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Rationale, aims, and objectives: In recent years, several expensive new health technologies have been introduced. The availability of those technologies intensifies the discussion regarding the affordability of these technologies at different decision‐making levels. On the meso level, both hospitals and clinicians are facing budget constraints resulting in a tension to balance between different patients' interests. As such, it is crucial to make optimal use of the available resources. Different strategies are in place to deal with this problem, but decisions on a macro level on what to fund or not can limit the role and freedom of clinicians in their decisions on a micro level. At the same time, without central guidance regarding such decisions, micro level decisions may lead to inequities and undesirable treatment variation between clinicians and hospitals. The challenge is to find instruments that can balance both levels of decision making. Discussion Clinicians are becoming increasingly aware that their decisions to spend more resources (like time and budget) on 1 particular patient group reduce the resources available to other patients. Involving clinicians in thinking about the optimal use of limited resources, also in an attempt to bridge the world of economic reasoning and clinical practice, is crucial therefore. We argue that clinical guidelines may prove a clear vehicle for this by including both clinical and economic evidence to support the recommendations made. The development of such guidelines requires cooperation of clinicians, and health economists are cooperating with each other. Conclusion: The development of clinical guidelines which combine economic and clinical evidence should be stimulated, to balance central guidance and uniformity while maintaining necessary decentralized freedom. This is an opportunity to combine the reality of budgets and opportunity costs with clinical practice. Missing this opportunity risks either variation and inequity or central and necessarily crude measures. [ABSTRACT FROM AUTHOR]

Objectives: To assess the cost effectiveness of home telemonitoring (HTM) and nurse telephone support (NTS) compared with usual care (UC) in the management of patients with chronic heart failure, from a third-party payer's perspective.Methods: We developed a Markov model with a 20-year time horizon to analyze the cost effectiveness using the original study (Trans-European Network-Home-Care Management System) and various data sources. A probabilistic sensitivity analysis was performed to assess the decision uncertainty in our model.Results: In the original scenario (which concerned the cost inputs at the time of the original study), HTM and NTS interventions yielded a difference in quality-adjusted life-years (QALYs) gained compared with UC: 2.93 and 3.07, respectively, versus 1.91. An incremental net monetary benefit analysis showed €7,697 and €13,589 in HTM and NTS versus UC at a willingness-to-pay (WTP) threshold of €20,000, and €69,100 and €83,100 at a WTP threshold of €80,000, respectively. The incremental cost-effectiveness ratios were €12,479 for HTM versus UC and €8,270 for NTS versus UC. The current scenario (including telenurse cost inputs in NTS) yielded results that were slightly different from those for the original scenario, when comparing all New York Heart Association (NYHA) classes of severity. NTS dominated HTM, compared with UC, in all NYHA classes except NYHA IV.Conclusions: This modeling study demonstrated that HTM and NTS are viable solutions to support patients with chronic heart failure. NTS is cost-effective in comparison with UC at a WTP of €9000/QALY or higher. Like NTS, HTM improves the survival of patients in all NYHA classes and is cost-effective in comparison with UC at a WTP of €14,000/QALY or higher. [ABSTRACT FROM AUTHOR]

Economic evaluations can inform decisions about the efficiency and allocation of resources to implementation strategies—strategies explicitly designed to inform care providers and patients about the best available research evidence and to enhance its use in their practices. These strategies are increasingly popular in health care, especially in light of growing concerns about quality of care and limits on resources. But such concerns have hardly motivated health authorities and other decision-makers to spend on some form of economic evaluation in their assessments of implementation strategies. This editorial addresses the importance of economic evaluation in the context of implementation science—particularly, how these analyses can be most efficiently incorporated into decision-making processes about implementation strategies. [ABSTRACT FROM AUTHOR]

Background: In the Netherlands, decisions about the reimbursement of new pharmaceuticals are based on cost effectiveness, as well as therapeutic value and budget impact. Since 1 January 2005, drug manufacturers are formally required to substantiate the cost effectiveness of drugs that have therapeutic added value in comparison with existing ones through pharmacoeconomic evaluations. Dutch guidelines for pharmacoeconomic research providemethods guidance, ensuring consistency in both the evidence and the decision-making process about drug reimbursement. Aim: This study reviewed the methodological quality of all 21 formally required pharmacoeconomic evaluations of new pharmaceuticals between 1 January 2005 and 1 October 2008, and verified whether these evaluations complied with pharmacoeconomic guidelines. Methods: Data on the quality of the pharmacoeconomic evaluations were extracted from the pharmacoeconomic reports published by the Dutch Health Care Insurance Board (CVZ). The Board's newsletters provided information on the advice to, and reimbursement decisions made by, the Dutch Minister of Health. All data extraction was carried out by two independent reviewers, and descriptive analyses were conducted. Results: The methodological quality was sound in only 8 of the 21 pharmacoeconomic evaluations. In most cases, the perspective of analysis, the comparator drugs, and the reporting of both total and incremental costs and effects were correct. However, drug indication, form (i.e. cost utility/cost effectiveness) and time horizon of the evaluations were frequently flawed. Moreover, the costs and effects of the pharmaceuticals were not always analysed correctly, and modelling studies were often non-transparent. Twelve drugs were reimbursed, and nine were not. Conclusions: The compliance with pharmacoeconomic guidelines in economic evaluations of new pharmaceuticals can be improved. This would improve the methodological quality of the pharmacoeconomic evaluations and ensureconsistency in the evidence and the decision-making process for drug reimbursement in the Netherlands [ABSTRACT FROM AUTHOR]

Abstract: Objectives: The purpose of this systematic review is primarily to identify published cost-effectiveness analyses and cost-utility analyses of endocrine therapies for the treatment of early breast cancer. A secondary objective is to identify whether differences in seven modeling characteristics are related to differences in outcome of these cost-effectiveness and cost-utility analyses. Methods: A systematic literature review was conducted to identify peer-reviewed full economic evaluations of endocrine treatments of early breast cancer published in the English language between 2000 and December 2010. Information from these publications was abstracted regarding outcome, quality, and modeling methods. Results: We identified 20 economic evaluations comprising 5 different endocrine therapeutic strategies, which are all assessed more then once. The incremental cost-effectiveness ratios (ICERs) of the reported outcomes varied widely for identical therapies. For anastrazole compared to tamoxifen, incremental life-years gained even ranged from 0.16 to 0.550 with an ICER ranging from €3,958 to €75,331. Incremental quality-adjusted life-years (QALYs) gained ranged from 0.092 to 0.378 with a cost per QALY gained varying from €3,696 to €120,265. These large differences in outcome were related to different modeling methods, with differences in time horizon and use of a carryover effect as most prominent causes. Conclusion: Despite similar comparators and logical differences due to transferability issues, the outcomes of the included studies varied widely. To increase comparability and transparency of pharmacoeconomic evaluations, standardization of modeling methods for different therapeutic groups/diseases and the availability of a detailed and complete description of the model used in the evaluation is advocated. Recommendations for standardization in modeling treatment strategies in early breast cancer are presented. [Copyright &y& Elsevier]

For at least two decades, there has been an intense debate on whether and how to include the value of lost productivity in economic evaluations. This debate is often reflected in pharmacoeconomic guidelines, which have been developed to indicate the methods and requirements for the design, execution, and reporting of economic evaluations in a particular country. Objective: To examine what various national pharmacoeconomic guidelines recommend regarding the identification, measurement, and valuation of lost productivity. Methods: First, the theoretical framework on how lost productivity can be identified, measured, and valued is described. Second, a summary sheet has been used to identify various pharmacoeconomic guidelines recommendations regarding the value of lost productivity. Results: Twenty-two of the 30 guidelines identified recommend performing economic evaluations using the societal perspective. Nevertheless, even if the societal perspective is recommended, it is not always clear how the value of lost productivity should be taken into account. Most guidelines recommend including the costs of absenteeism from paid and/or unpaid work. In addition, although no agreement exists on how lost productivity should be valued, none of the guidelines recommended using the US panel approach for the valuation of lost productivity. Discussion: The different recommendations hinder international transferability of the value of lost productivity. This difficulty is mainly caused by different recommendations regarding identification and valuation. These differences result from the debate and lack of consensus on including the value of lost productivity losses in economic evaluations. It will become easier to transfer data across jurisdictions if all data are reported transparently. [ABSTRACT FROM AUTHOR]

Objective: Discrete event simulation (DES) modeling has several advantages over simpler modeling techniques in health economics, such as increased flexibility and the ability to model complex systems. Nevertheless, these benefits may come at the cost of reduced transparency, which may compromise the model's face validity and credibility. We aimed to produce a transparent report on the construction and validation of a DES model using a recently developed model of ocular hypertension and glaucoma. Methods: Current evidence of associations between prognostic factors and disease progression in ocular hypertension and glaucoma was translated into DES model elements. The model was extended to simulate treatment decisions and effects. Utility and costs were linked to disease status and treatment, and clinical and health economic outcomes were defined. The model was validated at several levels. The soundness of design and the plausibility of input estimates were evaluated in interdisciplinary meetings (face validity). Individual patients were traced throughout the simulation under a multitude of model settings to debug the model, and the model was run with a variety of extreme scenarios to compare the outcomes with prior expectations (internal validity). Finally, several intermediate (clinical) outcomes of the model were compared with those observed in experimental or observational studies (external validity) and the feasibility of evaluating hypothetical treatment strategies was tested. Results: The model performed well in all validity tests. Analyses of hypothetical treatment strategies took about 30 minutes per cohort and lead to plausible health–economic outcomes. Conclusion: There is added value of DES models in complex treatment strategies such as glaucoma. Achieving transparency in model structure and outcomes may require some effort in reporting and validating the model, but it is feasible. [ABSTRACT FROM AUTHOR]

Objective: Markov models are increasingly used in economic evaluations of (new) treatments for chronic diseases. In this study we propose a Markov model with health states defined by the disease activity score (DAS) to be used to extrapolate efficacy data from short-term clinical trials in rheumatoid arthritis to longer term cost-effectiveness results. Moreover, we perform an initial validation of this model.Methods: To test the validity of the model, the expected disease course (according to the model) was first compared with the observed disease course in an inception cohort of newly diagnosed rheumatoid arthritis patients. Then the relationship of the health states with utility and costs was investigated. Finally, costs and QALYs were calculated for usual care of patients in the first 5 years of their disease using the model and compared with the literature.Results: The model seemed to be able to extrapolate 1-year efficacy data as seen by a comparable distribution over the Markov states between the model results and the observational data. The health states had a significant relationship with costs and utility, and population characteristics had only a moderate effect on the cost and utility values of the Markov states. The distribution over the Markov states resulted in 3.266 expected QALYs per patient over 5 years. The expected medical and total costs per patient over 5 years were 6754 euro (1997 values) and 12,641 euro, respectively, for standard rheumatoid arthritis care in The Netherlands.Conclusion: The developed Markov model seems a valid model for use in economic evaluations in rheumatoid arthritis. The model produced similar utilities, but lower total costs, to those in previously published studies. Although the steps to develop and validate this Markov model were applied in the context of rheumatoid arthritis, they can be generalised to other chronic diseases. [ABSTRACT FROM AUTHOR]

Cost-effectiveness acceptability curves (CEACs) are a method used to present uncertainty surrounding incremental cost-effectiveness ratios (ICERs). Construction of the curves relies on the assumption that the willingness to pay (WTP) for health gain is identical to the willingness to accept (WTA) health loss. The objective of this paper is to explore the impact that differences between WTP and WTA health changes have on CEACs. Previous empirical evidence has shown that the relationship between WTP and WTA is not 1 : 1. The discrepancy between WTP and WTA for health changes can be expressed as a ratio: the accept/reject ratio (which can vary between 1 and infinity). Depending on this ratio, the area within the southwest quadrant of the cost-effectiveness plane in which any bootstrap cost-effect pairs will be considered to be cost effective will be smaller, resulting in a lower CEAC. We used data from two clinical trials to illustrate that relaxing the 1 : 1 WTP/WTA assumption has an impact on the CEACs. Given the difficulty in assessing the accept/reject ratio for every evaluation, we suggest presenting a series of CEACs for a range of values for the accept/reject ratio, including 1 and infinite. Although it is not possible to explain this phenomenon within the extra-welfarist framework, it has been shown empirically that individuals give a higher valuation to the removal of effective therapies than to the introduction of new therapies that are more costly and effective. In cost-effectiveness analyses where uncertainty of the ICER covers the southwest quadrant of the cost-effectiveness plane, the discrepancy between societies’ WTP and WTA should be indicated by drawing multiple CEACs. [ABSTRACT FROM AUTHOR]

Given the potential role of economic information in healthcare decision making, it is of interest to assess its influence on decisions at a national or regional level (macro level), at a healthcare facility level (meso level) and at the healthcare provider level (micro level). This literature review summarises 36 empirical studies that examined the influence of economic evaluations on these three healthcare decision-making levels. Economic evaluations are considered useful and important; however, their direct influence on decision making (instrumental use) is moderate, especially at the macro and micro levels. A major influence was observed at the meso level, leading to the conclusion that economic evaluations have the most pronounced influence on decision making within healthcare organisations. However, unexpectedly, our literature search did not reveal an empirical study analysing the considerable influence of economic evaluations on decisions by the National Institute of Health and Clinical Excellence in the UK. Our findings indicate that results of economic evaluations cannot be considered the dominant decision criterion for healthcare decision makers at either the macro, meso or micro levels. Enlightenment use (where scientific evidence provides a background of information, ideas and concepts that affect the way policy makers view problems and solutions) of economic evaluations in decision making remains to be proven. [ABSTRACT FROM AUTHOR]

Discusses the theoretical and empirical arguments in determining the optimal solution regarding which discounting method and/or discounting rate should be use for economic evaluations in health care. Definition of discounting; Calculations for discounting; Basis of the concept of uniform discounting.

Examines the precision and accuracy of a retrospective self-administered questionnaire on sick leave. Importance of the impact of disease on the ability of a person to perform work; Use of different recall periods to analyze the relationship between memory and length of the recall period.

Purpose: To review types and frequencies of adverse events (AE) associated with bone-conduction hearing implants (BCHIs) and active middle-ear implants (aMEIs) as reported in the literature. Methods: Cochrane, PubMed, and EMBASE libraries were searched for primary articles in English or German language that reported on adverse events following BCHI or aMEI implantation, included at least five patients and were published between 1996 and 2016. Study characteristics, demographics, and counts of adverse events were tabulated and analyzed within the R statistical programming environment. Results: Following assessment of the reporting quality of adverse events, we present a brief guideline that potentially improves AE reporting in this field of research. For the full dataset, we summarize study-level adverse event frequencies in terms of ratio of events to ears (REE) by AE groups and by device. For a subset of studies, we also report cumulative incidence (risk) for minor- and major adverse-events by device and by device groups. Conclusions: Data analyzed in this review show that: (1) the reporting quality of adverse events associated with BCHI and aMEIs is often very low; (2) adverse events associated with BCHI and aMEIs are qualitatively different and not equally frequent among devices; (3) state-of-the-art implantable BCHIs and aMEIs are a safe treatment option for hearing loss. [ABSTRACT FROM AUTHOR]

Background: In health economic literature, checklists or best practice recommendations on model validation/credibility always declare verification of the programmed model as a fundamental step, such as 'is the model implemented correctly and does the implementation accurately represent the conceptual model?' However, to date, little operational guidance for the model verification process has been given. In this study, we aimed to create an operational checklist for model users or reviewers to verify the technical implementation of health economic decision analytical models and document their verification efforts.Methods: Literature on model validation, verification, programming errors and credibility was reviewed systematically from scientific databases. An initial beta version of the checklist was developed based on the checklists/tests identified from the literature and from authors' previous modeling/appraisal experience. Next, the first draft checklist was presented to a number of health economists on several occasions and was tested on different models (built in different software, developed by different stakeholders, including drug manufacturers, consultancies or academia), each time leading to an update of the checklist and culminating in the final version of the TECHnical VERification (TECH-VER) checklist, introduced in this paper.Results: The TECH-VER necessitates a model reviewer (preferably independent), an executable and transparent model, its input sources, and detailed documentation (e.g. technical report/scientific paper) in which the conceptual model, its implementation, programmed model inputs, and results are reported. The TECH-VER checklist consists of five domains: (1) input calculations; (2) event-state (patient flow) calculations; (3) result calculations; (4) uncertainty analysis calculations; and (5) other overall checks (e.g. validity or interface). The first four domains reflect the verification of the components of a typical health economic model. For these domains, as a prerequisite of verification tests, the reviewer should identify the relevant calculations in the electronic model and assess the provided justifications for the methods used in the identified calculations. For this purpose, we recommend completeness/consistency checks. Afterwards, the verification tests can be conducted for the calculations in each of these stages by checking the correctness of the implementation of these calculations. For this purpose, the following type of tests are recommended in consecutive order: (i) black-box tests, i.e. checking if model calculations are in line with a priori expectations; (ii) white-box testing, i.e. going through the program code details line by line, or cell by cell (recommended for some crucial calculations and if there are some unexpected results from the black-box tests); and (iii) model replication/parallel programming (recommended only in certain situations, and if the issues related to the identified unexpected results from black-box tests could not be resolved through white-box testing). To reduce the time burden of model verification, we suggest a hierarchical order in tests i-iii, where going to the next step is necessary when the previous step fails.Conclusions: The TECH-VER checklist is a comprehensive checklist for the technical verification of decision analytical models, aiming to help identify model implementation errors and their root causes while improving the transparency and efficiency of the verification efforts. In addition to external reviews, we consider that the TECH-VER can be used as an internal training and quality control tool for new health economists, while developing their initial models. It is the authors' aim that the TECH-VER checklist transforms itself to an open-source living document, with possible future versions, or 'bolt-on' extensions for specific applications with additional 'fit-for-purpose' tests, as well as 'tips and tricks' and some demonstrative error examples. For this reason, the TECH-VER checklist and the list of black-box tests created in this paper and a few model verification examples is uploaded to an open access, online platform (github and the website of the institute), where other users will also be able to upload their original verification efforts and tests. [ABSTRACT FROM AUTHOR]

The article comments on "Loss Aversion and Cost Effectiveness of Healthcare Programmes," by Afschin Gandjour which is in this issue of "PharmacoEconomics." The author's opinion is that Gandjuor's method for incorporating loss aversion into cost-effectiveness estimates for health initiatives is too simple because it does not apply a population-based incremental cost-effectiveness ratio.

Results of cost effectiveness analyses (CEA) studies are most useful for decision makers if they face only one constraint: the health care budget. However, in practice, decision makers wishing to use the results of CEA studies may face multiple resource constraints relating to, for instance, constraints in health care inputs such as a shortage of skilled labour. The presence of multiple resource constraints influences the decision rules of CEA and limits the usefulness of traditional CEA studies for decision makers. The goal of this paper is to illustrate how results of CEA can be interpreted and used in case a decision maker faces a health care input constraint. We set up a theoretical model describing the optimal allocation of the health care budget in the presence of a health care input constraint. Insights derived from that model were used to analyse a stylized example based on a decision about a surgical robot as well as a published cost effectiveness study on eye care services in Zambia. Our theoretical model shows that applying default decision rules in the presence of a health care input constraint leads to suboptimal decisions but that there are ways of preserving the traditional decision rules of CEA by reweighing different cost categories. The examples illustrate how such adjustments can be made, and makes clear that optimal decisions depend crucially on such adjustments. We conclude that it is possible to use the results of cost effectiveness studies in the presence of health care input constraints if results are properly adjusted. [ABSTRACT FROM AUTHOR]

In Europe, 1–3% of the population suffers from atrial fibrillation (AF) and has increased stroke risk. By 2060 a doubling in number of cases and great burden in managing this medical condition are expected. This paper offers an overview of data on epidemiology and management of AF and stroke in four European countries as well as the interconnection between these dimensions. A search index was developed to access multiple scientific and “grey” literatures. Information was prioritised based on strength of evidence and date. Information on country reports was double-checked with national experts. The overall prevalence of AF is consistent across countries. France has the lowest stroke incidence and mortality, followed by Netherland and UK, while Romania has higher rates. GPs or medical specialists are responsible for AF treatment; exception are the special thrombosis services in the Netherlands. Prevention measurements are only present in UK through screening programs. Although international and national guidelines are available, undertreatment is present in all countries. Despite differences in healthcare systems and management of AF, epidemiology is comparable between three of the countries. Romania is an outlier, by being limited in data accessibility. This knowledge can contribute to improved AF care in Europe. [ABSTRACT FROM AUTHOR]

A response from the authors of the article "Methodological quality of economic evaluations of new pharmaceuticals in the Netherlands," which discusses the use of economic evaluation information from clinical studies and other evaluative approaches in decision making is presented.

Background: Systematic reviews of cost-effectiveness analyses summarize results and describe study characteristics. Variability in the study results is often explained qualitatively or based on sensitivity analyses of individual studies. However, variability due to input parameters and study characteristics (e.g., funding or study quality) is often not statistically explained. As a case study, a systematic review on the cost-effectiveness of drug-eluting stents (DES) versus bare-metal stents (BMS) using meta-regression analyses is performed to explore the usefulness of such methods compared with conventional review methods.Methods: We attempted to identify and review all modelling studies published until January 2012 that compared costs and consequences of DES versus BMS. We extracted general study information (e.g., funding), modelling methods, values of input parameters, and quality of the model using the Philips et al. checklist. Associations between study characteristics and the incremental costs and effectiveness of individual analyses were explored using regression analyses corrected for study ID.Results: Sixteen eligible studies were identified, with a combined total of 508 analyses. The overall quality of the models was moderate (59% ± 15%). This study showed associations (e.g., type of lesion) that were expected (based on individual studies), however the meta-regression analyses revealed also unpredicted associations: e.g., model quality was negatively associated with repeat revascularizations avoided.Conclusions: Meta-regressions can be of added value, identifying significant associations that could not be identified using conventional review methods or by sensitivity analyses of individual studies. Furthermore, this study underlines the need to examine input parameters and perform a quality check of studies when interpreting the results. [ABSTRACT FROM AUTHOR]

Background The question of how to value lost productivity in economic evaluations has been subject of debate in the past twenty years. According to the Washington panel, lost productivity influences health-related quality of life and should thus be considered a health effect instead of a cost to avoid double counting. Current empirical evidence on the inclusion of income loss when valuing health states is not decisive. We examined the relationship between three aspects of lost productivity (work-status, absenteeism and presenteeism) and patient or social valuation of health-related quality of life (HRQoL). Methods Cross-sectional survey data were collected from a total of 830 respondents with a rheumatic disorder from four West-European countries. Health-related quality of life was expressed in either the European societal utility using EQ-5D-3 L or the patient valuation using EQ-VAS. The impact of work-status (four categories), absenteeism (absent from paid work during the past three months), and presenteeism (QQ method) on EQ-5D utilities and VAS scores was examined in linear regression analyses taking into account demographic characteristics and disease severity (duration, pain and restriction). Results The relationship between work-status, absenteeism or presenteeism and HRQoL was stronger for patient valuation than societal valuation. Compared to work-status and presenteeism the relationship between absenteeism and HRQoL was even less explicit. However, results for all measures of lost productivity are only marginally significant and negligible compared to the influence of disease-related restrictions. Conclusions This survey study in patients with a rheumatic disorder in four European countries, does not fully support the Washington panel's claim that lost productivity is a significantly related with HRQoL, and this is even more apparent for absenteeism than for work-status and presenteeism. For West-European countries, there is no reason, to include absenteeism in the QALY. Findings need to be confirmed in other disease areas. [ABSTRACT FROM AUTHOR]

Many national colorectal cancer screening campaigns have a similar structure. First, individuals are invited to take a noninvasive screening test, and, second, in the case of a positive screening test result, they are advised to undergo a more invasive follow-up test. The objective of this study was to investigate how much individuals' participation decision in noninvasive screening is affected by the presence or absence of detailed information about invasive follow-up testing and how this effect varies over screening tests. Methods We used a labeled discrete choice experiment of three noninvasive colorectal cancer screening types with two versions that did or did not present respondents with detailed information about the possible invasive follow-up test (i.e., colonoscopy) and its procedure. We used data from 631 Dutch respondents aged 55 to 75 years. Each respondent received only one of the two versions (N = 310 for the invasive follow-up test information specification version, and N = 321 for the no-information specification version). Results Mixed logit model results show that detailed information about the invasive follow-up test negatively affects screening participation decisions. This effect can be explained mainly by a decrease in choice shares for the most preferred screening test (a combined stool and blood sample test). Choice share simulations based on the discrete choice experiment indicated that presenting invasive follow-up test information decreases screening participation by 4.79%. Conclusions Detailed information about the invasive follow-up test has a negative effect on individuals' screening participation decisions in noninvasive colorectal cancer screening campaigns. This result poses new challenges for policymakers who aim not only to increase uptake but also to provide full disclosure to potential screening participants. [ABSTRACT FROM AUTHOR]

