Current Actions

Tell Policymakers to Support our Federal Newborn Screening System

Wed, 23 Apr 2025 04:00:00 GMT

The Administration recently 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 is a critical element of the U.S. newborn screening system, 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 as part of their universal newborn screening programs. Urge policymakers to protect our federal newborn screening system and immediately reinstate the ACHDNC!

Urge your Senators to support the Rare Pediatric Disease Priority Review Voucher Program

Mon, 07 Apr 2025 04:00:00 GMT

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. However, legislation extending this program must pass to ensure this important tool is reauthorized to address the significant unmet treatment needs that exist in the pediatric rare disease population does not end.

Urge Governor Hobbs to Sign the Arizona Rare Disease Advisory Council Bill!

Wed, 12 Mar 2025 04:00:00 GMT

Great news! House Bill 2380, which creates a Rare Disease Advisory Council in Arizona, has passed both the House and Senate - it can now head to the Governor's desk for signing. This Council will give rare disease patients, caregivers, healthcare providers, and other stakeholders an opportunity to make recommendations to state policy makers on improving public policy for our community. Contact the Governor TODAY to share why signing this bill is important for rare disease patients and families across Arizona!

Urge Congress - Protect Crucial Funding for NIH and FDA!

Mon, 10 Mar 2025 04:00:00 GMT

The Fiscal Year 2026 (FY26) appropriations process continues! Congress has important decisions to make in the upcoming months regarding the fiscal year 2026 funding for the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). Send a message to your Senators and Representative today urging them to make rare disease health a priority by supporting robust funding for the NIH and FDA. Many treatments for rare diseases have been developed from the work done by the NIH, and FDA plays a critical role in delivering on the promise of these new treatments. It is critically important to ensure this support continues in order to help current and future generations..

We need your help funding the Rare Disease Advisory Council in California!

Thu, 06 Mar 2025 05:00:00 GMT

AB 2613 (Zbur) was passed unanimously by both the Assembly and Senate, and signed into law by California Governor Newsom in September 2024, creating the Jacqueline Marie Zbur Rare Disease Advisory Council (RDAC). This Council will give California rare disease patients, families, caregivers, providers, and other stakeholders an opportunity to make formal recommendations to state decision-makers on how to improve public policy for our community. However, the RDAC must receive funding before it can become a reality! Californians, contact your Assemblymember on the Assembly Budget Committee today to share why funding the Jacqueline Marie Zbur Rare Disease Advisory Council is important and ask for their support!

Help Move the NY Rare Disease Advisory Council Bill Forward!

Mon, 03 Mar 2025 05:00:00 GMT

You can still make a difference in advancing S1287-A (Persaud) / A1296-A (Paulin) to establish a permanent Rare Disease Advisory Council (RDAC) in New York. This critical legislation ensures that the rare disease community has a dedicated platform within state health policy discussions. The temporary Rare Disease Working Group, which recently expired, recommended the creation of a permanent advisory body to continue addressing the needs of the 1.8 million New Yorkers living with a rare disease. The bill has passed the NY Senate, a major step forward in the legislative process! Meanwhile, the Assembly Ways and Means Committee still needs to act on its Same As. Your voice is needed— If your Assemblymember serves on Ways & Means, tell them this bill is a priority and ask for their YES vote.

Protect Medicaid for Rare Disease Patients

Fri, 21 Feb 2025 05:00:00 GMT

The U.S. House Energy & Commerce Committee passed a bill that would impose significant barriers to Medicaid, threatening access to quality, affordable health care for millions of rare disease patients. Now is the time to take action- contact your Congressional representative today and urge them to vote against any cuts to Medicaid. Even if you have already taken action, now is the time to remind your representatives of the importance of this issue.

SB207 is Almost There: Ask Governor Stitt to Support Oklahoma's Rare Disease Community!

Sun, 09 Feb 2025 05:00:00 GMT

Great news! Senate Bill 207 has passed both the Oklahoma Senate and House and may soon head to the Governor's desk. This bill will establish the Oklahoma Rare Disease Advisory Council and strengthen your state's newborn screening program. Your message to the Governor can help ensure nearly 1 in 10 Oklahomans living with rare diseases are given a voice in state government.

Give Rhode Islanders a Voice in State Government

Thu, 06 Feb 2025 05:00:00 GMT

Rhode Island has the opportunity to better support individuals with rare diseases by establishing a Rare Disease Advisory Council (RDAC) through H.5023. RDACs serve as a dedicated forum for patients, caregivers, providers, and experts to inform state policymakers on the unique challenges faced by the rare disease community. Thirty states have already created RDACs, and ten more have active bills under consideration. H.5023 ensures that Rhode Island joins this growing movement by creating an RDAC that is streamlined, effective, and able to tap into state grants and funding opportunities to support its work. This legislation will help increase awareness, improve access to healthcare networks, and empower rare disease patients to self-advocate while ensuring their voices are heard in state government.

Urge Congress to get lifesaving rare disease legislation over the finish line in 2025!

Thu, 06 Feb 2025 05:00:00 GMT

As we begin this year’s legislative session, now is the time to make your voice heard on pressing issues impacting our rare disease community. We urgently need your help in advocating for several rare disease bills that aim to increase access to quality health care and treatment for those living with rare diseases.

Join the Rare Action Network

Wed, 30 Apr 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!