Insulin therapy in type 2 diabetes patients failing oral agents: cost-effectiveness of biphasic insulin aspart 70/30 vs. insulin glargine in the US.

OBJECTIVES: To project the long-term clinical and economic outcomes of treatment with biphasic insulin aspart 30 (BIAsp 70/30, 30% soluble and 70% protaminated insulin aspart) vs. insulin glargine in insulin-naïve type 2 diabetes patients failing to achieve glycemic control with oral antidiabetic agents alone (OADs). METHODS: Baseline patient characteristics and treatment effect data from the recent 'INITIATE' clinical trial served as input to a peer-reviewed, validated Markov/Monte-Carlo simulation model. INITIATE demonstrated improvements in HbA1c favouring BIAsp 70/30 vs. glargine (-0.43%; p < 0.005) and greater efficacy in reaching glycaemic targets among patients poorly controlled on OAD therapy. Effects on life expectancy (LE), quality-adjusted life expectancy (QALE), cumulative incidence of diabetes-related complications and direct medical costs (2004 USD) were projected over 35 years. Clinical outcomes and costs were discounted at a rate of 3.0% per annum. Sensitivity analyses were performed. RESULTS: Improvements in glycaemic control were projected to lead to gains in LE (0.19 +/- 0.24 years) and QALE (0.19 +/- 0.17 years) favouring BIAsp 70/30 vs. glargine. Treatment with BIAsp 70/30 was also associated with reductions in the cumulative incidences of diabetes-related complications, notably in renal and retinal conditions. The incremental cost-effectiveness ratio was $46 533 per quality-adjusted life year gained with BIAsp 70/30 vs. glargine (for patients with baseline HbA1c >/= 8.5%, it was $34 916). Total lifetime costs were compared to efficacy rates in both arms as a ratio, which revealed that the lifetime cost per patient treated successfully to target HbA1c levels of <7.0% and

Multimedia quality of life assessment: advances with FLAIR.

Assessing impact of functional dependency on quality of life (QOL) among older adults can provide an in-depth understanding of health preferences. Utilities as a measure of preferences are necessary in conducting cost-effectiveness evaluations of healthcare interventions designed to improve overall QOL. We describe further development of a multimedia utility elicitation instrument that is highly portable and easily accessible. An earlier version, FLAIR1, introduced features designed for older adult, computer inexperienced users. FLAIR2 includes modifications such as migration to a web-based platform, consistency checks, audio/visual updates, and more response methods. As compared with FLAIR1, more FLAIR2 respondents (n=318) preferred using the computer and found the computer program to be enjoyable, easy to use, and easily understood. There were also fewer inconsistencies among FLAIR2 respondents. FLAIR2 enhancements have increased portability, minimized invariance and inconsistency, and produced a more user friendly design.

Quality of life assessment software for computer-inexperienced older adults: multimedia utility elicitation for activities of daily living.

Functional status as measured by dependencies in the Activities of Daily Living (ADLs) is an important indicator of overall health for older adults. Methodologies for outcomes-based medical-decision-making for public policy, such as decision modeling and cost-effectiveness analysis, require utilities for outcome health states. Utilities have been reported for many disease states, but have not been indexed by functional status, which is a strong predictor of outcome in geriatrics. We describe here a utility elicitation program developed specifically for use with computer-inexperienced older adults: Functional Limitation And Independence Rating (FLAIR1). FLAIR1 design features address common physical problems of the aged and computer attitudes of inexperienced users that could impede computer acceptance. We interviewed 400 adults ages 65 years and older with FLAIR1. In exit interviews with 154 respondents, 118 (76%) found FLAIR1 easy to use. Design features in FLAIR1 can be applied to other software for older adults

Cost-effectiveness of automated external defibrillators on airlines.

CONTEXT: Installation of automated external defibrillators (AEDs) on passenger aircraft has been shown to improve survival of cardiac arrest in that setting, but the cost-effectiveness of such measures has not been proven. OBJECTIVE: To examine the costs and effectiveness of several different options for AED deployment in the US commercial air transportation system. DESIGN, SETTING, AND SUBJECTS: Decision and cost-effectiveness analysis of a strategy of full deployment on all aircraft as well as several strategies of partial deployment only on larger aircraft, compared with a baseline strategy of no AEDs on aircraft (but training flight attendants in basic life support) for a hypothetical cohort of persons experiencing cardiac arrest aboard US commercial aircraft. Estimates for costs and outcomes were obtained from the medical literature, the Federal Aviation Administration, the Air Transport Association of America, a population-based cohort of Medicare patients, AED manufacturers, and the Bureau of Labor Statistics. MAIN OUTCOME MEASURES: Quality-adjusted survival after cardiac arrest; costs of AED deployment on aircraft and of medical care for cardiac arrest survivors. RESULTS: Adding AEDs on passenger aircraft with more than 200 passengers would cost $35 300 per quality-adjusted life-year (QALY) gained. Additional AEDs on aircraft with capacities between 100 and 200 persons would cost an additional $40 800 per added QALY compared with deployment on large-capacity aircraft only, and full deployment on all passenger aircraft would cost an additional $94 700 per QALY gained compared with limited deployment on aircraft with capacity greater than 100. Sensitivity analyses indicated that the quality of life, annual mortality rate, and the effectiveness of AEDs in improving survival were the most influential factors in the model. In 85% of Monte Carlo simulations, AED placement on large-capacity aircraft produced cost-effectiveness ratios of less than $50 000 per QALY. CONCLUSION: The cost-effectiveness of placing AEDs on commercial aircraft compares favorably with the cost-effectiveness of widely accepted medical interventions and health policy regulations, but is critically dependent on the passenger capacity of the aircraft. Placing AEDs on most US commercial aircraft would meet conventional standards of cost-effectiveness.

Evidence-based coverage policy.

Many health plans apply evidence-based approaches to coverage decisions. The foundation of such approaches is the systematic review of information about the effectiveness of medical interventions. This paper discusses the principles underlying evidence-based coverage policy and how they are applied by two major programs: the Technology Evaluation Center of the Blue Cross Blue Shield Association and the Medicare Coverage Advisory Committee. Although such policies likely have limited effects on spending, they can help to direct medical resources toward effective care.

Cost-effectiveness of androgen suppression therapies in advanced prostate cancer.

BACKGROUND: The costs and side effects of several antiandrogen therapies for advanced prostate cancer differ substantially. We estimated the cost-effectiveness of antiandrogen therapies for advanced prostate cancer. METHODS: We performed a cost-effectiveness analysis using a Markov model based on a formal meta-analysis and literature review. The base case was assumed to be a 65-year-old man with a clinically evident, local recurrence of prostate cancer. The model used a societal perspective and a time horizon of 20 years. Six androgen suppression strategies were evaluated: diethylstilbestrol (DES), orchiectomy, a nonsteroidal antiandrogen (NSAA), a luteinizing hormone-releasing hormone (LHRH) agonist, and combinations of an NSAA with an LHRH agonist or orchiectomy. Outcome measures were survival, quality-adjusted life years (QALYs), lifetime costs, and incremental cost-effectiveness ratios. RESULTS: DES, the least expensive therapy, had a discounted lifetime cost of $3600 and the lowest quality-adjusted survival, 4.6 QALYs. At a cost of $7000, orchiectomy was associated with 5.1 QALYs, resulting in an incremental cost-effectiveness ratio of $7500/QALY relative to DES. All other strategies-LHRH agonists, NSAA, and both combined androgen blockade strategies-had higher costs and lower quality-adjusted survival than orchiectomy. These results were sensitive to the quality of life associated with orchiectomy and the efficacy of combined androgen blockade, and they changed little when prostate-specific antigen results were used to guide therapy. Under a wide range of other assumptions, the cost-effectiveness of orchiectomy relative to DES was consistently less than $20 000/QALY. Androgen suppression therapies were most cost-effective if initiated after patients became symptomatic from prostate metastases. CONCLUSIONS: For men who accept it, orchiectomy is likely to be the most cost-effective androgen suppression strategy. Combined androgen blockade is the least economically attractive option, yielding small health benefits at high relative costs.

A concise review of the cost-effectiveness of coronary heart disease prevention.

Coronary heart disease is one of the largest sources of morbidity, mortality, and health care expenditure in the United States. This article reviews a number of studies that estimate the cost per unit of health benefits associated with different primary and secondary prevention strategies for coronary heart disease. Although prevention does not provide a panacea for rising health care spending, many preventive strategies are cost-effective when compared to other common clinical interventions. Prevention should be incorporated into regular clinical practice.

