The societal burden of haemophilia A. III – The potential impact of emicizumab on costs of haemophilia A in Australia

Abstract

IntroductionEmicizumab is a humanized monoclonal modified IgG4 antibody with bispecific antibody structure bridging Factor IXa and Factor X. Emicizumab has demonstrated efficacy and safety in adults, adolescents and paediatrics with HA, with or without inhibitors to Factor VIII (FVIII). There is currently no evidence that reports on the potential impact of the introduction of emicizumab on the societal costs of haemophilia A (HA). The purpose of this study was to explore the cost impact associated with the introduction of emicizumab on the current societal costs of people with HA (PwHA) in Australia.MethodsWe conducted an analysis of the impact of emicizumab on societal costs, based on changes in the direct and indirect costs incurred by PwHA. Potential impacts of emicizumab on outcomes in PwHA were modelled based on HAVEN 1, HAVEN 2 and HAVEN 3 studies. We assumed that eligible PwHA commenced use of emicizumab on 1 January 2018. The impact of emicizumab on costs of HA in Australia males was then estimated for the 12‐month period to 31 December 2018.ResultsOverall, uptake of emicizumab in its first year of use reduces annual costs associated with moderate/severe HA by AUD$69.197M (62.3%). This reflects 64.2% reduction in the cost of FVIII blood products and 92% reduction in cost of bypassing agents.ConclusionThe cost of emicizumab is likely to offset some or all of the projected reductions in treatment costs. However, we also found 30.7% reduction in non‐treatment direct costs (AUD$3.771M) and 19.1% reduction in indirect costs (AUD$2.732M).

Economic Evaluation of Immune Tolerance Induction in Children With Severe Hemophilia A and High-Responding Inhibitors: A Cost-Effectiveness Analysis of Prophylaxis With Emicizumab

“…In a non-ITI setting, pharmacoeconomic models have recently shown that prophylaxis with emicizumab for PsHAhri is associated with reduced costs of treatment of HA compared to prophylaxis with BpA. [21][22][23] An Australian study evaluated the impact of emicizumab on societal costs, based on changes in the direct and indirect costs incurred by people with moderate or severe HA. 22 The first year of emicizumab reduced annual costs associated with BpA by 92.0%.…”

“…[21][22][23] An Australian study evaluated the impact of emicizumab on societal costs, based on changes in the direct and indirect costs incurred by people with moderate or severe HA. 22 The first year of emicizumab reduced annual costs associated with BpA by 92.0%. 22 Compared to prophylaxis with BpA, the overall budget reduction of prophylaxis with emicizumab for people with HA and inhibitors who failed ITI was €45.4 million in a 3-year horizon (PPP US$56.8 million, calculated as mentioned before), according to an Italian study.…”

“…22 The first year of emicizumab reduced annual costs associated with BpA by 92.0%. 22 Compared to prophylaxis with BpA, the overall budget reduction of prophylaxis with emicizumab for people with HA and inhibitors who failed ITI was €45.4 million in a 3-year horizon (PPP US$56.8 million, calculated as mentioned before), according to an Italian study. 21 This same study showed that prophylaxis with emicizumab was cost-saving, compared to prophylaxis with BpA.…”

“…19,20 In a non-ITI setting, pharmacoeconomic models have recently shown that prophylaxis with emicizumab for PsHAhri is more cost-effective than prophylaxis with BpA. [21][22][23] However, we believe there has been no study to date that has evaluated the cost-effectiveness of the association between ITI with prophylaxis with emicizumab. Since there is a growing concern regarding ITI for PsHAhri with respect to its costs and effectiveness, there is an urgent need to estimate the impact of introducing emicizumab as a prophylactic agent during ITI.…”