Objectives: Although cost-utility analysis (CUA) has been recommended by some experts as the preferred technique for economic evaluation, there is controversy regarding what costs should be included and how they should be measured. The purpose of this study was to: a) identify the cost components that have been included in published CUAs; b) catalogue the sources of valuation used; c) examine the methods employed for estimating costs; and d) explore whether methods have changed over time.

Methods: We conducted a comprehensive search of the published literature and systematically collected data on the cost estimation of CUAs. We audited the cost estimates in 228 CUAs.

Results: In most studies (99%), analysts included some direct healthcare costs. However, the inclusion of direct non-healthcare and time costs (17%) was generally lacking, as was productivity costs (8%). Only 6% of studies considered future costs in added life-years. In general, we found little evidence of change in methods over time. The most frequently used source for valuation of healthcare services was published estimates (73%). Few studies obtained utilization data from RCTs (10%) or relied on other primary data (23%). About two-thirds of studies conducted sensitivity analyses on cost estimates.

Conclusions: We found wide variations in the estimation of costs in published CUAs. The study underscores the need for more uniformity and transparency in the field, and continued vigilance over cost estimates in CUAs on the part of analysts, reviewers, and journal editors.

Objectives: Although ethics has been on the agenda in health technology assessment (HTA) since its inception, the integration of moral issues is still not standard and is performed in a vast variety of ways. Therefore, there is a need for a procedure for integrating moral issues in HTA.

Methods: Literature review of existing approaches together with application of various theories in moral philosophy and axiology.

Results: The article develops a set of questions that addresses a wide range of moral issues related to the assessment and implementation of health technology. The issues include general moral issues and moral issues related to stakeholders, methodology, characteristics of technology, and to the HTA process itself. The questions form a kind of checklist for use in HTAs.

Conclusions: The presented approach for integrating moral issues in HTA has a broad theoretical foundation and has shown to be useful in practice. Integrating ethical issues in HTAs can be of great importance with respect to the dissemination of HTA results and in efficient health policy making.

Programs that disseminate information to health care practitioners often do so partly to encourage appropriate changes in practice. However, merely providing information is seldom enough to accomplish such changes. If information transfer programs are to influence practice, they must be designed to maximize the conditions facilitating change. Reliance on a diffusion model for thinking about how information reaches practitioners has led researchers to over-emphasize the importance of exposure to information and ignore other factors that determine whether change will occur, such as practitioners' motivation to change, the context in which clinical decisions are made, and how information is presented. The fact that successful dissemination will not necessarily produce change also has implications for how information transfer programs should be monitored and evaluated.

Objectives: Exoskeletons are electromechanical devices that are worn by a human operator to increase their physical performance. Several exoskeletons have been developed to restore functional movements, such as walking, for those with paralysis due to neurological impairment. However, existing exoskeletons have limitations with respect to affordability, size, weight, speed, and efficiency, which may reduce their functional application. Therefore, the aim of this scoping review is to collect and narratively synthesize the perspectives of users of exoskeleton technology.

Methods: A systematic literature search was conducted across several healthcare related online databases.

Results: A total of 4,619 articles were identified, of which 51 were selected for full review. Only three studies were identified that met the inclusion criteria. Of these, one showed an incongruence between users’ expectations and experiences of device use; another reported perspectives on potential rather than actual device use, ranking design features in order of perceived importance; and the other reported ratings of ease of device use in training.

Conclusions: The heterogeneity of studies included within this review, leave the authors unable to suggest consensus as to user perspectives of exoskeleton technology. However, it is apparent that users are able to suggest priorities for exoskeleton design and that users’ perspectives of exoskeleton technology might change in response to experience of use. The authors, therefore, suggest that exoskeleton design should be an iterative process, whereby user perspectives are sought, incorporated and refined by tangible experience, to ensure that devices developed are acceptable to and usable by the populations they seek to re-enable.

