Advocate for the Rare Disease Community: Urge Legislators to Address Critical Issues
We would like to highlight some recent and upcoming legislation that have a critical impact on our rare disease community. It is essential that we encourage our lawmakers to acknowledge their responsibility to support individuals with rare diseases.
Please find more information below and and I urge you to share your support for these important legislative efforts. Together, we can help ensure that the needs of the rare disease community are addressed and protected.
Urge your Senators to support the Rare Pediatric Disease Priority Review Voucher Program
This week, Senators on the Health, Education, Labor and Pensions (HELP) Committee have the opportunity to advance important legislation that supports rare disease innovation for children.
Since 2012, the FDA has awarded companies that develop life-saving and novel therapies for children with rare diseases with priority review vouchers (PRVs) to help spur innovation in this critical area. The Give Kids a Chance Act (S. 932) would reauthorize the Pediatric Rare Disease Priority Review Voucher program through 2029.
Legislation reauthorizing this critical program must pass to ensure that this vital tool continues to address the significant unmet treatment needs among the pediatric rare disease population. Without this program's reauthorization, FDA will no longer be allowed to initiate the process necessary to issue new rare pediatric disease PRVs and our community will lose a vital incentive that has been so successful in helping more children access safe and effective therapies for their condition.
Send a message to your Senators TODAY asking them to support the Give Kids a Chance Act of 2025!
Statement on U.S. Department of Health and Human Services Restructuring
The National Organization for Rare Disorders (NORD®), is a nonprofit, nonpartisan organization representing more than 30 million Americans living with rare diseases. As a member of NORD, the Jack McGovern Coats' Disease Foundation joins others in the growing concern about the impacts that the restructuring of the U.S. Department of Health and Human Services (HHS) will have on the rare disease community. This rapid, largescale reduction-in-force (RIF) poses great risk to the critical activities that public health agencies, the public health workforce, and researchers do to enable rare disease patient access to vital lifesaving care and treatments across the country.
Please find below a statement from NORD CEO, Pamela Gavin, regarding the recent and ongoing restructuring taking place within the Department of Health and Human Services.