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Rare Diseases and Orphan Products Breakthrough Summit, October 15th and 16th in Washington, D.C "A New Era of Patient-Focused Innovation"

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Monday, October 08, 2018

At NORD, we are busy gearing up for the 2018 Rare Diseases and Orphan Products Breakthrough Summit, October 15th and 16th in Washington, D.C.and I’m really looking forward to exploring this year’s Summit theme, “A New Era of Patient-Focused Innovation,” from a variety of perspectives. We’re in an exciting time, with rare disease patients and caregivers helping to build momentum in new ways, such as through patient-reported outcomes and real-world evidence. At the Summit, we’ll hear from FDA senior leadership regarding the status and future directions of Patient-Focused Drug Development and examine the patient/caregiver roles in terms of awareness, education, research, and advancing the dialogue on key policy issues.  Day 1 will begin with a Patient/Caregiver Opening Address, featuring a panel of young advocates I think you’ll find as inspiring as I do.

 You won’t want to miss our keynote speaker, FDA Commissioner Scott Gottlieb, or hearing from other leadership across the FDA and NIH as they share their current priorities and predictions for rare disease research and orphan drug development. Registration for the Summit has been very busy and this year we are looking forward to welcoming 700 individuals from across the rare disease space. This is the only conference where you can interact directly with thought leaders from more than 100 patient advocacy organizations, government, medical professionals, and the orphan product industry—in an environment of real collaboration and insight. 

 Through expert panels, we’ll delve into the biggest rare disease issues of the day at the Summit, such as how the FDA is modernizing its drug review processes, along with recently issued guidances. Gene therapy is another topic we’ll explore—including the perspectives of patients on this potentially curative treatment; for example, we’ll hear from Ashanthi DeSilva, who underwent the first human gene therapy as a child. In addition, there will be breakout sessions, 14 lunch and learns to choose from, and much more.

 There’s still time to register! If you are a part of the rare disease community or interested in advocating for it, I urge you to join us. Please visit NORDSummit.org for information on what promises to be the most important rare disease conference of the year. I hope to see you soon in DC!

Author: Mary Dunkle, Vice President of Educational Initiatives for the National Organization for Rare Disorders (NORD)
Source: NORD
1.0
3.0 from 2 votes
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