EveryLife Foundation for Rare Diseases

03/12/2026

The NYC Half is almost here!

This weekend the EveryLife Foundation Run Team will run on behalf of the 30 million Americans living with rare diseases.

Help us cheer them on and support their mission.

🔗 Make your donation today! https://hubs.li/Q046qwt40

03/05/2026

The EveryLife Foundation urges Congress to support funding and report language in FY 2027 for the Rare Disease Innovation Hub. The Hub facilitates collaboration between the FDA’s Center for Biologic Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) to improve outcomes for patients living with rare diseases. Our Chief Mission Officer, Annie Kennedy, urged Congress to resource the Hub in her witness testimony at the Senate Special Committee on Aging last Thursday during Rare Disease Week 2026.

Currently, only 5% of the more than 10,000 rare diseases have FDA-approved treatments. The approval process for rare disease drugs requires special knowledge and skills across FDA departments. Without clear collaboration, it is challenging to compete the approval process in a timeframe that meets rare disease patients’ needs. The Hub streamlines collaboration between departments to ensure the agency can meet patients' needs.

With proper funding and clear guidelines, the Rare Disease Innovation Hub can improve the FDA’s rare disease drug approval process and transform the lives of patients nationwide.

Encourage your Member of Congress to support funding for the Rare Disease Innovation Hub here: https://hubs.li/Q045KnwS0

To watch the full Senate Special Committee on Aging hearing here: https://hubs.li/Q045KrHK0

03/04/2026

We’re heading into the final stretch before the New York City Half Marathon!

Six dedicated runners are taking on the NYC Half on behalf of the EveryLife Foundation for Rare Diseases, running in honor of the 30 million Americans living with a rare disease and the families, caregivers, and advocates who stand beside them every day.
Because of this incredible community, we’ve already reached 84% of our fundraising goal. With race day quickly approaching, we’re inviting you to help us cross the finish line together.

✨ Support their journey with a donation here: https://hubs.li/Q045CP3y0

Every gift fuels our mission, advancing evidence-based policy, accelerating science-driven research, and strengthening the rare disease community so that no one has to navigate their journey alone.
Thank you for cheering on our runners and standing with the rare disease community every step of the way.

02/27/2026

What a way to spend the final days of Rare Disease Week 2026! Despite a little drizzle and some gray clouds, hundreds of rare disease advocates participated in more than 300 Hill visits on Thursday to share their stories and advocate for rare disease policies.

Yesterday started with our community in the Senate spotlight. The EveryLife Foundation for Rare Diseases was honored to help represent the community by participating in the Senate Special Committee on Aging’s hearing on the effects of FDA delays on rare disease therapy development. Annie Kennedy, our Chief Mission Officer, joined other expert witnesses, Dr. Jeremy Schmahmann, Director of Massachusetts General HospitalAtaxia Center; Bradley Campbell, President & CEO of Amicus Therapeutics; and Dr. Cara O'Neill, Chief Science Officer and Co-Founder of the Cure Sanfilippo Foundation, to give expert testimony.

We would like to extend our deepest gratitude to Chairman Scott, Ranking Member Gillibrand, Senator Johnson, Senator McCormick, Senator Alsobrooks, and Senator Kim for the important dialogue in this hearing.

The expert witnesses focused on issues regarding the utilization of existing regulatory tools, such as the accelerated approval pathway and advisory committees; the need for consistency and transparency in the drug review process; ensuring ethical practices in rare disease clinical trials that do not result in unnecessary disease progression; and increasing domestic manufacturing transparency and speed to fulfill the United States' innovation capabilities.

Our message, our stories, and our urgency were heard. Conversations with the Senate Special Committee on Aging will continue in the coming days. We will keep you updated on any future Committee interactions.

For more information, visit our website: https://everylifefoundation.org/rare-disease-week-2026-senate-hearing-recap/

There is still time to share your story about how regulatory delays and the lack of treatment options have affected your life before the Committee’s deadline. Share your story before March 3: https://everylifefoundation.quorum.us/campaign/155587/

02/24/2026

On Monday, the U.S. Food and Drug Administration released more details about the Plausible Mechanism Framework, first announced by FDA Commissioner Makary and Director Prasad in the New England Journal of Medicine in November 2025.

The Plausible Mechanism 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.

The EveryLife Foundation team will closely review the draft Guidance, alongside the Community Congress Regulatory Working Group in the coming weeks and together, we will continue to press for policy solutions that acknowledge the differences in how rare disease products must be developed and evaluated. Today’s release of the Plausible Mechanism Framework Draft Guidance was a meaningful step in that direction.

To learn more about yesterday's event at the U.S. Department of U.S. Department of Health and Human Services (HHS) visit: https://everylifefoundation.org/fda-takes-important-step-toward-individualized-therapies-2/

02/23/2026

Happening soon!

Tomorrow at 3 pm ET, The Washington Post is hosting conversations about the next frontiers of rare disease research.

Representative Doris Matsui, co-chair of the Rare Disease Congressional Caucus, joins Mark McClellan, the Duke-Margolis Institute for Health Policy, for one conversation. Then, our Chief Mission Officer, Annie Kennedy, joins Cate McCanless, Chief Corporate Affairs Officer at Harmony Biosciences, for a discussion focusing on the role of patients in rare disease policy in addressing their urgent needs.

