Lipid-Lowering Strategies for Primary Prevention of Coronary Heart Disease in the UK: A Cost-Effectiveness Analysis.

AIM: We aimed to assess the cost effectiveness of four different lipid-lowering strategies for primary prevention of coronary heart disease initiated at ages 30, 40, 50, and 60 years from the UK National Health Service perspective. METHODS: We developed a microsimulation model comparing the initiation of a lipid-lowering strategy to current standard of care (control). We included 458,692 participants of the UK Biobank study. The four lipid-lowering strategies were: (1) low/moderate-intensity statins; (2) high-intensity statins; (3) low/moderate-intensity statins and ezetimibe; and (4) inclisiran. The main outcome was the incremental cost-effectiveness ratio for each lipid-lowering strategy compared to the control, with 3.5% annual discounting using 2021 GBP (£); incremental cost-effectiveness ratios were compared to the UK willingness-to-pay threshold of £20,000-£30,000 per quality-adjusted life-year. RESULTS: The most effective intervention, low/moderate-intensity statins and ezetimibe, was projected to lead to a gain in quality-adjusted life-years of 0.067 per person initiated at 30 and 0.026 at age 60 years. Initiating therapy at 40 years of age was the most cost effective for all lipid-lowering strategies, with incremental cost-effectiveness ratios of £2553 (95% uncertainty interval: 1270, 3969), £4511 (3138, 6401), £11,107 (8655, 14,508), and £1,406,296 (1,121,775, 1,796,281) per quality-adjusted life-year gained for strategies 1-4, respectively. Incremental cost-effectiveness ratios were lower for male individuals (vs female individuals) and for people with higher (vs lower) low-density lipoprotein-cholesterol. For example, low/moderate-intensity statin use initiated from age 40 years had an incremental cost-effectiveness ratio of £5891 (3822, 9348), £2174 (772, 4216), and was dominant (i.e. cost saving; -2,760, 350) in female individuals with a low-density lipoprotein-cholesterol of ≥ 3.0, ≥ 4.0 and ≥ 5.0 mmol/L, respectively. Inclisiran was not cost effective in any sub-group at its current price. CONCLUSIONS: Low-density lipoprotein-cholesterol lowering from early ages is a more cost-effective strategy than late intervention and cost effectiveness increased with the increasing lifetime risk of coronary heart disease.

Societal health and economic burden of cardiovascular diseases in the population with type 2 diabetes in Qatar. A 10-year forecasting model.

AIMS: To predict the future health and economic burden of cardiovascular disease (CVD) in type 2 diabetes (T2D) in Qatar. MATERIALS AND METHODS: A dynamic multistate model was designed to simulate the progression of fatal and non-fatal CVD events among people with T2D in Qatar aged 40-79 years. First CVD events [i.e. myocardial infarction (MI) and stroke] were calculated via the 2013 Pooled Cohort Equation, while recurrent CVD events were sourced from the REACH registry. Key model outcomes were fatal and non-fatal MI and stroke, years of life lived, quality-adjusted life years, total direct medical costs and total productivity loss costs. Utility and cost model inputs were drawn from published sources. The model adopted a Qatari societal perspective. Sensitivity analyses were performed to test the robustness of estimates. RESULTS: Over 10 years among people with T2D, model estimates 108 195 [95% uncertainty interval (UI) 104 249-112 172] non-fatal MIs, 62 366 (95% UI 60 283-65 520) non-fatal strokes and 14 612 (95% UI 14 472-14 744) CVD deaths. The T2D population accrued 4 786 605 (95% UI 4 743 454, 4 858 705) total years of life lived and 3 781 833 (95% UI 3 724 718-3 830 669) total quality-adjusted life years. Direct costs accounted for 57.85% of the total costs, with a projection of QAR41.60 billion (US$11.40 billion) [95% UI 7.53-147.40 billion (US$2.06-40.38 billion)], while the total indirect costs were expected to exceed QAR30.31 billion (US$8.30 billion) [95% UI 1.07-162.60 billion (US$292.05 million-44.55 billion)]. CONCLUSIONS: The findings suggest a significant economic and health burden of CVD among people with T2D in Qatar and highlight the need for more enhanced preventive strategies targeting this population group.

