Background: The adoption of new medical devices and diagnostics is often hampered by lack of published evidence which makes conventional health technology assessment (HTA) difficult. We now have 5 years’ experience of the Medical Technologies Advisory Committee of the National Institute for Health and Care Excellence (NICE) in the United Kingdom, addressing this problem. This committee assesses devices and diagnostics against claims of advantage, to produce guidance on adoption for the health service.

Methods: We have reflected on the practical, technical, and intellectual processes we have used in developing guidance for the health service.

Results: When scientific and clinical evidence is sparse, promise and plausibility play an increased part in decision-making. Drivers of promise include a clear design and mechanism of action, the possibility of radical improvement in care and/or resource use, and improving health outcomes for large numbers of patients. Plausibility relates to judgements about the whether the promise is likely to be delivered in a “real world” setting. Promise and plausibility need to be balanced against the amount of evidence available. We examine the influence they may have on decision-making compared with other factors such as risk and cost.

Conclusions: Decisions about adoption of new devices and diagnostics with little evidence are influenced by judgements of their promise and the plausibility of claims that they will provide benefits in a real-world setting. This kind of decision making needs to be transparent and this article explains how these influences can be balanced against the use of more familiar criteria.

Objectives: While most studies have focused on analyzing the results of telemedicine use, it is crucial to consider the determinants of its use to fully understand the issue. This article aims to provide evidence on the determinants of telemedicine use in clinical practice.

Methods: The survey targeted a total population of 398 medical professionals from a healthcare institution in Spain. The study sample was formed by the ninety-three primary care physicians who responded. Using an extended Technology Acceptance Model and microdata for the ninety-six physicians, binary logistic regression analysis was carried out.

Results: The analysis performed confirmed the model's goodness-of-fit, which allowed 48.1 percent of the dependent variable's variance to be explained. The outcomes revealed that the physicians at the healthcare institution placed greater importance on telemedicine's potential to reduce costs, and on its usefulness to the medical profession. The perception of medical information security and confidentiality and the patients’ predisposition toward telemedicine were the second explanatory factors in order of importance. A third set of moderating effects would appear to corroborate the importance of the physicians’ own opinions.

Conclusions: These results have revealed the need for a dynamic approach to the design of telemedicine use, especially when it targets a variety of end-users. Hence, the importance of conducting studies before using telemedicine, and attempting to identify which of the above-mentioned predictors exert an influence and how.

Objectives: Although consideration of ethical issues is recognized as a crucial part of health technology assessment, ethics analysis for HTA is generally perceived as methodologically underdeveloped in comparison to other HTA domains. The aim of our study is (i) to verify existing tools for quality assessment of ethics analyses for HTA, (ii) to consider some arguments for and against the need for quality assessment tools for ethics analyses for HTA, and (iii) to propose a preliminary set of criteria that could be used for assessing the quality of ethics analyses for HTA.

Methods: We systematically reviewed the literature, reviewed HTA organizations’ Web sites, and solicited views from thirty-two experts in the field of ethics for HTA.

Results: The database and HTA agency Web site searches yielded 420 references (413 from databases, seven from HTA Web sites). No formal instruments for assessing the quality of ethics analyses for HTA purposes were identified. Thirty-two experts in the field of ethics for HTA from ten countries, who were brought together at two workshops held in Edmonton (Canada) and Cologne (Germany) confirmed the findings from the literature.

Conclusions: Generating a quality assessment tool for ethics analyses in HTA would confer considerable benefits, including methodological alignment with other areas of HTA, increase in transparency and transferability of ethics analyses, and provision of common language between the various participants in the HTA process. We propose key characteristics of quality assessment tools for this purpose, which can be applied to ethics analyses for HTA purposes.

Objectives: We explore how broader aspects of a treatment's value and the impact of the condition on patients not captured by routine health technology assessment (HTA) methods using clinical and economic evidence, defined as “other considerations,” may influence HTA processes in different settings.

Methods: Countries included were England, Scotland, Sweden, and France. Data sources were the publicly available reports on HTA recommendations. Ten drugs with European Medicines Agency orphan designation and appraised in England were selected. Qualitative thematic analysis was used to systematically identify and code all “other considerations” based on a previously developed methodological framework, which also coded whether it was provided by stakeholders, and how it influenced the decision.

Results: A classification framework of scientific and social value judgments was developed and used throughout the study. A total of 125 “other considerations” were identified and grouped into ten subcategories based on the information provided. Eighteen to 100 percent of these, depending on the agency, were put forward as one of the main reasons for the final decision potentially contributing to accepting a higher incremental cost-effectiveness ratio or uncertain evidence. Some of these were nonquantified or nonelicited and pertained to the assessor's judgment. A taxonomy of these value judgments was created to be used in future cases. Results also contributed to better defining the determinants of social value and improving accountability for reasonableness.