Objective: As health technology assessment (HTA) organizations in Canada and around the world seek to involve the public and patients in their activities, frameworks to guide decisions about whom to involve, through which mechanisms, and at what stages of the HTA process have been lacking. The aim of this study was to describe the development and outputs of a comprehensive framework for involving the public and patients in a government agency's HTA process.

Methods: The framework was informed by a synthesis of international practice and published literature, a dialogue with local, national and international stakeholders, and the deliberations of a government agency's public engagement subcommittee in Ontario, Canada.

Results: The practice and literature synthesis failed to identify a single, optimal approach to involving the public and patients in HTA. Choice of methods should be considered in the context of each HTA stage, goals for incorporating societal and/or patient perspectives into the process, and relevant societal and/or patient values at stake. The resulting framework is structured around four actionable elements: (i) guiding principles and goals for public and patient involvement (PPI) in HTA, (ii) the establishment of a common language to support PPI efforts, (iii) a flexible array of PPI approaches, and (iv) on-going evaluation of PPI to inform adjustments over time.

Conclusions: A public and patient involvement framework has been developed for implementation in a government agency’s HTA process. Core elements of this framework may apply to other organizations responsible for HTA and health system quality improvement.

Objectives: Guidelines for colorectal cancer screening and surveillance in people at average risk and at increased risk have recently been published by the American Gastroenterological Association. The guidelines for the population at average risk were evaluated using cost-effectiveness analyses.

Methods: Since colorectal cancers primarily arise from precancerous adenomas, a state transition model of disease progression from adenomatous polyps was developed. Rather than assuming that polyps turn to cancer after a fixed interval (dwell time), such transitions were modeled to occur as an exponential function of the age of the polyps. Screening strategies included periodic fecal occult blood test, flexible sigmoidoscopy, double-contrast barium enema, and colonoscopy. Screening costs in 1994 dollars were estimated using Medicare and private claims data, and clinical parameters were based upon published studies.

Results: Cost per life-year saved was $12,636 for flexible sigmoidoscopy every 5 years and $14,394 for annual fecal occult blood testing. The assumption made for polyp dwell time critically affected the attractiveness of alternative screening strategies.

Conclusions: Sigmoidoscopy every 5 years and annual fecal blood testing were the two most cost-effective strategies, but with low compliance, occult blood testing was less cost-effective. Lowering colonoscopy costs greatly improved the cost-effectiveness of colonoscopy every 10 years.

In this article, we review the development of health technology assessment (HTA) in England and Wales, France, The Netherlands, and Sweden, and we summarize the reaction to these developments from a variety of different disciplinary and stakeholder perspectives (political science, sociology, economics, ethics, public health, general practice, clinical medicine, patients, and the pharmaceutical industry). We conclude that translating HTA into policy is a highly complex business and that, despite the growth of HTA over the past two decades, its influence on policy making, and its perceived relevance for people from a broad range of different perspectives, remains marginal.

The objective of health technology assessment (HTA) is to support decision making in health care. HTA does not claim to provide a definite solution to a health care problem, but to assist decision makers with evidence-based information about the clinical, ethical, social, and economic implications of the development, diffusion, and use of health care technology.

Background: In many countries, the incremental cost-effectiveness ratio (ICER) is used to assess whether an intervention is worth its costs. At the same time, policy makers often feel uncomfortable with refusing reimbursement of any intervention purely on the basis of the fact that the ICER exceeds a specific threshold value. Reluctance to define a single threshold value for the ICER seems to have been stronger in social security systems than in national healthcare services systems. This study explores how basic differences between healthcare systems impact upon the potential usefulness of an ICER threshold value.

Methods: This study is a narrative review of literature about the theoretical foundations of the ICER threshold value approach and its practical relevance in different types of healthcare systems.

Results: A single ICER threshold value cannot be maintained, defined, or measured and should not be used as a policy-making tool. None of the solutions presented up until now to make the ICER threshold approach a valuable policy-making tool overcome the important weaknesses of the approach.

