Objectives: Equity is one of the founding principles in most healthcare systems. Financial constraints entail an increased risk of exacerbating inequities and a greater need for evidence-based decisions. It is, therefore, both important and timely to enquire how equity can be addressed in health technology assessment (HTA) practice. We aimed to explore related practices from a broad range of HTA agencies, identify exemplary approaches and common concerns, and offer insights for future considerations.

Methods: HTA agencies for which both methodological guides and HTA reports were publicly available were selected from an initial comprehensive pool. Information was extracted on issues ranging from a general commitment to fairness to specific measures targeting both methodological and process-related elements.

Results: Methodological documents and ninety-eight reports from nineteen agencies were analyzed. Our findings indicate that equity was not a standard consideration in HTA report production. The nature of specific approaches and the amount of resources invested into including an equity perspective varied considerably. Specific measures (e.g., appropriate information sources, analytical tools, and schemes) were mentioned by almost half of the agencies analyzed. Albeit sporadic, both horizontal and vertical equity considerations were identified in included HTA reports.

Conclusions: While varying legal contexts and institutional principles can lead to different interpretations of equity at the decision point, a combination of methodological and process-related practices could contribute to more equity-sensitive evaluations, especially in conjunction with enhanced dissemination of existing methodological tools. Networking initiatives on behalf of existing collaborating platforms could play an important role in this direction.

Objectives: The suitability of general HTA methodology for medical devices is gaining interest as a topic of scientific discourse. Given the broad range of medical devices, there might be differences between groups of devices that impact both the necessity and the methods of their assessment. Our aim is to develop a taxonomy that provides researchers and policy makers with an orientation tool on how to approach the assessment of different types of medical devices.

Methods: Several classifications for medical devices based on varying rationales for different regulatory and reporting purposes were analyzed in detail to develop a comprehensive taxonomic model.

Results: The taxonomy is based on relevant aspects of existing classification schemes incorporating elements of risk and functionality. Its 9 × 6 matrix distinguishes between the diagnostic or therapeutic nature of devices and considers whether the medical device is directly used by patients, constitutes part of a specific procedure, or can be used for a variety of procedures. We considered the relevance of different device categories in regard to HTA to be considerably variable, ranging from high to low.

Conclusions: Existing medical device classifications cannot be used for HTA as they are based on different underlying logics. The developed taxonomy combines different device classification schemes used for different purposes. It aims at providing decision makers with a tool enabling them to consider device characteristics in detail across more than one dimension. The placement of device groups in the matrix can provide decision support on the necessity of conducting a full HTA.

Objectives: Many of the currently used health technologies have never been systematically assessed or are misused, overused or superseded. Therefore, they may be ineffective. Active identification of ineffectiveness in health care is gaining importance to facilitate best care for patients and optimal use of limited resources. The present research analyzed processes and experiences of programs for identifying ineffective health technologies. The goal of this study was to elucidate factors that facilitate implementation.

Methods: Based on an overview article, a systematic literature search and unsystematic hand-search were conducted. Further information was gained from international experts.

Results: Seven programs were identified that include identification, prioritization and assessment of ineffective health technologies and dissemination of recommendations. The programs are quite similar regarding their goals, target groups and criteria for identification and prioritization. Outputs, mainly HTA reports or lists, are mostly disseminated by means of the internet. Top–down and bottom–up programs both have benefits in terms of implementation of recommendations, either as binding guidelines and decisions or as nonbinding information for physicians and other stakeholders. Crucial facilitators of implementation are political will, transparent processes and broad stakeholder involvement focusing on physicians.

Conclusions: All programs can improve the quality of health care and enable cost reduction in supportive surrounding conditions. Physicians and patients must be continuously involved in the process of evaluating health technologies. Additionally, decision makers must support programs and translate recommendations into concrete actions.

Objectives: The headroom approach to medical device development relies on the estimation of a value-based price ceiling at different stages of the development cycle. Such price-ceilings delineate the commercial opportunities for new products in many healthcare systems. We apply a simple model to obtain critical business information as the product proceeds along a development pathway, and indicate some future directions for the development of the approach.

Methods: Health economic modelling in the supply-side development cycle for new products.