Conclusions: The systematic identification of the scientific and social value judgments enables to better understanding the dimensions of value, which can be used to improve their transparency and consistent use across decisions and settings.

Objectives: Traditional economic evaluations for most health technology assessments (HTAs) have previously not included environmental outcomes. With the growing interest in reducing the environmental impact of human activities, the need to consider how to include environmental outcomes into HTAs has increased. We present a simple method of doing so.

Methods: We adapted an existing clinical-economic model to include environmental outcomes (carbon dioxide [CO2] emissions) to predict the consequences of adding insulin to an oral antidiabetic (OAD) regimen for patients with type 2 diabetes mellitus (T2DM) over 30 years, from the United Kingdom payer perspective. Epidemiological, efficacy, healthcare costs, utility, and carbon emissions data were derived from published literature. A scenario analysis was performed to explore the impact of parameter uncertainty.

Results: The addition of insulin to an OAD regimen increases costs by 2,668 British pounds per patient and is associated with 0.36 additional quality-adjusted life-years per patient. The insulin-OAD combination regimen generates more treatment and disease management-related CO2 emissions per patient (1,686 kg) than the OAD-only regimen (310 kg), but generates fewer emissions associated with treating complications (3,019 kg versus 3,337 kg). Overall, adding insulin to OAD therapy generates an extra 1,057 kg of CO2 emissions per patient over 30 years.

Conclusions: The model offers a simple approach for incorporating environmental outcomes into health economic analyses, to support a decision-maker's objective of reducing the environmental impact of health care. Further work is required to improve the accuracy of the approach; in particular, the generation of resource-specific environmental impacts.

In Husereau et al., the affiliation for Laura Sampietro-Colom was listed as “Hospital Clinic”. It should have instead been listed as, “Hospital Clinic, Barcelona, Spain”.

Objectives: Untreated obstructive sleep apnea-hypopnea syndrome (OSAHS) is associated with excessive daytime sleepiness, increased risk of cardiovascular (CV) disease, and road traffic accidents (RTAs), which impact survival and health-related quality of life. This study, funded by the French National Authority for Health (HAS), aimed to assess the cost-effectiveness of different treatments (i.e., continuous positive airway pressure [CPAP], dental devices, lifestyle advice, and no treatment) in patients with mild-to-moderate OSAHS in France.

Methods: A Markov model was developed to simulate the progression of two cohorts, stratified by CV risk, over a lifetime horizon. Daytime sleepiness and RTAs were taken into account for all patients while CV events were only considered for patients with high CV risk.

Results: For patients with low CV risk, incremental cost-effectiveness ratio (ICER) of dental devices versus no treatment varied between 32,976 EUR (moderate OSAHS) and 45,579 EUR (mild OSAHS) per quality-adjusted life-year (QALY), and CPAP versus dental devices, above 256,000 EUR/QALY. For patients with high CV risk, CPAP was associated with a gain of 0.62 QALY compared with no treatment, resulting in an ICER of 10,128 EUR/QALY.

Conclusion: The analysis suggests that it is efficient to treat all OSAHS patients with high CV risk with CPAP and that dental devices are more efficient than CPAP for mild-to-moderate OSAHS with low CV risk. However, out-of-pocket costs are currently much higher for dental devices than for CPAP (i.e., 3,326 EUR versus 2,430 EUR) as orthodontic treatment is mainly non-refundable in France.

Objectives: Herpes zoster (HZ) is characterized by a painful skin rash. Its main complication is postherpetic neuralgia (PHN), pain persisting or occurring after the rash onset. HZ treatment aims to reduce acute pain, impede the onset complications, and disease progression. The aim of this study was to assess the cost-effectiveness of HZ vaccination compared with no vaccination strategy, within the Italian context.

Methods: The natural history of HZ and PHN was mapped through a Markov model with lifetime horizon. A population of patients aged between 60 and 79 years was hypothesized. Third party payer (Italian National Health Service, I-NHS) and societal perspectives were adopted. Data were derived from literature.

Results and Conclusions: The incremental cost-effectiveness ratio of the vaccination equaled EUR 11,943 per quality-adjusted life-year (QALY) under the I-NHS perspective and EUR 11,248 per QALY under the societal perspective. Considering a cost-effectiveness threshold of EUR 30,000/QALY, the multi-way sensitivity analysis showed that vaccination is cost-effective regardless of the perspective adopted, in 99 percent of simulations.