Abstract: Productivity costs related to paid work are commonly calculated in economic evaluations of health technologies by multiplying the relevant number of work days lost with a wage rate estimate. It has been argued that actual productivity costs may either be lower or higher than current estimates due to compensation mechanisms and/or multiplier effects (related to team dependency and problems with finding good substitutes in cases of absenteeism). Empirical evidence on such mechanisms and their impact on productivity costs is scarce, however. This study aims to increase knowledge on how diminished productivity is compensated within firms. Moreover, it aims to explore how compensation and multiplier effects potentially affect productivity cost estimates. Absenteeism and compensation mechanisms were measured in a randomized trial among Dutch citizens examining the cost-effectiveness of reimbursement for smoking cessation treatment. Multiplier effects were extracted from published literature. Productivity costs were calculated applying the Friction Cost Approach. Regular estimates were subsequently adjusted for (i) compensation during regular working hours, (ii) job dependent multipliers and (iii) both compensation and multiplier effects. A total of 187 respondents included in the trial were useful for inclusion in this study, based on being in paid employment, having experienced absenteeism in the preceding six months and completing the questionnaire on absenteeism and compensation mechanisms. Over half of these respondents stated that their absenteeism was compensated during normal working hours by themselves or colleagues. Only counting productivity costs not compensated in regular working hours reduced the traditional estimate by 57%. Correcting for multiplier effects increased regular estimates by a quarter. Combining both impacts decreased traditional estimates by 29%. To conclude, large amounts of lost production are compensated in normal hours. Productivity costs estimates are strongly influenced by adjustment for compensation mechanisms and multiplier effects. The validity of such adjustments needs further examination, however. [Copyright &y& Elsevier]

. Purpose: To determine the cost-effectiveness of ocular hypertension (OH) treatment initiated with latanoprost compared to timolol. Methods: Two strategies for OH therapy are modelled, (1) 'starting with timolol' and (2) 'starting with latanoprost'. Therapy can be maintained or changed dependent on the achieved intraocular pressure (IOP) and side-effects. Adjustments of therapy to reach a target pressure involve monotherapy, combination therapy and laser. Four drugs are used: latanoprost, timolol, brimonidine and dorzolamide. Once the adjustments of therapy are completed, lifelong follow-up with IOP-dependent conversion to glaucoma and progression to blindness are modelled. Direct medical costs are assigned. The IOP-lowering effect of drugs is based on meta-analyses and applied by Monte Carlo simulation to a hypothetical cohort of patients with OH. The characteristics of the cohort, including the initial IOP distribution, are based on data of 1000 patients. Results: The IOP decreased from 25,4 mm Hg (mean) to 16.7 (±0.017) mm Hg (strategy 1) and to 16.5 (±0.013) mm Hg (strategy 2). Costs per patient within 15 months of therapy were € 367 and € 469, respectively. Lifetime blindness and costs were 0.0334 years and € 3 514 (strategy 1) and 0.0318 years and € 4 397 (strategy 2). Incremental costs per year of vision saved for strategy (2) in comparison with strategy (1) amount to, given the uncertainties in the model, approximately € 537 000. Conclusion: For saving 1 year of vision, high costs are needed when OH therapy is initiated with latanoprost compared to timolol, when the cost price of latanoprost remains high. [ABSTRACT FROM AUTHOR]

. Purpose: To evaluate the long-term effects and costs of four treatment strategies for primary open-angle glaucoma compared to usual care. Methods: Cost-effectiveness analyses with a lifelong horizon were made from a societal perspective. Data were generated with a patient-level model based on discrete event simulation. The model structure and parameter estimates were based on literature, particularly clinical studies on the natural course of glaucoma and the effect of treatment. We simulated heterogeneous cohorts of 3000 patients and explored the impact of uncertainty with sensitivity analyses. Results: The incremental cost-effectiveness ratio (ICER) of initial treatment with a prostaglandin analogue compared with a β-blocker was €12.931 per quality-adjusted life year (QALY) gained. A low initial target pressure (15 mmHg) resulted in 0.115 QALYs gained and €1550 saved compared to a gradual decrease from 21 to 15 mmHg upon progression. Visual field (VF) measurements every 6 rather than 12 months lead to health gains at increased costs (ICER €173 486 per QALY gained), whereas measurements every 24 months lead to health losses at reduced costs (ICER €21 516 per QALY lost). All treatment strategies were dominant over 'withholding treatment'. Conclusions: From a cost-effectiveness point of view, it seems advantageous to aim for a low intraocular pressure in all glaucoma patients. The feasibility of this strategy should therefore be investigated. Additionally, the cost-effectiveness outcomes of initiating monotherapy with a prostaglandin analogue and reducing the frequency of VF testing may be acceptable. [ABSTRACT FROM AUTHOR]

Rationale and objectives It is difficult to keep control over prescribing behaviour in general practice. The purpose of this study was to assess the initial effects of a behaviour independent financial incentive on the volume of drug prescribing of general practitioners (GPs). Design 2-Year Controlled Before After study with an intervention region and a concurrent control region. Setting and participants GPs in two regions in the Netherlands ( n = 119 and n = 118). Intervention A financial incentive for prescribing according to local guidelines on specific drugs or drug categories. The financial incentive consisted of a non-recurrent, behaviour-independent allowance. Main outcome measure Change in the number of prescriptions for 10 targeted drugs or drug groups. Results Significant changes were seen only in three types of antibiotics and in recommended gastric medicines. In almost all cases, effects were temporary. Conclusion Behaviour independent financial incentives can be a help in changing prescription behaviour of GPs, but effects are small-scale and temporary. [ABSTRACT FROM AUTHOR]

BackgroundThe impact of dementia constitutes a major public health challenge to our society. A multidisciplinary approach to dementia is generally recommended. ObjectiveThe objective of this review is to give an overview of the reported empirical studies on the added value of a multidisciplinary approach to diagnosing dementia, i.e. whether differences can be found between diagnostic multidisciplinary approaches, and/or monodisciplinary ones with respect to the diagnostic outcomes, the impact of diagnostics and their effects on quality of life and costs. MethodCritical review of studies which involve a multidisciplinary assessment of dementia. Literature was systematically searched in a number of international databases (Medline, PsychInfo, Pubmed and EconLit). A first selection was based on screening titles by one author. Interrater reliability was determined by scoring all abstracts by two authors. The reliability of selecting full articles was based on scoring a random sample by two authors. Kappa values were calculated. The Kappa statistic (k) was also used as an indicator of the diagnostic agreement between single disciplines and multidisciplinary teams. ResultsFive studies were identified as relevant for the purpose of this review. The most important outcome was the level of diagnostic agreement between single disciplines and multidisciplinary teams (MTs). Overall, there was substantial agreement (kappa: 0.37–0.76) on the diagnosis dementia (syndrome), but not on the subtypes (aetiology) (AD: 0.26–0.60; VD: 0.35–0.52; depression: 0.30–0.46). Diagnoses also differed between the disciplines. ConclusionsMerely studies on diagnostic accuracy were found, all using different reference standards. Kappa values were chosen as outcome measure and every diagnosis was considered of equal value. Therefore, the added value could not definitively be demonstrated. However, the potential added value of a multidisciplinary approach to diagnosing dementia probably lies in the ability to differentiate among the subtypes of dementia and to detect comorbid psychiatric conditions such as depression. Disciplines within an MT complement each other in establishing a specific diagnosis, which is considered to be the added value of such a multidisciplinary team. Copyright © 2006 John Wiley & Sons, Ltd. [ABSTRACT FROM AUTHOR]