A cost-effectiveness analysis of prescribing strategies in the management of gastroesophageal reflux disease.

OBJECTIVE: Patients who have uncomplicated gastroesophageal-reflux disease (GERD) typically present with heartburn and acid regurgitation. We sought to determine the cost-effectiveness of H2-receptor antagonists (H2RAs) and proton-pump inhibitors (PPIs) as first-line empiric therapy for patients with typical symptoms of GERD. METHODS: Decision analysis comparing costs and benefits of empirical treatment with H2RAs and PPIs for patients presenting with typical GERD was employed. The six treatment arms in the model were: 1) Lifestyle therapy, including antacids; 2) H2RA therapy, with endoscopy performed if no response to H2RAs; 3) Step up (H2RA-PPI) Arm: H2RA followed by PPI therapy in the case of symptomatic failure; 4) Step down arm: PPI therapy followed by H2RA if symptomatic response to PPI, and antacid therapy if response to H2RA therapy; 5) PPI-on-demand therapy: 8 wk of treatment for symptomatic recurrence, with no more than three courses per year; and 6) PPI-continuous therapy. Measurements were lifetime costs, quality-adjusted life years (QALYs) gained, and incremental cost effectiveness. RESULTS: Initial therapy with PPIs followed by on-demand therapy was the most cost-effective approach, with a cost-effectiveness ratio of $20,934 per QALY gained for patients with moderate to severe GERD symptoms, and $37,923 for patients with mild GERD symptoms. This therapy was also associated with the greatest gain in discounted QALYs. The PPI-on-demand strategy was more effective and less costly than the H2RA followed by PPI strategy or the other treatment arms. The results were not highly sensitive to cost of therapy, QALY adjustment from GERD symptoms, or the success rate of the lifestyle arm. However, when the success rate of the PPI-on-demand arm was < or =59%, the H2RA-PPI arm was the preferred strategy. CONCLUSION: For patients with moderate to severe symptoms of GERD, initial treatment with PPIs followed by on-demand therapy is a cost-effective approach.

Cost-effectiveness of alternative test strategies for the diagnosis of coronary artery disease.

BACKGROUND: The appropriate roles for several diagnostic tests for coronary disease are uncertain. OBJECTIVE: To evaluate the cost-effectiveness of alternative approaches to diagnosis of coronary disease. DESIGN: Meta-analysis of the accuracy of alternative diagnostic tests plus decision analysis to assess the health outcomes and costs of alternative diagnostic strategies for patients at intermediate pretest risk for coronary disease. DATA SOURCES: Studies of test accuracy that met inclusion criteria; published information on treatment effectiveness and disease prevalence. TARGET POPULATION: Men and women 45, 55, and 65 years of age with a 25% to 75% pretest risk for coronary disease. TIME HORIZON: 30 years. PERSPECTIVE: Societal. INTERVENTIONS: Diagnostic strategies were initial angiography and initial testing with one of five noninvasive tests--exercise treadmill testing, planar thallium imaging, single-photon emission computed tomography (SPECT), stress echocardiography, and positron emission tomography (PET)--followed by coronary angiography if noninvasive test results were positive. Testing was followed by observation, medical treatment, or revascularization. OUTCOME MEASURES: Life-years, quality-adjusted life-years (QALYs), costs, and costs per QALY. RESULTS OF BASE-CASE ANALYSIS: Life expectancy varied little with the initial diagnostic test; for a 55-year-old man, the best-performing test increased life expectancy by 7 more days than the worst-performing test. More sensitive tests increased QALYs more. Echocardiography improved health outcomes and reduced costs relative to stress testing and planar thallium imaging. The incremental cost-effectiveness ratio was $75,000/QALY for SPECT relative to echocardiography and was greater than $640,000 for PET relative to SPECT. Compared with SPECT, immediate angiography had an incremental cost-effectiveness ratio of $94,000/QALY. RESULTS OF SENSITIVITY ANALYSIS: Qualitative findings varied little with age, sex, pretest probability of disease, or the test indeterminancy rate. Results varied most with sensitivity to severe coronary disease. CONCLUSIONS: Echocardiography, SPECT, and immediate angiography are cost-effective alternatives to PET and other diagnostic approaches. Test selection should reflect local variation in test accuracy.

