EveryLife Foundation @EveryLifeOrg

The rare disease community was in the spotlight last week thanks to the power of your advocacy! In case you missed it, here are last week's regulatory and access policy highlights: ➡️ The Acting Commissioner of the Food and Drug Administration (FDA) hosted a Round Table for Rare Disease Community Leaders to discuss rare disease regulatory opportunities. For more details and a summary of the meeting, see our latest newspost: everylifefoundation.org/fda-rare-disea… ➡️ Just in time for the FDA Rare Disease Roundtable, the EveryLife Foundation released its Summary Action Report coming out of the 2026 Scientific Workshop: Making What Matters Count: Advancing Patient-Centered Rare Disease Therapy Development Methodologies, Tools, and Knowledge Management. This brief summary includes several key themes and priority actions identified during the workshop, organized around the three major focus areas that guided our discussions: everylifefoundation.org/events-schedul… ➡️ The Centers for Medicare and Medicaid Services (CMS) officially released the Interim Final Rule: Medicaid Program; Community Engagement Requirement for Certain Individuals to provide guidelines for states implementing work requirements for Medicaid expansion populations. The community has the opportunity to submit comments and help shape the final rule through July 31. Please visit our website for updates and action items as we work to digest this nearly 400-page document, including updates on upcoming webinars and resources: everylifefoundation.org/medicaid-inter… We’ll continue to keep the community posted as more information and opportunities to engage become available.

The EveryLife Foundation for Rare Diseases was honored to be invited by the FDA to participate in a closed roundtable discussion with FDA leadership on Wednesday, June 3. Our Chief Mission Officer, Annie Kennedy, and Board Member, James Valentine shared some of the challenges and opportunities prioritized by our community during our Community Congress partner engagements, PDUFA Ad hoc working group prioritization, and recent Scientific Workshop discussions. Linked below is a summary of this week's conversation with the agency. We are grateful to Acting Commissioner, Kyle Diamantas, and Associate Director for Rare Disease Strategy, Amy Rick, for convening this meeting. We are optimistic about what comes next and look forward to working closely with the agency to ensure the voices of our community are heard. Read our meeting summary here: everylifefoundation.org/fda-rare-disea… Participating organizations, pictured here, include: @CureSanfilippoF, @CFRI_CureCF, @cureangelman, Friedreich's Ataxia Research Alliance (FARA), @CancerResrch, @GlobalGenes, @HaystackProject, @iamalsorg, @stopbatten , @RareDiseases, @One_Rare_, @ParentProjectMD, @UncommonCures

On June 1, 2026, the Centers for Medicare and Medicaid Services (CMS) issued the Interim Final Rule (IFR): Medicaid Program; Community Engagement Requirement for Certain Individuals to establish federal guidelines for states as they implement these requirements. Read it here: cms.gov/newsroom/fact-… While the EveryLife Foundation team is still reading through the rule to understand its impact on the rare disease community, we would like to highlight several themes that will be particularly important to our community as states implement new guidance: ➡️ First, there are several definitions in the rule around disability, medical complexity, and medical frailty, and their connection to the ability to work. We are exploring the effect of these definitions on the eligibility of our rare community. ➡️ Second, the rule includes guidelines for how and when individuals receiving Medicaid can self-attest (or sign off) that they meet the exclusion requirements without further documentation. ➡️ Third, the rule outlines how states can decide whether someone qualifies as disabled or medically complex, encouraging the use of healthcare code lists that do not account for significant numbers of rare diseases. ➡️Fourth, the rule describes caregiving exemptions for parents of young children and those caring for people with disabilities. We are working to better understand how our rare disease community can qualify for these important exemptions from work requirements. Please visit our website to read our full breakdown of the rule: everylifefoundation.org/what-our-commu… We know that Medicaid is a lifeline for our rare disease community that enables our community members to live and thrive, and we will continue to keep the community updated as more information is made available.

Ready to share your story and take part in rare disease advocacy? Join the EveryLife Foundation for Rare Diseases for a virtual session, “How to Get Involved with EveryLife,” on Thursday, June 11 at 6 PM ET. You’ll hear about our programs, events, and the many ways to engage in advocacy through the Foundation. Register now at: everylifefoundation.org/intro Want to start connecting now? Sign up for our newsletter so you never miss an opportunity: everylifefoundation.org/newsletter-sig…

We are at a critical inflection point in therapy development, with the opportunity to build on scientific momentum to unlock complex rare diseases and save lives. Our rare disease community is eager to support FDA leaders who respond to unmet patient needs with urgency by empowering review staff to deploy the full range of tools, expertise, and infrastructure at their disposal. At the same time, we are committed to working with the agency to further improve upon these capabilities as science continues to evolve. Read our recommendations to achieve a regulatory environment that maintains scientific momentum in rare disease. Click Here ➡️ everylifefoundation.org/everylife-foun… Our rare disease community is eager to engage, and we stand ready to support new leadership to ensure that every potential rare disease innovation has an optimal pathway to becoming an effective, life-altering therapy.

Final Day to Apply for the Virtual Youth and Teen Advocacy Day! RDLA invites members of the rare disease community between 10 and 18 years old with a connection to rare diseases to participate in this amazing opportunity. Advocates will meet virtually with their Members of Congress and share their rare disease story. Prior to these meetings, participants will attend virtual trainings on how Congress creates laws, how to use their voice and share their story with legislators, and how to understand key issues affecting the rare disease community, designed for youth and teens. Registration closes today, May 22. Register now using the following link: hubs.li/Q04hw90G0

📅 Yesterday, the EveryLife Foundation for Rare Diseases hosted our North Carolina State Advocacy Day, where around 25 advocates gathered in Raleigh to advocate for rare disease priorities! This amazing group asked their legislators to fully fund the Rare Disease Advisory Council (H.B.116), expand biomarker testing (H.B.567), and ensure that rare disease patients are considered as the state implements Medicaid changes due to H.R.1. Thank you to our event partners @nbd_foundation of North Carolina, @MPSSociety, and American Cancer Society Cancer Action Network (@ACSCAN).  We look forward to seeing you all next time! To learn more about rare disease advocacy work happening in North Carolina, visit our website: linkedin.com/safety/go/?url…

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We’re proud to share that the EveryLife Foundation has officially secured spots for the 2026 TCS New York City Marathon on November 1 and four incredible advocates will raise awareness, amplify voices, and carry the rare disease community across the finish line with them. Building on the momentum of our inaugural team at the United Airlines NYC Half Marathon, our endurance program continues to grow, bringing our community together in new and meaningful ways to advance our mission. In the weeks ahead, we'll introduce you to each of our team members, the moments that moved them, the people who inspired them, and why they chose to show up for the rare disease community in this way. Because this movement belongs to all of us. Whether you're cheering from the sidelines, sharing a story, or advocating from wherever you are — every voice, every action, and every act of solidarity move this mission forward. This is what community looks like. Showing up together. For all of us. #RareDisease #TCSNYCMarathon #EveryLifeFoundation #RaceForRare

@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…

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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/