WASHINGTON, D.C. and NORWELL, Mass. — Three of the nation’s most influential federal health leaders are set to take center stage at the upcoming 2026 National Organization for Rare Disorders (NORD) Rare Diseases and Orphan Products Breakthrough Summit. The landmark event, now in its 16th year, stands as the nation’s largest and longest-standing gathering dedicated entirely to rare diseases and orphan products.
Scheduled for October 25–27 in Washington, D.C., the summit will draw more than 900 rare disease leaders representing patient advocacy groups, industry innovators, policymakers, researchers, and medical professionals. This year’s conference arrives at a critical juncture for the rare disease community, providing a collaborative forum to tackle pressing challenges and accelerate solutions for the more than 30 million children and adults across the United States living with rare conditions.
The high-profile federal delegation headlining the event includes Acting Commissioner of Food and Drugs Kyle Diamantas, J.D., representing the U.S. Food and Drug Administration (FDA); Director Jay Bhattacharya, M.D., Ph.D., leading the National Institutes of Health (NIH); and Dr. Stephanie E. Haridopolos, M.D., DABFM, Director of National Health Communications for the Office of the Surgeon General and Principal Deputy Assistant Secretary for Health Policy. Their collective presence underscores a deeply unified federal commitment to advancing rare disease research, streamlining regulatory pathways, and transforming public health outcomes.
Pam Gavin, Chief Executive Officer of NORD, emphasized the profound significance of having these key decision-makers present at the national gathering. Rare disease is at a pivotal moment, she noted, adding that it is essential for the broader advocacy and scientific community to hear directly from the federal leaders who are actively shaping what comes next. According to Gavin, their participation reflects a longstanding, robust partnership and continued collaboration among the FDA, the NIH, and the Office of the Surgeon General. The vital work these leaders oversee across research, regulation, and public health serves as the bedrock for building upon today’s unprecedented scientific breakthroughs and translating that progress into tangible, life-changing advances for individuals and families navigating rare conditions.
Shaping Regulatory Horizons: FDA Leadership Takes the Stage
As Acting Commissioner of Food and Drugs, Kyle Diamantas oversees the sprawling regulatory agency responsible for evaluating and approving the drugs, biologics, medical devices, and diagnostics that are fundamental to modern rare disease care. In one of his earliest official actions in the role, Diamantas demonstrated a hands-on commitment to the population by organizing a dedicated roundtable with rare disease advocates, including representatives from NORD. The session was designed to listen directly to stakeholders about how the FDA can better understand, address, and overcome the unique structural and clinical challenges faced by the rare disease community.
At the Breakthrough Summit, Diamantas’s participation will offer attendees an invaluable opportunity to hear firsthand about the agency’s evolving regulatory priorities and innovative pathways. Because the path from scientific discovery to patient access often hinges on regulatory frameworks, understanding how the FDA plans to adapt to rapidly advancing fields like gene therapy, personalized medicine, and advanced diagnostics remains a top priority for developers and patient advocates alike. His address will provide critical visibility into how the agency intends to balance rigorous safety standards with the urgent, unmet needs of patients who often have few or no approved treatment options.
Unlocking Scientific Frontiers: NIH and Biomedical Innovation
Representing the engine of America’s scientific discovery, Dr. Jay Bhattacharya brings his leadership as Director of the National Institutes of Health to the forefront of the summit. Directing the nation’s premier biomedical research enterprise, Dr. Bhattacharya oversees the foundational science that underpins tomorrow’s diagnostics, treatments, and potential cures.
The NIH has increasingly positioned itself at the cutting edge of scientific technology, leveraging artificial intelligence and machine learning to analyze vast electronic health record data sets. Furthermore, the agency has championed public-private partnerships designed to develop robust platforms and standardized methodologies for gene-targeted therapies. These collaborative efforts aim to radically accelerate the development, testing, and delivery of targeted gene therapies for rare diseases—a class of treatments that holds immense promise for conditions historically deemed untreatable.