Low-molecular-weight heparins compared with unfractionated heparin for treatment of acute deep venous thrombosis. A cost-effectiveness analysis.

BACKGROUND: Low-molecular-weight heparins are effective for treating venous thrombosis, but their cost-effectiveness has not been rigorously assessed. OBJECTIVE: To evaluate the cost-effectiveness of low-molecular-weight heparins compared with unfractionated heparin for treatment of acute deep venous thrombosis. DESIGN: Decision model. DATA SOURCES: Probabilities for clinical outcomes were obtained from a meta-analysis of randomized trials. Cost estimates were derived from Medicare reimbursement and other sources. TARGET POPULATION: Two hypothetical cohorts of 60-year-old men with acute deep venous thrombosis. TIME HORIZON: Patient lifetime. PERSPECTIVE: Societal. INTERVENTION: Fixed-dose low-molecular-weight heparin or adjusted-dose unfractionated heparin. OUTCOME MEASURES: Costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios. An in-patient hospital setting was used for the base-case analysis. Secondary analyses examined outpatient treatment with low-molecular-weight heparin. RESULTS OF BASE-CASE ANALYSIS: Total costs for inpatient treatment were $26,516 for low-molecular-weight heparin and $26,361 for unfractionated heparin. The cost of initial care was higher in patients who received low-molecular-weight heparin, but this was partly offset by reduced costs for early complications. Low-molecular-weight heparin treatment increased quality-adjusted life expectancy by approximately 0.02 years. The incremental cost-effectiveness of inpatient low-molecular-weight heparin treatment was $7820 per QALY gained. Treatment with low-molecular-weight heparin was cost saving when as few as 8% of patients were treated at home. RESULTS OF SENSITIVITY ANALYSIS: When late complications were assumed to occur 25% less frequently in patients who received unfractionated heparin, the incremental cost-effectiveness ratio increased to almost $75,000 per QALY gained. When late complications were assumed to occur 25% less frequently in patients who received low-molecular-weight heparin, this treatment resulted in a net cost savings. Inpatient low-molecular-weight heparin treatment became cost saving when its pharmacy cost was reduced by 31% or more, when it reduced the yearly incidence of late complications by at least 7%, when as few as 8% of patients were treated entirely as outpatients, or when at least 13% of patients were eligible for early discharge. CONCLUSIONS: Low-molecular-weight heparins are highly cost-effective for inpatient management of venous thrombosis. This treatment reduces costs when small numbers of patients are eligible for outpatient management.

Cost-effectiveness of 3 methods to enhance the sensitivity of Papanicolaou testing.

CONTEXT: ThinPrep, AutoPap, and Papnet are 3 new technologies that increase the sensitivity and cost of cervical cancer screening. OBJECTIVE: To estimate the cost-effectiveness of these technological enhancements to Papanicolaou (Pap) tests. DESIGN: We estimated the increase in sensitivity from using these technologies by combining results of 8 studies meeting defined criteria. We used published literature and additional sources for cost estimates. To estimate overall cost-effectiveness, we applied a 9-state time-varying transition state model to these data and information about specific populations. SETTING: A hypothetical program serving a cohort of 20- to 65-year-old women who begin screening at the same age and are representative of the US population. RESULTS: The new technologies increased life expectancy by 5 hours to 1.6 days, varying with the technology and the frequency of screening. All 3 technologies also increased the cost per woman screened by $30 to $257 (1996 US dollars). AutoPap dominated ThinPrep in the base case. At each screening interval, AutoPap increased survival at the lowest cost. The cost per year of life saved rose from $7777 with quadrennial screening to $166000 with annual screening. Papnet produced more life-years at a higher cost per year of life saved. However, when used with triennial screening, each of them produced more life-years at lower cost than conventional Pap testing every 2 years. The cost-effectiveness ratio of each technology improved with increases in the prevalence of disease, decreases in the sensitivity of conventional Pap testing, and increases in the improvement in sensitivity produced by the technology. CONCLUSIONS: Technologies to increase the sensitivity of Pap testing are more cost-effective when incorporated into infrequent screening. Increases in sensitivity and decreases in cost may eventually make each technology more cost-effective.

