Our community’s advocacy is working! Congress is listening to the powerful outreach from our rare disease community.
Lawmakers have released a draft legislative package that includes a five-year reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program, increased funding for rare disease research, and other important healthcare priorities that matter deeply to patients and families.
Most of the government is operating under a continuing resolution, which expires on January 30. Rather than passing a single bill to fund all remaining government agencies, Congress is attempting to pass a series of ‘minibus’ packages, each containing appropriations bills and related policies. If passed by Congress and signed by the President, the proposed legislation would fund key health agencies, including the NIH, CDC, and HRSA, through September 30, 2026.
This momentum reflects the power of sustained advocacy and the power of you sharing your voice. During Rare Disease Week 2025, more than 11,000 advocates signed a petition delivered to every member of Congress calling on lawmakers to protect and prioritize rare disease biomedical research and public health funding. The petition was followed by months of advocacy efforts to prevent major cuts to research funding proposed in the President’s budget request and in the House appropriations deliberations.
Those advocacy efforts are paying off. Under the draft of the Labor, Health and Human Services, Education, and Related Agencies Appropriations bill, the NIH would receive $48.7 billion, an increase of $415 million. Importantly for the rare disease community, the National Center for Advancing Translational Sciences (NCATS), the NIH’s home for rare disease research, would receive a $10 million increase dedicated to rare disease research. The proposal also continues protections against drastic cuts to the NIH’s indirect cost rate, and blocks a planned change to grant funding that would have reduced the number of grants awarded each year. At a time when funding for many critical initiatives is under threat, these investments in rare disease research send a strong signal that Congress considers rare disease issues a national priority.
Including the reauthorization of the PRV Program in this critical funding bill gives us a chance to preserve the PRV Program and ensure robust funding for rare disease research and public health initiatives.
Our job is not done until votes are cast and the President has signed a bill. Let’s make sure that Congress hears how important it is for them to complete their work, pass the draft healthcare package, and ensure that lifesaving research and programs for our community continue to drive innovation and improve the health of the over 30 million Americans living with rare diseases.
What else is included in the proposed legislation?
- The Accelerating Kids Access to Care Act: The widely supported legislation would streamline the process for physicians to enroll in other states’ Medicaid programs, making it easier for rare disease patients to access care across state lines.
- The full Give Kids a Chance Act was included. In addition to the reauthorization for the PRV Program, the Give Kids a Chance Act provides new pediatric cancer funding and FDA authorities, clarifies how the FDA applies the exclusivity provisions of the Orphan Drug Act, and gives the FDA the ability to provide more information in the development of generic drugs.
- ARPA-H Funding: The Advanced Research Projects Agency for Health would receive $ 1.5 billion, enabling the continuation of several innovative initiatives aimed at improving rare disease diagnosis and therapy development.
- Newborn Screening Funding: The CDC would receive the same funding as in FY25 to support their work in providing technical and quality assurance support for state’s newborn screening programs.
- Telehealth extensions: Medicare telehealth flexibilities would be extended until December 31, 2027.
- Community Health Centers: The bill includes the largest increase in mandatory funding for community health centers in the last 10 years.
- PBM Reform: Policies that place new restrictions on pharmacy benefit managers were included. The changes focus on price transparency, requiring PBMs to pass on rebate savings to employers, and ensuring PBMs provide realistic contracts to willing pharmacies to enhance network adequacy.
To urge your Congressional members to support this legislative package, visit our action alert here
