Fostering innovation for societal gain has been a core attribute of technology assessment (TA) since its inception. Although many observers were concerned that TA would be a means by which government would impede the development and use of technology, this was not the intent of policy makers or the agencies that conducted the original TAs.

Background: Identifying treatments that offer value and value for money is becoming increasingly important, with interest in how health technology assessment (HTA) and decision makers can take appropriate account of what is of value to patients and to society, and in the relationship between innovation and assessments of value.

Methods: This study summarizes points from an Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations, discussions among attendees, and background papers.

Results and Conclusions: Various perspectives on value were considered; most place patient health at the core of value. Wider elements of value comprise other benefits for: patients; caregivers; the health and social care systems; and society. Most decision-making systems seek to take account of similar elements of value, although they are assessed and combined in different ways. Judgment in decisions remains important and cannot be replaced by mathematical approaches. There was discussion of the value of innovation and of the effects of value assessments on innovation. Discussion also included moving toward “progressive health system decision making,” an ongoing process whereby evidence-based decisions on use would be made at various stages in the technology lifecycle. Five actions are identified: (i) development of a general framework for the definition and assessment of value; development by HTA/coverage bodies and regulators of (ii) disease-specific guidance and (iii) further joint scientific advice for industry on demonstrating value; (iv) development of a framework for progressive licensing, usage, and reimbursement; and (v) promoting work to better adapt HTA, coverage, and procurement approaches to medical devices.

Background: Two general alternative approaches, cost-effectiveness analysis and the therapeutic added value approach, link the pricing and approval of drugs to value. Value as assessed by payers is a function of: benefit less cost, willingness to pay for benefit, and how they handle uncertainty.

Methods: This study uses international examples to explore the elements of value that can be included in the assessment of health technologies, approaches to scoring the elements of value and how they can be combined to make a decision.

Results: A range of value elements, measures, and approaches to aggregation are identified across different HTA systems. We show that seemingly arbitrary differences in measurement and aggregation can lead to significantly different outcomes, and argue that the choice of values, measures, and decision-making processes should be informed by the societal values that underpin a health system.

Conclusions: We identify three areas for further research to improve both health system and industry R&D decision making: (i) whether more consistency could be achieved across health systems on the elements of value that matter; (ii) the relative merits of discrete versus continuous measures of value; and (iii) how structured decision making (to aggregate the elements of value) could or should become.

Background: In recent years, there has been growing interest in evaluating the health and economic impact of medical devices. Payers increasingly rely on cost-effectiveness analyses in making their coverage decisions, and are adopting value-based purchasing initiatives. These analytic approaches, however, have been shaped heavily by their use in the pharmaceutical realm, and are ill-adapted to the medical device context.

Methods: This study focuses on the development and evaluation of left ventricular assist devices (LVADs) to highlight the unique challenges involved in the design and conduct of device trials compared with pharmaceuticals.

Results: Devices are moving targets characterized by a much higher degree of post-introduction innovation and “learning by using” than pharmaceuticals. The cost effectiveness ratio of left ventricular assist devices for destination therapy, for example, decreased from around $600,000 per life year saved based on results from the pivotal trial to around $100,000 within a relatively short time period.

Conclusions: These dynamics pose fundamental challenges to the evaluation enterprise as well as the policy-making world, which this paper addresses.

The notion of value, in the evaluation of the clinical and cost-effectiveness of therapeutic interventions, was the subject of discussion at the HTAi Policy Forum in February 2013. A summary of its discussions and conclusions is published in this issue of the journal. This commentary considers the implications of the proposal that health technology assessment (HTA) agencies should include, in the value proposition, wider societal costs and benefits as well as incorporating innovative promise.

There is an expectation that the value of a new technology will be considered by decision makers in determining whether to provide subsidy. What constitutes the elements of value and how these may be weighted is not transparent, however, and further work needs to be done to determine the circumstances and mechanisms of their application. In the end, judgment will still be needed even if greater formalization of the value construct is developed.

The assessment of value within healthcare is undergoing a major transformation. Gone are the days when a new mechanism of action alone would be regarded as a high value innovation. Today, there is much more of an emphasis on what are the outcomes (i.e., mortality and morbidity benefits), how does this compare with the current standard of care and what is the impact to the usage of healthcare resources. This emphasis is quite understandable given the financial crisis we are experiencing with an ever growing and aging population that is placing considerable strain on the healthcare system.

