Current Actions

Urge Your Members of Congress to Support Lifesaving Access to Care for Rare Disease Patients!

Wed, 01 Oct 2025 04:00:00 GMT

As a result of the current U.S. government shutdown, key telehealth flexibilities that increased access to care for rare disease patients on Medicare have expired. If Congress fails to extend the telehealth flexibilities further, rare disease patients would be required to visit their treating physician in person, a journey of potentially hundreds of miles for some, and could also cause financial hardship for rare patients. Also connected to the shutdown are the potential expiration of the Affordable Care Act’s (ACA) enhanced Advance Premium Tax Credits (eAPTCs) at the end of this year. These tax credits reduce the cost of health insurance premiums on the individual marketplace. If the eAPTCs expire, health insurance premiums could spike over 100% for many enrollees. Take action TODAY and urge your lawmakers to protect rare disease patients’ access to care by permanently extending telehealth flexibilities and eAPTCS!

Urge Your U.S. Senator to Oppose Steep Budget Cuts to the National Institutes of Health (NIH)!

Wed, 30 Jul 2025 04:00:00 GMT

The Senate Appropriations Committee is considering the Administration’s request to cut NIH funding by nearly 40 percent for its fiscal year 2026 budget which could harm critical, lifesaving work at the National Institutes of Health (NIH). Please ask your members of Congress to support the rare disease community by asking for robust funding for NIH!

Take action today!

Tell Policymakers to Support our Federal Newborn Screening System

Wed, 23 Apr 2025 04:00:00 GMT

Earlier this year, the Administration eliminated the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), and many federal newborn screening activities may be impacted by other changes at the Department of Health and Human Services (HHS). The ACHDNC and these federal programs are a critical element of the U.S. newborn screening system, supporting state newborn screening programs and playing a central role in the process of adding conditions to the federal Recommended Uniform Screening Panel (RUSP), federal guidelines outlining what serious rare conditions states should screen for. Reach out to your policymakers to share why newborn screening matters to you and urge them to protect our federal newborn screening system.

Support the Rare Pediatric Disease Priority Review Voucher (PRV) Program

Mon, 07 Apr 2025 04:00:00 GMT

Members of Congress can help drive new treatments for children with rare diseases — but they need to act soon.

Since 2012, the FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) Program has helped spur innovation by rewarding companies that develop life-saving therapies for children with rare diseases. According to NORD’s White Paper, this program has helped generate 60 new and innovative treatment options for children living with one of 44 different conditions— real progress for kids living with rare diseases.

The Give Kids a Chance Act (H.R. 1262 / S. 932) would extend this program for at least five more years. Congress must act quickly to preserve this vital incentive and continue driving breakthroughs for children with rare diseases.

Join the Rare Action Network

Thu, 11 Sep 2025 04:00:00 GMT

Members of the Rare Action Network are part of 30+ million person community working towards improving the lives of patients with rare diseases. RAN members will positively improve the quality of life for rare disease patients and their families by ensuring rare diseases become a national priority through policy, education and awareness. JOIN TODAY!