Dr. Bhattacharya’s perspective is considered vital for attendees seeking to understand how the diverse individual centers within the NIH coordinate their resources and research agendas. By fostering cross-institute collaboration, the NIH strives to support both pediatric and adult patients living with one or more rare diseases, bridging the gap between basic laboratory science and clinical application.
Advancing Public Health and Policy: Recognizing Dr. Stephanie E. Haridopolos
Dr. Stephanie E. Haridopolos brings a profound, longstanding dedication to rare diseases into her influential roles within the Office of the Surgeon General and the Department of Health and Human Services. During the summit, Dr. Haridopolos will deliver remarks centering on her extensive work in newborn screening, an area of public health that has experienced landmark policy shifts in recent years.
In recognition of her transformative contributions, Dr. Haridopolos will be presented with NORD’s prestigious 2026 Rare Impact Award as a Policy Changemaker. Her advocacy and leadership played a decisive role in a major 2025 health milestone: the official addition of two severe rare conditions, Duchenne muscular dystrophy and metachromatic leukodystrophy, to the Recommended Uniform Screening Panel (RUSP). Inclusion on the RUSP establishes a standardized national benchmark, ensuring that newborns across the country can be tested for these conditions shortly after birth.
Beyond her work on the RUSP, Dr. Haridopolos has served as a critical contributor to the Newborn Screening Working Group. This ongoing initiative is tasked with systematizing how the RUSP operates, ultimately expanding opportunities for earlier diagnosis, timely clinical intervention, and improved long-term outcomes for children born with rare genetic disorders. Her presence at the summit highlights the powerful intersection of rigorous public health policy and compassionate patient advocacy.
A Comprehensive Federal Dialogue and Multi-Sector Collaboration
Complementing the keynote addresses by Diamantas, Bhattacharya, and Haridopolos, the summit will feature a specialized federal regulatory panel. This session will convene senior representatives from across the FDA, including leadership from the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, the Center for Devices and Radiological Health, and the specialized Rare Disease Innovation Hub. By bringing together experts from pharmaceuticals, biologics, cell and gene therapies, and medical devices, the panel will offer a holistic view of how the federal government is adapting its review processes to accommodate complex, highly specialized modern therapeutics.
For the more than 30 million Americans currently facing a rare disease diagnosis, the convergence of policy, science, and industry at the summit represents a moment of profound significance. Rapid advancements in biomedical science are continually unlocking new possibilities for earlier, more accurate diagnoses and targeted treatments. However, these emerging technologies and innovative therapeutic approaches also present formidable challenges, pushing the nation’s research, regulatory, and healthcare delivery systems to modernize and keep pace.
The active participation of these top federal health leaders underscores a shared recognition that true progress requires deep cooperation. By working across traditional boundaries—uniting government agencies, academic research institutions, clinical medicine, the biotechnology and pharmaceutical industries, and the patient community—stakeholders can effectively translate scientific breakthroughs into faster diagnoses, broader treatment access, and significantly better health outcomes for the roughly 1 in 10 Americans affected by a rare disease.
Now celebrating its 16th year, the NORD Breakthrough Summit continues to serve as the premier annual forum for the rare disease sector. The 2026 event will bring together the diverse ecosystem of stakeholders necessary to drive sustained momentum across research, therapeutic development, clinical care, policy formulation, and patient access. Complete agenda details, registration information, and additional event programming can be accessed through the official summit website at NORDSummit.org.
About the National Organization for Rare Disorders (NORD®)
Established in 1983, the National Organization for Rare Disorders is an independent, nonprofit, and nonpartisan organization dedicated to driving positive change for the health and well-being of the more than 30 million Americans living with rare diseases. Working in close collaboration with over 350 disease-specific member patient organizations and a comprehensive Rare Disease Centers of Excellence network spanning more than 170 premier medical institutions and children’s hospitals nationwide, NORD champions comprehensive advancements in patient care, cutting-edge research, public education, and legislative advocacy. Further information regarding NORD’s ongoing initiatives and resources can be found online at rarediseases.org.