Objective: In economic evaluation of health care programmes normally health-related quality of life is part of measurement of a programme's effectiveness and productivity part of its costs. In this paper the relationship between quality of life and productivity is highlighted and empirically assessed in persons suffering from low back pain.Methods: A secondary analysis was performed on data from a sample of 483 patients treated for low back pain. Periodically, both quality of life (EQ-5D) and productivity indicators for both paid and unpaid work were measured. Mean EQ-5D scores were compared for groups of patients classified by level of productivity. The relationship between quality of life and productivity was also assessed using Spearman rank correlation coefficients.Results: Mean EQ-5D scores for patients without productivity losses were a half to a full standard deviation higher than for patients with the lowest levels of productivity. The correlation between quality of life and productivity was moderate.Conclusion: Lower levels of quality of life were associated with efficiency loss and absenteeism. However, due to the moderate strength of the relation the use of information on quality of life to model productivity costs in case of absence of productivity data was not recommended. [ABSTRACT FROM AUTHOR]

Rationale, Aims, and Objectives: While different imaging and treatment options are available in acute coronary syndrome (ACS) care, there is a lack of data regarding their use across Europe. We examined the diagnostic and treatment strategies in patients with known or suspected ACS as reported by physicians and identified variations in responses across European countries and geographical areas. Method: A web‐based clinician survey focusing on ACS imaging and revascularization treatments was circulated through email distribution lists and websites of European professional societies in the field of cardiology. We collected information on respondents' clinical setting and specialty. Reported percentages of patients receiving imaging or treatment modalities and percentages of clinicians reporting to use modalities in a range of clinical scenarios were analyzed. Statistical comparisons were performed. Results: In total, 69 responses were received (Sweden [n = 20], United Kingdom [n = 16], Northern/Western Europe [n = 17], Southern Europe [n = 9], and Central Europe [n = 7]). Considerable variations between geographical areas were seen in terms of reported diagnostic modalities and treatment strategies. For example, when presented with the scenario of a theoretical 45‐year‐old smoking female with a suspected ACS, 56% of UK clinicians reported to use coronary computed tomography angiography, compared to only 10% of Swedish clinicians (P =.002). Large variations were observed regarding the reported use of fractional flow reserve by physicians for non‐culprit lesions during invasive management of myocardial infarction patients (44% in Sweden, 31% in the United Kingdom, and 30% in Northern/Western Europe vs non‐use in Central and Southern Europe). Conclusions: In this survey, respondents reported different diagnostic and treatment strategies in ACS care. These variations seem to have geographic components. Larger studies or real world data are needed to verify these observations and investigate their causes. More research is needed to compare the quality and efficiency of ACS care across countries and explore pathways for improvement. [ABSTRACT FROM AUTHOR]

Background: Empirical identification of the direct impact of hospitalisation in the change in utility could provide an interpretation for some of the unexplained variance in quality of life responses in clinical practice and clinical trials and provide assistance to researchers in assessing the impact of a hospitalisation in the context of economic evaluations. This study had the goal of determining the impact of nonfatal hospitalisations on the quality of life of a cohort of patients previously diagnosed with heart failure by using their quality of life measurements before and after hospitalisation.Methods: The impact of hospitalisation on health-related quality of life was estimated by calculating the difference in utility measured using the EQ-5D-3L in patients that were hospitalised and had records of utility before and after hospitalisation. The variation in differences between the utilities pre and post hospitalisation was explained through two multiple linear regression models using (1) the individual patient characteristics and (2) the hospitalisation characteristics as explanatory variables.Results: The mean difference between health-related quality of life measurement pre and post hospitalisation was found to be 0.020 [95% CI: - 0.020, 0.059] when measured with the EQ-5D index, while there was a mean decrease of - 0.012 [95% CI: - 0.043, 0.020] in the utility measured with the visual analogue scale. Differences in utility variation according to the primary cause for hospitalisation were found. Regression models showed a statistically significant impact of body mass index and serum creatinine in the index utility differences and of serum creatinine for utilities measured with the visual analogue scale.Conclusions: Knowing the impact of hospitalisation on health-related quality of life is particularly relevant for informing cost-effectiveness studies designed to assess health technologies aimed at reducing hospital admissions. Through using patient-level data it was possible to estimate the variation in utilities before and after the average hospitalisation and for hospitalisations due to the most common causes for hospital admission. These estimates for (dis) utility could be used in the calculations of effectiveness on economic evaluations, especially when discrete event simulations are the employed modelling technique. [ABSTRACT FROM AUTHOR]

Background Severe adverse events after treatment with ivermectin in individuals with high levels of Loa loa microfilariae in the blood preclude onchocerciasis elimination through community-directed treatment with ivermectin (CDTI) in Central Africa. We measured the cost of a community-based pilot using a test-and-not-treat (TaNT) strategy in the Soa health district in Cameroon. Methods Based on actual expenditures, we empirically estimated the economic cost of the Soa TaNT campaign, including financial costs and opportunity costs that will likely be borne by control programs and stakeholders in the future. In addition to the empirical analyses, we estimated base-case, less intensive, and more intensive resource use scenarios to explore how costs might differ if TaNT were implemented programmatically. Results The total costs of US$283 938 divided by total population, people tested, and people treated with 42% coverage were US$4.0, US$9.2, and US$9.5, respectively. In programmatic implementation, these costs (base-case estimates with less and more intensive scenarios) could be US$2.2 ($1.9–$3.6), US$5.2 ($4.5–$8.3), and US$5.4 ($4.6–$8.6), respectively. Conclusions TaNT clearly provides a safe strategy for large-scale ivermectin treatment and overcomes a major obstacle to the elimination of onchocerciasis in areas coendemic for Loa loa. Although it is more expensive than standard CDTI, costs vary depending on the setting, the implementation choices made by the institutions involved, and the community participation rate. Research on the required duration of TaNT is needed to improve the affordability assessment, and more experience is needed to understand how to implement TaNT optimally. [ABSTRACT FROM AUTHOR]

Systematic reviews with economic components are important decision tools for stakeholders seeking to evaluate technologies, such as breast cancer screening (BCS) programs. This overview of systematic reviews explores the determinants of the cost‐effectiveness of BCS and assesses the quality of secondary evidence. The search identified 30 systematic reviews that reported on the determinants of the cost‐effectiveness of BCS, including the costs of breast cancer and BCS. While the quality of the reviews varied widely, only four out of 30 papers were considered to be of a high quality. We did not identify publication bias in the original evidence on the cost‐effectiveness of mammography screening; however, we highlight a need for improved clarity in both reporting and data verification. The reviews consisted mainly of studies from high‐income countries. Breast cancer costs varied widely among the studies. Factors leading to higher costs included: time (diagnosis and last months before death), later stage or metastases, recurrence of the disease, age below 64 years and type of follow‐up (more intensive or more specialized). Overall, screening with mammography was considered cost‐effective in the age range 50‐69 years in Western European and Northern American countries but not for older or younger women. Its cost‐effectiveness was questionable for low‐income settings and Asia. Mammography screening was more cost‐effective with biennial screening compared to annual screening and single reading using computer‐aided detection vs double reading. No information on the cost‐effectiveness of ultrasonography was found, and there is much uncertainty on the cost‐effectiveness of CBE because of methodological limitations. [ABSTRACT FROM AUTHOR]

Objectives: Value of information (VOI) analysis provides information on opportunity cost of a decision in healthcare by estimating the cost of reducing parametric uncertainty and quantifying the value of generating additional evidence. This study is an application of the VOI methodology to the problem of choosing between home telemonitoring and nurse telephone support over usual care in chronic heart failure management in the Netherlands. Methods: The expected value of perfect information (EVPI) and the expected value of partially perfect information (EVPPI) analyses were based on an informal threshold of €20K per quality-adjusted life-year. These VOI-analyses were applied to a probabilistic Markov model comparing the 20-year costs and effects in three interventions. The EVPPI explored the value of decision uncertainty caused by the following group of parameters: treatment-specific transition probabilities between New York Heart Association (NYHA) defined disease states, utilities associated with the disease states, number of hospitalizations and ER visits, health state specific costs, and the distribution of patients per NYHA group. We performed the analysis for two population sizes in the Netherlands—patients in all NYHA classes of severity, and patients in NYHA IV class only. Results: The population EVPI for an effective population of 2,841,567 CHF patients in All NYHA classes of severity over the next 20 years is more than €4.5B, implying that further research is highly cost-effective. In the NYHA IV only analysis, for the effective population of 208,003 patients over next 20 years, the population EVPI at the same informal threshold is approx. €590M. The EVPPI analysis showed that the only relevant group of parameters that contribute to the overall decision uncertainty are transition probabilities, in both All NYHA and NYHA IV analyses. Conclusions: Results of our VOI exercise show that the cost of uncertainty regarding the decision on reimbursement of telehealth interventions for chronic heart failure patients is high in the Netherlands, and that future research is needed, mainly on the transition probabilities. [ABSTRACT FROM AUTHOR]

This report provides recommendations for budget holders and decision makers in high-, middle, and low-income countries requiring economic analyses of new vaccination programs to allocate scarce resources given budget constraints. ISPOR's Economic Evaluation of Vaccines Designed to Prevent Infectious Disease: Good Practices Task Force wrote guidelines for three analytic methods and solicited comments on them from external reviewers. Cost-effectiveness analyses use decision-analytic models to estimate cumulative changes in resource use, costs, and changes in quality- or disability-adjusted life-years attributable to changes in disease outcomes. Constrained optimization modeling uses a mathematical objective function to be optimized (e.g. disease cases avoided) for a target population for a set of interventions including vaccination programs within established constraints. Fiscal health modeling estimates changes in net present value of government revenues and expenditures attributable to changes in disease outcomes. The task force recommends that those designing economic analyses for new vaccination programs take into account the decision maker's policy objectives and country-specific decision context when estimating: uptake rate in the target population; vaccination program's impact on disease cases in the population over time using a dynamic transmission epidemiologic model; vaccination program implementation and operating costs; and the changes in costs and health outcomes of the target disease(s). The three approaches to economic analysis are complementary and can be used alone or together to estimate a vaccination program's economic value for national, regional, or subregional decision makers in high-, middle-, and low-income countries. [ABSTRACT FROM AUTHOR]

Background: The control or elimination of neglected tropical diseases (NTDs) has targets defined by the WHO for 2020, reinforced by the 2012 London Declaration. We estimated the economic impact to individuals of meeting these targets for human African trypanosomiasis, leprosy, visceral leishmaniasis and Chagas disease, NTDs controlled or eliminated by innovative and intensified disease management (IDM). Methods: A systematic literature review identified information on productivity loss and out-of-pocket payments (OPPs) related to these NTDs, which were combined with projections of the number of people suffering from each NTD, country and year for 2011–2020 and 2021–2030. The ideal scenario in which the WHO’s 2020 targets are met was compared with a counterfactual scenario that assumed the situation of 1990 stayed unaltered. Economic benefit equaled the difference between the two scenarios. Values are reported in 2005 US$, purchasing power parity-adjusted, discounted at 3% per annum from 2010. Probabilistic sensitivity analyses were used to quantify the degree of uncertainty around the base-case impact estimate. Results: The total global productivity gained for the four IDM-NTDs was I$ 23.1 (I$ 15.9 –I$ 34.0) billion in 2011–2020 and I$ 35.9 (I$ 25.0 –I$ 51.9) billion in 2021–2030 (2.5th and 97.5th percentiles in brackets), corresponding to US$ 10.7 billion (US$ 7.4 –US$ 15.7) and US$ 16.6 billion (US$ 11.6 –US$ 24.0). Reduction in OPPs was I$ 14 billion (US$ 6.7 billion) and I$ 18 billion (US$ 10.4 billion) for the same periods. Conclusions: We faced important limitations to our work, such as finding no OPPs for leprosy. We had to combine limited data from various sources, heterogeneous background, and of variable quality. Nevertheless, based on conservative assumptions and subsequent uncertainty analyses, we estimate that the benefits of achieving the targets are considerable. Under plausible scenarios, the economic benefits far exceed the necessary investments by endemic country governments and their development partners. Given the higher frequency of NTDs among the poorest households, these investments represent good value for money in the effort to improve well-being, distribute the world’s prosperity more equitably and reduce inequity. [ABSTRACT FROM AUTHOR]

Background: The purpose of this study is to assess the Business-to-Consumer (B2C) model for telemonitoring patients with Chronic Heart Failure (CHF) by analysing the value it creates, both for organizations or ventures that provide telemonitoring services based on it, and for society.Methods: The business model assessment was based on the following categories: caveats, venture type, six-factor alignment, strategic market assessment, financial viability, valuation analysis, sustainability, societal impact, and technology assessment. The venture valuation was performed for three jurisdictions (countries) - Singapore, the Netherlands and the United States - in order to show the opportunities in a small, medium-sized, and large country (i.e. population).Results: The business model assessment revealed that B2C telemonitoring is viable and profitable in the Innovating in Healthcare Framework. Analysis of the ecosystem revealed an average-to-excellent fit with the six factors. The structure and financing fit was average, public policy and technology alignment was good, while consumer alignment and accountability fit was deemed excellent. The financial prognosis revealed that the venture is viable and profitable in Singapore and the Netherlands but not in the United States due to relatively high salary inputs.Conclusions: The B2C model in telemonitoring CHF potentially creates value for patients, shareholders of the service provider, and society. However, the validity of the results could be improved, for instance by using a peer-reviewed framework, a systematic literature search, case-based cost/efficiency inputs, and varied scenario inputs. [ABSTRACT FROM AUTHOR]

The National Institute for Health and Care Excellence (NICE) invited Janssen, the company manufacturing abiraterone acetate (AA; tradename Zytiga®), to submit evidence for the clinical and cost effectiveness of AA in combination with prednisone/prednisolone (AAP) compared with watchful waiting (i.e. best supportive care [BSC]) for chemotherapy-naïve patients with metastatic castration-resistant prostate cancer (mCRPC). Kleijnen Systematic Reviews Ltd (KSR), in collaboration with Maastricht University Medical Center, was commissioned as the Evidence Review Group (ERG). This paper presents a summary of the company submission (CS), the ERG report, subsequent addenda, and the development of the NICE guidance for the use of this drug in England and Wales by the Appraisal Committee (AC). The ERG produced a critical review of the clinical and cost effectiveness of AAP based on the CS. An important question in this appraisal was, according to the ERG, whether AAP followed by docetaxel is more effective than BSC followed by docetaxel. In the COU-AA-302 trial, 239 of 546 (43.8 %) AAP patients and 304 of 542 (56.1 %) BSC patients received docetaxel as subsequent therapy, following AA or placebo. The results for this specific group of patients were not presented in the CS; therefore, the ERG asked the company to provide these data in the clarification letter; however, these data were presented as commercial-in-confidence and cannot therefore be reported here. The ERG's critical assessment of the company's economic evaluation highlighted a number of concerns, including (a) not using the intention-to-treat (ITT) population; (b) inconsistencies in estimating prediction equations; (c) not fully incorporating the impact of adverse events; (d) incorrectly incorporating the new patient access scheme (PAS); and (e) the assumption that AA non-compliance leads to recoverable drug costs. Although some of these issues were adjusted in the ERG base case, the ERG could not estimate the impact of all of these issues, and thus acknowledges that there are still uncertainties concerning the cost-effectiveness evidence. With the exception of the ERG's preference for using the ITT population, the AC agreed with the approach taken in the ERG base case. The original company and ERG base-case incremental cost-effectiveness ratios (ICERs) were £46,722 and £57,688 per QALY gained, respectively; these changed to £28,563 and £38,061 per QALY gained, respectively, in the revised base cases applying a new PAS. Regarding the end-of-life criteria, after 24 months approximately 63 % of patients in the control group of the COU-AA-302 trial were still alive, and the median survival was 30.1 months (95 % CI 27.3-34.1). Therefore, it is unlikely that life expectancy would be less than 24 months. The AC stated that the most plausible ICER is likely between £28,600 and £32,800 per QALY gained, and concluded that AAP at this stage in the treatment pathway did not meet the end-of-life criterion for short life expectancy. Moreover, in March 2016, the AC produced the final guidance, stating that AAP is recommended, within its marketing authorisation, as an option for treating mCRPC. [ABSTRACT FROM AUTHOR]

