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

• Published in: Haemophilia : the official journal of the World Federation of Hemophilia Vol. 21; no. 3; pp. 320 - 329
• Main Authors: Villarrubia, R., Oyagüez, I., Álvarez-Román, M. T., Mingot-Castellano, M. E., Parra, R., Casado, M. A.
• Format: Journal Article
• Language: English
• Published: England Blackwell Publishing Ltd 01.05.2015; Wiley Subscription Services, Inc
• Subjects: Bleeding; Blood Coagulation Factor Inhibitors - immunology; Blood Coagulation Factors - administration & dosage; Blood Coagulation Factors - therapeutic use; Coagulation factors; Cost analysis; Cost control; Cost-Benefit Analysis; Disease management; Disease prevention; Drug Costs; Drug dosages; Factor VIIa - administration & dosage; Factor VIIa - therapeutic use; Factor VIII - immunology; haemophilia; Health Care Costs; Hemophilia; Hemophilia A - complications; Hemophilia A - diagnosis; Hemophilia A - drug therapy; Hemophilia A - immunology; Hemorrhage - etiology; Hemorrhage - therapy; Humans; inhibitors; Isoantibodies - immunology; Models, Statistical; Muscles; on-demand; Patients; Premedication; Prophylaxis; Prothrombin; Recombinant Proteins - administration & dosage; Recombinant Proteins - therapeutic use; Sensitivity analysis; Severity of Illness Index; Soft tissues; Spain; haemophilia; cost-analysis; inhibitors; on-demand; prophylaxis
• ISSN: 1351-8216; 1365-2516; 1365-2516
• DOI: 10.1111/hae.12681

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 μg kg−1 (rFVIIa) and 235.28 U kg−1 (aPCC). The average bleeding cost (€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 (€2013) derived from local databases. Sensitivity analyses (SA) were performed. Results: Annual cost of aPCC prophylaxis (€524 358) was 16% lower than on‐demand treatment with rFVIIa (€627 876). Yearly drug costs were €497 017 for aPCC (€73 166 for on‐demand treatment and €423 850 for prophylaxis), and €548 870 for rFVIIa. Disease management cost (€2645 per year) and surgical procedures (€708 per year) were common for both strategies. In the SA prophylactic treatment led to savings between €26 225 and €‐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 €100 000 per‐patient per year, being 16% less costly than on‐demand treatment with rFVIIa, for the Spanish NHS.