas
Join the Rare Disease Congressional Caucus
The Rare Disease Congressional Caucus is a bipartisan group dedicated to championing the needs of the rare disease community. The Caucus:
- Fosters awareness and understanding on Capitol Hill
- Shapes legislation to address barriers to research, development, and access
- Ensures a permanent voice for rare disease patients and families in Washington
Reauthorize the Rare Pediatric Disease Priority Review Voucher
The Creating Hope Reauthorization Act (H.R. 7384 / S. 4583) renews a vital program that drives innovation for rare pediatric diseases—at no cost to taxpayers. Without reauthorization, the FDA will lose the ability to award Rare Pediatric PRVs after September 30, 2026.
Timely action is critical to protect innovation and ensure children with rare diseases have hope for future treatments.
Accelerating Kids’ Access to Care Act
The Accelerating Kids’ Access to Care Act (H.R. 4758 / S. 2372) streamlines the process for pediatric specialists to provide care across state lines. By allowing providers to more efficiently enroll in multiple Medicaid programs:
- Children with cancer and rare diseases gain faster access to specialists
- Families face fewer bureaucratic delays
- Providers can deliver timely, life-saving care under a five-year enrollment term
Medicaid Expansion
Expanding Medicaid coverage can transform outcomes for families by:
- Increasing access to essential care
- Reducing emergency room costs for the uninsured
- Creating new healthcare jobs and boosting local economies
- Narrowing the health coverage gap for vulnerable communities
Every person deserves access to affordable healthcare—regardless of income, geography, or diagnosis.
Incentivizing Medicaid Expansion Act
This legislation offers states the enhanced federal medical assistance percentage (FMAP) regardless of when they expand their Medicaid programs. By applying retroactively, the act encourages more states to expand coverage and improve healthcare access for newly eligible populations under the Affordable Care Act.
The National Economic Burden of Rare Disease Study
In 2019, the EveryLife Foundation for Rare Diseases conducted the most comprehensive study to date, estimating the total economic burden of rare diseases in the U.S. at nearly $1 trillion across 379 conditions.
Key findings revealed:
- Enormous direct medical costs
- Significant indirect costs (lost productivity, caregiving demands)
- An urgent need for increased federal funding in research and treatment development
Federal investment is crucial to mitigating this economic burden and promoting health equity for families affected by rare diseases nationwide.
✅ Take Action
Your voice makes a difference. Together, we can drive policy that transforms lives.
🤝 Join Our Advocacy Network – Stay informed, take action, and amplify the voices of rare disease families and professionals.
📢 Contact Your Representatives – Urge them to support rare disease legislation.
Source: Cover Alabama. www.coveralabama.org | EveryLife Foundation for Rare Diseases. www.everylifefoundation.org
The E.WE Foundation 