Results: The headroom can be used: initially as a ‘reality check’ on the viability of the device in the healthcare market; to support product development decisions using a real options approach; and to contribute to a pricing policy which respects uncertainties in the reimbursement outlook.

Conclusions: The headroom provides a unifying thread for business decisions along the development cycle for a new product. Over the course of the cycle attitudes to uncertainty will evolve, based on the timing and manner in which new information accrues. Within this framework the developmental value of new information can justify the costs of clinical trials and other evidence-gathering activities. Headroom can function as a simple shared tool to parties in commercial negotiations around individual products or groups of products. The development of similar approaches in other contexts holds promise for more rational planning of service provision.

Objectives: Model for ASsessment of Telemedicine Applications (MAST) is a health technology assessment (HTA) inspired framework for assessing the effectiveness and contribution to quality of telemedicine applications based on rigorous, scientific data. This study reports from a study of how it was used and perceived in twenty-one pilots of the European project RENEWING HEALTH (RH). The objectives of RH were to implement large-scale, real-life test beds for the validation and subsequent evaluation of innovative patient-centered telemedicine services. The study is a contribution to the appraisal of HTA methods.

Methods: A questionnaire was administered for project leaders of the pilots. It included questions about use and usefulness of MAST for (i) preceding considerations, (ii) evaluation of outcomes within seven domains, and (iii) considerations of transferability. Free text spaces allowed for proposals of improvement. The responses covered all pilots. A quantitative summary of use and a qualitative analysis of usefulness were performed.

Results: MAST was used and considered useful for pilot evaluations. Challenges included problems to scientifically determine alternative service options and outcome within the seven domains. Proposals for improvement included process studies and adding domains of technological usability, responsible innovation, health literacy, behavior change, caregiver perspectives and motivational issues of professionals.

Conclusions: MAST was used according to its structure. Its usefulness in patient centered pilots can be improved by adding new stakeholder groups. Interdependencies between scientific rigor, resources and timeliness should be addressed. Operational options for improvements include process studies, literature reviews and sequential mini-HTAs for identification of areas for more elaborate investigations.

Background: “Mapping” onto generic preference-based outcome measures is increasingly being used as a means of generating health utilities for use within health economic evaluations. Despite publication of technical guides for the conduct of mapping research, guidance for the reporting of mapping studies is currently lacking. The MAPS (MApping onto Preference-based measures reporting Standards) statement is a new checklist, which aims to promote complete and transparent reporting of mapping studies.

Methods: In the absence of previously published reporting checklists or reporting guidance documents, a de novo list of reporting items was created by a working group comprised of six health economists and one Delphi methodologist. A two-round, modified Delphi survey with representatives from academia, consultancy, health technology assessment agencies, and the biomedical journal editorial community was used to identify a list of essential reporting items from this larger list.

Results: From the initial de novo list of twenty-nine candidate items, a set of twenty-three essential reporting items was developed. The items are presented numerically and categorized within six sections, namely: (i) title and abstract, (ii) introduction, (iii) methods, (iv) results, (v) discussion, and (vi) other. The MAPS statement is best applied in conjunction with the accompanying MAPS explanation and elaboration document.

Conclusions: It is anticipated that the MAPS statement will improve the clarity, transparency. and completeness of reporting of mapping studies. To facilitate dissemination and uptake, the MAPS statement is being co-published by seven health economics and quality of life journals, and broader endorsement is encouraged. The MAPS working group plans to assess the need for an update of the reporting checklist in five years’ time.

Objectives: The objective of this study was to explore barriers and facilitators influencing the integration of ethical considerations in health technology assessment (HTA).

Methods: The study consisted of two complementary approaches: (a) a systematic review of the literature; and (b) an eighteen-item online survey that was distributed to fifty-six HTA agencies affiliated with the International Network of Agencies for Health Technology Assessment.

Results: The review identified twenty-six relevant articles. The most often cited barriers in the literature were: scarcity, heterogeneity and complexity of ethical analysis methods; challenges in translating ethical analysis results into knowledge that is useful for decision makers; and lack of organizational support in terms of required expertise, time and financial resources. The most frequently cited facilitators included: usage of value-based appraisal methods, stakeholder and public engagement, enhancement of practice guidelines, ethical expertise, and educational interventions.

Representatives of twenty-six (46.5 percent) agencies from nineteen countries completed the survey. A median of 10 percent (interquartile range, 5 percent to 50 percent) of the HTA products produced by the agencies was reported to include an assessment of ethical aspects. The most commonly perceived barriers were: limited ethical knowledge and expertise, insufficient time and resources, and difficulties in finding ethical evidence or using ethical guidelines. Educational interventions, demand by policy makers, and involvement of ethicists in HTA were the most commonly perceived facilitators.

Conclusions: Our results emphasize the importance of simplification of ethics methodology and development of good practice guidelines in HTA, as well as capacity building for engaging HTA practitioners in ethical analyses.

Objectives: The objective of this study was to compare evidence requirements for health technology assessment of pharmaceuticals by national agencies across Europe responsible for reimbursement decisions focusing specifically on relative effectiveness assessment.

Methods: Evidence requirements from thirty-three European countries were requested and twenty-nine national agencies provided documents to review. Data were extracted from national documents (manufacturer's submission templates and associated guidance) into a purpose-made framework with categories covering information about the health condition, the technology, clinical effectiveness and safety.

Results: The level of detail in the required evidence varies considerably across countries. Some countries include specific questions while others request information under general headings. Some countries include all information in a single document, which may or may not include guidance on how to complete the template. Others have specific guidance documents or methods and process manuals that help with the completion of the submission templates. Despite differences in quantity and detail, the content of the evidence requirements is broadly similar. All countries ask for information on the health technology, target disease, and clinical effectiveness and safety. However, one country only requests clinical effectiveness information as part of cost-effectiveness analyses. We found twenty-six evidence requirements for which generic answers may apply across borders and nineteen in which countries requested nationally specific information.

Conclusions: This work suggests that it would be possible to put together a minimum set of evidence requirements for HTA to support reimbursement decisions across Europe which could facilitate collaboration between jurisdictions.

Objectives: Our objective was to gather perspectives from payers on how comparative effectiveness research (CER) in the United States and relative effectiveness (RE) research in Europe will impact evidentiary standards for access decisions of new drugs by 2020.

Methods: We conducted semi-structured interviews with fourteen senior officials representing public and private payers, health technology assessment groups, and pricing and reimbursement bodies in the United States and Europe. An online survey assessed current use of CER/RE evidence and potential trends that might influence its use for decision making by 2020. A semi-structured interview elicited payers' definitions of CER/RE and was structured around four hypothetical cases resembling drugs expected to be more common or poised to create policy challenges by 2020. Topics included acceptance of study designs and analytic methods associated with CER/RE. A systematic content review was done to extract relevant information.

Results: According to key informants, randomization will remain an essential component for assessing comparative or relative effectiveness. They anticipate greater use of policy levers such as conditional reimbursement or prior authorization to manage diffusion of new drugs. Case studies provided important insights into situations when certain types of CER evidence may be acceptable (e.g., observational data when differences between drugs are largely convenience).

Conclusions: Industry perceptions that CER/RE will change payers' evidentiary requirements in the future are consistent with our findings. Growing investment in payers' own data and increased reliance on policy tools to control diffusion of new drugs may also influence the type of evidence industry will be required to produce by 2020.

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.

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: 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.

Background: Health-related quality of life (HRQoL) is an important endpoint of many healthcare interventions. This study develops guidance on how to select appropriate HRQoL measures for inclusion in a clinical trial, given the purposes of the HRQoL measurement.

Methods: The guidance is based on a systematic literature review, discussions with members of the European Network for Health Technology Assessment (EUnetHTA) and two rounds of public consultation.

Results: A set of twelve recommendations was developed, addressing the requirements for HRQoL data for relative effectiveness assessment, for cost-utility analyses and for informing clinical decision making. Recommendations relate to the choice of the type of measure as well as to aspects such as measurement frequency, target population and presentation.

Conclusions: The purpose and context of HRQoL measurement is crucial for the relevance of the data obtained with a specific HRQoL measure. It is recommended to always include a generic HRQoL instrument in clinical trials to cover a wide range of possible future uses of the HRQoL data.

Objectives: The aim of this study was to review and compare current health technology assessment (HTA) activities for medical devices across non-European Union HTA agencies.

Methods: HTA activities for medical devices were evaluated from three perspectives: organizational structure, processes, and methods. Agencies were primarily selected upon membership of existing HTA networks. The data collection was performed in two stages: stage 1–agency Web-site assessment using a standardized questionnaire, followed by review and validation of the collected data by a representative of the agency; and stage 2–semi-structured telephone interviews with key informants of a sub-sample of agencies.

Results: In total, thirty-six HTA agencies across twenty non-EU countries assessing medical devices were included. Twenty-seven of thirty-six (75 percent) agencies were judged at stage 1 to have adopted HTA-specific approaches for medical devices (MD-specific agencies) that were largely organizational or procedural. There appeared to be few differences in the organization, process and methods between MD-specific and non–MD-specific agencies. Although the majority (69 percent) of both categories of agency had specific methods guidance or policy for evidence submission, only one MD-specific agency had developed methodological guidelines specific to medical devices. In stage 2, many MD-specific agencies cited insufficient resources (budget, skilled employees), lack of coordination (between regulator and reimbursement bodies), and the inability to generalize findings from evidence synthesis to be key challenges in the HTA of medical devices.

Conclusions: The lack of evidence for differentiation in scientific methods for HTA of devices raises the question of whether HTA needs to develop new methods for medical devices but rather adapt existing methodological approaches. In contrast, organizational and/or procedural adaptation of existing HTA agency frameworks to accommodate medical devices appear relatively commonplace.

Objectives: Clinical research data are often collected on paper and later inputted onto an electronic database. This method is time consuming and potentially introduces errors. Therefore, to make primary data collection more efficient and less error prone we aimed to develop a touch-screen application for data collection in a psoriatic arthritis research clinic and compared it with the pre-existing paper-based system.

Methods: We developed a Web application using Java and optimized it for the iPad®. It highlights missing fields for physicians in real time, and only permits submission of data collection form after corrections are made. For its evaluation, seven physicians participated, and before each patient visit they were randomly assigned paper or iPad® data entry. Number of errors, length of visit, and time between clinic visit and completion of data entry were measured.

Results: A total of 106 patients seen in the clinic who agreed to participate were randomly assigned to be evaluated by clinic physicians using the iPad® (fifty-three patients) or a paper protocol (fifty-three patients). On average, 3.34 omissions were found per paper form, of which 2.24 would have been detected on the iPad®. The iPad® increased the mean patient encounter time from 37.2 minutes to 46.5 minutes, but eliminated delay between a clinic visit and its data entry.

Conclusions: Entering data using the iPad® application makes the patient encounter slightly longer, but reduces “missing fields.” It also eliminates the delay between clinic visit and data entry thus improving the efficiency of clinical data capture in a research setting.

Objectives: Colloquial evidence (CE) has been described as the informal evidence that helps provide context to other forms of evidence in guidance development. Despite challenges around quality, and the potential biases, the use of CE is becoming increasingly important in assessments where scientific literature is sparse and to also capture the experience of all stakeholders in discussions, including that of experts and patients. We aimed to ascertain how CE was being used at the National Institute for Health and Care Excellence (NICE).

Methods: Relevant data corresponding to the use of CE was extracted from all NICE technical and process manuals by two reviewers and quality assured and analyzed by a third reviewer. This was considered in light of the results of a focused literature review and a combined checklist for quality assessment was developed.

Results: At NICE, CE is utilised across all guidance producing programmes and at all stages of development. CE could range from information from experts and patient/carers, grey literature (including evidence from websites and policy reports) and testimony from stakeholders through consultation. Six tools for critical appraisal of CE were available from the literature and a combined best practice checklist has been proposed.

Conclusions: As decisions often need to be made in areas where there is a lack of published scientific evidence, CE is employed. Therefore to ensure its appropriateness the development of a validated CE data quality check-list to assist decision makers is essential and further research in this area is a priority.

Objectives: Economic evaluations, although not formally used in purchasing decisions for medical devices in Canada, are still being conducted and published. The aim of this study was to examine the way that prices have been included in Canadian economic evaluations of medical devices.

