The EveryLife Foundation will convene its 10th annual Rare Disease Scientific Workshop, titled “Conceptualizing a Rare Disease Center of Excellence at the FDA”, on Thursday, September 13th in Washington, DC. The goal of the workshop will be to gather key thought-leaders from industry, the Food and Drug Administration (FDA), and patient organizations to discuss potential models, best practices and the pathway forward. Utilizing input from stakeholders across the rare disease community, this workshop will prioritize the discussion and discovery of actionable takeaways.
Senior level officials from the FDA and National Institutes of Health (NIH) have been invited to present along with representatives from industry and patient organizations.
While attendance is free and open to the public, space is limited and the event fills up quickly. To be guaranteed seating, Please contact Carol Kennedy, Chief Development Officer, at ckennedy@rareadvocates.org for information on sponsorship opportunities.
10th Annual Rare Disease Scientific Workshop
Conceptualizing a Rare Disease Center of Excellence at the FDA
Thursday, September 13th, 2018
8:30am – 4:00pm (light breakfast and lunch included)
Willard Intercontinental Hotel
1401 Pennsylvania Ave NW, Washington, DC 20004
DRAFT AGENDA:
8:30 am Breakfast & Registration
9:00 am Success and Progress at the FDA
9:45 am Continued Challenges at the FDA, Patient & Academic Perspectives
11:00 am Continued Challenges at the FDA, Industry Case Studies
12:30 pm Networking Lunch
1:30 pm Potential Model of Center of Excellence for Rare Diseases
Panel Discussion on proposed model
2:45 pm Alternative Concepts for Improving FDA Drug Review
3:45 pm Final Discussion and Thoughts on Next Steps
4:00 pm Adjourn
CONFIRMED SPEAKERS:
- Wilson Bryan, MD, Director, Division of Clinical Evaluation and Pharmacology/Toxicology, Office of Cellular, Tissue, and Gene Therapies; CBER
- Celia M. Witten, PhD, MD, Director of the Office of Cellular, Tissue and Gene Therapy, CBER
- Frank Sasinowski, Director, Hyman, Phelps & McNamara, P.C.
- Brad Glasscock, Group Vice President, Global Regulatory Affairs, BioMarin
- Rich Moscicki, Chief Medical Officer and Executive Vice President, PhRMA
- ArmaGen
- Amyloidosis Research Consortium
- National Organization for Rare Disorders
- Parent Project Muscular Dystrophy
Thank you to our 2018 sponsors to date:
