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An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.n average the cost per patient for orphan drugs is six times that of non-orphan drugs, a clear indication of their pricing power.. Partly as a result of the 1983 US Orphan Drug Act, Japan adopted it in 1993 as did the European Union in 2000.
Currently, there are 32 orphan drugs designated for treatment some aspect of sickle cell disease. Only hydroxyurea has orphan drug approval to reduce the frequency of painful crises and the need for blood transfusions in adult patients with sickle cell anemia with recurrent moderate to severe painful crises.That was approved in 1998.ickle cell disease is a rare genetic condition that affects 90,000 to 100,000 Americans, mostly African Americans.