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.
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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%
Created with Highcharts 7.2.2Usage percentage98.4%98.4%98%98%97.2%97.2%97.2%97.2%96.2%96.2%96%96%95.9%95.9%95.6%95.6%94%94%93.4%93.4%92.9%92.9%92.7%92.7%92.6%92.6%90.4%90.4%84.5%84.5%83.8%83.8%83.5%83.5%82.3%82.3%81.1%81.1%78%78%74.8%74.8%64.1%64.1%62.8%62.8%62.6%62.6%54.9%54.9%54.3%54.3%43.9%43.9%43%43%42.1%42.1%37.5%37.5%34.9%34.9%23.1%23.1%22.6%22.6%19.8%19.8%12.5%12.5%9.3%9.3%7.8%7.8%4.5%4.5%3.2%3.2%2.2%2.2%1.9%1.9%1.8%1.8%1.1%1.1%0.3%0.3%0.1%0.1%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%10.3%10.3%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%0%21.9%21.9%0%0%57.3%57.3%63.6%63.6%52.9%52.9%74.9%74.9%66.7%66.7%72.7%72.7%18.1%18.1%93.2%93.2%0%0%96.6%96.6%90.2%90.2%0%0%33.4%33.4%61.9%61.9%90.5%90.5%1.6%1.6%2%2%2.8%2.8%2.8%2.8%3.8%3.8%4%4%4.1%4.1%4.4%4.4%6%6%6.6%6.6%7.1%7.1%7.3%7.3%7.4%7.4%9.6%9.6%5.2%5.2%16.2%16.2%16.5%16.5%17.7%17.7%18.9%18.9%22%22%25.2%25.2%35.9%35.9%37.2%37.2%37.4%37.4%45.1%45.1%45.7%45.7%56.1%56.1%57%57%36%36%62.5%62.5%7.8%7.8%13.3%13.3%24.5%24.5%5.2%5.2%20.8%20.8%18%18%74.1%74.1%2.3%2.3%96.8%96.8%1.2%1.2%7.9%7.9%98.2%98.2%65.6%65.6%37.8%37.8%9.5%9.5%Orphan useNon-orphan useOff-label useXiaflexVprivKalydecoAmpyraCaprelsaCaystonXalkoriVelcadeJakafiZelborafIclusig**SolirisLumizyme***MozobilPromactaNplateCinryzeTyvasoBerinertKalbitorFerriproxMakenaAdcircaFirazyrOnfiZirganBanzelXenazineSabrilCuvposaVotrientGleevecGamunexSensiparRituxanAfinitorAvastinRemicadeCoartemHerceptinHumiraGraliseHorizant*ColcrysViread
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* 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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AHIP. (October 12, 2016). Percentage of annual utilization among select orphan drugs in the U.S. from 2012 to 2014, by usage type [Graph]. In Statista. Retrieved July 09, 2025, from https://www.statista.com/statistics/874132/orphan-drugs-utilization-by-usage/
AHIP. "Percentage of annual utilization among select orphan drugs in the U.S. from 2012 to 2014, by usage type." Chart. October 12, 2016. Statista. Accessed July 09, 2025. https://www.statista.com/statistics/874132/orphan-drugs-utilization-by-usage/
AHIP. (2016). Percentage of annual utilization among select orphan drugs in the U.S. from 2012 to 2014, by usage type. Statista. Statista Inc.. Accessed: July 09, 2025. https://www.statista.com/statistics/874132/orphan-drugs-utilization-by-usage/
AHIP. "Percentage of Annual Utilization among Select Orphan Drugs in The U.S. from 2012 to 2014, by Usage Type." Statista, Statista Inc., 12 Oct 2016, https://www.statista.com/statistics/874132/orphan-drugs-utilization-by-usage/
AHIP, Percentage of annual utilization among select orphan drugs in the U.S. from 2012 to 2014, by usage type Statista, https://www.statista.com/statistics/874132/orphan-drugs-utilization-by-usage/ (last visited July 09, 2025)
Percentage of annual utilization among select orphan drugs in the U.S. from 2012 to 2014, by usage type [Graph], AHIP, October 12, 2016. [Online]. Available: https://www.statista.com/statistics/874132/orphan-drugs-utilization-by-usage/
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