Background: Generation of additional evidence may be necessary to access new promising technologies (marketing approval or coverage). Access with evidence generation (AEG) is a more recent concept with regard to coverage than to marketing approval.

Objectives: One aim of Work Package 7 (WP7) Strand A of the European network for Health Technology Assessment (EUnetHTA) was to provide an overview of national AEG mechanisms associated with marketing approvals and funding or coverage decisions.

Methods: A systematic literature review, surveys of WP7 Partners, and consultation of key people were used to obtain information on the AEG mechanisms used by twenty-three countries (twenty European countries, United States, Canada [Ontario], and Australia).

Results: Interest in the implementation of AEG policies, particularly at the coverage decision stage, is growing. An overview of national experiences was used to draw up a generally applicable five-step policy framework for AEG mechanisms that comprised (i) a first assessment identifying knowledge gaps; (ii) a decision conditional to evidence generation; (iii) generation of the evidence requested; (iv) re-assessment integrating the new evidence; (v) a revised decision. The critical factors for success that were identified were coordination, methodological guidance, funding, and a regulatory framework. Countries were categorized on the basis of current implementation of the proposed policy framework.

Conclusions: International collaboration is necessary to gather a critical mass of high-quality data quickly and to ensure timely access to new promising technologies. The overview produced by WP7A has led to development of tools to facilitate collaboration on evidence generation.

Objectives: To compare the cost-effectiveness of generic psychological therapy (counseling) with routinely prescribed antidepressant drugs in a naturalistic general practice setting for a follow-up period of 12 months.

Methods: Economic analysis alongside a randomized clinical trial with patient preference arm. Comparison of depression-related health service costs at 12 months. Cost-effectiveness analysis of bootstrapped trial data using net monetary benefits and acceptability curves.

Results: No significant difference between the mean observed costs of patients randomized to antidepressants or to counseling (£342 vs £302, p = .56 [t test]). If decision makers are not willing to pay more for additional benefits (value placed on extra patient with good outcome, denoted by K, is zero), then we find little difference between the treatment modalities in terms of cost-effectiveness. If decision makers do place value on additional benefit (K > £0), then the antidepressant group becomes more likely to be cost-effective. This likelihood is in excess of 90% where decision makers are prepared to pay an additional £2,000 or more per additional patient with a good global outcome. The mean values for incremental net monetary benefits (INMB) from antidepressants are substantial for higher values of K (INMB = £406 when K = £2,500).

Conclusions: For a small proportion of patients, the counseling intervention (as specified in this trial) is a dominant cost-effective strategy. For a larger proportion of patients, the antidepressant intervention (as specified in this trial) is the dominant cost-effective strategy. For the remaining group of patients, cost-effectiveness depends on the value of K. Since we cannot observe K, acceptability curves are a useful way to inform decision makers.

Objectives: The aim of this study was to assess the accuracy of the English National Horizon Scanning Centre (NHSC) in identifying and filtering pharmaceutical developments using end user and international collaborator databases of emerging technologies as proxies for new drugs of likely significance to health services and/or patients.

Methods: We used the NHSC information system and the list of National Institute for Health and Clinical Excellence (NICE) technology appraisals to estimate the false positive rate for NHSC identification, filtration, and reporting. We assessed the sensitivity of NHSC identification and filtration of pharmaceuticals for NICE technology appraisals from 1999 to the end of December 2010, and for pharmaceuticals entered into the EuroScan International Network database.

Results: We estimate that overall NHSC identification, filtration and reporting had a positive predictive value of 0.39 (95 percent CI, 0.36 to 0.43) and a false positive rate of 60 percent. Using NICE appraisals and EuroScan's database as proxies for pharmaceuticals of significance, we estimate the NHSC sensitivity over the 10-year period at 0.92 (95 percent CI, 0.89 to 0.95) and 0.89 (95 percent CI, 0.82 to 0.96) respectively.

Conclusions: Our results suggest that the NHSC has performed well in terms of sensitivity over the past decade, but that the false positive rate of 60 percent may indicate that the filtration criteria for pharmaceuticals could be tightened for increased efficiency. Future evaluations of EAA systems should include an element of external review and explore the level of accuracy acceptable to funders and customers of such systems.

This review of the advantages (peaks) and problems (pits) of large data bases to study quality contrasts their suitability with randomized control trials. Researchers need to advise policymakers and others about when statistically significant differences in quality are also politically and socially significant and deserve responsible reactions.

