Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors.
“The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told BioNews Services, publisher of this website, by phone from the District of Columbia.
“With all the new DNA modification therapies, small molecules, gene therapies, and acceleration in the diagnosis of rare diseases — we’re adding new ones weekly — this is the most exciting time to be in the field, but also the most dizzying,” said Summar, who’s also director of the Rare Disease Institute at Children’s National Medical Center. “So keeping up with it all requires a lot of attention.”
At least 800 people are expected to attend the 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit, set for Oct. 21-22 at Washington’s Marriott Wardman Park Hotel.
The event, whose theme is “The Time is Now,” features a keynote speech by Ned Sharpless, MD, acting commissioner of the U.S. Food and Drug Administration (FDA).
Also scheduled to speak are the directors of three key FDA divisions: the Center for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research (CBER), and the Center for Devices and Radiological Health (CDRH).
Other speakers on the agenda include: former FDA Commissioner Scott Gottlieb, MD, now with the American Enterprise Institute; Steven Pearson, MD, founder and president of the Institute for Clinical and Economic Review (ICER); Brad Wong, president and CEO of the Pulmonary Hypertension Association, and Dawn Rotellini, senior vice-president at the National Hemophilia Foundation.
In addition, the agenda includes speakers from the National Institutes of Health (NIH), patient advocacy groups, industry, and academia. There’s also an expansive poster session, one-to-one networking opportunities, and nine breakthrough topic sessions over two days.
These sessions cover topics such as gene therapy, newborn screening, curative therapies for rare cancers, investment opportunities in orphan products, social media platforms for organizing patients, and patient registries and natural history studies.
“It’s clear that now is the time for driving innovation, advocacy, research and new therapies,” said Peter L. Saltonstall, NORD’s president and CEO. “This year we’ll be taking on timely topics such as the challenges of gene therapy, retaining treatment affordability while sustaining innovation, and fostering dialogue among payers, providers, and patients.”
Two women who not only became patient advocates themselves, but went on to acquire the education and skills necessary to advance research on rare diseases, also will speak, as part of a patient/caregiver session titled “There is No Time Like the Present: Inspiration to Action.”
Karen Pignet-Aiach is founder and CEO of Lysogene, a Paris-based pharma company focused on rare neurogenerative diseases. A financial consultant, Pignet-Aiach changed careers in 2005 after her daughter, Ornella, was diagnosed with Sanfilippo syndrome, an incurable disease. Four years later, she founded Lysogene, which today has a gene therapy in development.
Terry Jo Bichell of Nashville was a practicing nurse midwife with a degree in public health — teaching women how to breastfeed and care for their babies — when her own son, Louie, was diagnosed with Angelman syndrome. Ultimately, Terry Jo went back to school at Vanderbilt University, earning a PhD in neuroscience from the university’s Brain Institute. She’s also the Tennessee volunteer ambassador for NORD’s Rare Action Network.
NORD, founded in 1983, speaks for nearly 300 patient advocacy groups, from the Alport Syndrome Foundation and the Cystic Fibrosis Foundation to the XLH Network, which represents patients with X-linked hypophosphatemia.
Among the participants at last year’s NORD Summit, 40% represented patient advocacy groups; 30% were from the pharmaceutical industry; 15% worked on consulting; 5% were government officials; another 5% were in research or academia; 3% were healthcare providers or investors; and 2% were journalists.
Sponsors of the NORD’s 2019 summit include Sanofi Genzyme, Eversana, Retrophin, and Takeda. For a complete listing of the more than 95 speakers, panel discussions, lunch and learn groups, and more, download the agenda. To register for the summit, click here; information about exhibiting and corporate sponsorship opportunities can be found here.
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