Cost of Emicizumab
The cost of emicizumab varies globally and can be considerable in some countries, representing a significant implementation barrier despite its clinical benefits. 1
Global Cost Variability
The International Society on Thrombosis and Haemostasis (ISTH) 2024 guidelines explicitly acknowledge that emicizumab costs differ substantially across healthcare systems and may not be affordable or even available in all countries. 1 This geographic variability in pricing and access is a major consideration when implementing prophylaxis recommendations for hemophilia A patients with inhibitors.
Cost-Effectiveness Evidence
Despite the high acquisition cost, multiple economic analyses demonstrate that emicizumab may be cost-saving compared to alternative treatments:
Comparison with Bypassing Agents
Emicizumab may be both more effective and less costly than bypassing agents to prevent bleeding events in patients with inhibitors. 1
In France, emicizumab was dominant (less costly and more effective) compared to bypassing agents, saving €234,191 while gaining 0.88 quality-adjusted life years (QALYs). 2
In Korea, lifetime emicizumab prophylaxis prevented 807 bleedings, extended 3.04 QALYs, and reduced costs by $2.6 million USD compared to bypassing agent on-demand treatment. 3
In Iran, emicizumab prophylaxis was dominant in all age groups with annual bleeding rates ≥18, with considerable cost savings. 4
Comparison with Factor VIII Concentrates
In Canada, emicizumab resulted in total lifetime costs of $26.2 million compared to $27.2 million for standard half-life FVIII and $36.7 million for extended half-life FVIII, while providing superior effectiveness (31.61 QALYs vs 31.30 and 31.16 QALYs respectively). 5
Probabilistic sensitivity analysis showed emicizumab was cost-saving 100% of the time compared to both FVIII options. 5
Clinical Context for Cost Considerations
The ISTH guidelines emphasize that shared decision-making should consider availability of products, costs, and patient preference when choosing between emicizumab and alternative therapies. 1 This is particularly relevant because:
Hemophilia care is costly, with factor replacement therapy accounting for approximately 80% of overall hemophilia care costs. 1
The large costs involved in prophylaxis for severe hemophilia A with inhibitors—particularly with bypassing agents—create substantial implementation challenges. 1
Low- and middle-income countries may not be able to afford standard prophylaxis options, making cost a critical barrier to optimal care. 1
Important Caveats
Emicizumab may not be available or registered in some countries, limiting its use regardless of cost-effectiveness. 1 Additionally, while economic analyses consistently show favorable cost-effectiveness ratios, these studies have limitations including: