A cost‐effectiveness analysis of sunitinib in patients with metastatic renal cell carcinoma intolerant to or experiencing disease progression on immunotherapy: perspective of the Spanish National Health System

Aim: To investigate the cost‐effectiveness of sunitinib (50 mg/day, schedule 4/2) vs. best supportive care (BSC) in patients with cytokine‐refractory metastatic renal cell carcinoma (mRCC), from the perspective of the Spanish National Health Service. Material and Methods: A Markov model compared the cost‐effectiveness (taking into account drugs; medical visits; laboratory tests; X‐rays; terminal care; adverse event management) of sunitinib and BSC across three disease states: no progression, survival with progression and death from mRCC or other causes. Results: The monthly incremental cost‐effectiveness ratio (ICER) values for sunitinib treatment were €6073/progression‐free survival month, €25 199/life years and €34 196/quality‐adjusted life years (QALY) gained. In 95% of cases, the ICER/QALY values were below the accepted €45 000/QALY threshold. Efficacy and cost of sunitinib had the greatest impact on cost‐effectiveness. Conclusion: Sunitinib has a good cost‐effectiveness profile in mRCC. The cost per life year and QALY gained is affordable according to current effectiveness thresholds in developed countries.     

Cost‐utility analysis of bevacizumab versus ranibizumab in neovascular age‐related macular degeneration using a Markov model

Objective To evaluate the cost‐effectiveness of intravitreal bevacizumab to ranibizumab in patients with neovascular age‐related macular degeneration (AMD). Methods A cost‐utility analysis using a Markov model was performed to evaluate incremental cost‐effectiveness ratio [ICER, $US per quality‐adjusted life year (QALY) gained] between bevacizumab and ranibizumab from a US payer perspective. Transition probabilities for ranibizumab and bevacizumab were extrapolated from published studies and local institutional data. Utility values, likewise, were obtained from another published study. Mortality rates were determined from the Centers for Disease Control 2003 Life Tables. Resource utilization and total direct costs were estimated using the Centers for Medicare and Medicaid Services and the Veterans Affairs Decision Support System. A hypothetical cohort of 1000 patients was simulated through the model for 20 years. Sensitivity analyses were performed using univariate and probabilistic sensitivity analysis (PSA) on all costs, transition probabilities and utility values. An acceptability curve was generated to illustrate the cost‐effectiveness probability of bevacizumab to ranibizumab with increasing willingness‐to‐pay (WTP). Results The cost‐effectiveness ratios (CER) for bevacizumab and ranibizumab were $1405 per QALY and $12 177 per QALY, respectively. The ICER for bevacizumab was dominant compared to ranibizumab. The base‐case CER was sensitive to drug costs of the study medications with a breakeven point of $44 for ranibizumab and $2666 for bevacizumab. PSA revealed a 95% probability of bevacizumab being more cost‐effective than ranibizumab at a WTP of $50 000 per QALY gained. Conclusion Based on a WTP defined at $50 000 per QALY gained, bevacizumab was cost‐effective versus ranibizumab 95% of the time because of lower acquisition costs and increased efficacy.     

A Cost-Utility Analysis of Pregabalin Versus Duloxetine for the Treatment of Painful Diabetic Neuropathy

ABSTRACT

The objective of the current study was to determine the cost-utility of pregabalin versus duloxetine for treating painful diabetic neuropathy (PDN) using a decision tree analysis. Literature searches identified clinical trials and real-world studies reporting the efficacy, tolerability, safety, adherence, opioid usage, health care utilization, and costs of pregabalin and duloxetine. The proportions of patients reported in the included studies were used to determine probabilities in the decision tree model. The base-case model included the Food and Drug Administration (FDA)-approved doses of pregabalin (300 mg/day) and duloxetine (60 mg/day), whereas “real-world” sensitivity analyses explored the effects over a range of doses (pregabalin 75–600 mg/day, duloxetine 20–120 mg/day). A 6-month time horizon and a US third-party payer perspective were chosen for the study. Outcomes from the model were expressed as cost per quality-adjusted life-year (QALY). In the base-case model, duloxetine cost less and was more effective than pregabalin (incremental cost ?$187, incremental effectiveness 0.011 QALYs). Results from two real-world sensitivity analyses indicated that duloxetine cost $16,300 and $20,667 more per additional QALY than pregabalin. Using a decision tree model that incorporated both clinical trial and real-world data, duloxetine was a more cost-effective option than pregabalin in the treatment of PDN from the perspective of third-party payers.     

