July 2025

FY 2026 Congressional Appropriations Process: In May, the President released a proposed budget for fiscal year 2026 which contained cuts and changes to federal agencies including the Food and Drug Administration, National Institutes of Health, and Centers for Disease Control and Prevention. In June, the appropriations process began in both the House and Senate. The full committee in the House began their markup of the Agriculture-FDA appropriations bill and released report language that includes multiple rare-related sections. The bill now heads to the full House for a vote. In the Senate, the Labor-Health and Human Services Senate Appropriations Subcommittee held a hearing to discuss the proposed budget.  Read the bill language and learn more about the federal budget process.

In the Senate, they released their version of the Agriculture-FDA appropriations bill and report language that includes many rare-related section, including support for the Rare Disease Innovation Hub. The report language recognizes the challenges of rare disease therapy development and the important role the Hub can play in that process. It supports the full implementation of the Hub's 2025 strategic agenda as well as calls on the Hub to submit a report to Congress within 12 months about any relevant information about the Hub's work.  

Several resources are now available to help explain the process and the rare disease community’s priorities: 

• RDLA Tip Sheet on Advocacy and the Appropriations Process 

• The EveryLife Foundation’s Statement on Understanding the Federal Budget Process and What’s at Stake for Rare Diseases 

• The EveryLife Foundation’s Statement on the President’s FY26 Budget Proposal 

Budget Reconciliation Bill: The “One Big Beautiful Bill Act”, H.R.1, was passed by the Senate and House and signed into law by the president on July 4, 2025. The bill includes many changes to Medicaid eligibility, enrollment, and financing, including new work reporting requirements mandating a minimum level of employment, hours worked, or volunteer activity in order to qualify for Medicaid health coverage. It is estimated that millions of people could lose health insurance as a result of these provisions. To learn more about the law, its impact on the rare disease community, and what’s next for advocates, listen to RDLA’s recent monthly webinar. 

Celebrate the Power of the Rare Disease Community - Nominations Now Open for the RareVoice Awards! The RareVoice Awards, presented by RDLA, recognize outstanding advocates who amplify the voices of the rare disease community in shaping state and federal policies on Capitol Hill. Nominations are open across several categories, including federal and state advocacy by patients, organizations, congressional and agency staff, youth advocates, and even artists who use their creativity to champion our cause. Learn more.   

Learn more about state-level legislation and its status below.  

Apply Now: State Medicaid Beneficiary Advisory Councils Seeking Patient Members: Several states have open applications for their Medicaid Beneficiary Advisory Councils, which provide patients with a voice in shaping Medicaid policy and program operations. State Medicaid leaders are especially encouraging applications from rural residents, working-aged adults, and men. For questions, contact bmcgowan@everylifefoundation.org   

Upcoming deadlines: 

• Ohio: Medicaid participants can apply for the council. There is no current deadline. For more information, click here. 

Prior Authorization: 

• CA AB 539: This bill would require prior authorization for a health care service to remain valid for a period of at least one year from the date of approval. It is currently scheduled for a committee hearing. Learn more about the bill. 

Find more ways to take action for the rare disease community and access all RDLA resources and policy primers.   

The EveryLife Foundation supports 501(c)(3) rare disease nonprofits that directly engage policymakers to advance patient needs. Rare Giving grants fund advocacy efforts and resources such as conferences, educational events, survey tools, patient data collection, and translation services, all aimed at strengthening public policy for rare diseases. Apply here.   

The EveryLife Foundation for Rare Diseases asks you to contact your legislators, in response to the termination of the Advisory Committee on Heritable Disorders in Newborns and Children, which facilitated the addition of conditions to the Recommended Uniform Screening Panel (RUSP) and ask them to restore the federal newborn screening system.   

Invite your Member of Congress to join the Rare Disease Congressional Caucus or thank them for being a member. View a full list of Caucus members or ask your members to join.  

The Alzheimer’s Impact Movement urges you to contact your legislators and encourage them to support the Credit for Caring Act (S. 925/H.R. 2036), which would create a new federal tax credit of up to $5,000 per year for eligible caregivers.  

The EveryLife Foundation  asks advocates to urge your members of Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher program by passing the Give Kids a Chance Act of 2025 (H.R. 1262/S.932). Send your Representative and Senators a message and encourage their support. 

The EveryLife Foundation  asks advocates to encourage your Members of Congress to protect rare disease experts, funding, and stability of our nation’s biomedical research and public health agencies. View the action alert.  

The Myositis Association encourages advocates to contact their representatives in support of H.Res. 277, which designates May as "National Myositis Awareness Month” and seeks to raise awareness for the disease. Contact your legislator and learn more.  

The Patient-Centered Outcomes Research Institute (PCORI) intends to issue a PCORI Funding Announcement on August 12, 2025, seeking to fund clinical effectiveness research studies that focus on rare diseases. Interested applicants should utilize this preannouncement to identify collaborators, obtain input on potential studies, and develop high quality proposals with PCORI’s special areas of emphasis considered in their submission. This initiative will fund studies that compare treatments or care strategies to improve symptom management, speed diagnosis, and strengthen care delivery. Applications are due January 13, 2026, and must include strong patient and caregiver engagement. For more information, visit the PCORI website. 

13th Annual Rally for Medical Research: Save the date! The 13th Annual Rally for Medical Research will be held on September 18, 2025. The event will bring patients, caregivers, researchers, clinicians, and other advocates together for meetings with congressional offices in support of funding for the National Institutes of Health (NIH). A reception for attendees will be held on Wednesday, September 17. Registration is open now on the Rally for Medical Research website. 

2025 PCORI Annual Meeting: Patient-Centered Outcomes Research Institute (PCORI) invites advocates to save the date for their Annual Meeting, held in Washington DC, from October 21-22, 2025. Registration will be available on July 7. Learn more and register on the PCORI website.  

RDLA Monthly Webinars Recap: On June 26, we heard a review of rare disease legislation and learned about how advocates can support the bills. On July 10, we heard from a panel of experts about H.R.1 and what it means for the rare disease community. The RDLA Monthly Webinars are an opportunity to inform patient advocates about pressing health policy topics so that they can be successful legislative advocates. Watch a recording of the June and July RDLA Webinars.