#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit.

The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the Marriott Wardman Park Hotel. BioNews Services, which publishes this website, will cover the conference onsite.

“Registration for the summit has been very busy,” Mary Dunkle, NORD’s vice-president of educational initiatives, said in an email. “This is the only conference where you can interact directly with thought leaders from over 100 patient advocacy organizations, government, medical professionals, and the orphan product industry — in an environment of real collaboration and insight.”

Scott Gottlieb, MD, commissioner of the U.S. Food and Drug Administration (FDA), will deliver the keynote speech on “A New Era of Patient-focused Innovation,” which is the theme of this year’s summit.

At least 85 other speakers are on the agenda, including:

• Christopher P. Austin, MD, director of the National Center for Advancing Translational Sciences (NCATS) at National Institutes of Health
• Tanisha Carino, PhD, executive director of FasterCures
• Pushkal Garg, MD, chief medical officer of Alnylam
• Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence
• Ashanthi De Silva, rare disease editor at The Mighty, and the first gene therapy patient
• Shannon Resetich, head of rare diseases for North America at Sanofi Genzyme
• Brendan Hayes, a mother of two children with hemophilia, representing the National Hemophilia Foundation
• NBC news correspondent Richard Engel, who will provide remarks by video

These speakers will join the directors of the FDA’s three medical centers — Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), and Center for Devices and Radiological Health (CDRH)— as well as leaders from the National Institutes of Health (NIH), patient groups, industry, and academia.

“It is clear that the pace of innovation in rare diseases is accelerating,” Peter L. Saltonstall, NORD president and CEO, said in a press release.

“With innovation comes new challenges such as how patients will access these new treatments, whether payers will cover them and what role patients will play in future drug development,” Saltonstall added. “These are among the topics to be discussed at our summit, which provides a unique opportunity to listen to cutting-edge speakers, network with peers, and hear directly from senior FDA staff about their current priorities.”

Among other things, the event features a poster session with 80 presenters sharing abstracts on the theme of life-transforming treatments. There will also be six breakout sessions over two days, and one-on-one networking opportunities. In addition, “Lunch & Learn” sessions will allow attendees to join roundtable discussions in a casual setting.

NORD, founded 35 years ago in the wake of the Orphan Drug Act of 1983, now represents more than 270 disease-specific patient organizations. It seeks to identify, treat, and cure the estimated 7,000 rare diseases that affect 30 million Americans, or nearly 1 in 10 people.

Sponsors of the 2018 NORD Summit include Dohmen, Sanofi Genzyme, Shire and Retrophin. For more information on the conference, download the complete agenda. To register, click here.