From VOICES to BREAKTHROUGHS
October 19–21 | Washington, DC
With Cyndi Powers, CMP
Learn more about NORD at rarediseases.org.
Pre-Summit Membership Meeting
The Summit began with the NORD Member Meeting. I am guessing there were over 150 NORD members from the rare disease community in the room. A highlight was the panel, “Executive Insights: Balancing Vision, Resources, and Well-Being,” which explored ways to manage teams, retain staff and volunteers, leverage technology, and build sustainable organizations. NORD leadership also presented member feedback collected over the past year, and attendees contributed real-time input on their top priorities for 2026—many aligning with NORD’s own strategic goals. The meeting closed with lively roundtable networking, where discussions ranged from fundraising and community engagement to artificial intelligence and patient registries. That evening’s welcome reception celebrated trailblazers across the rare disease community, honoring organizations and individuals who make a lasting impact. Later, at the Rare Cancer Alliance reception, I connected with NORD volunteer leaders, industry partners, and students pursuing rare disease studies. It was inspiring to introduce Waldenstrom’s macroglobulinemia (WM) to this next generation of changemakers.
Day One: Listening to Learn
Monday’s sessions emphasized patient-centered collaboration and building trust. From young advocates to industry executives, the shared message was clear: listen to patients, reduce barriers, and expand access to treatment.
The session on the new era of rare disease clinical trials was particularly powerful. Panelists agreed that “patients are the experts” and must be included in the clinical trial design process. COVID-19 accelerated innovation and flexibility in medicine, and now, with patient advocacy groups partnering alongside industry, those changes can continue to shape better, more inclusive trials.
Although the government shutdown prevented participation from the FDA and NIH, NORD seamlessly pivoted to a session featuring former FDA leaders, offering valuable insights on regulatory policy for rare therapies.
Day 2: Inspiring the Next Generation
Just like the IWMF, NORD is looking toward the next generation to advance care and research for rare diseases, in our case WM. A student panel—from a college freshman to a PhD candidate—shared how they discovered their passion for rare disease work.
One student’s high school internship at one of NORD Rare Disease Centers of Excellence set him on his path; another was inspired by an undergraduate conference dedicated to rare conditions. Their stories remind us that nurturing future leaders is essential to sustaining progress in rare disease and WM advocacy.
The session on AI in Drug Development was both enlightening and optimistic. While rare diseases have historically fallen outside traditional industry business models, AI has the potential to reduce the cost and time of drug development, making rare disease research more viable for industry investment.
A Surprise Visit from Senator Amy Klobuchar
A highlight of the conference was an unexpected visit from Senator Amy Klobuchar, whose energy and heartfelt words underscored her dedication to patients with rare diseases. It was a powerful reminder that champions for our community exist at every level—from the grassroots to the halls of Congress. The Summit concluded with “Next Steps for Optimal Impact,” moderated by NORD CEO Pam Gavin, encapsulating NORD’s powerful message: “Alone we are rare. Together we are strong.”
Together Toward Breakthroughs
This year’s NORD Breakthrough Summit reminded us that every voice matters—and when we unite those voices, breakthroughs happen. Through collaboration, innovation, and shared purpose, the rare disease community continues to build a stronger, more hopeful future for all.
Cyndi Powers, CMP, is IWMF’s director of conferences and events.