Acquired hemophilia A (AHA) is a rare coagulation disease characterized by frequent bleeding episodes treated with plasma-derived products and bypassing agents as rFVIIa and aPCC. Similar to the previous plasma-derived porcine FVIII and without its side effects, pFVIII (Obizur®) is a porcine recombinant factor VIII produced with the recombinant DNA technique. The study analyzes the economic impact of pFVIII compared to the other available therapies in order to manage the bleeding episodes in AHA patients.

To assess the impact of the introduction of pFVIII in the market-mix of products for the management of AHA a budget impact analysis was conducted from the perspective of the Italian National Health System (INHS) and considering a three-year time horizon. Consumption of products, products’ wastage, needs for additional treatment in case of failure of first line therapy, laboratory tests, hospitalization and drug wastage were considered for cost estimation. Model inputs were derived from literature, preliminary experience with the use of pFVIII for compassionate use, and from the updating of previous evidence by data collected among a panel of clinical experts. Univariate sensitivity analysis was performed to explore overall uncertainties in input parameters.

The management of a bleeding episode considering conventional treatment is EUR8,229,621 per year, with an overall cost over three years equal to EUR24,688,864. The introduction of pFVIII leads to an overall costs saving ranging from EUR2,253,938 and EUR1,196,985 when the treatment duration is varied between 5 and 6.5 days, according to data from compassionate use or literature, respectively.

The model outlined a significant reduction of all the components of direct costs for the INHS when Obizur® is introduced into the market with an ex-factory unit price equal to EUR2.32/IU.