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Introduction: Recent evidence indicates that Maternal Supplementation with Long-Chain n-3 Fatty Acids During Pregnancy Substantially Mitigates Offspring's Asthma. Adding information regarding its cost-utility will undoubtedly allow its adoption, or not, in clinical practice guidelines. This research aimed to determine the cost-utility of LCPUFA supplementation in the third trimester of pregnancy to reduce the risk of wheezing and asthma in infants in Colombia. Methods: A Markov model was formulated to estimate the cost and quality-adjusted life-years (QALYs) attributed to individuals with severe asthma in Colombia, with a time horizon of five years and a cycle length of two weeks. Probabilistic sensitivity analysis and a value of information (VOI) analysis were conducted to evaluate the uncertainties in the case base. Cost-utility was assessed at a willingness-to-pay (WTP) value of US$5180. All costs were adjusted to 2021 with a 5% annual discounting rate for cost and QALYs. Results: The mean incremental cost of LCPUFA supplementation versus no supplementation was US-43.65. The mean incremental benefit of LCPUFA supplementation versus no supplementation was 0.074 QALY. The incremental cost-utility ratio was estimated at US$590.68 per QALY. The outcomes derived from our primary analysis remained robust when subjected to variations in all underlying assumptions and parameter values. Conclusion: Supplementation strategy supplementation with long-chain n-3 fatty acids during pregnancy is cost-effective in reducing the risk of developing asthma during childhood in Colombia. [ABSTRACT FROM AUTHOR]

ORIENT-16, a phase III clinical trial conducted at 62 hospitals in China, reported that add-on sintilimab (Sin) to chemotherapy (Chemo) had favorable efficacy (p < 0.05) for patients with advanced HER2-negative gastric or gastroesophageal junction cancer (GC/GEJC). This study aimed to evaluate the cost-utility of the Sin+Chemo based on results of ORIENT-16 from the perspective of Chinese healthcare payers. A three-state partitioned survival model was developed to simulate the 10-year life expectancy and total healthcare costs for patients with advanced HER2-negative GC/GEJC. Primary measure outcomes were: cost, quality-adjusted life-years (QALYs), and incremental cost-utility ratios (ICURs). Sensitivity/scenario analyses were conducted to assess the model robustness. In all patients, Sin+Chemo vs Chemo increased costs by $6,472, additionally providing 0.61 QALYs, resulting in an ICUR of $10,610/QALY. While, in PD-L1 combined positive score ≥ 5 cohort, the ICUR was $9,738/QALYs. The ICUR was most sensitive to the utility of progression-free survival. The probabilistic sensitivity analysis showed that add-on Sin had a 100% probability of being cost-effective at a willingness-to-pay threshold of $18,625/QALY gained. Sin+Chemo is a cost-effective first-line treatment option for advanced HER2-negative GC/GEJC in China. ORIENT-16, , identifier is NCT03745170. [ABSTRACT FROM AUTHOR]

Antineutrophil cytoplasmic antibody-associated vasculitis (AAV) are rare autoimmune diseases characterized by inflammation of blood vessels. This study aimed to assess the cost-utility of avacopan in combination with rituximab (RTX) or cyclophosphamide (CYC) compared with glucocorticoids (GC) for the treatment of severe, active AAV in Spain. A 9-state Markov model was designed to reflect the induction of remission and sustained remission of AAV over a lifetime horizon. Clinical data and utility values were mainly obtained from the ADVOCATE trial, and costs (€ 2022) were sourced from national databases. Quality-adjusted life years (QALYs), and incremental cost-utility ratio (ICUR) were evaluated. An annual discount rate of 3% was applied. Sensitivity analyses were performed to examine the robustness of the results. Avacopan yielded an increase in effectiveness (6.52 vs. 6.17 QALYs) and costs (€16,009) compared to GC, resulting in an ICUR of €45,638 per additional QALY gained. Avacopan was associated with a lower incidence of end-stage renal disease (ESRD), relapse and hospitalization-related adverse events. Sensitivity analyses suggested that the model outputs were robust and that the progression to ESRD was a driver of ICUR. Avacopan is a cost-effective option for patients with severe, active AAV compared to GC in Spain. [ABSTRACT FROM AUTHOR]

Clinical outcomes in chronic lymphocytic leukemia (CLL) have improved with targeted therapy, including Bruton tyrosine kinase inhibitors such as acalabrutinib. A semi-Markov model with three health states (progression-free, progressed disease, and death) estimated cost per quality-adjusted life-year (QALY) gained for acalabrutinib ± obinutuzumab vs chlorambucil + obinutuzumab in treatment-naive CLL (based on ELEVATE-TN). The model used direct costs and resource utilization from the US Medicare perspective and utility values sourced from literature. Sensitivity analyses tested the robustness of the model. Over a 30-year lifetime horizon, the model base case analysis suggested that acalabrutinib monotherapy had an incremental cost of $206,329 and 2.52 QALYs gained versus chlorambucil + obinutuzumab, resulting in an incremental cost-effectiveness ratio (ICER) of $81,960/QALY. Acalabrutinib + obinutuzumab had an incremental cost of $423,747 and 2.79 QALYs gained (ICER: $152,153/QALY). Probabilistic sensitivity analysis showed the probability of acalabrutinib monotherapy being cost-effective as 59% to 73% at a $100,000-to-150,000/QALY willingness-to-pay threshold; the probability of acalabrutinib + obinutuzumab being cost-effective ranged from 34% to 51%. Although the analysis is limited by uncertainty in postprogression survival outcomes, acalabrutinib monotherapy is likely cost-effective vs chlorambucil + obinutuzumab in treatment-naive CLL in the US Medicare setting. [ABSTRACT FROM AUTHOR]

STN1013001 is an innovative latanoprost cationic emulsion for open-angle glaucoma/ocular hypertension (OAG/OHT) and ocular surface disease (OSD). A 5-year, 7 health states, 1-year cycle early Markov model-supported cost-utility analysis (CUA) of STN1013001 vs. other latanoprost formulations (Latanoprost) followed the Italian National Health Service (INHS) perspective. One-way, probabilistic and scenario sensitivity analyses tested the uncertainty of the baseline results. Value of information analysis (VOIA) investigated the potential cost-effectiveness of collecting further evidence. Over 5 years, the Markov model-supported CUA predicts STN1013001 to be potentially highly cost-effective vs. Latanoprost (+€57.60 cost at €2020 values; +0.089 Quality-Adjusted Life Years). The Incremental Cost-Utility Ratio (€647.65) falls well below the lower limit of the acceptability range proposed for Italy (€25,000-€40,000). Sensitivity analyses confirmed the robustness of the baseline findings. VOIA highlighted that further information might only be cost-effective for OAG/OHT utilities and OSD-related disutility. STN1013001 is potentially highly cost-effective and strongly dominant vs. Latanoprost for OAG/OHT+OSD patients from the INHS perspective. These findings should be re-assessed using the data from the ongoing Phase III trial (NCT04133311) comparing the efficacy and safety of STN1013001 vs. Latanoprost and with future real-world CUAs upon the availability of STN1013001 on the Italian market. [ABSTRACT FROM AUTHOR]

To analyse the cost-effectiveness (CE) of implementing different newborn hearing screening protocols in a low- to middle-income country. A decision analytical model with a 78-year time horizon. Direct medical, direct non-medical and indirect costs were collected from 126 subjects in southern Thailand. Various protocols involving universal newborn hearing screening (UNHS) and targeted newborn hearing screening (TNHS), using two technologies, namely automated otoacoustic emissions (aOAEs) and automated auditory brainstem responses (aABRs), were evaluated. Incremental cost-effectiveness ratios (ICERs) were calculated for all protocols in United States dollars (US$)/quality-adjusted life year (QALY) gained. Also, probabilistic sensitivity analyses with 1000 trials for each specific protocol were performed. The ICERs of UNHS with aOAE, UNHS with aABR, TNHS with aABR and UNHS with optimised baseline parameters were 3702, 3545, 1545 and 2483 US$/QALY gained, respectively. With the CE threshold of 5000 US$/QALY gained, the chances of ICERs to be cost-effective for UNHS with aOAE, UNHS with aABR, TNHS with aABR and UNHS with optimised baseline parameters were 72, 77, 93 and 94%, respectively. All screening protocols were considered as cost-effective, and a very high chance of being cost-effective for UNHS could be achieved when certain baseline parameters were optimised. [ABSTRACT FROM AUTHOR]

