This article dicusses the individual's choice to engage in informal care as an issue for economic evaluation. Traditional methods used in economic evaluation studies for valuing time spent on informal care are discussed and an alternative method is put forward that incorporates the quality of life of caregivers as an outcome measure to represent the effects on caregivers. The methodological issues concerning the valuation of informal caregivers' time are becoming more important as new drugs and other health care technologies are introduced for patients with diseases that are typically associated with informal care.

Menorrhagia can be treated by drug therapy or surgery. General practitioners (GPs) can prescribe drugs to reduce menstrual blood loss as first-line treatment, referring patients for surgical treatment if drug therapy fails. This study examined the efficacy of drugs used to treat menorrhagia and surveyed British GPs to discover the extent to which they prescribed the most effective drugs for this condition. The results suggest that treatment of this condition in primary care falls short of desirable standards. A meta-analysis of randomized trials of drug therapy revealed wide differences in efficacy and side effects. The most effective drug (tranexamic acid) is little used by British GPs, whereas the least effective drug (norethisterone) is the most frequently prescribed.

For the past several years, we have discussed the idea of producing a publication on the history of health technology assessment (HTA). It seemed important to us to develop this history now, while those who lived it can give their own accounts. An exception is Seymour Perry, the first president of ISTAHC and the Director of the first national public program in HTA, the U.S. National Center for Health Care Technology (NCHCT). See the In Memoriam in this issue.

In most of the cost-utility literature, quality-adjusted life-year (QALY) gains are interpreted as a measure of social value. Given this interpretation, the validity of different multi-attribute health-state scaling instruments may be tested by comparing the values they provide on the 0–1 QALY scale with directly elicited preferences for person trade-offs between different treatments (equivalence of numbers of different patients treated). Norwegian and Australian public preferences as measured by the person trade-off suggest that the EuroQol Instrument assigns excessively low values to health states. This seems to be even more true of the McMaster Health Classification System. The Quality of Well-being Scale appears to compress states toward the middle of the 0–1 scale. By contrast, the Rosser/Kind index fits reasonably well with directly measured person trade-off data.

Objectives: This study describes the health technology assessment (HTA) framework introduced by Regione Lombardia to regulate the introduction of new technologies. The study outlines the process and dimensions adopted to prioritize, assess and appraise the requests of new technologies.

Methods: The HTA framework incorporates and adapts elements from the EUnetHTA Core Model and the EVIDEM framework. It includes dimensions, topics, and issues provided by EUnetHTA Core Model to collect data and process the assessment. Decision making is instead supported by the criteria and Multi-Criteria Decision Analysis technique from the EVIDEM consortium.

Results: The HTA framework moves along three process stages: (i) prioritization of requests, (ii) assessment of prioritized technology, (iii) appraisal of technology in support of decision making. Requests received by Regione Lombardia are first prioritized according to their relevance along eight dimensions (e.g., costs, efficiency and efficacy, organizational impact, safety). Evidence about the impacts of the prioritized technologies is then collected following the issues and topics provided by EUnetHTA Core Model. Finally, the Multi-Criteria Decision Analysis technique is used to appraise the novel technology and support Regione Lombardia decision making.

Conclusions: The VTS (Valutazione delle Tecnologie Sanitarie) framework has been successfully implemented at the end of 2011. From its inception, twenty-six technologies have been processed.

Objectives: Progression-free survival (PFS) has not been validated as a surrogate endpoint for overall survival (OS) for anthracycline (A) and taxane-based (T) chemotherapy in advanced breast cancer (ABC). Using trial-level, meta-analytic approaches, we evaluated PFS as a surrogate endpoint.

Methods: A literature review identified randomized, controlled A and T trials for ABC. Progression-based endpoints were classified by prospective definitions. Treatment effects were derived as hazard ratios for PFS (HRPFS) and OS (HROS). Kappa statistic assessed overall agreement. A fixed-effects regression model was used to predict HROS from observed HRPFS. Cross-validation was performed. Sensitivity and subgroup analyses were performed for PFS definition, year of last patient recruitment, line of treatment, and constant rate assumption.

Results: Sixteen A and fifteen T trials met inclusion criteria, producing seventeen A (n = 4,323) and seventeen T (n = 5,893) trial-arm pairs. Agreement (kappa statistic) between the direction of HROS and HRPFS was 0.71 for A (p = .0029) and 0.75 for T (p = .0028). While HRPFS was a statistically significant predictor of HROS for both A (p = .0019) and T (p = .012), the explained variances were 0.49 (A) and 0.35 (T). In cross-validation, 97 percent of the 95 percent prediction intervals crossed the equivalence line, and the direction of predicted HROS agreed with observed HROS in 82 percent (A) and 76 percent (T). Results were robust in sensitivity and subgroup analyses.

Conclusions: This meta-analysis suggests that the trial-level treatment effect on PFS is significantly associated with the trial-level treatment effect on OS. However, prediction of OS based on PFS is surrounded with uncertainty.

Methods often used for the valuation of health states are the time trade-off (TTO) and the visual analog scale (VAS). The VAS is easier than the TTO and can be self-administered; however it usually leads to lower scores. In the literature a power transformation of group mean VAS scores to TTO scores has been proposed. We were able to replicate this finding of a power function. We found coefficients that were very similar to those from the literature, for 183 cancer patients. The relationship existed independently of disease state and health status.

Objective: To assess the effect of the use of cochlear implants (CI) on the health status of postlingually deaf adults.

Methods: Participants comprised 45 postlingually deaf adult multichannel CI users and 46 deaf candidates on the waiting list for a CI. The latter group acted as control subjects to corroborate the validity of retrospective completion of the questionnaires by the CI recipients. Three HRQOL instruments were used: a) a specially developed CI questionnaire (NCIQ); b) a generic HRQOL questionnaire (SF-36); and c) a health-state classification system (HUI-2) suited to estimate single preference scores.

Results: Retrospectively estimated pre-implant scores in the CI user group corresponded very well with the scores in the control group. Postimplant scores in the CI users were substantially higher in all six domains (p < .001) of the NCIQ than the scores in the control group. Effects due to a CI were also observed with the SF-36 in five of the seven domains (p < .01). Statistically significant differences between the two groups (p = .001) were observed in two of the six domains of the HUI-2.

Conclusions: All three questionnaires detected improvements in HRQOL due to CI use. To make a detailed assessment of the effect of a CI on functional outcomes and well-being, a special purpose HRQOL instrument is far more adequate than a general HRQOL instrument. This study also showed that a CI affects several other health domains besides auditory performance. The effect of CI use on general functioning and well-being proved to be considerable.

Objectives: The aim of this study was to develop a decision-analytic model to assess the cost-effectiveness of spinal cord stimulation (SCS), relative to nonsurgical conventional medical management (CMM), for patients with failed back surgery syndrome (FBSS).

Methods: A decision tree and Markov model were developed to synthesize evidence on both health-care costs and outcomes for patients with FBSS. Outcome data of SCS and CMM were sourced from 2-year follow-up data of two randomized controlled trials (RCTs). Treatment effects were measured as levels of pain relief. Short- and long-term health-care costs were obtained from a detailed Canadian costing study in FBSS patients. Results are presented as incremental cost per quality adjusted life year (QALY) and expressed in 2003 Euros. Costs were discounted at 6 percent and outcomes at 1.5 percent.

Results: Over the lifetime of the patient, SCS was dominant (i.e., SCS is cost-saving and gives more health gain relative to CMM); a finding that was robust across sensitivity analyses. At a 2-year time horizon, SCS gave more health gain but at an increased cost relative to CMM. Given the uncertainty in effectiveness and cost parameters, the 2-year cost-effectiveness of SCS ranged from €30,370 in the base case to €63,511 in the worst-case scenario.

Conclusions: SCS was found to be both more effective and less costly than CMM, over the lifetime of a patient. In the short-term, although SCS is potentially cost-effective, the model results are highly sensitive to the choice of input parameters. Further empirical data are required to improve the precision in the estimation of short-term cost-effectiveness.

Objectives: The feasibility and convergent validity of a cost diary and a cost questionnaire was investigated.

Methods: Data were obtained as part of a cost-utility analysis alongside a multicenter clinical trial in patients with resectable rectal cancer. A sample of 107 patients from 30 hospitals was asked to keep a weekly diary during the first 3 months after surgery, and a monthly diary from 3 to 12 months after surgery. A second sample of seventy-two patients from twenty-eight hospitals in the trial received a questionnaire at 3, 6, and 12 months after surgery, referring to the previous 3 or 6 months. Format and items of the questions were similar and included a wide range of medical and nonmedical items and costs after hospitalization for surgery.

Results: Small differences were found with respect to nonresponse (range, 79 to 86 percent) and missing questions (range, 1 to 6 percent between the diary and questionnaire). For most estimates of volumes of care and of costs, the diary and questionnaire did not differ significantly. Total 3-month nonhospital costs were €1,860, €1,280, and €1,050 in the diary sample and €1,860, €1,090, and €840 in the questionnaire sample at 3, 6, and 12 months after surgery, respectively (p =.50). However, with respect to open questions, the diary sample tended to report significantly more care.

Conclusions: For the assessment of health-care utilization in economic evaluations alongside clinical trials, a cost questionnaire with structured closed questions may replace a cost diary for recall periods up to 6 months.

Research in unconventional medicine requires a number of different questions to build up a “mosaic” of evidence. Choice of research design depends on the question being asked and is independent of the therapy under investigation. Despite the doubts of some practitioners, randomized trials are of value for determining certain questions in alternative medicine.

Objectives: Although measuring the utilization of ambulatory and home-based healthcare resources is an essential component of economic analyses, very little methodological attention has been devoted to the development and evaluation of resource costing tools. This study evaluated a newly developed tool, the Ambulatory and Home Care Record (AHCR), which comprehensively evaluates costs incurred by the health system and care recipients and their unpaid caregivers.

Methods: The level of agreement between self-reports from 110 cystic fibrosis care recipients and administrative data was assessed for four categories of health services: home-based visits with healthcare professionals, ambulatory visits with healthcare professionals, laboratory and diagnostic tests, and prescription medications.

Results: Agreement between care recipients' reports on the AHCR and administrative data ranged from moderate (kappa=0.41; 95 percent confidence interval, 0.16–0.61) for physician specialist visits to perfect (kappa=1.0) for physiotherapy visits.

Conclusions: By evaluating and standardizing a resource and costing tool, such as the AHCR, economic evaluations may be improved and comparisons of the resource implications for different services and for diverse populations are possible.

Objectives: The aim of this study was to investigate doctors' knowledge of the relative and absolute costs of diagnostic tests, medical consumables (e.g., syringes or intravenous tubing), and healthcare visits as well as to determine factors influencing awareness.

Methods: For this systematic review, we searched the Cochrane Library, EconoLit, EMBASE, and MEDLINE; reviewed reference lists; and had contact with authors. Studies were included if either doctors or trainees were surveyed, there were >10 survey respondents, costs of diagnostic or therapeutic items were estimated, results were expressed quantitatively, and a clear description was provided of how authors defined Accurate Estimates and determined True Cost. Two authors reviewed each article for eligibility and extracted data independently. Cost accuracy outcomes were summarized, but data were not combined due to extensive heterogeneity.

Results: Fourteen articles were included in the final analysis. Cost accuracy was low; 33 percent of estimates were within 20 percent or 25 percent of true cost and 50 percent were within 50 percent or in the 50–200 percent range of the true cost. Country, year of study, level of training, and specialty did not impact accuracy. The cost of items appears to have no impact on the accuracy (Fisher's exact test, p = .41) or pattern of estimation (binomial test, p = .92).

Conclusions: Doctors have a limited understanding of diagnostic and nondrug therapeutic costs, and we could not identify anything that impacts understanding of these costs. More focus is required in the education of physicians about costs and the access to cost information.

Objectives: Stroke is the third largest cause of death and a major factor in permanent disability. Disparities in access to healthcare services exist due to geographical barriers and limited resources. Rural locations often lack the resources for adequate acute stroke care. Telestroke is intended to enable the transfer of knowledge of acute stroke management to areas with limited neurological services. The objective of this study is to assess the feasibility, acceptability, and treatment delivery reliability of telemedicine systems in acute stroke management.

