Cost Comparison of Efanesoctocog Alfa with Existing Factor VIII Replacement Therapies for Major Surgeries in People with Severe Hemophilia a

Janice Staber; Alix Arnaud; Ion Agirrezabal; Lane Anson; Andrew Wilson; Nana Kragh; Doris V. Quon; Allison P Wheeler

doi:10.1182/blood-2024-207568

Introduction People with severe hemophilia A (HA) are susceptible to excessive bleeds during surgery and insufficient hemostatic control can lead to increased morbidity and mortality. Adequate factor VIII (FVIII) replacement therapy is required pre- and post-operatively to maintain hemostasis, prevent bleeds and facilitate healing. The World Federation of Hemophilia guidelines recommend a pre-operative FVIII level of 80%-100%, and a post-operative FVIII level of 60%-80% on days 1-3, 40%-60% on days 4-6, 30%-50% on days 7-14 for major surgeries in patients with severe HA. Perioperative care with standard half-life (SHL) and extended half-life (EHL) therapies usually require frequent injections to achieve the target FVIII levels, leading to high costs of surgery in patients with severe HA. Efanesoctocog alfa is a first-in-class high-sustained FVIII replacement therapy, designed to prolong half-life by decoupling FVIII from endogenous von Willebrand factor. Objective To estimate total costs associated with perioperative hemostatic management in patients with severe HA treated with SHL (octocog alfa), EHL (rurioctocog alfa pegol and efmoroctocog alfa), and high-sustained (efanesoctocog alfa) FVIII replacement therapies. Methods Data on dose, total factor consumption, frequency of administration, pre- and post-operative target FVIII level (IU/dL) associated with major surgeries for octocog alfa, rurioctocog alfa pegol, efmoroctocog alfa, and efanesoctocog alfa were collected from United States (US) prescribing information (PI) and phase 3 clinical trials included in PI. The 2024 wholesale acquisition costs (WAC) were used for calculating factor costs. Typically, to achieve the pre-operative target FVIII activity, octocog alfa, rurioctocog alfa pegol, and efmoroctocog alfa are administered every 8-24 hours to prevent bleeding and intermittently for at least another 7 days or until the wound has healed sufficiently. For major surgeries with efanesoctocog alfa, a pre-operative loading dose of 50 IU/kg was administered, followed by 30 or 50 IU/kg every 2 to 3 days, as needed. The total cost was estimated by multiplying the total factor consumed (IU/kg) during the reported perioperative period (7 days for rurioctocog alfa pegol, 14 days for efmoroctocog alfa and efanesoctocog alfa) by the average US adult male weight (assumed as 91 kg) and the cost per IU of SHL, EHL, and high-sustained FVIII replacement therapies used for major surgeries. The difference in total factor consumption and total costs between the therapies were then compared. Results Octocog alfa data (NCT00157053) included 58 patients (≥5 years old) with severe HA who underwent 65 surgical procedures (22 major surgeries). Rurioctocog alfa pegol data (NCT01913405) included 21 previously treated male patients (≥12-75 years old) with severe HA who underwent 21 major surgeries. Efmoroctocog alfa data (NCT01454739) included 21 patients (≥12 years old) with severe HA who underwent 23 major surgeries. Efanesoctocog alfa data (NCT04161495) included 12 patients (≥12 years old) with severe HA who underwent 13 major surgeries. The median (interquartile range) total factor consumption (IU/kg) per major surgery during the reported perioperative period for octocog alfa, rurioctocog alfa pegol, efmoroctocog alfa, and efanesoctocog alfa were 910 (228-1825), 629 (464-1457), 493 (121-733), and 163 (45-361), respectively. The total factor cost during the reported perioperative period for major surgery for octocog alfa, rurioctocog alfa pegol, efmoroctocog alfa, and efanesoctocog alfa were $157,339, $142,525, $115,747, and $78,165, respectively. The total factor consumption and cost savings demonstrated by efanesoctocog alfa were compared with SHL and EHL FVIII replacement therapies: 5.5 times fewer IU of factor and $79,174 savings vs octocog alfa, 3.9 times fewer IU of factor and $64,360 savings vs rurioctocog alfa pegol, and 3 times fewer IU of factor and $37,581 savings vs efmoroctocog alfa. Conclusion Despite the higher per IU WAC, perioperative management with efanesoctocog alfa was estimated to be less costly than SHL and EHL therapies. This is attributed to its high-sustained factor activity and reduced factor consumption during the reported perioperative period. The major limitations of the study were: the types of major surgeries varied among studies; the perioperative period data of octocog alfa were not found. Staber:Bayer: Membership on an entity's Board of Directors or advisory committees; BioMarin: Membership on an entity's Board of Directors or advisory committees; Sanofi: Membership on an entity's Board of Directors or advisory committees. Arnaud:Sanofi: Current Employment, Current holder of stock options in a privately-held company. Agirrezabal:Sanofi: Current Employment, Current holder of stock options in a privately-held company. Anson:Sanofi: Current Employment, Current holder of stock options in a privately-held company. Wilson:Sanofi: Current Employment, Current holder of stock options in a privately-held company. Kragh:Sobi: Current Employment, Current holder of stock options in a privately-held company. Quon:Bayer: Honoraria, Other: Consulting /Advisory Boards; Sanofi: Honoraria, Other: Consulting /Advisory Boards, Speakers Bureau; CSL Behring: Honoraria, Other: CSL Behring, Speakers Bureau; Hemabiologics/LFB: Honoraria, Other: Consulting /Advisory Boards, Speakers Bureau; Genentech/Roche: Honoraria, Other: Consulting /Advisory Boards; Novo Nordisk: Honoraria, Other: Consulting /Advisory Boards, Speakers Bureau; Pfizer: Honoraria, Other: Consulting /Advisory Boards, Speakers Bureau; Takeda: Honoraria, Other: Consulting /Advisory Boards. Wheeler:Novo Nordisk: Consultancy, Honoraria; Octapharma: Honoraria, Research Funding; CSL Behring: Honoraria; Bayer: Honoraria; HEMA Biologics: Honoraria; Genentech: Honoraria; Pfizer Inc: Honoraria; Sanofi-Aventis: Honoraria; Takeda Pharmaceuticals: Honoraria; UniQure: Honoraria; Vega Therapeutics, Inc.: Honoraria.

Cost Comparison of Efanesoctocog Alfa with Existing Factor VIII Replacement Therapies for Major Surgeries in People with Severe Hemophilia a

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