Lifetime Quality-adjusted Life Years Research Articles

Lifetime cost-effectiveness analysis of ticagrelor in patients with acute coronary syndromes based on the PLATO trial: a Singapore healthcare perspective

Ticagrelor is a novel antiplatelet drug developed to reduce atherothrombosis. The PLATO trial compared ticagrelor and aspirin to clopidogrel and aspirin in patients with acute coronary syndromes (ACS). Ticagrelor was found to be superior in the primary composite endpoint of cardiovascular death, myocardial infarction or stroke, without increasing major bleeding events. The current study estimates the lifetime cost-effectiveness of ticagrelor relative to generic clopidogrel from a Singapore public healthcare perspective. This study used a two-part cost-effectiveness model. The first part was a 12-month decision tree (using PLATO trial data) to estimate the rates of major cardiovascular events, healthcare costs and health-related quality of life. The second part was a Markov model estimating lifetime quality-adjusted survival and costs conditional on events during the initial 12 months. Daily drug costs applied were SGD 1.05 (generic clopidogrel) and SGD 6.00 (ticagrelor). Cost per quality-adjusted life years (QALY) was estimated from a Singapore public healthcare perspective using life tables and short-term costs from Singapore, and long-term costs from South Korea. Deterministic and probabilistic sensitivity analyses were performed. Ticagrelor was associated with a lifetime QALY gain of 0.13, primarily driven by lower mortality. The resulting incremental cost per QALY gained was SGD 10,136.00. Probabilistic sensitivity analysis indicated that ticagrelor had a > 99% probability of being cost-effective, given the lower recommended WHO willingness-to-pay threshold of one GDP/capita per QALY. Based on PLATO trial data, one-year treatment with ticagrelor versus generic clopidogrel in patients with ACS, relative to WHO reference standards, is cost-effective from a Singapore public healthcare perspective.

Cemented, cementless, and hybrid prostheses for total hip replacement: cost effectiveness analysis

Objective To compare the cost effectiveness of the three most commonly chosen types of prosthesis for total hip replacement.Design Lifetime cost effectiveness model with parameters estimated from individual patient data...

Cost-Effectiveness Analysis of Boceprevir for the Treatment of Chronic Hepatitis C Virus Genotype 1 Infection in Portugal

The recent approval of two protease inhibitors, boceprevir and telaprevir, is likely to change the management of chronic hepatitis C virus (HCV) genotype 1 infection. We evaluated the long-term clinical outcomes and the cost effectiveness of therapeutic strategies using boceprevir with peginterferon plus ribavirin (PR) in comparison with PR alone for treating HCV genotype 1 infection in Portugal. A Markov model was developed to project the expected lifetime costs and quality-adjusted life-years (QALYs) associated with PR alone and the treatment strategies outlined by the European Medicines Agency in the boceprevir summary of product characteristics. The boceprevir-based therapeutic strategies differ according to whether or not the patient was previously treated and whether or not the patient had compensated cirrhosis. The model simulated the experience of a series of cohorts of chronically HCV-infected patients (each defined by age, sex, race and fibrosis score). All treatment-related inputs were obtained from boceprevir clinical trials - SPRINT-2, RESPOND-2 and PROVIDE. Estimates of the natural history parameters and health state utilities were based on published studies. Portugal-specific annual direct costs of HCV health states were estimated by convening a panel of experts to derive health state resource use and multiplying the results by national unit costs. The model was developed from a healthcare system perspective with a timeframe corresponding to the remaining duration of the patients' lifetimes. Both future costs and QALYs were discounted at 5 %. To test the robustness of the conclusions, we conducted deterministic and probabilistic sensitivity analyses. In comparison with the treatment with PR alone, boceprevir-based regimens were projected to reduce the lifetime incidence of advanced liver disease, liver transplantation, and liver-related death by 45-51 % and increase life expectancy by 2.3-4.3 years. Although the addition of BOC increased treatment costs by €13,300-€19,700, the reduction of disease burden resulted in a decrease of €5,400-€9,000 in discounted health state costs and an increase of 0.68-1.23 in discounted QALYs per patient. The incremental cost-effectiveness ratios of the boceprevir-based regimens compared with PR among previously untreated and previously treated patients were €11,600/QALY and €8,700/QALY, respectively. The results were most sensitive to variations in sustained virologic response rates, discount rates and age at treatment. Adding boceprevir to PR was projected to reduce the number of liver complications and liver-related deaths, and to be cost effective in treating both previously untreated and treated patients.

Cost–utility analysis of oral deferasirox versus infusional deferoxamine in transfusion‐dependent β‐thalassemia patients

Deferasirox (DFX) is a novel iron chelator that has been shown to have similar efficacy and safety compared with deferoxamine (DFO) in patients with β-thalassemia. The aim of this study was to determine the cost utility of DFX versus DFO in β-thalassemia major patients from Iran's society perspective. A Markov model has been developed to determine lifetime cost and quality-adjusted life-years (QALYs) of patients. To estimate the annual cost of each method, a cross-sectional study was conducted among two groups of patients who received DFO and DFX (n = 100 and n = 45, respectively). Also a time trade-off method was used to estimate the utility of two strategies. Finally a one-way and probabilistic sensitivity analysis was conducted to examine the strength of the results. Our base-case analysis showed that estimated total lifetime costs per patient for DFX and DFO were 47,029 international dollar ($Int) and $Int143,522, respectively, while the estimated total discounted QALYs per person were 12.28 and 7.76, respectively. Calculated incremental cost-effectiveness ratio showed that DSX is a dominant therapy and its estimated lifetime net monetary benefit was $Int273,528. We conclude that the use of DFX instead of DFO represents a cost-effective use of resources for treatment of iron overload in patients with β-thalassemia from Iran's society perspective.

Lifetime QALY prioritarianism in priority setting

Two principles form the basis for much priority setting in health. According to the greater benefit principle, resources should be directed toward the intervention with the greater health benefit. According...

PCV28 Cost-Effectiveness of Presentation and Delayed Troponin Testing for Acute Myocardial Infarction

To estimate the cost-effectiveness of delayed troponin testing for myocardial infarction (MI), as recommended in current guidelines, compared to troponin testing and other biomarkers at presentation. We developed a decision analytic model to estimate the cost-effectiveness of diagnostic strategies for MI, measured as the incremental cost per quality-adjusted life year (QALY) gained by each strategy compared to the next most effective alternative. The model was applied to a hypothetical population of 1000 patients attending hospital with symptoms suggesting MI but a normal or non-diagnostic electrocardiogram (ECG) and no major co-morbidities requiring hospital treatment. Sensitivity and specificity of the strategies were estimated by meta-analysis of diagnostic cohort studies of presentation troponin T. The risk of reinfarction and death (with and without treatment) was determined using data from a study by Mills et al, Lifetime QALYs were estimated from life expectancy and corresponding annual utilities. The discounted life expectancy of patients with MI and MI with reinfarction was estimated from Polanczyk et al, while the utility of patients with MI was estimated from Ward et al. In all scenarios tested presentation high sensitivity troponin testing was the most effective strategy with an incremental cost-effectiveness ratio (ICER) below the £20,000/QALY threshold. Delayed troponin testing was only likely to be cost-effective if a discharge decision could be made as soon as a negative result was available and the £30,000/QALY threshold was used. Delayed troponin testing is unlikely to be cost-effective compared to high sensitivity troponin testing at presentation in most scenarios. Current guidelines recommending 10-12 hour troponin testing does not appear to promote cost-effective use of hospital resources, unless services are in place to allow rapid decision making once delayed test results are available.

Modeling the impact of the difference in cross-protection data between a human papillomavirus (HPV)-16/18 AS04-adjuvanted vaccine and a human papillomavirus (HPV)-6/11/16/18 vaccine in Canada

BackgroundIn Canada, two vaccines that have demonstrated high efficacy against infection with human papillomavirus (HPV) types −16 and −18 are available. The HPV-6/11/16/18 vaccine provides protection against genital warts (GW) while the HPV-16/18 vaccine may provide better protection against other oncogenic HPV types. In this analysis, the estimated clinical and economic benefit of each of these vaccines was compared in the Canadian setting.MethodsA Markov model of the natural history of HPV infection among women, cervical cancer (CC) and GW was used to estimate the impact of vaccinating a cohort of 100,000 12-year-old females on lifetime outcomes and healthcare system costs (no indirect benefit in males included). A budget impact model was used to estimate the impact of each vaccine by province.ResultsIn the base case, vaccination with the HPV-16/18 vaccine was predicted to prevent 48 additional CC cases, and 16 additional CC deaths, while vaccination with the HPV-6/11/16/18 vaccine was predicted to prevent 6,933 additional GW cases. Vaccination with the HPV-16/18 vaccine was estimated to save 1 additional discounted quality adjusted life year (QALY) at an overall lower lifetime cost to the healthcare system compared to the HPV-6/11/16/18 vaccine (assuming vaccine price parity). In sensitivity analyses, the HPV-6/11/16/18 vaccine was associated with greater QALYs saved when the cross-protection efficacy of the HPV-16/18 vaccine was reduced, or the burden of GW due to HPV-6/11 was increased. In most scenarios with price parity, the lifetime healthcare cost of the strategy with the HPV-16/18 vaccine was predicted to be lower than the HPV-6/11/16/18 vaccine. In the probabilistic sensitivity analyses, the HPV-16/18 vaccine provided more QALY benefit than the HPV-6/11/16/18 vaccine in 49.2% of scenarios, with lower relative lifetime costs in 83.5% of scenarios.ConclusionsOverall, the predicted lifetime healthcare costs and QALYs saved by implementing each of the vaccines are similar. Vaccination with the HPV-16/18 vaccine is expected to be associated with reduced CC disease morbidity and mortality compared to vaccination with the HPV-6/11/16/18 vaccine. Differences in these outcomes depend on the extent of cervical disease prevented by cross-protection and the burden of GW caused by HPV-6/11.

Lifetime Cost-Utility Analyses of Deferasirox in Beta-Thalassaemia Patients with Chronic Iron Overload

Regular blood transfusions for beta-thalassaemia patients lead to the accumulation of iron deposits in the body. In order to remove such deposits, iron chelation therapy is required. Subcutaneously administered deferoxamine has been the gold standard chelation therapy for over 40years. Deferasirox is a newer chelation therapy that is taken orally once daily. The objective of this study was to estimate the long-term costs and quality-adjusted life-years (QALYs) associated with deferoxamine and deferasirox in a cohort of transfusion-dependent beta-thalassaemia patients from a UK health service perspective. A 50-year annual cycle state transition model comprised three core health states: alive without cardiac complications, alive with cardiac complications, and dead, as well as representing other chronic complications of iron overload: diabetes, hypogonadism, hypoparathyroidism and hypothyroidism. The model was calibrated to identify sets of convergent input parameter values that predicted observed overall survival by mean lifetime compliance with chelation therapy. A pivotal non-inferiority trial informed the main estimates of the effectiveness of deferasirox, which were applied to the calibrated model. Using cost values for the year 2011, costs and utilities were summed over patients' lifetimes to estimate lifetime costs and QALY gains. Mean lifetime treatment costs for patients receiving deferoxamine were £70,000 higher than deferasirox. Drug acquisition costs were £100,000 higher for deferasirox, but administration costs associated with deferoxamine were £170,000 higher. Higher compliance associated with oral deferasirox administration led to fewer complications. Combined with the quality-of-life effects of an oral mode of administration, an average gain of 4.85QALYs for deferasirox was estimated. In the base case, deferasirox dominates deferoxamine, i.e., costs less and patients gain more QALYs. The key parameter is the proportion of deferoxamine patients using balloon infusers. Sensitivity analyses showed that even when the proportion of patients using balloon infusers is decreased from 79 to 25%, the incremental cost per QALY gained remains well under £20,000. Higher drug acquisition costs for deferasirox are offset by the avoidance of infusion-related equipment costs. Combined with health benefits derived from an oral mode of administration and improved compliance, deferasirox has a high probability of being a cost-effective intervention compared with deferoxamine.

Cost-effectiveness of alendronate for the treatment of osteopenic postmenopausal women in Japan

Many postmenopausal women have osteopenia, a condition characterized by loss of bone mineral density (BMD) that is not as severe as in osteoporosis. The objective of this study was to estimate the cost-effectiveness of alendronate to prevent fractures in osteopenic postmenopausal women without a history of fracture in Japan. An individual simulation model was developed to predict lifetime costs and quality-adjusted life years (QALYs) of 5 years of preventive alendronate therapy versus no preventive therapy. The risk of hip and vertebral fracture associated with age and BMD was derived from epidemiologic studies in Japan. We ran the model with different combinations of age (65, 70, and 75 years), BMD (70%, 75%, and 80% of young adult mean [YAM]), and additional clinical risk factors. For 70-year-old women with a BMD of 70% of the YAM having one of the following risk factors: a family history of hip fracture, high alcohol intake, or current smoking, the incremental cost-effectiveness ratio (ICER) of alendronate was $92,937, $126,251, and $129,067 per QALY, respectively. These results were sensitive to age, BMD, and number of clinical risk factors. Probabilistic sensitivity analysis for the base case showed that in the presence of one, two, and three risk factors, alendronate was cost-effective in 0.2% to 2.6%, 13.1% to 56.1%, and 99.1% of the simulations, respectively, if society is willing to pay $50,000 per QALY. Additional analysis indicated that alendronate can be a good value in osteopenic women if the 10-year probability for a osteoporotic hip or vertebral fracture is more than 26.2%. Our results indicate that whether to treat osteopenia with alendronate should be determined on the basis of age, BMD, and number of clinical risk factors in terms of cost-effectiveness.

Cost-Effectiveness of Truncated Therapy for Hepatitis C Based on Rapid Virologic Response

BackgroundShortened courses of treatment with pegylated interferon alfa and ribavirin for patients with hepatitis C virus infection who experience rapid virologic response can be effective in appropriately selected patients. The cost-effectiveness of truncated therapy is not known. ObjectiveTo assess the cost-effectiveness of response-guided therapy versus standard-duration therapy on the basis of best available evidence. MethodsWe developed a decision model for chronic hepatitis C virus infection representing two treatment strategies: 1) standard-duration therapy with pegylated interferon alfa and ribavirin for 48 weeks in patients with genotype 1 or 4 and for 24 weeks in patients with genotype 2 or 3 and 2) truncated therapy (i.e., 50% decrease in treatment duration) in patients with rapid virologic response. Patients for whom truncated therapy failed began standard-duration therapy guided by genotype. We used a Markov model to estimate lifetime costs and quality-adjusted life-years. ResultsIn the base-case analysis, mean lifetime costs were $46,623 ± $2,483 with standard-duration therapy and $42,354 ± $2,489 with truncated therapy. Mean lifetime quality-adjusted life-years were similar between the groups (17.1 ± 0.7 with standard therapy; 17.2 ± 0.7 with truncated therapy). Across model simulations, the probability of truncated therapy being economically dominant (i.e., both cost saving and more effective) was 78.6%. The results were consistent when we stratified the data by genotype. In one-way sensitivity analyses, the results were sensitive only to changes in treatment efficacy. ConclusionTruncated therapy based on rapid virologic response is likely to be cost saving for treatment-naive patients with chronic hepatitis C virus infection. Cost-effectiveness varied with small changes in relative treatment efficacy.

