The use of economic evaluation in health care: Australian decision makers' perceptions

Decision making about the alternative uses of health care resources is an issue of critical concern for governments and administrators in all health care systems. While many factors need to be taken into consideration when making these decisions, economic evaluation can help to determine the relative efficiency of different choices. Research in various countries suggests that economic evaluation is not being used by health care decision makers to the extent that health economists think that it should be. Interest in the use of economic evaluation is increasing in Australia but, to date, there has been no Australian research which looks at its use from the point of view of its potential users--the decision makers. This study fills that gap. It was found that there was a high level of awareness of economic evaluation among the group of decision makers interviewed and that some had used it in their decision making. However decisions often have to be made quickly and take into account factors other than efficiency, hence limiting the use of economics. Other problems limiting its use were availability of data and lack of expertise. Those interviewed suggested a number of ways in which the problems they identified could be overcome. In particular, they recommended that researchers doing economic evaluations should be more responsive to the needs of the decision makers using them.     

Economic Evaluation of Vaccination Programs: A Guide for Selecting Modeling Approaches

ObjectivesIllustrate 3 economic evaluation methods whose value measures may be useful to decision makers considering vaccination programs.MethodsKeyword searches identified example publications of cost-effectiveness analysis (CEA), fiscal health modeling (FHM), and constrained optimization (CO) for economic evaluation of a vaccination program in countries where at least 2 of the methods had been used. We examined the extent to which different value measures may be useful for decision makers considering adoption of a new vaccination program. With these findings, we created a guide for selecting modeling approaches illustrating the decision-maker contexts and policy objectives for which each method may be useful.ResultsWe identified 8 countries with published evaluations for vaccination programs using >1 method for 4 infections: influenza, human papilloma virus, rotavirus, and malaria. CEA studies targeted health system decision makers using a threshold to determine the efficiency of a new vaccination program. FHM studies targeted public sector spending decision makers estimating lifetime changes in government tax revenue net of transfer payments. CO studies targeted decision makers selecting from a mix of options for preventing an infectious disease within budget and feasibility constraints. Cost and utility inputs, epidemiologic models, comparators, and constraints varied by modeling method.ConclusionsAlthough CEAs measures of incremental cost-effectiveness ratios are critical for understanding vaccination program efficiency for all decision makers determining access and reimbursement, FHMs provide measures of the program’s impact on public spending for government officials, and COs provide measures of the optimal mix of all prevention interventions for public health officials.     

The influence of economic evaluation studies on decision making. A European survey. The EUROMET group

Despite the growing activity in the field of health economics very little is known about the influence of economic evaluation studies on health care decision making in the EU member states. Several investigations about the impact of health economic studies on decision making have been performed, but most of them did not involve decision makers themselves. In this paper the results of the EUROMET survey are reported and discussed. Different types of decision makers in nine European countries were surveyed by postal questionnaires, semi-structured interviews and focus group discussions. Questions include issues about the extent of knowledge about economic evaluation, the actual and potential use of study results as well as barriers and incentives in the use of studies. It is concluded that despite the general positive attitude knowledge about the formal methodology is rather limited. Accordingly, results of economic evaluation studies are not widely used in decision making. The results show that institutional dimensions, such as difficulties in transferring budgets, are viewed as important barriers. Also, the lack of credibility of studies is assigned a high relevance. Moreover, decision makers wish for a better explanation of the practical relevance of studies and feel that there is a need for more training in health economics. Considering these requirements a number of recommendations for enhancing the value of health economic studies are given.     

