The Future of Rare Disease R&D: Protecting Progress, Achieving Access

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