EveryLife Foundation

@RepJohnJoyce @SenatorWicker @RepBarragan

On May 13, the EveryLife Foundation hosted our inaugural Community Congress Leader Hill Day. These powerhouse advocates, who earlier in the week participated in the EveryLife Foundation's Scientific Workshop, met with key Congressional offices to preview our rare disease community's policy priorities as we prepare to engage in PDUFA VIII legislative activities. This united group of national rare disease patient advocacy organization leaders, who have also been active in coalition regulatory discussions, included: Andrew Rosen, @NAF_Ataxia; Brandi Underwood, International FOXP1 Foundation; Cara O'Neill, @CureSanfilippoF; Fabienne Antoine-Nasser, @SickCells; Jess Myers, @US_HAEA ; Dr. Kim Stephens, @Projectalive; Lynn Hano Albizo, @immunedeficienC; Pat Furlong, @ParentProjectMD; Roxanne Yaghoubi, @EpilepsyFdn; Ryan Fischer, Foundation for Angelman Syndrome Therapeutics (FAST); Terri L. Klein, @MPSSociety; Allie Ladd, @CureMPS1, Inc., and Tricha Shivas, Foundation Sarcoidosis Research. We are so grateful for their participation! And thank you to all members of our Community Congress whose efforts and expertise contributed to the policy priorities reflected in these Hill discussions. Our #raredisease community leadership’s collective voice is a force multiplier at a time when regulatory momentum is most critical. And we are just getting started. To learn more about Community Congress, visit: everylifefoundation.org/community-cong… To stay updated on what's happening in our community, sign up for our monthly newsletters: everylifefoundation.org/newsletter-sig…

Our biennial #ScientificWorkshop is a wrap! Thank you to our panelists for a great afternoon of discussions. Kicking things off, #raredisease stakeholders shared their experience navigating the regulatory process, focusing on the utilization of patient-relevant clinical endpoints. We also explored innovative trial designs that enable benefit capture as well as broad patient inclusion, taking a look at a number of relevant case studies. And the afternoon concluded with a robust discussion around increasing cross-stakeholder understanding about evidentiary thresholds and regulatory requirements in order to put together an action plan. Patient advocacy leaders and industry partners were joined by representatives from @fdacder. Thank you to everyone who participated in and attended the 2026 Scientific Workshop. The #EveryLife team will pull together a comprehensive recap in the coming weeks to share with the full community.

Applications are now open for the Paula Kovarick Segalman Family Scholarship for ALS, part of the EveryLife Foundation’s Community Scholarship Program. This renewable scholarship supports students living with ALS, or with an immediate family member with ALS, pursuing higher education during the 2026–2027 school year. Learn more and apply by June 18, 2026, at 2 PM ET: hubs.li/Q04gjyv20. Local to the DMV? Learn more about the Segalman family’s event supporting the scholarship this week on the scholarship website.

Registration for Rare Across America 2026 is officially open! Meet with your Members of Congress virtually and at their in-district offices and educate them on the issues that are most important to the rare community by sharing your story. Meetings will take place from August 10 to 21, and registration closes on Friday, July 17. Make an impact in your community at #RareAcrossAmerica2026 Register here: hubs.li/Q04gjsmq0

This morning's #ScientificWorkshop kicked off with panelists discussing the question: Roadmaps have been built but how do we create freeways? Answer: Early communication with regulators is key and the #raredisease patient voice needs to be included in decision making. We were also joined by panelists from @FDACBER and @fdacder providing their perspectives on patterns in rare disease therapy development. Our last morning session included PAG leaders, industry, and regulatory experts advocating for earlier endpoints to unlock #acceleratedapproval. Afternoon sessions are underway! Reminder: #EveryLifeFoundation will provide a full recap of today's workshop for the community in the coming weeks.

Our SVP of Policy + Advocacy, Jamie Sullivan kicks off the 2026 #ScientificWorkshop focused on advancing patient-centered #raredisease therapy development methodologies, tools, and knowledge management.

Today, we welcome leaders from the rare disease patient, scientific, government, and biopharma communities to Washington, DC, for our 15th Scientific Workshop, "Making What Matters Count: Advancing Patient-Centered Rare Disease Therapy Development Methodologies, Tools, and Knowledge Management," for specific community partners. Expert panels will review case studies on topics such as innovative clinical trial designs, novel surrogate endpoint development, and patient-centered clinical endpoints, discuss what enabled these achievements, and brainstorm ideas for creating more consistent and scalable progress across the rare disease community. From these discussions, we will produce an action report highlighting participants' suggestions for improving knowledge management systems, regulatory guidance, and legislative policy. To learn more about the Scientific Workshop Series and to view select recordings and the action report following the Workshop, please visit our website: everylifefoundation.org/events-schedul… Thank you to all of our 2026 Sponsors! This year's event is presented by @AlexionPharma. Our Priority Review sponsors are Otsuka Pharmaceuticals, @SanofiUS, and @Sarepta. Our Clinical Trial sponsors include @alliancerm, @AstellasUS, @IAmBiotech, @BridgeBioPharma, Burroughs Wellcome Fund, @ChiesiGroup , @HarmonyBio, ITF Therapeutics LLC, @IQVIA_global, @RareCoalition, Stealth Bio Therapeutics, @TakedaPharma, @ultragenyx, and @VertexPharma . Our Discovery sponsors include Azafaros, Beren Therapeutics, @FulcrumTx, @PalvellaTX, @Regenexx, and @StokeTx. We couldn't have done this without you!

The EveryLife Foundation submitted comments in response to the FDA’s draft guidance on the Plausible Mechanism Framework for the development of individualized therapies targeting specific genetic conditions with known biological causes. This framework is a set of recommendations to help those developing individualized therapies generate sufficient evidence that they are safe and effective, and that they can be manufactured appropriately. Our comments reflected input from our Community Congress coalition and included requests for FDA to clarify how the Framework will be applied to broader populations and technologies and suggestions for how to maximize the impact of the Framework, including: leveraging the Rare Disease Innovation Hub to convene stakeholders, publishing scenario-based case studies, ensuring adequate FDA staffing and scientific expertise, encouraging shared data platforms to leverage preclinical/toxicology/manufacturing knowledge, and clarifying how patient preference/experience data and patient organizations will be incorporated. We also urged continued investment in natural history studies, biomarkers, and clearer evidence standards for surrogate markers, including a more streamlined qualification pathway. To read our full comments, visit our website: everylifefoundation.org/the-everylife-…

‼️ The EveryLife Foundation has signed two letters highlighting the importance of Medicaid to our rare disease community. The patient advocacy and disability communities are united in urging Congress to avoid further cuts to critical healthcare programs in the budget reconciliation process. State Medicaid programs are facing difficult decisions as they begin implementing changes passed in last year's One Big Beautiful Bill Act (H.R. 1). Some states have proposed cutting critical Home and Community-Based Services programs that our community relies on to receive care in our homes, rather than being forced into institutional settings. Lately, we've seen claims that HCBS programs are vulnerable to fraud, and that hard-fought policy advances, such as paid family caregiving services, should be eliminated. For the rare disease community, Medicaid's Home and Community-Based Services programs mean greater independence, better quality of life, and care that actually reflects the needs of patients and families. Protecting program integrity is important, but it should never come at the expense of the people who rely on these services every day. As we continue to work on both the state and federal level to protect Medicaid funding, including HCBS programs, we urge you to share your Medicaid story and highlight the program's impact on the rare disease community ➡️ everylifefoundation.quorum.us/campaign/14592… To read both of the letters, visit our website ➡️ everylifefoundation.org/policy-papers/

📣 Last call! Applications for the #RAREis Scholarship Fund close on Tuesday, April 28, at 2 PM Eastern. 58 individuals in the rare disease community will receive $5,000 scholarships to support their education this fall. Don’t miss this opportunity to take the next step in your education and career. Apply now at hubs.li/Q04dbD1J0.

