Health Economic Review of Recombinant Activated Factor VII (Novoseven®) for Treatment of Bleeding Episodes in Hemophilia Patients with Inhibitors.

OBJECTIVE: Severe hemophilia with inhibitors is a rare disease with substantial clinical, humanistic, and economic consequences. Within the past few years, a number of formal economic analyses have emerged which examined the cost of treating bleeding episodes with recombinant activated factor VII (rFVIIa) versus plasma-derived agents. Thus the objective was to review the recent health economic analyses of rFVIIa in the management of patients with hemophilia and inhibitors.

DATA SOURCES: Published, English-language medical literature on the economics of rFVIIa was searched from January 1996 through July 2006 using: the MEDLINE/PubMed database, PubMed related articles feature, company knowledge of published medical literature for rFVIIa that was not indexed by MEDLINE/PubMed, and a thorough review of retrieved article bibliographies.

STUDY SELECTION AND DATA ABSTRACTION: Abstracts selected for full article retrieval/review were those that specifically mentioned (or implied that the full article would address) cost impact, cost of treatment, or cost-effectiveness for rFVIIa in patients with hemophilia and inhibitors. Of 70 abstracts reviewed, 30 articles were selected for retrieval, and from those, thirteen economic analyses (6 burden of illness and 7 comparative studies) met inclusion criteria for data abstraction and synthesis. Seventeen articles were excluded for the following reasons: reports of basic drug acquisition costs without formal economic analysis, single case reports, studies without inhibitor patients, or studies of immune tolerance therapy.

DATA SYNTHESIS: The economic impact of rFVIIa on hospitals and treatment centers occurs primarily during hospitalization to manage major bleeding episodes and allow for elective orthopedic surgeries that would not have been attempted without availability of rFVIIa. Six out of seven comparative analyses for on-demand treatment suggest that total cost of treating a bleeding episode with rFVIIa may be lower than that of using plasma-based agents due to faster bleeding resolution, higher initial efficacy rates, and avoidance of second and third lines of treatment. Dosing assumptions for the various agents compared in the economic analyses were the most sensitive variables.

CONCLUSIONS: The currently available literature suggests that rFVIIa is a cost-effective treatment option and may ultimately lead to improvement in clinical outcomes for patients with hemophilia and inhibitors.