Estimating the cost-effectiveness and return on investment of the Victorian Cardiac Outcomes Registry in Australia: a minimum threshold analysis.

OBJECTIVES: We sought to establish the minimum level of clinical benefit attributable to the Victorian Cardiac Outcomes Registry (VCOR) for the registry to be cost-effective. DESIGN: A modelled cost-effectiveness study of VCOR was conducted from the Australian healthcare system and societal perspectives. SETTING: Observed deaths and costs attributed to coronary heart disease (CHD) over a 5-year period (2014-2018) were compared with deaths and costs arising from a hypothetical situation which assumed that VCOR did not exist. Data from the Australian Bureau of Statistics and published sources were used to construct a decision analytic life table model to simulate the follow-up of Victorians aged ≥25 years for 5 years, or until death. The assumed contribution of VCOR to the proportional change in CHD mortality trend observed over the study period was varied to quantify the minimum level of clinical benefits required for the registry to be cost-effective. The marginal costs of VCOR operation and years of life saved (YoLS) were estimated. PRIMARY OUTCOME MEASURES: The return on investment (ROI) ratio and the incremental cost-effectiveness ratio (ICER). RESULTS: The minimum proportional change in CHD mortality attributed to VCOR required for the registry to be considered cost-effective was 0.125%. Assuming this clinical benefit, a net return of $A4.30 for every dollar invested in VCOR was estimated (ROI ratio over 5 years: 4.3 (95% CI 3.6 to 5.0)). The ICER estimated for VCOR was $A49 616 (95% CI $A42 228 to $A59 608) per YoLS. Sensitivity analyses found that the model was sensitive to the time horizon assumed and the extent of registry contribution to CHD mortality trends. CONCLUSIONS: VCOR is likely cost-effective and represents a sound investment for the Victorian healthcare system. Our evaluation highlights the value of clinical quality registries in Australia.

Projecting the Health and Economic Burden of Cardiovascular Disease Among People with Type 2 Diabetes, 2022-2031.

OBJECTIVE: The aim was to project the health and economic outcomes of cardiovascular disease (CVD) among people with type 2 diabetes from Australian public healthcare and societal perspectives over the next decade. METHODS: A dynamic multistate model with yearly cycles was developed to project cardiovascular events among Australians with type 2 diabetes aged 40-89 years from 2022 to 2031. CVD risk (myocardial infarction [MI] and stroke) in the type 2 diabetes population was estimated using the 2013 pooled cohort equation, and recurrent cardiovascular event rates in the type 2 diabetes with established CVD population were obtained from the global Reduction of Atherothrombosis for Continued Health (REACH) registry. Costs and utilities were derived from published sources. Outcomes included fatal and non-fatal MI and stroke, years of life lived, quality-adjusted life years (QALYs), total healthcare costs, and total productivity losses. The annual discount rate was 5%, applied to outcomes and costs. RESULTS: Between 2022 and 2031, a total of 83,618 non-fatal MIs (95% uncertainty interval [UI] 83,170-84,053) and 58,774 non-fatal strokes (95% UI 58,458-59,013) were projected. Total years of life lived and QALYs (discounted) were projected to be 9,549,487 (95% UI 9,416,423-9,654,043) and 6,632,897 (95% UI 5,065,606-7,591,679), respectively. Total healthcare costs and total lost productivity costs (discounted) were projected to be 9.59 billion Australian dollars (AU$) (95% UI 1.90-30.45 billion) and AU$9.07 billion (95% UI 663.53 million-33.19 billion), respectively. CONCLUSIONS: CVD in people with type 2 diabetes will substantially impact the Australian healthcare system and society over the next decade. Future work to investigate different strategies to optimize the control of risk factors for the prevention and treatment of CVD in type 2 diabetes in Australia is warranted.

Estimating the impact of Angelman syndrome on parental productivity in Australia using productivity-adjusted life years.