Conclusions: ICERs and ICER threshold values are insufficient for assessing interventions' value for money. Rather, they should be considered as one element in the decision-making process. Complete rationalization of the decision-making process by means of quantitative decision criteria is undesirable and not feasible. Increasing transparency in the criteria used for a decision and explicitness about the relative importance of each criterion should, therefore, be the major goal.

Objectives: To develop and test an appropriateness of indications tool for total knee replacement (TKR) in patients with osteoarthritis.

Methods: Criteria were developed using a modified Delphi panel judgment. Another panel rated the same indications, and the results were compared with the main panel. Test-retest of the main panel was performed. Regression models were used to assess the contribution of each algorithm variable. A classification tree was developed.

Results: The procedure was considered appropriate in 167 (26.8%) scenarios, and there was agreement on 112 (67.1%) of them. When the rates of the main panel were compared with those of a second panel, the result was a kappa statistic of 0.75. The test-retest kappa for the main panel was 0.78. Neither in the first case nor in the second was there an instance in which a scenario classified as appropriate shifted to inappropriate or vice versa. The regression models showed that symptomatology and radiology were the variables that explained most of the variability of appropriateness as determined by panelists. In the classification tree performed, the probability of misclassification was 3.8% with 150 scenarios, of the 156 analyzed and classified correctly.

Conclusions: The previous parameters tested showed acceptable results for an evaluation tool. These results support the use of this algorithm as an aid in formulating clinical practice guidelines and to promote the appropriateness of TKR.

Objectives: The aim of this study was to gather qualitative and quantitative data on criteria considered by healthcare decision makers.

Methods: Using snowball sampling and an online questionnaire with forty-three criteria organized into ten clusters, decision makers were invited by an international task force to report which criteria they consider when making decisions on healthcare interventions in their context. Respondents reported whether each criterion is “currently considered,” “should be considered,” and its relative weight (scale 0–5). Differences in proportions of respondents were explored with inferential statistics across levels of decision (micro, meso, macro), decision maker perspectives, and world regions.

Results: A total of 140 decision makers (1/3 clinical, 2/3 policy) from 23 countries in five continents completed the survey. The most relevant criteria (top ranked for “Currently considered,” “Should be considered,” and weights) were Clinical efficacy/effectiveness, Safety, Quality of evidence, Disease severity, and Impact on healthcare costs. Organizational and skill requirements were frequently considered but had relatively low weights. For almost all criteria, a higher proportion of decision makers reported that they “Should be considered” than that they are “Currently considered” (p < .05). For more than 74 percent of criteria, there were no statistical differences in proportions across levels of decision, perspectives and world regions. Statistically significant differences across several comparisons were found for: Population priorities, Stakeholder pressure/interests, Capacity to stimulate research, Impact on partnership and collaboration, and Environmental impact.

Conclusions: Results suggest convergence among decision makers on the relevance of a core set of criteria and on the need to consider a wider range of criteria. Areas of divergence appear to be principally related to contextual factors.

Objectives: Health Technology Assessment (HTA) is defined as a policy research approach that examines the short- and long-term social consequences of the application or use of technology. Internationally different institutions have translated this definition to local contexts. In Denmark, HTA is comprehensive with focus on four aspects of the problem in question (technology [clinical evidence], economy, patient, and organization). The objective of this study is to study how the application of HTA differs across leading countries and to study the extent to which Danish HTA reports differ from foreign HTAs.

Methods: A sample of 433 HTA reports published in the period 1989–2002 by eleven leading institutions or agencies in Denmark and eight other countries were reviewed. We looked at the characteristics of the HTA with respect to focus on the four main aspects and the manner in which each aspect has been approached.

Results: The study shows health technology procedures to be the most common type of health technology assessed in HTAs and literature review to be the most often used method of analysis. Policy recommendations are only present in approximately half of the HTA reports.

Conclusions: In the HTAs one generally sees a great focus on the clinical aspect of health technologies, leaving the economic, the patient-related, and the organizational aspect much more unanalyzed. The Danish HTAs generally have a wider scope than HTAs produced in other countries and tend to focus more frequently on patient-related and organizational dimensions.

Objective: To evaluate the sensitivity and precision of various extended search methods in identifying randomized controlled trials (RCTs) for systematic reviews.

Method: Prospective analysis of extended search methods (specialized databases or trial registries, reference lists, hand-searching, personal communication, and Internet) used in two systematic reviews of RCTs. The gold standard was the total number of RCTs identified by major databases (MEDLINE, EMBASE, etc.) and extended search strategies combined. Sensitivity was the proportion of all known RCTs identified by any extended search method. Precision reflected the proportion of all items uncovered by any extended search method that actually were RCTs.

Results: The extended search identified 94 additional RCTs for the systematic reviews beyond those identified with the major databases. Specialized databases and trial registries had the highest sensitivity and precision for the lipid-lowering project (13.6% and 52.7%, respectively; p < .05) followed by scanning of reference lists (7.2% sensitivity and 41.9% precision; p < .05). Hand-searching was more effective than personal communication and Internet searching (1.7% sensitivity and 12.2% precision; p < .05). The acupuncture project had slightly different results, with the specialized databases and trial registries tied with the review of reference lists for highest sensitivity (14.2%). The precision followed the same trend as the lipid-lowering project (17.6% specialized databases; 8.3% reference lists; p < .05). A post-hoc analysis showed that 75 of the 94 RCTs were indexed in the major databases but missed by the major database search.

Conclusions: Extended searching identified additional RCTs for the systematic reviews beyond those found in major databases. Specialized databases and trial registries were most effective. An important number of RCTs were missed by the major database search. Timing and accuracy of indexing may explain this finding. The definitive measure, whether there is an association between the method used to uncover RCTs, the quality of the items uncovered and their impact on systematic review results, is yet to be determined.

Medical-technical possibilities are often experienced as being strongly compelling. This imperative character of medical technology not only applies to the medical profession, but also to patients who frequently find it very difficult to refuse medical examinations or treatments. This article discusses the technological imperative with regard to patients. It attempts to discover relationships with decision theory, particularly concerning “anticipated decision regret:” The fact that prevention of regret plays an important role in the use of medical technology is illustrated through a number of examples: liver transplantation, prenatal diagnosis, screening, and in vitro fertilization.

Objectives: To estimate the excess cost of stroke in Sweden and the potential costs that could be avoided by preventing first-ever strokes.

Methods: We adopted the incidence approach for estimating the present value of both direct and indirect costs. Data on mortality, stroke recurrence, and inpatient care were estimated from a national register of patient data with a four-year follow-up period. To estimate costs for social services, we used survey data on living conditions before stroke onset and at three and at twenty-four months. Costs for outpatient visits, rehabilitation, drugs, and production losses due to premature death and early retirement were estimated on the basis of both published and nonpublished sources. Lifetime costs were based on life tables adjusted for excess mortality of stroke, and costs in year 4 were extrapolated to subsequent years.

Results: The present value direct cost for an average stroke patient is SEK513,800 (US$56,024 or Euro60,825). The corresponding indirect cost is SEK125,110 (US$13,640 or Euro14,810). Almost 45 percent of the direct costs were attributable to social services. Women had higher costs than men, and costs for survivors increased with age due to social services.

Conclusions: With an incidence of 213 first-ever strokes per 100,000 individuals, the total excess direct and indirect cost of stroke would be SEK12.3 billion (approximately US$1.3 billion or Euro1.5 billion). Hence, there are large potential cost offsets both in the health-care sector and in the social service sector if the incidence of first-ever stroke could be reduced.

Objective: The objective of this study was to elicit the factors related to worldwide variation in biomedical research productivity.

