Cost‐utility analysis of emicizumab for the treatment of severe hemophilia A patients in Canada

Abstract

Introduction: EHL FVIII products and emicizumab provide clinicians with other prophylactic options for treating hemophilia A, however, it is unclear if emicizumab is a cost-saving option. The objective of this study is to estimate the health and economic effects of using prophylactic EHL FVIII, SHL FVIII, and emicizumab in severe haemophilia A patients. Materials and Methods:A state-transition Markov model evaluated the costeffectiveness of prophylactic SHL FVIII, EHL FVIII, and emicizumab in a cohort of 2-year-old male patients over a lifetime horizon in the form of a cost-utility analysis using a Canadian provincial ministry of health payer perspective. The transition probabilities, costs, and utilities were obtained from literature and the Canadian Bleeding Disorders Registry. Probabilistic sensitivity and scenario analyses were performed to test the robustness of the model. Results:The base-case analysis, over a lifetime horizon, resulted in a total cost and utilities per person for SHL FVIII, EHL FVIII, and emicizumab of $27.2 million (M), $36. 7 M, and $26.2 M, respectively, and 31.30, 31.16, and 31.61 quality-adjusted life years, respectively. Emicizumab treatment resulted in 29 and 16 less bleeds in a lifetime compared to SHL FVIII and EHL FVIII, respectively. Probabilistic sensitivity analysis showed that emicizumab was cost-saving 100% of the time compared to SHL FVIII and EHL FVIII. Conclusion:The cost-utility analysis showed that emicizumab is more effective and may be less costly than FVIII for Canadian haemophilia A patients, conditional on drug cost assumptions. Our model indicates that emicizumab may be a potentially favourable treatment option for minimising healthcare costs and providing higher effectiveness.

Can German Health Insurance Claims Data Fill Information Gaps in Rare Chronic Diseases: Use Case of Haemophilia A

“…9 In Germany, the economic burden of direct costs for severe HA is estimated to be 79 times higher than the mean per capita health expenditure. 10 The increasing costs for innovative therapies such as bispecific monoclonal antibodies or gene therapies 11,12 raise questions about the value of innovative treatments compared to standard treatments for decisions on health care resource allocation. Basic updated information for treatment patterns, outcomes, costs of HA, and HA-specific comorbidities stratified by age and severity is a necessary basis for value assessment of innovative therapies.…”

Cost‐effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States

“…In a study by Yu and colleagues, emicizumab prophylaxis was found to be cost-saving in Canada among patients with severe hemophilia A, dominating the standard of care prophylaxis strategy. 7 In reports by the Institute for Clinical and Economic Review, emicizumab prophylaxis was found to be cost-saving in the United States among patients with severe hemophilia A without inhibitors as well as among patients with hemophilia A with inhibitors. 8,9 It is worth noting that the baseline prevalence of bleeding events and other hemophilia A-related complications is known to be less in the moderate or mild population than in the severe population.…”