PRO38 LONG-TERM Outcomes of Previously-Treated Adult and Adolescent Patients with Severe Haemophilia a Receiving Prophylaxis with Extended Half-Life FVIII Treatments: An Economic Analysis from a UK Perspective

Abstract

Standard of care for patients with haemophilia A is prophylaxis with factor VIII (FVIII) therapies.1,2 Extended half-life (EHL) FVIII products offer reduced infusion burden compared with standard FVIII treatments.3,4 However, comparative evidence between EHLs is lacking. The objective of this study was to develop a pharmacodynamic–pharmacokinetic decision model to predict comparative bleed outcomes of adults and adolescents with severe haemophilia A receiving treatment with EHL FVIIIs, capturing differences in cumulative bleeding episodes and breakthrough bleed resolution and resource costs. Clinical data from the Pathfinder 2 Phase III trial was used to understand the link between FVIII levels and annual bleeding rates (ABRs). This was combined with modelled FVIII levels for four EHL FVIIIs (turoctocog alfa pegol, rurioctocog alfa pegol, efmoroctocog alfa, damoctocog alfa pegol) to estimate comparative ABRs over a lifetime horizon. Breakthrough bleed resolution and resource costs were estimated from a UK NHS perspective. Turoctocog alfa pegol prophylaxis resulted in 8–19% fewer cumulative bleeding episodes versus comparators in the base case scenario. Assuming parity in annual prophylaxis costs, this treatment reduced breakthrough bleed resolution and resource costs by 12–32% versus comparators. Using a pharmacodynamic–pharmacokinetic model, turoctocog alfa pegol prophylaxis was associated with fewer cumulative bleeds and lower breakthrough bleed resolution and resource costs versus comparators. [1]Aledort L, et al. Blood Transfus. 2019; 17(6):479–86. [2]Srivastava A, et al. Haemophilia. 2013;19(1):e1-47. [3]Kumar R, et al. Semin Thromb Hemost. 2016;42(1):18-29. [4]Shah A, et al. Haemophilia. 2018;24(5):733-40.