Zanfina Ademi

A systematic review of economic evaluations of the detection and treatment of familial hypercholesterolemia.

OBJECTIVES To systematically evaluate the cost-effectiveness of screening and treatment of familial hypercholesterolaemia (FH). METHODS An extensive search strategy using MeSH terms was used to search Medline, Embase, EBM review (includes databases such as the Centre for Reviews and Dissemination database), the NHS Economic-Evaluation Database, the HTA database, the Cochrane Library and the Database of Abstracts of Reviews of Effects. Completed studies that evaluated cost-effectiveness of treatment and screening of FH were included. Two reviewers independently assessed the quality of the studies. The studies were assessed using the Consensus on Health-Economic Criteria and a published checklist for evaluating model-based economic evaluations (EE). RESULTS Nine studies were identified. Three studies that focused on lipid-lowering treatment among patients with known FH suggested this strategy is highly cost-effective. Six studies reported on the cost-effectiveness of FH screening, and subsequent treatment of those identified with the condition. Compared with no screening, the incremental cost-effectiveness ratio of screening ranged from €3177-€29,554 per life year gained. The results of modelled EE were sensitive to the underlying prevalence of FH among the population being screened, the validity of the screening test and the price and efficacy of lipid-lowering therapy. CONCLUSION Overall, cascade screening for new cases of FH appears to be cost-effective. However, there were uncertainties in the modelling methods, especially with regard to the underlying prevalence of FH, validity of the screening tests, and use of different approaches to assess the outcomes of treatment. Further health EE based on high quality and country-specific data are required.

Familial hypercholesterolaemia: A review with emphasis on evidence for treatment, new models of care and health economic evaluations

Familial hypercholesterolaemia (FH) is a condition that should be familiar to all health professionals involved in preventive medicine. FH is the most common and serious monogenic disorder of lipid metabolism that leads to premature coronary heart disease. However, most cases remain undetected or inadequately treated in our community. We provide an overview of FH, with emphasis on evidence for treatment, new models of care (MoCs) and health economic evaluations. Evidence for treatment is based on cohort studies; while this is a low level class of evidence, MoCs concur in recommending early intervention and lowering of plasma low-density lipoprotein-cholesterol levels by at least 40% with statins. Preliminary health economic evaluations suggest that detecting and treating FH is cost-effective, but further studies based on high-quality international data and standardised costing methods are needed. If the recommendations in the published MoCs are followed, there is likely to be significant improvement in the health and quality of life of patients with FH and their families, as well as major cost savings in healthcare for end-organ damage, including myocardial infarction, acute coronary syndromes and possibly stroke, but this requires to be verified.

Comparison of Health-Related Quality of Life, Work Status, and Health Care Utilization and Costs According to Hip and Knee Joint Disease Severity: A National Australian Study

Background No population-based studies have investigated how the impact of hip and knee joint disease may vary with increasing severity. Objective The purpose of this study was to evaluate health-related quality of life (HRQoL), work status, and health service utilization and costs according to severity of hip and knee joint disease. Design A national cross-sectional survey was conducted. Methods Five thousand individuals were randomly selected from the Australian electoral roll and invited to complete a questionnaire to screen for doctor-diagnosed hip arthritis, hip osteoarthritis (OA), knee arthritis, and knee OA. Severity was classified by means of Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) scores (range=0–100): <7=asymptomatic, 7–38=mild-moderate, and ≥39=severe. Health-related quality of life was evaluated by means of the Assessment of Quality of Life (AQoL) instrument (range=−0.04 to 1.00; scored worst-best). Self-reported data on work status and health service utilization were collected, with health care costs estimated with the use of government data. Results Data were available for 1,157 participants, with 237 (20%) reporting hip or knee joint disease. Of these, 16% (n=37) were classified as asymptomatic, 51% (n=120) as mild-moderate, and 27% (n=64) as severe. The severe group reported very low HRQoL (adjusted mean AQoL=0.43, 95% confidence interval [95% CI]=0.38–0.47) compared with the mild-moderate group (adjusted mean AQoL=0.72, 95% CI=0.69–0.75) and the asymptomatic group (adjusted mean AQoL=0.80, 95% CI=0.74–0.86). Compared with the asymptomatic group, the severe group was >3 times less likely to undertake paid work (adjusted odds ratio=0.28, 95% CI=0.09–0.88) and >4 times less likely to undertake unpaid work (adjusted odds ratio=0.24, 95% CI=0.10–0.62). Although physical therapy services were used infrequently, primary and specialist care utilization and costs were highest for the severe group. Limitations Other costs (including physical therapy consultations) were unavailable. Conclusions A clear pattern of worsening HRQoL, reduced work participation, and higher medical care utilization was seen with increasing severity of joint disease.

