Consensus Recommendations for the Off-Label Use of Recombinant Human Factor VIIa (NovoSeven®) Therapy

ABSTRACT Objective: To rationalize decision-making concerning the growing and costly off-label use of recombinant human factor VII (rFVIIa, NovoSeven®) therapy. Options: Using rFVIIa therapy for patients with bleeding or providing usual care (e.g., continued clotting factor replacement). Outcome: Morbidity and mortality related to continued bleeding or thrombosis. Evidence: A literature review was performed to assess the efficacy and safety of offlabel rFVIIa therapy. An expert consensus panel reviewed the data. Most studies were case series or reports. Only four randomized trials were available in the peer-reviewed literature at the time of the panel’s deliberations. The Rand Corporation/University of California at Los Angeles Appropriateness Method was used to rate 41 clinical scenarios as “appropriate,” “uncertain,” or “inappropriate” in a comparison of risks and benefits in an environment