Objectives: To examine the production of Health Technology Assessments (HTAs) with economic evaluations (EEs) conducted by Canadian HTA agencies.

Methods: This research used a three-step approach: (i) the Web sites of five Canadian organizations promoting HTA were searched to identify HTA reports with EEs; (ii) HTA agencies were surveyed to verify that our information was complete with respect to HTA activities and to describe the factors that influence the HTA process in Canada (i.e., selection of HTA topics, execution, dissemination of results and future trends); (iii) HTAs with EEs were appraised in terms of study design, retrieval of clinical and economic evidence, resource utilization and costing, effectiveness measures, treatment of uncertainty as well as presence of a budget impact analysis (BIA), and policy recommendations.

Results: Two hundred forty-nine HTA reports were identified of which 19 percent included EEs (n = 48). Decision analytic techniques were used in approximately 75 percent of the forty-eight EEs and probabilistic sensitivity analyses were commonly used by all agencies to deal with parameter uncertainty. BIAs or policy recommendations were given in 50 percent of the evaluations. Differences between agencies were observed in terms of selection of topics, focus of assessment and production of HTA (e.g., in-house activities). Major barriers to the conduct of HTAs with EEs were capacity, a lack of interest by decision makers and a lack of robust clinical information.

Conclusions: The results of this research point to the need for increased HTA training, collaboration, evidence synthesis, and use of pragmatic “real world” evaluations.

New models for determining the cost of care and length of stay in the neonatal intensive care unit (NICU) were developed using financial and clinical data from 588 admissions to our NICU. The model for determining costs explained 71% of the variability in total hospital costs. Models such as the ones developed in this study can be used to compare costs in different institutions, determine temporal trends in costs, and examine the financial impact of using new technologies. Such models can also be useful components of a rational prospective pricing system for the NICU.

Japanese surgeons have performed only one heart transplant in the quarter century since the procedure was developed. Possessing the requisite training and technology, transplant surgeons have been stymied by several factors that elude professional and political solution. Most critically, the lack of a brain death standard limits the availability of transplantable organs. Mistrust of the medical profession, traditional outlooks on death, and the primacy placed on consensual decision making have fueled debate about brain death and transplantation. Volatile and value laden, these issues have overwhelmed the discussion of health care resources, equal access to high-technology medical procedures, and insurance coverage for transplantation.

Objectives: The aim of this study was to explore the needs and requirements of decision makers in our regional healthcare system for health technology assessment (HTA) products to support portfolio development planning for a new HTA agency in Madrid, Spain.

Methods: A Delphi study was conducted during 2003. Questionnaires were developed based on a review of products and services offered by other agency members of the International Network of Agencies for Health Technology Assessment, and included preference and prioritization questions to evaluate twenty-two different products and services. The initial Delphi panel involved eighty-seven experts from twenty-one public hospitals, eleven primary healthcare centers, six private hospitals, and eight departments of the Regional Ministry of Health of the Community of Madrid.

Results: The global participation rate was 83.9 percent. Ten of the twenty-two possible products were rated of high interest by more than 80 percent of respondents. Important differences in preferences and priorities were detected across different settings. Public hospitals and primary healthcare centers shared a more “micro” perspective, preferring classic technology-centered HTA products, whereas private hospitals and Ministry representatives demanded more “macro” products and services such as organizational model and information system assessments.

Conclusions: The high participation rate supports the representativeness of the results for our regional context. The strategic development of an HTA portfolio based on decision makers’ needs and requirements as identified in this type of exercise should help achieve a better impact on policy development and decision making.

Objectives: The aim of this study was to evaluate available knowledge and identify knowledge gaps within the field of oral and maxillofacial surgery, by systematically collecting and evaluating systematic reviews. Twelve specific domains were selected: surgical removal of teeth, antibiotic and corticosteroid prophylaxis, orofacial infections, dental and facial trauma, orthognathic surgery, reconstructive surgery, benign tumors, cysts, premalignant lesions, oral complications of treatment of malignant tumors, hyperbaric oxygen therapy, temporomandibular joint surgery, cost effectiveness of different surgical treatments, and ethics.

Methods: The literature search, covering four databases, was conducted during September 2014: PubMed, The Cochrane library, Centre for Reviews and Dissemination and EBSCO dentistry and oral science source. Retrieved systematic reviews were quality assessed by AMSTAR.

Results: In all, 1,778 abstracts were identified, of which 200 met the inclusion criteria. Forty-five systematic reviews were assessed as of high to moderate quality. The results disclosed some existing evidence in a few domains, such as surgical removal of teeth and implant survival after sinus lifts. However, in all domains, the search revealed a large number of knowledge gaps. Also of concern was the lack of data regarding health economics and ethics.

Conclusions: In conclusion, there is a need for well-conducted clinical research in the fields of oral and maxillofacial surgery.

Objectives: The aim of this study, in the context of disinvestment related health technology assessment, is to examine whether analysis of Web 2.0—commercial media output, blogs, and discussion forums—can provide an understanding of media framing, community perspectives, and the sociopolitical aspects of an entrenched technology.

Methods: Thematic analysis of relevant data from fifty-nine media articles, thirty-nine discussion forums, thirteen blogs, and three Facebook pages relating to our case study: public funding for assisted reproductive technology services. Mainstream media and community-based social media responses were compared.

Results: Media responses were narrow, primarily describing emotive individual narratives or the political nexus of interests. Community (including patient) responses were broader including discussion of opportunity cost and vested interests but mostly reflected the polar ends of the debate, diverging strongly for or against disinvestment from public funding.

Conclusion: Web2.0 and media analysis offers an inexpensive method to capture media portrayal, divergent community responses both to that portrayal and independent of it, and insight into the sociopolitical aspects of an entrenched technology undergoing disinvestment debate.

The direct and indirect costs of acute myeloid leukemia were estimated for Sweden in 1989. The calculated total cost was SEK 460 million. Nearly half of the costs, or 1.7 million per patient diagnosed, were indirect costs due to premature mortality. Direct costs of relapses and indirect costs of mortality represent costs due to the absence of completely curable therapy. They also represent potential cost savings that could be ootained after introduction of new treatment options in the future.

Objectives: Legal analysis can highlight important issues that are relevant when deciding whether a medical technology should be implemented or reimbursed. Literature and studies show that even though the law is an acknowledged part of health technology assessment (HTA), legal issues are rarely considered in practice. One reason for this may be the lack of knowledge about the diversity of legal issues that are relevant for HTA. Therefore, this contribution aims primarily to identify and then explain the relevant legal issues in HTA. This study offers a framework for identifying the legal issues in HTAs in different jurisdictions and provides a basis for further research.

Methods: After extensive literature search, the authors review Swiss health law to identify legal issues that are relevant to HTA. The authors then categorize these legal issues using a framework with an inside and outside perspective. Finally, they explain a selection of these legal issues with several examples.

Results: This study reveals numerous legal issues that are relevant for HTA and underlines the necessity of incorporating legal analysis in HTAs. The suggested perspectival framework in this study provides a basis to structure the legal analysis. The identified legal issues are relevant in other countries and the perspectival framework is transferable to other jurisdictions.

Conclusions: The article underlines the importance of in-depth discussion about the role of law in HTA. It provides a structured overview of the legal issues in HTA and suggests a development of more concrete instruments toward a standardized legal technology assessment.

Objectives: Several studies have reported multiple barriers to and facilitators for the uptake of health technology assessment (HTA) information by policy makers. This study elicited, using best-worst scaling (BWS), the most important barriers and facilitators and their relative weight in the use of HTA by policy makers.

Methods: Two BWS object case surveys (one for barriers, one for facilitators) were conducted among sixteen policy makers and thirty-three HTA experts in the Netherlands. A list of twenty-two barriers and nineteen facilitators was included. In each choice task, participants were asked to choose the most important and the least important barrier/facilitator from a set of five. We used Hierarchical Bayes modeling to generate the mean relative importance score (RIS) for each factor and a subgroup analysis was conducted to assess differences between policy makers and HTA experts.