BACKGROUND: Angelman syndrome (AS) is a rare genetic condition characterized by global developmental delay, including severe intellectual disability. The parents of persons with AS experience increased stress, anxiety, and depression. This impacts parents' career choices and productivity. OBJECTIVE: To estimate, for the first time, the total productivity lost by the parents of persons with AS over a 10-year period in Australia and the corresponding cost to society. METHODS: A cost-of-illness model with simulated follow-up over a 10-year period was developed, with 2019 as the baseline year, facilitated by a Markov chain of life tables. The prevalence of persons with AS and their parents, the productivity-adjusted life years (PALYs) lost by parents, and the cost to society were estimated. Key data were obtained from a prospective cohort of AS families, peer-reviewed literature, and publicly available sources. RESULTS: The base-case productivity burden borne by the estimated 330 living parents of the 428 prevalent persons with AS totaled AUD$45.30 million, corresponding to a loss of 38.42% of PALYs per parent. CONCLUSIONS: Caring for a child with AS has a significant impact on the productivity of affected parents, with a large associated impact on the broader Australian economy.

Number needed to treat and cost per responder of Janus kinase inhibitors approved for the treatment of moderate-to-severe rheumatoid arthritis in Japan.

OBJECTIVE: This study evaluated the effectiveness and cost-effectiveness of baricitinib, tofacitinib, and upadacitinib regimens, compared to conventional synthetic disease-modifying antirheumatic drug (csDMARD) alone, among Japanese patients with moderate-to-severe rheumatoid arthritis (RA) inadequately responsive to csDMARD, measured in terms of number needed to treat (NNT) and cost per responder (CPR). METHODS: Efficacy data were derived from two recent network meta-analyses among global and Japanese population. The cost perspective was that of the Japanese Health Service. Both NNT and CPR were based on disease activity score for 28 joints with C-reactive protein (DAS28-CRP) remission and American College of Rheumatology (ACR) 20/50/70 at 12 and 24 weeks. RESULTS: Over 12 weeks, the median NNT and the median CPR to achieve DAS28-CRP remission were 4.3 and JPY 1,799,696 [USD 16,361], respectively, for upadacitinib 15 mg + csDMARD. The equivalent results were 6.0 and JPY 2,691,684 [USD 24,470] for baricitinib 4 mg + csDMARD and 5.6 and JPY 2,507,152 [USD 22,792] for tofacitinib 5 mg + csDMARD. Similar rankings were observed at 24 weeks and for other outcomes. CONCLUSIONS: Upadacitinib 15 mg was associated with the lowest NNT and CPR among the three Janus kinase inhibitors used in treatment regimens for Japanese patients with moderate-to-severe RA inadequately responsive to csDMARD.

A Systematic Review of Cost-Effectiveness of Non-Statin Lipid-Lowering Drugs for Primary and Secondary Prevention of Cardiovascular Disease in Patients with Type 2 Diabetes Mellitus.

Although studies of nonstatin lipid-lowering therapies have demonstrated cardiovascular benefits; whether these benefits provide good value has not been evaluated in type 2 diabetes mellitus patients. A systematic review was performed to include studies on the cost-effectiveness of non-statin lipid-lowering therapies in type 2 diabetes mellitus patients with/without cardiovascular disease. Thirteen studies were included; ezetimibe (n = 8), proprotein convertase subtilisin/kexin type 9 inhibitors (n = 4), fenofibrate (n = 2), nicotinic acid (n = 1), extended-release niacin/laropiprant (n = 1), and icosapent ethyl (n = 1). Six studies considered ezetimibe + statin to be a cost-effective compared to statins monotherapy, three studies suggested that proprotein convertase subtilisin/kexin type 9inhibitors + statins were not cost-effective compared to statin + ezetimibe. Fenofibrate was a cost-effective either as monotherapy or combined with a statin compared to statin or fenofibrate monotherapy. Nicotinic acid and proprotein convertase subtilisin/kexin type 9 compared to statin monotherapy were also cost-effective. Icosapent ethyl was cost-effective compared to standard care but not using the wholesale acquisition cost.

Projecting the incidence and costs of major cardiovascular and kidney complications of type 2 diabetes with widespread SGLT2i and GLP-1 RA use: a cost-effectiveness analysis.

AIMS/HYPOTHESIS: Whether sodium-glucose co-transporter 2 inhibitors (SGLT2is) or glucagon-like peptide-1 receptor agonists (GLP-1 RAs) are cost-effective based solely on their cardiovascular and kidney benefits is unknown. We projected the health and economic outcomes due to myocardial infarction (MI), stroke, heart failure (HF) and end-stage kidney disease (ESKD) among people with type 2 diabetes, with and without CVD, under scenarios of widespread use of these drugs. METHODS: We designed a microsimulation model using real-world data that captured CVD and ESKD morbidity and mortality from 2020 to 2040. The populations and transition probabilities were derived by linking the Australian Diabetes Registry (1.1 million people with type 2 diabetes) to hospital admissions databases, the National Death Index and the ESKD Registry using data from 2010 to 2019. We modelled four interventions: increase in use of SGLT2is or GLP-1 RAs to 75% of the total population with type 2 diabetes, and increase in use of SGLT2is or GLP-1 RAs to 75% of the secondary prevention population (i.e. people with type 2 diabetes and prior CVD). All interventions were compared with current use of SGLT2is (20% of the total population) and GLP-1 RAs (5% of the total population). Outcomes of interest included quality-adjusted life years (QALYs), total costs (from the Australian public healthcare perspective) and the incremental cost-effectiveness ratio (ICER). We applied 5% annual discounting for health economic outcomes. The willingness-to-pay threshold was set at AU$28,000 per QALY gained. RESULTS: The numbers of QALYs gained from 2020 to 2040 with increased SGLT2i and GLP-1 RA use in the total population (n=1.1 million in 2020; n=1.5 million in 2040) were 176,446 and 200,932, respectively, compared with current use. Net cost differences were AU$4.2 billion for SGLT2is and AU$20.2 billion for GLP-1 RAs, and the ICERs were AU$23,717 and AU$100,705 per QALY gained, respectively. In the secondary prevention population, the ICERs were AU$8878 for SGLT2is and AU$79,742 for GLP-1 RAs. CONCLUSIONS/INTERPRETATION: At current prices, use of SGLT2is, but not GLP-1 RAs, would be cost-effective when considering only their cardiovascular and kidney disease benefits for people with type 2 diabetes.

Economic Evaluation of the Community Benefit of the Australian Pregnancy Register of Antiseizure Medications.

BACKGROUND AND OBJECTIVE: The Raoul Wallenberg Australian Pregnancy Register (APR) was established to collect, analyze, and publish data on the risks to babies exposed to antiseizure medications (ASMs) and to facilitate quality improvements in management care over time. It is one of the seveal prospective observational pregnancy registers of ASMs that has been established around the world. Although the APR and other registries have contributed to knowledge gain that has been applied to decrease adverse pregnancy outcomes, their cost-effectiveness remains unknown. Here, we aimed to evaluate the economic impacts of the APR from both societal and health care system perspectives. METHODS: Using decision analytic modeling, we estimated the effectiveness (prevention of adverse pregnancy outcomes) and costs (costs of adverse pregnancy outcomes and the register itself) of the APR over a 20-year time horizon (2000-2019). The comparator was set as the adverse pregnancy outcomes collected by the APR between 1998 and 2002 (i.e., no APR derived improvements in care). In the scenario analysis, we conservatively assumed a 2.5% and 5% contribution of the APR to the savings in health care and societal costs. Adverse pregnancy outcomes included stillbirth, birth defects, and induced abortion. All cost data were derived from published sources. Health and economic outcomes were extrapolated to the total target Australian epilepsy population. The primary outcomes of interest were the return of investment (ROI) for the APR and incremental cost-effectiveness ratio (ICER) for cost per adverse outcome avoided. RESULTS: Over the 20-year time horizon, the ROI from the APR from a societal perspective was Australian dollars (AUD) 2,250 (i.e., every dollar spent on the program resulted in a return of AUD2,250). Over this time, it was estimated that 9,609 adverse pregnancy outcomes were avoided, and health care and societal costs were reduced by AUD 191 million and AUD 9.0 billion, respectively. Hence, from a health economic point of view, the APR was dominant, providing cost saving ICERs from both perspectives. DISCUSSION: Following its inception 20+ years ago, the APR has represented excellent value for investment for Australia, being also health-saving and cost saving from a societal and a health care perspective. With the growing number of marketed ASMs, the APR is expected to continue to have a major impact in the foreseeable future.

