RARE Foundation

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RARE Foundation We are a community of relentless advocates who drive public policy to improve the quality of life for all people living with rare diseases.

With robust, science-based evidence, we redefine what’s possible—accelerating equitable treatments and diagnosis. The RARE Foundation (formerly the EveryLife Foundation for Rare Diseases) is a community of relentless advocates, who drive public policy to improve the quality of life for all people living with rare diseases.

📣 Important Update: Today, the FDA announced it would launch a new initiative to build an improved framework for rare di...
10/14/2026

📣 Important Update: Today, the FDA announced it would launch a new initiative to build an improved framework for rare disease drug development and regulatory review.

In a white paper, the FDA outlined six guiding principles for the new framework that revolve around greater predictability and consistency, broader incorporation of the patient voice, improved use of external expertise, building training and knowledge management resources, using risk-based approaches, and championing innovative approaches.

The FDA will undertake a series of activities to collect feedback from the community, including a public workshop on October 28, a public request for information, and future sector-specific roundtables.

Today’s announcement reflects years of active and meaningful engagement from our entire rare disease community. Thank you to every family, advocate, clinician, researcher, industry partner, congressional leader and staffer, and rare disease community champion who has worked so hard to highlight the urgency and priorities of our community.

And thank you to the dedicated leaders and review teams within the FDA who have been listening to and engaging with our community.

Today represents an extraordinary inflection point for our community. And we are eager to engage with the FDA to further map out enhanced rare disease development and regulatory review processes that enable us to harness the potential in our pipelines for this generation of rare disease patients. We also look forward to the upcoming public meeting to better understand this new framework and when it will begin to be implemented.

We will continue to update the community as we receive more information. Together, as a community, we are redefining rare disease.

➡️ Learn more about today's announcement, as well as access FDA's white paper and public workshop information, by visiting our website: https://www.rareadvocates.org/news/fda-introduces-a-new-framework-for-rare-disease-review/

10/13/2026

Newborn Screening Awareness Month may have passed, but we wanted to share some wisdom from Teryn Suhr, Executive Director and Co-Founder of MLD Foundation. As a registered nurse and the parent of a child with a rare disease, Teryn knows how important newborn screening is for individual families and for public health as a whole. Listen to Teryn's testimony.

Newborn screening is more than an awareness month for our community -- it is critical for early intervention. Visit our new website to explore a number of helpful resources and to stay updated on newborn screening efforts federally and in your state: https://www.rareadvocates.org/issues/newborn-screening/

As many in the community gear up to attend APHL's Newborn Screening Symposium, we want to share more expert testimony on...
10/09/2026

As many in the community gear up to attend APHL's Newborn Screening Symposium, we want to share more expert testimony on the importance of newborn screening. Lesa Brackbill, M.A., Director of Advocacy at Patient Advocacy Strategies, and Board Member for KrabbeConnect, is a rare parent and knows how crucial newborn screening is for children like hers. Lesa also partnered with us to develop our Newborn Screening Roadmap: https://www.rareadvocates.org/newborn-screening-resource-roadmap/

10/08/2026

September might be over, but newborn screening advocacy continues! Natasha Bonhomme, Founder of Expecting Health, recently co-hosted our 8th annual Newborn Screening Bootcamp. Here Natasha describes the key to optimizing health outcomes in newborn screening. Hint: It's all about the data.

For a full recap of our Newborn Screening Recap, including a recording, visit our website: https://www.rareadvocates.org/news/recap-8th-annual-newborn-screening-bootcamp/

The RARE Foundation Is Hiring a Communications Assistant!Join our dedicated team in making a meaningful difference in th...
10/08/2026

The RARE Foundation Is Hiring a Communications Assistant!

Join our dedicated team in making a meaningful difference in the lives of more than 30 million Americans living with rare diseases.

At the RARE Foundation, we are committed to improving the lives of people and families affected by more than 10,000 known rare diseases through science, policy, and advocacy.

