Current status of orphan disease drug development.
The Orphan Drug Act has successfully stimulated the production of many orphan products for a number of orphan diseases. The success of its exclusive marketing provision in bringing otherwise unprofitable products to market has attracted the attention of manufacturers who use this provision to gain a monopoly for products with much larger annual sales than were contemplated by the original legislation. Corrective legislation to close this loophole is being prepared for introduction to Congress.
Department of Pediatrics, University of Michigan Medical Center, Ann Arbor 48109-2029.
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D000243:Adenosine Deaminase / Q000627:therapeutic use; D000287:Administration, Topical; D001905:Botulinum Toxins / Q000627:therapeutic use; D003254:Consumer Advocacy; D017373:Cyproterone Acetate / Q000627:therapeutic use; D004341:Drug Evaluation / Q000191:economics* / Q000331:legislation & jurisprudence*; D004345:Drug Industry / Q000191:economics; D004921:Erythropoietin / Q000627:therapeutic use; D005776:Gaucher Disease / Q000188:drug therapy; D005962:Glucosylceramidase / Q000627:therapeutic use; D006801:Humans; D008389:Marketing of Health Services / Q000191:economics; D008795:Metronidazole / Q000627:therapeutic use; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence* / Q000592:standards*; D014266:Trientine / Q000627:therapeutic use
D004921:Erythropoietin; D008795:Metronidazole; C070292:alglucerase; D017373:Cyproterone Acetate; D005962:Glucosylceramidase; D001905:Botulinum Toxins; D000243:Adenosine Deaminase; C051409:pegademase bovine; D014266:Trientine