Orphan drug utilization types U.S. 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

Characteristic	Orphan use	Non-orphan use	Off-label use
Xiaflex	98.1	0	1.9
Kalydeco	97.9	0	2.1
Ampyra	97.2	0	2.8
Caprelsa	96.7	0	3.3
Cayston	96.3	0	3.7
Xalkori	95.2	0	4.8
Vpriv	95.1	0	4.9
Iclusig**	95	0	5
Velcade	94.7	0	5.3
Soliris	94.2	0	5.8
Jakafi	92.2	0	7.8
Promacta	89.5	5.5	4.9
Zelboraf	88.6	0	11.4
Lumizyme***	88.1	0	11.9
Mozobil	87.5	0	12.5
Berinert	86.4	0	13.6
Nplate	84.8	0	15.2
Cinryze	83.1	0	16.9
Tyvaso	82.9	0	17.1
Kalbitor	81.8	0	18.2
Ferriprox	76.3	0	23.7
Firazyr	65.6	0	34.4
Makena	62.9	0	37.1
Adcirca	60.2	0	39.8
Onfi	54.5	0	45.5
Zirgan	52.2	0	47.8
Banzel	44.7	0	55.3
Sabril	43.8	22.9	33.3
Xenazine	39.1	0	60.9
Cuvposa	34.1	0	65.9
Votrient	32.4	60	7.6
Gamunex	23.7	52.9	23.4
Sensipar	19.4	75.9	4.7
Gleevec	19.3	67.6	13.1
Rituxan	12.8	64.9	22.3
Afinitor	10.7	70.4	18.9
Avastin	10.2	16.3	73.5
Herceptin	2.4	96.4	1.3
Humira	2.4	90.9	6.7
Coartem	2.2	0	97.8
Remicade	1.6	96	2.4
Gralise	1.5	0	98.5
Horizant*	1	28.4	70.5
Colcrys	0.3	62.8	36.9
Viread	0	90.9	9.1

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Release date

October 2016

More information

Region

United States

Survey time period

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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