Relative effectiveness and cost-effectiveness of methods of androgen suppression in the treatment of advanced prostate cancer.

OBJECTIVES: With 184,500 new cases and 39,200 deaths anticipated in 1998, prostate cancer is second only to lung cancer in cancer mortality for men. This report is a systematic review of the evidence from randomized controlled trials on the relative effectiveness of alternative strategies for androgen suppression as treatment of advanced prostate cancer. Three key issues are addressed: (1) the relative effectiveness of the available methods for monotherapy (orchiectomy, luteinizing hormone-releasing hormone [LHRH] agonists, and antiandrogens), (2) the effectiveness of combined androgen blockade compared to monotherapy, and (3) the effectiveness of immediate androgen suppression compared to androgen suppression deferred until clinical progression. Outcomes of interest are overall, cancer-specific, and progression-free survival; time to treatment failure; adverse effects; and quality of life. Two supplementary analyses were conducted for each key question: (1) meta-analysis of overall survival at 2 years (questions 1 and 2) and 5 years (questions 2 and 3), and (2) cost-effectiveness analysis. SEARCH STRATEGY: The MEDLINE, CANCERLIT, and EMBASE databases were searched from 1966 to March 1998, and Current Contents to August 24, 1998, for the terms: leuprolide (Lupron); goserelin (Zoladex); buserelin (Suprefact); flutamide (Eulexin); nilutamide (Anandron, Nilandron); bicalutamide (Casodex); cyproterone acetate (Androcur); diethylstilbestrol (DES); and orchiectomy (castration, orchidectomy). The search was then limited to human studies indexed under the MeSH term "prostatic neoplasms" and by the UK Cochrane Center search strategy for randomized controlled trials. Total yield was 1,477 references. SELECTION CRITERIA: We Reports of efficacy outcomes were limited to randomized controlled trials. Phase II studies that reported on withdrawals from therapy and all studies reporting on quality of life were also included. DATA COLLECTION AND ANALYSIS: The systematic review used a prospectively designed protocol conducted by two independent reviewers, with disagreements resolved by consensus. The meta-analysis combined data on overall survival using a random effects model. The cost-effectiveness analysis used a decision analysis model of advanced prostate cancer with health states and transitions derived from the literature and estimates of effectiveness derived from the meta-analysis. The cost-effectiveness analysis is conducted from a societal perspective, consistent with the guidelines of the U.S. Public Health Service Panel on Cost-Effectiveness in Health and Medicine. MAIN RESULTS: Survival after treatment with an LHRH agonist is equivalent to survival after orchiectomy. The available LHRH agonists are equally effective, and no LHRH agonist is superior to the other when adverse effects are considered. Survival may be somewhat lower with use of a nonsteroidal antiandrogen. There is no statistically significant difference in survival at 2 years between patients treated with combined androgen blockade or monotherapy. Meta-analysis of the limited data available shows a statistically significant difference in survival at 5 years that favors combined androgen blockade. However, the magnitude of this difference is of questionable clinical significance. For the subgroup of patients with good prognosis, there is no statistically significant difference in survival. Adverse effects leading to withdrawal from therapy occurred more often with combined androgen blockade. No evidence is yet available from randomized controlled trials of androgen suppression initiated at prostate-specific antigen (PSA) rise after definitive therapy for clinically localized disease. For patients who are newly diagnosed with locally advanced or asymptomatic metastatic disease, the evidence is insufficient to determine whether primary androgen suppression initiated at diagnosis improves outcomes. (ABSTRACT TRUNCATED)

Cost effectiveness of coronary heart disease prevention strategies in adults.

