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Creating the Costliest Orphan: The Orphan Drug Act in the Development of Ceredase™

Published online by Cambridge University Press:  10 March 2009

Dana P. Goldman ,
Ann E. Clarke  and
Alan M. Garber
Dana P. Goldman
Affiliation:
Stanford University
Ann E. Clarke
Affiliation:
Stanford University
Alan M. Garber
Affiliation:
Palo Alto Department of Veterans Affairs Medical Center and Stanford University
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Abstract

The FDA recently approved Ceredase™, a new treatment for Gaucher's disease, under the provisions of the Orphan Drug Act. Ceredase™ is unusually expensive, but there are no satisfactory alternative therapies. It appears likely that Ceredase™would not have become available without the protection of the Orphan Drug Act, but its expense and the lack of information about its long-term effects on health raise questions about whether the ODA provides appropriate incentives to develop cost-effective technologies.

Type
Special Section: Orphan Technologies
Information
International Journal of Technology Assessment in Health Care , Volume 8 , Issue 4 , Fall 1992 , pp. 583 - 597
Copyright
Copyright © Cambridge University Press 1992

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