title: Budget Impact Analysis of Prolonged Half-Life Recombinant FVIII Therapy for Hemophilia in the United States.
abstract: Hemophilia A is a factor VIII deficiency, associated with spontaneous, recurrent bleeding episodes. This may lead to comorbidities such as arthropathy and joint replacement, which contribute to morbidity and increased health care expenditure. Recombinant factor VIII Fc fusion protein (rFVIIIFc), a prolonged half-life factor therapy, requires fewer infusions, resulting in reduced treatment burden.
  
  
  
  Use a budget impact analysis to assess the potential economic impact of introducing rFVIIIFc to a formulary from the perspective of a private payer in the United States.
  
  
  
  The budget impact model was developed to estimate the potential economic impact of adding rFVIIIFc to a private payer formulary across a 2-year time period. The eligible patient population consisted of inhibitor-free adults with severe hemophilia A, receiving recombinant-based episodic or prophylaxis treatment regimens. Patients were assumed to switch from conventional recombinant factor treatment to rFVIIIFc. Only medication costs were included in the model.
  
  
  
  The introduction of rFVIIIFc is estimated to have a budget impact of 1.4% ($0.12 per member per month) across 2 years for a private payer population of 1,000,000 (estimated 19.7 individuals receiving treatment for hemophilia A). The introduction of rFVIIIFc is estimated to prevent 124 bleeds across 2 years at a cost of $1891 per bleed avoided.
  
  
  
  Hemophilia A is a rare disease with a low prevalence; therefore, the overall cost to society of introducing rFVIIIFc is small. Considerations for comprehensively assessing the budget impact of introducing rFVIIIFc should include episodic and prophylaxis regimens, bleed avoidance, and annual factor consumption required under alternative scenarios.
authors.affiliation: ICON plc, Vancouver, British Columbia, Canada. Electronic address: Suzanne.lane@iconplc.com.
authors.forename: Suzanne
authors.initials: S
authors.lastname: McMullen
authors.affiliation: Biogen, Weston, MA, USA.
authors.forename: Brieana
authors.initials: B
authors.lastname: Buckley
authors.affiliation: Biogen, Weston, MA, USA.
authors.forename: Eric
authors.initials: E
authors.lastname: Hall
authors.affiliation: Biogen, Weston, MA, USA.
authors.forename: Jon
authors.initials: J
authors.lastname: Kendter
authors.affiliation: ICON plc, Vancouver, British Columbia, Canada.
authors.forename: Karissa
authors.initials: K
authors.lastname: Johnston
mesh_terms: D000328:Adult; D002017:Budgets / Q000706:statistics & numerical data; D005169:Factor VIII / Q000191:economics* / Q000493:pharmacokinetics* / Q000627:therapeutic use; D005252:Fees, Pharmaceutical / Q000706:statistics & numerical data*; D006207:Half-Life; D006467:Hemophilia A / Q000191:economics / Q000628:therapy*; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D007141:Immunoglobulin Fc Fragments / Q000191:economics* / Q000627:therapeutic use; D007343:Insurance Carriers / Q000191:economics*; D008297:Male; D017059:Models, Econometric; D011993:Recombinant Fusion Proteins / Q000191:economics* / Q000493:pharmacokinetics* / Q000627:therapeutic use
chemical_list: D007141:Immunoglobulin Fc Fragments; D011993:Recombinant Fusion Proteins; C587014:factor VIII-Fc fusion protein; D005169:Factor VIII
keywords: budget impact; economic impact; hemophilia A; rFVIIIFc