Current Actions

Urge Your Senators and Representative to Cosponsor the RARE Act!

Sat, 13 Apr 2024 04:00:00 GMT

Since the inception of the Orphan Drug Act more than 40 years ago, the FDA has interpreted orphan exclusivity, a key incentive for rare disease drug development, to be granted based on the indication for which the drug was actually shown safe and effective, rather than on the much broader orphan drug designation. A recent court case questioned this interpretation, creating uncertainty about the breadth and scope of orphan drug exclusivity. The RARE Act would cement FDA’s long-standing interpretation of the ODA, maintaining critical incentives for continued orphan drug research and innovation.

Contact your elected officials TODAY and urge them to cosponsor the RARE Act to help incentivize the development of safe and effective treatments for all rare disease patients!

Urge Your Representative to Cosponsor the Creating Hope Reauthorization Act (H.R. 7384)

Sat, 13 Apr 2024 04:00:00 GMT

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 Creating Hope Reauthorization Act (H.R. 7384) would reauthorize the Pediatric Rare Disease Priority Review Voucher program for four more years. However, this legislation must pass before the September 30, 2024 deadline to ensure this important tool in ongoing efforts to address the significant unmet treatment needs that exist in the pediatric rare disease population is not lost.

Contact your Representative TODAY and urge them to cosponsor the Creating Hope Reauthorization Act!

Mississippi, Tell the Conference Committee the Time for Medicaid Expansion is Now

Mon, 04 Mar 2024 05:00:00 GMT

The Mississippi State House has passed House Bill 1725 which would expand health care coverage to up to 200,000 Mississippians by expanding the State’s Medicaid program. Mississippi is one of 10 states that have yet to take advantage of Medicaid expansion incentives. Your State Senator must hear from you – the person they represent. Reach out to them today and tell them they could change the reality for rare disease patients and families living in Mississippi and give them the chance they deserve to live a fulfilling, long and healthy life. Tell them the time to expand Medicaid is NOW!

Vermonters, Step Therapy Reform is Critical! Tell Senate Appropriations It's Time to Pass H.766!

Fri, 01 Mar 2024 05:00:00 GMT

Step therapy, sometimes referred to as "fail first,” is a process that requires patients to take one or more alternative medications chosen by their insurance provider before they can access the medication that was prescribed by their health care provider. This can cause delays to necessary care and may be harmful to patients. H.766 would protect patients who obtain insurance coverage through private insurance providers by establishing an exemption process, mandating certain exceptions to step therapy protocols, and requiring a timeframe for insurance plans to respond to exemption or appeal requests. The bill passed the House UNANIMOUSLY on March 13, 2024. It needs your support to get through the Senate. Contact Members of Senate Finance now to ask for their support of this bill and to reject amendments put forward by healthplans to waterdown critical patient protections that are a part of H.766.

California, Help Establish a Rare Disease Council!

Fri, 01 Mar 2024 05:00:00 GMT

Legislation to create a Rare Disease Advisory Council in California, AB 2613, was recently introduced by Assemblymember Zbur. Once enacted, this Council will give rare disease patients, families, caregivers, providers, and other stakeholders an opportunity to make formal recommendations to state policy decision-makers on how to improve public policy for our community. Contact your Assemblymember today to share why creating a Rare Disease Advisory Council is important and ask that AB2613 be advanced swiftly through the Legislature.

New Yorkers, Tell Governor Hochul You Need a Permanent and Robust RDAC

Fri, 01 Mar 2024 05:00:00 GMT

As part of the 2024 State of the State, Governor Kathy Hochul announced her intent to make New York State’s Rare Disease Working Group a permanent Rare Disease Advisory Council. New York State created its working group in 2019 however, due to the State’s unprecedented and critical response to the COVID-19 Pandemic, the group did not meet for the first time until March of 2023. We need your help to see New York’s Council made permanent. Reach out to Governor Hochul today and tell her rare New Yorkers have not seen their Council reach its full potential and need a permanent and robust Rare Disease Advisory Council.

Share Your State's State Report Card! Let Your Elected Officials Know How the State is Doing Serving the Rare Community!

Fri, 23 Feb 2024 05:00:00 GMT

Join us in sharing your state’s grade on rare disease policies with your elected officials.

One in 10 Americans are living with a rare disease, and half of these individuals are children. Studies show our community members face higher out-of-pocket health care costs than non-rare patients; unnecessarily long and challenging roads to an accurate diagnosis; and frequently must travel great distances or across state lines to receive necessary medical care.

Improving the lives of those living with a rare disease starts with advocacy and sharing your story. Join us in telling your elected officials why they should prioritize caring about rare disease issues. Then visit our take action page for opportunities to improve your state’s policies (check back often, as new action alerts are added frequently).

Ask Congress to Support Funding for the National Institutes of Health in Fiscal Year 2025!

Fri, 23 Feb 2024 05:00:00 GMT

The Fiscal Year 2025 (FY2025) appropriations process is getting started! Congress has important decisions to make this year regarding the FY 2025 funding for the National Institutes of Health (NIH). 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. Many treatments for rare diseases have been developed from the work done by the NIH, and it is critically important to ensure this support continues in order to help current and future generations.

Ask Your Members of Congress to Cosponsor the PASTEUR Act!

Wed, 13 Sep 2023 04:00:00 GMT

The Pioneering Antimicrobial Subscriptions to End Upsurging Resistance (PASTEUR) Act (H.R. 2940 / S. 1355) is a bipartisan bill that will bolster a new era of antibiotic research and development into novel treatments.

The PASTEUR Act will bring much-needed antimicrobials into the hands of patients and all Americans those who need them. Momentum has been gaining in Congress on this issue, but our help is needed to move the needle further. Urge your members of Congress to cosponsor and help pass this bill without delay.

Ask Congress to Support the Safe Step Act!

Wed, 15 Mar 2023 04:00:00 GMT

Step therapy, sometimes referred to as "fail first,” is a process that requires patients to take one or more alternative medications chosen by their insurance provider before they can access the medication prescribed by their health care provider. When used inappropriately, step therapy can prevent patients from accessing critical treatment. The Safe Step Act would protect patients who obtain insurance coverage through private insurance providers by establishing an exemption process, mandating certain exceptions to step therapy protocols, and requiring a timeframe for insurance plans to respond to exemption or appeal requests. Contact your elected officials today in support of this important legislation!

Join the Rare Action Network

Wed, 17 Apr 2024 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!