WASHINGTON, D.C. and NORWELL, Mass. — As scientific innovation accelerates at an unprecedented pace, the rare disease community faces a defining question: how to turn groundbreaking laboratory discoveries into tangible, accessible treatments for patients. This critical transition will take center stage later this month as the National Organization for Rare Disorders (NORD®) hosts a high-level CEO leadership dialogue at its upcoming national conference.
The panel, titled "Next Decade of Rare Disease: A CEO Leadership Dialogue," is set for 11 a.m. ET on Monday, October 26, in Washington, D.C. It forms a cornerstone of the 16th Annual NORD Rare Diseases & Orphan Products Breakthrough Summit®, a three-year-running event spanning October 25 through 27.
To guide this pivotal conversation, NORD has enlisted Becky Quick, co-anchor of CNBC’s “Squawk Box” and host of “CNBC Cures.” Quick will moderate a discussion featuring the chief executive officers of three major global biopharmaceutical companies—BioMarin, Jazz Pharmaceuticals, and Otsuka—alongside NORD President and CEO Pam Gavin.
The dialogue arrives at a crucial juncture for the rare disease sector. Over the past decade, unprecedented strides in biotechnology, genetic research, and molecular science have unlocked diagnostic and therapeutic possibilities that were once unimaginable. Yet, industry experts emphasize that scientific discovery is only the first step in a complex journey. Realizing the full potential of these advancements requires a synchronized evolution across regulatory frameworks, public and private investment, healthcare infrastructure, and global treatment delivery systems.
The panel will examine the strategic leadership, institutional decisions, and cross-sector partnerships necessary to navigate this moment. Specifically, the participants will address the profound challenge of scale—how the healthcare ecosystem can efficiently advance a greater number of treatments for a vastly broader array of rare conditions.
The invited biopharmaceutical executives bring diverse global perspectives to the table, each representing organizations with distinct rare disease portfolios, extensive clinical pipelines, and deep experience in shepherding complex therapies through development and commercialization. By pairing these industry leaders with Gavin, the discussion will bridge the gap between commercial biotechnology and the independent, cross-disease patient perspective. Gavin represents a patient and systems point of view that connects the scientific and economic realities of drug development with the diverse unmet medical needs, daily care hurdles, and profound access challenges experienced by individuals living with any of the more than 10,000 known rare diseases.
For moderator Becky Quick, the event also carries a deeply personal resonance. Beyond her decades of experience covering global business, financial markets, and high-profile industry figures, Quick is the parent of a child living with a rare disease. This dual background equips her to steer a conversation that balances hard-nosed commercial and regulatory realities with the urgent human stakes of patient care. She will weave these viewpoints together to explore how sector leaders can dismantle systemic barriers, maximize technological innovation, and translate individual scientific victories into broad-based progress for the entire rare disease community.
"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 Breakthrough Summit has established itself as a premier gathering place for the rare disease community, drawing more than 900 leaders from every corner of the ecosystem. Attendees include representatives from patient advocacy groups, academic research institutions, biotechnology and pharmaceutical companies, regulatory agencies, and healthcare delivery systems. Together, they convene to address the most pressing issues shaping the future of rare disease research, treatment development, public policy, patient care, and market access.
The comprehensive programming for the 2026 Summit spans a wide array of topics, including federal health and regulatory priorities, emerging therapies and cutting-edge technologies, investment trends, patient-industry partnerships, and the logistical systems required to safely and efficiently move breakthroughs from the laboratory bench to the patient’s bedside.
Adding further weight to the three-day conference, the 2026 Breakthrough Summit will feature prominent remarks and interactive conversations with senior federal health leaders. Among the key governmental figures scheduled to participate 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 presence underscores the vital intersection between federal oversight, scientific funding, and private-sector innovation in addressing unmet medical needs.
The entire framework of the NORD Breakthrough Summit is built around the premise of cross-sector collaboration. Organizers emphasize that the complex challenges inherent in rare disease research and treatment delivery are too vast for any single organization, corporation, or government agency to solve in isolation.
Founded in 1983, the National Organization for Rare Disorders (NORD) operates as an independent, nonpartisan, 501(c)(3) nonprofit organization dedicated to improving the health and lives of the more than 30 million Americans living with rare diseases. Working in close partnership with over 350 disease-specific member patient organizations, NORD serves as a unifying force, driving meaningful progress in rare disease research, education, patient support, and public policy.
Registration and further details regarding the event agenda, speaker lineups, and session schedules for the 2026 Rare Diseases & Orphan Products Breakthrough Summit are available online at NORDSummit.org.
