WASHINGTON, D.C. and NORWELL, Mass. — As the landscape of medical science continues to evolve at a breathtaking pace, the National Organization for Rare Diseases (NORD®) has announced a high-profile panel that will bring together top executives from major biopharmaceutical companies to address the future of rare disease treatment. 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, at the upcoming NORD® Rare Diseases & Orphan Products Breakthrough Summit®.
The landmark panel, titled “Next Decade of Rare Disease: A CEO Leadership Dialogue,” is scheduled to take place at 11 a.m. ET on Monday, October 26, in Washington, D.C. The session forms a centerpiece of the broader three-day Summit, which runs from October 25 through October 27. Industry professionals, researchers, clinicians, advocates, and patients interested in attending can view the full agenda and register for the event by visiting NORDSummit.org.
This year’s dialogue comes at a uniquely pivotal time for the rare disease community. Scientific and technological advancements are currently opening up unprecedented possibilities for earlier diagnoses and more targeted treatments. However, industry stakeholders increasingly recognize that discovery alone is insufficient. Realizing the full potential of these scientific breakthroughs will require regulatory frameworks, robust public and private policies, sustained investment, modern healthcare infrastructure, and efficient treatment delivery systems to keep pace with innovation.
The upcoming conversation will examine the critical leadership decisions, strategic choices, and cross-sector partnerships needed to capitalize on this current moment. Panelists are expected to tackle the complex challenge of scale—specifically, how the medical and business ecosystems can successfully advance and deliver more treatments across a greater number of rare conditions.
The distinguished panel brings together leaders from global biopharmaceutical companies with distinct rare disease portfolios, robust pipelines, and extensive real-world experience in developing and delivering therapies across a diverse spectrum of rare conditions. Alongside these industry leaders, Pam Gavin will bring NORD’s independent, cross-disease patient and systems perspective to the discussion. Her participation will help bridge the gap between the scientific and business questions facing modern rare disease innovation and the diverse, unmet needs, daily care hurdles, and access challenges experienced by individuals living with more than 10,000 distinct rare diseases.
Moderator Becky Quick brings decades of prominent journalistic experience covering global business, economics, and financial markets, alongside interviews with many of the world’s most influential corporate leaders. In addition to her professional expertise, Quick holds a deeply personal connection to the rare disease community as a parent of a child living with a rare condition. Her unique vantage point will guide the conversation, blending rigorous financial and strategic inquiry with a profound understanding of the human impact of rare diseases, exploring how leaders can maximize the potential of technological innovation and translate individual laboratory breakthroughs into widespread clinical 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, emphasizing the urgency behind the upcoming session. “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 celebrating its 16th year, the NORD Rare Disease & Orphan Products Breakthrough Summit has established itself as a premier annual gathering, bringing together more than 900 leaders from across the entire rare disease ecosystem. Attendees typically include representatives from patient advocacy organizations, pharmaceutical and biotechnology companies, regulatory agencies, clinical research institutions, and the investment community. Together, they address the most critical issues shaping the future of rare disease research, treatment development, public policy, patient care, and healthcare access.
Comprehensive programming for the three-day event spans federal health and regulatory priorities, emerging therapies and cutting-edge technologies, venture and institutional investment, genuine patient partnership, and the systemic infrastructure required to successfully move breakthroughs from initial laboratory discovery to the patients who need them most.
In addition to the CEO leadership dialogue, the 2026 Breakthrough Summit will feature prominent remarks and interactive conversations with senior federal health leaders. Among the key governmental figures slated 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 involvement underscores the vital partnership between federal regulators, public researchers, and private innovators necessary to address the regulatory and scientific hurdles unique to rare disease drug development.
The NORD Breakthrough Summit is fundamentally built around the premise of cross-sector collaboration, tackling complex systemic challenges that no single organization, company, or government agency can solve in isolation. Complete registration details, housing information, and the fully detailed event agenda remain publicly available at NORDSummit.org.
Founded in 1983, the National Organization for Rare Diseases (NORD®) operates as a leading independent, nonpartisan, 501(c)(3) nonprofit organization dedicated entirely to improving the health, well-being, and lives of the more than 30 million Americans who live with rare diseases. Working in active partnership with over 350 disease-specific member patient organizations, NORD drives continuous progress in rare disease medical research, patient advocacy, specialized care, and public policy reform.