Objectives: In this study, we explored crucial factors that explain a person's attitude toward and his or her assessment of telemedical systems. Special focus lies on the link between the perspective of physicians (telemedicine users) and technicians (telemedicine designers) to find potential barriers hindering the broad application of telemedical systems in hospitals and doctors’ offices.

Methods: A survey among medical professionals (n = 34), technical professionals (n = 39), and a control group (n = 44) was conducted. The collected data were assessed in terms of domain knowledge, attitudes toward telemedicine, and potential implementation barriers.

Results: Participants favored the conventional method over telemedical monitoring in regards to privacy, security, and time efficiency. In contrast, telemedicine was preferred with reference to efficiency of data analysis, long-term care, and emergency adequacy. Significant differences between the professional groups were found regarding perceived cost effectiveness, patients’ compliance, privacy protection, and false alarm sensitivity. Medical professionals exhibited the most reluctance toward using telemedical treatments.

Conclusions: The perceived drawbacks are attributed to a general uncertainty about the reliability of telemedical systems, in combination with concerns about personal data privacy, security, and loss of control. The reported fear of not being able to correctly use and handle the systems assumes a low usability of devices. To acquaint medical professionals with the benefits and limitations of telemedical systems, telemonitoring and tele-treatment should be included in the education of medical personnel at an early stage.

Objectives: Depression is common in diabetes and linked to a wide range of adverse outcomes. UK policy indicates that depression should be treated using conventional psychological treatments in a stepped care framework. This review aimed to identify current economic evidence of psychological treatments for depression among people with diabetes.

Method: Electronic search strategies (conducted in MEDLINE, EMBASE, PsycINFO, CINAHL, NHS EED) combined clinical and economic search terms to identify full economic evaluations of the relevant interventions. Prespecified screening and inclusion criteria were used. Standardized data extraction and critical appraisal were conducted and the results summarized qualitatively.

Results: Excluding duplicates, 1,516 studies for co-morbid depression and diabetes were screened. Four economic evaluations were identified. The studies found that the interventions improved health status, reduced depression and were cost-effective compared with usual care. The studies were all U.S.-based and evaluated collaborative care programs that included psychological therapies. Critical appraisal indicated limitations with the study designs, analysis and results for all studies.

Conclusions: The review highlighted the paucity of evidence in this area. The four studies indicated the potential of interventions to reduce depression and be cost-effective compared with usual care. Two studies reported costs per QALY gained of USD 267 to USD 4,317, whilst two studies reported the intervention dominated usual care, with net savings of USD 440 to USD 612 and net gains in patient free days or QALYs.

Objectives: The aim of this study was to evaluate the comparative effectiveness of anti-vascular endothelial growth factor therapy in the treatment of diabetic macular edema (DME).

Methods: Searches focused on reports concerning treatment of DME with aflibercept, bevacizumab, pegaptanib, or ranibizumab published between January 2000 and June 30, 2012, with comparisons to laser photocoagulation, sham injection, or other control (e.g., triamcinolone). Effectiveness was based on best-corrected visual acuity (BCVA), in terms of letters gained. Direct meta-analyses were conducted on BCVA for each anti-vascular endothelial growth factor (VEGF) agent; indirect comparisons also were performed for each anti-VEGF pair.

Results: A total of fifteen randomized controlled trials (eleven fair- or good-quality) and eight observational studies were included. No direct comparative studies were identified. Improvement in visual acuity versus control was seen with all agents (range: 4–9 letters); outcomes were consistent across multiple timepoints. Meta-analyses showed no statistically significant and/or consistent differences between agents in BCVA changes or the percentage of patients gaining more than ten letters. No discernible differences in the potential harms of anti-VEGFs, including ocular events, MI, stroke, and other cardiovascular events, as well as death, were noted between aflibercept, pegaptanib, and ranibizumab. Data on harms for bevacizumab were underreported.

Conclusions: All anti-VEGF agents are effective in improving visual acuity in comparison to laser photocoagulation. Evidence is insufficient to distinguish the performance of any single anti-VEGF agent over others. The safety of bevacizumab remains the greatest element of uncertainty.

Objectives: The aim of this study was to examine the potential cost-effectiveness of self-managed computer therapy for people with long-standing aphasia post stroke and to estimate the value of further research.

