December 7 at 5:45 am PT/8:45 am ET
More than 25 million Americans live with a rare disease. While scientific advances have improved our ability to diagnose them, and cell and gene therapies offer promise to those afflicted with them, the rare disease community faces unique challenges in accessing appropriate care—from the "diagnostic odyssey" to obtaining insurance coverage. What are the current strategies for advancing access to rare disease therapies? How have these strategies fared? Where are the greatest opportunities to coordinate efforts and resources?
Welcoming Remarks
Esther Krofah
Executive Director, FasterCures, Milken Institute
Opening Remarks
Rashmi Sinha
Co-Founder, Systemic JIA Foundation
Speakers
Christopher Austin
Director, National Center for Advancing Translational Sciences, National Institutes of Health
Sharon Barr
Head of Research, Alexion Pharmaceuticals
Doug Danison
Senior Vice President, Head of Access Value and Evidence Strategy, bluebird bio
Kari Rosbeck
President and CEO, Tuberous Sclerosis Alliance
Michael Sherman
Chief Medical Officer, Harvard Pilgrim Health Care Moderator: Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation
Moderator
Annie Kennedy
Chief of Policy and Advocacy, EveryLife Foundation