Cost-effectiveness of drotrecogin alfa (activated) in the treatment of severe sepsis

OBJECTIVE: To assess the cost-effectiveness of drotrecogin alfa (activated) therapy, which was recently shown to reduce mortality in severe sepsis. DESIGN: Estimates of effectiveness and resource use were based on data collected prospectively as part of a multicenter international trial. Estimates of hospital costs were based on a subset of the patients treated in the United States (33% of all enrolled patients). Lifetime projections were modeled from published sources and tested in sensitivity analyses. Analyses were conducted from the United States societal perspective, limited to healthcare costs, and using a 3% annual discount rate. SETTING: A total of 164 medical institutions in 11 countries. PATIENTS: Adults > or = 18 yrs of age with severe sepsis INTERVENTIONS: Eligible patients were randomly assigned to receive a 96-hr intravenous infusion of drotrecogin alfa (activated) at 24 microg/kg/hr (n = 850) or placebo (n = 840). MEASUREMENTS AND MAIN RESULTS: Base Case: incremental short-term (days 1-28) healthcare costs per day-28 survivor; Panel on Cost-Effectiveness in Health and Medicine Reference Case: incremental lifetime healthcare costs per quality-adjusted life-year. Over the first 28 days (short-term Base Case), drotrecogin alfa (activated) increased the costs of care by $9,800 and survival by 0.061 lives saved per treated patient. Thus, drotrecogin alfa (activated) cost $160,000 per life saved (with 84.7% probability that ratio is <$250,000 per life saved). Projected to lifetime (lifetime Reference Case), drotrecogin alfa (activated) increased the costs of care by $16,000 and quality-adjusted survival by 0.33 quality-adjusted life-years per treated patient. Thus, drotrecogin alfa (activated) cost $48,800 per quality-adjusted life-year (with 82% probability that ratio is <$100,000 per quality-adjusted life-year). Estimates were generally robust to sensitivity analyses, although cost-effectiveness deteriorated to >$100,000 per quality-adjusted life-year if survivors lived <4.6 yrs on average. Drotrecogin alfa (activated) cost $27,400 per quality-adjusted life-year when limited to patients with an Acute Physiology and Chronic Health Evaluation II score > or = 25 and was cost-ineffective when limited to patients with a score <25. CONCLUSIONS: Drotrecogin alfa has a cost-effectiveness profile similar to that of many well-accepted healthcare strategies and below commonly quoted thresholds.     

Cost-effectiveness of Osimertinib vs Docetaxel-bevacizumab in Third-line Treatment in EGFR T790M Resistance Mutation Advanced Non–Small Cell Lung Cancer in China

PurposeThis study aimed to evaluate the cost-effectiveness of osimertinib vs docetaxel and bevacizumab in third-line treatment in EGFR T790M resistance mutation advanced non–small cell lung cancer in China from the perspective of the health care system.MethodsTo explore modeling uncertainty, 2 different model methods (a Markov model and a partitioned survival [PS] model) were developed to simulate costs and health outcomes during a lifetime. Both models consisted of 3 health states: progression-free survival, postprogression survival, and death. Efficacy and safety data of osimertinib vs docetaxel and bevacizumab in patients who had acquired EGFR T790M resistance mutation were derived from a key head-to-head clinical trial. Cost and utility values were derived from local charges, the literature, the China Drug Bidding Database, and patients’ health care documents. Two scenario analyses and sensitivity analyses were performed to explore the robustness of the results.FindingsIn the Markov model, compared with docetaxel and bevacizumab, osimertinib yielded 0.69 additional quality-adjusted life-years (QALYs) at an additional cost (in US dollars) of $17,311 for an incremental cost-utility ratio (ICUR) of $25,463 per QALY. In the PS model, osimertinib yielded an additional 0.69 QALYs with an incremental cost of $17,827 for an ICUR of $25,951 per QALY. From the Markov model, the ICUR was $29,416 per QALY in scenario 1 and $25,543 per QALY in scenario 2. From the PS model, the ICUR was $30,264 per QALY and $25,947 per QALY for scenarios 1 and 2, respectively. In the probabilistic sensitivity analysis, osimertinib treatment had a 21%–63% probability of being cost-effective at a willingness-to-pay threshold of $9777 to $29,330 per QALY (1–3 times the gross domestic product per capita).ImplicationsThe findings from the present analysis suggest that osimertinib could be cost-effective vs docetaxel and bevacizumab in third-line treatment in EGFR T790M resistance mutation advanced non–small cell lung cancer in China.     

