WASHINGTON, D.C. and NORWELL, Mass. — As scientific breakthroughs open unprecedented possibilities for diagnosing and treating rare conditions, turning those laboratory milestones into accessible patient treatments remains one of the healthcare sector’s most pressing challenges. To address this critical hurdle, the National Organization for Rare Disorders (NORD®) has announced a high-profile leadership dialogue for its upcoming annual event.
CNBC “Squawk Box” co-anchor and “CNBC Cures” host Becky Quick will moderate a rare disease CEO leadership dialogue featuring the chief executive officers of BioMarin, Jazz Pharmaceuticals, and Otsuka, alongside NORD CEO Pam Gavin. The panel is scheduled for 11 a.m. ET on Monday, October 26, in Washington, D.C., as a centerpiece of the three-day NORD® Rare Diseases & Orphan Products Breakthrough Summit®, running from October 25 to 27.
Titled “Next Decade of Rare Disease: A CEO Leadership Dialogue,” the session comes at a pivotal juncture for the rare disease community. While scientific and technological advancements are rapidly expanding the boundaries of modern medicine, realizing their full potential depends heavily on whether regulatory frameworks, public policies, private investment, healthcare infrastructure, and treatment delivery models can keep pace. The panel will examine the strategic leadership, complex corporate decisions, and cross-sector partnerships required to capitalize on this scientific momentum and solve the immense challenge of scale—ultimately advancing more treatments for a greater number of rare diseases.
The participating biopharmaceutical leaders bring extensive global perspectives, representing organizations with diverse rare disease portfolios, robust pipelines, and deep operational experience in developing and delivering therapies across a wide spectrum of conditions. Complementing their industry viewpoints, Pam Gavin will bring NORD’s independent, cross-disease patient and systems perspective. Her contributions will connect the commercial and scientific questions facing industry innovators directly with the diverse, often unmet needs, daily care hurdles, and profound access challenges experienced by individuals and families navigating more than 10,000 distinct rare diseases.
Moderator Becky Quick brings decades of distinguished journalism experience covering global business, economics, and financial markets, alongside her work interviewing many of the world’s most influential corporate leaders. In addition to her professional expertise, Quick brings a deeply personal connection to the cause as the parent of a child living with a rare disease. This unique vantage point will allow her to guide the conversation adeptly, bridging corporate strategy with human impact to explore how leaders can maximize the promise of modern innovation and translate individual laboratory breakthroughs into widespread, systemic progress.
“We are at an important moment for rare disease. Science is opening doors that would have been unimaginable even a decade ago, but discovery alone does not ensure those advances reach the people who need them,” said Pam Gavin, CEO of NORD. “Realizing that potential will require leadership across the rare disease ecosystem to remove barriers, keep pace with innovation and translate progress into better outcomes for more people across more rare diseases. That is why this conversation—and the Breakthrough Summit—matter right now.”
Now in its 16th consecutive year, the NORD Rare Disease & Orphan Products Breakthrough Summit has established itself as a premier gathering, drawing more than 900 leaders from every corner of the rare disease ecosystem. Attendees include representatives from patient advocacy groups, biotechnology and pharmaceutical firms, regulatory agencies, clinical research institutions, and the investment community. Together, they convene to address the critical issues shaping the future of rare disease research, therapeutic development, healthcare policy, patient care, and market access.
The comprehensive multi-day programming spans a wide array of topics, including federal health and regulatory priorities, emerging therapies and cutting-edge technologies, venture investment trends, meaningful patient partnerships, and the systemic infrastructure needed to shepherd breakthroughs smoothly from initial discovery to clinical availability.
Adding to the significance of the 2026 Breakthrough Summit, the event will also feature keynote remarks and prominent discussions with senior federal health leaders. Among them are Kyle Diamantas, J.D., Acting Commissioner of the U.S. Food and Drug Administration (FDA), and Dr. Jay Bhattacharya, M.D., Ph.D., Director of the National Institutes of Health (NIH). Their participation highlights the ongoing, vital dialogue between government agencies, regulatory bodies, researchers, and industry stakeholders necessary to foster a supportive environment for medical innovation.
The NORD Breakthrough Summit is intentionally built around the principle of cross-sector collaboration, tackling complex systemic challenges that no single organization, company, or sector could hope to solve in isolation. By bringing diverse stakeholders together under one roof, the summit fosters an environment where cooperative solutions can take root.
Founded in 1983, the National Organization for Rare Disorders (NORD) is a leading independent, nonpartisan, 501(c)(3) nonprofit organization dedicated to identifying, treating, and curing rare disorders through education, advocacy, research, and patient services. Representing and partnering with more than 350 disease-specific member patient organizations, NORD works tirelessly to improve the health and lives of the estimated over 30 million Americans living with rare diseases, driving continuous progress in research, care, and public policy.
