Background: Hemophilia A (HA) is an inherited bleeding disorder, for which the standard of care (SOC) in Canada is prophylactic infusion of clotting factor (CF) VIII (FVIII). Additional on-demand infusions of FVIII or other CF may be administered to treat bleeds as needed. Up to 35% of patients with HA (PwHA) will develop inhibitors to FVIII. These patients are treated with bypassing agents, which may involve frequent and/or large doses infusions. Although HA is a rare disease, there is a substantial economic burden.

OBJECTIVES: Determine the average annual cost for the treatment of severe or moderate patients with HA with inhibitors (inh+) and without inhibitors (inh-) in Canada.

METHODS: Retrospective study using the Canadian Bleeding Disorder Registry data and medical records from 2014 to 2016 at 4 Canadian Hemophilia Treatment Centres. Healthcare resource utilization (HRU) was quantified in Canadian dollars and included: CF, healthcare visits, tests, hospitalizations, and emergency department (ED) visits.

RESULTS: N=56 HA (7 moderate, 49 severe). Five of the 56 patients were inh+, all severe. There were 31 hospitalizations among 16 inh- and 4 among 2 inh+. Median annual ED visits was 0 (0.7) and 1 (0.5) for inh + and 20 inh-. Three inh+ had at least one surgery. Average total healthcare cost was $310,774 in 2014, $327,331 in 2015 and $369,018 in 2016 for inh-; for inh+ the average total cost increased from $1,049,488 in 2014 to $1,707,589 in 2016. More than 97% of the cost was attributed to CF.

CONCLUSIONS: Direct HRU and costs for SOC for treatment of HA in Canada increased from 2014 to 2016. The cost for inh+ patients is consistently higher than for inh- patients. Exploring treatments that reduce bleeding rates and require fewer infusions may be beneficial in lowering the overall cost of treatment for PwHA.