EFFICIENCY AND EQUITY: A STATED PREFERENCE APPROACH

Outcome measurement in the economic evaluation of health care considers outcomes independent of to whom they accrue. This article reports on a discrete choice experiment designed to elicit population preferences regarding the allocation of health gain between hypothetical groups of potential patients. A random‐effects probit model is estimated, and a technique for converting these results into equity weights for use in economic evaluation is adopted. On average, the modelling predicts a relatively high social value on health gains accruing to nonsmokers, carers, those with a low income and those with an expected age of death less than 45 years. Respondents tend to favour individuals with similar characteristics to themselves. These results challenge the conventional practice of assuming constant equity weighting. For decision makers, whether a formal equity weighting system represents an improvement on more informal approaches to weighing up equity and efficiency concerns remains uncertain. Copyright © 2012 John Wiley & Sons, Ltd.     

Do economic evaluation studies inform effective healthcare resource allocation in Iran? A critical review of the literature

To aid informed health sector decision-making, data from sufficient high quality economic evaluations must be available to policy makers. To date, no known study has analysed the quantity and quality of available Iranian economic evaluation studies. This study aimed to assess the quantity, quality and targeting of economic evaluation studies conducted in the Iranian context.The study systematically reviewed full economic evaluation studies (n?=?30) published between 1999 and 2012 in international and local journals. The findings of the review indicate that although the literature on economic evaluation in Iran is growing, these evaluations were of poor quality and suffer from several major methodological flaws. Furthermore, the review reveals that economic evaluation studies have not addressed the major health problems in Iran.While the availability of evidence is no guarantee that it will be used to aid decision-making, the absence of evidence will certainly preclude its use. Considering the deficiencies in the data identified by this review, current economic evaluations cannot be a useful source of information for decision makers in Iran. To improve the quality and overall usefulness of economic evaluations we would recommend; 1) developing clear national guidelines for the conduct of economic evaluations, 2) highlighting priority areas where information from such studies would be most useful and 3) training researchers and policy makers in the calculation and use of economic evaluation data.     

A critical review of health-related economic evaluations in Australia: implications for health policy

In Australia, as in many other countries, economic evaluation is increasingly seen by health care policy makers as a useful aid to priority setting and resource allocation. In Australia, economic evaluation is now a requirement for new drugs to be listed on the Pharmaceutical Benefits Scheme which provides a government subsidy on the price of listed drugs for purchasers. Yet, despite recognition of the importance of economic evaluation by policy makers, there is a paucity of published evaluations in Australia. We reviewed all of the 33 health-related economic evaluations conducted in Australia and subsequently published since 1978. This study assesses how well informed decision makers might be if they used the results and conclusions of published economic evaluations as an aid to resource allocation. The review highlights several issues: (i) it is difficult to interpret the conclusions or assess the generalisability of individual papers without information on the context of the original study; (ii) the choice of comparator(s) was often unexplained and most papers did not employ marginal analysis; (iii) in the absence of marginal analysis, the comparability of cost-effectiveness ratios in league tables must be questioned as well as the completeness (were all the relevant alternatives included?) of studies; and (iv) the quality of effectiveness evidence varies enormously, with some authors content to use the best available evidence (even if it is of poor quality). The development of standards for economic evaluation methods might ensure a more consistent and scientific approach to evaluative work, but they cannot guarantee it. A more concerted effort to disseminate the principles and methods of economic evaluation to policy makers and non-economist evaluators might be a more important precursor to improving the credibility and usefulness of economic evaluations in priority setting.     

Increasing decision-makers' access to economic evaluations: alternative methods of communicating the information

OBJECTIVES: Although the importance of economic evaluations is recognized, research suggests the ways in which studies are summarized may not be optimal for a busy decision maker with little training in economics methodology. Therefore, the objective of this study was to seek decision makers' views on different summary formats, including a score, short summary, and structured abstracts of different degrees of detail. METHODS: We contacted 2,400 people, of which 84 decision makers volunteered and were presented, cumulatively, with different formats and asked whether these provided sufficient detail on the methodology and results of an economic study. RESULTS: From the fifty decision makers who responded to the questionnaire, it was found that the preferred combination was a very short summary, plus a more detailed structured abstract. It was also found that decision makers with economics training preferred the most detailed format, partly reflecting their reasons for consulting economic evaluations. CONCLUSIONS: Decision makers require both an initial screen of study content, plus more detail should they find the study relevant or interesting.     

