Uniting Leaders to Discuss Equitable Access and Innovation in Rare Disease

SAVE THE DATE: OCTOBER 20-21, 2025

SEE HIGHLIGHTS FROM THE 2024 SUMMIT

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ATTENDEES

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SPEAKERS

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SPONSORS AND EXHIBITORS

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RESEARCH POSTERS

Shape the future of rare disease treatments, research and policy at the NORD® Rare Diseases and Orphan Products Breakthrough Summit.® Together, we can advance equity and innovation for the more than 30 million Americans – and more than 400 million people worldwide – with rare diseases!

Join Us

Some of the rare disease leaders and innovators you heard from at the 2024 NORD Breakthrough Summit

Robert M. Califf, MD

Commissioner of Food and Drugs, Food and Drug Administration (FDA)

Meena Seshamani, MD, PhD

Deputy Administrator and Director, Center for Medicare, Centers for Medicare & Medicaid Services (CMS)

Joni L. Rutter, PhD

Director, National Center for Advancing Translational Sciences (NCATS), NIH

Rachid Izzar

Executive Vice President, Global Product Strategy & Commercialization, Biogen

Peter Marks, MD, PhD

Director, Center for Biologics Evaluation & Research (CBER), US Food and Drug Administration (FDA)

Michelle Tarver, MD, PhD

Interim Director, Center for Devices and Radiological Health (CDRH), US Food and Drug Administration

Patrizia Cavazzoni, MD

Director, Center for Drug Evaluation & Research (CDER), U.S. Food & Drug Administration

Hilary Marston, MD, MPH

Chief Medical Officer, FDA

See Full Speaker List

Why Attend?

Collaborate & Influence

Contribute to critical discussions, forge valuable partnerships, and help advance equitable access to rare disease research and
patient care.

Engage with Experts

Connect with influential voices and stakeholders shaping the future of rare disease treatments, research and policy.

Discover Innovations

Learn about cutting-edge advancements to improve access to clinical trials, and discover emerging trends and issues that are poised to redefine the landscape of rare disease.