Results: The five most important barriers (RIS > 6.00) were “no explicit framework for decision-making process,” “insufficient support by stakeholders,” “lack of support,” “limited generalizability,” and “absence of appropriate incentives.” The six most important facilitators were: “availability of explicit framework for decision making,” “sufficient support by stakeholders,” “appropriate incentives,” “sufficient quality,” “sufficient awareness,” and “sufficient support within the organization.” Overall, perceptions did not differ markedly between policy makers and HTA experts.

Conclusions: Our study suggests that barriers and facilitators related to “policy characteristics” and “organization and resources” were particularly important. It is important to stimulate a pulse at the national level to create an explicit framework for including HTA in the decision-making context.

Objectives: This article provides a detailed understanding of the differences in selected formulary submission guidelines supplied by various health technology assessment (HTA) agencies and indicates how these differences can impact the evidence base used to populate the HTA.

Methods: Detailed summaries of the recommended methods for evidence generation, organized by topic areas relevant for clinical and economic data, for twelve countries in Europe, North America, and Australia where HTA processes are well developed were prepared. Using these summaries, we provide examples of the likely impact these differences in recommended methods could have on the evidence base used to evaluate new health technologies.

Results: Areas where recommendations differed included methodologies for systematic literature reviews (e.g., preferred databases and study designs for inclusion); selection of appropriate comparators; guidance on critical appraisal and synthesis of clinical evidence; appropriate sources for health value measures, resource use, and cost data; and approaches to uncertainty analyses. Performing literature searches that capture all relevant studies and then creating subsets of the literature based on a listing of country-specific requirements could allow for direct comparison of the evidence bases associated with the different guidelines.

Conclusions: If the formulary submission guidelines were followed as written, different (although overlapping) bodies of evidence likely would be generated for each country, which could contribute to disparate assessments and recommendations. This comparison of the formulary submission guidelines could contribute to an understanding of why clinical and reimbursement decisions vary across countries.

Objectives: The diffusion of cost-utility analyses (CUAs) through the medical literature was examined, documenting visible patterns and determining how they correspond with expectations about the diffusion of process innovations.

Methods: This study used 539 CUAs from a registry. It includes data elements comprising year of publication, the research center in which the study was performed, the clinical area covered by the CUA, and the specific journal. Finally, each paper was assigned to a journal type that could be one of the three categories: health services research, general medicine, or clinical specialty.

Results: When the average number of publications is plotted against time, the plot reveals an S-shaped curve. It appears that, whereas CUAs initially were published more frequently in general medical or health services research journals, there was a clear increase in the diffusion of CUA into subspecialty journals over time. The concentration ratio for research centers as measured by the Herfindhal–Hirschman Index decreased over time.

Conclusions: The spread of CUA through the medical literature follows patterns identified for the diffusion of other new technologies and processes. Future research should focus on what impact this spread has had on the practice of medicine and formulation of health policy.

Objectives: The aim of this study was to analyze the current status of population screening for colon/rectum cancer in Europe to compare the different strategies, the coverage, the existence of pilot experiences, regional coverages, and the risk factors considered in each strategy.

Methods: A comprehensive, systematic search was performed in the literature for documents addressing population screening for colon/rectum cancer in Europe. An ad hoc questionnaire was prepared including questions considered relevant. The questionnaire was reviewed by experts in the area. To identify key informants, colleague members of the International Network of Agencies for Health Technology Assessment (INAHTA), participants in the EUnetHTA project, or representatives of the ministries of health of the different European countries were contacted. The information provided by key informants was checked with information directly obtained from the ministries of health, gray literature, and research documents.

