Introduction & Context
The National Organization for Rare Disorders (NORD) has announced its second annual Rare Disease Scientific Symposium, set to take place on April 14–15, 2026, in Arlington, Virginia. This event aims to bring together leading researchers, clinicians, industry innovators, government leaders, and patient advocates to advance solutions for the over 30 million Americans living with rare diseases. A highlight of the symposium will be a keynote address by NIH Director Dr. Jay Bhattacharya, underscoring the urgency of accelerating research, diagnosis, and therapeutic development in this field.
Background & History
NORD has been at the forefront of rare disease advocacy and research since its founding in 1983. The inaugural Rare Disease Scientific Symposium in 2025 received positive feedback for fostering collaboration across multiple rare diseases and creating a shared scientific forum. Building on this momentum, the 2026 symposium aims to bridge gaps in the rapidly evolving rare disease landscape, particularly in collaborative clinical research, regulatory innovation, and data utilization.
Key Stakeholders & Perspectives
The symposium will feature sessions exploring innovative clinical trial designs, alternatives to placebo controls, and emerging regulatory pathways. Attendees will gain insights 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. Speakers will include experts from the NORD Rare Disease Centers of Excellence network, comprising 46 leading U.S. medical centers and research institutions.
Analysis & Implications
The symposium's focus on regulatory innovation, including drug repurposing and the FDA's emerging plausible mechanism pathway, reflects a strategic shift towards more efficient therapeutic development processes. By fostering collaboration across sectors and strengthening models that move discoveries forward, the event aims to translate scientific breakthroughs into real-world treatments for rare and ultra-rare diseases.
Looking Ahead
As the symposium approaches, stakeholders are encouraged to participate and contribute to the ongoing dialogue aimed at accelerating rare disease research and therapeutic development. Registration is now open, with early bird rates available until February 17, 2026. The event represents a significant opportunity to shape the future of rare disease research and care through collaborative efforts.