#United States #Washington, D.C. #NORD #Rare Disease #FDA CBER

NORD's Inaugural Rare Disease Scientific Symposium

The National Organization for Rare Disorders (NORD®) is set to launch its first-ever Rare Disease Scientific Symposium, a two-day gathering of leading voices from science, industry, and regulatory bodies. Taking place from June 2-3, 2025, in Washington, D.C., the event aims to accelerate both translational and clinical research efforts focused on rare diseases.

Keynote Speaker and Agenda Highlights

The symposium will kick off with a keynote address from Dr. Vinay Prasad, the newly appointed Director of the FDA's Center for Biologics Evaluation and Research (CBER). His participation underscores the importance of regulatory perspectives in advancing research for rare diseases.

Attendees can expect a comprehensive program featuring over 20 sessions led by renowned experts. Topics will cover innovative research designs tailored for small patient populations, the role of real-world evidence and patient-reported data, and strategies for fostering collaboration across sectors—from academia to biopharmaceutical companies and governmental agencies.

Pamela K. Gavin, NORD's Chief Executive Officer, emphasized the organization's history of facilitating cooperation in the rare disease scientific community. "For more than four decades, we have pushed forward the frontiers of knowledge in this challenging arena. As the landscape becomes increasingly complex, our symposium will be a critical platform for collaboration among various stakeholders," she remarked.

Addressing Urgent Needs in Rare Disease Research

With over 10,000 recognized rare diseases and a staggering 95% lacking FDA-approved treatments, the need for innovation in research has reached a critical juncture. Key speakers—drawing from prestigious institutions, top pharmaceutical companies, and notable agencies such as the NIH and FDA—will tackle difficult questions and share insights that could lead to breakthroughs.

Tracey Sikora, NORD's Vice President for Clinical and Research Programs, reiterated the importance of a collaborative approach: "The challenges in rare disease research are daunting when faced alone, but collective efforts can significantly speed up progress. This symposium embodies our core belief in partnerships as the catalyst for advancing science and improving patient lives."

A report from the National Academies of Sciences, Engineering, and Medicine has influenced the symposium's agenda, calling for enhanced collaboration between the FDA and the rare disease community, greater regulatory flexibility, and the incorporation of patient-centered data into research and development processes.

Additional Features and Registration Details

In addition to the keynote address and expert-led discussions, the symposium is aligned with NORD's mission to advocate for those living with rare diseases. Various leading medical institutions that form the NORD® Rare Disease Centers of Excellence will also participate, contributing to discussions aimed at enhancing diagnosis and treatment.

Registration for the symposium is now open, with details available at nordscience.org, where attendees can also explore the full agenda and the list of speakers. This event is an important opportunity for professionals across the field to network and collaborate, setting the stage for future advancements in the treatment of rare diseases.

About NORD

Founded in 1983, the National Organization for Rare Disorders (NORD®) is a pivotal non-profit organization dedicated to advocate for the health and well-being of the estimated 30 million Americans living with rare diseases. By partnering with over 350 disease-specific organizations, NORD strives to enhance research, care, and policy related to rare disorders, reinforcing its commitment to drive measurable progress in this essential health arena.