Cost effectiveness of intensive care in a low resource setting: A prospective cohort of medical critically ill patients

AIM: To calculate cost effectiveness of the treatment of critically ill patients in a medical intensive care unit (ICU) of a middle income country with limited access to ICU resources. METHODS: A prospective cohort study and economic evaluation of consecutive patients treated in a recently established medical ICU in Sarajevo, Bosnia and Herzegovina. A cost utility analysis of the intensive care of critically ill patients compared to the hospital ward treatment from the perspective of the health care system was subsequently performed. Incremental cost effectiveness was calculated using estimates of ICU vs non-ICU treatment effectiveness based on a formal systematic review of published studies. Decision analytic modeling was used to compare treatment alternatives. Sensitivity analyses of the key model parameters were performed. RESULTS: Out of 148 patients, seventy patients (47.2%) survived to one year after critical illness with a median quality of life index 0.64 [interquartile range(IQR) 0.49-0.76]. Median number of life years gained per patient was 30 (IQR 16-40) or 18 quality adjusted life years (QALYs) (IQR 7-28). The cost of treatment of critically ill patients varied between 1820 dollar and 20109 dollar per hospital survivor and between 100 dollar and 2514 dollar per QALY saved. Mean factors that influenced costs were: Age, diagnostic category, ICU and hospital length of stay and number and type of diagnostic and therapeutic interventions. The incremental cost effectiveness ratio for ICU treatment was estimated at 3254 dollar per QALY corresponding to 35% of per capita GDP or a Very Cost Effective category according to World Health Organization criteria. CONCLUSION: The ICU treatment of critically ill medical patients in a resource poor country is cost effective and compares favorably with other medical interventions. Public health authorities in low and middle income countries should encourage development of critical care services.     

Cost-effectiveness of long-acting bronchodilators for chronic obstructive pulmonary disease

OBJECTIVE: To evaluate and compare the cost-effectiveness of long-acting bronchodilators by estimating incremental costs per quality-adjusted life-year (QALY) gained in patients with moderate to severe chronic obstructive pulmonary disease. METHODS: This cost-effective analysis was conducted from a third-party payer's perspective. The study was a retrospective pooled analysis, and the effectiveness evidence was derived from a systematic review of literature published from January 1, 1980, to April 14, 2006. Incremental QALYs were estimated by converting the St George's Respiratory Questionnaire scores into EuroQoL-5D scores and using these combined scores as the summary benefit measure. RESULTS: The incremental cost per additional QALY was $26,094 (range, $11,780-$77,214) for tiotropium and $41,000 (range, $23,650-$98,750) for salmeterol compared with placebo. The cost per QALY gained was lower with tiotropium compared with salmeterol or ipratropium based on either the pooled data of available trials or a head-to-head trial. Treatment with tiotropium could save $391 per year while gaining 13 quality-adjusted days compared with ipratropium. CONCLUSION: Tiotropium appears to be more cost-effective than the alternatives and may be the preferred agent for maintenance therapy in patients with moderate to severe chronic obstructive pulmonary disease. Compared with ipratropium, tiotropium could be cost saving. Because of the wide ranges of cost-effectiveness ratios for tiotropium and salmeterol and the significant overlap between them, a large prospective head-to-head trial would help address the uncertainty and confirm the results of this analysis.     

Cost–Effectiveness of Helicopter Transport of Stroke Patients for Thrombolysis

OBJECTIVES: Treatment with intravenous (IV) or intra-arterial (IA) thrombolysis in patients with acute ischemic stroke demands careful patient selection and specialized institutional capabilities. Physicians at hospitals without these resources may prefer patient transfer for acute treatment. Helicopter transport for these patients has been described but without analysis of the effects of its additional cost. The authors examined the cost-effectiveness of helicopter transport for patients with acute stroke. METHODS: Costs per additional good outcome and per quality-adjusted life-year (QALY) were calculated using a computer model. Input variables included flight, thrombolytic agent, and angiography costs; annual cost per patient for long-term care of symptomatic stroke; percentage of transported patients treated; percentage of patients receiving IV versus IA therapy; discount rate; absolute probability of good outcome; annual mortality with and without treatment; and quality-of-life modifier. Sensitivity analysis was performed. RESULTS: Helicopter transport of acute stroke patients to tertiary care centers for thrombolytic therapy costs $35,000 per additional good outcome and $3,700 per QALY for the reference case. Cost-effectiveness was sensitive to the effectiveness of thrombolysis but minimally sensitive to most other input values. Cost per QALY ranged from $0 to $50,000, as the absolute increase in good outcomes (minimal or no deficit) ranged from 20% to 5%. Cost-effectiveness was not sensitive to ranges of helicopter flight costs or the proportion of flown patients undergoing treatment. CONCLUSIONS: This model indicates helicopter transfer of patients with suspected acute ischemic stroke for potential thrombolysis is cost-effective for a wide range of system variables.     

