Washington, DC, October 9, 2020—On October 8 and 9, the National Organization for Rare Disorders (NORD®) virtually hosted the 2020 Rare Diseases and Orphan Products Breakthrough Summit, the first time the organization representing over 25 million Americans impacted by rare diseases has presented the conference exclusively online. Nearly 900 registrants from more than 20 countries around the world gathered online for the annual event, the most impactful rare disease conference in the United States.
Each year the NORD Summit brings together participants spanning the rare community, including rare disease experts and leaders from patient advocacy groups, government, industry and academia to discuss the most current and critical topics related to rare diseases and orphan products. The 2020 theme, “Entering a New Era,” highlighted the current climate of scientific innovation that allows us to envision life-altering or even curative treatments, as well as today’s awareness of the importance of addressing global public health challenges.
The two–day event kicked off on Thursday morning with a greeting from NORD’s President and CEO, Peter L. Saltonstall, followed by a moving patient/caregiver opening address. “Innovation Transforms Lives” featured Jennifer Beck of the American Porphyria Foundation, Amy Medina of Cure SMA, and Mark Sleeper, an individual living with cystic fibrosis, discussing how their lives have been dramatically altered by recent medical advancements.
In his keynote, US Food and Drug Administration (FDA) Commissioner Stephen Hahn, MD touched on topics including COVID-19, the collaboration between NORD and FDA on patient listening sessions and in rare disease research, and the 51 orphan indications covering a broad range of rare diseases that have been approved by FDA through July of this year. “It is essential that even as we continue to support the development of safe and effective treatments and vaccines for COVID-19 during this public health emergency, we also advance FDA’s other priorities including the development of treatments for patients with rare diseases,” said Dr. Hahn, who went on to praise NORD for the organization’s “important role in shining a light on, encouraging and forging opportunities to advance new and groundbreaking scientific research in areas where there might otherwise not be attention and action.”
Other highlights from day one of the Summit included discussions on what has been learned from COVID-19 and how it can be applied for a stronger health care system, perspectives from Capitol Hill staffers on 2020 and what is ahead, and breakout sessions on topics ranging from designing a regulatory-ready natural history study, virtual fundraising and grassroots advocacy to advancing access to diagnostic testing, streamlining orphan drug development and the latest news on gene therapy and genome editing research.
Sponsors of the 2020 NORD Summit included Eversana, Insmed, Retrophin and Takeda. To sign up to receive communications on the 2021 Rare Diseases and Orphan Products Breakthrough Summit, please click here.
Tonight at 7:00pm EDT, NORD will premiere a streaming version of the Rare Impact Awards, with 2020 honorees including legislators, advocates, researchers, physicians and a rock and roll legend, all being celebrated for making extraordinary strides for the rare disease community. Janet Woodcock, MD, Director of the Center for Drug Evaluation and Research, FDA, will be honored with a Lifetime Achievement Award. The 2020 Rare Impact Awards program is free and open to the public, with registration required.
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