Methods: The incremental cost-effectiveness ratio of computer therapy in addition to usual stimulation compared with usual stimulation alone was considered in people with long-standing aphasia using data from the CACTUS trial. A model-based approach was taken. Where possible the input parameters required for the model were obtained from the CACTUS trial data, a United Kingdom-based pilot randomized controlled trial that recruited thirty-four people with aphasia and randomized them to computer treatment or usual care. Cost-effectiveness was described using an incremental cost-effectiveness ratio (ICER) together with cost-effectiveness acceptability curves. A value of information analysis was undertaken to inform future research priorities.

Results: The intervention had an ICER of £3,058 compared with usual care. The likelihood of the intervention being cost-effective was 75.8 percent at a cost-effectiveness threshold of £20,000 per QALY gained. The expected value of perfect information was £37 million.

Conclusions: Our results suggest that computer therapy for people with long-standing aphasia is likely to represent a cost-effective use of resources. However, our analysis is exploratory given the small size of the trial it is based upon and therefore our results are uncertain. Further research would be of high value, particularly with respect to the quality of life gain achieved by people who respond well to therapy.

Introduction: Spontaneous adverse drug events (ADE) reporting is the main source of data for assessing the risk/benefit of drugs available in the pharmaceutical market. However, its major limitation is underreporting, which hinders and delays the signal detection by Pharmacovigilance (PhV).

Objectives: To identify the techniques of educational intervention (EI) for promotion of PhV by health professionals and to assess their impact.

Methods: A systematic review was performed in the PUBMED, PAHO, LILACS and EMBASE databases, from November/2011 to January/2012, updated in March/2013. The strategy search included the use of health descriptors and a manual search in the references cited by selected papers.

Results: 101 articles were identified, of which 16 met the inclusion criteria. Most of these studies (10) were conducted in European hospitals and physicians were the health professionals subjected to most EI (12), these studies lasted from one month to two years. EI with multifaceted techniques raised the absolute number, the rate of reporting related to adverse drug reactions (ADR), technical defects of health technologies, and also promoted an improvement in the quality of reports, since there was increased reporting of ADR classified as serious, unexpected, related to new drugs and with high degree of causality.

Conclusion: Multifaceted educational interventions for multidisciplinary health teams working at all healthcare levels, with sufficient duration to reach all professionals who act in the institution, including issues related to medication errors and therapeutic ineffectiveness, must be validated, with the aim of standardizing the Good Practice of PhV and improve drug safety indicators.

Background: Health technology reassessment (HTR) is “a structured, evidence-based assessment of the clinical, social, ethical, and economic effects of a technology currently used in the healthcare system, to inform optimal use of that technology in comparison to its alternatives.” The purpose of this study is to describe the key themes in the context of current HTR activities and propose a way forward for this newly emerging field.

Methods: Data were gathered from a workshop held as part of the 2012 Canadian Agency for Drugs and Technology in Health (CADTH) symposium. The workshop consisted of two panel presentations followed by discussion; data gathered, including presentations and rich audience discussion transcripts, were analyzed for key themes emerging in the field of HTR using constant comparative analysis.

Results: The language chosen to describe HTR will set the tone for engagement. The identification of champions at multiple levels and political will are essential. Key lessons from international experience are: disinvestment is difficult, focus on clinical areas not specific technologies, identify clear goals of the HTR agenda. Six key themes were identified to move the HTR agenda forward: emphasize integration over segregation, focus on development of HTR methods and processes, processes are context-specific but lessons must be shared, build capacity in synergistic interdisciplinary fields, develop meaningful stakeholder engagement, strengthen postimplementation monitoring and evaluation.

Conclusions: To move this field forward, we must continue to build on international experiences with a focus on developing novel methodological approaches to generating, incorporating, and implementing evidence into policy and practice.

Objectives: The aim of this study was to develop and apply an instrument to map the level of health technology assessment (HTA) development at country level in selected countries. We examined middle-income countries (Argentina, Brazil, India, Indonesia, Malaysia, Mexico, and Russia) and countries well-known for their comprehensive HTA programs (Australia, Canada, and United Kingdom).

Methods: A review of relevant key documents regarding the HTA process was performed to develop the instrument which was then reviewed by selected HTAi members and revised. We identified and collected relevant information to map the level of HTA in the selected countries. This was supplemented by information from a structured survey among HTA experts in the selected countries (response rate: 65/385).

Results: Mapping of HTA in a country can be done by focusing on the level of institutionalization and the HTA process (identification, priority setting, assessment, appraisal, reporting, dissemination, and implementation in policy and practice). Although HTA is most advanced in industrialized countries, there is a growing community in middle-income countries that uses HTA. For example, Brazil is rapidly developing effective HTA programs. India and Russia are at the very beginning of introducing HTA. The other middle-income countries show intermediate levels of HTA development compared with the reference countries.