Cost–Utility of Angiotensin-Converting Enzyme Inhibitor-Based Treatment Compared With Thiazide Diuretic-Based Treatment for Hypertension in Elderly Australians Considering Diabetes as Comorbidity

AbstractThe objective of this study was to examine the cost-effectiveness of angiotensin-converting enzyme inhibitor (ACEI)-based treatment compared with thiazide diuretic-based treatment for hypertension in elderly Australians considering diabetes as an outcome along with cardiovascular outcomes from the Australian governments perspective.We used a cost–utility analysis to estimate the incremental cost-effectiveness ratio (ICER) per quality-adjusted life-year (QALY) gained. Data on cardiovascular events and new onset of diabetes were used from the Second Australian National Blood Pressure Study, a randomized clinical trial comparing diuretic-based (hydrochlorothiazide) versus ACEI-based (enalapril) treatment in 6083 elderly (age ≥65 years) hypertensive patients over a median 4.1-year period. For this economic analysis, the total study population was stratified into 2 groups. Group A was restricted to participants diabetes free at baseline (n = 5642); group B was restricted to participants with preexisting diabetes mellitus (type 1 or type 2) at baseline (n = 441). Data on utility scores for different events were used from available published literatures; whereas, treatment and adverse event management costs were calculated from direct health care costs available from Australian government reimbursement data. Costs and QALYs were discounted at 5% per annum. One-way and probabilistic sensitivity analyses were performed to assess the uncertainty around utilities and cost data.After a treatment period of 5 years, for group A, the ICER was Australian dollars (AUD) 27,698 (&OV0556; 18,004; AUD 1–&OV0556; 0.65) per QALY gained comparing ACEI-based treatment with diuretic-based treatment (sensitive to the utility value for new-onset diabetes). In group B, ACEI-based treatment was a dominant strategy (both more effective and cost-saving). On probabilistic sensitivity analysis, the ICERs per QALY gained were always below AUD 50,000 for group B; whereas for group A, the probability of being below AUD 50,000 was 85%.Although the dispensed price of diuretic-based treatment of hypertension in the elderly is lower, upon considering the potential enhanced likelihood of the development of diabetes in addition to the costs of treating cardiovascular disease, ACEI-based treatment may be a more cost-effective strategy in this population.

A cost-effectiveness analysis of trametinib plus dabrafenib as first-line therapy for metastatic BRAF V600-mutated melanoma in the Swiss setting

The treatment of patients with metastatic melanomas that harbour BRAF V600E or V600K mutations with trametinib plus dabrafenib appears to be superior to treatment with vemurafenib alone. This treatment regimen is likely to become available in Switzerland in the near future.

Prolonged effectiveness of coronary artery bypass surgery versus drug-eluting stents in diabetics with multi-vessel disease: An updated systematic review and meta-analysis

BACKGROUND Currently, the appropriateness of percutaneous coronary intervention (PCI) using drug-eluting stents (DES) versus coronary artery bypass grafting (CABG) for patients with diabetes (DM) and multi-vessel disease (MVD) is uncertain due to limited evidence from few randomised controlled trials (RCTs). We aimed to compare the clinical effectiveness of CABG versus PCI-DES in DM-MVD patients using an evidence-based approach. METHODS A systematic review and meta-analyses were conducted to compare the risk of all-cause mortality, myocardial infarction (MI), repeat revascularisation, cerebrovascular events (CVE), and major adverse cardiac or cerebrovascular events (MACCE). RESULTS A total of 1,837 and 3,052 DM-MVD patients were pooled from four RCTs (FREEDOM, SYNTAX, VA CARDS, and CARDia) and five non-randomised studies. At mean follow-up of 3 years, CABG compared with PCI-DES was associated with a lower risk of all-cause mortality and MI in RCTs. By contrast, no significant differences were observed in the mean 3.5-year risk of all-cause mortality and MI in non-randomised trials. However, the risk of repeat revascularisations following PCI-DES compared with CABG was 2.3 (95% CI=1.8-2.8) and 3.0 (2.3-4.2)-folds higher in RCTs and non-randomised trials, respectively. Accordingly, the risk of MACCE at 3 years following CABG compared with PCI-DES was lower in both RCTs and non-randomised trials [0.65 (: 0.55-0.77); and 0.77 (0.60-0.98), respectively]. CONCLUSIONS Based on our pooled results, we recommend CABG compared with PCI-DES for patients with DM-MVD. Although non-randomised trials suggest no additional survival-, MI-, and CVE- benefit from CABG over PCI-DES, these results should be interpreted with care.

