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NIH Director Dr. Jay Bhattacharya to Open Second Annual NORD® Rare Disease Scientific Symposium

The National Organization for Rare Disorders (NORD®) has officially announced its agenda for the much-anticipated second annual NORD® Rare Disease Scientific Symposium, set for April 14-15, 2026, in Arlington, Virginia. This event will welcome prominent figures from the realms of research, clinical care, and patient advocacy to forge pathways for breakthroughs in rare disease treatment.

Dr. Jay Bhattacharya, the esteemed Director of the National Institutes of Health (NIH), will deliver opening remarks emphasizing the critical need to enhance research, diagnosis, and therapeutic strategies for the approximately 30 million Americans affected by rare diseases. The symposium promises a rich agenda filled with discussions centered on innovative clinical trial methodologies, efficient regulatory pathways, and the shared goal of transforming scientific achievements into tangible patient benefits.

Tracey Sikora, NORD's Vice President of Research and Clinical Programs, conveyed the positive sentiment surrounding last year's inaugural symposium, noting that participants highlighted the sense of connection across various rare diseases and the imperative of communal scientific exchange. The 2026 event aims to build upon this momentum, addressing the evolving needs of researchers, clinicians, and advocates engaged with rare diseases.

What to Expect

This year's agenda features innovative topics such as:

• - Bayesian and Adaptive Trials: These sessions will illustrate how Bayesian methodologies can optimize clinical trials by making them more efficient, particularly in scenarios with very small patient populations.
• - FDA Regulatory Innovations: Discussions on the FDA's emerging plausible mechanism pathways will shed light on how regulatory authorities may assess therapies based on biological rationale and platform approaches.
• - Alternatives to Placebo Controls: Aimed at exploring non-traditional control strategies, these sessions address critical regulatory considerations for rare disease studies.
• - Funding Models: Presentations will evaluate diverse funding strategies and highlight the roles of advocacy organizations, academia, and industry in supporting research for underrepresented patient groups.
• - Rescued Therapies: Case studies on therapies that, despite being overlooked, found new life through thoughtful academic and clinical strategies.

The Drive for Collaboration

NORD's Chief Executive Officer, Pamela Gavin, emphasized the organization's longstanding commitment to fostering collaboration within the rare disease community. For over 40 years, NORD has played a pivotal role in advancing science and policy related to rare diseases. This symposium is a continuation of that mission, bringing together experts, advocates, and innovators to cultivate partnerships that will ultimately enhance patient care and research outcomes.

Prominent speakers from the NORD® Rare Disease Centers of Excellence, which encompasses 46 leading U.S. medical centers, will share their insights and findings. Last year's inaugural symposium attracted over 600 attendees, reflecting the growing urgency and commitment toward developing solutions for rare diseases.

Registration Now Open

Those interested in participating can now register for the 2026 NORD Rare Disease Scientific Symposium online. Early-bird registration is available until February 17, 2026. This event presents an invaluable opportunity for attendees to collaborate with stakeholders from diverse sectors, share knowledge, and explore innovative solutions for rare disease challenges.

To learn more about the National Organization for Rare Disorders and find registration details, visit their official website at NORD. The time is now to unite our efforts and reshape the future of rare disease treatment through collaborative innovation.