Abstract

Management of Hemophilia B requires intravenous factor IX (FIX) infusions to replenish missing coagulation factor. The introduction of extended half-life (EHL) replacement products in the US, with longer half-lives compared to standard half-life (SHL) product enabled comparison of expenditures and dispensation of clotting factor concentrates for the treatment of Hemophilia B with two EHL products versus an SHL product. De-identified claims from a large national specialty pharmacy claims database were used to identify male patients with severe or moderate hemophilia B who received FIX replacement from Apr 2015 (month first EHL FIX dispensed) to Sep 2017 and had data for at least 1 month of dispensation. SHL vs EHL groups were compared. Key outcome measures were direct expenditures and factor IUs dispensed. Expenditures and IUs dispensed were measured over monthly increments. Descriptive statistics were used to analyze results. Medians for expenditures and IUs were used to accommodate for the skewness of data distribution. 386 Hemophilia B patients met the inclusion criteria and were included in the analysis. The SHL group comprised of 293 nonacog alfa patients and the EHL group included 107 eftrenonacog alfa patients and 45 albutrepenonacog alfa patients. The albutrepenonacog group had a proportionately higher number of pediatric patients. The median FIX product dispensation per patient per calendar month was 16,134 IU (IQR, 26,903 IU) (nonacog) versus 16,380 IU (IQR, 17,287 IU) (eftrenonacog) and 10,039 IU (IQR, 10,620 IU) (albutrepenonacog). Median expenditures per patient per calendar month were higher for EHLs (eftrenonacog $48,336; IQR, $49,970; and albutrepenonacog $42,664; IQR, $45,135) than for SHL ($21,849; IQR, $36,102). This real-world data analysis, unadjusted for treatment regimen, showed higher expenditure among U.S. moderate/severe hemophilia B patients who used EHL, compared to SHL FIX. Further analyses, incorporating essential clinical characteristics and drug regimen, should be explored.

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