title: Pharmacoeconomic analysis of recombinant factor VIIa versus APCC in the treatment of minor-to-moderate bleeds in hemophilia patients with inhibitors.
abstract: OBJECTIVE
  To compare the cost-effectiveness of three treatment regimens using recombinant activated Factor VII (rFVIIa), NovoSeven, and activated prothrombin-complex concentrate (APCC), FEIBA VH, for home treatment of minor-to-moderate bleeds in hemophilia patients with inhibitors.
  
  
  METHODS
  A literature-based, decision-analytic model was developed to compare three treatment regimens. The regimens consisting of first-, second-, and third-line treatments were: rFVIIa-rFVIIa-rFVIIa; APCC-rFVIIa-rFVIIa; and APCC-APCC-rFVIIa. Patients not responding to first-line treatment were administered second-line treatment, and those failing second-line received third-line treatment. Using literature and expert opinion, the model structure and base-case inputs were adapted to the US from a previously published analysis. The percentage of evaluable bleeds controlled with rFVIIa and APCC were obtained from published literature. Drug costs (2005 US$) based on average wholesale price were included in the base-case model. Univariate and probabilistic sensitivity analyses (second-order Monte Carlo simulation) were conducted by varying the efficacy, re-bleeding rates, patient weight, and dosing to ascertain robustness of the model.
  
  
  RESULTS
  In the base-case analysis, the average cost per resolved bleed using rFVIIa as first-, second-, and third-line treatment was $28 076. Using APCC as first-line and rFVIIa as second- and third-line treatment resulted in an average cost per resolved bleed of $30 883, whereas the regimen using APCC as first- and second-line, and rFVIIa as third-line treatment was the most expensive, with an average cost per resolved bleed of $32 150. Cost offsets occurred for the rFVIIa-only regimen through avoidance of second and third lines of treatment. In probabilistic sensitivity analyses, the rFVIIa-only strategy was the least expensive strategy more than 68% of the time.
  
  
  CONCLUSIONS
  The management of minor-to-moderate bleeds extends beyond the initial line of treatment, and should include the economic impact of re-bleeding and failures over multiple lines of treatment. In the majority of cases, the rFVIIa-only regimen appears to be a less expensive treatment option in inhibitor patients with minor-to-moderate bleeds over three lines of treatment.
authors.affiliation: Novo Nordisk Inc., Princeton, NJ, USA. ajsh@novonordisk.com
authors.forename: Ashish V
authors.initials: AV
authors.lastname: Joshi
authors.forename: Jennifer M
authors.initials: JM
authors.lastname: Stephens
authors.forename: Vicki
authors.initials: V
authors.lastname: Munro
authors.forename: Prasad
authors.initials: P
authors.lastname: Mathew
authors.forename: Marc F
authors.initials: MF
authors.lastname: Botteman
mesh_terms: D001779:Blood Coagulation Factors / Q000191:economics* / Q000627:therapeutic use; D016527:Drug Costs; D005167:Factor VII / Q000191:economics* / Q000627:therapeutic use; D015942:Factor VIIa; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy* / Q000191:economics; D006470:Hemorrhage / Q000188:drug therapy* / Q000191:economics / Q000209:etiology; D006490:Hemostatics / Q000191:economics* / Q000627:therapeutic use; D006801:Humans; D011994:Recombinant Proteins / Q000191:economics / Q000627:therapeutic use; D016896:Treatment Outcome; D014481:United States
chemical_list: D001779:Blood Coagulation Factors; D006490:Hemostatics; D011994:Recombinant Proteins; C025667:prothrombin complex concentrates; D005167:Factor VII; C103587:recombinant FVIIa; D015942:Factor VIIa