The advantage of tenecteplase (TNK) over alteplase (ALT) in managing acute ischemic stroke (AIS) has been reported, but the cost-effectiveness of these two strategies has not received as much attention. The objective of this study was to compare TNK and ALT for the management of AIS patients in Iran in terms of cost-effectiveness. This study was carried out from the payer's perspective in Iran, with a lifetime horizon. A full economic evaluation model was designed as a decision tree and a Markov model. After defining different Markov states, each health state was assigned a utility value, and quality-adjusted life year (QALY) was estimated using that value. The incremental cost-effectiveness ratio (ICER) was ultimately used for evaluating the comparative cost-effectiveness. Both deterministic and probabilistic sensitivity analyses were carried out. Compared to ALT, TNK can save approximately 4333.81 USD, and is able to increase one unit of QALY while saving approximately 17,450.29 USD. So, Base-case results showed that TNK strongly dominates ALT. Moreover, the base case results were strongly confirmed by deterministic and probabilistic sensitivity analysis. Base-case and sensitivity analysis showed that TNK is the dominant strategy compared to ALT for the management of AIS patients. [ABSTRACT FROM AUTHOR]

To assess the cost-effectiveness of tezepelumab as add-on maintenance therapy compared with standard of care (SoC) for the treatment of patients with severe asthma in Canada. A cost utility analysis was conducted using a Markov cohort model with five health states ("controlled asthma", "uncontrolled asthma", "previously controlled asthma with exacerbation", "previously uncontrolled asthma with exacerbation", and "death"). Tezepelumab plus SoC was compared to SoC (high-dose inhaled corticosteroids plus long-acting beta agonist) using efficacy estimates derived from the NAVIGATOR (NCT03347279) and SOURCE (NCT03406078) trials. The model included the costs of therapy, administration, resource use for disease management, and adverse events. Utility estimates were calculated using a mixed-effects regression analysis of the NAVIGATOR and SOURCE trials. A Canadian public payer perspective was used with a 50-year time horizon, a 1.5% annual discount rate, and the base case analysis was conducted probabilistically. A key scenario analysis assessed the cost-effectiveness of tezepelumab compared with currently reimbursed biologics informed by an indirect treatment comparison. The base case analysis suggested that tezepelumab plus SoC was associated with a quality-adjusted life-year (QALY) gain of 1.077 compared with SoC alone at an incremental cost of $207,101 (2022 Canadian dollars), resulting in an incremental cost-utility ratio of $192,357/QALY. The key scenario analysis demonstrated that tezepelumab was dominant against all currently reimbursed biologics, with higher incremental QALYs (ranging from 0.062 to 0.407) and lower incremental costs (ranging from −$6,878 to −$1,974). Additionally, when compared against currently reimbursed biologics in Canada, tezepelumab had the highest probability of being cost-effective across all willingness-to-pay (WTP) thresholds. Tezepelumab provided additional life years and QALYs at additional cost compared with SoC in Canada. In addition, tezepelumab dominated (i.e. more effective, less costly) the other currently reimbursed biologics. [ABSTRACT FROM AUTHOR]

Paediatric growth hormone deficiency (pGHD) manifests as growth failure associated with inadequate growth hormone (GH) production. Daily injections of recombinant human GH (dGH) [somatropin] is the current standard of care, which has been shown to be well tolerated and effective, but associated with suboptimal adherence, leading to reduced effectiveness. Somatrogon, a once-weekly injectable long-acting human GH, has demonstrated clinical non-inferiority and significantly lower life interference (i.e. treatment burden) vs. somatropin in two Phase 3 studies. This work evaluated cost-effectiveness and cost-utility of somatrogon vs dGHs from an Irish payer perspective. A Markov model was developed for patients starting somatrogon or dGHs treatment at 3–12 years and continuing up to achievement of near adult height (NAH), with growth driven by trial-based height velocity (HV) and treatment-specific adherence. Patients could discontinue treatment at the end of Year 1 (4%). DGH adherence (95.3%–65% over treatment duration) and adherence-growth relationship were based on published evidence. Higher Year 1 adherence of 4%, tapering over time, for somatrogon vs. dGHs was based on clinical consultation. Treatment costs, monitoring costs and costs due to different wastage types (device setting and adherence) were sourced from local data. Health utilities based on height and injection frequency were derived from published literature. Scenario analysis, deterministic and probabilistic sensitivity analysis were performed. Somatrogon treatment led to 1.87–3.66 cm greater NAH gain and 0.21–0.50 higher quality adjusted life years (QALYs) vs. dGHs, across the base case and scenarios evaluated. Somatrogon treatment was associated with cost savings of €5,699–€21,974 and lower cost per cm gained vs. dGHs (€197–€527), per patient. Somatrogon was cost-effective vs. dGHs, with the result consistent across the sensitivity analyses conducted. Somatrogon weekly injections were estimated to result in higher NAH, higher QALYs, lower overall costs and lower costs per cm gained than dGHs, in pGHD. [ABSTRACT FROM AUTHOR]

Despite the availability of several commercial rapid diagnostic tests (RDTs) developed to detect typhoid fever, the cost-effectiveness in resource-limited settings is unclear. This review aimed to explore the literature on typhoid economic evaluations in order to assess the cost-effectiveness of using typhoid RDTs in resource-limited settings. A systematic review was conducted focusing on the identification of economic evaluations of typhoid RDTs to diagnose patients with suspected typhoid fever. Two studies were identified and included for narrative synthesis. Whilst highlighting a gap in the published literature, this review shows the use of typhoid RDTs to potentially be cost-effective in resource-limited settings. Factors that appeared as significant in determining cost-effectiveness related to test characteristics (such as sensitivity, specificity, and cost) and the prevalence of typhoid fever and should factor into any future evaluations. Concerted effort is needed in resource-limited settings with regard to medical device regulation to ensure that clinically effective and cost-effective typhoid RDTs are widely available and introduced into clinical practice. Typhoid modeling (with respect to typhoid testing and treatment strategies) represents an understudied area and further work is needed. [ABSTRACT FROM AUTHOR]

Background: TNF inhibitors are costly drugs supplied generally on health systems or private insurances. Performance analysis is essential to verify the results achieved by health technologies in these systems. The objective of the study was to compare the two most used biological drugs for the treatment of psoriatic arthritis (PsA) in Brazil. Methods: A cost-utility analysis was built using a Markov model, with a five-year time horizon, a discount rate of 5%, and from the perspective of the Unified Health System. Deterministic and probabilistic sensitivity analyses were performed. Results: Etanercept was the most cost-effective drug. Adalimumab became the most cost-effective drug in one of the four analysis scenarios with a willingness to pay from one gross domestic product per capita. The deterministic sensitivity analysis identified that the cost parameters had the greatest impact on the most effective drug. The probabilistic sensitivity analysis indicated that etanercept is the drug most likely to be cost-effective. Conclusion: The difference between the drugs in terms of utility was minimal and the costs were the main factor that impacted the cost-utility ratio, which points to the benefits of price renegotiation for the efficient allocation of resources in the health system. [ABSTRACT FROM AUTHOR]

A new 20-valent pneumococcal conjugate vaccine (PCV20) provides protection against 20 pneumococcal serotypes. The vaccine has the potential to decrease the impact of pneumococcal diseases in society and to increase health among vulnerable persons. This study investigates the cost-effectiveness of vaccinating Danish adults in different age groups and risk of pneumococcal disease with PCV20 compared to the 23-valent pneumococcal polysaccharide vaccine (PPV23) – either as PCV20 compared to PPV23 or as PPV23 followed by PCV20 compared to PPV23. A Markov model adapted to the Danish setting was developed to estimate clinical outcomes and costs of vaccinating the Danish population in specific age and risk groups. The model used a restricted societal perspective and estimated outcomes and costs using a lifetime time horizon. To estimate the clinical outcomes and costs, inputs on vaccine effectiveness and waning were retrieved from other studies whereas data on risk groups, coverage and costs were based on real-world data. The results showed that in all scenarios the incidence and mortality of pneumococcal disease were reduced when vaccinating with PCV20, resulting in lower costs. For the vaccine target group of adults aged ≥18 years at moderate or high risk and all adults aged ≥65 years both in the case of PPV23+PCV20 compared to PPV23 and in case of PCV20 compared to PPV23 vaccination with PCV20 was found to be a dominant strategy gaining 1,350 or 5,821 quality-adjusted life years (QALYs), respectively, and reducing total costs by 60 or 396 million EUR, respectively, as compared to PPV23 vaccination alone. Similar results of dominant PCV20 strategy were found for other age and risk group comparisons. Both deterministic and probabilistic sensitivity analyses confirmed the results being robust to changes in input parameters and applied assumptions. Like other modelling studies, this analysis has limitations such as lack of detailed data for some inputs. Vaccination with PCV20 reduced the incidence and mortality of pneumococcal diseases in Danish adults compared to PPV23. This reduction has the potential to reduce the financial burden related to managing diseases while also increasing public health. [ABSTRACT FROM AUTHOR]

We evaluated the availability of transcatheter aortic valve replacement (TAVR) to determine its value across all severe symptomatic aortic stenosis (SSAS) patients, especially those untreated because of concerns regarding invasive surgical AVR (SAVR) and its impact on active aging. We performed payer perspective cost-utility analysis (CUA) and societal perspective cost-benefit analysis (CBA). The CBA's benefit measure is active time: salaried labor, unpaid work, and active leisure. The study population is a cohort of US elderly SSAS patients. We compared a "TAVR available" scenario in which SSAS patients distribute themselves across TAVR, SAVR, and medical management (MM); and a "TAVR not available" scenario with only SAVR and MM. We structured each scenario with a decision-tree model of SSAS patient treatment allocation. We measured the association between health and active time in the US Health and Retirement Study and used this association to impute active time to SSAS patients given their health. The incremental cost-effectiveness ratio (ICER) and rate of return (RoR) of TAVR availability were $8,533 and 395%, respectively. CUA net monetary benefits (NMB) were $212,199 per patient and $43.4 billion population-wide. CBA NMB were $50,530 per patient and $10.3 billion population-wide. Among study limitations were scarcity of evidence regarding key parameters and the lack of long-term survival, health utility, and treatment cost data. Our analysis did not account for TAVR durability, retreatments, and valve-in-valve treatments. Across risk-, age-, and treatment-eligibility groups, TAVR is the economically optimal treatment choice. It represents strong value-for-money per patient and population-wide. The vast majority of TAVR value involves raising treatment uptake among the untreated. Aortic stenosis (AS) is a common and lethal heart disease. Surgical treatment has long been available, but its invasiveness limits uptake. More recently, transcatheter aortic valve replacement (TAVR) has emerged as a treatment alternative. Its minimal invasiveness has significantly increased treatment rates, but economic evaluations omit this benefit, risking undervaluation. We evaluated TAVR in elderly US severe symptomatic AS patients, using payer perspective cost-utility analysis (CUA) and societal perspective cost-benefit analysis (CBA). Both CUA and CBA incorporated TAVR's impact on treatment rates. Given patient preferences for treatment options promoting active aging, our CBA used the value of active time as a benefit measure. We found that CUA/CBA net monetary benefits are $212,199/$50,530 per patient. Across risk-, age-, and treatment-eligibility groups, TAVR is the economically optimal treatment choice over surgery and medical management. It represents strong value-for-money per patient and population-wide. Increased treatment uptake accounts for the vast share of TAVR's value. [ABSTRACT FROM AUTHOR]

Peristomal skin complications (PSCs) and leakages are major issues for people living with a stoma. The purpose of this study is to understand how these stoma-linked complications impact health-related quality of life (HRQoL) in a UK population. The study used time trade-off (TTO) methodology to quantify health state utilities associated with two stoma-related complications: PSC and leakages. Respondents assessed 10 different health states with different PSC severity levels (no, mild, moderate or severe PSC) and frequencies of leakage events (2, 12 or 48 leakages onto clothes per year, and no leakage due to a digital solution). The average disutility value for each health state was also assessed. The study was conducted via a web-based survey in the UK adult general population. The analysis included 758 respondents. Respondents considered living with a stoma with no PSC to be more favorable than the other health states. Severe pain, itching and/or burning (PIB) was associated with the largest disutility compared to no PSC. The disutility (0–1 scale) compared to no PSC was 0.287 (p <.0001), 0.106 (p <.0001) and 0.025 (p=.0005) for PIB scores of 8, 5 and 2, respectively, on a 1–10 scale. More frequent leakage events were associated with lower utility. The utility decreases compared to no PSC were 0.114 (p <.0001), 0.057 (p <.0001) and 0.022 (p <.0001) for 48, 12 and 2 leakage events per year, respectively. The health state with a digital notification solution that notifies the user before a leakage event happens was considered as good as no PSC. Experiencing mild, moderate, and severe levels of PSC or leakage onto clothes is associated with a significant reduction in HRQoL compared to no PSC and/or no leakage. Stoma appliances that reduce the skin complications or keep leakage from reaching the clothes are likely to improve HRQoL. [ABSTRACT FROM AUTHOR]

For hospitalized patients with chronic obstructive pulmonary disease (COPD), albuterol and levalbuterol can both be used as relievers to alleviate bronchoconstriction. This study aimed to evaluate levalbuterol and albuterol's cost-utility and budget impact in hospitalized patients with COPD. A cost-utility analysis was used to evaluate the impact on the costs of nebulized levalbuterol verse albuterol in hospitalized patients with COPD. The decision tree model was employed to estimate the incremental cost per quality-adjusted life year in the admission setting. A budget impact model was used to examine the impact of budget on levalbuterol's entry into the Chinese market from the healthcare system's perspective. One-way sensitivity and probabilistic sensitivity analyses were performed to test the uncertainty of the parameters. The cost-utility results showed that levalbuterol saved ¥495.7 ($105.1) per hospitalization, while the budget impact analysis revealed a potential saving of ¥22.3 ($6.8) million in 3 years. The sensitivity analysis indicated that the results were robust to the changes in input parameter values. Levalbuterol is a cost-saving option for treating hospitalized patients with COPD in China. Chronic obstructive pulmonary disease (COPD) is a common disease in China, with an increased financial burden over the years. Nebulized albuterol is the most commonly used short-acting beta2-agonist, often regarded as the initial bronchodilator to treat hospitalized COPD patients. Its R-isomer, levalbuterol, entered the Chinese market in 2019. The new intervention always impacts the expenditure of the health system. We built a cost-utility and budget impact model to analyze the difference between albuterol and levalbuterol. The cost-utility results showed that levalbuterol saved ¥495.7 ($105.1) per hospitalization compared with albuterol, while the budget impact analysis revealed a potential saving of ¥22.3 ($6.8) million in 3 years. [ABSTRACT FROM AUTHOR]

Health state utilities associated with weight change are needed as inputs for cost-utility analyses (CUAs) examining the value of treatments for obesity and type 2 diabetes (T2D). Although some pharmaceutical treatments currently in development are associated with substantial weight loss, little is known about the utility impact of weight decreases greater than 10%. The purpose of this study was to estimate utilities associated with body weight decreases up to 20% based on preferences of individuals with obesity, with and without T2D. Health state vignettes were developed to represent respondents' own current weight and weight decreases of 2.5, 5, 10, 15, and 20%. Health state utilities were elicited in time trade-off interviews in two UK locations (Edinburgh and London) with a sample of participants with obesity, with and without T2D. Mean utility increases associated with each amount of weight decrease were calculated. Regression analyses were performed to derive a method for estimating utility change associated with weight decreases. Analyses were conducted with data from 405 individuals with obesity (202 with T2D, 203 without T2D). Utility increases associated with various levels of weight decrease ranged from 0.011 to 0.060 in the subgroup with T2D and 0.015 to 0.077 in the subgroup without T2D. All regression models found that the percentage of weight decrease was a highly significant predictor of change in utility (p <.0001). The relationship between weight change and utility change did not appear to be linear. Equations are recommended for estimating utility change based on the natural logarithm of percentage of weight decrease. Results of this study may be used to provide inputs for CUAs examining and comparing the value of treatments that are associated with substantial amounts of weight loss in patients with obesity, with or without T2D. [ABSTRACT FROM AUTHOR]

Efficacy of lenalidomide plus rituximab (R-LEN) compared to rituximab monotherapy (R-mono) for patients with previously treated follicular lymphoma (FL) was investigated in AUGMENT (NCT01938001). Our aim was to evaluate the cost-effectiveness of R-LEN versus R-mono in this setting from a Dutch perspective. Cost-effectiveness was assessed through a partitioned survival model from three perspectives (i.e. societal, healthcare, and societal, including future non-medical costs). Patient-level data from AUGMENT informed effectiveness parameters (i.e. long-term survival) and health state utilities. Resource use and prices were based on AUGMENT and the literature. Clinical experts validated efficacy input parameters and results. Uncertainty was explored through sensitivity and scenario analyses. R-LEN resulted in 1.7 incremental discounted quality-adjusted life years (QALYs). Total incremental discounted costs were 67,161 EUR from a societal perspective. In conclusion, R-LEN was cost-effective at a willingness-to-pay (WTP) threshold of 50,000 EUR/QALY in the base-case analyses(incremental cost-effectiveness ratio = 40,493 EUR/QALY). Scenario and sensitivity analyses indicated some level of uncertainty regarding this conclusion, depending on the chosen WTP-threshold and perspective. [ABSTRACT FROM AUTHOR]

Background: Non-invasive prenatal testing (NIPT) is a relatively new technology for diagnosis of fetal aneuploidies in the context of a developing country like Vietnam. Currently, first-trimester screening (FTS) is more conventional in Vietnam. NIPT is assumed to be more accurate than other maternal screening but is also more costly. The purpose of the study was to perform a cost-utility analysis comparing the FTS and NIPT for Down syndrome for high-risk pregnant women (over 35-year-old) in Vietnam. Methods: Decision tree model and probabilistic sensitivity analysis were used. Results: With the modelling population of 27,232 over 35 years of age pregnant women, FTS helps detect 218.5 cases of the fetus with Down syndrome compared with 247.2 cases when screening with NIPT. In addition, NIPT reduced 1384 false-positive cases compared to FTS. From a societal perspective, NIPT-dominated FTS. Compared to FTS, adopting an NIPT would cost an additional of USD 3,800 for each additional quality-adjusted life-year gained. Conclusions: NIPT is a cost-effective alternative to FTS for prenatal screening for Down syndrome with high-risk pregnant women. This is a very first attempt on cost-utility analysis of prenatal screening methods for Down syndrome in Vietnam and recommendations are made for future research to determine the most cost-effectiveness methods. [ABSTRACT FROM AUTHOR]

Objectives: To examine the cost-effectiveness of three mutually exclusive reproductive health interventions (named level A, B and C) implemented in Chi Linh for adolescents. Methods: This research involved the standard techniques of cost-utility analysis. A Markov state-transition model was used to show how a hypothetical cohort of 100,000 adolescents moved between 11 different health states over time, beginning at the year the intervention commenced (2011) and continuing in 3-month increments. The uncertainty of model input parameters was examined in probabilistic sensitivity analyses. Results: The deterministic analysis and uncertainty analysis revealed that implementing intervention level B compared to level A was highly cost effective. The ICER of intervention level B in relation to level A was USD 915/QALY gained. Implementing intervention level C compared to level B was cost-effective. The ICER of intervention level C over level B was USD 1911/QALY gained. Conclusions: If the decision makers including the intervention team, Ministry of Health and Ministry of Education and Training, change their current practice of educating adolescents on reproductive health to the best option, costs would be incurred at the lowest amount and health benefits would be brought back to the highest level. [ABSTRACT FROM AUTHOR]

Background: Spending on drugs provided by the Brazilian Public Health System (BPHS) for the treatment of rheumatoid arthritis (RA) increased substantially with the beginning of the supply of biological disease-modifying anti-rheumatic drugs (bDMARD). This study aims to perform a cost-utility analysis of the most used biological drugs for the treatment of RA in Brazil.Methods: a Markov model was used to carry out the cost-utility analysis. The data were obtained from a prospective cohort of RA patients using adalimumab, etanercept, and golimumab in Brazil. The BPHS perspective was adopted and the time horizon was five years. Deterministic and probabilistic sensitivity analyses were performed to evaluate the uncertainty.Results: golimumab was the most cost-effective drug. Etanercept was dominated by golimumab. Adalimumab presented an incremental cost-utility ratio (ICUR) of $95,095.37 compared to golimumab in five years of follow-up. These results were confirmed by sensitivity analyses.Conclusion: the utility among adalimumab, etanercept, and golimumab was similar and the cost was the component that most impacted the economic model. Therefore, depending on the agreed price with the drug manufacturers, the incremental cost-utility ratio may vary among them. [ABSTRACT FROM AUTHOR]

Background: This study aimed to estimate the cost-utility of stereotactic body radiotherapy (SBRT) plus cetuximab for patients with previously irradiated recurrent squamous cell carcinoma of the head and neck.Methods: We constructed a Markov health-state transition model to simulate costs and clinical outcomes of recurrent squamous cell carcinoma of the head and neck. Model parameters were derived from the published literature and the National Health Insurance Administration reimbursement price list. Incremental cost-effectiveness ratio and the net monetary benefit were calculated from a health payer perspective. The impact of uncertainty was modeled with one-way and probabilistic sensitivity analyses.Results: In the base-case, SBRT plus cetuximab compared to SBRT alone resulted in an ICER of NT$ 840,455 per QALY gained. In the one-way sensitivity analysis, the utility of progression-free state for patients treated with SBRT plus cetuximab or SBRT alone and the cost of progression-free survival for SBRT+Cet were the most sensitive parameters in the model. Probabilistic sensitivity analysis showed that the probability of cost-effectiveness at a willingness-to-pay threshold of NT$ 2,252,340 per QALY was 100% for SBRT plus cetuximab but 0% for SBRT alone.Conclusions: This study showed that SBRT+Cet was cost-effective and benefited patients with previously irradiated rSCCHN. [ABSTRACT FROM AUTHOR]

Background/objectives: Major depression has a negative impact on quality of life, increasing the risk of premature death. It imposes social and economic costs on individuals, families and society. Mental illness is now the leading cause globally of disability/lost quality life and premature mortality. Finding cost-effective treatments for depression is a public health priority. We report an economic evaluation of a dietary intervention for treating major depression. Methods: This economic evaluation drew on the HELFIMED RCT, a 3-month group-based Mediterranean-style diet (MedDiet) intervention (including cooking workshops), against a social group-program for people with major depression. We conducted (i) a cost-utility analysis, utility scores measured at baseline, 3-months and 6-months using the AQoL8D, modelled to 2 years (base case); (ii) a cost-effectiveness analysis, differential cost/case of depression resolved (to normal/mild) measured by the DASS. Differential program costs were calculated from resources use costed in AUD2017. QALYs were discounted at 3.5%pa. Results: Best estimate differential cost/QALY gain per person, MedDiet relative to social group was AUD2775. Probabilistic sensitivity analysis, varying costs, utility gain, model period found 95% likelihood cost/QALY less than AUD20,000. Estimated cost per additional case of depression resolved, MedDiet group relative to social group was AUD2,225. Conclusions: A MedDiet group-program for treating major depression was highly cost-effective relative to a social group-program, measured in terms of cost/QALY gain and cost per case of major depression resolved. Supporting access by persons with major depression to group-based dietary programs should be a policy priority. A change to funding will be needed to realise the potential benefits. [ABSTRACT FROM AUTHOR]

Introduction: Early biological treatment of rheumatoid arthritis (RA) may reverse the autoimmune response in some patients resulting in favorable long-term outcomes. Although the cost-effectiveness of this strategy has been questioned, biosimilar entries warrant the revision of clinical and pharmaco-economic evidence. Areas covered: We conducted a systematic review of randomized controlled trials (RCTs) published up to 24 May 2018 in Pubmed, EMBASE and Cochrane CENTRAL, comparing infliximab with non-biological therapy in patients with RA naïve to methotrexate. We performed meta-analyses for efficacy outcomes at month 6 and years 1 and 2. Six RCTs were identified, involving 1832 patients. At month 6 ACR70 response and remission, and at year 1 ACR20/ACR70 responses and remission were improved significantly with first-line infliximab versus control. The differences were not significant at year 2. We reviewed cost-utility studies, up to 31 October 2018 in PubMed, Cochrane CENTRAL and the CRD HTA databases. Four studies indicated that first-line use of originator infliximab calculated at 2005–2008 prices was not cost-effective. Expert opinion: We demonstrated the efficacy benefits of first-line infliximab therapy up to 1 year in methotrexate-naïve RA. We highlighted the need for standardized reporting of outcomes and conducting cost-effectiveness analyses of first-line biosimilar therapy in RA. [ABSTRACT FROM AUTHOR]

Introduction: Innovative drugs have been approved in ophthalmology. Thus, the number and importance of economic evaluation studies of ophthalmic drugs have been growing. This study aims to assess the methodological quality of pharmacoeconomic studies of ophthalmic drugs.Areas Covered: A systematic search was conducted in Pubmed/Embase until November 2018 to identify full pharmacoeconomic studies evaluating ophthalmic drugs. The quality of studies was evaluated using the British Medical Journal (BMJ) checklist. Quality indicators were evaluated by Fisher's exact test. Ninety-five studies were included, 50 (52.6%) cost-utility analysis, 28 (29.5%) cost-effectiveness and 17 (17.9%) cost-effectiveness/cost-utility. All studies presented, at least, three methodological limitations. Cost-utility studies, studies conducted from a health system perspective, with time horizons longer than one-year and that rely on observational or observational and experimental data simultaneously are associated with higher quality. Only eight (8.4%) studies considered two eyes in the economic analysis and only 13 (13.7%) considered the natural history of the disease when extrapolating results for long-term analysis.Expert Opinion: The majority of the pharmacoeconomic studies were assessed as having good methodological quality, however, the methodological quality scores were sensitive to several indicators. Therefore, improving the quality of studies would enhance their usefulness in the decision-making processes. [ABSTRACT FROM AUTHOR]

Background: Outpatient parenteral antimicrobial therapy (OPAT) has been used for decades in different countries to reduce hospitalization rates, with favorable clinical and economic outcomes. This study assesses the cost-utility of OPAT compared to inpatient parenteral antimicrobial therapy (IPAT) from the perspective of a public university hospital and the Brazilian National Health System (Unified Health System -SUS).Methods: Prospective study with adult patients undergoing OPAT at an infusion center, compared to IPAT. Clinical outcomes and quality-adjusted life year (QALY) were assessed, as well as a micro-costing. Cost-utility analysis from the hospital and SUS perspectives were conducted by means of a decision tree, within a 30-day horizon time.Results: Forty cases of OPAT (1112 days) were included and monitored, with a favorable outcome in 97.50%. OPAT compared to IPAT generated overall savings of 31.86% from the hospital perspective and 26.53% from the SUS perspective. The intervention reduced costs, with an incremental cost-utility ratio of -44,395.68/QALY for the hospital and -48,466.70/QALY for the SUS, with better cost-utility for treatment times greater than 14 days. Sensitivity analysis confirmed the stability of the model.Conclusion: Our economic assessment demonstrated that, in the Brazilian context, OPAT is a cost-saving strategy both for hospitals and for the SUS. [ABSTRACT FROM AUTHOR]

Introduction: To evaluate the potential economic value and likely impact of a hypothetical rapid test in its early stages of development requires the use of models. The model structure and the type of model (dynamic/static) to employ are key considerations. The aim of the review was to explore the literature on typhoid economic evaluations and to explore the types of models that have been previously adopted in this setting for test-treat evaluations and to capture data on model inputs that may be useful for a de novo model.Areas Covered: A systematic review was conducted to identify economic evaluations focused on typhoid in established literature databases. Eight studies were identified and included for narrative synthesis. The review has revealed that there have been relatively few economic evaluations that have focused on typhoid fever, all of which have focused on the impact of interventions at the population level (vaccination) but not the individual level (test-treat strategies).Expert Commentary: Under certain circumstances, either a static model or a transmission dynamic model may be appropriate in the evaluation of an intervention for typhoid fever. Typhoid test-treat modeling represents a gray area where further work is needed. [ABSTRACT FROM AUTHOR]

Objective: Rifaximin-α 550 mg twice daily plus lactulose has demonstrated efficacy in reducing recurrence of episodes of overt hepatic encephalopathy (OHE) and the risk of hepatic encephalopathy (HE)-related hospitalizations compared with lactulose alone. This analysis estimated the cost effectiveness of rifaximin-α 550 mg twice daily plus lactulose versus lactulose alone in United Kingdom (UK) cirrhotic patients with OHE.Method: A Markov model was built to estimate the incremental cost-effectiveness ratio (ICER). The perspective was that of the UK National Health Service (NHS). Clinical data was sourced from a randomized controlled trial (RCT) and an open-label maintenance study in cirrhotic patients in remission from recurrent episodes of OHE. Health-related utility was estimated indirectly from disease-specific quality of life RCT data. Resource use data describing the impact of rifaximin-α on hospital admissions and length of stay for cirrhotic patients with OHE was from four single-center UK audits. Costs (2012) were derived from published sources; costs and benefits were discounted at 3.5%. The base-case time horizon was 5 years.Results: The average cost per patient was £22,971 in the rifaximin-α plus lactulose arm and £23,545 in the lactulose arm, a saving of £573. The corresponding values for benefit were 2.35 quality adjusted life years (QALYs) and 1.83 QALYs per person, a difference of 0.52 QALYs. This translated into a dominant base-case ICER. Key parameters that impacted the ICER included number of hospital admissions and length of stay.Conclusion: Rifaximin-α 550 mg twice daily in patients with recurrent episodes of OHE was estimated to generate cost savings and improved clinical outcomes compared to standard care over 5 years. [ABSTRACT FROM AUTHOR]

Background: To assess the cost-utility of vortioxetine versus relevant comparators (agomelatine, bupropion SR, sertraline, and venlafaxine XR) in the finnish setting in major depressive disorder (MDD) patients with inadequate response to selective serotonin- /serotonin-norepinephrine reuptake inhibitors.Methods: A one-year analysis was conducted using a decision tree with a Markov state transition component. The health states were remission, relapse and recovery. A Finnish healthcare payer perspective was adopted.Results: Vortioxetine was less costly and more effective versus all comparators in both direct and societal perspectives. Vortioxetine reduced the average annual direct costs by 4% versus venlafaxine XR and 8% versus sertraline. The greater efficacy associated with vortioxetine was translated into a higher percentage of patients in remission and recovery. The model was most sensitive to changes in remission rates at 8 weeks.Conclusion: This cost-utility analysis showed vortioxetine to be a good alternative for MDD patients switching therapy in Finland. [ABSTRACT FROM AUTHOR]

Purpose: The aim of this study was to analyse the cost-effectiveness of prescribing powered mobility devices (PMDs) to elderly users.Methods: Forty-five persons participated in the pre- and post-intervention study with a follow-up at four months. All participants were prescribed a scooter model and were offered individual support to get started using the PMD. In the analysis, the use of the PMD was compared to the situation prior to its use. The cost-utility analysis takes a societal perspective and considers costs, savings and quality of life (QoL) using answers to the EQ-5D questionnaire.Results: Costs for the first year with the PMD were 1395 USD and then 592 USD per subsequent year. There was a significant decrease in transportation costs and in relatives' time use, but the increase in QoL of 0.041 was not significant. Costs per gained quality adjusted life year (QALY) were 12 400–14 700 USD/QALY if the value of time saved not was considered and 600–2900 USD/QALY when an hour was valued at 3.6 USD.Conclusion: Prescription of PMDs to elderly users might be cost-effective. However, there are shortcomings in measuring QALY gains from the use of a PMD, and it is unclear how time savings among relatives should be valued.Implications for RehabilitationPrescription of powered mobility devices for the elderly with disability seems to be cost effective and should be a standard intervention.In economic evaluations of powered mobility devices and other assistive devices, the commonly used analyses methods are not always appropriate and therefore need to be adjusted. [ABSTRACT FROM PUBLISHER]

Objective: Across Japan, around 2 million people are infected with hepatitis C virus (HCV) with long-term complications such as cirrhosis, hepatocellular carcinoma (HCC) and liver transplant (LT). Current treatment options have several limitations due to side effects, interferon intolerability and ineligibility, long treatment durations and low sustained virological responses (SVR) rates, especially for the most severe patients. Sofosbuvir (SOF) is the first nucleotide analog NS5B polymerase inhibitor with pan-genotypic activity. SOF, administered in combination with ribavirin (RBV) with or without pegylated interferon (PEGIFN) resulted in high SVR rates across genotype (GT) 1-6 patients. It is also the first available regimen for patients that are unsuitable for interferon. This analysis assessed the cost-utility ratio of sofosbuvir in GT2 patients in Japan.Research Design and Methods: A Markov model followed a cohort of 10,000 GT2 patients until patients reached 100 years of age. Approximately 20% of patients initiated treatment at the cirrhotic stage. Comparators were based on the current recommendations in Japan, including PEGIFN with ribavirin (RBV), telaprevir (TVR) in combination with PEGIFN + RBV and no treatment. Costs and outcomes were discounted at 2%.Results: Sofosbuvir was cost-effective across all the studied indications, especially in patients unsuitable for interferon, with incremental cost-effectiveness ratios (ICERs) lower than JPY 5,000,000. Compared to the other treatments included in the analysis, SOF + RBV resulted in improved clinical outcomes. Results were robust to sensitivity analyses.Conclusion: SOF combined with RBV was shown to be cost-effective in GT2 patients in Japan. Compared to PEGIFN + RBV, TVR + PEGIFN + RBV and no treatment SOF offers a more efficacious, shorter and better tolerated treatment option and extends treatment to reach HCV-infected patients who are ineligible for interferon-based regimens. Although adverse events were not included in the analyses, this would not make any changes to our conclusion. [ABSTRACT FROM AUTHOR]

Objective: Fabry disease is a rare inherited lysosomal storage disorder caused by deficiency of α-galactosidase A. Effective enzyme replacement therapies are available that are administered intravenously. However, a new oral treatment is being developed as an alternative option for patients with amenable mutations. This study was designed to understand the value that people place on the different features of treatments for Fabry disease.Research Design and Methods: A discrete choice experiment (DCE) was designed to assess the importance of different aspects of treatments for Fabry disease. The attributes included overall survival, mode of administration, treatment related reactions, treatment related headaches and risk of antibody formation. Attributes were combined using a published orthogonal array into choice sets. A research panel was used to survey the UK general public. The mixed logit model was used to estimate strength of preference for the attributes and marginal rates of substitution (MRSs). Disutilities were estimated from the DCE data for changes in each attribute.Results: The sample (n = 506) was broadly representative of UK demographics. The logit model revealed that all attributes were significant predictors of choice. Participants were significantly more likely to choose a treatment which meant an increase in their life expectancy by 1 year (odds ratio = 1.574; 95% CI = 1.504-1.647) and significantly less likely to choose self-administered intravenous (IV) treatment compared to an every other day tablet (OR = 0.426 95% CI = 0.384-0.474). Estimated disutilities were -0.0543 (self-administered infusion), treatment related headaches 12 times a year (-0.0361) and infusion reactions six times a year (-0.0202).Conclusions: The survey revealed a significant preference for oral treatment compared with IV even in the context of a treatment that can extend overall survival. MRSs were used as a basis for estimating disutilities associated with changes in attribute levels which could be used to weight QALYs. It is possible that other important treatment attributes are missing from this research which may have provided further insights. It would also be useful to extend this research to include Fabry disease patients so their preferences can be assessed against the societal perspective. [ABSTRACT FROM AUTHOR]

Objective: Hepatitis C is the result of a ribonucleic acid (RNA) virus (hepatitis C virus; HCV). The Japan Society of Hepatology (JSH) estimated that 1.5-2 million people in Japan carry HCV. Six major HCV genotypes (GT) and a large number of subtypes have been described in the literature. In Japan, around 70% to 80% of people are infected with HCV genotype 1b. The progress of the disease primarily affects the liver and may lead to liver cirrhosis, hepatocellular carcinoma (HCC) and death. Sofosbuvir (SOF) is a nucleotide analogue NS5B inhibitor and ledipasvir (LDV) is an inhibitor of the HCV NS5A protein. They are combined in a single tablet regimen for the treatment of GT1 patients and resulted in sustained virological response (SVR) above 94% in large phase III trials. This analysis assesses the cost-utility of LDV/SOF in GT1 patients in Japan.Research Design and Methods: A cohort of 10,000 patients was followed through a Markov model until they reached 100 years of age. GT1 treatment-naïve and experienced, non-cirrhotic and cirrhotic patients were studied separately. LDV/SOF was compared to several treatment regimens containing pegylated interferon (PEGIFN), telaprevir (TVR), simeprevir (SMV), daclatasvir (DCV), asunaprevir (ASV) and ribavirin (RBV). Discount rates of 2% were applied to costs and outcomes according to the Japanese guidelines.Results: LDV/SOF was cost-effective against most comparators with incremental cost-effectiveness ratios (ICERs) below JPY 5,000,000. By applying a societal perspective, LDV/SOF was the dominant treatment strategy in all cases. Moreover, LDV/SOF reduced the number of cases of advanced liver disease. These results were robust to sensitivity analyses.Conclusions: LDV/SOF was cost-effective compared to most of the currently recommended treatments. Furthermore, LDV/SOF extends treatments to HCV-infected patients who are ineligible for interferon and RBV-based regimens. LDV/SOF thus has the potential to help reduce the burden of HCV in Japan. [ABSTRACT FROM AUTHOR]

With a low breast cancer incidence and low population density, Greenland is geographically and organisationally challenged in implementing a cost effective breast cancer screening programme where a large proportion of the Greenlandic women will have to travel far to attend. The aim of this paper is to evaluate the cost effectiveness and cost utility of different strategies for implementing population-based breast cancer screening in Greenland. Two strategies were evaluated: Centralised screening in the capital Nuuk and decentralised screening in the five municipal regions of Greenland. A cost effectiveness and cost utility analysis were performed from a societal perspective to estimate the costs per years of life saved and per QALY gained. Two accommodation models for the women's attendance were examined; accommodation in ordinary hotels or in patient hotels. The least costly accommodation model was the hotel model compared with the patient hotel model, regardless of screening strategy. The decentralised strategy was more cost effective compared with the centralised strategy, resulting in 0.5 million DKK per years of life saved (YLS) and 4.1 million DKK per quality-adjusted life year (QALY) gained within the hotel model. These ratios are significantly higher compared with findings from other countries. The sensitivity analysis showed a substantial gap between the most and least favourable model assumptions. The investigated strategies were all estimated to be extremely costly, mostly due to high transportation and accommodation costs and loss of productivity, and none would be accepted as cost-effective per YLS/QALY gained within a conventional threshold level. The least expensive strategy was regional screening with hotel accommodation. [ABSTRACT FROM AUTHOR]

