On-Label Versus Off-Label Use of Recombinant Activated Factor VII: A Comprehensive Review of Use in Two Canadian Centers

Recombinant activated factor VII (rFVIIa; NovoSeven®, NiaStase®, Novo Nordisk, Bagsværd, Denmark) was originally developed for the treatment of bleeds in patients with hemophilia and inhibitors. However, the agent is increasingly being employed in “off-label”/unlicensed indications. Consequently there is a need to undertake comprehensive reviews of rFVIIa use; the resulting information will facilitate understanding of how the agent is currently being employed and help to determine trends in its use. This article considers two recently reported reviews describing the use of rFVIIa in two heavily populated regions of Canada-regions with a combined population capture area of approximately 8.5 million people. The reviews report rFVIIa use in a total of 196 patients who collectively received 15,262.8 mg of rFVIIa. Both reviews obtained similar findings and reached similar conclusions: the majority of patients receiving rFVIIa are being treated for “off-label” indications, with numbers of such patients having grown rapidly between the years 2000 and 2005. However, hemophilia patients still account for the vast majority of rFVIIa use, as small numbers of hemophilia patients can consume large quantities of the agent. It is important to be aware of the increasing use of rFVIIa in off-label indications.