Methods: Biomedical research articles published in 1990–2000 were accessed through the MEDLINE database. The number of articles (journal articles) originating from each of the countries of the world was elicited and normalized to publication per million population per year.

Results: In a multiple regression model, where publication per million population was the dependent variable and 10 social and economic indicators were independent variables, gross national product (GNP) per capita and research and development (R&D) expenditure emerged as significant factors. In separate simple linear regression analyses, all but two independent variables were found significant.

Conclusions: To increase biomedical research output, GNP per capita and R&D expenditure are the most important factors to address.

With the growing international literature in economic evaluation and the rapid spread of new health technologies, there is a need to undertake, or at least interpret, economic evaluations on the international level. However, the ways in which cross-national differences affect the cost-effectiveness of health technologies or their evaluations have never been studied. This paper explores these issues by taking advantage of a unique situation in which the same economic evaluation of a new indication for a health technology was conducted simultaneously in four countries using an identical methodology. The study showed that if prior agreement on methods can be reached and local data applied, economic evaluations can be undertaken in a way that facilitates the extrapolation of results from country to country.

Background: Since its inception, the field of health technology assessment (HTA) has stressed the need for consideration of ethical and social issues. However, few concepts or analytic tools have been developed, and because of the complexity of the endeavor and a lack of integration of work already produced, such concepts remain difficult to apply in HTA.

Objectives: Through a descriptive “map” of concepts, tools, and processes, we summarize the most tangible efforts on the part of HTA producers to address social and ethical issues.

Methods: A literature review and content analysis of HTA reports in the Centre for Reviews and Dissemination database enables a synthesis of the reflections on, initiatives around, and gaps in knowledge related to the integration of social and ethical issues in HTA.

Results: We examine: (i) the aim of integrating ethical and social issues in HTA, (ii) the theoretical approaches used, (iii) the methods and processes applied, and (iv) the implications for HTA producers. We highlight two levels at which social and ethical issues can be considered: throughout the production process of HTA reports and as part of the organizational structure of HTA agencies.

Conclusions: Given the profound societal changes that occur in relation to healthcare technology development, HTA producers have a responsibility to inform and enlighten technology-related public and policy debates. Fulfilling this role, though, requires that socioethical dimensions of technology and HTA are made explicit.

Objectives: The objective of this study is to apply a Markov model to compare cost-effectiveness of total knee replacement (TKR) using computer-assisted surgery (CAS) with that of TKR using a conventional manual method in the absence of formal clinical trial evidence.

Methods: A structured search was carried out to identify evidence relating to the clinical outcome, cost, and effectiveness of TKR. Nine Markov states were identified based on the progress of the disease after TKR. Effectiveness was expressed by quality-adjusted life years (QALYs). The simulation was carried out initially for 120 cycles of a month each, starting with 1,000 TKRs. A discount rate of 3.5 percent was used for both cost and effectiveness in the incremental cost-effectiveness analysis. Then, a probabilistic sensitivity analysis was carried out using a Monte Carlo approach with 10,000 iterations.

Results: Computer-assisted TKR was a long-term cost-effective technology, but the QALYs gained were small. After the first 2 years, the incremental cost per QALY of computer-assisted TKR was dominant because of cheaper and more QALYs. The incremental cost-effectiveness ratio (ICER) was sensitive to the “effect of CAS,” to the CAS extra cost, and to the utility of the state “Normal health after primary TKR,” but it was not sensitive to utilities of other Markov states. Both probabilistic and deterministic analyses produced similar cumulative serious or minor complication rates and complex or simple revision rates. They also produced similar ICERs.

Conclusions: Compared with conventional TKR, computer-assisted TKR is a cost-saving technology in the long-term and may offer small additional QALYs. The “effect of CAS” is to reduce revision rates and complications through more accurate and precise alignment, and although the conclusions from the model, even when allowing for a full probabilistic analysis of uncertainty, are clear, the “effect of CAS” on the rate of revisions awaits long-term clinical evidence.