Introduction: Economic evaluations conducted in children have unique features compared to adults. Important developments in pediatric economic evaluation in recent years include new options for valuing health states for cost-utility analysis (CUA) and shifting child health priorities. The Pediatric Economic Database Evaluation (PEDE) project includes a comprehensive database of pediatric health economic evaluations published since 1980. The objective of this scoping review was to identify trends over time in the use of CUA and other analytic techniques, and the therapeutic areas chosen for study. Areas covered: Medical and grey literature were searched, key characteristics were extracted, frequencies were tabulated and cross-tabulations were performed. Differences between early (1980 and 1999) and late (2000 and 2014) periods were assessed using a chi-squared statistic. Of the 2,776 pediatric economic evaluations published between 1980 and 2014, substantially more were cost-effectiveness analyses (CEAs) and CUAs than cost benefit analyses and cost minimization analyses (63.9 and 24.9% versus 7.6 and 3.6%, respectively). This pattern was consistent regardless of the type of intervention, disease or age group studied. A trend toward higher proportions of CUAs and CEAs was evident in the later period (X2 p < 0.0001). Other significant trends included a higher proportion of studies of preventive interventions (X2 p < 0.0001), and more studies in children aged 1 to 12 years and fewer in perinates in the later period (X2 p < 0.0001). Overall the most common disease class studied was infectious diseases (29.2%). Expert commentary: Pediatric economic evaluation continues to grow in volume and methodologic complexity. While CUAs have increased, whether their quality has improved remains unknown. Although most studies are in infectious disease, the volume of publications may not align with emerging child health priorities such as adolescent health, injury, developmental disabilities, mental health, and the use of personalized medicine. Increasing economic evaluations in these areas will enhance pediatric decision-making. [ABSTRACT FROM AUTHOR]

Introduction: For patients with late-stage (metastatic) breast cancer, the impact of treatment on health-related quality of life is a key factor in decision-making. A systematic review was conducted to identify health state utility values (HSUVs) for late-stage breast cancer, derived using methods preferred by health technology assessment (HTA) agencies, by treatment line. The aim was to generate a list of HSUVs, that could help to justify the values used to populate cost-utility models. Areas covered: Ten electronic databases, international congress websites and online HSUV databases were searched (January 1995-May 2014) for HSUVs for adults with late-stage breast cancer that had been derived from methods favoured by HTA agencies. Publications were included only if they reported studies that originated HSUVs. Expert commentary: Large numbers of HSUVs are available for late-stage breast cancer in the published literature. Contrary to expectations, the HSUVs reported in the literature vary greatly for some health states. As a result, the choice of HSUV can have considerable implications for the outcomes of economic evaluations. Standardization of HSUV methodology is expected to reduce variability; however, further research is recommended for assessing the sensitivity of generic preference-based measures in late-stage (metastatic) breast cancer. [ABSTRACT FROM AUTHOR]

Objective: This study aims to compare the lifetime costs and health outcomes of both first-line and sequential combination treatments with standard treatment for pulmonary arterial hypertension (PAH) associated with congenital heart disease (CHD) (PAH-CHD) patients.Methods: A cost-utility analysis was performed using a Markov model based on a societal perspective. One-way and probabilistic sensitivity analyses were performed to investigate the effect of parameter uncertainty.Results: As first-line treatments, both beraprost (incremental cost-effectiveness ratio (ICER) = 192,752 and 201,308 Thai baht (THB) per quality-adjusted life year (QALY) gained) and sildenafil (ICER = 249,770 and 226,802 THB per QALY gained) seemed cost-effective for PAH-CHD patients aged ≤30 years in functional classes II and III, respectively, while no treatment was cost-effective for the sequential combination therapy.Conclusions: Sildenafil should be included in the National Drug List of Essential Medicines as the first-line treatment for PAH-CHD, and its price per dose should be negotiated to be reduced by 43-57%. [ABSTRACT FROM AUTHOR]

Children are not mini-adults, and thus require studies to be conducted in the population of interest to inform decisions about their care. The paucity of such studies for clinical efficacy lead them to be termed ‘therapeutic orphans’. Following the introduction of the ‘fourth hurdle’ of reimbursement approval on the basis of cost-utility analysis, utility data is now a key requirement for patients to access treatments in England and many other countries. This special report considers whether a paucity of utility valuation studies in children may have made them ‘economic orphans’ as well and presents results of a review of NICE appraisals as a window on this problem over time. [ABSTRACT FROM PUBLISHER]

Although economic evaluations have been performed alongside factorial trials, there seems to be limited guidance/consensus on appropriate methods of analysis. Following Centre for Review and Dissemination guidance, a systematic review of published literature for all years was performed to explore how economic evaluation alongside factorial trials have been conducted and only full economic evaluations conducted alongside factorial trials were included. A total of 16 relevant studies were identified, and an assessment of these indicated that two methods: within-the-table and at-the-margins approaches were used for the analysis. With the exception of one study, all others did not consider interactions in costs and outcomes or give a detailed explanation of why a particular approach was adopted. The authors recommend that additional guidance is needed, and further research is required to evaluate the impact of alternative methods on policy recommendations and establish good practice methods for the economic analysis of factorial trials. [ABSTRACT FROM AUTHOR]

Background: In Thailand, there has been no economic evaluation study of adjuvant chemotherapy for stage III colon cancer patients after resection. Objective: This study aims to evaluate the cost-utility of all chemotherapy regimens currently used in Thailand compared with the adjuvant 5-fluorouracil/leucovorin (5-FU/LV) plus capecitabine as the first-line therapy for metastatic disease in patients with stage III colon cancer after resection. Methods: A cost-utility analysis was performed to estimate the relevant lifetime costs and health outcomes of chemotherapy regimens based on a societal perspective using a Markov model. Results: The results suggested that the adjuvant 5-FU/LV plus capecitabine as the first-line therapy for metastatic disease would be the most cost-effective chemotherapy. Conclusions: The adjuvant FOLFOX and FOLFIRI as the first-line treatment for metastatic disease would be cost-effective with an incremental cost-effectiveness ratio of 299,365 Thai baht per QALY gained based on a societal perspective if both prices of FOLFOX and FOLFIRI were decreased by 40%. [ABSTRACT FROM AUTHOR]

Purpose: To perform a short-term and a long-term cost-utility analysis of rhegmatogenous retinal detachment (RRD) surgery in Shanghai, China Methods: A total of 117 patients who underwent RRD surgery at Shanghai First People's Hospital, Affiliated Shanghai Jiaotong University, and then underwent follow-up for at least 1 year, were included in the study. Costs associated with RRD surgery and time trade-off utility values before and after surgery were recorded. Short-term analysis was made for 1 year. Quality-adjusted life years (QALYs) gained by RRD surgery over the lifetime were calculated at a 3% discount rate in the long-term analysis. This study used the bootstrap method in statistical analysis and one-way sensitivity analyses to test robustness of the results. Results: Compared with no treatment, the mean incremental costs of RRD surgery was 11,384 Chinese yuan (CN¥) (US$1751); the mean additional QALYs gained was 0.05 (95% confidence interval [CI] 0.04-0.06) for 1 year and 0.88 (95% CI 0.64-1.13) for life expectancy; the incremental cost effectiveness ratio (ICER) was CN¥224,921 (US$34,603)/QALY for the short-term, and CN¥13,794 (US$2122)/QALY for the long-term. In short-term analysis, the ICER was CN¥150,087 (US$23,090)/QALY for scleral buckling surgery, and CN¥507,727 (US$78,112)/QALY for vitreous surgery. In the long-term analysis, the ICER was CN¥6280 (US$966)/QALY for scleral buckling, and CN¥30,756 (US$4732)/QALY for vitreous surgery. Conclusions: For RRD patients, surgery increases QALYs. In a 1-year analysis, surgery is not a cost-effective treatment, however it is cost-effective over the lifetime. [ABSTRACT FROM AUTHOR]