Methods: We conducted a review of the economic concepts and implications of methods used for economic evaluations of the eleven most implanted medical devices from the Canadian perspective.

Results: We found Canadian economic studies for five of the eleven medical devices and identified nineteen Canadian studies. Overall, the device costs were important components of total procedure cost, with an average ratio of 44.1 %. Observational estimates of the device costs were obtained from buyers or sellers in 13 of the 19 studies. Although most of the devices last more than 1 year, standard costing methods for capital equipment was never used. In addition, only eight studies included a sensitivity analysis for the device cost. None of the sensitivity analyses were based on actual price distributions.

Conclusions: Economic evaluations are potentially important for policy making, but although they are being conducted, there is no standardized approach for incorporating medical device prices in economic analyses. Our review provides suggestions for improvements in how the prices are incorporated for economic evaluations of medical devices.

Objectives: Developing a search strategy for use in a systematic review is a time-consuming process requiring construction of detailed search strings using complicated syntax, followed by iterative fine-tuning and trial-and-error testing of these strings in online biomedical search engines.

Methods: Building upon limitations of existing online-only search builders, a user-friendly computer-based tool was created to expedite search strategy development as part of production of a systematic review.

Results: Search Builder 1.0 is a Microsoft Excel®-based tool that automatically assembles search strategy text strings for PubMed (www.pubmed.com) and Embase (www.embase.com), based on a list of user-defined search terms and preferences. With the click of a button, Search Builder 1.0 automatically populates the syntax needed for functional search strings, and copies the string to the clipboard for pasting into Pubmed or Embase. The offline file-based interface of Search Builder 1.0 also allows for searches to be easily shared and saved for future reference.

Conclusions: This novel, user-friendly tool can save considerable time and streamline a cumbersome step in the systematic review process.

Objectives: A rapid scoping review was performed to support the development of a new clinical technology platform. An iterative sifting approach was adopted to address the challenges posed by the nature of the review question and the extremely large volume of search results to be sifted within the timescales of the review.

Methods: This study describes the iterative sifting approach applied in the scoping review and a preliminary validation of the methods applied.

Results: The searches performed for the rapid scoping review retrieved 27,198 records. This was the full set of records subjected to the staged, iterative sifting approach and the subsequent validation process. The iterative sifting approach involved the screening for relevance of 17,354 (i.e., 63.8 percent) of the 27,198 records. A list of fifty-three potential biomarker names was generated as a result of this iterative sifting method, of which nineteen were selected by clinical specialists for further scrutiny. The preliminary validation involved the exhaustive sifting of the remaining 9,844 previously unsifted records. The validation process identified sixteen additional potential biomarker names not identified by the iterative sifting process. The clinical specialists subsequently concluded that none were of further clinical interest.

Conclusions: This study describes an approach to the screening of search records that can be successfully applied in appropriate review and decision problems to allow the prioritization of the most relevant search records and achieve time savings. Following further refinement and standardization, this iterative sifting method may have potential for further applications in reviews and other decision problems.

Objectives: The aim of this study was to explore stakeholders’ points of views regarding the applicability and relevance of a framework for user involvement in health technology assessment (HTA) at the local level. We tested this framework in the context of the assessment of alternative measures to restraint and seclusion among hospitalized adults and those living in long-term-care facilities.

Methods: Twenty stakeholders (health managers, user representatives, and clinicians) from seven regions of Quebec participated in a semi-structured interview. A thematic analysis of the transcribed interviews was performed.

Results: The findings highlighted the relevance and applicability of the framework to this specific HTA. According to interviewees, direct participation of users in the HTA process allows them to be part of the decision-making process. User consultation makes it possible to consider the views of a wide variety of people, such as marginalized and vulnerable groups, who do not necessarily meet the requirements for participating in HTA committees. However, some user representatives emphasized that user consultation should be integrated into a more holistic and participatory perspective. The most frequent barrier associated with user involvement in HTA was the top-down health system, which takes little account of the user's perspective.

Conclusions: The proposed framework was seen as a reference tool for making practitioners and health managers aware of the different mechanisms of user involvement in HTA and providing a structured way to classify and describe strategies. However, there is a need for more concrete instruments to guide practice and support decision making on specific strategies for user involvement in HTA at the local level.