Genetic testing in patients with acute coronary syndrome undergoing percutaneous coronary intervention: a cost‐effectiveness analysis

Summary. Background: The CYP2C19 genotype is a predictor of adverse cardiovascular events in acute coronary syndrome (ACS) patients undergoing percutaneous coronary intervention (PCI) treated with clopidogrel. Objectives: We aimed to evaluate the cost‐effectiveness of a CYP2C19*2 genotype‐guided strategy of antiplatelet therapy in ACS patients undergoing PCI, compared with two ‘no testing’ strategies (empiric clopidogrel or prasugrel). Methods: We developed a Markov model to compare three strategies. The model captured adverse cardiovascular events and antiplatelet‐related complications. Costs were expressed in 2010 US dollars and estimated using diagnosis‐related group codes and Medicare reimbursement rates. The net wholesale price for prasugrel was estimated as $5.45 per day. A generic estimate for clopidogrel of $1.00 per day was used and genetic testing was assumed to cost $500. Results: Base case analyses demonstrated little difference between treatment strategies. The genetic testing‐guided strategy yielded the most QALYs and was the least costly. Over 15 months, total costs were $18 lower with a gain of 0.004 QALY in the genotype‐guided strategy compared with empiric clopidogrel, and $899 lower with a gain of 0.0005 QALY compared with empiric prasugrel. The strongest predictor of the preferred strategy was the relative risk of thrombotic events in carriers compared with wild‐type individuals treated with clopidogrel. Above a 47% increased risk, a genotype‐guided strategy was the dominant strategy. Above a clopidogrel cost of $3.96 per day, genetic testing was no longer dominant but remained cost‐effective. Conclusions: Among ACS patients undergoing PCI, a genotype‐guided strategy yields similar outcomes to empiric approaches to treatment, but is marginally less costly and more effective.     

Cost‐effectiveness of Acupuncture for Chronic Nonspecific Low Back Pain

Cost‐effectiveness is a major criterion underpinning decisions in mainstream health care. Acupuncture is increasingly used in patients with chronic lower back pain (LBP), but there is a lack of evidence on cost‐effectiveness. The objective of this study was to assess the cost‐effectiveness of acupuncture in alleviating chronic LBP either alone or in conjunction with standard care compared with patients receiving routine care, and/or sham. To determine effectiveness, we undertook meta‐analyses which found a significant improvement in pain in those receiving acupuncture and standard care compared with those receiving standard care alone. For acupuncture and standard care vs. standard care and sham, a weak positive effect was found for weeks 12 to 16, but this was not significant. For acupuncture alone vs. standard care alone, a significant positive effect was found at week 8, but not at weeks 26 or 52. The main outcome parameters for our cost‐effectiveness analysis were the incremental cost‐effectiveness ratio (ICER) of acupuncture treatment presented as cost (A$) per disability‐adjusted life‐year (DALY) saved. The WHO benchmark for a very highly cost‐effective intervention is one that costs less than gross domestic product per capita per quality‐adjusted life‐year (QALY) gained or DALY averted, or less than around $A52,000 in 2009 (the base year for the analysis). According to this threshold, acupuncture as a complement to standard care for relief of chronic LBP is highly cost‐effective, costing around $48,562 per DALY avoided. When comorbid depression is alleviated at the same rate as pain, cost is around $18,960 per DALY avoided. Acupuncture as a substitute for standard care was not found to be cost‐effective unless comorbid depression was included. According to the WHO cost‐effectiveness threshold values, acupuncture is a cost‐effective treatment strategy in patients with chronic LBP.     

