Orphan Drugs:
Improving the Health of Every Patient
June 4, 2009
U.S. Chamber of Commerce | Washington, D.C.
An orphan or rare disease is one that affects fewer than 200,000 Americans a year. It is estimated that 25 million Americans are affected by one of 6,800 rare or “orphan” conditions. Yet, many suffer for years before being diagnosed with a condition that had eluded doctors. Developing treatments for these life threatening and chronically debilitating rare “orphan” diseases cost millions of dollars.
As the demand for health care reform is being addressed by the current administration, it is important that responsible decisions are being made to care for patients with particular needs. An important aspect of healthcare reform concerns how to continue incentivizing and fostering discovery of new medical therapies treating a rare disease or condition as prescribed in the Orphan Drug Act.
Keynote addresses were delivered by Social Security Administration Commissioner Michael J. Astrue, former general counsel of BioGen and CEO of Transkaryotic Therapies, Inc. (TKT), and Angus Russell, CEO of Shire plc, a leading global pharmaceuticals company.
Download Agenda (PDF)
Speaker Biographies (PDF)
Speaker Presentation Materials:
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W. Bradford Gary (PPT)
Vice President, Government Operations, Allergan, Inc.
Diane Edquist Dorman (PPT)
Vice President, Public Policy, National Organization for Rare Disorders
Stephen C. Groft, Pharm.D. (PPT)
Director, Office of Rare Diseases Research, National Institutes of Health
Jean R. Slutsky, P.A., M.S.P.H. (PPT)
Director, Center for Outcomes and Evidence, Agency for Healthcare Research and Quality, U.S. Department of Health and Human Services
Barry H. Thompson, M.D., F.A.A.P., F.A.C.M.G. (PPT)
Medical Director, American College of Medical Genetics
§ Handout 1 – Newborn Screening Act Sheet (PDF)
§ Handout 2 – Primary or Secondary Hypergalactosemia (PDF)
From left to right: Diane Edquist Dorman, W. Bradford Gary, Winston Wong, and James O'Leary
From left to right: Jeffrey A. Kelman, Stephen C. Groft, and Jean Slutsky
From left to right: Jean Slutsky, Barry H. Thompson, and Timothy R. Cote
A video presentation by Canadian MPS Society board member Simon Ibell.
Event sponsor:
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Event marketing partners:
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For more information about this program, please contact Ben Askin at (202) 463-5500 or ncfevents@uschamber.com.
