Targeted Cures: A Policy Discussion on Treating Patients with Rare Diseases

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Approximately 30 million Americans live with a rare disease, and many more may go undiagnosed or misdiagnosed. With treatments currently available for only 5 percent of rare diseases, how can policymakers and the healthcare industry collaborate to develop innovative new treatments and bring them to market for the patients who need them most? What are the challenges of developing and delivering new treatment options for those suffering from rare diseases? Policymakers, government regulators, and industry leaders will explore the cutting-edge science and medicine driving rare disease advances, and discuss policy considerations for enhancing the research and drug development path and ensuring patient access to innovative treatments for rare diseases.

8:45 AM: Keynote interview with CONGRESSMAN FRED UPTON in conversation with The Hill’s BOB CUSACK

9:05 AM: Keynote interview with SENATOR BOB CASEY in conversation with BOB CUSACK

9:20 AM: Sponsor Perspective presented by STEPHEN J. UBL, President and CEO, PhRMA

9:25 AM: Keynote interview with DR. GEORGE A. SCANGOS, CEO of Biogen, in conversation with BOB CUSACK

9:40 AM: Panel discussion with:
CHRISTOPHER P. AUSTIN, Director, National Center for Advancing Translational Sciences, National Institutes of Health
LORRI KNOX CAREY, ALS Advocate
PETER PITTS, President & Co-Founder, Center for Medicine in the Public Interest
Moderated by The Hill’s SARAH FERRIS

10:05 AM: Program concludes