This article describes the findings of an ongoing assessment of prostatectomy that relied on the use of administrative data bases. Examples of the use of claims data for monitoring outcomes and treatment comparisons are provided, as well as a discussion of the strengths and limitations of administrative data for technology assessment.

The paper outlines the context in which patient's views have been emphasized and the evidence from survey research about the criteria users employ to evaluate health care. Evidence from qualitative research shows that users are ambivalent about modern medicine and are particularly sceptical about the value of drugs and medicine.

Objectives: Constructive Technology Assessment (CTA) is a means to guide early implementation of new developments in society, and can be used as an evaluation tool for Coverage with Evidence Development (CED). We used CTA for the introduction of a new diagnostic test in the Netherlands, the 70-gene prognosis signature (MammaPrint®) for node-negative breast cancer patients.

Methods: Studied aspects were (organizational) efficiency, patient-centeredness and diffusion scenarios. Pre-post structured surveys were conducted in fifteen community hospitals concerning changes in logistics and teamwork as a consequence of the introduction of the 70-gene signature. Patient-centeredness was measured by questionnaires and interviews regarding knowledge and psychological impact of the test. Diffusion scenarios, which are commonly applied in industry to anticipate on future development and diffusion of their products, have been applied in this study.

Results: Median implementation-time of the 70-gene signature was 1.2 months. Most changes were seen in pathology processes and adjuvant treatment decisions. Physicians valued the addition of the 70-gene signature information as beneficial for patient management. Patient-centeredness (n = 77, response 78 percent): patients receiving a concordant high-risk and discordant clinical low/high risk-signature showed significantly more negative emotions with respect to receiving both test-results compared with concordant low-risk and discordant clinical high/low risk-signature patients. The first scenario was written in 2004 before the introduction of the 70-gene signature and identified hypothetical developments that could influence diffusion; especially the “what-if” deviation describing a discussion on validity among physicians proved to be realistic.

Conclusions: Differences in speed of implementation and influenced treatment decisions were seen. Impact on patients seems especially related to discordance and its successive communication. In the future, scenario drafting will lead to input for model-based cost-effectiveness analysis. Finally, CTA can be useful as a tool to guide CED by adding monitoring and anticipation on possible developments during early implementation, to the assessment of promising new technologies.

The growing evidence of the efficacy and effectiveness of dental implants calls for economic evaluations to determine the economic efficiency of this technology for different indications. Such studies must be integrated with the clinical evaluations in order to produce the relevant data. In most cases, dental implants will produce a better outcome than the best alternative technology, but this improvement will only come at greater cost. Cost–benefit evaluations of dental implants must therefore address the difficult task of assessing the value of the improvement in oral health.

Objectives: The aim of this study was to assess the cost-effectiveness of drotrecogin alfa (activated) compared with best supportive care in a UK cohort of adult intensive-care patients with severe sepsis.

Methods: A systematic review of evidence on the clinical- and cost-effectiveness of drotrecogin alfa (activated) was undertaken, and a decision-analytic model was developed to estimate the cost-effectiveness of treatment in the United Kingdom. Trial data from the Recombinant Human Activated Protein C Worldwide Evaluation in Severe Sepsis (PROWESS) study have been synthesized with other data, including UK data on severe sepsis, to estimate the costs and consequences of treatment over time.

Results: For patients with severe sepsis and multiple organ dysfunction, the estimates of cost per life year and cost per quality-adjusted life year (QALY) are £4,931 and £8,228, respectively. For patients with severe sepsis alone, the cost per life-year and cost per QALY are £5,495 and £9,161, respectively.

Conclusions: Whereas the therapeutic cost for drotrecogin alfa (activated) appears high (at around £5,000 per patient) and the potential impact on the provider budget is considerable, drotrecogin alfa (activated) is clinically effective, represents a cost-effective use of resources, and is a significant advance in the treatment of severe sepsis in patients requiring intensive care.

The major aim of this study was to estimate and compare the patient-borne costs of lifelong subcutaneous gamma globulin therapy at the hospital and at home. Thirty patients were included and the data were collected with a questionnaire. The introduction of self-therapy at home reduced the total yearly costs by approximately 50% and the out-of-pocket expenses for the palients by 85%.