Cost-Utility Analysis of Sacubitril/Valsartan Use Compared With Standard Care in Chronic Heart Failure Patients With Reduced Ejection Fraction in South Korea

PurposeSacubitril/valsartan, the first-in-class angiotensin receptor neprilysin inhibitor (ARNI), is a possible treatment option for chronic heart failure patients with reduced ejection fraction (HFrEF). The aim of this study was to estimate the cost-effectiveness of sacubitril/valsartan use in South Korea for treating patients with HFrEF compared with that of enalapril, an angiotensin-converting enzyme inhibitor, and with angiotensin receptor blockers (ARBs).MethodsA Markov model was designed to estimate the lifetime cost-effectiveness of treatment for patients with HFrEF. Cohorts in the alive-state incurred a monthly risk of hospitalization because of deteriorated HF, adverse events (AEs), or death. Transition probabilities of sacubitril/valsartan and enalapril were estimated by using data from the PARADIGM-HF (Prospective Comparison of ARNI With ACEI to Determine Impact on Global Mortality and Morbidity in Heart Failure) trial. The effectiveness of ARBs (eg, reduction in mortality and hospitalization rates) was assumed to be identical to that of enalapril, according to the results of the meta-analysis. However, there was no comparative evidence for AEs. We therefore conducted a Bayesian network meta-analysis and adjusted the incidence rate of AEs for ARBs. The utility for estimating quality-adjusted life years (QALYs) was elicited by the survey of the general South Korean population by using EuroQol-5 dimensions. We calculated the medical costs, including medication, monitoring, hospitalization, AEs, and terminal care, from the health care sector perspective. Costs and effectiveness were discounted by 5%. One-way sensitivity analyses and a probabilistic sensitivity analysis were conducted to determine the model robustness.FindingsThe total cost per patient for sacubitril/valsartan and enalapril was $25,832 and $18,295, respectively. Sacubitril/valsartan was associated with an ∼8- month longer life expectancy compared with enalapril and a QALY gain of 0.59. As a result, the incremental cost-effectiveness ratio for sacubitril/valsartan versus enalapril was $12,722 per QALY. The incremental cost-effectiveness ratio of sacubitril/valsartan versus ARB was $11,970 with an incurred cost of $18,741 for the ARB group. The main results and those of various sensitivity analyses were lower than a threshold of $20,000.ImplicationsFrom a health care sector perspective, sacubitril/valsartan is a cost-effective treatment for HFrEF compared with enalapril and ARBs. This finding could be helpful for cardiologists or decision makers in reaching cost-effective choices regarding the treatment selection process.     

Comparison of the metabolic and economic consequences of long‐term treatment of schizophrenia using ziprasidone,olanzapine, quetiapine and risperidone in Canada: a cost‐effectiveness analysis

Rationale, aims and objectives Second‐generation antipsychotic agents have varying propensities to cause weight gain, elevated lipid levels and associated long‐term complications. This study evaluates the cost‐effectiveness of four second‐generation antipsychotic agents used in Canada for the treatment of schizophrenia (ziprasidone, olanzapine, quetiapine, risperidone) with a focus on their long‐term metabolic consequences. Method Using data from the Clinical Antipsychotic Trials of Intervention Effectiveness Study, a semi‐Markov model was developed to predict the incidence and associated costs of type 2 diabetes, cardiovascular complications (e.g. angina, myocardial infarction, stroke, cardiovascular disease death), and acute psychiatric hospitalizations in patients with chronic schizophrenia treated over 5 years. Incremental costs per quality‐adjusted life year (QALY) gained were calculated from the perspective of the Canadian provincial ministries of health. Scenario and probabilistic sensitivity analyses were performed. Results The total average cost of treatment with ziprasidone was $25 301 versus $28 563 with olanzapine, $26 233 with quetiapine and $21 831 with risperidone. Ziprasidone had the lowest predicted number of type 2 diabetes cases and cardiovascular disease events, and the highest QALY gains. Patients receiving quetiapine had the highest predicted number of hospitalizations. Ziprasidone was less costly and resulted in more QALYs compared with olanzapine and quetiapine. Compared with risperidone, ziprasidone was more costly and had higher QALYs, with an incremental cost per QALY gained of $218 060. Conclusion Compared with olanzapine and quetiapine, ziprasidone produced savings to the health care system. Although ziprasidone generated incremental expenditures versus risperidone, it resulted in more QALYs. Based on this analysis, ziprasidone treatment possesses cost and therapeutic advantages compared with olanzapine and quetiapine.     