Pre-assessment to assess the match between cost-effectiveness results and decision makers' information needs: an illustration using two cases in rehabilitation medicine in The Netherlands

OBJECTIVE: To determine if a pre-assessment can be used to establish whether cost-effectiveness results would meet the actual information needs of Dutch healthcare decision makers. METHODS: Two recent studies in rehabilitation medicine served as study material. Based on Wholey, a limited pre-assessment was performed in which the potential impact of cost-effectiveness analysis (CEA) results on intended users' decision making was assessed. Desk research and semi-structured interviews with several intended users of CEA results were performed. These included general practitioners, representatives of health insurance companies, the Health Care Insurance Board (CvZ), and medical guidelines committees. RESULTS: In day-to-day decision making of the interviewed decision makers, a cost-effectiveness criterion seemed to be of limited importance. Instead, results from clinical effectiveness studies and budget impact studies appeared to be sufficient. CvZ, however, preferred relative cost-effectiveness to be a criterion for inclusion in future reimbursement guidelines. In both cases the limited pre-assessments changed the expectations of the investigators regarding decision-making impact of an economic evaluation. CONCLUSION: This study revealed that the use of CEA results for Dutch micro- and meso-level healthcare decision making is not self-evident. The main purpose of CEA results is to support health policy making and planning at a macroeconomic level. Pre-assessment can be a valuable tool in designing a CEA to support the actual information needs of the decision makers.     

Methods for public health economic evaluation: A Delphi survey of decision makers in English and Welsh local government

Standard reference case methods recommended for health technology appraisals do not translate well to a public health setting. This paper reports on a Delphi survey designed to elicit views of public health decision makers in England and Wales, about different methodological elements of economic evaluation. This is important as methods should align with the objective function of decision makers. The Delphi survey comprised two rounds, with round 1 allowing open‐ended recommendations in addition to 5‐point Likert scale responses. The final survey comprised 36 questions, and levels and strength of agreement were assessed using median values and mean absolute deviation of the median. The Delphi panel (n = 66) achieved high levels of agreement for costs, health, well‐being, and productivity impact to be important elements within an economic evaluation. The panel agreed that evaluations should be relevant to the local context and include costs and consequences over a lifetime horizon. There was a call for the transparent reporting of costs and effects for different population subgroups, and for different sectors. Overall, the panel revealed a preference for a flexible approach, understanding that economic evidence fits within a dynamic process of decision making. These results provide empirical evidence to inform guidelines for public health economic evaluation.     

Elements for assessment of telemedicine applications.

OBJECTIVES: As an initiative of the International Network of Agencies for Health Technology Assessment, an approach to assessment of telemedicine applications was prepared to assist decision makers who are considering introduction and use of this technology. METHODS: Review and commentary drawing on published assessment frameworks and reports of primary evaluations of telemedicine, with particular reference to experience in Finland and Canada. RESULTS: Elements of the approach included development of a business case (considering population and services, personnel and consumers, delivery arrangements, specifications and costs); subsequent evaluation of the telemedicine application; and follow-up (covering the domains of technical assessment, effectiveness, user assessment of the technology, costs of telemedicine, trials, economic evaluation methods, and sensitivity analysis). CONCLUSIONS: Decision makers should link introduction of new and often costly technology to appraisal of its feasibility, followed by evaluation of the application, including longer term consideration of its sustainability and impact on the healthcare system. As the effectiveness and efficiency of telemedicine applications will often be strongly influenced by local issues, results of assessments may not be generalizable.     

