Leukoreduction, filtering white blood cells from transfusion blood, effectively avoids leukocyte-related complications of blood transfusion. The technology has proven its relative cost-effectiveness for specific patient populations. With the advent of variant Creutzfeldt–Jakob disease, a transmittable spongiform encephalopathy caused by mad cow disease (bovine spongiform encephalopathy), the hard hit United Kingdom introduced universal leukoreduction for all patients as a precaution for transmission of prions in 1999. This costly policy was followed by many other countries, in the absence of much evidence of an actual health problem or of a more than presumed effectiveness of leukoreduction in preventing prion transmission. The core problem proved to be legal. The blood banks are legally accountable for blood safety. This accountability is absolute, based on avoidance of all possible risks, regardless of costs. This strategy leads to inefficiencies in health care: (i) blood safety management is guided by available rather than cost-effective technology, and (ii) private insurance premiums for civil liability are sharply increasing, while they are in no way related to the expected returns and the high and increasing blood safety. A rational safety policy is to be optimal, taking into account costs and effects of the safety procedures. This issue will need an open discussion with the general public of the real risks and a clear and unambiguous definition of proportionality in the precautionary principle, based on the European law.

Background: Despite the growing medical and public interest in reducing exposure to allogeneic blood, little is known about the use of alternatives to allogeneic transfusion. This study was conducted to determine the availability of these technologies in Canadian hospitals and was undertaken under the auspices of the International Study of Peri-Operative Transfusion (ISPOT), a 10-country study of the effectiveness of, attitudes toward, and practices related to the use of alternatives to allogeneic transfusion.

Methods: A cross-sectional national mail survey of Canadian hospitals with greater than 50 medical/surgical beds. Chiefs of anesthesia, surgery, and the divisions of cardiac, orthopedic, vascular, and urology were initially mailed a brief postcard asking which of seven technologies were used in their center. This was then followed up with a one-page questionnaire asking how frequently the technologies were used, their thoughts on the appropriateness of the use of the technologies, barriers to their greater use, and reasons for nonuse of the technologies.

Results: Response rates to the postcard survey ranged from 70%–98%, depending on the technology and type of surgery, and ranged from 27%–53% for the follow-up questionnaire. All technologies were used most frequently in cardiac surgery. Aprotinin, tranexamic acid, aminocaproic acid, desmopressin, and cell salvage were reported used in over 70% of cardiac surgery centers. Of these, tranexamic acid and cell salvage were the only ones used routinely in some centers. Acute normovolemic hemodilution and erythropoietin were used in 45% and 20% of cardiac centers, respectively. The drugs were used in less than 15% of orthopedic, vascular, and urologic divisions, with the exception of desmopressin in urologic and vascular surgery and aminocaproic acid in urologic surgery. The techniques of cell salvage and acute normovolemic hemodilution were used in 30%–45% of these divisions, with the exception of cell salvage, which was used in less than 15% of urology units. In more than 60% of cases, the technologies were considered to be used “about right,” although an important minority felt that they were underused.

Conclusions: In general, alternatives to perioperative allogeneic transfusion were rarely used except in cardiac surgery.

There has been an increased focus on the relationship between health technology assessment (HTA) and regulatory assessments and how regulatory, HTA and coverage bodies, and industry can work better together to improve efficiency and alignment of processes. There is increasingly agreement across sectors that improved communication and coordination could contribute to facilitating timely patient access to effective, affordable treatments that offer value to the health system. Discussions on aspects of this relationship are being held in different forums and various forms of coordination and collaboration are being developed or piloted within several jurisdictions. It is therefore both timely and of value to stakeholders to describe and reflect on current initiatives intended to improve interactions between regulatory, HTA and coverage bodies, and industry. Drawing on 2011 meetings of the HTAi Policy Forum and the Center for Innovation in Regulatory Science (CIRS), this study aims to describe and compare initiatives, and point to success factors and challenges that are likely to inform future work and collaboration.

