Objectives: The AIFA (Agenzia Italiana del Farmaco—Italian Medicines Agency) Monitoring Registries track the eligibility of patients and the complete flow of treatments, guaranteeing appropriateness in use of pharmaceutical products, according to approved indications.

Methods: This study describes the Italian pharmaceutical context and the aims and functioning of AIFA Monitoring Registries, focusing on the applications to the Managed Entry Agreements (MEAs) and HTA approaches.

Results: The AIFA Monitoring Registries System has been operational in Italy since 2005. In 2012, the system became part of the NHS Information Technology system, aiming at enhancing appropriate use of pharmaceuticals and efficiency of the administrative activity. Currently, seventy-six medicines are monitored through the system, corresponding to fifty-eight therapeutic indications; individual treatments recorded are more than 515,000, for a population of approximately 505,000 patients. For each monitored product, patients eligible for treatment are registered in the specific therapeutic indication dynamic monitoring database to collect epidemiologic and clinical data, including data on the safety profile, and ex-post information missing at first evaluation stage.

Conclusions: AIFA Monitoring Registries allow the evaluation of the pharmaceuticals’ performance in clinical practice and may promote innovation and quicker access to medicines at affordable prices, for the benefit of patients.

Objectives: The aim of this study was to assess the cost-effectiveness of screening for open-angle glaucoma (OAG) in the United Kingdom, given that OAG is an important cause of blindness worldwide.

Methods: A Markov model was developed to estimate lifetime costs and benefits of a cohort of patients facing, alternatively, screening or current opportunistic case finding strategies. Strategies, varying in how screening would be organized (e.g., invitation for assessment by a glaucoma-trained optometrist [GO] or for simple test assessment by a technician) were developed, and allowed for the progression of OAG and treatment effects. Data inputs were obtained from systematic reviews. Deterministic and probabilistic sensitivity analyses were performed.

Results: Screening was more likely to be cost-effective as prevalence increased, for 40 year olds compared with 60 or 75 year olds, when the re-screening interval was greater (10 years), and for the technician strategy compared with the GO strategy. For each age cohort and at prevalence levels of ≤1 percent, the likelihood that either screening strategy would be more cost-effective than current practice was small. For those 40 years of age, “technician screening” compared with current practice has an incremental cost-effectiveness ratio (ICER) that society might be willing to pay when prevalence is 6 percent to 10 percent and at over 10 percent for 60 year olds. In the United Kingdom, the age specific prevalence of OAG is much lower. Screening by GO, at any age or prevalence level, was not associated with an ICER < £30,000.

Conclusions: Population screening for OAG is unlikely to be cost-effective but could be for specific subgroups at higher risk.

This article describes criteria used by several organizations to select technologies for assessment and the factors that are common to most lists of criteria. It presents a framework or model (called TAPSS) that relates the most important factors. The model can be used to estimate the expected impact of an assessment on health and economic outcomes for a population. It incorporates information on such factors as the health and economic importance of the disease addressed by the technology, the expected impact of the technology on health and economic outcomes, variations in the technology's current use, and factors affecting the production and reception of an assessment. The article includes an example of an application of the method—screening for maple syrup urine disease.

Objectives: The evaluation of telemedicine from the patient's point of view has focused on the patient pathway and patient satisfaction. However, research in this field does not provide us with the means to assess a patient's perception of the procedure if their reasoning ability is impaired. In this study, we use direct observation of a patient's behavior and mood to assess their perception of an oral teleconsultation procedure.

Methods: This study has been conducted in the context of a pilot project using an asynchronous teleconsultation to improve access to dental care for the dependent elderly, disabled people, and prisoners, some of whom may be cognitively impaired. We use a direct observation form consisting of five behavioral variables and eight affect variables to reflect the patient's experience of the oral teleconsultation procedure.

Results: A total of 135 patients were evaluated; 10 refused the procedure. Psychotic patients (n = 33) had a somewhat negative experience during the oral teleconsultation procedure. Patients who were not psychotic had a positive experience; this decreased as we moved from the autonomous to the semi-autonomous and then to the dependent sub-group. Some gender differences were also noted.

Conclusions: Improving evidence on evaluating the acceptance of the cognitively impaired is required to improve the technology development process so that it can be translated into an improved patient experience and adherence. Although the study was specifically focused on teledentistry, the approach described in this study could be adapted to other forms of teleconsultation.