Register to watch here: https://hubs.li/Q044fQbF0

02/20/2026

‼️Our Chief Mission Officer, Annie Kennedy, will be testifying at the Senate Special Committee on Aging's hearing that looks to examine the FDA's role in the rare disease community.

The hearing will be held on February 26 at 9:30 am in the Hart Senate Office Building, room SH-216. If you are attending Rare Disease Week, join us for a breakfast and a meet and greet at 8:30 am in the Dirksen Senate Office Building, Room G-11, before or between your Hill meetings. If you are not attending Rare Disease Week and would still like to tune in, you can watch the livestream here: https://www.aging.senate.gov/hearings

In recent months, several difficult regulatory decisions have affected clinical trials and review timelines for products with direct implications for the rare disease patient community. Remember, your story can help inform what happens next! The Aging Committee is collecting stories about what rare disease research and treatments mean to patients, highlighting the real-world impact of regulatory delays on individual rare disease communities.

Share your story here ➡️
https://everylifefoundation.quorum.us/campaign/155587/

02/18/2026

‼️ The Senate Special Committee on Aging has announced it will hold a hearing to examine the FDA's role in the rare disease community.

The hearing will be held on February 26 at 9:30 am in the Hart Senate Office Building, room SH-216. If you are attending Rare Disease Week, join us for a breakfast and a meet and greet at 8:30am in Dirksen Senate Office Building, Room G-11 before or between your Hill meetings. We will be watching a livestream of the hearing from this room as well, if the main room reaches capacity. If you are not attending Rare Disease Week, you can watch the livestream here: https://www.aging.senate.gov/hearings

❓ Why this Hearing is Important
In recent months, several difficult regulatory decisions have affected clinical trials and review timelines for products with direct implications on the rare disease patient community (for example, complete response letters, refusals to file, and incomplete response letters).

If you are a member of a patient community affected by these decisions, we want to hear from you. To help inform the hearing, the Aging Committee is collecting stories about what rare disease research and treatments mean to patients, highlighting the real-world impact of regulatory delays on individual rare disease communities.

Share your story here ➡️ https://everylifefoundation.quorum.us/campaign/155587

EveryLife Foundation for Rare Diseases

02/16/2026

Why is Katie Kundrat running:

"Katie and Kathleen are running together as a fundraising team, combining their efforts to support the rare disease community and amplify their collective impact.

I run because every day I work with children and families navigating rare neuromuscular conditions, and I see how much strength and perseverance their journeys require. The families and patients endless dedication to their care and improving their quality of life, inspires me to move and run for this cause. As a physical therapist on the neuromuscular clinical team at Children’s National, I support patients as they work toward mobility, strength, and maintaining quality of life and those experiences stay with me long after the clinic day ends.

Running with the EveryLife Foundation feels like another way for me to stand alongside the patients and families I care for. This race is about showing up, raising awareness, and helping advance a future where individuals affected by rare disease have the support, research, and resources they deserve. I am proud to represent a foundation that embodies the same values my patients exhibit every day."

To help Katie reach her goal, click here: https://hubs.li/Q043cy7H0

To learn more about the rest of the EveryLife Foundation for Rare Diseases Run Team, click here: https://hubs.li/Q043cybp0

02/12/2026

Why is Katie Barrett-Stevens running?

"Running has always been a way that I could connect my mind and body to be fully present in both. I started running when we lost my brother-in-law in a hiking accident – if he could no longer run, then I would. This activity has deep meaning as a mother who has worked to find the best treatments for her child and in turn became the first Executive Director of the leading patient advocacy organization for Telomere Biology Disorders. Running means I run for and beside those in the rare community. If they can’t run, then I will. To put it simply, I love it and it allows me to think about all that my life and career have asked of me and to show up in the best way possible for myself and those I love and serve."

To help Katie reach her goal, click here: https://fundraisers.nyrr.org/fundraisers/85a6b8c627920c26745e

To learn more about the rest of the EveryLife Foundation for Rare Diseases Run Team, click here: https://everylifefoundation.org/run/?utm_content=368558642&utm_medium=social&utm_source=linkedin&hss_channel=lcp-16143911

02/05/2026

Why is Donta running:

“I wanted to run the NYC Half with the EveryLife Foundation because their mission aligns deeply with my commitment to advocacy, equity, and community-driven impact. Rare disease patients and families too often navigate fragmented systems without adequate support or representation, and the EveryLife Foundation works tirelessly to change that by amplifying patient voices and advancing meaningful policy solutions. Running this race is my way of honoring the resilience of the rare disease community and helping ensure that lived experience is at the center of research, policy, and care. Every mile represents progress toward a future where no rare disease patient is left behind.”

To help Donta reach his goal, click here: https://hubs.li/Q0420hY-0

To learn more about the rest of the EveryLife Foundation for Rare Diseases Run Team, click here: https://hubs.li/Q0420gNp0

02/05/2026

Hiring Now!

The EveryLife Foundation for Rare Diseases is hiring for a Director of Events. We are looking for an experienced leader in event management, preferably in a non-profit, healthcare, or advocacy setting. The EveryLife Foundation hosts in-person, hybrid, and virtual events that empower rare disease advocates to make their voice heard with policymakers. The Director of Events is the bridge between complex policy work and the people it impacts.

Interested applicants can use the following link to learn more about the position and apply: https://hubs.li/Q041_Nxf0