Objectives: In 2012, Colombia experienced an important institutional transformation after the establishment of the Health Technology Assessment Institute (IETS), the disbandment of the Regulatory Commission for Health and the reassignment of reimbursement decision-making powers to the Ministry of Health and Social Protection (MoHSP). These dynamic changes provided the opportunity to test Multi-Criteria Decision Analysis (MCDA) for systematic and more transparent resource-allocation decision-making.

Methods: During 2012 and 2013, the MCDA framework Evidence and Value: Impact on Decision Making (EVIDEM) was tested in Colombia. This consisted of a preparatory stage in which the investigators conducted literature searches and produced HTA reports for four interventions of interest, followed by a panel session with decision makers. This method was contrasted with a current approach used in Colombia for updating the publicly financed benefits package (POS), where narrative health technology assessment (HTA) reports are presented alongside comprehensive budget impact analyses (BIAs).

Results: Disease severity, size of population, and efficacy ranked at the top among fifteen preselected relevant criteria. MCDA estimates of technologies of interest ranged between 71 to 90 percent of maximum value. The ranking of technologies was sensitive to the methods used. Participants considered that a two-step approach including an MCDA template, complemented by a detailed BIA would be the best approach to assist decision-making in this context. Participants agreed that systematic priority setting should take place in Colombia.

Conclusions: This work may serve as the basis to the MoHSP on its interest of setting up a systematic and more transparent process for resource-allocation decision-making.

Objectives: The objective of this study was to conduct a cost-effectiveness analysis of Herceptin® from the hospital's point of view. This new biotechnological pharmaceutical is a humanized monoclonal antibody that targets the HER2 receptor, an important anti-cancer target.

Methods: A cost model with standard diagnostic and treatment options for breast cancer was set up for a Belgian university hospital in close collaboration with its specialists. Direct and indirect costs were calculated for each diagnostic and treatment option using the micro-costing method. Effectiveness was estimated through a literature study. The model allowed us to take cost consequences in other stages of the model into account and to calculate changes in monthly treatment costs from different “starting points.” With an incremental cost-effectiveness analysis, differences in costs and effectiveness with and without Herceptin® were compared.

Results: Over the complete treatment period from diagnosis until the metastatic phase, monthly costs for the hospital rose from €85.07 to €90.35 for stage I diagnosed breast cancer when adding Herceptin® to the model. In the metastatic phase alone, these costs rose from €1,132.33 to €1,256.23. With Herceptin®, we found an extra cost of €3,981.44 per extra life-month.

Conclusions: This cost-effectiveness ratio was rather high, because Herceptin® was quite expensive and the product was additive in its current use and did not replace existing treatments. Future research will concentrate on alternative applications of Herceptin® based on ongoing Herceptin® trials and expert opinions.

The issue of health benefits coverage—and its relation to health technology assessment (HTA)—has gained increasing attention in recent years. Economic constraints on health care, as well as the rapid pace of technological change, have forced European countries to face difficult choices in providing such care. The active use of coverage decision making has been proposed as a tool to help rationalize health care, and HTA has been advocated as a necessary activity to improve coverage decisions.

Objectives: After 4 years of deepening recession, Argentina's economy plummeted after default in 2002. This crisis critically affected health expenditures and triggered acute rationing. Our objective was to explore health decision-makers' knowledge and attitudes about economic evaluations (EE) and whether health technology assessment (HTA) were increasingly used for decision making.

Methods: A qualitative design based on semistructured interviews and focus groups was used to explore how decision makers belonging to different health sectors implement resource allocation decisions.

Results: Informants were mostly unaware of EE. The most important criteria mentioned to adopt a treatment were evidence of effectiveness, social/stakeholder demand, or resource availability. Despite general positive attitudes about EE, knowledge was rather limited. Although cost considerations were widely accepted by purchasers and managers, clinicians argued about these issues as interfering with the doctor–patient relationship. Other important perceived barriers to HTA use were lack of confidence in the transferability of studies conducted in developed countries and institutional fragmentation of the Argentine healthcare system. The new macroeconomic context was cited as a justification of implicit rationing measures. Although explicit priority setting was implemented by many purchasers and managers, HTA was not used to improve technical and/or allocative efficiency.

Conclusions: The crisis seems to be a strong incentive to extend the use of HTA in Argentina, provided decision makers are aware as well as involved in the generation of local studies.

The differences in survival rates of colon cancer patients between hospital districts were used as an indicator of equity in cancer treatment in Finland. The merits of two different assessment methods were evaluated. The large variation was mainly due to confounding by age and the extent of the disease and to random variation.

The need for better communication and collaboration between health technology assessment agencies led to the formation of an International Network of Agencies for Health Technology Assessment (INAHTA). The network now comprises 27 agencies and has been successful in improving exchange of information and in undertaking joint health technology assessment projects. Issues for the future include possible changes to criteria for membership and identification of resources for more extensive programs.

Objectives: Lifestyle changes to prevent type 2 diabetes among high-risk persons have been shown to be cost-effective. This study investigates the cost-effectiveness of a community-based program promoting general population lifestyle changes to prevent diabetes.

Methods: The 10-year program was implemented in three municipalities in Sweden. Effectiveness was measured with a quasiexperimental cohort design, that is, risk factor levels in a population group aged 36–56 years at baseline and 8–10 years later (2,149 men; 3,092 women) in the program municipalities and a control area were compared. The incremental cost-utility analysis included future diabetes and cardiovascular disease-related health effects and societal costs (discounted 3 percent), estimated by a Markov model.

Results: In all areas, risk factor levels increased during follow-up, leading to increased societal costs of between SEK40,000 and 90,000 (1 Euro 2004 = SEK9.13; 1 US$ = SEK 7.35) and quality-adjusted life-year (QALY) losses between 0.12 and 0.48 per individual. Compared with the control area, the cost increases and QALY losses for women were more favorable in two program areas but less favorable in one, and less favorable for men in both areas (data unavailable for one municipality). The findings indicate that the program was cost-effective in only two female study groups.

Conclusions: Conflicting results on the cost-effectiveness of the program were obtained. As several potentially valuable aspects of the program are not included in the cost-effectiveness analysis, the societal value of the program might not be adequately reflected.

Objectives: The objective of this exploratory study was to survey international health technology assessment (HTA) professionals to determine attitudes toward ethics in HTA.

Methods: An exploratory, quantitative, cross-sectional study design was developed. The sample population (n = 636) was selected from authors of the 206 articles published in the International Journal of Technology Assessment in Health Care between 2005 and 2007. A survey instrument was piloted and e-mailed.

Results: The response rate was low (n = 104; 16.4 percent). Respondents were primarily middle-aged (46 ± 11 years) male (62 percent) health professionals from Western countries (n = 92; 88.5 percent), with a mean of 10 (± 6 years) years of HTA experience. Although at least 90 percent of respondents agreed that healthcare decisions involved value judgments and that ethical analysis was important to HTA, respondents were divided as to whether normative (n = 45; 44.6 percent) or descriptive (n = 38; 37.6 percent) ethical recommendations were necessary. Most respondents (n = 83; 81.4 percent) believed that HTA should include citizen participation, but two thirds (n = 67; 67.0 percent) agreed that the final decision should be restricted to decision makers. A majority of respondents thought that ethical analysis could be discussed anywhere within the HTA process, either by an expert trained in ethics (n = 62; 60.8 percent) or by an external consultant (n = 80; 78.4 percent).

Conclusions: This study showed that ethical discourse in HTA is constrained by practical considerations, which serves to limit moral inquiry. To increase ethical analysis, a positive attitude toward ethics needs to be fostered within the HTA community.

The dream of artificial blood has existed since the 1960s. Disease-free hemoglobin solutions will be commercially available in 1991. A willingness-to-pay (WTP) survey was undertaken to assess the tangible and intangible benefits to the public from this new product. The positive results suggest that third-party payers should cover this benefit and assist the diffusion of this new technology. From the perspective of industrial marketing, results suggest that there will be little consumer price resistance for hemoglobin solutions within the suggested price range of $225–300 per unit. Management implications of this new product are discussed.

Objectives: The aim of this study was to identify and discuss appropriate approaches to integrate ethical inquiry in health technology assessment (HTA).

Methods: The key question is how ethics can be integrated in HTA. This is addressed in two steps: by investigating what it means to integrate ethics in HTA, and by assessing how suitable the various methods in ethics are to be integrated in HTA according to these meanings of integration.

Results: In the first step, we found that integrating ethics can mean that ethics is (a) subsumed under or (b) combined with other parts of the HTA process; that it can be (c) coordinated with other parts; or that (d) ethics actively interacts and changes other parts of the HTA process. For the second step, we found that the various methods in ethics have different merits with respect to the four conceptions of integration in HTA.

Conclusions: Traditional approaches in moral philosophy tend to be most suited to be subsumed or combined, while processual approaches being close to the HTA or implementation process appear to be most suited to coordinated and interactive types of integration. The article provides a guide for choosing the ethics approach that appears most appropriate for the goals and process of a particular HTA.

Objectives: The aim of this study was to summarize the current evidence for the cost-effectiveness of primarily human papillomavirus (HPV) -based cervical cancer screening in settings with already established Papanicolaou test (Pap) programs. Emphasis was placed on the German situation with annual Pap screening.

Methods: Medical, economic, and health technology assessment (HTA) databases were systematically searched for cost-effectiveness studies comparing HPV to Pap screening. Study data were extracted, standardized, and summarized in cost-effectiveness plots contrasting HPV strategies to Pap screening with 1-, 2-, 3-, and 5-years interval. For each Pap setting, the likelihood of cost-effective HPV screening was assessed depending on willingness-to-pay.

Results: We reviewed twelve decision-analytic cost-effectiveness models. Study results showed wide variation due to methodical heterogeneity. Data synthesis revealed that the cost-effectiveness of HPV screening depends on the interval of the established Pap screening strategy. In comparison with Pap screening every 2 years, only 25 percent of the HPV-based screening strategies were cost-effective. However, in comparison with Pap screening every 1, 3, or 5 years, 83 percent, 55 percent, and 92 percent of HPV screening strategies were cost-effective, respectively. Results for settings with annual Pap screening are based on models assuming 100 percent screening coverage.

Conclusions: The introduction of HPV-based screening programs is cost-effective if the screening interval of the established Pap program exceeds 2 years. In settings with biennial Pap screening, introduction of HPV-based screening is unlikely to be cost-effective. Results also suggest cost-effectiveness of HPV-based screening in settings with annual Pap screening; however, this finding should be confirmed under realistic screening adherence assumptions.

Objectives: The EuroScan International Network is a global network of publicly funded early awareness and alert (EAA) systems for health technologies. We describe the EuroScan member agency systems and methods, and highlight the potential for increased collaboration.

Methods: EuroScan members completed postal questionnaires supplemented with telephone interviews in 2012 to elicit additional information and check equivalence of responses. Information was updated between March and May 2013.

Results: Fifteen of the seventeen member agencies responded. The principal purpose of agencies is to inform decisions on coverage or reimbursement of health services and decisions on undertaking secondary research. The main users of information are national governments; health professionals; health services purchasers, commissioners, and decision makers; and healthcare providers. Most EuroScan agencies are small with almost half having fewer than two whole time equivalent staff. Ten agencies use both active and passive identification approaches, four use only active approaches. Most start identification in the experimental or investigational stages of the technology life cycle. All agencies assessed technologies when they are between the investigational and established, but under diffusion stages. Barriers to collaboration revolve around different system aims, purposes, and requirements; a lack of staff, finance, or opportunity; language differences; and restrictions on dissemination.

Conclusions: Although many barriers to collaboration were identified, the majority of agencies were supportive of increased collaboration either involving the whole EuroScan Network or between individual agencies. Despite differences in the detailed identification processes, members thought that this was the most feasible phase to develop additional collaboration.

Objectives: An overview was produced of approaches currently used to evaluate chronic disease management in selected European countries. The study aims to describe the methods and metrics used in Europe as a first to help advance the methodological basis for their assessment.

Methods: A common template for collection of evaluation methods and performance measures was sent to key informants in twelve European countries; responses were summarized in tables based on template evaluation categories. Extracted data were descriptively analyzed.

Results: Approaches to the evaluation of chronic disease management vary widely in objectives, designs, metrics, observation period, and data collection methods. Half of the reported studies used noncontrolled designs. The majority measure clinical process measures, patient behavior and satisfaction, cost and utilization; several also used a range of structural indicators. Effects are usually observed over 1 or 3 years on patient populations with a single, commonly prevalent, chronic disease.

Conclusions: There is wide variation within and between European countries on approaches to evaluating chronic disease management in their objectives, designs, indicators, target audiences, and actors involved. This study is the first extensive, international overview of the area reported in the literature.

Objectives: Europe has many health technology assessment (HTA) agencies, each producing their own HTA reports. Adapting HTA reports for different contexts could reduce the need for multiple reports on the same health technology with resultant saving of time and resources. This study aims to examine and understand the process of adaptation, and to develop a toolkit that would help the adaptation of reports produced by other countries.

Methods: The methods used were a review of the literature; a survey of twenty-nine European HTA organizations, two rounds of a Delphi survey, a face-to-face meeting of twenty-one European network for Health Technology Assessment (EUnetHTA) representatives, iterative rounds of review, and two rounds of quality assurance testing (termed applicability testing).

Results: Descriptions of previous examples of adaptation in the literature are sparse. Most respondents had previous experience in adapting reports, and all believed that adaptation was useful, and there was the ability to benefit from the use of a toolkit to aid in the process. EUnetHTA Partners developed and tested an adaptation toolkit. The toolkit is composed of a series of checklists and resources that identify or clarify the relevance, reliability, and transferability of data and information from existing reports.

Conclusions: Consensus of opinion from twenty-nine European organizations/networks has indicated that the adaptation of HTA reports would be desirable and beneficial. A toolkit was developed to help with the adaptation of HTA reports produced in other settings. This collection of resources is available for use by all HTA agencies and can be accessed at: http://www.eunethta.net/upload/WP5/EUnetHTA_HTA_Adaptation_Toolkit_October08.pdf.

Objectives: Our aim was to review the recommendations given by health technology assessment (HTA) institutions in their methodological guidelines concerning the use of surrogate outcomes in their assessments. In a second step, we aimed at quantifying the role surrogate parameters take in assessment reports.

Methods: We analyzed methodological papers and guidelines from HTA agencies with International Network of Agencies for Health Technology Assessment membership as well as from institutions related to pharmaceutical regulation (i.e., reimbursement, pricing). We analyzed the use of surrogate outcomes in a sample of HTA reports randomly drawn from the HTA database. We checked methods, results (including evidence tables), and conclusions sections and extracted the outcomes reported. We report descriptive statistics on the presence of surrogate outcomes in the reports.

Results: We identified thirty-four methodological guidelines, twenty of them addressing the issue of outcome parameter choice and the problematic of surrogate outcomes. Overall HTA agencies call on caution regarding the reliance on surrogate outcomes. None of the agencies has provided a list or catalog of acceptable and validated surrogate outcomes. We extracted the outcome parameter of 140 HTA reports. Only around half of the reports determined the outcomes for the assessment prospectively. Surrogate outcomes had been used in 62 percent of the reports. However, only 3.6 percent were based upon surrogate outcomes exclusively. All of them assessed diagnostic or screening technologies and the surrogate outcomes were predominantly test characteristics.

Conclusions: HTA institutions seem to agree on a cautious approach to the use of surrogate outcomes in technology assessment. Thorough assessment of health technologies should not rely exclusively on surrogate outcomes.

Objectives: To determine if published figures on the proportion of articles included in systematic reviews and identified in electronic databases are applicable to an example from medical imaging.

Methods: A systematic review was performed. Additionally, sensitivity and precision of a MEDLINE search were compared with values from three published searches, each customized for a specific field.

Results: All articles included in the systematic review were in electronic databases. The MEDLINE search had low precision compared with searches in other fields.

Conclusions: In a specific area of medical imaging, electronic databases, including MEDLINE, are reliable sources of articles.