Can economic evaluation guidelines improve efficiency in resource allocation? The cases of Portugal, The Netherlands, Finland, and the United Kingdom

The use of economic evaluation in decision making appears to have increased over the past few years and economic evaluation is looked upon as another measure to help contain costs and improve efficiency in an evidence-based decision-making environment. Following the examples of Australia and the Canadian Province of Ontario, four European Union (EU) countries (Finland, the Netherlands, Portugal, and the United Kingdom) have recently introduced economic evaluation guidelines. In addition to the Australian and Canadian guidelines, which constitute a hurdle to reimbursement, the paradigm that seems to be evolving in the four EU countries follows a similar route. Finland and the Netherlands seem to be moving toward the notion of a fourth hurdle to reimbursement, whereas the National Institute for Clinical Excellence in England and Wales was in principle meant to influence practice, although in reality this essentially acts as a hurdle to reimbursement, requiring a different data set to that used by regulatory authorities. Whereas the Portuguese guidelines were developed to assist in preparing economic submissions to support reimbursement decisions, they are unclear about when such evidence will be required and also discuss the dissemination of economic evidence to broader audiences. The introduction of these guidelines poses a number of challenges to policy makers, the implications of which are analyzed in the paper: a) to ensure that economic evaluations are carried out scientifically without industrial or political bias; b) to define an acceptable methodology that would increase their credibility; and c) to address certain practical issues ranging from deciding how to use economic evaluations in policy making to setting up new institutions or improving the coordination and dissemination of evidence. The variation in the use of economic evaluation guidelines in the four EU countries highlights the differences in national pharmaceutical policies and is in line with policy makers' continuous attempts to contain costs. While the paper critically discusses the guidelines, it also points out that a series of methodologic issues need to be addressed if economic criteria are to be introduced in policy making with the aim to improve resource allocation. The paper concludes that economic evaluation as a discipline is beginning to impact on policy, whereas the consistent use of economic evaluation results is, in principle, being adopted by policy makers but needs to go a step further to reach practitioners.     

Long-Term Drug Treatment of GERD

Gastroesophageal reflux disease (GERD) is one of the most common disorders of the gastrointestinal tract. Clinical presentation can vary from simple heartburn to erosive esophagitis. Some patients require long-term, possibly life-long, therapy. The cost of treatment is substantial, as patients with moderate to severe disease are high consumers of healthcare resources. The goal of therapy is to control symptoms, and to prevent complications, at a reasonable and manageable cost. Formulary decision making is more complex than simply choosing between proton pump inhibitors (PPIs) and histamine H₂ receptor antagonists (H₂RAs); more importantly, it involves choosing the most cost-effective treatment strategy for a patient population. Since there are no long-term prospective economic studies available on the management of GERD, modeling studies provide the primary source of data for decision makers. A critical review of selected studies found that conclusions can vary based on a number of factors. These include the severity of GERD in the population being modeled, effectiveness endpoints, costs (drug and nondrug), and the inclusion of anti-reflux surgery. Effectiveness variables used in these models are generally based on randomized control trials in which endoscopic findings are used to judge success. This differs from the management of symptoms, as is common in clinical practice. In addition, the selection of which randomized clinical trial is used to define effectiveness endpoints can have a significant effect on the outcome. Thus, generalizability of economic studies often limits their value to decision makers. Pharmaceutical manufacturers have funded most of the studies in this area. For formulary decision makers to apply long-term economic analysis to their clinical practice, a critical review of these analyses is essential. Economic analysis can be a supplement to rational clinical judgment and experience in formulary decision making.     

Pharmacoeconomic modelling in schizophrenia

Pharmacoeconomic evaluations are important elements in the decision making process, and decision tree analyses are statistical models that analyse both clinical and economic consequences of medical actions. Using one theoretic model, key confounding variables were identified that constituted a standardised framework for economic evaluation of schizophrenia management. The extent to which they were included in several previously published schizophrenia models was appraised. Five different models were developed, and a systematic review of schizophrenia modelling studies was conducted. Results indicate that atypical antipsychotics may be more or less cost-effective depending upon whether key confounding variables were taken into account, but vigilance is warranted when assessing data because serious discrepancies can occur between different methods of analysis. A need for standardised schizophrenia pharmacoeconomic models exists. Additionally, social rehabilitation should be considered because this may also influence outcomes. Standardising modelling techniques will facilitate adherence to guidelines issued by decision makers.     

