title: Cost effectiveness of recombinant factor VIIa for treatment of intracerebral hemorrhage.
abstract: BACKGROUND
  Phase I/II placebo-controlled clinical trials of recombinant Factor VIIa (rFVIIa) suggested that administration of rFVIIa within 4 hours after onset of intracerebral hemorrhage (ICH) is safe, limits ICH growth, and improves outcomes. We sought to determine the cost-effectiveness of rFVIIa for acute ICH treatment, using published Phase II data. We hypothesized that rFVIIa would have a low marginal cost-effectiveness ratio (mCER) given the poor neurologic outcomes after ICH with conventional management.
  
  
  METHODS
  We performed an incremental cost-effectiveness analysis from the societal perspective, considering conventional management vs. 80 ug/kg rFVIIa treatment for acute ICH cases meeting Phase II inclusion criteria. The time frame for the analysis was 1. 25 years: data from the Phase II trial was used for 90 day outcomes and rFVIIa complications - arterial thromboembolic events (ATE). We assumed no substantial cost differences in care between the two strategies except: 1) cost of rFVIIa (for an 80 mcg/kg dose in an 80 kg patient, assumed cost of $6,408); 2) cost of ATE side effects from rFVIIa (which also decrease quality of life and increase the chance of death); and 3) differential monetary costs of outcomes and their impact on quality of life, including disposition (home vs. nursing home), and outpatient vs. inpatient rehabilitation. Sensitivity analyses were performed to explore uncertainty in parameter estimates, impact of rFVIIa cost, direct cost of neurologic outcomes, probability of ATE, and outcomes after ATE.
  
  
  RESULTS
  In the "base case", treating ICH with rFVIIa dominates the usual care strategy by being more effective and less costly. rFVIIa maintained a mCER < $50,000/QALY over a wide range of sensitivity analyses. Sensitivity analyses showed that the cost of rFVIIa must exceed $14,500, or the frequency of ATE exceed 29%, for the mCER to exceed $50,000/QALY. Varying the cost and/or reducing the utility of health states following ATE did not impact results.
  
  
  CONCLUSIONS
  Based on data from preliminary trials, treating selected ICH patients with rFVIIa results in lower cost and improved clinical outcomes. This potential cost-effectiveness must be considered in light of the Phase III trial results.
authors.affiliation: Department of Neurology, University of Cincinnati, 260 Stetson Street, Suite 2300, Cincinnati, Ohio 45267-05525, USA. brett.kissela@uc.edu
authors.forename: Brett M
authors.initials: BM
authors.lastname: Kissela
authors.forename: Mark H
authors.initials: MH
authors.lastname: Eckman
mesh_terms: D002543:Cerebral Hemorrhage / Q000188:drug therapy* / Q000191:economics*; D017322:Clinical Trials, Phase II as Topic / Q000191:economics; D003362:Cost-Benefit Analysis / Q000191:economics / Q000379:methods; D015942:Factor VIIa / Q000191:economics* / Q000627:therapeutic use*; D006801:Humans; D018803:Models, Economic; D011994:Recombinant Proteins / Q000191:economics / Q000627:therapeutic use
chemical_list: D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa