Background: The importance of economic evaluation in decision making is growing with increasing budgetary pressures on health systems. Diverse economic evidence is available for a range of interventions across national contexts within Europe, but little attention has been given to identifying evidence gaps that, if filled, could contribute to more efficient allocation of resources. One objective of the Research Agenda for Health Economic Evaluation project is to determine the most important methodological evidence gaps for the ten highest burden conditions in the European Union (EU), and to suggest ways of filling these gaps.

Methods: The highest burden conditions in the EU by Disability Adjusted Life Years were determined using the Global Burden of Disease study. Clinical interventions were identified for each condition based on published guidelines, and economic evaluations indexed in MEDLINE were mapped to each intervention. A panel of public health and health economics experts discussed the evidence during a workshop and identified evidence gaps.

Results: The literature analysis contributed to identifying cross-cutting methodological and technical issues, which were considered by the expert panel to derive methodological research priorities.

Conclusions: The panel suggests a research agenda for health economics which incorporates the use of real-world evidence in the assessment of new and existing interventions; increased understanding of cost-effectiveness according to patient characteristics beyond the “-omics” approach to inform both investment and disinvestment decisions; methods for assessment of complex interventions; improved cross-talk between economic evaluations from health and other sectors; early health technology assessment; and standardized, transferable approaches to economic modeling.

Objectives: Our study addresses the important issue of estimating treatment costs from historical data. It is a problem frequently faced by health technology assessment analysts. We compared four approaches used to estimate current costs when good quality contemporary data are not available using liver transplantation as an example.

Methods: First, the total cost estimates extracted for patients from a cohort study, conducted in the 1990s, were inflated using a published inflation multiplier. Second, resource use estimates from the cohort study were extracted for hepatitis C patients and updated using current unit costs. Third, expert elicitation was carried out to identify changes in clinical practice over time and quantify current resource use. Fourth, routine data on resource use were obtained from National Health Service Blood and Transplant (NHSBT).

Results: The first two methods did not account for changes in clinical practice. Also the first was not specific to hepatitis patients. The use of experts confirmed significant changes in clinical practice. However, the quantification of resource use using experts is challenging as clinical specialists may not have a complete overview of clinical pathway. The NHSBT data are the most accurate reflection of transplantation and posttransplantation phase; however, data were not available for the whole pathway of care. The best estimate of total cost, combining NHSBT data and expert elicitation, is £121,211.

Discussion: Observational data from routine care are potentially the most reliable reflection of current resource use. Efforts should be made to make such data readily available and accessible to researchers. Expert elicitation provided reasonable estimates.

Objectives: The study aims to develop an understanding of the views of children and adolescents, parents, and professionals on upper limb prosthetic devices to develop and improve device design. Previous research has found that children are dissatisfied with prostheses but has relied heavily on parent proxy reports and quantitative measures (such as questionnaires) to explore their views.

Methods: Thirty-four participants (eight children aged 8–15 years with upper limb difference, nine parents, eight prosthetists, and nine occupational therapists) contributed to the development of new devices through the BRIDGE methodology of participatory design, using focus groups and interviews.

Results: The study identified areas for improving prostheses from the perspective of children and adolescents, developed prototypes based on these and gained feedback on the prototypes from the children and other stakeholders (parents and professionals) of paediatric upper limb prostheses. Future device development needs to focus on ease of use, versatility, appearance, and safety.

Conclusions: This study has demonstrated that children and adolescents can and should be involved as equal partners in the development of daily living equipment and that rapid prototyping (three-dimensional printing or additive manufacturing), used within a participatory design framework, can be a useful tool for facilitating this.

Objectives: Patient production losses occur when individuals’ capacities to work, whether paid or unpaid, are impaired by illness, treatment, disability, or death. There is controversy about whether and how to include patient production losses in economic evaluations in health care. Patient production losses have not previously been considered when evaluating medications for reimbursement under the U.K. National Health Service. Proposals for value-based assessment of health technologies in the United Kingdom created renewed interest in whether and how to include costs from a wider societal perspective, such as patient production losses, within economic evaluation of healthcare interventions.

Methods: A narrative review was undertaken of theoretical, ethical, and policy issues that might inform decisions that involve the normative question of whether or not to include patient production losses in economic evaluation.

Results: It seems difficult to reconcile the implications of including patient production losses with the objectives of a healthcare system dedicated to providing universal healthcare coverage without regard to patients’ ability to pay.

Conclusions: Tax payer funded healthcare systems may legitimately adopt maximands other than health gain, but these will be at the opportunity cost of less than maximum health gains.

Objectives: The health technology assessment (HTA) Core Model® is a tool for defining and standardizing the elements of HTA analyses within several domains for producing structured reports. This study explored the parallels between the Core Model and a national HTA report. Experiences from various European HTA agencies were also investigated to determine the Core Model's adaptability to national reports.

Methods: A comparison between a national report on Genetic Counseling, produced by the Cellule d'expertise médicale Luxembourg, and the Core Model was performed to identify parallels in terms of relevant and comparable assessment elements (AEs). Semi-structured interviews with five representatives from European HTA agencies were performed to assess their user experiences with the Core Model.

Results: The comparative study revealed that 50 percent of the total number (n = 144) of AEs in the Core Model were relevant for the national report. Of these 144 AEs from the Core Model, 34 (24 percent) were covered in the national report. Some AEs were covered only partly. The interviewees emphasized flexibility in using the Core Model and stated that the most important aspects to be evaluated include characteristics of the disease and technology, clinical effectiveness, economic aspects, and safety.

Conclusions: In the present study, the national report covered an acceptable number of AEs of the Core Model. These results need to be interpreted with caution because only one comparison was performed. The Core Model can be used in a flexible manner, applying only those elements that are relevant from the perspective of the technology assessment and specific country context.

Objectives: The reduction in cognitive decline depends on timely diagnosis. The aim of this systematic review was to analyze the current available information and communication technologies-based instruments for cognitive decline early screening and detection in terms of usability, validity, and reliability.

Methods: Electronic searches identified 1,785 articles of which thirty-four met the inclusion criteria and were grouped according to their main purpose into test batteries, measures of isolated tasks, behavioral measures, and diagnostic tools.

Results: Thirty one instruments were analyzed. Fifty-two percent were personal computer based, 26 percent tablet, 13 percent laptop, and 1 was mobile phone based. The most common input method was touchscreen (48 percent). The instruments were validated with a total of 4,307 participants: 2,146 were healthy older adults (M = 73.59; SD = 5.12), 1,104 had dementia (M = 74.65; SD = 3.98) and 1,057 mild cognitive impairment (M = 74.84; SD = 4.46). Only 6 percent were administered at home, 19 percent reported outcomes about usability, and 22 percent about understandability. The methodological quality of the studies was good, the weakest methodological area being usability. Most of the instruments obtained acceptable values of specificity and sensitivity.

Conclusions: It is necessary to create home delivered instruments and to include usability studies in their design. Involvement of people with cognitive decline in all phases of the development process is of great importance to obtain valuable and user-friendly products. It would be advisable for researchers to make an effort to provide cutoff points for their instruments.

Objectives: Disinvestment of existing healthcare technologies that deliver low or no health benefit for their cost can be used as a tool to improve access to effective technologies, while ensuring the long-term sustainability of healthcare systems. The objective of this research was to identify disinvestment initiatives in Latin American countries (LAC).

Methods: First, a systematic literature review (SLR) was conducted. In February 2015, MEDLINE, MEDLINE In-Process, EMBASE, The Cochrane Library, and LILACS were searched for relevant journal articles, including terms related to “disinvestment,” “reallocation,” “obsolete technologies,” and “Latin America.” Additionally, a manual search of documents from Latin American health technology assessment agencies was performed. Second, an online questionnaire was sent to experts in LAC to assess whether unpublished real-life disinvestment initiatives exist. Questionnaire results were collected in September 2015.

Results: From the SLR, 350 records were selected for screening following de-duplication and eleven articles fulfilled inclusion criteria. Only two of these reported information on initiatives potentially identifiable as disinvestment-investment activities in Brazil and Peru. Nine respondents completed the questionnaire, and four reported that disinvestment initiatives had been conducted in their respective organizations in Argentina, Brazil, and Mexico. This lack of agreement between the SLR and the questionnaire responses shows that disinvestment initiatives are ongoing, despite being under reported.

Conclusions: Many challenges need to be overcome for a disinvestment initiative to be successful, and sharing particular experiences with the international community would increase the chances of positive outcomes. The present study highlights the need for publication of such experiences in LAC.

Introduction: Optimizing radiotherapy with or without chemotherapy through advanced imaging and accelerated radiation schemes shows promising results in locally advanced non–small-cell lung cancer (NSCLC). This study compared the cost-effectiveness of positron emission tomography-computed tomography based isotoxic accelerated sequential chemo-radiation (SRT2) and concurrent chemo-radiation with daily low-dose cisplatin (CRT2) with standard sequential (SRT1) and concurrent chemo-radiation (CRT1).

Methods: We used an externally validated mathematical model to simulate the four treatment strategies. The model was built using data from 200 NSCLC patients treated with curative sequential chemo-radiation. For concurrent strategies, data from a meta-analysis and a single study were included in the model. Costs, utilities, and resource use estimates were obtained from literature. Primary outcomes were the incremental cost-effectiveness and cost-utility ratio (ICUR) of each strategy. Scenario analyses were carried out to investigate the impact of uncertainty.

Results: Total undiscounted costs and quality-adjusted life-years (QALYs) for SRT1, CRT1, SRT2, and CRT2 were EUR 17,288, EUR 18,756, EUR 19,072, EUR 17,360 and QALYs 1.10, 1.15, 1.40, and 1.40, respectively. Compared with SRT1, the ICURs were EUR 38,024/QALY for CRT1, EUR 6,249/QALY for SRT2, and EUR 346/QALY for CRT2. CRT2 was highly cost-effective compared with SRT1. Moreover, CRT2 was more effective and less costly than CRT1 and SRT2. Therefore, these strategies were dominated by CRT2.

Conclusion: Optimized sequential and concurrent chemo-radiation strategies are more effective and cost-effective than the current conventional sequential and concurrent strategies. Concurrent chemo-radiation with a daily low dose cisplatin regimen is the most cost-effective treatment option for locally advanced inoperable NSCLC patients.

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: In recent years, numerous frameworks have been developed to enhance the legitimacy of health technology assessment processes. Despite efforts to implement these “legitimacy frameworks,” medicines funding decisions can still be perceived as lacking in legitimacy. We, therefore, sought to examine stakeholder views on factors that they think should be considered when making decisions about the funding of high-cost breast cancer therapies, focusing on those that are not included in current frameworks and processes.

Methods: We analyzed published discourse on the funding of high-cost breast-cancer therapies. Relevant materials were identified by searching the databases Google, Google Scholar, and Factiva in August 2014 and July 2016 and these were analyzed thematically.

Results: We analyzed fifty published materials and found that stakeholders, for the most part, want to be able to access medicines more quickly and at the same time as other patients and for decision makers to be more flexible with regards to evidence requirements and to use a wider range of criteria when evaluating therapies. Many also advocated for existing process to be accelerated or bypassed to improve access to therapies.

Conclusions: Our results illustrate that a stakeholder-derived conceptualization of legitimacy emphasizes principles of accelerated access and is not fully accounted for by existing frameworks and processes aimed at promoting legitimacy. However, further research examining the ethical, political, and clinical implications of the stakeholder claims raised here is needed before firm policy recommendations can be made.

Objectives: Medical devices are ubiquitous in modern medical care. However, little is known about the epidemiology of medical devices in the healthcare marketplace, including the rate at which medical devices are subject to recalls or other advisories. We sought to study the epidemiology of medical devices in Canada, focusing on device recalls. In Canada, a recall may signify a variety of events, ranging from relatively minor field safety notifications, to removal of a product from the marketplace.

Methods: We used data from Health Canada to study medical device recalls in Canada from 2005 to 2015. We analyzed the risks of medical device recalls according to the risk class of the device (I lowest; IV highest) and the hazard priority of the recall (Type I highest potential harm; Type III lowest potential harm).

Results: During a 10-year period, there were 7,226 medical device recalls. Most recalls were for intermediate risk class (Class II, 40.1 percent; Class III, 38.7 percent) medical devices. Among recalled devices, 5.0 percent were judged to have a reasonable probability of serious adverse health consequences or death (Type I recall Hazard Priority classification). While the number of medical devices marketed in Canada is not known, over a similar 10-year period, 24,849 new Class II, II, and IV medical device licenses were issued by Health Canada.

Conclusions: Several hundred medical device recalls occur in Canada each year. Further research is needed to characterize the nature of medical device recalls, and to explore how consumers use information about recalls.

Objectives: Although there is increased awareness of patient and public involvement (PPI) among health technology assessment (HTA) organizations, evaluations of PPI initiatives are relatively scarce. Our objective as members of Health Technology Assessment International's (HTAi's) Patient and Citizen Involvement Group (PCIG) was to advance understanding of the range of evaluation strategies adopted by HTA organizations and their potential usefulness.

Methods: In March 2016, a survey was sent to fifty-four HTA organizations through the International Network of Agencies for Health Technology Assessment (INAHTA) and contacts of members of HTAi's PCIG. Respondents were asked about their organizational structure; how patients and members of the public are involved; whether and how PPI initiatives have been evaluated, and, if so, which facilitators and challenges to evaluation were found and how results were used and disseminated.

Results: Fifteen (n = 15) programs from twelve countries responded (response rate 27.8 percent) that involved patients (14/15) and members of the public (10/15) in HTA activities. Seven programs evaluated their PPI activities, including participant satisfaction (5/7), process (5/7) and impact evaluations (4/7). Evaluation results were used to improve PPI activities, identify education and training needs, and direct strategic priorities. Facilitators and challenges revolved around the need for stakeholder buy-in, sufficient resources, senior leadership, and including patients in evaluations.

Conclusions: A small but diverse set of HTA organizations evaluate their PPI activities using a range of strategies that reflect the range of rationales and approaches to PPI in HTA. It will be important for HTA organizations to draw on evaluation theories and methods.