Lost Therapeutic Benefit of Delayed Low-Density Lipoprotein Cholesterol Control in Statin-Treated Patients and Cost-Effectiveness Analysis of Lipid-Lowering Intensification.

OBJECTIVES: Attainment of low-density lipoprotein cholesterol (LDL-C) therapeutic goals in statin-treated patients remains suboptimal. We quantified the health economic impact of delayed lipid-lowering intensification from an Australian healthcare and societal perspective. METHODS: A lifetime Markov cohort model (n = 1000) estimating the impact on coronary heart disease (CHD) of intensifying lipid-lowering treatment in statin-treated patients with uncontrolled LDL-C, at moderate to high risk of CHD with no delay or after a 5-year delay, compared with standard of care (no intensification), starting at age 40 years. Intensification was tested with high-intensity statins or statins + ezetimibe. LDL-C levels were extracted from a primary care cohort. CHD risk was estimated using the pooled cohort equation. The effect of cumulative exposure to LDL-C on CHD risk was derived from Mendelian randomization data. Outcomes included CHD events, quality-adjusted life-years (QALYs), healthcare and productivity costs, and incremental cost-effectiveness ratios (ICERs). All outcomes were discounted annually by 5%. RESULTS: Over the lifetime horizon, compared with standard of care, achieving LDL-C control with no delay with high-intensity statins prevented 29 CHD events and yielded 30 extra QALYs (ICERs AU$13 205/QALY) versus 22 CHD events and 16 QALYs (ICER AU$20 270/QALY) with a 5-year delay. For statins + ezetimibe, no delay prevented 53 CHD events and gave 45 extra QALYs (ICER AU$37 271/QALY) versus 40 CHD events and 29 QALYs (ICER of AU$44 218/QALY) after a 5-year delay. CONCLUSIONS: Delaying attainment of LDL-C goals translates into lost therapeutic benefit and a waste of resources. Urgent policies are needed to improve LDL-C goal attainment in statin-treated patients.

The Hospitalizations for Cardiovascular and Respiratory Conditions, Emergency Department Presentations and Economic Burden of Bushfires in Australia Between 2021 and 2030: A Modelling Study.

The health and environmental impacts of bushfires results in substantial economic costs to society. The present analysis sought to estimate the burden of bushfires in Australia over 10 years from 2021 to 2030 inclusive. A dynamic model with yearly cycles was constructed to simulate follow-up of the entire Australian population from 2021 to 2030, capturing deaths and years of life lived. Estimated numbers of bushfire-related-deaths, costs of related-hospitalizations, and broader economic costs were derived from published sources. A 5% annual discount rate was applied to all costs incurred and life years lived from 2022 onwards. Over the 10 years from 2021 to 2030, the modelled analysis predicted that 2418 [95% confidence interval (CI) 2412 - 2422] lives would be lost to bushfires, as well as 8590 [95% CI 8573 - 8606] years of life lost (discounted). Healthcare costs arising from deaths for smoke-related conditions, hospitalizations amounted to AUD $110 million [95% CI 91-129 million] (discounted). The impact on gross domestic product (GDP) totaled AUD $17.2 billion. A hypothetical intervention that reduces the impact of bushfires by 10% would save $11 million in healthcare costs and $1.9 billion in GDP. The health and economic burden of bushfires in Australia looms large during 2021 and 2030. This underscores the importance of actions to mitigate bushfire risk. The findings are useful for the future design and delivery and help policy makers to make informed decisions about investment in strategies to reduce the incidence and severity of future bushfires.

Cost-Effectiveness Analysis of Tobacco Control Strategies in Indonesia.

OBJECTIVES: In Indonesia, tobacco smoking is a significant public health problem that continues to grow, with a prevalence among the highest worldwide. This study aimed to assess the cost-effectiveness of government-funded varenicline, smoking bans in public places, and an additional 10% tobacco tax in Indonesia. METHODS: Markov modeling of Indonesians aged 15 to 84 years was undertaken, with simulated follow-up until age 85 years. Data on demographics, smoking prevalence, and mortality were drawn from the Global Burden of Disease Study 2017. Data regarding the efficacy and costs of the 3 interventions were gathered from published sources. Costs and benefits accrued beyond one year were discounted at 3% per annum. The year value of costing data was 2020. RESULTS: Government-funded varenicline, smoking bans in public places, and an additional 10% tobacco tax were predicted to save 5.5 million, 1.6 million, and 1.7 million years of life, respectively (all discounted). In terms of quality-adjusted life-years, 3 tobacco interventions were predicted to gain 11.9 million, 3.47 million, and 3.78 million in quality-adjusted life-years, respectively. The savings in smoking-related healthcare costs amounted to US $313.8 billion, US $97.5 billion, and US $106 billion, respectively. Hence, from the perspective of the healthcare system, all 3 interventions were cost saving (dominant). CONCLUSIONS: In Indonesia, tobacco control measures are likely to be highly cost-effective and even cost saving from the healthcare system's perspective. These cost savings can be balanced against economic losses that would result from the impact on the sizable Indonesian tobacco industry.

Healthcare utilisation and costing for decompensated chronic liver disease hospitalisations at a Victorian network.

BACKGROUND: The economic burden of decompensated chronic liver disease (CLD) on Australian healthcare services is poorly characterised. AIMS: To evaluate the in-patient healthcare utilisation costs associated with decompensated CLD at Monash Health, an Australian tertiary healthcare service. METHODS: The current retrospective cost analysis examined patients with decompensated CLD admitted between 1 January 2012 and 31 December 2018. Hospitalisations were identified using CLD-specific International Classification of Diseases, Tenth Revision, codes. Cost measures were estimated using the Victorian Weighted Inlier Equivalent Separation funding data based on the Australian Refined Diagnosis Related Groups cost weights. RESULTS: There were 707 hospitalisations in 435 adult patients. The mean age was 56.7 ± 11.7 years and the mean length of stay was 10.28 ± 11.2 days. Median survival was 31 months (interquartile range, 2-94 months) and 177 (40.8%) patients died within 1 year of admission. The cost of admission varied according to decompensation: hepatorenal syndrome ($20 162 AUD), variceal bleed ($16 630 AUD), spontaneous bacterial peritonitis ($12 664 AUD), hepatic encephalopathy ($9973 AUD) and ascites ($9001 AUD). There was no significant difference in the admissions or 30-day readmission rate from 2012 to 2018 financial year (FY). The total adjusted cost of cirrhotic admissions per year increased by 78% from FY2012 to FY2018. CONCLUSION: Hospital admission and readmission for decompensated CLD is common and associated with 40.8% 1-year mortality and high costs. Clearer delineation of goals of care and alternative ambulatory care models for decompensated CLD are urgently required to reduce the high costs and burden on health services.

Cost-Effectiveness of Empagliflozin in Patients With Heart Failure and Preserved Ejection Fraction.

BACKGROUND: Empagliflozin is the first medication to demonstrate clinical benefit in patients with heart failure with preserved ejection fraction, but its cost-effectiveness is unknown. We aimed to evaluate the cost-effectiveness of adding empagliflozin to standard therapy in patients with heart failure with preserved ejection fraction. METHODS: A Markov model from the perspective of the Australian health care system was constructed to compare empagliflozin plus standard care to standard care alone among a hypothetical cohort of patients with heart failure with preserved ejection fraction. Clinical probabilities were derived from The EMPEROR-Preserved (Empagliflozin Outcome Trial in Patients with Chronic Heart Failure with Preserved Ejection Fraction). Costs and utilities were derived from published sources. The main outcome was the incremental cost-effectiveness ratio per quality-adjusted life-year gained. Deterministic and probabilistic sensitivity analyses were performed to assess model uncertainty. Costs and benefits were discounted at 5% annually. RESULTS: Over a lifetime, the addition of empagliflozin to standard care prevented 167 heart failure hospitalizations and 155 heart failure-related urgent care visits for every 1000 patients treated and increased mean quality-adjusted survival by 0.16 quality adjusted life-years per patient. Mean lifetime costs in the empagliflozin and standard care groups were AUD$63 218 and AUD$58 478 per patient, respectively. This resulted in an incremental cost-effectiveness ratio of AUD$29 202 per quality adjusted life-year gained. In probabilistic sensitivity analyses, empagliflozin was cost-effective in 85% of 10 000 Monte Carlo simulations at a willingness-to-pay threshold of AUD$50 000 per quality adjusted life-year gained. CONCLUSIONS: In patients with heart failure with preserved ejection fraction, adding empagliflozin to standard care is likely to be cost-effective when compared with standard care alone in the Australian health care setting.

Estimating the economic impact of acute coronary syndrome in New Zealand over time (ANZACS-QI 64): a national registry-based cost burden study.

OBJECTIVES: To estimate the changes in costs associated with acute coronary syndrome (ACS) admissions in New Zealand (NZ) public hospitals over a 12-year period. DESIGN: A cost-burden study of ACS in NZ was conducted from the NZ healthcare system perspective. SETTING: Hospital admission costs were estimated using relevant diagnosis-related groups and their costs for publicly funded casemix hospitalisations, and applied to 190 364 patients with ACS admitted to NZ public hospitals between 2007 and 2018 identified from routine national hospital datasets. Trends in the costs of index ACS hospitalisation, hospital admissions costs, coronary revascularisation and all-cause mortality up to 1 year were evaluated. All costs were presented as 2019 NZ dollars. PRIMARY OUTCOME MEASURES: Healthcare costs attributed to ACS admissions in NZ over time. RESULTS: Between 2007 and 2018, there was a 42% decrease in costs attributed to ACS (NZ$7.7 million (M) to NZ$4.4 M per 100 000 per year), representing a decrease of NZ$298 827 per 100 000 population per year. Mean admission costs associated with each admission declined from NZ$18 411 in 2007 to NZ$16 898 over this period (p<0.001) after adjustment for key clinical and procedural characteristics. These reductions were against a background of increased use of coronary revascularisation (23.1% (2007) to 38.1% (2018)), declining ACS admissions (366-252 per 100 000 population) and an improvement in 1-year survival post-ACS. Nevertheless, the total ACS cost burden remained considerable at NZ$237 M in 2018. CONCLUSIONS: The economic cost of hospitalisations for ACS in NZ decreased considerably over time. Further studies are warranted to explore the association between reductions in ACS cost burden and changes in the management of ACS.

Integrating the Biology of Cardiovascular Disease into the Epidemiology of Economic Decision Modelling via Mendelian Randomisation.

Health economic analyses are essential for health services research, providing decision-makers and payers with evidence about the value of interventions relative to their opportunity cost. However, many health economic approaches are still limited, especially regarding the primary prevention of cardiovascular disease (CVD). In this article, we discuss some limitations to current health economic models and then outline an approach to address these via the incorporation of genomics into the design of health economic models for CVD. We propose that when a randomised clinical trial is not possible or practical, health economic models for primary prevention of CVD can be based on Mendelian randomisation analyses, a technique to assess causality in observational data. We discuss the advantages of this approach, such as integrating well-known disease biology into health economic models and how this may overcome current statistical approaches to assessing the benefits of interventions. We argue that this approach may provide the economic argument for integrating genomics into clinical practice and the efficient targeting of newer therapeutics, transforming our approach to the primary prevention of CVD, thereby moving from reactive to preventive healthcare. We end by discussing some limitations and potential pitfalls of this approach.

The Cost of Control: Cost-effectiveness Analysis of Hybrid Closed-Loop Therapy in Youth.

