Real‐world cost estimates of initiating emicizumab in US patients with haemophilia A

Abstract

Introduction: Emicizumab is the first approved non-factor therapy for haemophilia A.It provides superior prophylactic bleeding control compared to other products in both patients with and patients without inhibitors. However, there is no real-world data about the monetary consequences of starting emicizumab.Aim: To examine the estimated costs of starting emicizumab in a cohort of real-world haemophilia A patients with and without inhibitors. Methods:The cost of haemostatic therapy for 6 months before and after initiating emicizumab for participants in a multicentre observational study was calculated based on the type of product and dosing that was used for prophylaxis and treating breakthrough bleeds, the number of treated bleeds and the participant weight.

Emicizumab state‐of‐the‐art update

“…The clinical correlates of emicizumab's PK and pharmacodynamic(PD) profile are demonstrated in the HAVEN study program results 12 and several subsequent real-world evidence data sets. [15][16][17][18]28,[43][44][45] Given at a fixed dose of 1.5 mg/kg weekly (or equivalent dosing for longer time intervals), emicizumab provides robust and consistent protection from bleeding. Additionally, standard clotting assays available to most clinical laboratories are unsuitable for measuring emicizumab or its effect and cannot be used for dose adjustments.…”

“…The clinical correlates of emicizumab's PK and pharmacodynamic(PD) profile are demonstrated in the HAVEN study program results 12 and several subsequent real‐world evidence data sets 15–18,28,43–45 . Given at a fixed dose of 1.5 mg/kg weekly (or equivalent dosing for longer time intervals), emicizumab provides robust and consistent protection from bleeding.…”

Impact of novel hemophilia therapies around the world

“…According to Hermans and Makris, 75 25% to 35% of people with hemophilia A without inhibitors are under prophylaxis with emicizumab in Israel, the United Kingdom, and Belgium. In a real‐world cost estimate in the United States, emicizumab seems to be economically favorable compared to other agents for prophylaxis in people with or without inhibitors 76 …”

A new harmony for hemorrhagic disorders: The Dutch SYMPHONY consortium

“…11,12 This streamlined dosing likely contributed to the reduced estimated cost of prophylaxis when patients switched to emicizumab in the United States. 13 However, with accumulating clinical experience, the question has recently been raised if there is a role for personalizing emicizumab dosing. 12 Indeed, a recent abstract reported marked clinical benefit of low-dose emicizumab (<25% recommended dose) in a resource-scarce setting.…”