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Oct. 21, 2021

TOPIC: Press Releases

Record-Setting NORD Breakthrough Summit Welcomes Nearly 1,000 Members of the Rare Disease Community

Posted by Rohan Narayanan

Two days of insightful and engaging speakers, sessions, and networking at the most important event in rare disease saw record-breaking attendance

October 21, 2021This week, the National Organization for Rare Disorders (NORD®) hosted the virtual Rare Diseases and Orphan Products Breakthrough Summit, welcoming nearly 1,000 registrants from 33 countries for one of the largest and most important global events in rare disease. 

The Breakthrough Summit was launched as an annual event 12 years ago, offering an unparalleled opportunity to learn from all rare disease stakeholders – medical and academic experts, regulators and policymakers, innovators, patients, caregivers, and industry leaders. The 2021 NORD Summit covered many critical and relevant topics, including drug pricing, health technology and patient-generated data, diversity and inclusivity in clinical trials, public health and COVID-19, international collaboration, genetic testing, and more. 

On Monday, October 19, the Summit kicked off with the plenary, Together We Are Strong, featuring Dr. Titilope Fasipe, Co-Director, Sickle Cell and Thalassemia Program, Texas Children’s Cancer and Hematology Centers, and Kim McClellan, Arkansas NORD Rare Action Network® Volunteer State Ambassador. This event set the collaborative and innovative tone for the week, as all Summit attendees and speakers work together to learn, share, and usher in the new era of progress for the rare disease world.  

The keynote address on Tuesday, October 19 from Janet Woodcock, Acting Commissioner, US Food and Drug Administration (FDA) emphasized the importance of partnership and progress on behalf of the over 25 million Americans living with a rare condition. “Sometimes necessity forces us to find new approaches… At the FDA, we created the Office of Orphan Products Development to focus on those [rare] patients and developments of products needed to treat them,” said Janet Woodcock, MD, Acting Commissioner, FDA. 

Other highlights from the Summit included panels featuring leaders from Capitol Hill discussing drug pricing, a Town Hall with experts from the National Institutes of Health (NIH), a conversation with the three FDA Center Directors, and breakout sessions on issues ranging from healthcare system savings to advancing rare cancer awareness. 

Debra Houry, MD, MPH, Acting Principal Deputy Director of Centers for Disease Control and Prevention (CDC) provided a special message to the rare disease community, as did leadership from NIH.  

“The pandemic has emphasized what a problem we have with health disparities and health inequities… I just want to encourage you, despite the challenges we face right now, to look at where science is going and feel optimistic that we are on a path toward circumstances where many more of the rare diseases will begin to have therapeutic opportunities come forward. The future is bright but it’s going to require participation of all of us,” said Francis S. Collins, MD, PhD, Director, NIH. 

Sponsors for the conference included Sanofi Genzyme, Horizon, Takeda and Travere Therapeutics.  

All content from the NORD Breakthrough Summit is available on-demand for 30 days after the conclusion of the conference. Access is free for members of the media. Register now for full access, to revisit lessons learned, and hear from all key experts and speakers.


About the National Organization for Rare Disorders (NORD)
The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 300 disease-specific member organizations, more than 15,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit