On the last day of February every year is Rare Disease Day, a dedicated day to celebrate the rare disease community, including patients, families, caregivers, clinicians, researchers, regulators, and more. The day inspires multiple events, such as fundraisers and scientific symposia, to bring together all the critical stakeholders who tirelessly work to make life better for those affected by rare diseases. The goal is to build community, share ideas and resources, and advance research and innovation.
How Worldwide Clinical Trials’ Rally for Rare Event Came About
One of the most influential events in rare disease is the FDA–NIH Rare Disease Day conference, which allows the rare disease community to collaborate directly with leading scientists, researchers, and physicians from within the NIH and FDA regulators to showcase industry success and goals for the future. Unfortunately, this year’s event, scheduled for February 27-28, 2025, in Washington, DC, was abruptly postponed less than one week before it began.
This conference often sets a precedent for global engagement and awareness in rare disease research, discussing endpoints, and removing barriers and red tape to help drive research forward. That’s why Worldwide Clinical Trials was committed to ensuring the rare disease community still had a space to share knowledge, resources, and support on Rare Disease Day 2025.
When the FDA announced the event cancellation, our rare disease team and members of our marketing team quickly came together to find a solution to this unexpected hurdle. In an incredible feat of collaboration, they were able to rapidly put together a new event called Rally for Rare. With the goal of complementing and supporting the efforts of the FDA and NIH, the team secured a venue, gathered speakers and presenters, spread the word about the event through swift marketing efforts, and secured registrations within just four days.
Our community-led event served as the perfect opportunity for those planning to attend the FDA-NIH conference to continue the conversation. At our nearby venue at the Hyatt Regency Bethesda in Maryland, on February 28th, 2025, the presentations included:
| You Are Not Alone | Michele Herndon, DNP, RN, Interim Director, Undiagnosed Disease Network Foundation |
| Beyond DNA and Diagnosis: The Expanding Role of Genetic Counselors in Advocacy | Ashley Thompson, MS, CGC, Founder and Principal Consultant, ATGC Insights, LLC |
| Funding, Progress, and the Unknowns: The Porphyria Research Journey: A Patient Advocacy Perspective on Research Sustainability | Kristen Wheeden, MBA, MPH, PhD, President, United Porphyrias Association |
| Patient-Led Research: Creating Meaningful Endpoints | Gabi Conecker, Executive Director and Cofounder, Decoding Developmental Epilepsies JayEtta Hecker, Board Chair and Cofounder, Decoding Developmental Epilepsies |
| The Rare Disease Diagnostic Odyssey and Innovations in Genetic Testing | Lindsey Wahlstrom-Edwards, Head of Patient Engagement and Strategic Partnerships, Sano Genetics; Cofounder and Chief Momatologist, Rona’s FUN LAB Cody Barnett, MPH, Patient Advocacy Specialist, Illumina Lukas Lange, PhD, CEO and Cofounder, Probably Genetic |
| Corporate America and Time Off for Clinical Trials | Mary McGowan, CEO, Foundation for Sarcoidosis Research |
| The Patient-Led Revolution | Susannah Fox, Author of Rebel Health (MIT Press) |
| Translating the Sibling Experience | Noah Siedman, Community & Database Manager, BDSRA Foundation |
To view complete presentation information and videos, fill out the form to get exclusive access.
The response to this event from the rare disease community has been overwhelming and positive. It was a fantastic forum to encourage collaboration between advocates and researchers, and we look forward to the potential of organizing again in 2026!
The Rare Moments
In addition to our Rally for Rare event, Worldwide facilitated Rare Moments, a platform for those in the rare disease community to share their stories, experiences, and even hopes for the future to help others expand their understanding of what it means to live with and work with rare diseases.
Here are some notable moments from 2025:
Find Out How Worldwide Can Help Advance Your Rare Disease Research
As a CRO dedicated to helping accelerate treatments for patients in need, Worldwide is proud to share that over half of the clinical studies we support are in rare and ultra-rare indications. We welcome opportunities to engage with the rare disease community and help contribute to the conversation between advocates, researchers, drug developers, and more.
For any collaboration inquiries or questions about the 2025 Rally for Rare event, reach out to rare@worldwide.com.