We’re currently looking for a Communications Assistant to join our team and support our communications efforts. If you or someone you know may be interested, please share this opportunity or visit our website to apply! https://hubs.li/Q04zyXcs0

‼️2026 Newborn Screening Bootcamp Recap & Recording Now Available!On Thursday, September 24, the RARE Foundation and Exp...
10/06/2026

‼️2026 Newborn Screening Bootcamp Recap & Recording Now Available!

On Thursday, September 24, the RARE Foundation and Expecting Health co-hosted the 8th Annual Newborn Screening Bootcamp! We welcomed 56 in-person and 89 virtual attendees, bringing together experts across the ecosystem to discuss the evolving newborn screening policy and regulatory environment.

The RARE Foundation and Expecting Health were thrilled to host our partners in newborn screening for this educational day. The best advocacy happens when our rare disease community can be in the same room and exchange ideas, concerns, and lessons. We look forward to seeing you all next year!

If you were unable to attend this year’s Newborn Screening Bootcamp, you can watch the full day’s events and read our blog here: https://www.rareadvocates.org/news/recap-8th-annual-newborn-screening-bootcamp/

Team RARE 26.2 is almost here! 🎖️ Members of our community are already registering, creating their participant pages, an...
10/06/2026

Team RARE 26.2 is almost here! 🎖️

Members of our community are already registering, creating their participant pages, and planning how they’ll complete 26.2 miles their way. We invite you to join them!

From October 12 through November 13, run, walk, bike, hike, dance, roll, or choose another way to complete your miles. Do all 26.2 at once or spread them throughout the month and bring a friend or family member along!

Every registered participant who completes the challenge will receive a Team RARE medal, with additional rewards available for reaching fundraising milestones.

Registration is free, and fundraising is optional but encouraged. Join Team RARE 26.2 today and start planning how you’ll Do It Your Way.

Proudly supported by Rocket Pharmaceuticals.

Register here: https://www.rareadvocates.org/events/team-rare-26-2/

What changed in the FDA’s updated substantial evidence guidance, and what could it mean for rare disease therapy develop...
09/29/2026

What changed in the FDA’s updated substantial evidence guidance, and what could it mean for rare disease therapy development?

The FDA recently updated an important guidance document commonly referred to as the “substantial evidence guidance.”

In our latest blog, we explore the background of the substantial evidence standard, why it matters for rare disease therapy development, and what the RARE Foundation recommended in our comments to the FDA on the draft updates.

Read blog post here: https://www.rareadvocates.org/news/the-updated-substantial-evidence-guidance/

🏅 Advocates, you are invited to participate in Team RARE 26.2, a virtual endurance opportunity with one rule: Do It Your...
09/28/2026

🏅 Advocates, you are invited to participate in Team RARE 26.2, a virtual endurance opportunity with one rule: Do It Your Way!

From October 12 through November 13, run, walk, bike, roll, hike, dance, or combine activities as you complete 26.2 miles on your own or with family, friends, coworkers, or your community.

Proudly supported by Rocket Pharmaceuticals, Team RARE 26.2 is built around connection, inclusion, and shared purpose in support of individuals and families impacted by rare diseases.

🏅 Every registered participant who completes Team RARE 26.2 will receive a Team RARE medal. Participation is free, and fundraising is optional but highly encouraged!

👟 Together, Every Mile Counts.

Register and learn more here: https://www.rareadvocates.org/events/team-rare-26-2/

What’s behind our new name and logo? 💎RARE stands for Relentless Advocates. Robust Evidence. It reflects what has always...
09/26/2026

What’s behind our new name and logo? 💎

RARE stands for Relentless Advocates. Robust Evidence. It reflects what has always powered our work: bringing together the voices of the rare disease community to drive meaningful change.

Our new diamond-shaped logo tells that same story. Its interconnected shapes represent strength, unity, and collaboration, reminding us that when we bring our distinct voices together, we create something stronger than any one of us could achieve alone.

New look. Same commitment. Together, we’re Redefining Rare Disease.

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1012 14th NW, Suite 500
Washington D.C., DC
20005

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Tuesday 9am - 5pm
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