Glucosamine sulphate in the treatment of knee osteoarthritis: cost‐effectiveness comparison with paracetamol

Introduction: The aim of this study was to explore the cost‐effectiveness of glucosamine sulphate (GS) compared with paracetamol and placebo (PBO) in the treatment of knee osteoarthritis. For this purpose, a 6‐month time horizon and a health care perspective was used. Material and methods: The cost and effectiveness data were derived from Western Ontario and McMaster Universities Osteoarthritis Index data of the Glucosamine Unum In Die (once‐a‐day) Efficacy trial study by Herrero‐Beaumont et al. Clinical effectiveness was converted into utility scores to allow for the computation of cost per quality‐adjusted life year (QALY) For the three treatment arms Incremental Cost‐Effectiveness Ratio were calculated and statistical uncertainty was explored using a bootstrap simulation. Results: In terms of mean utility score at baseline, 3 and 6 months, no statistically significant difference was observed between the three groups. When considering the mean utility score changes from baseline to 3 and 6 months, no difference was observed in the first case but there was a statistically significant difference from baseline to 6 months with a p‐value of 0.047. When comparing GS with paracetamol, the mean baseline incremental cost‐effectiveness ratio (ICER) was dominant and the mean ICER after bootstrapping was ?1376 €/QALY indicating dominance (with 79% probability). When comparing GS with PBO, the mean baseline and after bootstrapping ICER were 3617.47 and 4285 €/QALY, respectively. Conclusion: The results of the present cost‐effectiveness analysis suggested that GS is a highly cost‐effective therapy alternative compared with paracetamol and PBO to treat patients diagnosed with primary knee OA.     

Assessing the Cost-effectiveness of a Hypothetical Disease-modifying Therapy With Limited Duration for the Treatment of Early Symptomatic Alzheimer Disease

PurposeClinical trials have produced promising results for disease-modifying therapies (DMTs) for Alzheimer’s disease (AD); however, the evidence on their potential cost-effectiveness is limited. This study assesses the cost-effectiveness of a hypothetical DMT with a limited treatment duration in AD.MethodsWe developed a Markov state–transition model to estimate the cost-effectiveness of a hypothetical DMT plus best supportive care (BSC) versus BSC alone among Americans living with mild cognitive impairment (MCI) due to AD or mild AD. AD states included MCI due to AD, mild AD, moderate AD, severe AD, and death. A hypothetical DMT was assumed to confer a 30% reduction in progression from MCI and mild AD. The base case annual drug acquisition cost was assumed to be $56,000. Other medical and indirect costs were obtained from published literature or list prices. Utilities for patients and caregivers were obtained from the published literature and varied by AD state and care setting (community care or long-term care). We considered 3 DMT treatment strategies: (1) treatment administered until patients reached severe AD (continuous strategy), (2) treatment administered for a maximum duration of 18 months or when patients reached severe AD (fixed-duration strategy), and (3) 40% of patients discontinuing treatment at 6 months because of amyloid plaque clearance and the remaining patients continuing treatment until 18 months or until they reached severe AD (test-and-discontinue strategy). Incremental cost-effectiveness ratios (ICERs) were calculated as the incremental cost per quality-adjusted life-year (QALY) gained.FindingsFrom the health care sector perspective, continuous treatment with a hypothetical DMT versus BSC resulted in an ICER of $612,354 per QALY gained. The ICER decreased to $157,288 per QALY gained in the fixed-duration strategy, driven by large reductions in treatment costs. With 40% of patients discontinuing treatment at 6 months (test-and-discontinue strategy), the ICER was $125,631 per QALY gained. In sensitivity and scenario analyses, the ICER was the most sensitive to changes in treatment efficacy, treatment cost, and the initial population AD state distribution. From the modified societal perspective, ICERs were 6.3%, 20.4%, and 25.1% lower than those from the health care sector perspective for the continuous, fixed-duration, and test-and-discontinue strategies, respectively.ImplicationsUnder a set of assumptions for annual treatment costs and the magnitude and duration of treatment efficacy, DMTs used for a limited duration may deliver value consistent with accepted US cost-effectiveness thresholds.     