This article reviews the developments in HTA in four countries, France, The Netherlands, Sweden, and United Kingdom, in relation to public health. It emphasizes that the majority of assessments made are concerned with individual clinical care rather than with the optimization of health. Possible reasons for the neglect of public health issues are that these are inherently more complex than the assessment of individual procedures or drugs. They are usually multisectoral, politically charged, and often considered mundane and “common sense” and, thus, not requiring evaluation (although when evaluations are done they are often counterintuitive). Unless more emphasis is given to the development and evaluation of public health measures, it is unlikely that there will be any major advances in health status. Possible areas for future assessment should include such issues as smoking, drug and other substance misuse, nutrition, and health inequalities. However, it is unlikely that these major areas of concern will be included in the future unless the methods of choice for priorities of development and assessment are changed to include measures that improve health status rather than only clinical services.

Objectives: The aim of this study was to identify risk points in the different stages of the smart infusion pump implementation process to prioritize improvement measures.

Methods: Failure modes and effects analysis (FMEA) in the pediatric intensive care unit (PICU) of a General and Teaching Hospital. A multidisciplinary team was comprised of two intensive care pediatricians, two clinical pharmacists and the PICU nurse manager. FMEA was carried out before implementing CareFusion infusion smart pumps and eighteen months after to identify risk points during three different stages of the implementation process: creating a drug library; using the technology during clinical practice and analyzing the data stored using Guardrails® CQI v4.1 Event Reporter software.

Results: Several actions for improvement were taken. These included carrying out periodical reviews of the drug library, developing support documents, and including a training profile in the system so that alarms set off by real programming errors could be distinguished from those caused by incorrect use of the system. Eighteen months after the implementation, these measures had helped to reduce the likelihood of each risk point occurring and increase the likelihood of their detection.

Conclusions: Carrying out an FMEA made it possible to detect risk points in the use of smart pumps, take action to improve the tool, and adapt it to the PICU. Providing user training and support tools and continuously monitoring results helped to improve the usefulness of the drug library, increased users’ compliance with the drug library, and decreased the number of unnecessary alarms.

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: To examine the feasibility of retrieving missing outcome data for summary meta-analyses using an example dealing with the effects of aerobic exercise on lipids and lipoproteins in adults.

Methods: Missing lipid and/or lipoprotein data from a currently developed meta-analytic data base were requested by means of electronic mail from 39 of 174 (22.4 percent) eligible studies. Binary logistic regression was used to examine whether year of publication and country were significant predictors for whether data would be provided.

Results: Of the thirty-nine studies from which data were requested, usable data were received for thirteen (33.3 percent) of the studies. The addition of these previously missing data decreased the percentage of eligible studies that would have had to be excluded by 33.5 percent (from 22.4 percent to 14.9 percent). Neither year of publication nor country in which the study was conducted (United States versus other) were significant predictors of whether missing data would be provided or not (p>.05).

Conclusions: Moderate success was achieved in the acquisition of missing outcome data dealing with the effects of aerobic exercise on lipids and lipoproteins in adults. However, whether this level of response is true in other areas of research needs to be determined by additional research.

Background: Walking is a good way to meet physical activity guidelines. We examined the effectiveness of walking in groups compared with walking alone or inactive controls in physically healthy adults on physical activity and quality of life. (PROSPERO CRD42016033752).

Methods: We searched Medline, Embase, Cinahl, Web of Knowledge Science Citation Index, and Cochrane CENTRAL until March 2016, for any comparative studies, in physically healthy adults, of walking in groups compared with inactive controls or walking alone, reporting any measure of physical activity. We searched references from recent relevant systematic reviews. Two reviewers checked study eligibility and independently extracted data. Disagreements were resolved through discussion. Quality was assessed using likelihood of selection, performance, attrition, and detection biases. Meta-analysis was conducted using Review Manager 5.3.

Results: From 1,404 citations, 18 studies were included in qualitative synthesis and 10 in meta-analyses. Fourteen compared group walking to inactive controls and four to walking alone. Eight reported more than one measure of physical activity, none reported according to current guidelines. Group walking compared with inactive controls increased follow-up physical activity (9 randomized controlled trials, standardized mean difference [SMD] 0.58 [95 percent confidence interval {CI}, 0.34–0.82] to SMD 0.43 [95 percent CI, 0.20–0.66]). Compared with walking alone, studies were too few and too heterogeneous to conduct meta-analysis, but the trend was improved physical activity at follow-up for group walking participants. Seven (all inactive control) reported quality-of-life: five showed statistically significantly improved scores.

Discussion: Better evidence may encourage government policy to promote walking in groups. Standardized physical activity outcomes need to be reported in research.

