Cost analysis of prophylaxis with activated prothrombin complex concentrate vs. on-demand therapy with activated factor VII in severe haemophilia A patients with inhibitors, in Spain

Objective: A cost analysis model was developed to compare annual cost of prophylaxis with activated prothrombin complex concentrate (aPCC) vs. on-demand therapy with activated recombinant factor VII (rFVIIa) in severe haemophilia A patients with inhibitors for the Spanish National Health System (NHS). Methods: Model inputs were drug cost for prophylaxis (aPCC) and for on-demand treatment (rFVIIa or aPCC); bleeding episodes management (excluding bypassing agent cost); surgical costs and disease management (excluding bleeding episodes). Annual bleeding episodes treated on-demand was assumed to be 25, whereas breakthrough bleeds on prophylaxis was 8. Dose for prophylaxis was 75.72 U kg(-1), three times per week. The total on-demand dose/bleeding episode was 679.66 lg kg(-1) (rFVIIa) and 235.28 U kg(-1) (aPCC). The average bleeding cost ((sic)2998) considered different bleeding sites (62.5% joints, 28.6% muscles and soft tissues, 3.6% mucocutaneous tissues and 5.4% other areas). A 7.5% deduction was applied to ex-factory drug prices. Unitary costs ((sic)2013) derived from local databases. Sensitivity analyses (SA) were performed. Results: Annual cost of aPCC prophylaxis ((sic)524 358) was 16% lower than on-demand treatment with rFVIIa ((sic)627 876). Yearly drug costs were (sic)497 017 for aPCC ((sic)73 166 for on-demand treatment and (sic)423 850 for prophylaxis), and (sic)548 870 for rFVIIa. Disease management cost ((sic)2645 per year) and surgical procedures ((sic)708 per year) were common for both strategies. In the SA prophylactic treatment led to savings between (sic)26 225 and (sic)-1 008 960. Conclusion: Prophylaxis with aPCC reduces number of bleeding episodes in severe haemophilia A patients with inhibitors. aPCC prophylaxis resulted in savings in excess of (sic)100 000 per-patient per year, being 16% less costly than on-demand treatment with rFVIIa, for the Spanish NHS.