Creating the costliest orphan. The Orphan Drug Act in the development of Ceredase.
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.
Goldman
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DP
Stanford University.
Clarke
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AE
Garber
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AM
D000293:Adolescent; D000328:Adult; D002648:Child; D003365:Costs and Cost Analysis; D005776:Gaucher Disease / Q000188:drug therapy* / Q000453:epidemiology; D005962:Glucosylceramidase / Q000191:economics*; D006801:Humans; D015994:Incidence; D007223:Infant; D009965:Orphan Drug Production / Q000191:economics* / Q000331:legislation & jurisprudence*; D015995:Prevalence; D014481:United States / Q000453:epidemiology
C070292:alglucerase; D005962:Glucosylceramidase