Leaders across research, clinical care, industry, and patient advocacy convene to accelerate rare disease breakthroughs from discovery to real-world patient impact

NORWELL, Mass., Feb. 5, 2026 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®) today announced the agenda for its second annual NORD® Rare Disease Scientific Symposium, taking place April 14–15, 2026, in Arlington, Va., and convening leading researchers, clinicians, industry innovators, government leaders, and patient advocates to advance solutions for people living with rare diseases.

The symposium will feature remarks from NIH Director Dr. Jay Bhattacharya, MD, PhD, underscoring the urgency of accelerating research, diagnosis, and therapeutic development for the more than 30 million Americans living with rare diseases.

The 2026 Scientific Symposium will feature sessions exploring innovative clinical trial designs, alternates to placebo controls, and emerging regulatory pathways. Attendees will also gain insight into funding models, drug repurposing, and how registries, real-world data, and patient-generated data can inform endpoint selection, evidence generation, and decisions related to access and coverage.

"We received incredibly positive feedback from last year's inaugural symposium," said Tracey Sikora, NORD Vice President of Research & Clinical Programs. "Attendees consistently cited the sense of camaraderie across multiple rare diseases and the importance of bringing together the community in a shared scientific forum. This year's program builds on that momentum and is designed to bridge gaps across the rapidly evolving rare disease landscape, particularly in collaborative clinical research, regulatory innovation, and the use of data across the development lifecycle. We're excited to continue fostering conversations that lead to real solutions."

New additions to this year's program focus on regulatory innovation, including drug repurposing and repositioning, rescued therapies, and the U.S. Food and Drug Administration (FDA)'s emerging plausible mechanism pathway.

Designed as a working forum, the symposium will focus on turning scientific breakthroughs into real-world treatments by fostering collaboration across sectors and strengthening models that move discoveries forward for rare and ultra-rare diseases as well as into scalable solutions benefiting multiple diseases.

"We believe in the power of collaboration and connection," said Pamela Gavin, NORD Chief Executive Officer. "For more than 40 years, NORD has driven progress in rare disease science. The NORD Rare Disease Scientific Symposium continues this tradition by fostering cross-sector collaboration and helping shape the future of rare disease research and care."

Speakers include research and clinical experts from the NORD® Rare Disease Centers of Excellence network, comprised of 46 U.S. leading medical centers and research institutions. Last year's inaugural event drew more than 600 attendees, underscoring the growing urgency and global interest in advancing rare disease science and accelerating therapeutic development.

Agenda Highlights

• Bayesian and Adaptive Trial Designs for Ultra-Rare Populations: Introduces how Bayesian and adaptive methods can make trials more efficient and informative when working with extremely small patient populations.
• FDA Regulatory Innovation: Plausible Mechanism & Platform Pathways: How regulators may rely on biological rationale and platform-based evidence approaches when evaluating therapies for rare diseases with limited clinical data.
• Innovative Alternatives to Placebo Controls: Exploration of non-traditional control strategies and regulatory considerations for rare disease trials.
• Funding Rare Disease Research: Examines diverse funding models and the roles of advocacy groups, academia, and industry in sustaining research for small and underserved patient populations.
• Abandoned, Shelved, and Rescued Therapies for Ultra-Rare Diseases: Case studies of once discontinued therapies that were later revived through academic leadership, public benefit approaches, or strategic repositioning.
• Drug Repurposing Innovation: Advances in phenotypic screening and computational tools identifying new uses for existing therapies.
• Access and Coverage: Data and Clinical Development: Examination of how clinical and real-world data intersect with payer and coverage considerations.

Registration Now Open 
Registration for the 2026 NORD Rare Disease Scientific Symposium is now open at https://nordscience.org. Earlybird registration is available until February 17, 2026. 

About the National Organization for Rare Disorders
Founded in 1983, the National Organization for Rare Disorders (NORD®) is a leading independent, nonpartisan, nonprofit and patient advocacy organization dedicated to improving the health and lives of over 30 million Americans living with rare diseases. In partnership with more than 350 disease-specific member patient organizations, NORD drives progress in rare disease research, care, and policy. Learn more at https://rarediseases.org/

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SOURCE National Organization for Rare Disorders (NORD®)