Results: An 88 percent response rate was obtained. In countries for which no questionnaire data were collected, information was directly retrieved from the Web sites of the corresponding ministries. Four countries were found to perform population screenings: Austria, France, Germany, and the United Kingdom. However, they used different strategies. Five countries had begun regional or local strategies: Denmark, Finland, Italy, Spain, and Switzerland, and two additional countries (the Netherlands and Norway) reported ongoing research studies intended to determine the best strategy to implement a population-based screening program. Differences were found in age range, procedure chosen, and follow-up period.

Conclusions: Even though the European Council recommended a wider implementation of population screening for colon/rectum cancer, our results suggest that this recommendation continues to be valid. The differences found in screening strategies (in terms of age range, procedures, risk factors considered, and follow-up periods) are not warranted by the results obtained in research studies or regional-national cancer registries.

The Danish healthcare services are mainly provided by public sector institutions. The system is highly decentralized. The state has little direct influence on the provision of healthcare services. State influence is exercised through legislation and budget allocations. The main task of the state is to initiate, co-ordinate, and advise. Counties, which run the hospitals, also decide on the placement of services. The hospital sector is controlled within the framework of legislation and global budgets. General practitioners occupy a central position in the Danish healthcare sector, acting as gatekeepers to the rest of the system. The system works well, and its structure has resulted in steady costs of health care for a long period. There is no regulatory mechanism in the Danish health services requiring use of health technology assessment (HTA) as a basis for policy decisions, planning, or administrative procedures. However, since the late 1970s a number of comprehensive assessments of health technology have formed the basis for national health policy decisions. In 1997, after years of public criticism of the quality of hospital care and health technologies, and on the basis of a previously developed national HTA strategy, a national institute for HTA (DIHTA) was established. There seems to be a growing awareness of evidence-based healthcare among health professionals and a general acceptance of health economic analyses as a basis for health policy decision making. This progress is coupled with growing regional HTA activity in the health services. HTA seems to have a bright future in Denmark.

Objectives: The aim of this study was to develop a decision support tool to assess the potential benefits and costs of new healthcare interventions.

Methods: The Canadian Partnership Against Cancer (CPAC) commissioned the development of a Cancer Risk Management Model (CRMM)—a computer microsimulation model that simulates individual lives one at a time, from birth to death, taking account of Canadian demographic and labor force characteristics, risk factor exposures, and health histories. Information from all the simulated lives is combined to produce aggregate measures of health outcomes for the population or for particular subpopulations.

Results: The CRMM can project the population health and economic impacts of cancer control programs in Canada and the impacts of major risk factors, cancer prevention, and screening programs and new cancer treatments on population health and costs to the healthcare system. It estimates both the direct costs of medical care, as well as lost earnings and impacts on tax revenues. The lung and colorectal modules are available through the CPAC Web site (www.cancerview.ca/cancerrriskmanagement) to registered users where structured scenarios can be explored for their projected impacts. Advanced users will be able to specify new scenarios or change existing modules by varying input parameters or by accessing open source code. Model development is now being extended to cervical and breast cancers.

Objectives: This work has investigated under what conditions cost-effectiveness data from a cluster randomized trial (CRT) are suitable for analysis using a cluster-adjusted nonparametric bootstrap. The bootstrap's main advantages are in dealing with skewed data and its ability to take correlations between costs and effects into account. However, there are known theoretical problems with a commonly used cluster bootstrap procedure, and the practical implications of these require investigation.

Methods: Simulations were used to estimate the coverage of confidence intervals around incremental cost-effectiveness ratios from CRTs using two bootstrap methods.

Results: The bootstrap gave excessively narrow confidence intervals, but there was evidence to suggest that, when the number of clusters per treatment arm exceeded 24, it might give acceptable results. The method that resampled individuals as well as clusters did not perform well when cost and effectiveness data were correlated.

Conclusions: If economic data from such trials are to be analyzed adequately, then there is a need for further investigations of more complex bootstrap procedures. Similarly, further research is required on methods such as the net benefit approach.

Objectives: The aim of this study was to analyze whether health technology assessment (HTA) reports published by SBU have influenced decisions, guidelines, clinical practice, or research priorities in Sweden.

