OBJECTIVES: To assess the cost-utility of turoctocog alfa pegol for the prophylactic treatment of severe hemophilia A patients without inhibitors in the Colombian health system.

METHODS: The study considered the recombinant factor VIII (rFVIII) extended half-life (EHL) and standard half-life (SHL) products for hemophilia A without inhibitors in prophylaxis, available in the Colombian market. A previously developed pharmacodynamic/pharmacokinetic decision model was adapted. Patients can transit through four health states according to their factor VIII activity levels and, also, through three additional states depending on age stratification; the cycle length was 28 days, and the time horizon was 70 years. Clinical events considered were mortality, total bleeds, traumatic bleeds, joint bleeds and spontaneous bleeds. Resource costs were estimated from a Colombian health system perspective based on the national drug pricing information system and expressed in US dollars. Deterministic and probabilistic sensitivity analyses were implemented.

RESULTS: Turoctocog alfa pegol prophylaxis was the alternative associated with more QALYs, the incremental QALYs resulted in a range of 0.22-1.76 compared with the other treatments. The use of turoctocog alfa pegol prophylaxis resulted in a 9.7-50.7% reduction in total bleeding episodes, resulting in lower associated costs for the treatment of spontaneous bleeds. Turoctocog alfa pegol prophylaxis, compared to EHL factors, was a dominant strategy, that is, less costly and more effective.

CONCLUSIONS: Given the assumptions of the study, the use of turoctocog alfa pegol for hemophilia A patients without inhibitors in Colombia results in lower treatment costs of spontaneous bleeds, fewer total bleeds and more QALYs.