5 Takeaways from the NORD Summit

What happens when you bring hundreds of rare disease patients and advocates together in Washington, D.C.?

NORD Summit 2019 speakers
Courtesy of the National Organization for Rare Disorders (NORD)
  1. Turnout broke previous records with more than 900 people attending the Rare Diseases & Orphan Products Breakthrough Summit, according to the National Organization for Rare Disorders.
  2. Grit recognizes grit. Terry Jo Bichell went back to college to get a Ph.D. in neuroscience after her son was diagnosed with the rare disease Angelman Syndrome. Bichell, who is now searching for a molecular cure, gave a keynote address and told attendees: “Many of you got bad news about a rare disease at some point in your lives. But what you did was roll up your sleeves and go to work. It takes a village to cure a rare disease.” 
  3. People are getting down into the details when it comes to promising new treatments, such as gene therapy. At a session about developing partnerships between patients and payers (insurance companies/governments) and providers (doctors/hospitals), the discussion was on providing access to lifesaving treatment and exploring new payment methods such as value- based arrangements and amortization of costs to relieve high short-term costs. A common theme throughout the meeting was rewarding innovation but keeping patient out-of-pocket costs down,” said CSL Behring’s Patrick Collins, Senior Director of Healthcare Policy and External Affairs, who was among the panelists.
  4. NORD welcomed the most senior government official ever to the event. U.S. Health and Human Services Secretary Alex Azar delivered remarks and emphasized the role of newborn screening. “The sooner we identify cases of rare diseases, the earlier babies can be treated, potentially preventing death or disability and enabling them to reach their full potential,” he said.
  5. Technology links the rare disease community. Through Facebook Live, NORD enabled anyone interested in the rare disease advocacy to watch summit presentations remotely. Visit NORD’s Facebook page to watch recorded sessions. Also, it’s not too soon to think about attending next year’s Breakthrough Summit, planned for October 8-9, 2020!