Purpose: To evaluate the cost-effectiveness for a screening interval longer than 1 year detecting diabetic retinopathy (DR) through the estimation of incremental costs per quality-adjusted life year (QALY) based on the best available clinical data in Japan. Methods: A Markov model with a probabilistic cohort analysis was framed to calculate incremental costs per QALY gained by implementing a screening program detecting DR in Japan. A 1-year cycle length and population size of 50,000 with a 50-year time horizon (age 40-90 years) was used. Best available clinical data from publications and national surveillance data was used, and a model was designed including current diagnosis and management of DR with corresponding visual outcomes. One-way and probabilistic sensitivity analyses were performed considering uncertainties in the parameters. Results: In the base-case analysis, the strategy with a screening program resulted in an incremental cost of 5,147 Japanese yen (¥; US$64.6) and incremental effectiveness of 0.0054 QALYs per person screened. The incremental cost-effectiveness ratio was ¥944,981 (US$11,857) per QALY. The simulation suggested that screening would result in a significant reduction in blindness in people aged 40 years or over (−16%). Sensitivity analyses suggested that in order to achieve both reductions in blindness and cost-effectiveness in Japan, the screening program should screen those aged 53-84 years, at intervals of 3 years or less. Conclusions: An eye screening program in Japan would be cost-effective in detecting DR and preventing blindness from DR, even allowing for the uncertainties in estimates of costs, utility, and current management of DR. [ABSTRACT FROM AUTHOR]

That the maximization of quality-adjusted life years violates concerns for fairness is well known. One approach to face this issue is to elicitfairness preferencesof the public empirically and to incorporate the correspondingequity weightsinto cost-utility analysis (CUA). It is thereby sought to encounter the objections by means of an axiological modification while leaving the value-maximizing framework of CUA intact. Based on the work of Lübbe (2005, 2009a, 2009b, 2010, forthcoming), this paper questions this strategy and scrutinizes the concomitant assumptions concerning the nature of prioritization decisions. Empirical studies indicate that these premises are in fact unwarranted. People chose a certain resource allocation because they perceive it as a fair way totreatthe persons concerned, not because it maximizes something valuable, and it is questionable if prioritization decision can be represented as value-maximizing choices at all. This reflection on the fundamental distinction between deontological and consequentialist reasoning bears general implications for the scope of ‘economic imperialism.’ [ABSTRACT FROM AUTHOR]

The objective is to examine the cost-utility of sipuleucel-T immunotherapy in asymptomatic or minimally symptomatic castration-resistant prostate cancer patients. The addition of sipuleucel-T immunotherapy to standard treatment led to a gain of 0.37 quality-adjusted life-year (QALY) at an additional cost of US$104,536. The incremental cost-utility ratio was US$283,000 per QALY saved. Threshold sensitivity analyses indicated that a price reduction of at least 53%, or application in a group of patients resulting in the relative reduction in the mortality rate of at least 39%, ought to augment the economic value of this regimen. Sipuleucel-T immunotherapy treatment at the current price with 96.5% certainty is not cost-effective. The specific group of patients who will benefit more from the treatment should be revealed and treated, or the cost of the vaccine should be lowered significantly to increase its economic value. Accounting for crossover treatment in control patients improves sipuleucel-T's value (US$132,000 per QALY saved) although further investigation is necessary. [ABSTRACT FROM AUTHOR]

Conventional coronary angiography (CCA) is the standard diagnostic for coronary artery disease (CAD), but multi-detector computed tomography coronary angiography (CTCA) is a non-invasive alternative. Methods: A multi-center coverage with evidence development study was undertaken and combined with an economic model to estimate the cost-effectiveness of CTCA followed by CCA vs CCA alone. Alternative assumptions were tested in patient scenario and sensitivity analyses. Results: CCA was found to dominate CTCA, however, CTCA was relatively more cost-effective in females, in advancing age, in patients with lower pre-test probabilities of CAD, the higher the sensitivity of CTCA and the lower the probability of undergoing a confirmatory CCA following a positive CTCA. Conclusions: Results were very sensitive to alternative patient populations and modeling assumptions. Careful consideration of patient characteristics, procedures to improve the diagnostic yield of CTCA and selective use of CCA following CTCA will impact whether CTCA is cost-effective or dominates CCA. [ABSTRACT FROM AUTHOR]

Objectives. The number of new health technologies has risen over the past decade. These new technologies usually are more effective but they also cost more compared to existing ones. In a publicly funded health care system such as Canada, the aim is to maximize the health of the population within the resources available. As a result, it is unavoidable that choices and trade-offs have to be made because there will always be more treatment options than resources will allow (i.e., scarcity of resources) as well as alternative uses for those resources (i.e., opportunity costs). The objective of this paper is to provide an overview of economic evaluations and how these tools can be used to help inform payers of the best use of scarce health care resources. Study design. This descriptive paper includes a summary of key consepts and definitions in economic appraisal and draws upon recently published papers as illustrations. Methods. Background on the necessity and role of economic evaluations is provided, followed by a description of the approaches for, and types of, economic evaluations. Two illustrative examples are used and some implications for rural, remote and circumpolar communities are discussed. Results. There are 2 main approaches for conducting an economic evaluation (trial- and model-based) and 3 types of evaluations which can be considered to inform payers of the best use of health care resources (cost-effectiveness, cost-utility and cost-benefit analyses). Conclusions. Techniques of economic evaluation are useful tools and an important input into the decision- making process. Although these techniques have universal application, there are issues specific to rural, remote and circumpolar communities which can affect the results of economic appraisals. [ABSTRACT FROM AUTHOR]

The Pharmaceutical Management Agency in New Zealand, PHARMAC, was established in 1993 at a time when growth in pharmaceutical expenditure was very high and arguably unsustainable. PHARMAC was charged with finding new and effective ways to manage expenditure growth, while also obtaining the best health outcomes for the New Zealand population. In order to help achieve this goal, PHARMAC has used Programme Budgeting Marginal Analysis. The use of Programme Budgeting Marginal Analysis, together with a capped budget and tools to generate savings, has significantly contributed to PHARMAC achieving its objective. However, there are implications of using Programme Budgeting Marginal Analysis with a capped budget. In particular, a different approach is required when undertaking and using cost--utility analysis (focused strongly on relative cost--effectiveness), and the opportunity cost of poor decisions is magnified significantly. As the demand on pharmaceutical expenditure continues to rise, the opportunity cost of not having a capped budget and tools for controlling pharmaceutical subsidies will only increase. [ABSTRACT FROM AUTHOR]

Purpose: To describe the methods and innovations used in constructing the VisQoL, a vision-related utility instrument for the health economic evaluation of eye care and rehabilitation programs. Methods: The VisQoL disaggregates vision into six items. Utilities were estimated for item worst responses (the worst level for each item, with all other items at their best level) and VisQoL all-worst responses (all items at their worst level) using the time trade-off procedure. Time trade-off questions require people to imagine living a fixed number of years with a particular health condition and then indicate how many of those years of life they would be willing to trade to have perfect health. Where respondents indicated a health state was “worse than death” negative utilities were estimated. Time trade-off questions minimized the “focusing effect,” which occurs if respondents discount the fact that all other aspects of health are at their best when answering questions, by using pictorial and verbal aids. Results: Item utilities were combined using a multiplicative model, and VisQoL model utilities placed on a scale where 0.00 and 1.00 represent full health and death, respectively. The VisQoL allows utilities to be calculated for a wide range of vision-related conditions. Conclusion: The 6-item VisQoL has excellent psychometric properties and is specifically designed to be sensitive to vision-related quality of life. It is the first instrument to permit the rapid estimation of utility values for use in economic evaluations of vision-related programs. [ABSTRACT FROM AUTHOR]

Economic evaluation in the form of reports of cost-effectiveness of the treatment and prevention of disease has only recently found widespread application in the visual sciences. While economic evaluation takes a number of forms: cost-minimization analysis, cost-benefit analysis, and cost-effectiveness analysis - it is the latter that is seen most often in the evaluation of vision-related health programs. Cost-effectiveness analysis is in particular seen most commonly in its very particular form of cost-utility analysis. Decision analysis is the analytic method most commonly used to perform cost-effectiveness analysis. In decision analysis, the expected cost and effectiveness of a health program are estimated in a rigorous fashion. In this report, we take the reader through the process of decision analysis including building the tree; populating the model with parameters for risk, cost and benefit; estimating expected cost and benefit; and deterministic and probabilistic sensitivity analysis. Examples employed include prominent studies of the cost-effectiveness of photodynamic therapy for treatment of neovascular macular degeneration and the treatment ocular hypertension to prevent glaucoma. [ABSTRACT FROM AUTHOR]