title: 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.
abstract: 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.
  
  
  CONCLUSIONS
  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.
authors: Pharmacoeconomics & Outcomes Research Iberia, Madrid, Spain.
authors: R
authors: R
authors: Villarrubia
authors: Pharmacoeconomics & Outcomes Research Iberia, Madrid, Spain.
authors: I
authors: I
authors: Oyagüez
authors: La Paz University Hospital, Madrid, Spain.
authors: M T
authors: MT
authors: Álvarez-Román
authors: Malaga Regional University Hospital, Málaga, Spain.
authors: M E
authors: ME
authors: Mingot-Castellano
authors: Vall d'Hebron University Hospital, Barcelona, Spain.
authors: R
authors: R
authors: Parra
authors: Pharmacoeconomics & Outcomes Research Iberia, Madrid, Spain.
authors: M A
authors: MA
authors: Casado
mesh_terms: D019774:Blood Coagulation Factor Inhibitors / Q000276:immunology; D001779:Blood Coagulation Factors / Q000008:administration & dosage / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D016527:Drug Costs; D005169:Factor VIII / Q000276:immunology; D015942:Factor VIIa / Q000008:administration & dosage / Q000627:therapeutic use*; D017048:Health Care Costs; D006467:Hemophilia A / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy* / Q000276:immunology; D006470:Hemorrhage / Q000209:etiology / Q000628:therapy; D006801:Humans; D007518:Isoantibodies / Q000276:immunology; D015233:Models, Statistical; D011292:Premedication; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use; D012720:Severity of Illness Index; D013030:Spain
chemical_list: D019774:Blood Coagulation Factor Inhibitors; D001779:Blood Coagulation Factors; D007518:Isoantibodies; D011994:Recombinant Proteins; C025667:prothrombin complex concentrates; D005169:Factor VIII; C103587:recombinant FVIIa; D015942:Factor VIIa
keywords: cost-analysis; haemophilia; inhibitors; on-demand; prophylaxis