Creating the Costliest Orphan:The Orphan Drug Act in the Development of Ceredase™
- 1 January 1992
- journal article
- Published by Cambridge University Press (CUP) in International Journal of Technology Assessment in Health Care
- Vol. 8 (4) , 583-597
- https://doi.org/10.1017/s0266462300002294
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.Keywords
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