Objectives: In the next decades, the number of stroke patients is expected to increase. Furthermore, organizational changes, such as stroke services, are expected to be implemented on a large scale. The purpose of this study is to estimate the future healthcare costs by taking into account the expected increase of stroke patients and a nationwide implementation of stroke services.

Methods: By means of a dynamic multistate life table, the total number of stroke patients can be projected. The model calculates the annual number of patients by age and gender. The total healthcare costs are calculated by multiplying the average healthcare costs specified by age, gender, and healthcare sector with the total number of stroke patients specified by age and gender.

Results: In the year 2000, the healthcare costs for stroke amounted to €1.62 billion. This amount is approximately 4.4 percent of the total national healthcare budget. Projections of the total costs of stroke based on current practice result in an increase of 28 percent (€2.08 billion) in the year 2020. A nationwide implementation of stroke services in 2020 would result in a substantial reduction of the costs of stroke (€1.81 billion: 13 percent cost reduction) compared with the regular care scenario.

Conclusions: A nationwide implementation of stroke services is a strong policy tool for cost containment of health care in an aging population like that in the Netherlands. Policy makers should optimize the organization of stroke care.

The time invested by patients and informal caregivers in treatment and rehabilitation should be valued in a cost-effectiveness analysis (CEA). The Washington Panel gives recommendations on incorporation of time costs that may misrepresent true societal costs. This article provides alternative recommendations for incorporating costs of time in CEA. Following an opportunity costs approach, time is separated into three parts, each with its own valuation methods: time spent on paid work, unpaid work, and leisure.

Objective: To develop and test a decision-support tool for prioritizing new competing Health Technologies (HTs) after their assessment using the mini-HTA approach.

Methods: A two layer value/risk tool was developed based on the mini-HTA. The first layer included 12 mini-HTA variables classified in two dimensions, namely value (safety, clinical benefit, patient impact, cost-effectiveness, quality of the evidence, innovativeness) and risk (staff, space and process of care impacts, incremental costs, net cost, investment effort). Weights given to these variables were obtained from a survey among decision-makers (at National/Regional level and hospital settings). A second layer included results from mini-HTA (scored as higher, equal or lower), which compares the performance of the new HT (in terms of the abovementioned 12 variables) with the available comparator. An algorithm combining the first (weights) and second (scores) layers was developed to obtain an overall score for each HT, which was then plotted in a value/risk matrix. The tool was tested using results from the mini-HTAs for three new HTs (Surgical Robot, Platelet Rich Plasma, Deep Brain Stimulation).

Results: No significant differences among decision-makers were observed as regards the weights given to the 12 variables, therefore, the median aggregate weights from decision-makers were introduced in the first layer. The dot plot resulting from the mini-HTA presented good power to visually discriminate between the assessed HTs.

Conclusion: The decision-support tool developed here makes possible a robust and straightforward comparison of different competing HTs. This facilitates hospital decision-makers deliberations on the prioritization of competing investments under fixed budgets.

Objectives: Assessment of ethical aspects of a technology is an important component of health technology assessment (HTA). Nevertheless, how the implementation of ethical assessment in HTA is to be organized and adapted to specific regulatory and organizational settings remains unclear. The objective of this study is to present a framework for systematic identification of ethical aspects of health technologies. Furthermore, the process of developing and adapting the framework to a specific setting is described.

Methods: The framework was developed based on an inventory of existing approaches to identification and assessment of ethical aspects in HTA. In addition, the framework was adapted to the Swedish legal and organizational healthcare context, to the role of the HTA agency and to the use of non-ethicists. The framework was reviewed by a group of ethicists working in the field as well as by a wider set of interested parties including industry, interest groups, and other potential users.

Results: The framework consists of twelve items with sub-questions, short explanations, and a concluding overall summary. The items are organized into four different themes: the effects of the intervention on health, its compatibility with ethical norms, structural factors with ethical implications, and long term ethical consequences of using the intervention.

Conclusions: In this study, a framework for identifying ethical aspects of health technologies is proposed. The general considerations and methodological approach to this venture will hopefully inspire and present important insights to organizations in other national contexts interested in making similar adaptations.

