Bleeding in patients with hemophilia who have inhibitors: Modeling US medical system utilization and cost avoidance between recombinant factor VIIa products with different clinical dosing requirements.

A mainstay of treatment in patients with hemophilia with inhibitors (PWIs) is the use of a recombinant factor VIIa (rFVIIa) bypassing agent. A new rFVIIa product may allow reduced rFVIIa utilization for on-demand treatment of bleeding episodes (BEs). A decision analytic health economic model was developed to compare the utilization and consequent need for bleed-related clinical encounters of 2 rFVIIa products, with the International Nomenclature Name of eptacog alfa (EA) and eptacog beta (EB). This study uses recent, peer-reviewed, and published data from clinical trials with similar endpoints for 1 million insured male lives in the United States. rFVIIa product utilization was modeled in hemophilia (A and B) PWI for on-demand treatment of BEs with rFVIIa treatment. Estimated annual BE rates were modeled to include prophylaxis and on-demand management. The clinical encounter avoidance estimates are based on refractory bleeding through 24 hours. In a cohort of 1 million insured, 5-6 patients with hemophilia A or B with inhibitors annually receive on-demand treatment for a total of 59 mild/moderate BEs. The model suggests that EB requires less unit utilization per BE (13,125 μg and 17,850 μg for the 75μg/kg and 225μg/kg dose regimens, respectively) than EA 90 μg/kg dosing (20,178μg), with wholesale acquisition costs expanding the difference. Further, both EB initial dose regimens would permit decreased total nonmedication health plan spending for the acute treatment of BEs by reducing the need for clinical encounters arising from BEs that fail to respond within 24 hours. With reduced infusion requirements, the model consistently shows that EB could generate lower insured-cohort drug utilization, as well as reduce costly clinical encounters by keeping mild and moderate BEs amenable to home bypassing agent management. The article was funded by HEMA Biologic, LLC. The authors approved all content and results in this article without being subject to sponsor censorship. Mr Jensen, Mr Cyr, and Ms Hathway are employees of PRECISIONheor, which provides consulting services to the pharmaceutical industry, including HEMA Biologics, LLC. Dr Batt is an advisor to PRECISIONheor. Dr Alexander is a former employee of HEMA Biologics, LLC, and provides consulting services to the pharmaceutical industry.