Methods: A systematic review was undertaken.

Results: Eighteen studies were included in this systematic review. Telestroke services have been reported to lead to better functional health outcomes, including reduced mortality and dependency, compared with conventional care. Most studies report that systemic tissue plasminogen activator (tPA) treatment increased in hospitals providing telestroke services, although patients were often transferred to a stroke center for continuing monitoring and surveillance. Patients and healthcare providers reported high levels of satisfaction. There was limited evidence regarding the impact on resource utilization and cost-effectiveness.

Conclusions: Telemedicine systems can be safe, feasible, and acceptable in acute stroke management. Telestroke is associated with increased delivery of tPA. The lack of standardized measuring and reporting of resources and health outcomes hinder comparisons between telestroke networks and the determination of best practices. More research is needed to explore the clinical and economic impact of telemedicine technologies in acute stroke management, so as to support policy makers in making informed decisions.

Objectives: The aim of this study was to promote approaches to health technology assessment (HTA) that are both evidence-based and values-based. We conducted a systematic review of published studies describing formal methods to consider equity in the context of cost-effectiveness analysis (CEA).

Methods: Candidate studies were identified through an unrestricted search of the Pub Med and EMBASE databases. The search closed on January 20, 2011. We identified additional studies by consulting experts and checking article bibliographies. Two authors independently reviewed each candidate study to determine inclusion and extracted data from studies retained for review. In addition to documenting methods, data extraction identified implicit and explicit notions of fairness. Data were synthesized in narrative form. Study quality was not assessed.

Results: Of the 695 candidate articles, 51 were retained for review. We identified three broad methods to facilitate quantitative consideration of equity concerns in economic evaluation: integration of distributional concerns through equity weights and social welfare functions, exploration of the opportunity costs of alternative policy options through mathematical programming, and multi-criteria decision analysis.

Conclusions: Several viable techniques to integrate equity concerns within CEA now exist, ranging from descriptive approaches to the quantitative methods studied in this review. Two obstacles at the normative level have impeded their use in decision making to date: the multiplicity of concepts and values discussed under the rubric of equity, and the lack of a widely accepted normative source on which to ground controversial value choices. Clarification of equity concepts and attention to procedural fairness may strengthen use of these techniques in HTA decision making.

Objectives: Australia, Canada, and many European countries now use various forms of health technology assessment (HTA) in decision making regarding the reimbursement of drugs and other health technologies. To achieve a better understanding of the potential for use of HTA in this context, an analytical framework was developed to describe and classify existing fourth hurdle systems.

Methods: Based on a review of published literature, and official documentation, the key aspects of a fourth hurdle system were identified at two levels: policy implementation and individual technology decision. Characteristics of the systems were grouped under four main headings: constitution and governance, objectives, use of evidence and decision processes, and accountability. The comprehensiveness and relevance of this framework was assessed by an independent group of experts in HTA. A pilot study was undertaken, using only published sources, to test the feasibility of obtaining the information needed to complete the framework.

Results: The framework was found to be sufficiently broad to encompass all the issues of interest regarding the systems, but the proportion of information available from published sources was variable between sections of the framework and between countries, with average availability of 45 percent.

Conclusions: The analytical framework will help researchers and policy-makers in individual countries to understand their own systems and will allow some preliminary sharing of experience between countries. More experience of its application is needed to judge whether it will provide the basis for more formal comparison of systems and whether it will determine the appropriateness for particular decision contexts.

Objective: To determine the extent to which public health decision makers used five systematic reviews to make policy decisions, and to determine which characteristics predict their use.

Methods: This cross-sectional follow-up study of public health decision makers in Ontario collected primary data using a telephone survey and a short, self-administered organizational demographics questionnaire completed by the administrative assistant for each Medical Officer of Health. Independent variables included characteristics of the innovation, organization, environment, and individual. Data were entered into a computerized database developed specifically for this study, and multiple logistic regression analysis was conducted.

Results: The participation rate was very high, with 85% of public health units and 96% of available decision makers completing the survey. In addition, 63% of respondents stated they had used at least one of the systematic reviews in the previous 2 years to make a decision. The most important predictors of use were one's position, expecting to use a review in the future, and perceptions that the reviews were easy to use and that they overcame the barrier of limited critical appraisal skills.