Economic evaluation of aquatic exercise for persons with osteoarthritis

OBJECTIVES: To estimate cost and outcomes of the Arthritis Foundation aquatic exercise classes from the societal perspective. DESIGN: Randomized trial of 20-week aquatic classes. Cost per quality-adjusted life year (QALY) gained was estimated using trial data. Sample size was based on 80% power to reject the null hypothesis that the cost/QALY gained would not exceed $50,000. SUBJECTS AND METHODS: Recruited 249 adults from Washington State aged 55 to 75 with a doctor-confirmed diagnosis of osteoarthritis to participate in aquatic classes. The Quality of Well-Being Scale (QWB) and Current Health Desirability Rating (CHDR) were used for economic evaluation, supplemented by the arthritis-specific Health Assessment Questionnaire (HAQ), Center for Epidemiologic Studies-Depression Scale (CES-D), and Perceived Quality of Life Scale (PQOL) collected at baseline and postclass. Outcome results applied to life expectancy tables were used to estimate QALYs. Use of health care facilities was assessed from diaries/questionnaires and Medicare reimbursement rates used to estimate costs. Nonparametric bootstrap sampling of costs/QALY ratios established the 95% CI around the estimates. RESULTS: Aquatic exercisers reported equal (QWB) or better (CHDR, HAQ, PQOL) health-related quality of life compared with controls. Outcomes improved with regular class attendance. Costs/QALY gained discounted at 3% were $205,186 using the QWB and $32,643 using the CHRD. CONCLUSION: Aquatic exercise exceeded $50,000 per QALY gained using the community-weighted outcome but fell below this arbitrary budget constraint when using the participant-weighted measure. Confidence intervals around these ratios suggested wide variability of cost effectiveness of aquatic exercise.     

Daily cost of an intensive care unit day: the contribution of mechanical ventilation

OBJECTIVE: To quantify the mean daily cost of intensive care, identify key factors associated with increased cost, and determine the incremental cost of mechanical ventilation during a day in the intensive care unit. DESIGN: Retrospective cohort analysis using data from NDCHealth's Hospital Patient Level Database. SETTING: A total of 253 geographically diverse U.S. hospitals. PATIENTS: The study included 51,009 patients >/=18 yrs of age admitted to an intensive care unit between October 1, 2002, and December 31, 2002. INTERVENTIONS: None. MEASUREMENTS AND MAIN RESULTS: Days of intensive care and mechanical ventilation were identified using billing data, and daily costs were calculated as the sum of daily charges multiplied by hospital-specific cost-to-charge ratios. Cost data are presented as mean (+/-sd). Incremental daily cost of mechanical ventilation was calculated using log-linear regression, adjusting for patient and hospital characteristics.Approximately 36% of identified patients were mechanically ventilated at some point during their intensive care unit stay. Mechanically ventilated patients were older (63.5 yrs vs. 61.7 yrs, p < .0001) and more likely to be male (56.1% vs. 51.8%, p < 0.0001), compared with patients who were not mechanically ventilated, and required mechanical ventilation for a mean duration of 5.6 days +/- 9.6. Mean intensive care unit cost and length of stay were 31,574 +/- 42,570 dollars and 14.4 days +/- 15.8 for patients requiring mechanical ventilation and 12,931 +/- 20,569 dollars and 8.5 days +/- 10.5 for those not requiring mechanical ventilation. Daily costs were greatest on intensive care unit day 1 (mechanical ventilation, 10,794 dollars; no mechanical ventilation, 6,667 dollars), decreased on day 2 (mechanical ventilation:, 4,796 dollars; no mechanical ventilation, 3,496 dollars), and became stable after day 3 (mechanical ventilation, 3,968 dollars; no mechanical ventilation, 3,184 dollars). Adjusting for patient and hospital characteristics, the mean incremental cost of mechanical ventilation in intensive care unit patients was 1,522 dollars per day (p < .001). CONCLUSIONS: Intensive care unit costs are highest during the first 2 days of admission, stabilizing at a lower level thereafter. Mechanical ventilation is associated with significantly higher daily costs for patients receiving treatment in the intensive care unit throughout their entire intensive care unit stay. Interventions that result in reduced intensive care unit length of stay and/or duration of mechanical ventilation could lead to substantial reductions in total inpatient cost.     

Cost-effectiveness of pegaptanib compared to photodynamic therapy with verteporfin and to standard care in the treatment of subfoveal wet age-related macular degeneration in Canada

