Societal costs of primary progressive multiple sclerosis in Australia and the economic impact of a hypothetical disease-modifying treatment that could delay disease progression.

AIMS: Primary progressive multiple sclerosis (PPMS) has a progressive course of disability with continuous neurological worsening. We investigated societal costs of PPMS in Australia and the economic impact of increasing the independence of people with PPMS through delaying disease progression. METHODS: This prevalence-based retrospective cost-of-illness analysis used observational data from publicly available secondary data sources and literature findings. Direct and indirect costs of PPMS were considered. A replica estimated population was created using the National Centre for Social and Economic Modelling (NATSEM) microsimulation model of the Australian tax and transfer system (STINMOD+). Using a budget impact analysis approach, we modelled the effect on PPMS costs of an effective hypothetical disease-modifying treatment (DMT) that delays disease progression by a year from mild to moderate and a further year from moderate to severe PPMS. RESULTS: An estimated 31,650 Australians have multiple sclerosis (MS) including 4,430 with PPMS. The proportion with PPMS was estimated to increase with age and disease severity. Overall 25% of males with MS, and 10% of females, were estimated to have PPMS. Societal cost of PPMS in Australia in 2018 was estimated at AU$418.1 million. Indirect costs contributed 67.5% of total costs, attributable to reduced workforce participation and need for informal care. The modelled DMT was estimated to create savings of AU$14.9 million (3.6%). Fewer people had moderate and severe PPMS resulting in major cost savings, partially offset by increased costs of treatment, care and support for a relative increase in the number of people with mild PPMS and their increased productivity losses. LIMITATIONS: Publicly available data may be incomplete. The potential cost of the DMT was not considered. CONCLUSIONS: The economic burden of PPMS was estimated at AU$418 million in 2018. An effective DMT that delayed progression from disease severity states by one year could provide significant cost savings.

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).

Rising midlife obesity will worsen future prevalence of dementia.

BACKGROUND: Midlife body weight status has been found to affect late life dementia outcomes. A cohort projections model was developed to assess the impact of midlife body mass index (BMI) profile on dementia in older Australians. METHODS: A baseline projection using age-sex specific dementia prevalence rates was constructed and the results of scenarios that took account of midlife BMI were compared with those from population ageing only. RESULTS: This modelling predicts that if the rising trend in midlife obesity and declining trend in midlife normal weight in Australia are to be taken into account in projecting future numbers of Australians with dementia then the number of people aged 65 or more years with dementia, by 2050, would be 14% higher than that expected from demographic ageing only. If midlife obesity prevalence was decreased to 20% and normal weight increased to 40% over the period of 2015-2025, then dementia cases among persons aged 65-69 years would be lower by about 10% in 2050 compared with the "doing nothing to stop current trends in obesity" projection. CONCLUSION: The rising tide of obesity in Australian adults will increase the dementia epidemic expected in future years.

Australian adults use complementary and alternative medicine in the treatment of chronic illness: a national study.

OBJECTIVES: The objectives of this study were to identify the prevalence of the use of vitamin/mineral supplements or natural/herbal remedies, concurrent use of pharmaceutical medication, and to profile those most likely to use these complementary and alternative medicines (CAM) in the treatment of five chronic conditions identified as national health priorities (asthma, diabetes, arthritis, osteoporosis, heart or circulatory condition) within the Australian adult population. METHODS: Analysis of the Australian National Health Survey database, 2004-05. RESULTS: Approximately 24% (1.3 million) of Australian adults with a chronic condition regularly applied CAM to treatment. CAM was most often used exclusively or in combination with pharmaceutical medicine in the treatment of arthritis and osteoporosis. Fewer than 10% of adults with asthma, diabetes or a heart or circulatory condition used CAM, most preferring pharmaceutical medicine. Regular CAM users were more likely to be aged ≥60, female, have a secondary school education and live in households with lower incomes than non-users. Non-users were more likely to be 30-59 years old and tertiary educated. CONCLUSION AND IMPLICATIONS: Arthritis, osteoporosis and, to a lesser extent, heart or circulatory conditions are illnesses for which doctors should advise, and patients need to be most aware about the full effects of CAM and possible interactive effects with prescribed medicine. They are also conditions for which research into the interactive effects of CAM and pharmaceutical medication would seem of most immediate benefit.