Why is Cerezyme so expensive? One reason is that it targets an ultra-rare disease. Drug companies generally avoid developing products for such "orphan diseases" because there is little consumer demand. To spur development of orphan drugs, the U.S. and other countries have enacted legislation granting market exclusivity (in the U.S., seven years) for orphan drug products. Gene transfer products aimed at genetic diseases will likely have orphan disease status, which should prompt us to think carefully about how access to this promising technology platform will be e(i)nsured. (photo credit: klynslis, 2007)
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