Drug Treatment and Cost of Cardiovascular Disease in Australia

Australias Pharmaceutical Benefits Scheme supports the use of effective drugs for the prevention and control of cardiovascular risk factors. However, there are little data available describing per person costs of medication in primary prevention and secondary prevention in the community. We aim to understand annual expenditure on cardiovascular medicines according to the level and extent of cardiovascular disease, using participants enrolled in the Reduction of Atherothrombosis for Continued Health (REACH) registry. 2873 participants were recruited into the REACH registry through 273 Australian general practices. Cardiovascular medicines review was undertaken at baseline. Average weighted costs of medications were estimated using government-reimbursed prices. Annual costs were stratified by disease extent and location. The annual mean cost of pharmaceuticals per person was 1307 AU dollars. The average reported medicine use per person across all states and participants groups varied significantly. Participants with cerebrovascular or peripheral arterial disease were prescribed less cardiovascular medication than those with coronary artery disease (CAD) (mean number of drugs 3.5 vs. 4.5, P < 0.0001) and (3.6 vs. 4.5, P < 0.0001), while those with risk factor alone had the same medication use as those with CAD (mean number 4.5). Medication use was lower in Western Australia in comparison to eastern States. Participants with existing cerebrovascular disease and peripheral vascular disease receive less preventive therapy than those with CAD or even risk factors alone. This observation is consistent across all mainland states. Given the evidence of the effectiveness and cost-effectiveness of treating all types of vascular diseases, the present study suggests that there is scope to improve the treatment of these high-risk participants in Australia.

Predictors of increasing waist circumference in an Australian population

OBJECTIVE To identify predictors of increasing waist circumference (WC) over a 5-year period in a contemporary population of Australian adults. DESIGN Longitudinal national cohort of adults participating in the Australian Diabetes, Obesity and Lifestyle Study (AusDiab). SETTINGS Australian adults in 2000 and 2005. SUBJECTS A total of 2521 men and 2726 women aged ≥25 years at baseline who participated in AusDiab and provided anthropometric measurements at baseline (1999-2000) and follow-up (2005). RESULTS A ≥5% increase of baseline WC occurred in 27 % of men and 38% of women over the 5-year period. In the multivariate analysis of the total population, there was a higher risk of ≥5% gain in baseline WC in women, younger people, people with a lower baseline WC, people who never married compared with married/de facto, current smokers compared with never smokers, people with a poorer diet quality and people with a low energy intake. However, there was no significant association with many expected predictors of waist gain such as physical activity. There were some associations between other lifestyle factors and change of WC by sex, age, level of education and across WC categories, but the associations differed across these groups. CONCLUSIONS A ≥5% increase of baseline WC occurred in a significant proportion of men and women over the 5-year period. Of the behavioural factors, poor diet quality was the key predictor of the ≥5% increase of baseline WC in this cohort. The findings highlight the need to understand better the causal role of lifestyle in regard to increasing WC over time.

Cost-effectiveness of apixaban compared to warfarin in the management of atrial fibrillation in Australia:

Objective To determine the cost-effectiveness of apixaban versus warfarin in patients with atrial fibrillation (AF) with a moderate to severe risk of stroke, from an Australian government-perspective. Methods A decision-analytic Markov model was constructed to assess the cost-effectiveness of apixaban versus warfarin, based on data from the Apixaban for Reduction in Stroke and Other Thromboembolic Events in AF (ARISTOTLE) trial. The model comprised five health states: ‘Alive, no major bleeding or stroke’, ‘Alive, no major bleeding, post stroke/systemic embolism’, ‘Alive, post major bleeding, no stroke’, ‘Alive, post-major bleeding and stroke’ and ‘Dead’. Disease cost data was derived from the North-East Melbourne Stroke Incidence Study and the Australian Refined Diagnose Related Groups. Costs of medications were based on data from the Pharmaceutical Benefit Scheme. Utility data was derived from published sources, and an annual discount rate of 5% was applied to costs and benefits. The main outcome of interest was incremental cost-effectiveness ratios per life year gained (LYG) and quality adjusted life years (QALYs) gained. Results Over 20 years, in the sample of 1000 subjects the model predicted that compared to warfarin, apixaban led to a (discounted) of 0.33 LYG and 0.31 QALYs gained, at a net cost of

Quantifying the excess cost and resource utilisation for patients with complications associated with elective knee arthroscopy: a retrospective cohort study.

4,308 per-person. These equated to ICERs of