OBJECTIVE: Hybrid closed-loop (HCL) therapy is an efficacious management strategy for young people with type 1 diabetes. However, high costs prevent equitable access. We thus sought to evaluate the cost-effectiveness of HCL therapy compared with current care among young people with type 1 diabetes in Australia. RESEARCH DESIGN AND METHODS: A patient-level Markov model was constructed to simulate disease progression for young people with type 1 diabetes using HCL therapy versus current care, with follow-up from 12 until 25 years of age. Downstream health and economic consequences were compared via decision analysis. Treatment effects and proportions using different technologies to define "current care" were based primarily on data from an Australian pediatric randomized controlled trial. Transition probabilities and utilities for health states were sourced from published studies. Costs were considered from the Australian health care system's perspective. An annual discount rate of 5% was applied to future costs and outcomes. Uncertainty was evaluated with probabilistic and deterministic sensitivity analyses. RESULTS: Use of HCL therapy resulted in an incremental cost-effectiveness ratio of Australian dollars (AUD) $32,789 per quality-adjusted life year (QALY) gained. The majority of simulations (93.3%) were below the commonly accepted willingness-to-pay threshold of AUD $50,000 per QALY gained in Australia. Sensitivity analyses indicated that the base-case results were robust. CONCLUSIONS: In this first cost-effectiveness analysis of HCL technologies for the management of young people with type 1 diabetes, HCL therapy was found to be cost-effective compared with current care in Australia.

Patient Perceived Financial Burden in Haematological Malignancies: A Systematic Review.

Advances in scientific understanding have led to novel therapies and improved supportive care for many patients with haematological malignancies. However, these new drugs are often costly, only available at centralised health care facilities, require regular specialist reviews and lengthy treatment regimens. This leads to a significant financial burden. Understanding the impact of financial burden on haematological patients is important to appreciate the urgency of alleviating this systemic issue. METHOD: Eligible studies were identified by systematically searching Medline, PsycINFO, CINAHL and Embase. Self-reported data reported in both quantitative and qualitative studies that described the financial burden for patients with haematological malignancies were included. Quality appraisal of the included studies was undertaken using the Joanna Briggs Institute tools. A narrative synthesis was employed. For quantitative studies, outcomes were extracted, tabulated and categorised to find similarities and differences between the studies. For qualitative studies, quotations, codes and themes were extracted and then clustered. An inductive approach derived qualitative themes. RESULTS: Twenty studies were identified for inclusion. Of the quantitative studies most (83%) employed un-validated researcher-generated measures to assess financial burden. Between 15-59% of patients experienced a financial burden. Out-of-pocket expenditure was frequent for clinical appointments, prescription and non-prescription medication, and travel. Financial burden was associated with a worsening quality of life and living in metropolitan areas, but there was no evidence for impact on survival. Patient-centred experiences from the qualitative inquiry complemented the quantitative findings and five themes were determined: familial or household impact; reliance on others; barriers to care due to cost; and barriers to accessing financial assistance and sources of out-of-pocket expenses. CONCLUSION: The impacts of financial burden are yet to be fully appreciated in haematological malignancies, exacerbated by the heterogeneous methods employed by researchers. Future work should focus on identifying the long-term ramifications of financial burden for patients and should trial interventions to reduce its prevalence and patient impacts.

Current Issues in Health Technology Assessment of Cancer Therapies: A Survey of Stakeholders and Opinion Leaders in Australia.

OBJECTIVE: The aim of this study was to find ways of bridging the gap in opinions concerning health technology assessment (HTA) in reimbursement submission between manufacturers and payers to avoid access delays for patients of vital medicines such as oncology drugs. This was done by investigating differences and similarities of opinion among key stakeholders in Australia. METHODS: The survey comprised of nine sections: background demographics, general statements on HTA, clinical claim, extrapolations, quality of life, costs and health resource utilization, agreements, decision making, and capability/capacity. Responses to each question were summarized using descriptive statistics and comparisons were made using chi-square statistics. RESULTS: There were ninety-seven respondents in total, thirty-seven from the public sector (academia/government) and sixty from the private sector (industry/consultancies). Private and public sector respondents had similar views on clinical claims. They were divided when it came to extrapolation of survival data and costs and health resource utilization. However, they generally agreed that rebates are useful, outcomes-based agreements are difficult to implement, managed entry schemes are required when data are limited, and willingness to pay is higher in cancer compared to other therapeutic areas. They also agreed that training mostly takes place through on the job training and that guideline updates were a least favored opportunity for continued training. CONCLUSIONS: Private sector respondents favor methods that reduce the incremental cost-effectiveness ratio when compared to the public sector respondents. There still exist a number of challenges for HTA in oncology and many research opportunities as a result of this study.