To determine the cost-effectiveness of colorectal cancer screening strategies in Japan and to determine the influence of long-term compliance with screening programs on the selection of strategies, the natural history of a simulated cohort of 40-year-old Japanese of both genders was modeled with and without colorectal cancer screening until age 75 years. Survival, number of complications, and direct medical costs were compared among several combinations of screening examinations. In addition, the age of initiating screening was varied, as was the long-term compliance rate. Strategies using immunoiogical fecal occult blood test were found to be the most cost-effective. Immunological fecal occult blood test followed by colonoscopy, if positive, would save 24.05 (5.88 discounted) days of life and cost 28,420 yen (US $210) per screened person, thus offering a cost-effectiveness ratio of 1.765 million yen (US $13,100) per year of life saved. If long-term compliance is 100%, initiating screening at age 40 years offers more years of life saved and a low incremental cost of screening. However, if more likely dropout rates are considered, initiation at age 40 years is dominated by later initiation of screening.

Objectives: The aim of this study was to obtain information from members of the International Network of Agencies for Health Technology Assessment (INAHTA) on their involvement of consumers (patients, carers, and related organizations) in their programs.

Methods: A questionnaire for a survey was developed and sent to member agencies in October 2005.

Results: Of the thirty-seven agencies that provided responses, 57 percent involve consumers in some aspects of their HTA programs and 83 percent intend to involve consumers in the future. Summaries of HTA reports that are intended to be easily understood by consumers are prepared by 49 percent of the agencies, and 36 percent involve consumers in dissemination of HTA material.

Conclusions: Most INAHTA members involve consumers in some aspects of their programs, although not always routinely. Involvement seems likely to increase in the future.

Objective: The U.K. NHS Economic Evaluation Database (EED) project is commissioned to identify papers on economic evaluations of health technologies and to disseminate their findings to NHS decision makers by means of structured abstracts that are available through a public database and the Cochrane Library. This paper discusses current issues relating to the economic aspects of producing NHS EED abstracts.

Methods: A review of NHS EED was undertaken between 1994 and 1999 to determine the methodologies adopted and issues that influence the usefulness of economic evaluations. Methods adopted to improve the quality of NHS EED abstracts are also reported.

Results: Eighty-five percent of NHS EED abstracts are cost-effectiveness analyses (CEAs), 9.3% are cost-utility analyses (CUAs), and only 1.4% are cost-benefit analyses (CBAs). Of the total abstracts, 65.9% are based on single studies, 19.5% on reviews, 3.9% on estimates of effectiveness, and 10.7% on combinations of these sources. Models are utilized in 16.7% of CEAs, 60.2% of CUAs, and 20% of CBAs. Analyses of CBA studies reveal a degree of misuse of well-established definitions. NHS EED internal control mechanisms are reported that provide a means of ensuring that abstracts are based on sound academic principles.

Conclusions: Most economic evaluations are conducted by means of CEA, followed by CUA, while CBA accounts for an extreme minority of cases. Single studies form the principal source of effectiveness data, although models are widely used, principally in CUA. The structure of NHS EED abstracts provides decision makers with the principal results and an interpretation of the relative strengths and weaknesses of economic evaluations.

We discuss some of the issues that have arisen during the development and introduction into practice of information materials for health professionals and patients that aim to promote clinical effectiveness and informed patient participation in clinical decision making.

Background: The University College of Southeast Norway has an on-going project to develop a smart house welfare system to allow older adults and people with disabilities to remain in their homes for as long as they wish in safe, dignified, living conditions.

Objectives: This article reviews reported ethical challenges to implementing smart houses for older adults.

Methods: A systematic literature review identified twenty-four articles in English, French, Spanish, and Norwegian, which were analyzed and synthesized using Hofmann's question list to investigate the reported ethical challenges.

Results: Smart houses offer a promising way to improve access to home care for older adults and people with disabilities. However, important ethical challenges arise when implementing smart houses, including cost-effectiveness, privacy, autonomy, informed consent, dignity, safety, and trust.

Conclusions: The identified ethical challenges are important to consider when developing smart house systems. Due to the limitations of smart house technology, designers and users should be mindful that smart houses can achieve a safer and more dignified life-style but cannot solve all the challenges related to ageing, disabilities, and disease. At some point, smart houses can no longer help persons as they develop needs that smart houses cannot meet.

