Treatment preferences in people with haemophilia A or caregivers of people with haemophilia A: A discrete choice experiment.

INTRODUCTION: The standard of care for people with haemophilia A (PwHA) is regular prophylactic therapy with Factor VIII. However, very little is known about the treatment preferences of PwHA with and without inhibitors, or their caregivers, when presented with the choice between intravenous and subcutaneous prophylactic treatments with different modes of action, side effect risk and frequency of administration. METHODS: We conducted a discrete choice experiment to assess the preferences of PwHA or their caregivers. The survey was conducted in Australia, New Zealand, Canada, France, Italy and the United Kingdom. RESULTS: There were 56 respondents. The majority of PwHA had hereditary HA (78%) rather than spontaneous HA (22%). Most PwHA were diagnosed when they were under 12 months old (65%). The model identified two segments based on treatment preferences. There is heterogeneity between PwHA in their treatment preferences. People with and without inhibitors prefer to receive treatment via a subcutaneous injection compared to an intravenous infusion. However, for PwHA, efficacy is key; they value a reduction in the annual bleed rate. For those without inhibitors, this also includes a reduction in the risk of developing inhibitors. The side effect risk, administration risk and storage requirements were prioritised differently in each segment. CONCLUSIONS: Results from this study may inform decisions about the value of existing and new treatments for PwHA.

The societal burden of haemophilia A. I - A snapshot of haemophilia A in Australia and beyond.

INTRODUCTION: Few studies, both in Australia and overseas, have examined the social impacts of living with haemophilia A (HA) or the economic costs associated with the disorder. The purpose of this paper is to examine the epidemiology and societal burden of people with HA (PwHA) in Australia, with a particular focus on men with this disorder. METHODS: The epidemiology and societal burden of HA in Australia, with a particular focus on men with this disorder, were assessed, using data available in the Australian and international literature and publicly available data. RESULTS: The mean annual prevalence of HA is approximately 1-2 per 10 000 males. Prophylactic treatment is used in one-quarter (25.1%) of people with moderate HA, and 82.2% of people with severe HA. Within the latter group, 16.1% have inhibitors for Factor VIII, predisposing them to worse morbidity, mortality and quality of life when compared to the non-inhibitor population. Joint pain and joint disease occur commonly in PwHA, with up to 70% of adults with HA experiencing joint problems. HA is associated with poor physical health, and PwHA miss school and work due to bleeding-related events. CONCLUSION: HA is associated with substantial economic burden; with large differences in costs reported between countries. Overall, HA imposes a significant burden of disease on PwHA, their families and the community at large.

The societal burden of haemophilia A. II - The cost of moderate and severe haemophilia A in Australia.

INTRODUCTION: Although the costs for people with haemophilia A (PwHA) in Europe and the United States have been well characterized, to date, there are no cost estimates for PwHA in Australia. The purpose of this study was to estimate direct and indirect costs of moderate and severe haemophilia A (HA) in Australia under current treatment practices. METHODS: The number of Australian males with moderate or severe HA was projected from Australian Bleeding Disorders Registry (ABDR) data. We estimated the prevalence in 2018 of adults with moderate HA to be 159 people, severe to be 416; and 68 and 283, respectively, in the paediatric (aged < 18 years) population. We used a 'bottom-up prevalence based cost of illness approach' to estimate costs; that is, we estimated the per capita cost for different groups of PwHA; for example, by age and disease severity, and these per capita costs were scaled up to the estimated population with HA. Costs were estimated based on publicly available secondary data and literature review. RESULTS: The treatment-related costs, direct and indirect costs, of moderate to severe HA are significant, totalling over AUD$111M in 2018, equating to a yearly per patient cost of approximately AUD$120 000 (equivalent to ~EUR€74 000 or ~USD$85 000). CONCLUSION: Although HA affects a relatively small number of people within the Australian population, it is associated with high aggregate costs and imposes a high economic burden.

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

INTRODUCTION: Emicizumab 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. METHODS: We 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. RESULTS: Overall, 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. CONCLUSION: The 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).