Today, our rare disease community has a reason to celebrate, as Congress passed legislation that includes a five-year reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program, increased funding for rare disease research, and other healthcare priorities that matter deeply to patients and families.
The PRV Program, established by the FDA over a decade ago, has been instrumental in advancing therapies for more than 40 rare pediatric conditions, many of which would likely never attract investment without this program. Importantly, the program costs taxpayers nothing, yet it provides immense hope to families – and has improved health outcomes for thousands of children in our community living with rare diseases. Its reauthorization, after more than two years of your advocacy, means the program can continue catalyzing pediatric therapy development and clinical trials, and investment decisions that were on hold since the program’s lapse can be unleashed.
In another achievement directly tied to robust advocacy from the patient and scientific communities, the NIH will receive $48.7 billion for FY26, 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, will receive a $10 million increase dedicated to rare disease research. The bill also includes 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.
With today’s action, Congress has helped strengthen the foundation for rare disease discovery and innovation. 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.
What else is included in the legislation?
- The Accelerating Kids Access to Care Act: The widely supported legislation will streamline the process for physicians to enroll in other states’ Medicaid programs, making it easier for patients with rare diseases to access care across state lines.
- The Give Kids a Chance Act: 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 will receive $ 1.5 billion, enabling the continuation of several innovative initiatives to improve rare disease diagnosis and therapy development.
- Newborn Screening Funding: The CDC received the same funding as in FY25 to support its work providing technical and quality assurance support for states’ newborn screening programs.
- The Joe Fiandra Access to Home Infusion Act: Coverage for home infusion services for rare disease patients with Medicare will improve, enabling more patients to receive medically necessary infused medications at home with appropriate support, rather than traveling to hospitals or clinics.
- Telehealth extensions: Medicare telehealth flexibilities were extended until December 31, 2027. These flexibilities, implemented during COVID, enable patients to receive care from home, permit audio-only for most visits, and expand eligible providers
- 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.
The EveryLife Foundation for Rare Diseases applauds the reauthorization of the PRV Program and renewed investments in critical health research and public health programs. In a complex and rapidly evolving legislative environment, this achievement reflects the collective strength and persistence of our rare disease community. Patients, families, advocates, researchers, and policymakers have worked tirelessly, through uncertainty and shifting priorities, to advance the policies enacted today.
Our nation’s sustained investments in biomedical research, public health infrastructure, and the PRV Program transformed scientific possibility into tangible progress for many of the 30 million Americans living with rare diseases. We are grateful to congressional champions who have partnered with the rare disease community to secure these advances. While significant work remains to enable all those living with rare diseases to thrive, today’s progress will accelerate innovation, expand access to life-changing therapies, and offer renewed hope to children and families whose futures once seemed beyond reach.
