Orphan drug utilization types U.S. 2012-2014

This statistic depicts the percentage of select orphan drugs that were utilized in select way in the U.S. between 2012 to 2014, by utilization type. According to the data, 95 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. from 2012 to 2014, by usage type

Characteristic	Orphan use	Non-orphan use	Off-label use
Xiaflex	98.4%	0%	1.6%
Vpriv	98%	0%	2%
Kalydeco	97.2%	0%	2.8%
Ampyra	97.2%	0%	2.8%
Caprelsa	96.2%	0%	3.8%
Cayston	96%	0%	4%
Xalkori	95.9%	0%	4.1%
Velcade	95.6%	0%	4.4%
Jakafi	94%	0%	6%
Zelboraf	93.4%	0%	6.6%
Iclusig**	92.9%	0%	7.1%
Soliris	92.7%	0%	7.3%
Lumizyme***	92.6%	0%	7.4%
Mozobil	90.4%	0%	9.6%
Promacta	84.5%	10.3%	5.2%
Nplate	83.8%	0%	16.2%
Cinryze	83.5%	0%	16.5%
Tyvaso	82.3%	0%	17.7%
Berinert	81.1%	0%	18.9%
Kalbitor	78%	0%	22%
Ferriprox	74.8%	0%	25.2%
Makena	64.1%	0%	35.9%
Adcirca	62.8%	0%	37.2%
Firazyr	62.6%	0%	37.4%
Onfi	54.9%	0%	45.1%
Zirgan	54.3%	0%	45.7%
Banzel	43.9%	0%	56.1%
Xenazine	43%	0%	57%
Sabril	42.1%	21.9%	36%
Cuvposa	37.5%	0%	62.5%
Votrient	34.9%	57.3%	7.8%
Gleevec	23.1%	63.6%	13.3%
Gamunex	22.6%	52.9%	24.5%
Sensipar	19.8%	74.9%	5.2%
Rituxan	12.5%	66.7%	20.8%
Afinitor	9.3%	72.7%	18%
Avastin	7.8%	18.1%	74.1%
Remicade	4.5%	93.2%	2.3%
Coartem	3.2%	0%	96.8%
Herceptin	2.2%	96.6%	1.2%
Humira	1.9%	90.2%	7.9%
Gralise	1.8%	0%	98.2%
Horizant*	1.1%	33.4%	65.6%
Colcrys	0.3%	61.9%	37.8%
Viread	0.1%	90.5%	9.5%

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Release date

October 2016

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Region

United States

Survey time period

01 January 2012 to 31 December 2014

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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