Objectives: Uncertainty is pervasive in decision making on new health technologies; therefore, some countries have put systems in place to support decision makers with timely information. An important, but as yet undocumented, determinant of the potential value for decision making of these so-called horizon scanning systems (HSSs) is how the most significant health technologies are selected.

Methods: All thirteen member organizations of EuroScan, a collaborative network for HSSs, were surveyed and interviewed on how they prioritize technologies for assessment.

Results: The majority of HSSs directly serves a customer. Some customers actively request early assessments of new health technologies, thereby diminishing the need for priority setting for the HSSs. All systems express a concern to miss an important technology and/or to select an unimportant technology. Almost all HSSs use explicit selection criteria, but these criteria hardly ever are operationalized. The number of criteria used varies, but costs and health benefit of the technology are always taken into account. The process of reaching a final decision is implicit, undocumented in all but one system, and is based on agreement by consensus.

Conclusions: The process of making the final decision on which technologies to assess can be improved by applying existing criteria more consistently and transparently. Current practice does not safeguard against missing an important technology. This finding is probably most important to act upon for systems with customers that do not actively request assessment of specific technologies.

Objectives: The aim of this study was to get insight in what criteria as presented in Health technology assessment (HTA) studies are important for decision makers in healthcare priority setting.

Methods: We performed a discrete choice experiment among Dutch healthcare professionals (policy makers, HTA experts, advanced HTA students). In twenty-seven choice sets, we asked respondents to elect reimbursement of one of two different healthcare interventions, which represented unlabeled, curative treatments. Both treatments were incrementally compared with usual care. The results of the interventions were normal outputs of HTA studies with a societal perspective. Results were analyzed using a multinomial logistic regression model. Upon completion of the questionnaire, we discussed the exercise with policy makers.

Results: Severity of disease, costs per quality-adjusted life-year gained, individual health gain, and the budget impact were the most decisive decision criteria. A program targeting more severe diseases increased the probability of reimbursement dramatically. Uncertainty related to cost-effectiveness was also important. Respondents preferred health gains that include quality of life improvements over extension of life without improved quality of life. Savings in productivity costs were not crucial in decision making, although these are to be included in Dutch reimbursement dossiers for new drugs. Regarding subgroups, we found that policy makers attached relatively more weight to disease severity than others but less to uncertainty.

Conclusions: Dutch policy makers and other healthcare professionals seem to have reasonably well articulated preferences: six of seven attributes were significant. Disease severity, budget impact, and cost-effectiveness were very important. The results are comparable to international studies, but reveal a larger set of important decision criteria.

Objectives: Schizophrenia imposes a great burden on society, and while evaluation should play an important role in informing society's efforts to alleviate these burdens, it is unclear what “endpoints” should be chosen as the objective of such analyses. The objectives of the study were to elicit endpoints directly from patients with schizophrenia, to ascertain whether patients are sufficiently cognoscente to express what endpoints are and are not important to them and to rank the relevant endpoints.

Methods: We applied principles of patient-centered health technology assessment to identify and value endpoints from the patient's perspective. Focus groups were conducted to elicit endpoints, using interpretive phenomalogical analysis (IPA) to guide the collection, analysis and interpretation of data. Patient interviews were subsequently used to elicit patient preference over endpoints. Respondents were presented with cards outlining the endpoints and asked to remove irrelevant cards. They where then asked to identify and rank their five most relevant endpoints in order of importance. Interviews were recorded for the purposed of triangulation, and data was analyzed using descriptive statistics. Patients were recruited from five geographically diverse cities in Germany. Eligibility required a diagnosis of schizophrenia by a physician and treatment with an antipsychotic medication for at least one year. Respondents were excluded if they were experiencing an acute episode.

Results: Thirteen endpoints emerged as important from the focus groups spanning side-effects, functional status, processes of care and clinical outcomes. Respondents could clearly identify relevant and irrelevant endpoints, and rank which factors were important to them. Triangulation between field notes of the ranking exercise and recordings confirmed that rankings were not arbitrary, but justified from the respondents' point of view.

Conclusions: Patients with schizophrenia can express preferences over endpoints. Our results show that qualitative methods such as IPA can be used to identify factors, but ranking exercises provide a more robust method for ranking the importance of endpoints. Future research involving patients with schizophrenia ranking outcomes is needed to identify variations across patients and methods such as conjoint analysis could prove beneficial in identifying acceptable tradeoffs across endpoints.

