In 1983, the Orphan Drug Act was promulgated, and the FDA Office of Orphan Drug Development was created and incentives were created for manufacturers to develop drugs and biological products (Fig. 7.13). There are estimated to be greater than 6,000 rare diseases and related conditions. A sponsor can request the FDA grant an orphan drug designation for a product to treat a rare disease or condition. The sponsor must demonstrate the disease is prevalent in less than 200,000 people in the United States, or if it is prevalent in more than 200,000 people, that upon commercialization, the sponsor would likely not be able to recover the cost of development. If orphan drug designation is granted, once the product is approved, the product will have 7 years of market exclusivity. The exclusivity does not allow a competitor to market the same drug for the same indication until the end of the 7-year exclusivity period. The Genzyme company particularly has used this regulatory approach in the rare, serious, and untreatable enzyme deficiency diseases; for example, Cerezyme® in Gaucher disease, Fabrazyme® in Fabry disease, and Aldurazyme® in Hurler syndrome. Serono used this process for Serostim®, growth hormone, for AIDS wasting syndrome. Amgen used this approach for Epogen® in anemia of renal disease because in the 1980s, the condition was documented to be within the 200,000 patient limit.
There is also an opportunity for a sponsor to receive tax credits on up to 50% of the dollars spent for certain clinical testing in the United States to develop the orphan drug. The FDA may offer financial grants to investigators or sponsors to defray some of the costs of developing an orphan drug. The orphan drug regulations are intended to provide incentives to pharmaceutical and biotechnology companies to develop drugs for treatment of diseases where there is little or no commercial incentive to do so. The product pipeline and commercialization of drugs for rare diseases has been affected significantly by the Orphan Drug Act. Prior to the act, there were 15 products approved for orphan disease. Today, there are more than 250 orphan drug products approved. Examples include Vidaza® (azacitidine) for the treatment of myelodysplastic syndrome and Clolar® (clofarabine) for the treatment of acute lymphoblastic leukemia. New molecular entities (NMEs) for orphan drugs also are approved faster by the FDA versus all other NMEs, about 10-12 months faster in 2000 to 2001.
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