Cost-Effectiveness of Early Colectomy With Ileal Pouch-Anal Anastamosis Versus Standard Medical Therapy in Severe Ulcerative Colitis

Inflammatory bowel diseases are costly chronic gastrointestinal diseases. We aimed to determine whether immediate colectomy with ileal pouch-anal anastamosis (IPAA) after diagnosis of severe ulcerative colitis (UC) was cost-effective compared to the standard medical therapy. We created a Markov model simulating 2 cohorts of 21-year-old patients with severe UC, following them until 100 years of age or death, comparing early colectomy with IPAA strategy to the standard medical therapy strategy. Deterministic and probabilistic analyses were performed. Standard medical care accrued a discounted lifetime cost of $236,370 per patient. In contrast, early colectomy with IPAA accrued a discounted lifetime cost of $147,763 per patient. Lifetime quality-adjusted life-years gained (QALY-gained) for standard medical therapy was 20.78, while QALY-gained for early colectomy with IPAA was 20.72. The resulting incremental cost-effectiveness ratio (Δcosts/ΔQALY) was approximately $1.5 million per QALY-gained. Results were robust to one-way sensitivity analyses for all variables in the model. Quality-of-life after colectomy with IPAA was the most sensitive variable impacting cost-effectiveness. A low utility value of less than 0.7 after colectomy with IPAA was necessary for the colectomy with IPAA strategy to be cost-ineffective. Under the appropriate clinical settings, early colectomy with IPAA after diagnosis of severe UC reduces health care expenditures and provides comparable quality of life compared to exhaustive standard medical therapy.

Cost-effectiveness of treating acute coronary syndrome patients with ticagrelor for 12 months: results from the PLATO study

The efficacy and safety of ticagrelor vs. clopidogrel in patients with acute coronary syndromes (ACS) are well documented in the PLATelet inhibition and patient Outcomes trial (PLATO). The aim of this study was to assess the long-term cost-effectiveness of treating ACS patients for 12 months with ticagrelor compared with generic clopidogrel. Event rates, health-care costs, and health-related quality of life during 12 months of therapy with either ticagrelor or generic clopidogrel were estimated from PLATO. Beyond 12 months, quality-adjusted survival and costs were estimated conditional on whether a non-fatal myocardial infarction (MI), a non-fatal stroke, or no MI or stroke occurred during the 12 months of therapy. Lifetime costs, life expectancy, and quality-adjusted life years (QALYs) were estimated for both treatment strategies. Incremental cost-effectiveness ratios were presented from a health-care perspective in 2010 Euros (€) applying unit costs and life tables from a Swedish setting in the base-case analysis. Treatment with ticagrelor was associated with increased health-care costs of €362 and a QALY gain of 0.13 compared with generic clopidogrel, yielding a cost per QALY gained with ticagrelor of €2753. The cost per life year gained was €2372. The results were consistent in major subgroups. Sensitivity analyses showed a cost per QALY gained with ticagrelor of ∼€7300 under certain scenarios. Based on clinical and health-economic evidence from the PLATO study, treating ACS patients with ticagrelor for 12 months is associated with a cost per QALY below generally accepted thresholds for cost-effectiveness. ClinicalTrials.gov Identifier: NCT00391872.

Cost-Effectiveness of Expanded Newborn Screening in Texas

ObjectiveTexas House Bill 790 resulted in the expansion of the newborn screening panel from 7 disorders to 27 disorders. Implementation of this change began in 2007. The objective of this study was to estimate the incremental cost-effectiveness of the expanded newborn screening program compared with the previous standard screening in Texas. MethodsA Markov model (for a hypothetical cohort of Texas births in 2007) was constructed to compare lifetime costs and quality-adjusted life-years (QALYs) between the expanded newborn screening and preexpansion newborn screening. Estimates of costs, probabilities of sequelae, and utilities for disorder categories were obtained from a combination of Texas statistics, the literature, and expert opinion. A baseline discount rate of 3% was used for both costs and QALYs, with a range of 0% to 5%. Analyses were conducted from a payer's perspective, and so only direct medical cost estimates were included. ResultsThe lifetime incremental cost-effectiveness ratio for expanded versus preexpansion screening was about $11,560 per QALY. The results remained robust to both deterministic and probabilistic sensitivity analyses. ConclusionsExpanded newborn screening does result in additional expenses to the payer, but it also improves patient outcomes by preventing avoidable morbidity and mortality. The screened population benefits from greater QALYs as compared with the unscreened population. Overall, expanded newborn screening in Texas was estimated to be a cost-effective option as compared with unexpanded newborn screening.

Comparison of lifetime incremental cost:utility ratios of surgery relative to failed medical management for the treatment of hip, knee and spine osteoarthritis modelled using 2-year postsurgical values

Demand for surgery to treat osteoarthritis (OA) of the hip, knee and spine has risen dramatically. Whereas total hip (THA) and total knee arthroplasty (TKA) have been widely accepted as cost-effective, spine surgeries (decompression, decompression with fusion) to treat degenerative conditions remain underfunded compared with other surgeries. An incremental cost-utility analysis comparing decompression and decompression with fusion to THA and TKA, from the perspective of the provincial health insurance system, was based on an observational matched-cohort study of prospectively collected outcomes and retrospectively collected costs. Patient outcomes were measured using short-form (SF)-36 surveys over a 2-year follow-up period. Utility was modelled over the lifetime, and quality-adjusted life years (QALYs) were determined. We calculated the incremental cost per QALY gained by estimating mean incremental lifetime costs and QALYs of surgery compared with medical management of each diagnosis group after discounting costs and QALYs at 3%. Sensitivity analyses were also conducted. The lifetime incremental cost:utility ratios (ICURs) discounted at 3% were $5321 per QALY for THA, $11,275 per QALY for TKA, $2307 per QALY for spinal decompression and $7153 per QALY for spinal decompression with fusion. The sensitivity analyses did not alter the ranking of the lifetime ICURs. In appropriately selected patients with leg-dominant symptoms secondary to focal lumbar spinal stenosis who have failed medical management, the lifetime ICUR for surgical treatment of.

Cost-effectiveness of zoledronic acid (ZOL) in postmenopausal women with early breast cancer receiving adjuvant letrozole.

553 Background: In the ZO-FAST trial, postmenopausal women with early breast cancer and a bone mineral density (BMD) T-score ≥ –2 and receiving adjuvant letrozole (2.5 mg/day) were randomized to either immediate ZOL (4 mg/6 months) treatment (upfront ZOL) or to the same therapy but only when BMD T-score decreased to < –2 or fracture occurrence (delayed ZOL). After 60 months, upfront ZOL increased both BMD and disease-free survival (P < .05) relative to delayed ZOL. The present analysis assessed, from a US payer perspective, the cost effectiveness of upfront ZOL vs delayed ZOL in this population. Methods: A Markov state-transition model was developed to estimate the lifetime costs and quality-adjusted life-years (QALYs) for a hypothetical cohort of postmenopausal women with early breast cancer receiving letrozole with upfront or delayed ZOL. Consistent with ZO-FAST, patients were 57 years of age and breast cancer recurrence-free at baseline. Patients could progress over time to Local Recurrence, Contra-lateral Tumor, Distant Recurrence, or Death. Transition probabilities were derived from ZO-FAST, supplemented with literature. Costs and utilities were literature based. All outcomes were discounted 3% per year. Results: Compared to delayed ZOL, upfront ZOL resulted in better overall survival, disease-free survival, and QALYs, but at a higher cost (Table). In the base case, the incremental cost/QALY gained with upfront vs delayed ZOL was $7,967. In > 95% of 1,000 probabilistic sensitivity analysis runs, upfront ZOL costs less than $38,376/QALY gained. Conclusions: Upfront ZOL may increase survival and QALY and, at a cost per QALY well under the $50,000/QALY threshold, is very cost-effective in this population. [Table: see text]

Cost‐Effectiveness of an Electronic Medical Record Based Clinical Decision Support System

