COMPARISON OF REAL-WORLD DOSE AND CONSUMPTION OF TWO EXTENDED HALF-LIFE RECOMBINANT FACTOR VIII PRODUCTS FOR THE TREATMENT OF HEMOPHILIA A IN THE UNITED STATES
Author(s)
Wu Y, Sun S, Fan T
Shire US Inc., a Takeda company, Cambridge, MA, USA
Presentation Documents
OBJECTIVES: US patients with hemophilia A can receive prophylaxis with extended half-life (EHL) recombinant factor VIII (rFVIII) products, including antihemophilic factor (recombinant), Fc fusion protein (rFVIII-Fc; Bioverativ Therapeutics Inc., Waltham, MA) and antihemophilic factor (recombinant), PEGylated, rurioctocog alfa pegol (TAK-660, BAX 855; Baxalta US Inc., a Takeda company, Lexington, MA, USA). This retrospective analysis of US specialty pharmacy data examined dosing patterns. METHODS: Patients with hemophilia A and ≥1 rFVIII dispensing record for prophylaxis (January 2015–December 2018) were identified from US specialty pharmacy data. Dosing patterns, including distribution of dosing frequencies and dispensed weekly dose, were assessed in patients with weight information and ≥2 consecutive monthly rFVIII prophylaxis dispensing records. RESULTS: This analysis included 774 patients (506 receiving rFVIII-Fc, 268 receiving TAK-660); respective mean (SD): age, 24.2 (15.8) and 26.3 (14.9) years; weight, 68.4 (36.8) and 79.8 (37.7) kg. The most frequent rFVIII-Fc regimens dispensed were twice weekly (2x/wk; 45.7%), followed by every 4 days (q4d; 20.6%), every 3 days (q3d; 9.1%), and 3 times per week (3x/wk; 7.5%). The most frequent TAK-660 regimens were 2x/wk (72.4%), followed by 3x/wk (8.7%), q4d (7.6%), and q3d (5.5%). During 2015–2018 (after US approval in June 2014), the proportion of rFVIII-Fc 2x/wk dispensing records increased from 31.5% to 50.9% and q4d dispensing records decreased from 31.3% to 14.5%. During 2016–2018 (after US approval in November 2015), the proportion of TAK-660 dispensing records were relatively stable (2x/wk, 67.0% and 72.2%; q4d, 10.7% and 7.9%). Mean (SD) weekly prophylactic dose was 105.4 (77.9; median 92.6) IU/kg with rFVIII-Fc, and 96.8 (41.9; median 90.9) IU/kg with TAK-660. CONCLUSIONS: This retrospective database analysis found that mean weekly consumption and its variability were higher in patients receiving rFVIII-Fc versus TAK-660. These findings may help payers better understand cost implications for EHL rFVIII therapies.
Conference/Value in Health Info
2019-11, ISPOR Europe 2019, Copenhagen, Denmark
Code
PRO158
Topic
Real World Data & Information Systems
Topic Subcategory
Health & Insurance Records Systems
Disease
Rare and Orphan Diseases, Systemic Disorders/Conditions