Utilization of orphan drugs in the U.S. by usage type 2014

This statistic depicts the percentage of select orphan drugs that were utilized in select way in the U.S. during 2014, by utilization type. According to the data, 98.5 percent of Gralise usage was for off-label use. Orphan drugs, once approved, are sometimes expanded to cover several other indications. In fact, many orphan drugs are utilized more often in non-orphan areas than in the original aimed orphan area.

Percentage of annual utilization among select orphan drugs in the U.S. in 2014, by usage type

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Release date

October 2016


United States

Survey time period


Supplementary notes

* The cumulative price change is based on changes in the unit price for NDC #00173-0806-01 & #53451-0101-01.
** As Iclusig was not approved by the FDA until December, 2012 it was included in the study; however, its pricing and utilization data were from 2013 to 2014 only.
*** Includes patients prescribed Myozyme.

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