‼️ Today, Congress is taking the first step on appropriations to fund the Food and Drug Administration’s FY27 operations, including the critical work the FDA does to evaluate therapies for rare diseases. In advance of the markup, the EveryLife Foundation submitted a statement to the subcommittee, urging lawmakers to support the inclusion of funding for the Rare Disease Innovation Hub, an ask first made by the hundreds of advocates who joined us in Washington, DC for Rare Disease Week 2026. The rare disease community is requesting that the Hub receive $5 million in funding to carry out its agenda, which includes hosting additional RISE Workshops, creating opportunities for rare disease patient organizations and experts to inform the therapy development and evaluation process, and streamlining navigation of the FDA’s rare disease resources. Dedicating funds to the RDIH ensures that the Hub can continue to grow its role in improving coordination across the FDA, leading to more consistent approaches to rare disease therapy development. This request is supported by more than 25 Congressional members who sent a letter to the subcommittee in March, and we look forward to continuing to work with Congress to ensure that the Rare Disease Innovation Hub is resourced and can work to its fullest potential. Share your support for rare disease funding at the FDA ➡️ everylifefoundation.quorum.us/campaign/15729… Read the full statement on our website➡️ everylifefoundation.org/the-everylife-…

The #RAREis Scholarship supports individuals with rare diseases in building their dreams. Beyond financial support, recipients are invited to participate in a mentorship program that connects them with peers who share similar experiences or areas of study. Apply by April 28 at 2 PM ET at hubs.li/Q049_HtF0

‼️ Last Friday, April 2, the White House released the President’s proposed Budget for Fiscal Year 2027. It proposes $15.8 Billion in reductions to Health and Human Services (HHS), including significant funding cuts to key agencies for the rare disease community, and an increase to the Food and Drug Administration (FDA). This budget is by no means final. Congress views the President's budget as a starting point for the budget process, but often chooses to draft based on Congressional priorities instead. While there is still much we will learn and distill over the coming days and weeks as negotiations on the Hill evolve, we wanted to take this opportunity to provide an overview of the significance of the release of the President’s Budget Proposal and the process that comes next. To read the full post, please visit our website ➡️ everylifefoundation.org/the-presidents… Our EveryLife Foundation team is here with you. We will keep you updated as new developments are shared, hearings are posted, and key opportunities arise to ensure that the funding priorities of our community are heard on the Hill.

‼️Attention California residents‼️ The California General Assembly has introduced A.B. 1798, a critical bill that protects patients from genetic discrimination and ensures continued participation in lifesaving research. Members of the Assembly Committee on Insurance have the opportunity right now to support this important legislation. Your story matters! Share why genetic nondiscrimination matters to you and how it impacts your life. Urge committee members to support this bill by filling out our action alert ➡️ everylifefoundation.quorum.us/campaign/15954… Read more about the bill ➡️ calmatters.digitaldemocracy.org/.../ca... We look forward to following this bill's progress as it advances through the California legislature.

📅 The RISE Workshop 3 convened on March 30, 2026, to discuss optimizing data sharing for therapy development and the collaboration, infrastructure, and education opportunities to promote data sharing as a consistent reality in the rare disease space. The EveryLife Foundation was delighted to support Duke Margolis Institute for Health Policy and the FDA Rare Disease Innovation Hub during the planning of this workshop, as the meeting topic was based on topic submissions from the community, including our July submission to the Federal Register. Annie Kennedy, Chief Mission Officer, was invited to provide opening remarks to help set the tone for the day's discussions. These hybrid RISE workshops are co-convened by the @DukeMargolis Institute for Health Policy and the U.S. Food and Drug Administration (@FDA) Rare Disease Innovation Hub under a cooperative agreement with the FDA. RISE workshops bring together innovators in drug development, rare disease research, patient advocacy, and regulatory science to discuss challenges in the development of medical products for rare diseases that are common to multiple rare diseases or a class of diseases and for which evolving science offers innovative solutions. Thank you to our partners at the Rare Disease Innovation Hub and at Duke-Margolis for convening this workshop and facilitating important conversations around this topic. To learn more about this week's workshop, click here ➡️ everylifefoundation.org/rise-workshop-…