Background: Lymphatic filariasis (LF), onchocerciasis, schistosomiasis, soil-transmitted helminths (STH) and trachoma represent the five most prevalent neglected tropical diseases (NTDs). They can be controlled or eliminated by means of safe and cost-effective interventions delivered through programs of Mass Drug Administration (MDA)—also named Preventive Chemotherapy (PCT). The WHO defined targets for NTD control/elimination by 2020, reinforced by the 2012 London Declaration, which, if achieved, would result in dramatic health gains. We estimated the potential economic benefit of achieving these targets, focusing specifically on productivity and out-of-pocket payments. Methods: Productivity loss was calculated by combining disease frequency with productivity loss from the disease, from the perspective of affected individuals. Productivity gain was calculated by deducting the total loss expected in the target achievement scenario from the loss in a counterfactual scenario where it was assumed the pre-intervention situation in 1990 regarding NTDs would continue unabated until 2030. Economic benefits from out-of-pocket payments (OPPs) were calculated similarly. Benefits are reported in 2005 US$ (purchasing power parity-adjusted and discounted at 3% per annum from 2010). Sensitivity analyses were used to assess the influence of changes in input parameters. Results: The economic benefit from productivity gain was estimated to be I$251 billion in 2011–2020 and I$313 billion in 2021–2030, considerably greater than the total OPPs averted of I$0.72 billion and I$0.96 billion in the same periods. The net benefit is expected to be US$ 27.4 and US$ 42.8 for every dollar invested during the same periods. Impact varies between NTDs and regions, since it is determined by disease prevalence and extent of disease-related productivity loss. Conclusion: Achieving the PCT-NTD targets for 2020 will yield significant economic benefits to affected individuals. Despite large uncertainty, these benefits far exceed the investment required by governments and their development partners within all reasonable scenarios. Given the concentration of the NTDs among the poorest households, these investments represent good value for money in efforts to share the world’s prosperity and reduce inequity. [ABSTRACT FROM AUTHOR]

Objective. To evaluate to what extent rheumatologists consider economic aspects and patients' preferences when choosing drug treatments in patients with active RA. Methods. In a discrete choice experiment, rheumatologists were asked to choose between two unlabelled drug treatment options for a hypothetical RA patient with moderate disease activity who failed two synthetic DMARDs. Attributes and levels of drug treatments were selected based on existing literature, rheumatologists' opinion and expert consensus. This resulted in five attributes each described by three levels: efficacy (level of improvement and achieved state on DAS28), safety (probability of a serious adverse event), patients' preference (level of agreement), annual medication costs and cost-effectiveness (incremental cost-effectiveness ratio). An efficient experimental design generated 14 treatment choices and a random parameter logit model estimated the relative importance of attributes. Results. Sixty-three rheumatologists from the Netherlands contributed to the analysis; 44% were female and mean (S.D.) age was 49 (8) years. Drug efficacy had the strongest relative contribution to the drug choice (44%) followed by medication costs (24%), patients' preference (17%) and cost-effectiveness (14%). Patients' preferences were most relevant when patients disliked a proposed treatment. The risk of serious but uncommon or rare side effects only played a minor role in the treatment choice (1 %). Conclusion. In addition to drug efficacy, rheumatologists account for economic aspects and for patients' preferences when deciding on drugs. Decisions are more influenced by absolute costs than relative cost-effectiveness and by patients' disliking as opposed to favouring the treatment. [ABSTRACT FROM AUTHOR]

Background: Limited information is available on short-term prognosis of Alzheimer's disease (AD) biomarkers in cerebrospinal fluid (CSF) in addition to routine diagnostic workup.Objective: This study aims to investigate the added prognostic value of AD CSF biomarkers.Methods: In a prospective cohort study, clinical experts predicted cognitive and functional symptoms in 114 memory clinic patients by assessing comprehensive routine diagnostic test information (patient history, and physical, neurological, psychiatric, neuropsychological, and MRI examinations), without and with CSF biomarkers. The reference standard was the 'observed clinically relevant decline' using baseline and 1- and 2-year follow-up information.Results: Decline over a 2-year period was observed in 51% of all participants (3% in SMC, 48% in MCI, 90% in mild dementia). In the total sample, the accuracy of predicted decline did not differ significantly between routine assessment without (79% correctly predicted) and with (74% correctly predicted) CSF biomarkers. Subgroup analyses revealed 25 (83%) correct predictions in SMC, 30 (68%) in MCI, and 35 (88%) in dementia without the use of CSF; and 21 (70%), 27 (61%), and 36 (90%), respectively, with the use of CSF in addition to the routine assessment.Conclusion: AD CSF biomarkers did not increase accuracy of 2-year prognosis of cognitive and functional decline when added to routine diagnostic workup. This suggests that the standard diagnostic workup without CSF biomarkers allows fairly accurate predictions for the short-term course of symptoms. Routine AD biomarkers in CSF have limited prognostic value over 2 years in persons with a suspected cognitive disorder. [ABSTRACT FROM AUTHOR]

Background: Neglected Tropical Diseases (NTDs) not only cause health and life expectancy loss, but can also lead to economic consequences including reduced ability to work. This article describes a systematic literature review of the effect on the economic productivity of individuals affected by one of the five worldwide most prevalent NTDs: lymphatic filariasis, onchocerciasis, schistosomiasis, soil-transmitted helminths (ascariasis, trichuriasis, and hookworm infection) and trachoma. These diseases are eligible to preventive chemotherapy (PCT). Methodology/Principal Findings: Eleven bibliographic databases were searched using different names of all NTDs and various keywords relating to productivity. Additional references were identified through reference lists from relevant papers. Of the 5316 unique publications found in the database searches, thirteen papers were identified for lymphatic filariasis, ten for onchocerciasis, eleven for schistosomiasis, six for soil-transmitted helminths and three for trachoma. Besides the scarcity in publications reporting the degree of productivity loss, this review revealed large variation in the estimated productivity loss related to these NTDs. Conclusions: It is clear that productivity is affected by NTDs, although the actual impact depends on the type and severity of the NTD as well as on the context where the disease occurs. The largest impact on productivity loss of individuals affected by one of these diseases seems to be due to blindness from onchocerciasis and severe schistosomiasis manifestations; productivity loss due to trachoma-related blindness has never been studied directly. However, productivity loss at an individual level might differ from productivity loss at a population level because of differences in the prevalence of NTDs. Variation in estimated productivity loss between and within diseases is caused by differences in research methods and setting. Publications should provide enough information to enable readers to assess the quality and relevance of the study for their purposes. [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 article presents the study on the willingness of the consumer to invest money and time for health and public health intervention at lifestyle behaviour change (LBC) through contingent valuation (CV). CV survey and internal validity test were conducted to explore the association between willingness and explanatory variables including paid work, income, and experience. Results were outlined and conclude the relevance of broader quality of life outcomes to consumers decision making.

Background: Sequential treatment of ankylosing spondylitis (AS) that includes tumour necrosis factor-α antagonists (anti-TNF agents) has been applied in most of the Western countries. Existing cost-effectiveness (CE) models almost exclusively presented the incremental CE of anti-TNF agents using a closed cohort while budget impact studies are mainly lacking. Notwithstanding, information on impact on total population health and societal budget as well as on actual incremental CE for a given decision time span are important for decision makers. This study aimed at quantifying, for different decision time spans starting from January 1, 2014 in the Dutch society, (1) impact of sequential drug treatment strategies without and with inclusion of anti-TNF agents (Strategies 1 and 2, respectively) on total population health and societal cost, and (2) the actual incremental CE of Strategy 2 compared to Strategy 1. Methods: Dynamic population modelling was used to capture total population health and cost, and the actual incremental CE. Distinguishing the prevalent AS population on January 1,2014 and the incident AS cohorts in the subsequent 20 years, the model tracked individually an actual number of AS patients until death or end of the simulation time. During the simulation, data on patient characteristics, history of drug use, costs and health at discrete time points were generated. In Strategy 1, five nonsteroidal anti-inflammatory drugs (NSAIDs) were available but anti-TNF agents withdrawn. In Strategy 2, five NSAIDs and two anti-TNF agents continued to be available. Results: The predicted size of the prevalent AS population in the Dutch society varied within the range of 67,145-69,957 with 44-46% of the patients receiving anti-TNF agents over the period 2014-2034. The use of anti-TNF agents resulted in an increase in the annual drug costs (168.54-205.28 million Euros), but at the same time caused a decrease in the annual productivity costs (12.58-31.21 million Euros) and in annual costs of healthcare categories other than drugs (7.23-11.90 million Euros). Incremental cost (Euros) per QALY gained in Strategy 2 compared to Strategy 1 corresponding to decision time spans of 5,10,15 and 20 years improved slightly from 75,379 to 67,268, 63,938 and 61,129, respectively. At willingness-to-pay thresholds of 118,656,112,067,110,188 and 110,512 Euros, it was 99 % certain that Strategy 2 was cost-effective for decision time spans of 5,10,15 and 20, respectively. Conclusions: Using the dynamic population approach, the present model can project real-time data to inform a healthcare system decision that affects all actual number of AS patients eligible for anti-TNF agents within different decision time spans. The predicted total population costs of different categories in the present study can help plan the organization of the healthcare resources based on the national budget for the disease. [ABSTRACT FROM AUTHOR]