Methods: All twenty-six SBU reports between 2006 and 2010 were analyzed. For each project, we searched publications and documentation that reflected impact on decisions, guidelines, research or clinical practice. Written documentation, before–after surveys or register-based time series data were used when available. Based on a conceptual model and on the available evidence, we determined whether HTA reports had a high, moderate, or low impact.

Results: HTA reports influenced comprehensive decisions to a high or moderate degree. In the case of fortifying flour with folic acid to a high degree. In ten cases, HTA reports were the primary source of clinical guidelines developed by the National Board of Health and Welfare (NBHW) or professional associations. In the cases of dyspepsia and gastro-esophageal reflux, as well as mild head injury, the HTA reports had a high impact on clinical practice. It was also obvious from this review that research had been initiated as a result of the knowledge gaps identified by HTA reports. In three cases, we had no adequate documentation, suggesting that the impact of the HTA report had been low.

Conclusions: Many interrelated forces change practice, but the cases presented here indicate that HTA reports have had a high impact on clinical guidelines, as well as a moderate or high impact on comprehensive decisions, the initiation of research and changes in clinical practice.

Over the last few years, the pharmaceutical industry has financed an increasing number of health economic evaluations. The purpose of this article is to describe how and why the pharmaceutical industry is applying health economic evaluations. Seven different fields of application are identified, and the varying reasons for use are discussed.

Objectives: Although endoscopic ultrasound (EUS) staging of esophageal cancer is established in clinical practice, high-quality evidence about its impact on patient outcomes is not available. This study aims to determine the impact of EUS for esophageal cancer staging on patient management and survival.

Methods: A systematic review was conducted using Medline, PreMedline, Embase, and The Cochrane Library. Included studies were (i) comparative studies reporting survival following EUS esophageal cancer staging, (ii) therapeutic impact studies reporting change in patient management following EUS. The quality of included studies was critically appraised.

Results: One systematic review, five studies reporting therapeutic impact, and two studies reporting patient survival were identified. The design and quality of the therapeutic impact studies varied widely. Management changed in 24–29 percent of patients following EUS staging of esophageal cancer (two studies). No studies provided data on the avoidance of surgery for this indication. One retrospective cohort study with historical control found EUS staging of esophageal cancer improved patient survival; a second study with similar design limitations did not find a survival benefit for EUS staging in patients undergoing resection. These studies had a high potential for bias, limiting the value of these findings.

Conclusions: Two studies provided evidence of a change in patient management following EUS for staging esophageal cancer, a higher level of evidence for a clinical benefit than can be obtained from accuracy studies alone. This evidence contributed to a recommendation for public funding of EUS in staging esophageal cancer in Australia.

Objectives: The cost-effectiveness of prenatal diagnosis intervention for Down's syndrome (DS) in China was assessed and evidence-based information for policy makers and providers is presented.

Methods: Based on field surveys in four selected cities in China and a literature review, the economic evaluation of prenatal diagnosis for DS from a societal perspective is conducted by cost-effectiveness analysis.

Results: In current clinical practice, for a cohort of 10,000 pregnant women, the strategy that delivers karyotyping by chorionic villus sampling (CVS) or amniocentesis (AC) only to those pregnant women 35 years of age and older (maternal age screening strategy) can detect .67 DS births. The strategy that offers the diagnostic test after maternal serum screening with α-fetoprotein and human chorionic gonadotrophin (maternal serum screening strategy) can detect 1.41 DS births. The cost per prevented DS birth by the maternal age screening strategy and maternal serum screening strategy is US$13,091 and US$56,048, respectively. Sensitivity analysis shows that the maternal serum screening strategy can be cost-effective if uptake rate of CVS or AC for patients with positive serum tests increase while the cost of serum screening decreases.

Conclusions: Although, in general, serum screening has been found to be more cost-effective than maternal age screening, this appears not to be the case in China. The reasons appear to be low uptake rate of the maternal serum strategy, low uptake rate of CVS or AC, and the high price of serum screening. Our findings are that health system factors concerning technology utilization are important determinants of the technology's efficiency.