Off-label (OL) use is the practice of prescribing authorised drugs for an unauthorised use to meet the special needs of an individual. Off-label use of medications is very common: some 40 – 60% of all drugs are prescribed off-label (different indication, different dose, different administration, different population...). Not all rare diseases benefit from an authorised and well-evaluated medicinal product, and off-label use of medicines can help many patients. But when a drug is used commonly as OL, it may be better to start a trial than continue with OL use. Otherwise, its ‘new’ use will never be validated. OL use may have a different side effects profile and different efficacy than what is known about the authorised drug and the user should be aware of that and be able to let the doctor know in a quick fashion.
Off-label use cannot be promoted by a company. Companies are frequently fined for off-label promotion. Many approved drugs are first considered useful in other indications but with time, it is seen that in fact, the contrary may be true.
One example is recombinant factor 7a (NovoSeven®, marketed by Novo Nordisk A/S of Denmark) authorised for use in hemophilia. Stanford University saw use of this drug grow by 125 times between 2000 and 2008, although hemophiliacs were using it only 4% of the time. The $10,000-a-dose drug was used during heart surgery and to treat medical problems such as trauma and bleeding in the brain not related to hemophilia. The Stanford researchers contend that such use puts patients at risk for heart attack and stroke because the drug can boost the risk of blood clots.
Dr. Veronica Yank, first author of one of two new studies, said, "Because it's such a powerful clotting agent, it has the potential when used off-label to damage the lives of patients without providing any real benefit."
Yank and her colleagues said that physicians should be cautious with the drug until there's more research into whether it is appropriate to use in certain cases.
The studies, published April 19 2011 in Annals of Internal Medicine, are accompanied by a commentary from Harvard Medical School researchers, which says the research provides "compelling data about the runaway use, uselessness and risk for this expensive treatment."
The studies were funded by the U.S. Agency for Healthcare Quality and Research after previous research raised concerns that RF7a increased the risk for blood clots. The agency asked for an assessment of five off-label uses of RF7a: heart surgery, intracranial hemorrhage, body and brain trauma, liver transplantation and prostate surgery.
SOURCE: Stanford University School of Medicine, news release, April 18, 2011, HealthDay, Copyright (c) 2011 HealthDay. All rights reserved.http://www.nlm.nih.gov/medlineplus/news/fullstory_111100.html (until 17.07.11).