Last month, the National Organization for Rare DisordersⓇ (NORD) broke previous records with its 2019 Rare Diseases & Orphan Products Breakthrough Summit, welcoming over 900 registrants to two days of unforgettable keynotes, powerful panels, breakout sessions and networking in Washington, DC.
The NORD Summit is the largest and most innovative multi-stakeholder event of its kind, drawing together participants from across the rare disease community—including patient advocacy groups, government, industry and academia—to discuss the most current and urgent topics related to rare diseases and orphan products. The 2019 theme focused on why “The Time is Now” to drive innovation, collaboration, advocacy and research. These topics included perspectives from a diverse group of thought leaders.
There is No Time Like the Present – Inspiration to Action
The 2019 Summit kicked off the morning of Monday, October 21 with a greeting from NORD’s President and CEO, Peter L. Saltonstall, followed by an emotional Patient/Caregiver Opening Address, “There is No Time Like the Present – Inspiration to Action.” Speakers Terry Jo Bichell and Karen Aiach are mothers and rare disease warriors whose stories illustrated how families impacted by rare diseases are helping to drive progress in research, an increased understanding of diseases and the development of treatments.
Keynote speeches were informative and inspiring, with speakers including CDER’s Associate Director for Strategic Initiatives, Theresa Mullin, PhD and FDA Acting Director Ned Sharpless, MD. Tuesday morning marked an impassioned keynote speech delivered by Health and Human Services Secretary Alex Azar, the highest ranking government official ever to speak at a NORD Summit.
“Effective treatments are often hard to come by, requiring years of expensive maintenance therapies. When successful therapies are developed, they’re not cheap. We need to ensure that Americans who suffer from rare diseases have ways to finance their care–while also making sure that our financing system can support innovation toward the cures we need,” said Secretary Azar.
Health and Human Services Secretary Alex Azar speaking at the NORD Summit on October 22, 2019 (Photo courtesy of National Organization for Rare Disorders)
Other highlights from the Summit included panels featuring key opinion leaders, an NIH conversation on why it is a unique moment for rare disease research, a town hall with the three FDA Center Directors and an interactive dialogue with FDA senior staff.
Rare Revolution Editor