Abstract

Management of hemophilia B requires replacement therapy with factor IX (FIX) coagulation products. Extended half-life (EHL) recombinant FIX products are now available along with existing standard half-life (SHL) FIX products. The aim is to examine factor dispensation and expenditure for Japanese patients who switched from SHL to EHL product in a real world setting. Japanese real world data provided by Medical Data Vision Co., Ltd (MDV) were used to compute number of IUs dispensed, direct product and total health care expenditure (¥)among individuals with data for ≥3 months before and after switching from an SHL (nonacog alfa, nonacog gamma, freeze-dried human blood-coagulation factor IX) to EHL (eftrenonacog alfa) product (April 2010–November 2017). MDV is a commercially available large scale real-world hospital medical administrative and claims database in Japan, that includes claims data from hospitals, regardless of insurance types, from >10 million inpatients and outpatients. We report medians for IUs and expenditures to accommodate for skewness of data distribution. Seventeen patients switched from an SHL to EHL product and had ≥3 months of data pre and post switch. Median quarterly IUs dispensed increased from 29,600 IUs on SHL to 36,750 IUs on EHL when considering available data on the 4 quarters pre and post switch. Median FIX expenditures increased 398% from ¥1,522,368 to ¥7,589,255. FIX replacement expenditures represented 96% of total expenditures pre and 99% post switch. Similar patterns held when analyses were limited to 7 individuals who switched from nonacog alfa (Medians= 18,000 IUs; ¥1,896,510) to eftrenonacog alfa (Medians=36,000 IUs; ¥7,480,296) (294% increase in expenditures). This analysis shows that switching from an SHL to EHL product increased FIX replacement IUs dispensed. Expenditures on FIX replacement products increased post switch by 294% - 398%. Further analyses in larger patient populations should be explored.

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