Medical groups have invested billions of dollars in electronic medical records (EMRs), but few studies have examined the cost-effectiveness of EMR-based clinical decision support (CDS). This study examined the cost-effectiveness of EMR-based CDS for adults with diabetes from the perspective of the health care system. Clinical outcome and cost data from a randomized clinical trial of EMR-based CDS were used as inputs into a diabetes simulation model. The simulation cohort included 1,092 patients with diabetes with A1c above goal at baseline. The United Kingdom Prospective Diabetes Study Outcomes Model, a validated simulation model of diabetes, was used to evaluate remaining life years, quality-adjusted life years (QALYs), and health care costs over patient lifetimes (40-year time horizon) from the health system perspective. Patients in the intervention group had significantly lowered A1c (0.26 percent, p=.014) relative to patients in the control arm. Intervention costs were $120 (SE=45) per patient in the first year and $76 (SE=45) per patient in the following years. In the base case analysis, EMR-based CDS increased lifetime QALYs by 0.04 (SE=0.01) and increased lifetime costs by $112 (SE=660), resulting in an incremental cost-effectiveness ratio of $3,017 per QALY. The cost-effectiveness of EMR-based CDS persisted in one-way, two-way, and probabilistic sensitivity analyses. Widespread adoption of sophisticated EMR-based CDS has the potential to modestly improve the quality of care for patients with chronic conditions without substantially increasing costs to the health care system.

A pharmaco-economic evaluation of deferasirox for treating patients with iron overload caused by transfusion-dependent thalassemia in Taiwan

The newly available iron chelator deferasirox (Exjade, Novartis) is expected to provide better long-term clinical outcomes and improved quality of life for patients with thalassemia than its predecessor, deferoxamine (Desferal, Novartis), because of its oral tablet form. We used the Markov model to estimate total additional lifetime costs and quality-adjusted life years (QALYs) gained with deferasirox versus deferoxamine in patients with transfusion-dependent thalassemia. Patients were assumed to be 2 years of age at initiation of chelation therapy. Clinical outcomes in terms of morbidity and mortality from associated complications and life expectancy for the study population were estimated using the databases of the Bureau of National Health Insurance and the Health and Vital Statistics of Taiwan. Treatment costs were based on analyses of health insurance claims for patients with transfusion-dependent thalassemia. Utilities in terms of quality of life were also included in the model. The incremental cost-utility ratio of deferasirox versus deferoxamine was defined by the ratio of the difference in expected lifetime costs to the difference in QALYs. One-way sensitivity analyses were performed to examine the robustness of the results to key assumptions. Patients treated with deferasirox are expected to experience a lower incidence of associated complications and obtain 2.3 QALYs (discounted) at an additional lifetime cost of US$36,291 per patient (US$15,596 per QALY). Sensitivity analyses showed that the unit drug cost of deferasirox had the greatest impact on the incremental cost-utility ratio. In addition, the incremental cost-utility ratio will increase by delaying the starting age (2 years of age in our study) of chelation therapy. Compared with infusional deferoxamine, oral deferasirox improved clinical outcomes and quality of life in terms of iron chelation in transfusion-dependent patients with thalassemia at a reasonable cost from a healthcare perspective.

Cost-Effectiveness of Varenicline Compared to Placebo as an Aid to Smoking Cessation in Patients with Cardiovascular Disease

Background: Varenicline has been shown to be an effective and well-tolerated intervention for smoking cessation in smokers with stable cardiovascular disease (CVD). The objective of this study was to evaluate the cost- effectiveness of varenicline and counselling compared to placebo and counselling based on extrapolating the results of a 52-week randomised controlled trial. Method: A Markov model was developed to simulate the outcomes of smokers with CVD. Outcomes included major forms of CVD (e.g. Coronary Heart Disease (CHD)) and other key chronic conditions attributable to smoking (e.g. Lung Cancer). The lifetime costs, Life-Years gained (LY) and Quality-Adjusted Life Years (QALY) were evaluated from a payer's perspective in Austria, Hungary and Germany. Additional analyses included a societal perspective, disease sub- group, and both one-way and probabilistic sensitivity analyses (PSA). Costs and outcomes were discounted at 3% per year. Results: From a payer's perspective, the incremental cost-effectiveness ratio per LY (or QALY) gained was � 4 112 (� 5 278), � 2 507 (� 3 183), and � 4 567 (� 5 867) for Austria, Hungary and Germany, respectively. Sub-group analyses demonstrated that smoking cessation in patients with CHD was more cost-effective than in patients with baseline stroke or peripheral vascular disease. Sensitivity analysis demonstrated that outcomes were not sensitive to modelling assumptions. Varenicline was less costly and more effective than placebo from a societal perspective, and more costly and more effective than placebo in all iterations of the payer PSA. Conclusions: Varenicline is a cost-effective adjunct to counselling compared to counselling alone in patients with stable CVD.

