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

On Wednesday, May 11, The Hill convened a discussion on delivering innovative new treatments to rare disease patients. Topics of discussion included: public-private partnerships to develop treatments and bring them to market, the cutting-edge science and medicine driving rare disease advances, and the path to making care accessible to the patients who need it most.

Featured speakers included:

SENATOR BOB CASEY (D-PA)

CONGRESSMAN FRED UPTON (R-MI-6), Chairman, House Committee on Energy & Commerce

CHRISTOPHER P. AUSTIN, M.D., 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

DR. GEORGE A. SCANGOS, CEO, Biogen

STEPHEN J. UBL, President & CEO, PhRMA