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.
Thoene
J G
JG
Department of Pediatrics, University of Michigan Medical Center, Ann Arbor 48109-2029.
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