Background: Sequential treatment of ankylosing spondylitis (AS) that includes tumour necrosis factor-α antagonists (anti-TNF agents) has been applied in most of the Western countries. Existing cost-effectiveness (CE) models almost exclusively presented the incremental CE of anti-TNF agents using a closed cohort while budget impact studies are mainly lacking. Notwithstanding, information on impact on total population health and societal budget as well as on actual incremental CE for a given decision time span are important for decision makers. This study aimed at quantifying, for different decision time spans starting from January 1, 2014 in the Dutch society, (1) impact of sequential drug treatment strategies without and with inclusion of anti-TNF agents (Strategies 1 and 2, respectively) on total population health and societal cost, and (2) the actual incremental CE of Strategy 2 compared to Strategy 1. Methods: Dynamic population modelling was used to capture total population health and cost, and the actual incremental CE. Distinguishing the prevalent AS population on January 1, 2014 and the incident AS cohorts in the subsequent 20 years, the model tracked individually an actual number of AS patients until death or end of the simulation time. During the simulation, data on patient characteristics, history of drug use, costs and health at discrete time points were generated. In Strategy 1, five nonsteroidal anti-inflammatory drugs (NSAIDs) were available but anti-TNF agents withdrawn. In Strategy 2, five NSAIDs and two anti-TNF agents continued to be available. Results: The predicted size of the prevalent AS population in the Dutch society varied within the range of 67,145-69,957 with 44-46 % of the patients receiving anti-TNF agents over the period 2014-2034. The use of anti-TNF agents resulted in an increase in the annual drug costs (168.54-205.28 million Euros), but at the same time caused a decrease in the annual productivity costs (12.58-31.21 million Euros) and in annual costs of healthcare categories other than drugs (7.23-11.90 million Euros). Incremental cost (Euros) per QALY gained in Strategy 2 compared to Strategy 1 corresponding to decision time spans of 5, 10, 15 and 20 years improved slightly from 75,379 to 67,268, 63,938 and 61,129, respectively. At willingness-to-pay thresholds of 118,656, 112,067, 110,188 and 110,512 Euros, it was 99 % certain that Strategy 2 was cost-effective for decision time spans of 5, 10, 15 and 20, respectively. [ABSTRACT FROM AUTHOR]

Background: Most studies on birth settings investigate the association between planned place of birth at the start of labor and birth outcomes and intervention rates. To optimize maternity care it also is important to pay attention to the entire process of pregnancy and childbirth. This study explores the association between the initial preferred place of birth and model of care, and the course of pregnancy and labor in low-risk nulliparous women in the Netherlands. Methods: As part of a Dutch prospective cohort study (2007-2011), we compared medical indications during pregnancy and birth outcomes of 576 women who initially preferred a home birth (n = 226), a midwife-led hospital birth (n = 168) or an obstetrician-led hospital birth (n = 182). Data were obtained by a questionnaire before 20 weeks of gestation and by medical records. Analyses were performed according to the initial preferred place of birth. Results: Low-risk nulliparous women who preferred a home birth with midwife-led care were less likely to be diagnosed with a medical indication during pregnancy compared to women who preferred a birth with obstetrician-led care (OR 0.41 95% CI 0.25-0.66). Preferring a birth with midwife-led care - both at home and in hospital - was associated with lower odds of induced labor (OR 0.51 95% CI 0.28-0.95 respectively OR 0.42 95% CI 0.21-0.85) and epidural analgesia (OR 0.32 95% CI 0.18-0.56 respectively OR 0.34 95% CI 0.19-0.62) compared to preferring a birth with obstetrician-led care. In addition, women who preferred a home birth were less likely to experience augmentation of labor (OR 0.54 95% CI 0.32-0.93) and narcotic analgesia (OR 0.41 95% CI 0.21-0.79) compared to women who preferred a birth with obstetrician-led care. We observed no significant association between preferred place of birth and mode of birth. Conclusions: Nulliparous women who initially preferred a home birth were less likely to be diagnosed with a medical indication during pregnancy. Women who initially preferred a birth with midwife-led care - both at home and in hospital - experienced lower rates of interventions during labor. Although some differences can be attributed to the model of care, we suggest that characteristics and attitudes of women themselves also play an important role. [ABSTRACT FROM AUTHOR]

Background In the absence of a gold standard, a panel of experts can be invited to assign a reference diagnosis for use in research. Available literature offers limited guidance on assembling and working with an expert panel for this purpose. We aimed to develop a protocol for an expert panel consensus diagnosis and evaluated its applicability in a pilot project. Methods An adjusted Delphi method was used, which started with the assessment of clinical vignettes by 3 experts individually, followed by a consensus discussion meeting to solve diagnostic discrepancies. A panel facilitator ensured that all experts were able to express their views, and encouraged the use of argumentation to arrive at a specific diagnosis, until consensus was reached by all experts. Eleven vignettes of patients suspected of having a primary neurodegenerative disease were presented to the experts. Clinical information was provided stepwise and included medical history, neurological, physical and cognitive function, brain MRI scan, and follow-up assessments over 2 years. After the consensus discussion meeting, the procedure was evaluated by the experts. Results The average degree of consensus for the reference diagnosis increased from 52% after individual assessment of the vignettes to 94% after the consensus discussion meeting. Average confidence in the diagnosis after individual assessment was 85%. This did not increase after the consensus discussion meeting. The process evaluation led to several recommendations for improvement of the protocol. Conclusion A protocol for attaining a reference diagnosis based on expert panel consensus was shown feasible in research practice. [ABSTRACT FROM AUTHOR]

Purpose To investigate the long-term health and economic consequences of direct treatment initiation in ocular hypertension patients. Methods A cost-effectiveness analysis with a societal perspective and a lifelong horizon was performed. The primary outcomes were the incremental quality-adjusted life years ( QALYs) and costs of direct pressure-lowering treatment for ocular hypertension, compared to a strategy where treatment is postponed until conversion to glaucoma has been observed. We used a decision analytic model based on individual patient simulation to forecast disease progression and treatment decisions in both strategies in a representative heterogeneous patient population and in 18 patient subgroups stratified by initial intraocular pressure and additional risk factors for conversion. Results The incremental discounted health gain of direct treatment was 0.27 QALYs, whereas the incremental discounted costs were −€ 649 during an average lifetime of 26 years. In the simulations of patient subgroups, the model outcomes moved towards higher health gains and lower incremental costs with increasing risk of conversion in the patient population. The incremental cost-effectiveness ratio of direct treatment ranged from € 15,425 per QALY gained in the lowest-risk subgroup to dominance in the highest-risk subgroup. Probabilistic sensitivity analysis indicated that uncertainty surrounding the model input parameters did not affect the conclusions. Conclusion Direct, early, pressure-lowering treatment is a dominant cost-effective treatment strategy over a strategy to start the same treatment approach later, after glaucoma has occurred for patients with ocular hypertension. Its implementation and consequences should be discussed with ophthalmologists and individual patients. [ABSTRACT FROM AUTHOR]

Background. The introduction of innovative non-invasive screening tests (e.g. tests based on stool and blood samples or both) may be a solution to increase colorectal cancer (CRC) screening uptake. However, preferences for these non-invasive screening tests have not been investigated in great detail yet. The purpose of this article therefore is to elicit individuals' preferences for different non-invasive screening tests in a Dutch screening campaign context. Material and methods. We investigate preferences by means of a labeled discrete choice experiment. Data of 815 individuals, aged 55-75 years, are used in the analysis. Results. Multinomial logit model analysis showed that the combi-test is generally preferred over the blood-test and the (currently available) stool-test. Furthermore, besides the large effect of screening test type, there are significant differences in preference depending on participants' socio-demographic background. Finally, the analysis showed a significant positive effect on screening test choice for the attributes sensitivity, risk reduction, and level of evidence and a non-significant effect for the attribute unnecessary follow-up test. Conclusion. Introducing new non-invasive screening tests that are based on a combination of stool and blood samples (or blood sample only) has the potential to increase CRC screening participation compared to the current standard stool-based test. [ABSTRACT FROM AUTHOR]