Objectives: A patient reported outcome (PRO) is “any report of the status of a patient's health condition that comes directly from the patient without interpretation of the patient's response by a clinician or anyone else” (USFDA 2009). PROs are discussed widely, and many regard the patients’ perspective on treatment benefit as very valuable. Although many PROs have shown satisfactory measurement properties including reliability, validity, and responsiveness, there is great concern about risk of bias, that is, in clinical trials.

Methods: Differences in perspectives of PRO measurement in rare diseases are given arising from methodology, clinical, HTA, and patient advocacy views.

Results: PROs are playing an important role in dealing with treatment benefit especially in small sample size as occurring often in rare diseases. Challenges remain especially regarding lack of responsiveness of generic measures, limited capture of all patient relevant aspects, study design and high risk of bias.

Conclusions: PROs seem a valuable instrument to detect patient relevant aspects in rare diseases. They should be seen in addition to other approved assessment methods as randomized controlled trials but not as their substitute.

Objectives: The majority of patients in India access private sector providers for curative medical services. However, there is scanty information on the cost of treatment of critically ill patients in this setting. The study evaluates the cost and extent of financial subsidy required for patients admitted to an intensive care unit (ICU) in India.

Methods: Data on direct medical, direct nonmedical, and indirect cost were prospectively collected from critically ill patients admitted to a tertiary teaching hospital in India. Willingness-to-pay (WTP) amount was obtained from the next-of-kin following admission and the actual cost paid by the family at discharge was recorded.

Results: The main diagnoses (n = 499) were infection (26 percent) and poisoning (21 percent). The mean APACHE-II score was 13.9 (95 percent confidence interval [CI], 13.3–14.5); 86 percent were ventilated. ICU stay was 7.8 days (95 percent CI, 7.3–8.3). Hospital mortality was 27.9 percent. Direct medical cost accounted for 77 percent (US$ 2164) of the total treatment cost (US$ 2818). Indirect cost and direct nonmedical cost contributed to 19 percent (US$ 547.5) and 4 percent (US$ 106.5), respectively. Average total and daily ICU cost were US$ 1,897 and US$ 255, respectively. Although the family's WTP was 53 percent (US$ 1146; 95 percent CI, 1090–1204) of direct medical cost, their final contribution was 67.7 percent (US$ 1465; 95 percent CI, 1327–1604).

Conclusions: The cost of an ICU admission in our setting is US$ 2818. Although the family's contribution to expenses exceeded their initial WTP, a substantial subsidy (33 percent) is still required. Alternate financing strategies for the poor and optimization of ICU resources are urgently required.

Objectives: There is little specific guidance on performing an early cost-effectiveness analysis (CEA) of medical tests. We developed a framework with general steps and applied it to two cases.

Methods: Step 1 is to narrow down the scope of analysis by defining the test's application, target population, outcome measures, and investigating current test strategies and test strategies if the new test were available. Step 2 is to collect evidence on the current test strategy. Step 3 is to develop a conceptual model of the current and new test strategies. Step 4 is to conduct the early-CEA by evaluating the potential (cost-)effectiveness of the new test in clinical practice. Step 5 involves a decision about the further development of the test.

Results: The first case illustrated the impact of varying the test performance on the headroom (maximum possible price) of an add-on test for patients with an intermediate-risk of having rheumatoid arthritis. Analyses showed that the headroom is particularly dependent on test performance. The second case estimated the minimum performance of a confirmatory imaging test to predict individual stroke risk. Different combinations of sensitivity and specificity were found to be cost-effective; if these combinations are attainable, the medical test developer can feel more confident about the value of further development of the test.

Conclusions: A well-designed early-CEA methodology can improve the ability to develop (cost-)effective medical tests in an efficient manner. Early-CEAs should continuously integrate insights and evidence that arise through feedback, which may convince developers to return to earlier steps.

Objectives: External experts can be consulted at different stages of an HTA. When using vague information sources, it is particularly important to plan, analyze, and report the information processing in a standardized and transparent way. Our objective was to search and analyze recommendations regarding where and how to include expert data in HTA.

Methods: We performed a systematic database search and screened the Internet pages of seventy-seven HTA organizations for guidelines, recommendations, and methods papers that address the inclusion of experts in HTA. Relevant documents were downloaded, and information was extracted in a standard form. Results were merged in tables and narrative evidence synthesis.

Results: From twenty-two HTA organizations, we included forty-two documents that consider the use of expert opinion in HTA. Nearly all documents mention experts in the step of preparation of the evidence report. Six documents address their role for priority setting of topics, fifteen for scoping, twelve for the appraisal of evidence and results, another twelve documents mention experts when considering the dissemination of HTA results.

During the assessment step, experts are most often asked to amend the literature search or to provide expertise for special data analyses. Another issue for external experts is to appraise the HTA results and refer them back to a clinical and social context. Little is reported on methods of expert elicitation when their input substitutes study data.