Objectives: The aim of this study was to estimate the cost-effectiveness of rituximab in patients not responding adequately to the first tumor necrosis factor (TNF) inhibitor using a model constructed to predict resource consumption and health outcomes in a population-based registry of biological treatments in Southern Sweden (SSATG).

Methods: The model was developed as a discrete event simulation model, using SSATG data for the years 1999–2007. The data set included 1,903 patients with complete data on treatments (up to three treatment lines), functional capacity (HAQ), disease activity (DAS28), and utility (EQ-5D). Resource consumption was based on a regular population-based survey of patients in Southern Sweden. Rituximab was incorporated as second line treatment, using effectiveness data for the active group (N = 311) in a clinical trial comparing rituximab to placebo (REFLEX). It is thus compared to the mix of second line biologics used in SSATG. The analysis starts after failure of the first TNF inhibitor. Results are reported as costs (€2008) per quality-adjusted life-year (QALY; both discounted 3 percent), for the societal perspective in Sweden.

Results: Total costs in the rituximab strategy are estimated at €401,100 compared with €403,000 in the TNF-inhibitor arm. Total QALYs are 5.98 and 5.78, respectively. The findings were found to be robust in extensive sensitivity analysis.

Conclusions: In our model, a strategy where rituximab is used as second line treatment after failure of the first TNF inhibitor provides a small saving (essentially due to the lower price of rituximab) and a QALY gain (due to better effect than the mix of second line TNF inhibitors).

Objectives: Evidence exists that telehealth interventions (e.g., telemonitoring, telediagnostics, telephone care) in disease management for chronic heart failure patients can improve medical outcomes, and we aim to give an overview of the cost-effectiveness of these interventions.

Methods: Based on the literature search on “heart failure” in combination with “cost” and “telehealth” we selected 301 titles and abstracts. Titles and abstracts were screened for a set of inclusion criteria: telehealth intervention, heart failure as the main disease, economic analysis present and a primary study performed. In the end, thirty-two studies were included for full reading, data extraction, and critical appraisal of the economic evaluation.

Results: Most studies did not present a comprehensive economic evaluation, consisting of the comparison of both costs and effects between telehealth intervention and a comparator. Data on telehealth investment costs were lacking in many studies. The few studies that assessed costs and consequences comprehensively showed that telehealth interventions are cost saving with slight improvement in effectiveness, or comparably effective with similar cost to usual care. However, the methodological quality of the studies was in general considered to be low.

Conclusions: The cost-effectiveness of telehealth in chronic heart failure is hardly ascertained in peer reviewed literature, the quality of evidence is poor and there was a difficulty in capturing all of the consequences/effects of telehealth intervention. We believe that without full economic analyses the cost-effectiveness of telehealth interventions in chronic heart failure remains unknown.

Background: Comprehensive health technology assessments (HTAs) include thorough reflections on ethical issues associated with health technologies, their use, and value-based decisions in the assessment process. As methods of information retrieval for effectiveness assessments are not applicable to information retrieval on ethical issues, a specific methodological approach is necessary.

Objectives: In the absence of existing adapted methods, our objective was to develop a methodological approach for the systematic retrieval of information on ethical issues related to health technologies.

Method and Results: A literature search was conducted to verify the non-existence of published comprehensive methodological approaches for the information retrieval on ethical issues for HTAs, and resulted in no hits. We, therefore, developed a step-by-step workflow following the workflow of information retrieval for effectiveness assessments: Step 1: Translation of the search question using the PICO scheme and additional components. Step 2: Concept building by modeling and linking search components. Step 3: Identification of synonyms in all relevant languages. Step 4: Selection of relevant information sources. Step 5: Design of search strategies for bibliographic databases. Step 6: Execution of search strategies and information seeking, including hand-searching. Step 7: Saving of retrieval results and standardized reporting of the process and results. Step 8: Final quality check and calculation of precision and recall.

Conclusions: Systematic searching for information on ethical issues related to health technologies can be performed following the common retrieval workflow for effectiveness assessments, but should be performed separately applying adapted procedures and search terms on ethical issues relevant to the research question.