OBJECTIVES : As new treatment options for hemophilia A become available, this analysis compared drug-related costs of the current treatment mix including antihemophilic factor (recombinant) and rurioctocog alfa pegol (SHP660, BAX 855; both Baxalta US Inc. [a Takeda company, Lexington, MA, USA]) with costs of an estimated future treatment mix including antihemophilic factor (recombinant) PEGylated-aucl (Bayer, Whippany, NJ, USA) and emicizumab (Genentech, Inc., South San Francisco, CA, USA).

METHODS : A literature-based model was developed from US commercial health insurer perspective: using a hypothetical plan of 1,000,000 insured patients, 25 patients were estimated to have severe hemophilia A, treated on demand or prophylactically; labeled dosing regimens guided product utilization; rFVIII prophylaxis utilization was adjusted to real-world compliance rates, while 100% compliance was assumed for emicizumab. FVIII inhibitor development was not considered. Market share of current treatments was estimated using prescription data. Future market share (FMS) was forecasted; 17% on-demand patients were assumed to switch to prophylaxis. The model used average sales price (ASP) for product costs. Outputs: per member per month (PMPM) costs and 1-year total annual health plan costs (HPC).

RESULTS : Based on 17% emicizumab FMS, the FM had 16.4% HPC increase ($741,583 overall, $0.06 PMPM) versus the current market. In the FM, emicizumab costs $1,151,245 overall, $0.096 PMPM; antihemophilic factor (recombinant) PEGylated-aucl costs $101,237 overall, $0.0084 PMPM. Antihemophilic factor (recombinant) had a 9.6% decrease ($1,351,454 overall, $0.11 PMPM) and rurioctocog alfa pegol had a 8.8% decrease ($503,200 overall, $0.04 PMPM). Based on 30% emicizumab FMS, the FM had a 26.9% HPC increase ($1,217,164 overall, $0.10 PMPM).

CONCLUSIONS : Based on estimated prophylaxis and compliance rates, adding emicizumab and antihemophilic factor (recombinant) PEGylated-aucl to the managed care drug formulary may be associated with increased PMPM costs versus current rFVIII treatment, given product cost. This analysis didn’t consider clinical outcomes and medical costs.