title: COST-UTILITY ANALYSIS OF PRIMARY PROPHYLAXIS, COMPARED WITH ON-DEMAND TREATMENT, FOR PATIENTS WITH SEVERE HEMOPHILIA TYPE A IN COLOMBIA.
abstract: OBJECTIVE
  This article presents a cost-utility analysis from the Colombian health system perspective comparing primary prophylaxis to on-demand treatment using exogenous clotting factor VIII (FVIII) for patients with severe hemophilia type A.
  
  
  METHODS
  We developed a Markov model to estimate expected costs and outcomes (measured as quality-adjusted life-years, QALYs) for each strategy. Transition probabilities were estimated using published studies; utility weights were obtained from a sample of Colombian patients with hemophilia and costs were gathered using local data. Both deterministic and probabilistic sensitivity analysis were performed to assess the robustness of results.
  
  
  RESULTS
  The additional cost per QALY gained of primary prophylaxis compared with on-demand treatment was 105,081,022 Colombian pesos (COP) (55,204 USD), and thus not considered cost-effective according to a threshold of up to three times the current Colombian gross domestic product (GDP) per-capita. When primary prophylaxis was provided throughout life using recombinant FVIII (rFVIII), which is much costlier than FVIII, the additional cost per QALY gained reached 174,159,553 COP (91,494 USD).
  
  
  CONCLUSIONS
  using a decision rule of up to three times the Colombian GDP per capita, primary prophylaxis (with either FVIII or rFVIII) would not be considered as cost-effective in this country. However, a final decision on providing or preventing patients from primary prophylaxis as a gold standard of care for severe hemophilia type A should also consider broader criteria than the incremental cost-effectiveness ratio results itself. Only a price reduction of exogenous FVIII of 50 percent or more would make primary prophylaxis cost-effective in this context.
authors.affiliation: Instituto de Evaluación Tecnológica en Salud - IETShcastro@hsph.harvard.edu;dochecj@hotmail.com.
authors.forename: Héctor Eduardo
authors.initials: HE
authors.lastname: Castro Jaramillo
authors.affiliation: Instituto de Evaluación Tecnológica en Salud - IETS.
authors.forename: Mabel
authors.initials: M
authors.lastname: Moreno Viscaya
authors.affiliation: Instituto de Evaluación Tecnológica en Salud - IETS.
authors.forename: Aurelio E
authors.initials: AE
authors.lastname: Mejia
mesh_terms: D003105:Colombia; D003362:Cost-Benefit Analysis; D005169:Factor VIII / Q000191:economics* / Q000627:therapeutic use; D006467:Hemophilia A / Q000188:drug therapy*; D006801:Humans; D008390:Markov Chains; D018803:Models, Economic; D019057:Quality-Adjusted Life Years; D012720:Severity of Illness Index
chemical_list: C427184:recombinant factor VIII SQ; C078147:F8 protein, human; D005169:Factor VIII
keywords: Colombia; Cost-benefit analysis; Healthcare rationing; Hemophilia A