Economic appraisal in the National Health Service: a survey.

Considerable emphasis is currently being placed on the pursuit of efficiency in health service provision. This paper reports the results of a survey of the use of economic appraisal to assist decision makers in choosing efficient courses of action. The survey group comprised National Health Service staff who had undertaken a correspondence course in health economics. The respondents were asked to identify issues arising locally where economic appraisal could have been applied but was not, and to suggest reasons why economic appraisal had not been used. They were then asked to give local examples of attempts to use economic appraisal and to indicate whether they were successful or what problems had been encountered. The results suggest that there is greater use of economic appraisal than is apparent from published sources but there is still not very much. The paper also summarises comments from the respondents on the decision making process in the National Health Service.     

Economic,Patient Preference,and Health-Related Quality of Life Considerations for Intranasal Corticosteroids in Allergic Rhinitis

Allergic rhinitis, as a medical condition, merits attention because of its prevalence in the population as well as the substantial economic impact of treating it. By virtue of their efficacy and low adverse effect profile, intranasal corticosteroids have gained recognition by healthcare providers as the first-line therapy for allergic rhinitis. For managed care decision makers, the use of intranasal corticosteroids as the gold standard of treatment in allergic rhinitis makes comparative economic and humanistic (patient preference or health-related quality of life [HR-QOL]) data between the various intranasal corticosteroids increasingly important for formulary decisions.Although the equal efficacy and safety of intranasal corticosteroid products in the treatment of allergic rhinitis is well documented, research that compares the different economic and humanistic aspects of intranasal corticosteroid products is limited and less conclusive. In this article, we review published studies reporting pharmacoeconomic and humanistic analyses of intranasal corticosteroids in the treatment of allergic rhinitis and make recommendations for managed care decision makers in the selection of intranasal corticosteroids for allergic rhinitis. Based on inclusion/exclusion criteria, 15 pharmacoeconomic and 19 patient preference/HR-QOL studies were selected and reviewed.The literature reviewed does not provide evidence of the superiority of a single intranasal corticosteroid product with respect to pharmacoeconomic, patient preference, or HR-QOL considerations. This finding is primarily owing to the lack of published head-to-head studies comparing pharmacoeconomic or humanistic outcomes between the different intranasal corticosteroids. Without further head-to-head studies on intranasal corticosteroids for the treatment of allergic rhinitis, cost minimization results may be the best decision strategy for managed care organizations (MCOs). Ideally, the results of cost-effectiveness or cost-utility studies comparing the different intranasal corticosteroids should guide the final formulary decision. In the absence of such studies, pharmacoeconomic and humanistic outcomes data from studies reported in the literature should be included into a pharmacoeconomic model, which considers the prevalence of allergic rhinitis in the MCOs to guide formulary inclusion decisions. Managed care decision makers will increasingly need to request this information from drug manufacturers if an informed, evidence-based decision is to be made.     

Methodological challenges posed by economic evaluations of early childhood intervention programmes

Early childhood intervention programmes have emerged in recent years with the aim of fostering the cognitive and social-emotional functioning and physical health of preschool children and enhancing their emerging competencies. This article presents a structured critical appraisal of economic evaluations of early childhood intervention programmes. It highlights a range of methodological issues in the field. These include: the fidelity of the evaluation process; the selection of the appropriate comparison group given the complexity of care routinely provided; the appropriate perspective and coverage of the study; methodological concerns relating to cost and benefit measurement and valuation; analytical requirements relating to the form of sensitivity analysis and the decision rules adopted by decision makers; and the interpretation of the results in the light of contextual factors. It is concluded that more transparent methodological guidance is required for analysts conducting economic evaluations of early childhood intervention programmes in particular and of public health interventions in general. Greater multidisciplinary collaboration between social scientists should also enhance the development of ground-breaking methods in this field.     