Although risk-factor modification has gained wide acceptance as an effective approach to the prevention of coronary heart disease (CHD), health planners, physicians and patients confront considerable uncertainty over the most appropriate and efficient preventive strategies. Some preventive approaches are both inexpensive and effective; others are expensive while their effectiveness is slight or unproven. Effectiveness varies with an individual's age, gender and other risk factors. Information provided by a cost-effectiveness analysis can clarify the value of alternative strategies for CHD prevention in specific populations, thereby helping to choose among them. It does so by producing a standard measure of value--the cost per year of life saved (YLS) or cost per quality-adjusted life-year (QALY) saved--that reveals which of several alternative interventions provides the greatest health benefit from a given expenditure. This article summarises the extensive literature on the cost effectiveness of CHD prevention with an emphasis on primary prevention. Published work indicates that smoking-cessation programmes, particularly those that rely on counselling with or without nicotine supplements, are highly cost effective in many settings. Although the evidence is limited, exercise programmes also appear to be cost effective. The detection and treatment of hypertension is highly cost effective, particularly when inexpensive drugs with proven effectiveness, such as diuretics or beta-blockers, are used. Hormone-replacement therapy is a cost-effective approach to CHD prevention in most postmenopausal women, although direct clinical trial data are lacking and it is uncertain which hormone preparation is best. Cholesterol reduction is a cost-effective strategy for the prevention of CHD in individuals without other treatable risk factors who are at very high risk of developing CHD. For individuals with multiple CHD risk factors, the choice of risk-modification strategies is complex and depends upon the interactions of risk and the relative costs of treating each risk.

Cost-effectiveness and cost-benefit analysis of using methotrexate vs Goeckerman therapy for psoriasis. A pilot study.

OBJECTIVE: To analyze the net benefit and cost-effectiveness of methotrexate use and Goeckerman therapy for psoriasis. DESIGN: Net benefit and cost-effectiveness depend on the costs, efficacy, and utilities of therapy. Utilities are quantitative measures of patient preferences. We obtained costs by using resource-based accounting techniques. Efficacy was estimated from literature reports. We surveyed patients with psoriasis, dermatologists, and healthy subjects using utility assessment methods. All assumptions were examined in a sensitivity analysis. MAIN OUTCOME MEASURES: For net benefit, if benefits out-weighed the costs, it was deemed worth providing. For the cost-effectiveness analysis, the ratio of costs-to-effectiveness of less than $35,000 was considered cost-effective. RESULTS: Using utilities from healthy nonexperts, the costs of both therapies exceeded the benefits in mild and moderate psoriasis. In severe psoriasis, only methotrexate demonstrates a net benefit. Both therapies were cost-effective compared with no therapy. Liquid methotrexate should be chosen over the tablet form since it was cheaper and had the same outcome. Goeckerman was cost-effective against liquid methotrexate in severe, but not mild or moderate psoriasis. There was a trend for therapies to be more cost-effective when using patient utilities and less with dermatologist utilities. The results were highly sensitive to efficacy and utilities. CONCLUSIONS: The results of this study need to be confirmed in other settings, but they demonstrate that the tools of cost-effectiveness and cost-benefit analysis have great potential value in dermatology. Once efficacy is better characterized and utilities better quantified, these types of analyses will be crucial for health care policy.

Mortality, hospital admissions, and medical costs of end-stage renal disease in the United States and Manitoba, Canada.

OBJECTIVES: National registry data suggest that mortality rates among patients with end-stage renal disease are lower in Canada than in the United States. Casemix and treatment variables, although limited in such instances, do not explain this difference. Using a more complete set of casemix and treatment variables from clinical databases, this study assesses mortality, hospital admission, and the cost of medical care for patients with end-stage renal disease treated in Manitoba, Canada and the United States. METHODS: Mortality rates were compared in patients with end-stage renal disease treated in the Province of Manitoba and a random sample of US patients enrolled in the US Renal Data System Casemix Severity Study. Hospital admission rates and costs of care were compared in Manitoba patients and in patients with end-stage renal disease in a large health care organization in Detroit, Michigan. RESULTS: Levels of serum creatinine, urea, and estimated glomerular filtration rate indicated more severe renal impairment at the outset of treatment in Manitoba than in the United States. Manitoba patients were more than twice as likely to receive kidney transplants as US Renal Data System patients. No patients in Manitoba used reprocessed dialyzers, compared with 57% of US Renal Data System patients. After adjustment for all casemix and treatment variables, the mortality rate was 47% higher in the United States. The hospital admission rate in Detroit was 41% lower than the hospital admission rate in Manitoba, which primarily reflects the doubled rate of transplantation in Manitoba. Adjusted total monthly costs were $503 higher in Detroit than in Manitoba. CONCLUSIONS: The higher mortality rates in the United States cannot be fully explained by adjustments for observable casemix or treatment variables. Further research is needed to identify factors that explain how Manitoba achieves a lower mortality rate while paying less for end-stage renal disease care than the United States.