Comparison of Real-World Dose and Consumption for Two Extended Half-Life Recombinant Factor VIII Products for the Treatment of Hemophilia A in the United States

Yanyu Wu,1 Shawn X Sun,1 Tao Fan2 1Global Evidence & Outcomes, Data Sciences Institute, Research and Development, Takeda Development Center Americas, Inc, Cambridge, MA, USA; 2US Health Economics and Outcomes Research, Takeda Pharmaceuticals USA, Inc, Lexington, MA, USACorrespondence: Yanyu Wu, Global Evidence & Outcomes, Data Sciences Institute, Research and Development, Takeda Development Center Americas, Inc, 40 Landsdowne Street, Cambridge, MA, USA, Tel +1 617-955-6408, Email yanyu.wu@takeda.comBackground: US patients with hemophilia A can receive prophylaxis with extended half-life recombinant factor VIII (rFVIII) products, including efmoroctocog alfa (fragment crystallizable fusion protein) and rurioctocog alfa pegol (antihemophilic factor [recombinant], PEGylated).Objective: To evaluate dosing patterns and weekly consumption of extended half-life rFVIII products in the United States.Methods: We performed a retrospective analysis using the US Specialty Pharmacy Database (2015– 2018). Included patients had a diagnosis of hemophilia A, ≥ 2 consecutive monthly claims for efmoroctocog alfa or rurioctocog alfa pegol for prophylaxis, and weight data. Outcome measures included weekly dosing frequency and dispensed weekly dose.Results: The analysis included 774 patients (efmoroctocog alfa, 506; rurioctocog alfa pegol, 268). Mean (SD) age was 24.2 (15.8) and 26.3 (14.9) years for patients receiving efmoroctocog alfa and rurioctocog alfa pegol, respectively; mean (SD) weight was 68.4 (36.8) and 79.8 (37.7) kg, respectively. The most frequent efmoroctocog alfa regimen was twice weekly (45.7%), followed by every 4 days (20.6%), every 3 days (9.1%), and 3 times per week (7.5%). The most frequent rurioctocog alfa pegol regimen was twice weekly (72.4%), followed by 3 times per week (8.7%), every 4 days (7.6%), and every 3 days (5.5%). The proportion of efmoroctocog alfa twice-weekly dispensing records increased from 31.5% to 50.9%, and every 4 days dispensing records decreased from 31.3% to 14.5% (2015– 2018). The proportion of rurioctocog alfa pegol dispensing records remained broadly stable (2016– 2018). Overall, mean (SD; median) weekly prophylactic dose was 105.4 (77.9; 92.6) IU/kg with efmoroctocog alfa, and 96.8 (41.9; 90.9) IU/kg with rurioctocog alfa pegol.Conclusion: In this database study, the most frequently observed dosing frequency was twice weekly for patients receiving efmoroctocog alfa or rurioctocog alfa pegol. The observed mean weekly consumption was slightly higher, and variation was greater, in patients receiving efmoroctocog alfa versus rurioctocog alfa pegol.Keywords: hemophilia A, fragment crystallizable fusion protein, efmoroctocog alfa, antihemophilic factor (recombinant), PEGylated, rurioctocog alfa pegol, dosing patterns