Conclusions: Despite existing recommendations on the use of expert opinion in HTA, common standards for elicitation are scarce in HTA guidelines.

Objectives: The regularly structured adaptation of health technology assessment (HTA) programs is of utmost importance to sustain the relevance of the products for stakeholders and to justify investment of scarce financial resources. This study describes internal adjustments and external measures taken to ensure the Horizon Scanning Programme in Oncology (HSO) is current.

Methods: Formal evaluation methods comprising a survey, a download, an environmental analysis, and a Web site questionnaire were used to evaluate user satisfaction.

Results: The evaluation showed that users were satisfied with HSO outputs in terms of timeliness, topics selected, and depth of information provided. Discussion of these findings with an expert panel led to changes such as an improved dissemination strategy and the introduction of an additional output, that is, the publication of a league table of emerging oncology drugs. The rather high level of international usage and the environmental analysis highlighted a considerable overlap in topics assessed and, thus, the potential for international collaboration. As a consequence, thirteen reports were jointly published based on eleven “calls for collaboration.” To further facilitate collaboration and the usability of reports for other agencies, HSO reports will be adjusted according to tools developed at a European level.

Conclusions: Evaluation of the impact of HTA programs allows the tailoring of outputs to fit the needs of the target population. However, within a fast developing HTA community, estimates of impact will increasingly be determined by international collaborative efforts. Refined methods and a broader definition of impact are needed to ultimately capture the efficiency of national HTA programs.

Objectives: To review and compare current Health Technology Assessment (HTA) activities for medical devices (MDs) across European HTA institutions.

Methods: A comprehensive approach was adopted to identify institutions involved in HTA in European countries. We systematically searched institutional Web sites and other online sources by using a structured tool to extract information on the role and link to decision making, structure, scope, process, methodological approach, and available HTA reports for each included institution.

Results: Information was obtained from eighty-four institutions, forty-seven of which were analyzed. Fifty-four methodological documents from twenty-three agencies in eighteen countries were identified. Only five agencies had separate documents for the assessment of MDs. A few agencies made separate provisions for the assessment of MDs in their general methods. The amount of publicly available HTA reports on MDs varied by device category and agency remit.

Conclusions: Despite growing consensus on their importance and international initiatives, such as the EUnetHTA Core Model®, specific tools for the assessment of MDs are rarely developed and implemented at the national level. Separate additional signposts incorporated in existing general methods guides may be sufficient for the evaluation of MDs.

Objectives: We describe a new evidence-based method for screening and evaluating emerging medical technologies. Washington State agencies, under legislative direction, have granted authority to its agency Medical Directors and policy leaders to make coverage decisions on medical technologies using a “dossier” process. The dossier process is employed when technology advocates or manufacturers request Washington State healthcare purchasers to pay for new and emerging technologies. This offers the advocate an opportunity to submit scientific evidence and information classically associated with a more formal health technology assessment.

Methods: The submitted information is independently reviewed and summarized for Washington State's public healthcare purchasers allowing a more standardized coverage decision for all public purchasers in Washington State.

Results: This process has allowed Washington State to make twelve evidence-based coverage decisions at a fraction of the cost of classic technology assessment. To date, of twelve reviews over 6 years, one health technology was approved for coverage, ten were not covered and one did not require a coverage decision.

Conclusions: This evidence-based dossier process has yielded high-value coverage decisions of new and emerging medical technologies for public healthcare purchasers in Washington State.

Objectives: The aim of this study was to develop criteria for the prioritization of topics for health technology assessment (HTA) in the healthcare system of Kazakhstan.

Methods: Initial proposals for criteria were suggested through consultation with Ministry of Health (MoH) policy areas. These were refined through a workshop attended by HTA department staff, persons from medical universities and research institutes, and MoH policy makers. The workshop included discussion on methods used in international HTA practice. Opinions of participants on selection of criteria from those specified in a review of prioritization processes were used to define a list for inclusion in an instrument for routine use. A scoring system was established in later discussion.

Results: Selected criteria for HTA prioritization were burden of disease, availability of alternative technology, clinical effectiveness, economic efficiency, budget impact, and ethical, legal, and/or psychosocial aspects. For each criterion, a health technology under consideration is given a score from 3 (High) to 1 (Low). The total score determines whether the technology is of high to medium priority or of low priority. Determination of priorities for assessment, using the instrument, should be carried out by an expert group appointed by the MoH. The process was applied in 2014 to a selection of topics, and three health technologies were chosen for full assessments.

Conclusions: Criteria for prioritization have evolved with development of the HTA program in Kazakhstan. A method for HTA prioritization has been developed that is easy to apply, requires comparatively few resources, and is compatible with processes required by the MoH.