EveryLife Foundation for Rare Diseases

Earlier this month, leaders from state-level rare disease organizations, traveling from 9 states, joined the EveryLife Foundation team in the RareHub in D.C. to participate in an annual in-person workshop. This event fostered valuable discussions and reinforced our commitment to advocating for the rare disease community. Thank you to all of the advocates who joined us! To learn more and take action please visit: https://hubs.ly/Q030pBM80

The EveryLife Foundation for Rare Diseases released the summary paper from the 2024 Scientific Workshop: Therapy Development for Small Populations: Evidence, Implications, & Policy in Characterizing Ultra-Rare. The paper highlights key takeaways, including: ✅ Defining ultra-rare characteristics ✅ Innovative trial designs ✅ Ethical considerations ✅ Risk-sharing paradigm shifts ✅ Regulatory flexibilities & real-world evidence This milestone wouldn’t have been possible without YOU! Thank you for your participation—whether in-person or virtual—in driving solutions for the rare disease community. #ScientificWorkshop 📝 Read the full summary now on our Scientific Workshop page here: https://hubs.li/Q030pwFN0

We’re thrilled to introduce the new co-chairs of the Community Congress Working Groups! These leaders bring invaluable expertise and dedication to advancing policies that impact the rare disease community. 🌟 Public Policy: Gina Loud 🌟 Regulatory: Jewell Martin 🌟 Newborn Screening: Andrew Hebert 🌟 Access & Value: Parisa Sanandaji The EveryLife Foundation's Community Congress unites patient organizations, industry leaders, and other stakeholders to drive meaningful change. With their leadership, we look forward to making strides in federal and state policy solutions for the rare disease community. Learn more: https://hubs.li/Q030mDvr0 #communitycongress

There are no words to express our heartbreak and devastation at the fact that last night’s Congressional votes did not include an extension of the Rare Pediatric Disease Priority Review Voucher Program (PRV) program. We, as a community, have done so much to ensure Congress understands the vital importance of this incentive program to ensure lifesaving therapies for rare disease patients are possible. To ensure hope is possible. We didn’t expect this outcome. Earlier this week, the progress made on the PRV Program reauthorization, as well as the Accelerating Kids Access to Care Act, by the efforts of our powerful community reaching out to Congress, sharing your stories, and raising awareness, had yielded great success. As of Wednesday morning, a five-year extension of the PRV program had been included among the health care provisions in the year-end funding package being considered by Congress. But then outside forces unrelated to our community, health care, or therapeutic development shifted the debate in Congress and caused the health care provisions to be removed. We are grateful to all who responded to our calls to action. Yesterday’s engagement alone caused Senate champions to attempt one more approach at extending the PRV overnight, even after not being included in the House package. While this attempt was also unsuccessful, it again demonstrated the power of our collective advocacy and the fact that we have garnered some steadfast champions on Capitol Hill. This is our community’s new reality, but we will continue to work with you and our champions in Congress to right this wrong. Progress has been made on the PRV Program, thanks to the efforts of over 200 patient organizations and thousands of advocates who have shared their stories and made their voices heard. And we’re not stopping. Our community deserves every opportunity for treatments. We can’t afford to have our lives caught in the middle of political jockeying. We don’t have the luxury of time. This experience has only strengthened our commitment to this community – and today we begin working to identify our path forward. We will need every member of this community to continue to advocate alongside us. United. Amplified. Stronger than ever.

📢 This is NOT a drill. The Rare Pediatric Priority Review Voucher #PRV Program is set to expire TODAY unless Congress acts and we need your help. 📞 Call the U.S. Capitol Switchboard - (202) 224-3121 Use the script below: “Hi my name is _____________ from____________. I’m a member of the rare disease community and am calling today to urge you to include an extension of the Pediatric PRV program in the continuing resolution because it gives kids hope for treatments and it doesn’t cost taxpayers or the government money. Please vote to support the Rare Pediatric PRV extension in the continuing resolution.” Hear Annie Kennedy, EveryLife Foundation's Chief of Policy, Advocacy, and Patient Engagement, explain why we need your voice to extend the #PRV program TODAY! #Cures4RareKids

Thanks to our friends at Biotechnology Innovation Organization for highlighting the EveryLife Foundation for Rare Diseases event about PRV and other life-saving legislation that must be acted on by Congress immediately! These children's incredible stories illustrate perfectly why the stakes are so high.

URGENT ACTION ALERT - Call your members of Congress and tell them the rare disease community needs them to pass the end-of-year legislative package now. The newly released draft bipartisan legislative package proposes a 5-year extension of the Rare Pediatric Disease Priority Review Voucher Program and other critical rare disease policies, like the Accelerating Kids Access to Care Act! There is an urgent need to contact members of Congress to let them know how important it is to pass the end-of-year legislative package now.    Let’s keep up the momentum and get these policies over the finish line! Click here to find your members of Congress and place a call today. https://lnkd.in/gCxsFtmY

📢 Exciting #PRV Update! The newly released draft bipartisan legislative package proposes a 5-year extension of the Rare Pediatric Disease Priority Review Voucher Program and other critical rare disease policies, like the Accelerating Kids Access to Care Act! We now urge Congress to act swiftly and pass this vital package into law this week.   This milestone wouldn’t have been possible without the tireless efforts of 200+ patient organizations and the thousands advocates who’ve activated, connected with their members of Congress, and shared their powerful stories. And while not yet passed, a huge thank you to our champions in Congress for their leadership. Your voices are driving hope and innovation for kids with rare diseases everywhere.   Let’s keep up the momentum and get these policies over the finish line! https://lnkd.in/gyQJv_K3 #Cures4RareKids!

As the year comes to an end, we celebrate 15 years of impact and reflect on EveryLife Foundation's journey toward creating real, lasting change for the rare disease community. Thanks to advocates like YOU, we’ve made strides in life-saving newborn screening, driving critical policy reforms, and supporting programs that bring hope and treatment to families nationwide. 💡 Your support changes lives. Lives like Anneliese’s and Paloma’s. 🌟Double Your Impact! Now through the end of the year, every donation will be matched dollar-for-dollar—up to $50,000—thanks to the generosity of our Board of Directors. Together, we can make an even greater difference for individuals and families living with rare diseases. Learn more: https://hubs.li/Q02_Qf5q0

Congratulations to the 2024 RareVoice Awards recipients! All of our nominees are true rare disease champions. #RareVoice2024 Thank you to the rare disease community for nominating these outstanding individuals and thank you to the nominations committee who performed the difficult task of selecting finalists and awardees in each category. ✨ Missed the show? Check out the show recording here: https://lnkd.in/gwuQ7-5m