Objectives: To assess the cost-effectiveness of alternative initial strategies in the management of uninvestigated dyspepsia in adult patients presenting to a primary care physician in Canada.

Methods: Clinical decision-making was modeled based on outcomes data from the literature and expert opinion. Costs were obtained from official reimbursement lists, and a detailed microcosting analysis of a gastroscopy. Costs were analysed over a one-year period after initial presentation, including consideration of a single relapse of symptoms. The main outcome was a symptomatic cure. The seven management strategies based on different initial tests were endoscopy, double-contrast barium meal, empirical eradication therapy, empirical antisecretory treatment, urea breath test (UBT), laboratory serology, and sequential testing (laboratory serology followed, if Helicobacter pylori positive, by UBT).

Results: In patients under age 45, endoscopy, double-contrast barium meal, and sequential testing were not cost-effective approaches. UBT was the most effective and most costly strategy costing $8,238 per additional patient cure compared with laboratory serology. In patients over age 45, UBT was again the most effective and most costly strategy, but endoscopy was the most reliable in detecting gastric cancers. Clinical variables that impacted these findings were the probability of symptomatic relapse in patients with nonulcer dyspepsia (NUD) after successful versus failed H. pylori eradication, the probability of finding a duodenal ulcer (DU) in a young dyspeptic patient, the specificity of UBT, and the prevalence of H. pylori in patients with DU.

Conclusions: A “test-and-treat” approach was favored, with UBT being the most effective but most costly initial test in adult dyspeptics under age 45. The choice of the most cost-effective approach is dependent on the benefits of H. pylori eradication in patients with NUD. In patients over age 45, UBT is the most effective and most costly, but endoscopy results in the early detection of most gastric cancers.The authors are indebted to the other members of the UBTAN Group, including Drs. Phil Sherman, Alan Thomson, and Sander Veldhuyzen van Zanten. The authors also thank Paul Sinclair, Eileen Grace, and Lawrence Joseph for their much valued comments and opinions.AB and CAF are research scholars of the Fonds de la Recherche en Santé du Québec. This work was supported in part by an “at arms length” grant from AstraZeneca Canada.

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.

A controversial issue in cost-effectiveness analysis is the discounting of gained life-years. What has not been realized, however, is that the different methods used for discounting this measurement provide fundamentally different results. The method used is seldom explicitly stated. In the present article the four main methods for the discounting of gained life-years are reviewed and compared. The conclusion is that if we wish to continue comparing results, researchers must employ the same methodology.

Health outcome assessment may be used for a variety of purposes: to evaluate the effectiveness of medical practices: to assess the quality of services provided; to educate providers, purchasers, and users of health services about the sequelae of treated and untreated disease; to guide reimbursement and regulatory policy; and to characterize the health status of a population. This article focuses on the use of health outcomes to evaluate the effectiveness of medical practices and to assess the quality of services provided.

Purpose: The aim of this study was to compare the general health-related quality of life (HRQoL), the metabolic control (HbA1c), the anthropometric measurement, and the cardiorespiratory fitness (expressed by VO2max) in youths with type 1 diabetes treated with continuous subcutaneous insulin infusion (CSII) to those receiving multiple daily injections (MDI). We looked for factors influencing the HRQoL and metabolic control.

Methods: A total of 239 patients treated with CSII (51 girls and 53 boys) or MDI (64 girls and 71 boys) between ages 8 and 18 years were assessed with the Pediatric Quality of Life Inventory, Generic Core Scales, and Diabetes Module. VO2max was evaluated using the 20-meter shuttle run test.

Results: CSII group had significantly better HRQoL according to both child self-report and parent proxy-report. Youths with CSII reported better physical, emotional, and school-related functioning, and had less diabetes-related fear and symptoms than the MDI group. There were no significant differences in body mass index z-scores, insulin doses, HbA1c, and VO2max between the groups. HRQoL was predicted by the CSII therapy (β = −0.220; p = .000) and the VO2max (β = 0.386; p = .000), other clinical and anthropometric parameters had no effect; the HbA1c was predicted only by VO2max (β = −0.353; p = .000).

Conclusions: Diabetic youths treated with CSII therapy have better HRQoL than those treated with MDI. There are no differences between the investigated groups in anthropometric data, glycated hemoglobin, and physical fitness. Moreover, good physical fitness has an important role in achieving better metabolic control and HRQoL, which underlines the importance of regular aerobic exercise in the treatment and care of type 1 diabetes in childhood.