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The National Institute for Health and Care Excellence (NICE) is only one of several organisations internationally that uses economic evaluation as part of decision making regarding funding and pricing of new medical technologies. However, it can be argued that NICE has developed a more prominent international profile than most in their use of economics. After 20 years of operation, it is timely to assess the extent of NICE's achievements, including the economic evaluation methods it has used and its willingness to adapt these as new evaluative approaches emerge and when NICE faces particular policy challenges. This paper considers some of the important policy and contextual developments in the UK over the last 20 years and how these may have shaped NICE's approach to economic evaluation. It then assesses key areas of NICE methods, including perspective, defining benefits, modelling and uncertainty. The paper concludes that NICE has provided important support for the development of new methods, in particular through its role in identifying priorities for methods research funding and its sponsorship of the NICE Decision Support Unit. However, potentially important developments in methods in a number of important areas have yet to be formally included in NICE's methods guidance and this should be addressed in the Institute's 2019/2020 methods review. [ABSTRACT FROM AUTHOR]

Objective: The Swedish Dental and Pharmaceutical Benefits Agency (TLV) is the government body responsible for deciding whether outpatient drugs are to be included in the pharmaceutical benefits scheme. This paper analyzes the impact of cost effectiveness and severity of disease on reimbursement decisions for new pharmaceuticals.Methods: Data has been extracted from all decisions made by the TLV between 2005 and 2011. Cost effectiveness is measured as the cost per quality-adjusted life-year (QALY) gained, whereas disease severity is a binary variable (severe-not severe). In total, the dataset consists of 102 decisions, with 86 approved and 16 declined reimbursements.Results: The lowest cost per QALY of declined reimbursements is Swedish kronor (SEK) 700,000 (€ 79,100), while the highest cost per QALY of approved reimbursements is SEK1,220,000 (€ 135,600). At a cost per QALY of SEK702,000 Swedish kronor (non-severe diseases) and SEK988,000 (severe diseases), the likelihood of approval is estimated to be 50/50 (€ 79,400 and € 111,700).Conclusions: The TLV places substantial weight on both the cost effectiveness and the severity of disease in reimbursement decisions, and the implied willingness to pay for a QALY is higher than the often cited 'rule of thumb' in Swedish policy debates. [ABSTRACT FROM AUTHOR]

Background: Pre-eclampsia is a pregnancy complication affecting both mother and fetus. Although there is no proven effective method to prevent pre-eclampsia, early identification of women at risk of pre-eclampsia could enhance appropriate application of antenatal care, management and treatment. Very little is known about the cost effectiveness of these and other tests for pre-eclampsia, mainly because there is no clear treatment path. The aim of this study was to provide a comprehensive overview of the existing evidence on the health economics of screening, diagnosis and treatment options in pre-eclampsia.Methods: We searched three electronic databases (PubMed, EMBASE and the Cochrane Library) for studies on screening, diagnosis, treatment or prevention of pre-eclampsia, published between 1994 and 2014. Only full papers written in English containing complete economic assessments in pre-eclampsia were included.Results: From an initial total of 138 references, six papers fulfilled the inclusion criteria. Three studies were on the cost effectiveness of treatment of pre-eclampsia, two of which evaluated magnesium sulphate for prevention of seizures and the third evaluated the cost effectiveness of induction of labour versus expectant monitoring. The other three studies were aimed at screening and diagnosis, in combination with subsequent preventive measures. The two studies on magnesium sulphate were equivocal on the cost effectiveness in non-severe cases, and the other study suggested that induction of labour in term pre-eclampsia was more cost effective than expectant monitoring. The screening studies were quite diverse in their objectives as well as in their conclusions. One study concluded that screening is probably not worthwhile, while two other studies stated that in certain scenarios it may be cost effective to screen all pregnant women and prophylactically treat those who are found to be at high risk of developing pre-eclampsia.Discussion: This study is the first to provide a comprehensive overview on the economic aspects of pre-eclampsia in its broadest sense, ranging from screening to treatment options. The main limitation of the present study lies in the variety of topics in combination with the limited number of papers that could be included; this restricted the comparisons that could be made. In conclusion, novel biomarkers in screening for and diagnosing pre-eclampsia show promise, but their accuracy is a major driver of cost effectiveness, as is prevalence. Universal screening for pre-eclampsia, using a biomarker, will be feasible only when accuracy is significantly increased. [ABSTRACT FROM AUTHOR]

Many healthcare organizations are now making good use of electronic health record (EHR) systems to record clinical information about their patients and the details of their healthcare. Electronic data in EHRs is generated by people engaged in complex processes within complex environments, and their human input, albeit shaped by computer systems, is compromised by many human factors. These data are potentially valuable to health economists and outcomes researchers but are sufficiently large and complex enough to be considered part of the new frontier of 'big data'. This paper describes emerging methods that draw together data mining, process modelling, activity-based costing and dynamic simulation models. Our research infrastructure includes safe links to Leeds hospital's EHRs with 3 million secondary and tertiary care patients. We created a multidisciplinary team of health economists, clinical specialists, and data and computer scientists, and developed a dynamic simulation tool called NETIMIS (Network Tools for Intervention Modelling with Intelligent Simulation; http://www.netimis.com ) suitable for visualization of both human-designed and data-mined processes which can then be used for 'what-if' analysis by stakeholders interested in costing, designing and evaluating healthcare interventions. We present two examples of model development to illustrate how dynamic simulation can be informed by big data from an EHR. We found the tool provided a focal point for multidisciplinary team work to help them iteratively and collaboratively 'deep dive' into big data. [ABSTRACT FROM AUTHOR]

There is a growing appetite for large complex databases that integrate a range of personal, socio-demographic, health, genetic and financial information on individuals. It has been argued that 'Big Data' will provide the necessary catalyst to advance both biomedical research and health economics and outcomes research. However, it is important that we do not succumb to being data rich but information poor. This paper discusses the benefits and challenges of building Big Data, analysing Big Data and making appropriate inferences in order to advance cancer care, using Cancer 2015 (a prospective, longitudinal, genomic cohort study in Victoria, Australia) as a case study. Cancer 2015 has been linked to State and Commonwealth reimbursement databases that have known limitations. This partly reflects the funding arrangements in Australia, a country with both public and private provision, including public funding of private healthcare, and partly the legislative frameworks that govern data linkage. Additionally, linkage is not without time delays and, as such, achieving a contemporaneous database is challenging. Despite these limitations, there is clear value in using linked data and creating Big Data. This paper describes the linked Cancer 2015 dataset, discusses estimation issues given the nature of the data and presents panel regression results that allow us to make possible inferences regarding which patient, disease, genomic and treatment characteristics explain variation in health expenditure. [ABSTRACT FROM AUTHOR]

This paper outlines recent advances in the methods of cost-benefit analysis (CBA). Economic evaluations in healthcare can be criticised for, amongst other things, the inappropriate use of incremental cost-effectiveness ratios and the reporting of benefits in terms of cost savings, such as treatment costs averted. Many such economic evaluations are, according to the ‘scientific’ definition, CBAs. The ‘balance-sheet’ (or opportunity cost) approach is a form of CBA which can be used to identify who bears the costs and who reaps the benefits from any change. Whilst the next stage in a CBA, as defined in health economics, would require that all costs and benefits be valued in monetary terms, the balance-sheet approach, however, advocates that available monetary values can be augmented by other measures of cost and benefit. As such, this approach, which has a theoretical basis, is proposed as a practical prescription for CBA and highlights the notion that unquantified benefits are important and can be included within CBAs even when monetarisation is not possible. Recent methodological developments in monetary valuation for use in CBA are the development of the technique of willingness to pay, the use of conjoint analysis (CA) to elicit willingness-to-pay (WTP) values and advances in the debate on the inclusion of production gains in CBAs. Whilst acknowledging that there have been developments in each of these areas, it is claimed there has also been progress in using CBA as a framework for evaluation, as reflected by the balance-sheet approach. The paper concludes by stating that almost all types of economic evaluation have an element of the ‘cost-benefit’ approach in them. The important issue is to focus on the policy question to be addressed and to outline the relevant costs and benefits in a manner which assists the evaluation of welfare changes resulting from changes in healthcare delivery. The focus should not be on moulding a question to fit a hybrid definition of an analytical technique. [ABSTRACT FROM AUTHOR]

In many countries, including Thailand, there is an increasing impetus to use economic evaluation to allow more explicit and transparent healthcare priority setting. However, an important question for policy makers in low- and middle-income countries is whether it is appropriate and feasible to introduce economic evaluation data into healthcare priority-setting decisions. In addition to ethical, social and political issues, information supply challenges need to be addressed. This paper systematically reviewed the literature on economic evaluation of health technology in Thailand published between 1982 and 2005. Its aim was to analyse the quantity, quality and targeting of economic evaluation studies that can provide a framework for those conducting similar reviews in other settings. The review revealed that, although the number of publications reporting economic evaluations has increased significantly in recent years, serious attention needs to be given to the quality of reporting and analysis. Furthermore, there is an absence of economic evaluation publications for 15 of the top 20 major health problems in Thailand, indicating a poor distribution of research resources towards the determination of cost-effective interventions for diminishing the disease burden of certain major health problems. If economic evaluation is only useful for policy makers when performed correctly and reported accurately, these findings depict information barriers to using economic evaluation to assist health decision-making processes in Thailand. [ABSTRACT FROM AUTHOR]

Chronic hepatitis C virus (HCV) infection affects 170 million individuals worldwide. As it is detected incidentally through the evaluation of liver function tests or at the time of blood donor testing, it is usually clinically silent until the advanced stages of liver disease have occurred, when treatment is less effective and shortages of donor liver organs limit the therapeutic options. Combination therapy with ribavirin and pegylated interferon has resulted in sustained viral negative response rates of 54–61%. Because treatment is expensive and not uniformly effective, and because not all chronically infected patients will develop complications, concerns have arisen regarding the cost effectiveness of combination therapy.This paper reviews the public health and individual implications of HCV infections. Because of the latency of infection, numerous country-specific population analyses suggest that HCV will cause an increasing number of liver-related deaths over the next 10 years, despite the dramatic drop in incidence over the past 10–15 years. These deaths will be related to prevalent HCV infection from transfusion and injection drug use prior to identification of the virus and availability of screening tests in the late 1980s and early 1990s. HCV can reduce life expectancy and impair quality of life, yet not all patients will develop progressive liver disease, and antiviral treatment may have associated adverse effects.Finally, to assess the value of antiviral drugs for HCV infection, this paper reviews studies examining the costs of antiviral drugs and of the disease itself along with response to antiviral therapy and the cost effectiveness of antiviral therapy. Although antiviral therapy appears to be expensive, when also considering the likelihood of sustained viral response to therapy, and the cost savings, quality-of-life improvement and prolongation of life expectancy from the prevention of HCV complications, antiviral treatment for HCV appears to be cost effective when compared with other well accepted medical interventions. [ABSTRACT FROM AUTHOR]

The National Institute for Health and Care Excellence (NICE) invited the manufacturer of lenalidomide (Celgene) to submit evidence of the clinical and cost effectiveness of the drug for treating adults with myelodysplastic syndromes (MDS) associated with deletion 5q cytogenetic abnormality, as part of the Institute's single technology appraisal (STA) process. Kleijnen Systematic Reviews Ltd (KSR), in collaboration with Erasmus University Rotterdam, was commissioned to act as the Evidence Review Group (ERG). This paper describes the company's submission, the ERG review, and the NICE's subsequent decisions. The ERG reviewed the evidence for clinical and cost effectiveness of the technology, as submitted by the manufacturer to the NICE. The ERG searched for relevant additional evidence and validated the manufacturer's decision analytic model to examine the robustness of the cost-effectiveness results. Clinical effectiveness was obtained from a three-arm, European, randomized, phase III trial among red blood cell (RBC) transfusion-dependent patients with low-/intermediate-1-risk del5q31 MDS. The primary endpoint was RBC independence for ≥26 weeks, and was reached by a higher proportion of patients in the lenalidomide 10 and 5 mg groups compared with placebo (56.1 and 42.6 vs 5.9 %, respectively; both p < 0.001). The option of dose adjustments after 16 weeks due to dose-limiting toxicities or lack of response made long-term effectiveness estimates unreliable, e.g. overall survival (OS). The de novo model of the manufacturer included a Markov state-transition cost-utility model implemented in Microsoft Excel. The base-case incremental cost-effectiveness ratio (ICER) of the manufacturer was £56,965. The ERG assessment indicated that the modeling structure represented the course of the disease; however, a few errors were identified and some of the input parameters were challenged. In response to the appraisal documentation, the company revised the economic model, which increased the ICER to £68,125 per quality-adjusted life-year. The NICE Appraisal Committee (AC) did not recommend lenalidomide as a cost-effective treatment. Subsequently, the manufacturer submitted a Patient Access Scheme (PAS) that provided lenalidomide free of charge for patients who remained on treatment after 26 cycles. This PAS improved the ICER to £25,300, although the AC considered the proportion of patients who received treatment beyond 26 cycles, and hence the ICER, to be uncertain. Nevertheless, the AC accepted a commitment from the manufacturer to publish, once available, data on the proportion of patients eligible for the PAS, and believed this provided reassurance that lenalidomide was a cost-effective treatment for low- or intermediate-1-risk MDS patients. [ABSTRACT FROM AUTHOR]

The National Institute for Health and Care Excellence (NICE) invited Dendreon, the company manufacturing sipuleucel-T, to submit evidence for the clinical and cost effectiveness of sipuleucel-T for asymptomatic or minimally symptomatic, metastatic, non-visceral hormone-relapsed prostate cancer patients in whom chemotherapy is not yet clinically indicated, as part of NICE's single technology appraisal process. The comparator was abiraterone acetate (AA) or best supportive care (BSC). The School of Health and Related Research at the University of Sheffield was commissioned to act as the Evidence Review Group (ERG). This paper describes the company submission (CS), ERG review, and subsequent decision of the NICE Appraisal Committee (AC). The ERG produced a critical review of the clinical and cost-effectiveness evidence of sipuleucel-T based upon the CS. Clinical-effectiveness data relevant to the decision problem were taken from three randomised controlled trials (RCTs) of sipuleucel-T and a placebo (PBO) comparator of antigen-presenting cells (APC) being re-infused (APC-PBO) (D9901, D9902A and D9902B), and one RCT (COU-AA-302) of AA plus prednisone vs. PBO plus prednisone. Two trials reported a significant advantage for sipuleucel-T in median overall survival compared with APC-PBO: for trial D9901, an adjusted hazard ratio (HR) 0.47; (95 % confidence interval [CI] 0.29, 0.76) p < 0.002; for D9902B, adjusted HR 0.78 (95 % CI 0.61, 0.98) p = 0.03. There was no significant difference between groups in D9902A, unadjusted HR 0.79 (95 % CI 0.48, 1.28) p = 0.331. Sipuleucel-T and APC-PBO groups did not differ significantly in time to disease progression, in any of the three RCTs. Most adverse events developed within 1 day of the infusion, and resolved within 2 days. The CS included an indirect comparison of sipuleucel-T (D9902B) and AA plus prednisone (COU-AA-302). As trials differed in prior use of chemotherapy, an analysis of only chemotherapy-naïve patients was included, in which the overall survival for sipuleucel-T and AA was not significantly different, HR 0.94 (95 % CI 0.69, 1.28) p = 0.699. The ERG had several concerns regarding the data and assumptions incorporated within the company's cost-effectiveness analyses and conducted exploratory analyses to quantify the impact of making alternative assumptions or using alternative data inputs. The deterministic incremental cost-effectiveness ratio (ICER) for sipuleucel-T vs. BSC when using the ERG's preferred data and assumptions was £ 108,585 per quality-adjusted life-year (QALY) in the whole licensed population and £ 61,204/QALY in the subgroup with low prostate-specific antigen at baseline. The ERG also conducted an incremental analysis comparing sipuleucel-T with both AA and BSC in the chemotherapy-naïve subgroup. Sipuleucel-T had a deterministic ICER of £ 111,682/QALY in this subgroup, when using the ERG's preferred assumptions, and AA was extendedly dominated. The ERG also concluded that estimates of costs and benefits for AA should be interpreted with caution given the limitations of the indirect comparison. The AC noted that the ICER for sipuleucel-T was well above the range usually considered cost effective, and did not recommend sipuleucel-T for the treatment of asymptomatic or minimally symptomatic, metastatic, non-visceral hormone-relapsed prostate cancer. [ABSTRACT FROM AUTHOR]

Objectives: In Australia, no therapeutic agents were subsidised for the treatment of idiopathic pulmonary artery hypertension (iPAH), a rare progressive and severe disease with short life expectancy, until 1 March 2004, when bosentan (a dual endothelin receptor antagonist of high cost) was listed on the Pharmaceutical Benefits Scheme (PBS). Bosentan, in addition to conventional therapy, has been shown to slow iPAH progression and improve clinical and haemodynamic status and symptomatology, compared with placebo and conventional therapy. The objective of this paper is to describe the process of the Australian Pharmaceutical Benefits Scheme listing for bosentan (Tracleer), which included a health economic model assessing the cost effectiveness of bosentan from a healthcare payer perspective, and a risk-sharing arrangement based on the establishment of a patient registry.Methods: The health economic model predicted the cost, hospitalisation and mortality rates of a population of iPAH patients treated with either the conventional therapy regimen used in Australia or bosentan plus the conventional therapy regimen. The model was implemented as a first-order Monte Carlo simulation with mortality modelled directly as the main clinical outcome. The impacts of proposed continuation criteria, restricting the ongoing use of the drug, were evaluated. Costs and outcomes were discounted at 5% and a sensitivity analysis examined the robustness of the key assumptions.Results: The model predicted that after 5, 10 and 15 years, the difference in average cumulative costs between bosentan plus conventional therapy and conventional therapy alone would be 116,929 Australian dollars (A dollars), A181,808 dollars and A216,331 dollars for each patient, respectively. There would be an associated increase in average life expectancy of 1.39, 2.93 and 3.87 years at 5, 10 and 15 years, respectively, with an incremental cost-effectiveness ratio at 15 years of A55,927 dollars for each life-year gained. Removing the continuation criteria from the model increased the incremental cost-effectiveness ratio to A62,267 dollars (1996-2002 values).Conclusions: Economic modelling based on improved survival suggests bosentan to be a potentially cost-effective treatment for iPAH. However, the structure of the model and its inputs should be reviewed and updated as more data become available. [ABSTRACT FROM AUTHOR]

A number of recent findings from the literature imply that the value of a QALY varies depending on the concentration or dispersion of that QALY over treated individuals. Given that funding decisions are currently made under either the assumption of distributive-neutrality or some combination of explicit decision criteria and implicit adjustment for distributional concerns, it is likely that substantial social welfare gains are available if distributional objectives could be more accurately reflected in funding decisions. This paper considers three alternative approaches to explicitly adjust for distributional concerns with regard to the concentration or dispersion of individual health gains. Including non-health arguments in the objective function by 'weighting' QALYs for distributional effects or imposing differential funding thresholds for interventions with different distributional effects might be considered first- and second-best solutions, and would likely deliver the greatest social welfare gains. However, there is some doubt that first- or second-best solutions would be: (i) feasible given current data gaps; and (ii) politically acceptable. Rather, a simple and transparent approach is suggested wherein the sponsors of interventions that deliver health gains that are of questionable 'welfare-significance' for the treated individual would be required to provide decision-makers with an estimate of willingness to pay for the QALYs in question and would only be eligible for funding in the event that the positive net present value criterion is met. [ABSTRACT FROM AUTHOR]

Aromatase inhibitors (AIs) have been evaluated clinically in a wide range of breast cancer treatment settings. Although these agents appear to have clinical superiority, they are more expensive than the therapies (primarily tamoxifen) they have been compared with, thus economic evaluation is required to consider their incremental value to the payer. This paper reviews published economic evaluations of AIs as first-line therapy for advanced cancer, and as adjuvant therapy for early breast cancer. The evaluations in the advanced setting demonstrate a range of different modelling techniques and consider the payer's perspective in three healthcare systems. There is broad similarity in the application of standard cohort Markov modelling techniques to evaluate AIs in the early breast cancer setting, covering four separate health system perspectives. AIs appear cost effective compared with current practice. An analysis in the advanced setting suggests that letrozole may be the more cost-effective AI. No direct comparisons of alternative AIs in the adjuvant setting are reported, although indirect comparisons may be feasible. Future evaluations of treatment strategies over the entire course of the disease may also be needed. [ABSTRACT FROM AUTHOR]

Background: Rising public and private expenditure on antipsychotic medications is concentrated on the cost of second generation or 'atypical' medications, which are more expensive than first generation medications and make up a rapidly growing share of all antipsychotic prescriptions. Previous studies have examined whether the higher acquisition costs of atypicals are offset by other cost and/or utilisation benefits. This paper extends this literature by examining possible effects of atypicals on earnings and related measures of labour supply in a large naturalistic study with a long-term follow-up period.Methods: We analysed data on earnings and other characteristics from the Schizophrenia Care and Assessment Program (SCAP), a 3-year longitudinal study (with data collection during the years 1997-2003) of 2327 adults with schizophrenia (including schizoaffective and schizophreniform disorders) recruited from behavioural healthcare provider systems in six areas of the US. We used empirical criteria and data from the SCAP database to identify 336 patients aged < 50 years who were in the stable or 'maintenance' phase of their antipsychotic treatment during the 6 months prior to baseline. Effects of atypicals compared with typicals were estimated from Tobit regression models that included additional covariates and the baseline-dependent variable values. Regression-dependent variables were reported earnings per month, hours worked per month, days worked per month and a binary indicator of employment. To control for the effect of selection bias in choice of type of atypical, we employed an instrumental variables (IV) estimation procedure.Results: For all dependent variables, our IV Tobit regressions yielded consistently positive coefficient estimates for atypical use that were either marginally significant (p < 0.1) or significant (p < 0.05) for earnings, significant for hours and days of work and not as consistently significant for employment status. Results from these regressions imply a positive effect of atypical use on monthly earnings in the range of Dollars US 107-122. In regressions that did not control for selection bias by using IVs, coefficients for atypical use were often negative and never statistically significant.Conclusions: Our results indicate that higher drug costs of atypicals for maintenance-phase treatment are at least partially offset by higher earnings among patients. These effects represent benefits to consumers as well as savings to taxpayer-supported income transfer programmes. Future studies should seek to determine if treatment with atypicals increases patients' earnings via better control over negative symptoms and/or improved patient cognition. Both appear to be connected with employment and labour supply in patients with schizophrenia, and both may be improved through use of atypicals. [ABSTRACT FROM AUTHOR]

Objective: To estimate the cost effectiveness (from the UK NHS and personal social services perspective) of the cholinesterase inhibitors donepezil, rivastigmine and galantamine compared with usual care in the treatment of mild to moderately severe Alzheimer's disease. Patients had a mean age of 74 years, a mean disease duration of 1 year and a mean Alzheimer's disease assessment scale-cognitive subscale score of 24.Methods: A pharmacoeconomic model was used to predict long-term outcomes over a 5-year time horizon and to estimate the cost effectiveness of cholinesterase inhibitors for the management of Alzheimer's disease. The model structure is informed by a systematic review of the literature on the clinical and cost effectiveness of cholinesterase inhibitors and a review of the literature on the costs and outcomes associated with treatment for Alzheimer's disease. The main outcome measure used was the cost per quality-adjusted life-year (QALY) gained. All healthcare costs (excluding cholinesterase inhibitor costs) were indexed to pounds sterling (2003 values). Drug costs are 2005 values. Multivariate probabilistic sensitivity analysis and scenario analysis were undertaken to assess uncertainty in the results.Results: The clinical benefits on cognition from treatment with cholinesterase inhibitors resulted in an incremental cost per QALY gained ranging from 53,780 pounds sterling to 74,735 pounds sterling, over 5 years (vs usual care). Uncertainty analysis suggests that the probability of any of these treatments having an incremental cost per QALY of < 30,000 pounds sterling is < 21%. The key determinants of cost effectiveness were the effectiveness of treatment, the mean treatment cost and the cost savings associated with an expected delay in disease progression.Conclusions: Results presented in this paper suggest that the use of cholinesterase inhibitors may not be a cost-effective use of NHS resources. Guidance from the National Institute for Health and Clinical Effectiveness (NICE) in the UK on their judgements surrounding the acceptability of technologies as an effective use of resources, indicates there would need to be special reasons for accepting cholinesterase inhibitors as a cost-effective use of NHS resources. [ABSTRACT FROM AUTHOR]

The economic burden of influenza-related illness has been estimated to be 71.3-166 billion US dollars in the US, the majority of which is attributable to indirect costs as a result of lost productivity. There are currently four antiviral drugs available for the treatment of influenza: two ion channel blockers, amantadine and rimantadine; and two neuraminidase inhibitors, zanamivir and oseltamivir. The objective of this paper was to review the studies evaluating the cost effectiveness of currently available antiviral treatment and prophylaxis management strategies for influenza. Published studies that reported both costs and effectiveness of influenza management were extracted using MEDLINE, pre-MEDLINE and EMBASE. To facilitate a broad comparison, all costs were inflated to 2003 US dollars. Fifteen studies met the inclusion criteria of the review, with 14 analyses based on decision-analytic modelling and one economic analysis performed alongside a clinical trial. Management strategies included antiviral influenza prophylaxis or vaccination, empiric treatment of suspected disease, or antiviral treatment following rapid influenza testing. Study populations included healthy adults, adults at risk of influenza-related adverse outcomes, institutionalised and non-institutionalised elderly, and children. The comparator in all studies was standard care (i.e. over-the-counter medications only), and analyses were carried out from both the societal and payer perspectives. The only dominant strategy relative to standard care was vaccination of the institutionalised elderly. All other strategies in all populations were both more costly and more effective than standard care. Depending on the population and the perspective, the incremental cost-effectiveness ratios (ICERs) for antiviral treatment strategies ranged from 5000 US dollars/QALY for amantadine in test-and-treat studies to >400,000 US dollars/QALY for zanamivir or oseltamivir treatment in children. Sensitivity analysis in all studies consistently reported a strong influence of the population prevalence or diagnostic accuracy of influenza on the cost effectiveness of all strategies. Baseline influenza prevalence varied widely between studies, ranging from 15% to 68%. There was also a wide variation in the assumption about the disutility of influenza (ranging from -0.137 to -0.983 for the elderly requiring hospitalisation), which also impacted the cost effectiveness. Given the variation in the ICERs of antiviral treatment and prophylaxis, the uncertainty around many model parameters, and the dynamic nature of influenza from year to year, one can only conclude that antiviral treatment or prophylaxis for influenza is likely to be more cost effective in specific populations at specific times during the influenza season, and during influenza seasons when the population prevalence reaches epidemic levels or there is mismatch between the vaccine and the circulating virus. [ABSTRACT FROM AUTHOR]

The anogenital human papillomavirus (HPV) is estimated to be the most commonly occurring sexually transmitted infection in the US. Comprehensive estimates of the annual economic burden associated with the prevention and treatment of anogenital HPV-related disease in the US population are currently unavailable. The purpose of this paper is to (i) outline an analytic framework from which to estimate the annual economic burden of preventing and treating anogenital HPV-related disease in the US; (ii) review available US literature concerning the annual economic burden of HPV; and (iii) highlight gaps in current knowledge where further study is particularly warranted. Among eight US studies identified that describe the annual economic burden pertaining to one or more aspects of anogenital HPV-related disease, three met the review eligibility criteria (published between 1990 and 2004, examined multiple facets of annual anogenital HPV-related economic burden, and clearly articulated the data and methods used in the estimation process). All costs were adjusted to 2004 US dollars. Estimates of the annual direct medical costs associated with cervical cancer were comparable across studies (range 300-400 million US dollars). In contrast, there was a wide range across studies for estimates of the annual direct medical costs associated with cervical intraepithelial neoplasia (range 700 million US dollars-2.3 billion US dollars). Only one study reported direct medical costs for anogenital warts (200 million US dollars) and routine cervical cancer screening (2.3 billion US dollars). No studies examined direct medical costs attributable to HPV-related anal, penile, vaginal or vulvar cancers, or the work and productivity losses resulting from time spent receiving medical care, morbidity or mortality. Current economic burden estimates would suggest annual direct medical costs associated with the prevention and treatment of anogenital warts and cervical HPV-related disease of at least 4 billion US dollars. This figure would likely rise to at least 5 billion US dollars per year if direct medical costs associated with other disease entities caused by the sexual transmission of HPV were included, with further additions to the economic burden totalling in the billions of dollars if work and productivity losses were incorporated, a research priority for future studies. [ABSTRACT FROM AUTHOR]

To date, decision trees and Markov models have been the most common methods used in pharmacoeconomic evaluations. Both of these techniques lack the flexibility required to appropriately represent clinical reality. In this paper an alternative, more natural, way to model clinical reality--discrete event simulation--is presented and its application is illustrated with a real world example.A discrete event simulation represents the course of disease very naturally, with few restrictions. Neither mutually exclusive branches nor states are required, nor is a fixed cycle. All relevant aspects can be incorporated explicitly and efficiently. Flexibility in handling perspectives and carrying out sensitivity analyses, including structural variations, is incorporated and the entire model can be presented very transparently. The main limitations are imposed by lack of data to fit realistic models. Discrete event simulation, though rarely employed in pharmacoeconomics today, should be strongly considered when carrying out economic evaluations, particularly those aimed at informing policy makers and at estimating the budget impact of a pharmaceutical intervention. [ABSTRACT FROM AUTHOR]

There is a lack of emphasis on health-related QOL (HR-QOL) changes associated with acute exacerbation of chronic bronchitis (CB) or chronic obstructive pulmonary disease (COPD). The aim of this review is to examine the use of HR-QOL instruments to evaluate acute exacerbation of CB or COPD, so as to form recommendations for future research. A literature search of papers published between 1966 and July 2003 identified more than 300 articles that used acute exacerbation of CB or COPD as the search term. However, only 21 of these studies employed HR-QOL measures as predictors of outcome or in the assessment of the impact, evolution or treatment of acute exacerbations of COPD or CB. A variety of HR-QOL measures were used, both generic and disease specific. The disease-specific St George’s Respiratory Questionnaire (SGRQ), devised for patients with stable CB and with a recall period of 1–12 months, was the most widely used measure, with the Chronic Respiratory disease Questionnaire (CRQ) and the Baseline and Transitional Dyspnoea Index (BDI, TDI) being the only other disease-specific measures used. Most measures, both generic and disease specific, performed adequately when used during acute exacerbation of CB or COPD and indicated poor HR-QOL during acute exacerbation, which improved on resolution of the exacerbation. Relationships were evident between HR-QOL during an acute exacerbation and various outcomes, including post-exacerbation functional status, hospital re- admission for acute exacerbation or COPD, and mortality. There is a need for studies of treatments for acute exacerbation of CB or COPD to include an appropriate HR-QOL instrument to aid in the stratification of patients so as to target the right treatment to the right patient group. While a new instrument could be developed to measure HR-QOL during acute exacerbation of CB or COPD, currently available disease-specific measures such as the CRQ and the SGRQ appear to be acceptable to patients during acute exacerbation. However, the recall period of the SGRQ symptoms component should be shortened to make it more appropriate for use during acute exacerbation. [ABSTRACT FROM AUTHOR]

The resources available for healthcare are limited compared with demand, if not need, and all healthcare systems, regardless of their financing and organisation, employ mechanisms to ration or prioritise finite healthcare resources. This paper reviews alternative approaches that can be used to allocate healthcare resources. It discusses the problems encountered when allocating healthcare resources according to free market principles. It then proceeds to discuss the advantages and disadvantages of alternative resource allocation approaches that can be applied to public health systems. These include: (i) approaches based on the concept of meeting the needs of the population to maximising its capacity to benefit from interventions; (ii) economic approaches that identify the most efficient allocation of resources with the view of maximising health benefits or other measures of social welfare; (iii) approaches that seek to ration healthcare by age; and (iv) approaches that resolve resource allocation disputes through debate and bargaining. At present, there appears to be no consensus about the relative importance of the potentially conflicting principles that can be used to guide resource allocation decisions. It is concluded that whatever shape tomorrow's health service takes, the requirement to make equitable and efficient use of finite healthcare resources will remain. [ABSTRACT FROM AUTHOR]

The introduction of expensive disease-modifying agents for the treatment of multiple sclerosis (MS) has created the potential for patients with MS to become higher contributors to healthcare spending. In an attempt to make formulary and reimbursement choices for these agents, decision-makers may look to the literature for guidance. This critical review attempts to decipher a consistent message from the available economic literature regarding the relationship between disease severity and cost in MS. In the 2 studies that have examined MS disease severity, a positive correlation with total (direct and indirect) cost, indirect cost and some, if not all, components of direct cost was reported. In studies taking the societal perspective, the majority of total costs were indirect. This paper documents the high burden of MS on society and serves to guide the decision-maker in interpreting the MS economic literature such that this information can be optimally utilised to make informed resource allocation decisions. [ABSTRACT FROM AUTHOR]

Model-based economic evaluations should capture the impact on all costs and outcomes relevant to the chosen study perspective and time horizon. This editorial defines what is meant by direct treatment disutility (DTD) and describes why it could be an important harm that those designing model-based evaluations should consider. Some existing estimates of DTD identified from the current literature are summarised in terms of the methods used to elicit the values and the size of the estimated DTD. Model-based studies that include DTDs are also summarised. It was found that the values used within model-based economic evaluations (ranging from 0.00384 to 0.02) were typically smaller than the directly elicited values from the existing literature (0-0.033). Yet even with conservative estimates of DTDs, cost-effectiveness results were sensitive to their inclusion. The editorial concludes by discussing future methodological and empirical research needed to estimate more robust DTD values. [ABSTRACT FROM AUTHOR]

Objectives: This study provides the results of a cost-effectiveness analysis of levetiracetam as an adjunctive treatment for refractory epilepsy from the Canadian Ministry of Health perspective. The main objective is to estimate the expected cost-effectiveness ratio expressed as the incremental cost per seizure-free day gained when using levetiracetam. In addition, this study examines the potential savings that might result by reducing the number of surgical evaluations and surgery when using levetiracetam.Methods: A 1-year dose escalation decision-tree model comparing levetiracetam plus standard therapy with standard therapy alone was designed in order to combine probability, resource use and unit cost data (1999 Canadian dollars [$Can]). The short-term outcomes were derived from three phase III randomised, double-blind, placebo-controlled trials performed in 904 patients, aged 16-70 years, with at least 1 year history of epilepsy, two to four partial seizures per month, and receiving a maximum of two classic antiepileptic drugs.Results: The average gain in seizure-free days attributed to levetiracetam was 19 days per patient per year and the incremental cost-effectiveness ratio (ICER) for levetiracetam add-on in the base-case scenario was $Can80.7 per seizure-free day gained per patient per year. Moreover, when surgical investigation and surgery are considered in the model, the use of levetiracetam may be dominant, with substantial savings to the overall healthcare budget. All univariate sensitivity analyses show that the model was robust to the assumptions made.Conclusions: The economic analysis presented in this paper suggests, given a wide range of assumptions, that the increased cost of treating patients (with refractory epilepsy) with levetiracetam may be partially offset by a reduction in other direct medical costs (from the Canadian Ministry of Health perspective), as a consequence of an increase in the number of seizure-free days. Moreover, potential cost savings may be foreseen when it is assumed that levetiracetam may reduce the number of candidates for surgical evaluation and surgery through a reduction of seizure frequency. [ABSTRACT FROM AUTHOR]

In schizophrenia, modelling techniques may be needed to estimate the long-term costs and effects of new interventions. However, it seems that a simple direct link between symptoms and costs does not exist. Decisions about whether a patient will be hospitalized or admitted to a different healthcare setting are based not only on symptoms but also on social and environmental factors. This paper describes the development of a model to assess the dependencies between a broad range of parameters in the treatment of schizophrenia. In particular, the model attempts to incorporate social and environmental factors into the decision-making process for the prescription of new drugs to patients. The model was used to analyse the potential benefits of improving compliance with medication by 20% in patients in the UK. A discrete event simulation (DES) model was developed, to describe a cohort of schizophrenia patients with multiple psychotic episodes. The model takes into account the patient's sex, disease severity, potential risk of harm to self and society, and social and environmental factors. Other variables that change over time include the number of psychiatric consultations, the presence of psychotic episodes, symptoms, treatments, compliance, side-effects, the lack of ability to take care of him/herself, care setting and risk of harm. Outcomes are costs, psychotic episodes and symptoms. Univariate and multivariate sensitivity analyses were performed. Direct medical costs were considered (year of costing 2002), applying a 6.0% discount rate for costs and a 1.5% discount rate for outcome. The timeframe of the model is 5 years. When 50% of the decisions about the patient care setting are based on symptoms, a 20% increase in compliance was estimated to save 16,147 pounds and to avoid 0.55 psychotic episodes per patient over 5 years. Sensitivity analysis showed that the costs savings associated with increased compliance are robust over a range of variations in parameters. DES offers a flexible structure for modelling a disease, taking into account how a patient's history affects the course of the disease over time. This approach is particularly pertinent to schizophrenia, in which treatment decisions are complex. The model shows that better compliance increases the time between relapses, decreases the symptom score, and reduces the requirement for treatment in an intensive patient care setting, leading to cost savings. The extent of the cost savings depends on the relative importance of symptoms and of social and environmental factors in these decisions. [ABSTRACT FROM AUTHOR]

Decision makers increasingly require cost-effectiveness evidence to inform resource allocation and the need for systematic reviews of economic evaluations (SREEs) has grown accordingly. The objective of this article is to describe current practice and identify unique challenges in conducting and reporting SREEs. Current guideline documents for SREEs were consulted and summarised. A rapid review of English-language SREEs, using MEDLINE and EMBASE, published in 2017/2018, containing at least 20 studies was undertaken to describe current practice. Information on data extraction methods, quality assessment (QA) tools and reporting methods were narratively summarised. Lessons learned from a recently conducted SREE of weight loss interventions for severely obese adults were discussed. Sixty-three publications were included in the rapid review. Substantial heterogeneity in review methods, reporting standards and QA approaches was evident. Our recently conducted SREE on weight loss interventions identified scope to improve process efficiency, opportunity for more transparent and succinct reporting, and potential to improve consistency of QA. Practical solutions may include (1) using pre-piloted data extraction forms linked explicitly to results tables; (2) consistently reporting on key assumptions and sensitivity analyses that drive results; and (3) using checklists that include topic-specific items where relevant and allow reviewers to distinguish between reporting, justification and QA. The lack of a mutually agreed, standardised set of best practice guidelines has led to substantial heterogeneity in the conduct and reporting of SREEs. Future work is required to standardise the approach to conducting SREEs so that they can generate efficient, timely and relevant evidence to support decision-making. We suggest only data extracting information that will be reported, focusing discussion around the key drivers of cost-effectiveness, and improving consistency in QA by distinguishing between what is reported, justified by authors and deemed appropriate by the reviewer. [ABSTRACT FROM AUTHOR]

Background: An increasing number of technologies are obtaining marketing authorisation based on sparse evidence, which causes growing uncertainty and risk within health technology reimbursement decision making. To ensure that uncertainty is considered and addressed within health technology assessment (HTA) recommendations, uncertainties need to be identified, included in health economic models, and reported.Objective: Our objective was to develop the TRansparent Uncertainty ASsessmenT (TRUST) tool for systematically identifying, assessing, and reporting uncertainties in decision models, with the aim of making uncertainties and their impact on cost effectiveness more explicit and transparent.Methods: TRUST was developed by drawing on the uncertainty and risk assessment literature. To develop and validate this tool, we conducted HTA stakeholder discussion meetings and interviews and applied it in six real-world HTA case studies in the Netherlands and the UK.Results: The TRUST tool enables the identification and categorisation of uncertainty according to its source (transparency issues, methodology issues, and issues with evidence: imprecision, bias and indirectness, and unavailability) in each model aspect. The source of uncertainty determines the appropriate analysis. The impact of uncertainties on cost effectiveness is also assessed. Stakeholders found using the tool to be feasible and of value for transparent uncertainty assessment. TRUST can be used during model development and/or model review.Conclusion: The TRUST tool enables systematic identification, assessment, and reporting of uncertainties in health economic models and may contribute to more informed and transparent decision making in the face of uncertainty. [ABSTRACT FROM AUTHOR]

Background: Dengue disease poses a great economic burden in Malaysia.Methods: This study evaluated the cost effectiveness and impact of dengue vaccination in Malaysia from both provider and societal perspectives using a dynamic transmission mathematical model. The model incorporated sensitivity analyses, Malaysia-specific data, evidence from recent phase III studies and pooled efficacy and long-term safety data to refine the estimates from previous published studies. Unit costs were valued in $US, year 2013 values.Results: Six vaccination programmes employing a three-dose schedule were identified as the most likely programmes to be implemented. In all programmes, vaccination produced positive benefits expressed as reductions in dengue cases, dengue-related deaths, life-years lost, disability-adjusted life-years and dengue treatment costs. Instead of incremental cost-effectiveness ratios (ICERs), we evaluated the cost effectiveness of the programmes by calculating the threshold prices for a highly cost-effective strategy [ICER <1 × gross domestic product (GDP) per capita] and a cost-effective strategy (ICER between 1 and 3 × GDP per capita). We found that vaccination may be cost effective up to a price of $US32.39 for programme 6 (highly cost effective up to $US14.15) and up to a price of $US100.59 for programme 1 (highly cost effective up to $US47.96) from the provider perspective. The cost-effectiveness analysis is sensitive to under-reporting, vaccine protection duration and model time horizon.Conclusion: Routine vaccination for a population aged 13 years with a catch-up cohort aged 14-30 years in targeted hotspot areas appears to be the best-value strategy among those investigated. Dengue vaccination is a potentially good investment if the purchaser can negotiate a price at or below the cost-effective threshold price. [ABSTRACT FROM AUTHOR]

Objectives: This study responds to a request in the National Institute for Health and Care Excellence (NICE) guidance to assess the impact of using alternative sources of utility values, applied to multiple sclerosis (MS).Methods: Incremental cost-effectiveness ratios (ICERs) were calculated using utility values based on UK and Dutch values of EQ-5D, two UK mappings and one Dutch mapping of EQ-5D and two condition-specific instruments: the UK eight-dimensional Multiple Sclerosis Impact Scale (MSIS-8D) and the Dutch Multiple Sclerosis Impact Scale Preference-Based Measure (MSIS-PBM). Deterministic and Monte-Carlo simulation-based ICERs were estimated for glatiramer acetate versus symptom management using a lifetime Markov model.Results: For both UK and Dutch perspectives, mapped and condition-specific utility values expressed significantly higher quality of life for the worst health state of the model than did EQ-5D. The ICER of glatiramer acetate with EQ-5D was US$182,291 for The Netherlands and US$153,476 for the UK. Ratios for mapped and condition-specific utilities were between 20 and 60 % higher.Conclusion: The overestimation of quality of life of patients with MS by mapped EQ-5D or condition-specific utility values, relative to observed EQ-5D, increases the ICER substantially in a lifetime Markov model. [ABSTRACT FROM AUTHOR]

Background: Ulcerative colitis (UC) is the most common form of inflammatory bowel disease in the UK. Medical management aims to induce and maintain remission and to avoid complications and the necessity for surgical intervention. Colectomy removes the source of inflammation but is associated with morbidity and mortality. Newer anti-tumour necrosis factor (TNF)-α therapies may improve medical outcomes, albeit at an increased cost.Objective: Our objective was to assess the incremental cost effectiveness of infliximab, adalimumab and golimumab versus conventional therapy and surgery from a National Health Service (NHS) and Personal Social Services (PSS) perspective over a lifetime horizon.Methods: A Markov model was developed with health states defined according to whether the patient is alive or dead, current treatments received, history of colectomy and level of disease control. Transition probabilities were derived from network meta-analyses (NMAs) of trials of anti-TNF-α agents in the moderate-to-severe UC population. Health utilities, colectomy rates, surgical complications and resource use estimates were derived from literature. Unit costs were drawn from standard costing sources and literature and were valued at year 2013/2014 values.Results: For patients in whom surgery is an option, colectomy is expected to dominate all medical treatment options. For patients in whom colectomy is not an option, infliximab and golimumab are expected to be ruled out due to dominance, whilst the incremental cost-effectiveness ratio (ICER) for adalimumab versus conventional treatment is expected to be approximately £50,278 per quality-adjusted life-year (QALY) gained.Conclusions: Based on the NMAs, the ICERs for anti-TNF-α therapy versus conventional treatment or surgery are expected to be at best, in excess of £50,000 per QALY gained. The cost effectiveness of withdrawing biologic therapy upon remission and re-treating relapse is unknown. [ABSTRACT FROM AUTHOR]

Background: Trivalent influenza vaccines encompass one influenza B lineage; however, predictions have been unreliable on which of two antigenically distinct circulating lineages will dominate. Quadrivalent seasonal influenza vaccines contain strains from both lineages. This analysis assesses the cost effectiveness of switching from trivalent inactivated influenza vaccination (TIV) in Finland to quadrivalent vaccination, using inactivated (QIV) or live-attenuated (Q-LAIV) vaccines.Methods: A transmission model simulated the dynamics of influenza infection while accounting for indirect (herd) protection. Prior distributions for key transmission parameters were repeatedly sampled and simulations that fitted the available information on influenza in Finland were recorded. The resulting posterior parameter distributions were used in a probabilistic sensitivity analysis in which economic parameters were sampled, simultaneously encompassing uncertainty in the transmission and economic parameters. The cost effectiveness of a range of trivalent and quadrivalent vaccine policies over a 20-year time horizon was assessed from both a societal and payer perspective in 2014 Euros.Results: The simulated temporal incidence pattern of symptomatic infections corresponded well with case surveillance data. A switch from the current TIV to Q-LAIV in children (2 to <18 years) and to QIV in other ages was estimated to annually avert approximately 76,100 symptomatic infections (95 % range 36,700-146,700), 11,500 primary care consultations (6100-20,000), 540 hospitalisations (240-1180), and 72 deaths (32-160), and was cost-saving relative to TIV (€374 million averted [€161-€752], in 2014 Euros, discounted at 3 %). This scenario had the highest probability of being the most cost-effective scenario considered.Conclusions: This analysis demonstrates that quadrivalent vaccination is expected to be highly cost effective, reducing the burden of influenza-related disease. [ABSTRACT FROM AUTHOR]

Background: Chronic spontaneous urticaria (CSU) negatively impacts patient quality of life and productivity and is associated with considerable indirect costs to society.Objective: The aim of this study was to assess the cost utility of add-on omalizumab treatment compared with standard of care (SOC) in moderate or severe CSU patients with inadequate response to SOC, from the UK societal perspective.Methods: A Markov model was developed, consisting of health states based on Urticaria Activity Score over 7 days (UAS7) and additional states for relapse, spontaneous remission and death. Model cycle length was 4 weeks, and total model time horizon was 20 years in the base case. The model considered early discontinuation of non-responders (response: UAS7 ≤6) and retreatment upon relapse (relapse: UAS7 ≥16) for responders. Clinical and cost inputs were derived from omalizumab trials and published sources, and cost utility was expressed as incremental cost-effectiveness ratios (ICERs). Scenario analyses included no early discontinuation of non-responders and an altered definition of response (UAS7 <16).Results: With a deterministic ICER of £3183 in the base case, omalizumab was associated with increased costs and benefits relative to SOC. Probabilistic sensitivity analysis supported this result. Productivity inputs were key model drivers, and individual scenarios without early discontinuation of non-responders and adjusted response definitions had little impact on results. ICERs were generally robust to changes in key model parameters and inputs.Conclusions: In this, the first economic evaluation of omalizumab in CSU from a UK societal perspective, omalizumab consistently represented a treatment option with societal benefit for CSU in the UK across a range of scenarios. [ABSTRACT FROM AUTHOR]

The authors discuss the implications of the 2015 proposed revision of the Cancer Drugs Fund (CDF), established to provide a means by which National Health Service (NHS) patients could get cancer drugs that are not available on the NHS in Great Britain. They argues against the threats of the revised scheme on public health. They describe how the revised CDF could maximize the value of the benefit to cancer patients while minimizing its negative impact on the equity and efficiency of the NHS.

Background: In 2014, lurasidone, an atypical antipsychotic, was approved for the treatment of schizophrenia in adults. It is an alternative treatment option to aripiprazole, and when compared with aripiprazole, lurasidone was associated with improved symptom reduction and reduced risk of weight gain and relapse. We conducted a cost-utility analysis of lurasidone versus aripiprazole from the perspective of healthcare services, using Scotland and Wales as specific case studies.Methods: A 10-year Markov model, incorporating a 6-week acute phase and a maintenance phase across three health states (discontinuation, relapse, death) was constructed. Six-week probabilities of discontinuation and adverse events were based on a published independent mixed-treatment comparison; long-term risks of relapse and discontinuation were from an indirect comparison. Costs included drug therapy, relapse, and outpatient, primary and residential care. Costs and benefits were discounted at 3.5 %. Utility estimates were taken from published literature, and cost effectiveness was expressed as total 10-year incremental costs and quality-adjusted life-years (QALYs).Results: Lurasidone yielded a cost saving of £3383 and an improvement of 0.005 QALYs versus aripiprazole, in Scotland. Deterministic sensitivity analysis demonstrated that results were sensitive to relapse rates, while probabilistic sensitivity analysis suggested that lurasidone had the highest expected net benefit at willingness-to-pay thresholds of £20,000-30,000 per QALY. The probability that lurasidone was a cost-effective treatment strategy was approximately 75 % at all willingness-to-pay thresholds, with similar results being obtained for the Welsh analysis.Conclusions: Our analysis suggests that lurasidone would provide an effective, cost-saving alternative for the healthcare service in the treatment of adult patients with schizophrenia. [ABSTRACT FROM AUTHOR]

Background: A trade-off exists between building confidence in health-economic (HE) decision models and the use of scarce resources. We aimed to create a practical tool providing model users with a structured view into the validation status of HE decision models, to address this trade-off.Methods: A Delphi panel was organized, and was completed by a workshop during an international conference. The proposed tool was constructed iteratively based on comments from, and the discussion amongst, panellists. During the Delphi process, comments were solicited on the importance and feasibility of possible validation techniques for modellers, their relevance for decision makers, and the overall structure and formulation in the tool.Results: The panel consisted of 47 experts in HE modelling and HE decision making from various professional and international backgrounds. In addition, 50 discussants actively engaged in the discussion at the conference workshop and returned 19 questionnaires with additional comments. The final version consists of 13 items covering all relevant aspects of HE decision models: the conceptual model, the input data, the implemented software program, and the model outcomes.Conclusions: Assessment of the Validation Status of Health-Economic decision models (AdViSHE) is a validation-assessment tool in which model developers report in a systematic way both on validation efforts performed and on their outcomes. Subsequently, model users can establish whether confidence in the model is justified or whether additional validation efforts should be undertaken. In this way, AdViSHE enhances transparency of the validation status of HE models and supports efficient model validation. [ABSTRACT FROM AUTHOR]

As part of its single technology appraisal (STA) process, the National Institute for Health and Care Excellence (NICE) invited the manufacturer of vedolizumab (Takeda UK) to submit evidence of the clinical effectiveness and cost effectiveness of vedolizumab for the treatment of patients with moderate-to-severe active ulcerative colitis (UC). The Evidence Review Group (ERG) produced a critical review of the evidence for the clinical effectiveness and cost effectiveness of the technology, based upon the company's submission to NICE. The evidence was derived mainly from GEMINI 1, a Phase 3, multicentre, randomised, double-blinded, placebo-controlled study of the induction and maintenance of clinical response and remission by vedolizumab (MLN0002) in patients with moderate-to-severe active UC with an inadequate response to, loss of response to or intolerance of conventional therapy or anti-tumour necrosis factor (TNF)-α. The clinical evidence showed that vedolizumab performed significantly better than placebo in both the induction and maintenance phases. In the post hoc subgroup analyses in patients with or without prior anti-TNF-α therapy, vedolizumab performed better then placebo (p value not reported). In addition, a greater improvement in health-related quality of life was observed in patients treated with vedolizumab, and the frequency and types of adverse events were similar in the vedolizumab and placebo groups, but the evidence was limited to short-term follow-up. There were a number of limitations and uncertainties in the clinical evidence base, which warrants caution in its interpretation--in particular, the post hoc subgroup analyses and high dropout rates in the maintenance phase of GEMINI 1. The company also presented a network meta-analysis of vedolizumab versus other biologic therapies indicated for moderate-to-severe UC. However, the ERG considered that the results presented may have underestimated the uncertainty in treatment effects, since fixed-effects models were used, despite clear evidence of heterogeneity among the trials included in the network. Results from the company's economic evaluation (which included price reductions to reflect the proposed patient access scheme for vedolizumab) suggested that vedolizumab is the most effective option compared with surgery and conventional therapy in the following three populations: (1) a mixed intention-to-treat population, including patients who have previously received anti-TNF-α therapy and those who are anti-TNF-α naïve; (2) patients who are anti-TNF-α naïve only; and (3) patients who have previously failed anti-TNF-α therapy only. The ERG concluded that the results of the company's economic evaluation could not be considered robust, because of errors in model implementation, omission of relevant comparators, deviations from the NICE reference case and questionable model assumptions. The ERG amended the company's model and demonstrated that vedolizumab is expected to be dominated by surgery in all three populations. [ABSTRACT FROM AUTHOR]

Objectives: The Hospital Episode Statistics (HES) dataset is a source of administrative 'big data' with potential for costing purposes in economic evaluations alongside clinical trials. This study assesses the validity of coverage in the HES outpatient dataset.Methods: Men who died of, or with, prostate cancer were selected from a prostate-cancer screening trial (CAP, Cluster randomised triAl of PSA testing for Prostate cancer). Details of visits that took place after 1/4/2003 to hospital outpatient departments for conditions related to prostate cancer were extracted from medical records (MR); these appointments were sought in the HES outpatient dataset based on date. The matching procedure was repeated for periods before and after 1/4/2008, when the HES outpatient dataset was accredited as a national statistic.Results: 4922 outpatient appointments were extracted from MR for 370 men. 4088 appointments recorded in MR were identified in the HES outpatient dataset (83.1%; 95% confidence interval [CI] 82.0-84.1). For appointments occurring prior to 1/4/2008, 2195/2755 (79.7%; 95% CI 78.2-81.2) matches were observed, while 1893/2167 (87.4%; 95% CI 86.0-88.9) appointments occurring after 1/4/2008 were identified (p for difference <0.001). 215/370 men (58.1%) had at least one appointment in the MR review that was unmatched in HES, 155 men (41.9%) had all their appointments identified, and 20 men (5.4%) had no appointments identified in HES.Conclusions: The HES outpatient dataset appears reasonably valid for research, particularly following accreditation. The dataset may be a suitable alternative to collecting MR data from hospital notes within a trial, although caution should be exercised with data collected prior to accreditation. [ABSTRACT FROM AUTHOR]

In the era of the Information Age and personalized medicine, healthcare delivery systems need to be efficient and patient-centred. The health system must be responsive to individual patient choices and preferences about their care, while considering the system consequences. While dynamic simulation modelling (DSM) and big data share characteristics, they present distinct and complementary value in healthcare. Big data and DSM are synergistic-big data offer support to enhance the application of dynamic models, but DSM also can greatly enhance the value conferred by big data. Big data can inform patient-centred care with its high velocity, volume, and variety (the three Vs) over traditional data analytics; however, big data are not sufficient to extract meaningful insights to inform approaches to improve healthcare delivery. DSM can serve as a natural bridge between the wealth of evidence offered by big data and informed decision making as a means of faster, deeper, more consistent learning from that evidence. We discuss the synergies between big data and DSM, practical considerations and challenges, and how integrating big data and DSM can be useful to decision makers to address complex, systemic health economics and outcomes questions and to transform healthcare delivery. [ABSTRACT FROM AUTHOR]

Background: Prognostic classification approaches are commonly used in clinical practice to predict health outcomes. However, there has been limited focus on use of the general approach for predicting costs. We applied a grouping algorithm designed for large-scale data sets and multiple prognostic factors to investigate whether it improves cost prediction among older Medicare beneficiaries diagnosed with prostate cancer.Methods: We analysed the linked Surveillance, Epidemiology and End Results (SEER)-Medicare data, which included data from 2000 through 2009 for men diagnosed with incident prostate cancer between 2000 and 2007. We split the survival data into two data sets (D0 and D1) of equal size. We trained the classifier of the Grouping Algorithm for Cancer Data (GACD) on D0 and tested it on D1. The prognostic factors included cancer stage, age, race and performance status proxies. We calculated the average difference between observed D1 costs and predicted D1 costs at 5 years post-diagnosis with and without the GACD.Results: The sample included 110,843 men with prostate cancer. The median age of the sample was 74 years, and 10% were African American. The average difference (mean absolute error [MAE]) per person between the real and predicted total 5-year cost was US$41,525 (MAE US$41,790; 95% confidence interval [CI] US$41,421-42,158) with the GACD and US$43,113 (MAE US$43,639; 95% CI US$43,062-44,217) without the GACD. The 5-year cost prediction without grouping resulted in a sample overestimate of US$79,544,508.Conclusion: The grouping algorithm developed for complex, large-scale data improves the prediction of 5-year costs. The prediction accuracy could be improved by utilization of a richer set of prognostic factors and refinement of categorical specifications. [ABSTRACT FROM AUTHOR]

We reviewed the clinical measures used in rheumatoid arthritis (RA) economic evaluations with respect to their relevance and sensitivity to changes in survival, health-related quality of life (HR-QOL) and costs. We compared the measures from the economic perspective and discussed the validity of methods used to extrapolate beyond the trial data. Cost-effectiveness evaluations of disease-modifying antirheumatic drugs in RA were identified by searching MEDLINE, EMBASE, Econlit and NHS EED databases. Studies were retained if they extrapolated beyond randomized controlled trial evidence using relationships between clinical measures, costs and utilities.In the 22 studies identified, clinical severity was measured using the Health Assessment Questionnaire (HAQ) Disability Index, the American College of Rheumatology (ACR) response criteria, the Disease Activity Score (DAS) or a combination of the HAQ and DAS. The HAQ is correlated with mortality, costs and HR-QOL instruments, and several studies used linear relationships to model these associations. However, a polynomial relationship or discrete states may be more appropriate for patients at the extremes of the disease spectrum, and numerous HAQ health states may be required to capture differences in mortality risk. While the ACR response criteria is a more comprehensive measure than the HAQ, it is a relative measure, which creates difficulties when estimating absolute changes in HR-QOL, costs and mortality risk. The evidence base linking DAS scores with HR-QOL instruments, costs and mortality is less robust, possibly due to the comparatively recent development of the measure and the limited number of possible scores (mild/moderate/severe). While there is some evidence of a relationship between DAS scores and costs, the DAS does not capture all aspects of HR-QOL, and no significant relationship has been established with mortality risk.Evidence suggests the HAQ to be the primary clinical measure for use in economic evaluations as it is measured in almost all clinical studies, and is closely correlated to health utilities, mortality and costs. While new developments suggest the sensitivity of health states may be improved by combining the HAQ with measures such as the DAS, further research is required in this area. Further research is also required to explore the advantages in using either continuous or discrete health states. [ABSTRACT FROM AUTHOR]

Objective: To determine the cost utility of treating schizophrenic patients with olanzapine compared with other antipsychotics in a naturalistic outpatient setting.Methods: The pan-European SOHO study is a 3-year, prospective, outpatient, observational study of outcomes associated with antipsychotic treatment, focusing on olanzapine, in ten European countries. For the cost-utility analysis, healthcare resource use (inpatient care, day care, outpatient psychiatric consultations and antipsychotic and concomitant medication use) and EQ-5D data were collected at baseline and at 3, 6 and 12 months. The perspective was that of the health service payer. UK healthcare unit costs (year 2004 values) were applied to the resource use data for the ten countries. UK population tariffs were applied to the EQ-5D data to determine utility values.An Epoch analysis was used to analyze the longitudinal data. Multivariate regression analyses that adjusted for baseline covariates were used to estimate the incremental cost and utility gains for patients treated with olanzapine compared with each of the other antipsychotics (risperidone, quetiapine, amisulpride, clozapine and oral or depot typical antipsychotics).Results: A total of 10 972 patients were enrolled at baseline, of which 9107 completed the 12-month study period. Treatment with olanzapine was more effective in terms of QALYs gained than all of the other antipsychotic treatments. Treatment with olanzapine dominated quetiapine and amisulpride. The incremental cost for olanzapine compared with risperidone was pound sterling 226 per patient over 12 months and the incremental cost per QALY gained was pound 5156, with bootstrap analyses showing 100% of the replications falling below a pound sterling 30 000 per QALY gained threshold. Compared with treatment with clozapine, olanzapine was found to be marginally more effective, at an additional cost of pound sterling 13 per patient over 12 months and to have an incremental cost per QALY gained of pound sterling 775. Bootstrap analyses showed that 81% of replications fell below a pound sterling 30 000 per QALY gained threshold. Comparing olanzapine with oral and depot typical antipsychotics, the incremental cost was pound sterling 849 and pound sterling 1106 per patient over 12 months and the incremental cost per QALY gained was pound sterling 15 696 and pound sterling 23 331, respectively. Bootstrap analyses showed that 98% of the replications fell below a pound sterling 30 000 per QALY gained threshold for the comparison with oral typical antipsychotics, and 79% of replications for the comparison with depot preparations.Conclusions: Among SOHO patients, if a funding threshold of pound sterling 30 000 per QALY gained is assumed, this analysis suggests that olanzapine has a high probability of being the most cost-effective treatment compared with other antipsychotic treatments. However, comparison of olanzapine with clozapine and typical depot antipsychotics should be viewed with caution because clozapine is a second-line treatment and depot treatment is used for patients who do not adhere to their oral medication. [ABSTRACT FROM AUTHOR]

Background: Palivizumab is one of the most costly paediatric medications for Medicaid and other health plans. It is uncertain whether the costs of palivizumab administration are justified in specific risk groups. Ongoing investigations of palivizumab require identification of reliable data sources.Objective: To estimate the reliability between Medicaid paid claims data and medical records documentation in a cost analysis of palivizumab.Study Design: A cross-sectional study of data reliability was performed within a cost analysis study of palivizumab in a cohort of at-risk patients during the US 2002/3 respiratory syncytial virus season. Exposure classification (to palivizumab prophylaxis) was compared using Medicaid claims and medical records data.Methods: The study was performed in 28 widely dispersed paediatric medical practices serving North Carolina, USA Medicaid patients within the AccessCare/Community Care of North Carolina (CCNC) Program, an enhanced primary care case management programme. Patients were eligible if they were born between 1 March 2002 and 28 February 2003 at 32-35 weeks estimated gestational age, were enrolled in the North Carolina primary care case management Medicaid programme during the study period and were patients of the participating practices. Medicaid healthcare claims were obtained in December 2003 for services provided between October 2002 and May 2003. Medical records were abstracted by community-based case managers. Primary variables included frequency, dates and dose of palivizumab injections. The main outcomes measures were agreement in the number of palivizumab injections, dates of administration and doses of palivizumab between Medicaid paid claims and medical record data.Results: Injection frequencies matched between medical record and Medicaid claims data for only 46.2% of study participants. Congruence in injection service dates occurred between data sources for only 1% of injections. Doses were similar between data sources for 81.9% of injections.Conclusions: In Medicaid recipients receiving palivizumab injection, Medicaid claims data were inconsistent with medical records data. Use of multiple data sources and validation are recommended to identify temporal relationships between drug administration and endpoints of interest. [ABSTRACT FROM AUTHOR]

Introduction: The Functional Assessment of Cancer Therapy-Lung (FACT-L) is a validated, sensitive and reliable patient questionnaire that evaluates and quantifies quality of life (QOL) across several domains, including lung cancer-related symptoms. The FACT-L was not designed for use in economic evaluation and does not incorporate preferences into its scoring system.Objective: To derive a set of Dutch preference weights for FACT-L health states that can be used to convert FACT-L into a single value that can be used in cost-utility analyses.Methods: A representative sample of the Dutch population (n = 1076) directly valued an orthogonal set of eight FACT-L health states on a 100-point rating scale with the anchor points 'worst imaginable health state' and 'best imaginable health state'. Eleven FACT-L items were selected to describe the FACT-L health states that were directly valued. Regression analysis was used to interpolate values for all other possible health states. Scores were transformed into values on a scale where 0 indicated dead and 1 indicated full health.Results: The estimated values for FACT-L health states ranged from 0.08 to 0.93. The estimated value sets were applied to FACT-L data of lung cancer patients participating in a clinical study. Significant differences in the mean value and mean gain of 0.12 and 0.07, respectively, were found between patients in remission and patients with progressive disease at 4 weeks' follow-up.Conclusion: Our results reaffirmed that the methodology used here is a feasible option to convert data collected with a disease-specific outcome measure into preferences. We concluded that the sensitivity of the derived set of societal preferences to capture differences and changes in clinical health states is an indication of its construct validity. [ABSTRACT FROM AUTHOR]

Background: Oseltamivir is effective in the treatment of influenza. Utilisation in The Netherlands is limited, but increasing.Objective: To estimate the cost effectiveness of oseltamivir treatment (vs symptom relief only) for patients with influenza-like illness (ILI) who are at increased risk for serious complications of influenza.Methods: A cost-effectiveness analysis was used, building on a previously developed model (decision tree) that was applied for evaluating influenza vaccination and pandemic preparedness plans. Three patient subgroups were assessed (elderly patients [aged > or = 65 years] without chronic disease, elderly patients with chronic disease, and chronically ill, non-elderly patients). Inputs for the model were taken from various sources including a meta-analysis. A societal perspective was adopted and costs were expressed in euro per life-year gained (year 2003 values). Life-years lost were discounted at 4% in accordance with Dutch guidelines. Deterministic and probabilistic sensitivity analyses were employed to assess the robustness of the results.Results: For chronically ill patients with ILI, visits to the GP for oseltamivir treatment were cost saving. For non-chronically ill elderly patients, incremental cost-effectiveness was estimated at 1759 euros per life-year gained. Cost savings and favourable cost effectiveness were robust in a deterministic and stochastic sensitivity analysis.Conclusion: Our model-based analysis suggests that at-risk people presenting with ILI to a GP could be offered oseltamivir at favourable cost effectiveness or even cost savings in the Dutch setting compared with symptom relief with analgesics only. [ABSTRACT FROM AUTHOR]

Background: There are many effective interventions, via various routes (intravenous [IV], oral [OR], intravitreal injection [IVT] and intraocular implantation [IMP]), for treating cytomegalovirus retinitis (CMVR) that have become available. There are large variations in treating CMVR in clinical practice in Thailand.Objective: To evaluate the incremental cost-effectiveness ratio (ICER) of providing (i) IVT, (ii) IV/OR and (iii) IMP ganciclovir to patients with HIV/AIDS and CMVR versus providing no treatment.Design: A simulation study for which the input parameters were derived from a systematic review of the literature, a hospital-based survey and patient interviews.Setting: The analysis assumed a Thai healthcare system perspective. However, the model was run using both societal and healthcare provider perspectives.Results: Our results suggest that IVT ganciclovir was cost effective and the best option for treating patients with CMVR irrespective of whether patients received antiretroviral treatment (ART). In patients receiving ART, moving from IVT to IV/OR ganciclovir was also likely to be a cost-effective option. Offering IMP ganciclovir was not likely to be cost effective. Providing treatments for patients with bilateral CMVR was more cost effective than providing treatments for those with unilateral CMVR, and offering treatments for patients receiving ART was better value for money than treating patients without ART.Conclusions: Our models suggest that IV/OR ganciclovir should be recommended for the treatment of unilateral and bilateral CMVR for patients receiving ART in the Thai healthcare system. IVT ganciclovir may also have a role in the treatment of CMVR patients not receiving ART. [ABSTRACT FROM AUTHOR]

BACKGROUND: Olanzapine and risperidone are two commonly prescribed atypical antipsychotics for schizophrenia. Prior studies have shown inconsistent results in terms of advantage in cost saving in prescribing these agents. Our preliminary analysis showed that a small percentage of intensive healthcare utilizers had substantial impact on healthcare costs. This study analysed the cost effects of olanzapine and risperidone among those who had intensive utilisation of medical care prior to drug initiation, and the relationship between the choice of the two drugs and patients’ co-morbid condition.Based on the records of 18 499 patients with schizophrenia prescribed either olanzapine or risperidone between 1 April 1999 and 31 March 2000 we defined intensive users of healthcare as those who incurred an annual total cost in the top tenth percentile.We measured co-morbidity by number of diseases, and healthcare costs ($US, 1998–2001 values) in the year prior and the year after treatment initiation in six categories of care (inpatient medical/surgical, inpatient psychiatric care, other inpatient, outpatient psychiatric care, other outpatient and outpatient pharmacy). METHODS: We retrospectively investigated patients first treated with either risperidone or olanzapine between 1 April 1999 and 31 March 2000. According to patients’ medication history during the 6 months prior to initial prescription of a study medication we categorised patients into three groups: (i) not receiving olanzapine or risperidone; (ii) not receiving any atypical antipsychotic agent; or (iii) not receiving any antipsychotic agent. We then compared the difference in cost saving by type of care in the 10% most expensive patients through bivariate and multivariate analyses.Based on the records of 18 499 patients with schizophrenia prescribed either olanzapine or risperidone between 1 April 1999 and 31 March 2000 we defined intensive users of healthcare as those who incurred an annual total cost in the top tenth percentile.We measured co-morbidity by number of diseases, and healthcare costs ($US, 1998–2001 values) in the year prior and the year after treatment initiation in six categories of care (inpatient medical/surgical, inpatient psychiatric care, other inpatient, outpatient psychiatric care, other outpatient and outpatient pharmacy). RESULTS: The top 10% most expensive patients accounted for about half of the total cost of the entire cohort and had nearly a 40% cost reduction for the year after treatment initiation versus the prior year, while the entire cohort increased cost between 2% and 12%. Compared with those prescribed olanzapine, patients prescribed risperidone had more medical co-morbidities, higher inpatient medical/surgical costs and lower psychiatric costs. Patients taking olanzapine had greater cost reduction in inpatient psychiatric care, whereas those taking risperidone had greater reduction in inpatient medical/surgical care. CONCLUSIONS: Among the top 10% most expensive patients, olanzapine and risperidone treatments were associated with comparable cost reductions in inpatient care. The choice of agent was associated with patients’ co-morbid condition and was correlated with cost reduction in inpatient medical/surgical or psychiatric care. [ABSTRACT FROM AUTHOR]

This article reviews the development of economic evaluation of health technologies in the UK and its impact on decision making. After a long period of limited impact from studies mainly carried out as academic exercises, the advent of the National Institute for Health and Clinical Excellence (NICE) in 1999 provided a transparent decision-making context where economic evaluation plays a central role. This article reviews some of the key characteristics about the way NICE works, for example, the way NICE has defined the form of analysis that it requires, reflecting its objective of maximising health gain (QALYs) from the predetermined and limited UK NHS budget.Two broad areas of widespread concern are noted. The first relates to the cost-effectiveness thresholds that NICE uses and the basis for them. The second is the patchy implementation of NICE guidance and the possible reasons for this. But even within the UK, NICE is the exception in making extensive and explicit use of economic evaluation and this article goes on to suggest that if there is to be a more widespread and consistent use of economic evaluation at both central and local levels, then health economists and others need to address three issues.The first is to be clear about what is the correct conceptual basis for determining the cost-effectiveness threshold and then to ensure that NICE has the empirical evidence to set it appropriately. The second is to recognise that even using the limited view of costs adopted by NICE, economic evaluations imply temporal and cross-service budgetary flexibility that the NHS locally does not in practice enjoy. The third issue is that with academic pressures for ever-increasing sophistication of ‘state of the art’ economic evaluation analysis, the NHS has more and more precise understanding of the cost effectiveness of just a few new technologies and little or no analysis of most. This limits the value of the former by reducing further the scope for appropriately disinvesting from cost-ineffective technologies to meet the additional costs of investing in cost-effective new ones.Whilst NICE stands out as an example of a context where high-quality economic evaluation plays a major role in decision making, the process is far from perfect and certainly is not representative of the use made of economic evaluation by the NHS as a whole. Health economists need to engage with the public and the health service to better understand their perspectives, rather than focusing on academic concerns relating to details of theory and analytical method. [ABSTRACT FROM AUTHOR]

Introduction: Inflammatory bowel disease (IBD) is a chronic condition that afflicts young adults in their economically productive years. The goal of this study was to determine the costs of IBD in Germany from a societal perspective, using cost diaries.Methods: Members of the German Crohn's Disease and Ulcerative Colitis Association who had IBD were recruited by post, and those who agreed to participate documented their IBD-associated costs prospectively in a diary over 4 weeks. They documented their use of healthcare facilities, medications, sick leave and out-of-pocket expenditures, as well as general demographic information, the status and history of their IBD, and long-term disability. Item costs were calculated according to national sources. Cost data were calculated using non-parametric bootstrapping and presented as mean costs (year 2004) over 4 weeks.Results: The cost diaries were returned by 483 subjects (Crohn's disease: n = 241, ulcerative colitis: n = 242) with a mean age of 42 years and an average disease duration of 13 years (SD +/- 8.09). The cost diaries were regarded as 'easy to complete' by 89% of participants. The mean 4-week costs per subject were 1,425 Euros(95% CI 1201, 1689) for Crohn's disease and 1,015 Euros(95% CI 832, 1258) for ulcerative colitis. Of the total costs for Crohn's disease, 64% were due to indirect costs such as early retirement or sick leave and 32% were due to direct medical costs. In contrast, of the total costs for ulcerative colitis, 41% were due to direct medical costs and 54% to indirect costs.Conclusions: This is the first comprehensive cost study for Crohn's disease and ulcerative colitis in Germany. The most important economic factors that influenced the cost profiles of both diseases were the long-term productivity losses due to an ongoing inability to work and the cost of medications. Results indicate significant cost differences between Crohn's disease and ulcerative colitis. This data provides initial cost estimates that can be analysed further with respect to cost determinants and disease-specific costs in the future. [ABSTRACT FROM AUTHOR]

Background: MA17 was a randomised placebo-controlled trial of letrozole 2.5 mg/day in 5187 estrogen receptor-positive, 50% node-negative, postmenopausal women (median age 62 years at enrollment) with early breast cancer, post-5 years' adjuvant tamoxifen therapy. The objective of this evaluation was to extrapolate the findings from the MA17 trial to estimate the lifetime cost effectiveness of letrozole in this setting.Methods: A Markov model was used to estimate the incremental cost per QALY gained with extended adjuvant letrozole versus no therapy. Probabilities of disease progression and death were estimated using data from the MA17 study and other secondary sources. Costs of breast cancer care (letrozole therapy, surveillance, recurrences, terminal care) and treatment of osteoporosis and utilities were derived from literature. A full probabilistic sensitivity analysis was undertaken. The analysis was conducted from the perspective of the UK National Health Service (NHS) and cost estimates reflect 2004 values. All costs and outcomes were discounted at 3.5%.Results: Extended adjuvant letrozole resulted in a gain of 0.36 QALYs per patient (13.66 vs 13.30 with no therapy). These benefits were obtained at an additional expected lifetime cost of 3732 pounds per patient (10,833 pounds letrozole vs 7101 pounds with no therapy). Cost effectiveness was estimated at 10,338 pounds per QALY gained (95% CI 5276, 43,828). The results were robust to sensitivity analyses.Conclusion: Five years of letrozole therapy appears to be cost effective from the NHS perspective and should be considered in women with early breast cancer, following tamoxifen adjuvant therapy. [ABSTRACT FROM AUTHOR]

Background: Streptococcus pneumoniae and Neisseria meningitidis group B are among the main causes of invasive bacterial meningitis infections in infants. Worldwide, these diseases lead to significant mortality, morbidity and costs. The societal impact is especially severe since the majority of cases occur in very young infants. A combination vaccine consisting of 9-valent conjugated pneumococcal and meningococcal B components is currently being developed. The aim of this study was to estimate the potential impact and cost effectiveness from the societal perspective of vaccinating infants in The Netherlands with this combination pneumococcal and meningococcal B vaccine versus no vaccination.Methods: A Markov cycle model was developed using epidemiological and healthcare resource use data from 1996 to 2001. This model was used to project the annual costs, benefits and health gains associated with vaccinating all newborns. The base year for the costing was 2003 and all costs and health effects were discounted at 4%. The results of the analysis are expressed in costs per QALY and both probabilistic and univariate sensitivity analyses were used to identify the robustness of the results.Results: Annually, an average of 755 cases of invasive pneumococcal and meningococcal B infection occurred in infants aged 0-10 years in The Netherlands. Introduction of the combination vaccine would prevent 201 cases of meningococcal B meningitis and 165 cases of invasive pneumococcal disease per year. Additionally, 3410 cases of pneumococcal pneumonia and 46,350 cases of otitis media would be prevented. Vaccination would save 35 lives per year and prevent 71 cases of severe sequelae. This translates into 860 life-years gained, or 1128 QALYs gained. Alongside these health gains, vaccination would prevent euro 17,681,370 of direct medical and indirect costs attributable to meningococcal and pneumococcal infections in The Netherlands. Depending on vaccine price, cost effectiveness varied from euro 3160 (vaccine price per dose euro 20) to euro 32,170 (vaccine price euro 60 per dose) per QALY. Base-case cost effectiveness (vaccine price euro 40) was euro 17,700 per QALY. The model was most sensitive to changes in incidence, vaccine price and duration of protective efficacy.Conclusion: Our results suggest that the introduction of a combination meningococcal B and pneumococcal vaccine into the Dutch infant vaccination programme is potentially cost effective compared with no vaccination. [ABSTRACT FROM AUTHOR]

Introduction: Visual impairment is a severe disability that puts a heavy burden on individuals, families and society. In developed countries, the two major diseases leading to irreversible visual impairment are glaucoma and age-related macular degeneration. Their prevalence will increase dramatically with population aging. The economic consequences of visual impairment are considerable, but have rarely been documented, apart from some ‘top-down’ estimates based on national statistics. We estimated the non-medical costs related to visual impairment in four European countries: France, Italy, Germany and the UK. Methods: Prevalence rates of visual impairment, defined according to local regulations, were taken from national registers and, for France, from two recent nationwide surveys conducted by the French Institute for National Statistics and Economic Studies (Institut National de la Statistique et des Etudes Economiques [INSEE]). Estimates of the number of non-registered persons were obtained from the literature and expert opinion. Estimates of non-medical costs included institutional care, non-medical devices, residential adaptations, burden on carer, paid home help, loss of income and social allowances related to visual impairment. Unit costs (year 2004) were extracted from national databases and manufacturers. Healthcare professionals were interviewed to estimate the duration of assistance required by visually impaired persons. These durations were used to evaluate the cost of paid assistance at home in the four countries. Results: The numbers of visually impaired persons were 1.27 million in France, 0.73 million in Germany, 1.03 million in Italy and 1.11 million in the UK, including, respectively, 56%, 11%, 80% and 72% non-registered persons. The frequency of institutionalisation for visually impaired persons were, respectively, 7.8%, 9.6%, 10.9% and 10%. Total annual costs for visually impaired persons were estimated at euro10 749 million in France, euro9214 million in Germany, euro12 069 million in Italy and euro15 180 million in the UK. This translated into average annual costs per affected individual of euro8434, euro12 662, euro11 701 and euro13 674, respectively. The main cost components of visual impairment in the community were ‘loss of income’ (23–43% of community costs), ‘burden on carer’ (24–39%) and ‘paid assistance’ (13–29%). Conclusion: Total non-medical costs associated with visual impairment are considerable. The present analysis demonstrates that the preponderant economic consequences of visual impairment lie beyond healthcare systems, and that visual impairment has a considerable negative impact on productivity. Considering the non-medical social dimensions of visual impairment related to the consequent incapacity and dependency should encourage payers to finance health innovations that aim to preserve vision. [ABSTRACT FROM AUTHOR]