Clinical outcomes, quality of life, and costs associated with implantation of vagus nerve stimulation therapy in pediatric patients with drug-resistant epilepsy

BackgroundVNS (Vagus Nerve Stimulation Therapy) is approved in the USA to treat refractory epilepsy as adjunctive to antiepileptic drugs (AEDs) in patients ≥12 years with complex partial seizures. AimsTo evaluate clinical outcomes, quality-adjusted life years (QALY), and costs associated with VNS in pediatric patients with drug-resistant epilepsy in a real-world setting. MethodsA retrospective analysis was conducted using Medicaid data (USA). Patients had ≥1 neurologist visits with epilepsy diagnosis (ICD-9 345.xx, 780.3x), ≥1 procedure claims for VNS implantation, ≥1 AEDs, ≥6-months of Pre- and Post-VNS continuous enrollment. Pre-VNS period was 6-months and Post-VNS period extended from implantation until device removal, death, Medicaid disenrollment, or study end (up to 3 years). Incidence rate ratios (IRR) and costs ($2010) were estimated. QALYs were estimated using number of seizure-related events. ResultsFor patients 1–11 years old (N = 238), hospitalizations and emergency room visits were reduced Post-VNS vs. Pre-VNS (adjusted IRR = 0.73 [95% CI: 0.61–0.88] and 0.74 [95% CI: 0.65–0.83], respectively). Average total healthcare costs were lower Post-VNS vs. Pre-VNS ($18,437 vs. $18,839 quarterly [adjusted p = 0.052]). For patients 12–17 years old (N = 207), hospitalizations and status epilepticus events were reduced Post-VNS vs. Pre-VNS (adjusted IRR = 0.43 [95% CI: 0.34–0.54] and 0.25 [95% CI: 0.16–0.39], respectively). Average total healthcare costs were lower Post-VNS vs. Pre-VNS period ($14,546 vs. $19,695 quarterly [adjusted p = 0.002]). Lifetime QALY gain after VNS was 5.96 (patients 1–11 years) and 4.82 years (patients 12–17 years). ConclusionsVNS in pediatric patients is associated with decreased resource use and epilepsy-related events, cost savings, and QALY gain.

Cost-Effectiveness of Multiple Sclerosis Disease-Modifying Therapies: A Systematic Review of the Literature

Objective. To provide a current and comprehensive understanding of the cost-effectiveness of DMTs for the treatment of MS by quantitatively evaluating the quality of recent cost-effectiveness studies and exploring how the field has progressed from past recommendations. Methods. We assessed the quality of studies that met our systematic literature search criteria using the Quality of Health Economic Studies validated instrument. Results. Of the 82 studies that met our initial search criteria, we included 22 in this review. Four studies (18%) achieved quality category 2, three studies (14%) achieved quality category 3, and 15 studies (68%) achieved the highest quality category 4. 91% of studies were simulation models. 13 studies (59%) had quality-adjusted life years (QALYs) as the primary outcome measure, included a societal perspective in the analysis, and utilized time horizons of 10 years to lifetime. Conclusions. To continue to improve the cost-effectiveness evidence of DMTs, we recommend: lifetime horizons, societal perspectives, and QALYs; supplemental evidence with shorter horizons, payer perspectives, and clinical outcomes to inform multiple decision makers; development of modeling and input standards for comparability; head-to-head RCTs between DMTs and long-term prospective studies; and comprehensive cost-effectiveness studies that compare all appropriate DMTs.