WASHINGTON, D.C. and NORWELL, Mass. — As scientific and technological advancements open unprecedented frontiers in medicine, turning laboratory discoveries into accessible 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. Becky Quick, co-anchor of CNBC’s “Squawk Box” and host of “CNBC Cures,” is set to moderate a rare disease CEO leadership panel featuring the chief executive officers of BioMarin, Jazz Pharmaceuticals, and Otsuka, alongside NORD CEO Pam Gavin.
The spotlight session, titled “Next Decade of Rare Disease: A CEO Leadership Dialogue,” will take place at 11 a.m. ET on Monday, October 26, in Washington, D.C. The discussion forms a centerpiece of the 16th annual NORD® Rare Diseases & Orphan Products Breakthrough Summit®, a comprehensive three-day conference running from October 25 to October 27. Registration details and the full event agenda are available to healthcare professionals, researchers, industry leaders, and advocates at NORDSummit.org.
This year’s summit arrives at a pivotal juncture for the rare disease community. Rapid progress in fields such as precision medicine, genetic therapies, and digital health tools is creating entirely new possibilities for the diagnosis and treatment of conditions that were once thought untreatable. However, industry stakeholders increasingly recognize that scientific innovation alone is not enough. Realizing the full potential of these advancements requires modernizing regulatory frameworks, securing sustained investment, expanding healthcare infrastructure, and smoothing pathways for treatment delivery to ensure they keep pace with scientific discovery.
The upcoming CEO dialogue will examine the executive leadership, strategic decisions, and cross-sector partnerships necessary to capitalize on this unique historical moment. A central theme of the conversation will be addressing the profound challenge of scale—specifically, how the biopharmaceutical industry and patient advocacy groups can work in tandem to advance more treatments for a greater number of rare diseases.
The panel brings together top executives from global biopharmaceutical companies that maintain distinct rare disease portfolios, robust pipelines, and extensive real-world experience in developing and delivering specialized therapies. By pairing these industry leaders with NORD CEO Pam Gavin, the panel ensures a comprehensive view of the landscape. Gavin brings NORD’s independent, cross-disease patient and systems perspective to the table, effectively connecting the complex scientific and business questions facing rare disease innovation with the diverse, urgent unmet needs, care, and access challenges experienced by people living with more than 10,000 known rare diseases.
Moderator Becky Quick brings decades of distinguished experience covering global business, economics, and financial markets, alongside a reputation for interviewing the world’s most influential corporate leaders. In addition to her extensive journalistic background, Quick brings a deeply personal connection to the rare disease community as the parent of a child living with a rare disease. This unique vantage point allows her to guide the conversation adeptly, bridging corporate strategy with human impact and exploring how industry leaders can maximize the potential of technological innovation to translate individual scientific breakthroughs into widespread progress.
“We are at an important moment for rare disease,” said Pam Gavin, CEO of NORD. “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. 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 year, the NORD Rare Disease & Orphan Products Breakthrough Summit has established itself as a premier gathering, drawing more than 900 leaders from across the entire rare disease ecosystem. Attendees include representatives from patient advocacy groups, biotechnology and pharmaceutical companies, academic research institutions, federal agencies, and investment firms. Together, they convene to address the critical issues shaping the future of rare disease research, treatment development, public policy, patient care, and healthcare access.
The extensive summit programming spans a wide array of vital topics, including federal health and regulatory priorities, emerging therapies and cutting-edge technologies, venture investment, effective patient partnerships, and the robust systems required to smoothly move breakthroughs from initial laboratory discovery to the patients who depend on them.
Reflecting the critical intersection of public policy and private innovation, the 2026 Breakthrough Summit will also feature keynote remarks and interactive conversations with senior federal health leaders. Among the prominent government officials scheduled to participate are Kyle Diamantas, J.D., Acting Commissioner of the U.S. Food and Drug Administration (FDA), and Jay Bhattacharya, M.D., Ph.D., Director of the National Institutes of Health (NIH). Their participation underscores the collaborative nature of the summit, highlighting the ongoing dialogue between regulatory agencies, research institutions, and the private sector.
Organizers emphasize that the NORD Breakthrough Summit is intentionally built around cross-sector collaboration designed to tackle complex systemic challenges that no single organization, company, or government agency can solve in isolation. By fostering an environment where diverse stakeholders can exchange ideas, debate policy, and forge strategic partnerships, the summit aims to accelerate the pace of progress for the broader rare disease community.
Founded in 1983, the National Organization for Rare Disorders (NORD®) is a leading independent, nonpartisan, 501(c)(3) nonprofit organization dedicated to improving the health and lives of over 30 million Americans living with rare diseases. Working in active partnership with more than 350 disease-specific member patient organizations, NORD drives continuous progress in rare disease research, patient support, medical care, and public policy.
Individuals interested in attending the summit, viewing the comprehensive schedule of panel discussions, or learning more about registration can visit NORDSummit.org.