Conclusions: This study presents a set of indicators for documenting the current level and trends in HTA at country level. The findings can be used as a baseline measurement for future monitoring and evaluation. This will allow a variety of stakeholders to assess the development of HTA in their country, help inform strategies, and justify expenditure for HTA.

Objectives: The use of value of information methods to inform trial design has been widely advocated but there have been few empirical applications of these methods and there is little evidence they are widely used in decision making. This study considers the usefulness of value of information models in the context of a real clinical decision problem relating to alternative diagnostic strategies for patients with a recent non-ST elevated myocardial infarction.

Methods: A pretrial economic model is constructed to consider the cost-effectiveness of two competing strategies: coronary angiography alone or in conjunction with fractional flow reserve measurement. A closed-form solution to the expected benefits of information is used with optimal sample size estimated for a range of models reflecting increasingly realistic assumptions and alternative decision contexts.

Results: Fractional flow reserve measurement is expected to be cost-effective with an incremental cost-effectiveness ratio of GBP 1,621, however, there is considerable uncertainty in this estimate and consequently a large expected value to reducing this uncertainty via a trial. The recommended sample size is strongly affected by the reality of the assumptions of the expected value of information (EVI) model and the decision context.

Conclusions: Value of information models can provide a simple and flexible approach to clinical trial design and are more consistent with the constraints and objectives of the healthcare system than traditional frequentist approaches. However, the variation in sample size estimates demonstrates that it is essential that appropriate model parameters and decision contexts are used in their application.

Objectives: To promote the further development of HTA, this study aims to design a model for HTA priority setting, which would address national needs for a country with little experience in the field of HTA, and assess its feasibility for the health system.

Methods: Literature search and review, as well as qualitative research have been used in the design and testing of the model for HTA priority setting. To test the model and the methodology, a three-round Delphi study was conducted in 2011 in the form of an electronic questionnaire, which was distributed to the panel of eleven national experts. The panel was composed of experts representing various fields of the health care sector: policy-makers, health care service professionals and academics, with diverse professional roles.

Results: The designed model consists of four stages: (i) selection of experts for the panel, (ii) indication and selection of health policy topics, (iii) identification of health technologies, and (iv) priority setting. Three rounds of the Delphi study were performed to test the model and reach expert consensus on a list of health technologies for assessment, including pharmaceuticals, vaccines, medical devices, diagnostic methods, public health interventions, organizational systems, etc.

Conclusions: Based on the Delphi technique as a method for consensus building, the model for HTA priority setting was developed for Lithuania; however, it could also be used for other countries with little experience in the field of HTA.

Objectives: The aim of this study was to gather qualitative and quantitative data on criteria considered by healthcare decision makers.

Methods: Using snowball sampling and an online questionnaire with forty-three criteria organized into ten clusters, decision makers were invited by an international task force to report which criteria they consider when making decisions on healthcare interventions in their context. Respondents reported whether each criterion is “currently considered,” “should be considered,” and its relative weight (scale 0–5). Differences in proportions of respondents were explored with inferential statistics across levels of decision (micro, meso, macro), decision maker perspectives, and world regions.

Results: A total of 140 decision makers (1/3 clinical, 2/3 policy) from 23 countries in five continents completed the survey. The most relevant criteria (top ranked for “Currently considered,” “Should be considered,” and weights) were Clinical efficacy/effectiveness, Safety, Quality of evidence, Disease severity, and Impact on healthcare costs. Organizational and skill requirements were frequently considered but had relatively low weights. For almost all criteria, a higher proportion of decision makers reported that they “Should be considered” than that they are “Currently considered” (p < .05). For more than 74 percent of criteria, there were no statistical differences in proportions across levels of decision, perspectives and world regions. Statistically significant differences across several comparisons were found for: Population priorities, Stakeholder pressure/interests, Capacity to stimulate research, Impact on partnership and collaboration, and Environmental impact.

Conclusions: Results suggest convergence among decision makers on the relevance of a core set of criteria and on the need to consider a wider range of criteria. Areas of divergence appear to be principally related to contextual factors.

In the original publication of ‘Assessing the Impacts of Citizen Deliberations on the Health Technology Process’ the title was published incorrectly. The correct title should be: Assessing the Impacts of Citizen Deliberations on the Health Technology Assessment Process. The publisher regrets this error.