Methodological Challenges to Economic Evaluations of Vaccines: Is a Common Approach Still Possible?

Economic evaluation of vaccination is a key tool to inform effective spending on vaccines. However, many evaluations have been criticised for failing to capture features of vaccines which are relevant to decision makers. These include broader societal benefits (such as improved educational achievement, economic growth and political stability), reduced health disparities, medical innovation, reduced hospital beds pressures, greater peace of mind and synergies in economic benefits with non-vaccine interventions. Also, the fiscal implications of vaccination programmes are not always made explicit. Alternative methodological frameworks have been proposed to better capture these benefits. However, any broadening of the methodology for economic evaluation must also involve evaluations of non-vaccine interventions, and hence may not always benefit vaccines given a fixed health-care budget. The scope of an economic evaluation must consider the budget from which vaccines are funded, and the decision-maker’s stated aims for that spending to achieve.     

Five minutes with the Governor.

This paper aims to initiate a dialog among readers regarding the positioning and promotion of health-related decision analysis in the public sector. It is motivated by the author's personal observations that quantitative and economic evaluation methods continue to be viewed with skepticism by some public officials and that no consensus exists within our community as to what an appropriate message to such decision makers might be. A personal view of some key themes to be stressed in defining a vision for the field is presented.     

Questionnaire to Assess Relevance and Credibility of Modeling Studies for Informing Health Care Decision Making: An ISPOR-AMCP-NPC Good Practice Task Force Report

The evaluation of the cost and health implications of agreeing to cover a new health technology is best accomplished using a model that mathematically combines inputs from various sources, together with assumptions about how these fit together and what might happen in reality. This need to make assumptions, the complexity of the resulting framework, the technical knowledge required, as well as funding by interested parties have led many decision makers to distrust the results of models. To assist stakeholders reviewing a model’s report, questions pertaining to the credibility of a model were developed. Because credibility is insufficient, questions regarding relevance of the model results were also created. The questions are formulated such that they are readily answered and they are supplemented by helper questions that provide additional detail. Some responses indicate strongly that a model should not be used for decision making: these trigger a “fatal flaw” indicator. It is hoped that the use of this questionnaire, along with the three others in the series, will help disseminate what to look for in comparative effectiveness evidence, improve practices by researchers supplying these data, and ultimately facilitate their use by health care decision makers.     

Future Directions in Valuing Benefits for Estimating QALYs: Is Time Up for the EQ-5D?

The widespread adoption of the EuroQol 5-dimensional questionnaire (EQ-5D) has been important for the comparability, transparency, and consistency of economic evaluations for informing resource allocation in healthcare. The objectives of this article were to (1) critically assess whether the widespread adoption of the EQ-5D and its time trade-off–based value sets to inform economic evaluation is likely to continue and (2) speculate about how benefits may be measured and valued to inform economic evaluation in the future. Evidence supports the use of the EQ-5D in many areas of health, but there are notable gaps. Furthermore, there has been interest among some policy makers in measuring changes in well-being, and in using common outcomes across sectors. Possibilities for measuring well-being alongside health can be achieved through bolt-on dimensions or an entirely new measure capturing both health and well-being. Nevertheless, there are significant concerns about the logic of estimating a common utility function. The development of online valuation methods has had a major impact on the field, which is likely to continue. We, however, recommend more allowance for respondents to consider their answers. There is an ongoing debate on the role of patient values or experience-based values. To date, this has seen limited take-up by decision makers and there are significant technical problems to obtaining representative and meaningful values. Policy makers and the general population must decide on the focus and scope of benefits that are incorporated into economic evaluation, and current evidence on this is mixed. In part, this will determine whether the widespread adoption will continue.