Conclusions: Utilization of the systematic reviews in Ontario was very high. The utilization rates found in this study were significantly higher than those reported in previous utilization studies. One's position was found to be the strongest predictor of use, identifying program managers and directors as the most appropriate audience for systematic reviews.

Objectives: The European network on Health Technology Assessment (EUnetHTA) aimed to produce tangible and practical results to be used in the various phases of health technology assessment and to establish a framework and processes to support this. This article presents the background, objectives, and organization of EUnetHTA, which involved a total of sixty-four partner organizations.

Methods: Establishing an effective and sustainable structure for a transnational network involved many managerial, policy, and methodological tools, according to the objective of each task or Work Package. Transparency in organization, financial transactions, and decision making was a key principle in the management of the Project as was the commitment to appropriately involve stakeholders.

Results: EUnetHTA activities resulted in a clear management and governance structure, efficient partnership, and transnational cooperation. The Project developed a model for sustainable continuation of the EUnetHTA Collaboration.

Conclusions: The EUnetHTA Project achieved its goals by producing a suite of practical tools, a strong network, and plans for continuing the work in a sustainable EUnetHTA Collaboration that facilitates and promotes the use of HTA at national and regional levels. Responsiveness to political developments in Europe should be balanced with maintaining a high level of ambition to promote independent, evidence-based information and well-tested tools for best practice based on a strong network of HTA institutions.

Objectives: The use of economic evaluation studies (EE) in the decision-making process within the health-care system of nine Latin American (LA) and three European countries was investigated. The aim was to identify the opportunities, obstacles, and changes needed to facilitate the introduction of EE as a formal tool in health-care decision-making processes in LA.

Methods: A comparative study was conducted based on existing literature and information provided through a questionnaire applied to decision makers in Argentina, Brazil, Colombia, Cuba, Mexico, Nicaragua, Peru, Portugal Spain, United Kingdom, Uruguay, and Venezuela. Systematic electronic searches of HEED, NHS EED, and LILACS were conducted to identify published economic evaluation studies in LA from 1982 onward.

Results: There is relatively little evidence of the conduct and use of EE within the health care systems in LA. Electronic searches retrieved 554 records; however, only 93 were EE. In the nine LA participating countries, broad allocation of health-care resources is primarily based on political criteria, historical records, geographical areas, and specific groups of patients and diseases. Public-health provision and inclusion of services in health-insurance package are responsibilities of the Ministry of Health. Decisions regarding the purchase of medicines are primarily made through public tenders, and mainly based on differences in clinical efficacy and the price of health technologies of interest.

Conclusions: To expedite the process of incorporating EE as a formal tool to inform decision-making processes within the health-care systems in LA countries, two main conditions need to be fulfilled. First, adequate resources and skills need to be available to conduct EE of good quality. Second, decision-making procedures need to be modified to accommodate “evidence-based” approaches such as EE.

Background: Identifying treatments that offer value and value for money is becoming increasingly important, with interest in how health technology assessment (HTA) and decision makers can take appropriate account of what is of value to patients and to society, and in the relationship between innovation and assessments of value.

Methods: This study summarizes points from an Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations, discussions among attendees, and background papers.

Results and Conclusions: Various perspectives on value were considered; most place patient health at the core of value. Wider elements of value comprise other benefits for: patients; caregivers; the health and social care systems; and society. Most decision-making systems seek to take account of similar elements of value, although they are assessed and combined in different ways. Judgment in decisions remains important and cannot be replaced by mathematical approaches. There was discussion of the value of innovation and of the effects of value assessments on innovation. Discussion also included moving toward “progressive health system decision making,” an ongoing process whereby evidence-based decisions on use would be made at various stages in the technology lifecycle. Five actions are identified: (i) development of a general framework for the definition and assessment of value; development by HTA/coverage bodies and regulators of (ii) disease-specific guidance and (iii) further joint scientific advice for industry on demonstrating value; (iv) development of a framework for progressive licensing, usage, and reimbursement; and (v) promoting work to better adapt HTA, coverage, and procurement approaches to medical devices.