BACKGROUND: Age-related macular degeneration (AMD) is characterized by loss of central vision and is the leading cause of blindness among persons over the age of 50 years in Canada. The wet form of AMD has 3 subtypes-occult, minimally classic, and predominantly classic. Photodynamic therapy (PDT) with verteporfin is indicated only for the category of predominantly classic wet AMD. Currently, there are no treatments available for the other AMD subtypes. Pegaptanib sodium was the first pharmacologic therapy approved in Canada for the treatment of subfoveal wet AMD regardless of subtype. OBJECTIVE: The aim of this study was to examine the cost-effectiveness of pegaptanib versus PDT with verteporfin and versus standard care for the treatment of subfoveal wet AMD in patients aged 65 years in Canada. METHODS: A Markov model based on visual acuity in the better-seeing eye was developed. Clinical efficacy was taken from the clinical trials. Costs of treatment, comorbidities (eg, depression, fractures, need for assisted living), vision rehabilitation, visual aids, and adverse events were considered. Costs, utilities, and mortality were estimated from data from the available published literature. Costs were reported in 2004 Canadian dollars, and costs and outcomes were discounted at 3% per annum. Lifetime costs, quality-adjusted life-years (QALYs), and vision years gained (VYGs) were estimated. Sensitivity analyses were performed to determine model robustness. RESULTS: Patients who received pegaptanib experienced more QALYs gained (4.17) and VYGs (3.83) compared with patients who received PDT (3.87 and 3.01, respectively) or standard care (3.96 and 3.26). Mean total costs per patient were greater in patients who received pegaptanib compared to those who received PDT or standard care ($20,016 vs $15,345 or $7669, respectively). The incremental cost per QALY in patients receiving pegaptanib compared to those receiving PDT was $49,052 and $59,039 for patients receiving pegaptanib versus standard care. The incremental cost per VYG was $20,401 and $21,559 with pegaptanib versus PDT and standard care, respectively. Sensitivity analyses found that the model was relatively robust to changes in various model parameters. CONCLUSION: The results of this analysis suggest that in Canada, pegaptanib is a cost-effective treatment for subfoveal wet AMD in elderly patients, regardless of lesion subtype, compared to PDT with verteporfin and to standard care.     

What does the value of modern medicine say about the $50,000 per quality-adjusted life-year decision rule?

BACKGROUND: In the United States, $50,000 per Quality-Adjusted Life-Year (QALY) is a decision rule that is often used to guide interpretation of cost-effectiveness analyses. However, many investigators have questioned the scientific basis of this rule, and it has not been updated. METHODS: We used 2 separate approaches to investigate whether the $50,000 per QALY rule is consistent with current resource allocation decisions. To infer a lower bound for the decision rule, we estimated the incremental cost-effectiveness of recent (2003) versus pre-"modern era" (1950) medical care in the United States. To infer an upper bound for the decision rule, we estimated the incremental cost-effectiveness of unsubsidized health insurance versus self-pay for nonelderly adults (ages 21-64) without health insurance. We discounted both costs and benefits, following recommendations of the Panel on Cost-Effectiveness in Health and Medicine. RESULTS: Our base case analyses suggest that plausible lower and upper bounds for a cost-effectiveness decision rule are $183,000 per life-year and $264,000 per life-year, respectively. Our sensitivity analyses widen the plausible range (between $95,000 per life-year saved and $264,000 per life-year saved when we considered only health care's impact on quantity of life, and between $109,000 per QALY saved and $297,000 per QALY saved when we considered health care's impact on quality as well as quantity of life) but it remained substantially higher than $50,000 per QALY. CONCLUSIONS: It is very unlikely that $50,000 per QALY is consistent with societal preferences in the United States.     

Cost-effectiveness of pegfilgrastim versus filgrastim primary prophylaxis in women with early-stage breast cancer receiving chemotherapy in the united states

Background: Prophylaxis with granulocyte colony-stimulating factor reduces the risk for febrile neutropenia (FN) in patients receiving myelosuppressive chemotherapy.Objective: We estimated the incremental cost-effectiveness of primary prophylaxis (starting in cycle 1 of chemotherapy) with pegfilgrastim versus filgrastim in women with early-stage breast cancer receiving myelosuppressive chemotherapy in the United States.Methods: A decision-analytic model was constructed from a health payer's perspective with a lifetime study horizon. The model considered direct medical costs and outcomes related to reduced FN and potential survival benefits due to reduced FN-related mortality and on-time receipt of full-dose chemotherapy. Sensitivity analyses were conducted.Results: Pegfilgrastim was cost-saving and more effective (ie, dominant strategy) than 11-day filgrastim. The incremental cost-effectiveness ratio (ICER) for pegfilgrastim versus 6-day filgrastim was $12,904 per FN episode avoided. Adding the survival benefit due to reduced FN mortality and receipt of optimal chemotherapy dose yielded an ICER of $31,511 per quality-adjusted life year (QALY) gained and $14,415 per QALY gained, respectively. The most influential factors included inpatient FN case-fatality rate, cost of pegfilgrastim and filgrastim, baseline probability of FN, relative risk for FN between filgrastim and pegfil-grastim, and cost of administration of filgrastim.Conclusion: Pegfilgrastim was cost-saving compared with 11-day filgrastim and cost-effective compared with 6-day filgrastim from a health payer's perspective for the primary prophylaxis of FN in these women with early-stage breast cancer receiving myelosuppressive chemotherapy.