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.

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.

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.

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: 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.

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%.

Objectives: Treatment switching occurs when patients in a randomized clinical trial switch from the treatment initially assigned to them to another treatment, typically from the control to experimental treatment. This study discusses the issues this raises and possible approaches to addressing them in trials of cancer drugs.

Methods: Stakeholders from around the world were invited to a 1.5-day Workshop in Adelaide, Australia. This study attempts to capture the key points from the discussion and the perspectives of the various stakeholder groups, but is not a formal consensus statement.

Results: Treatment switching raises challenging ethical issues with arguments for and against allowing it. It is increasingly common in cancer drug trials and presents challenges for the interpretation of results by regulators, clinicians, patients, and payers. Proposals are offered for good practice in the design, management, and analysis of trials and wider development programs for cancer drugs in which treatment switching has occurred or is likely to. Recommendations are also offered for further action to improve understanding of the importance and challenges of treatment switching and to promote agreement between key stakeholders on guidelines and other steps to address these challenges.

Conclusions: The handling of treatment switching in trials is of concern to all stakeholders. On the basis of the discussions at the Adelaide International Workshop, there would appear to be common ground on approaches to addressing treatment switching in cancer trials and scope for the development of formal guidelines to inform the work of regulators, payers, industry, trial designers and other stakeholders.

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.

Objective: Renal replacement therapy (RRT) is essential for maintenance of life for those with end-stage renal disease. However, there remain many areas of uncertainty about which method of RRT should be chosen. This paper reports an economic model based on a systematic review that attempts to determine which method of dialysis, continuous ambulatory peritoneal dialysis (CAPD) or hemodialysis, a patient should have as the initial method of RRT.

Methods: A systematic review and a costing exercise carried out in a Scottish hospital were used to populate a Markov model. Scenario analysis was used to model plausible variations in variables included in the model.

Results: In 8 of the 16 scenarios developed hemodialysis dominated CAPD. In a further eight scenarios, the cost per life-year displayed considerable variability, ranging from between £5,000 to £51,000. The higher costs per life-year were associated with minimum estimates of additional survival for a patient starting RRT on hospital hemodialysis.

Conclusion: It may be more cost-effective to manage patients starting on RRT with hospital hemodialysis than CAPD. This has particular implications for the United Kingdom, given that up to 50% of new patients receive CAPD.

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.

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 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.

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.

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.

Objectives: To compare the cost-effectiveness of the treatment of ankylosing spondylitis (AS) with infliximab in the United Kingdom over lifetime estimated from two different clinical trials and adjusted for clinical practice guidelines.

Methods: A cost-effectiveness model was developed to incorporate clinical, epidemiological, and economic data and allow extrapolation of trial results and incorporation of long-term treatment. Assumptions regarding treatment beyond the trials were based on open extensions from the trials and treatment guidelines by the British Society for Rheumatology. Results are presented for both the societal perspective and the perspective of the National Health Service (UK £, discounted 3.5 percent).

Results: Under the assumption that disease activity would be controlled and functional capacity would remain stable while on drug, treatment with infliximab (5 mg/kg every 6 weeks) dominates standard treatment in the societal perspective. In the National Health Service perspective, the cost per quality-adjusted life-year (QALY) gained over lifetime was £28,300 and £26,800 for the two trials. If functional capacity were to deteriorate at half the rate of untreated patients, the cost per QALY gained would be £35,300 and £34,100, respectively. The results are sensitive to the dosing regimen adopted, the discontinuation rate, and assumptions concerning disease progression while on treatment.

Conclusions: The two clinical trials yield the same cost-effectiveness results and the cost per QALY gained with treatment was found to be in the acceptable range.

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.

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.

A colorimetric instrument for the noninvasive quantification of hemoglobin, designed using color shades resembling those observed in the conjunctiva, was tested. The instrument's colors are contrasted against the color of the conjunctiva to measure hemoglobin content. Sensitivity, specificity, negative predictive value, and false-negative value were estimated to test the instrument's accuracy; kappa coefficients were used to estimate inter- and intraobserver variability. Physician field evaluations of conjunctiva color for the screening of anemia, reported in the literature, have had sensitivities and specificities as high as 70%. Readings with the instrument demonstrated a 63% sensitivity, 72% specificity, and 38% false negatives for screening hemoglobin values of less than or equal to 13 g/dl. The interobserver kappa coefficients for three pairs of readers were good to excellent for the same hemoglobin screening value. Statistically significant differences were noted, however, between observers during the reliability test. The instrument can be used by unskilled personnel to improve their decision-making about whom to send for futher care or for supplementation with iron.

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.

Objectives: Evaluation is crucial for integration of e-Health/m-Health into healthcare systems and health technology assessment (HTA) could offer sound methodological basis for these evaluations. Aim of this study was to look for HTA reports on e-Health/m-Health technologies and to analyze their transparency, consistency and thoroughness, with the goal to detect areas that need improvement.

Methods: PubMed, ISI-WOS, and University of York – Centre for Reviews and Dissemination–electronic databases were searched to identify reports on e-Health/m-Health technologies, published up until April 1, 2016. The International Network of Agencies for Health Technology Assessment (INAHTA) checklist was used to evaluate transparency and consistency of included reports. Thoroughness was assessed by checking the presence of domains suggested by the European network for Health Technology Assessment (EUnetHTA) HTA Core Model.

Results: Twenty-eight reports published between 1999 and 2015 were included. Most were delivered by non-European countries (71.4 percent) and only 35.7 percent were classified as full reports. All the HTA reports defined the scope of research whereas more than 80 percent provided author details, summary, discussed findings, and conclusion. On the contrary, policy and research questions were clearly defined in around 30 percent and 50 percent of reports. With respect to the EUnetHTA Core Model, around 70 percent of reports dealt with effectiveness and economic evaluation, more than 50 percent described health problem and approximately 40 percent organizational and social aspects.

Conclusions: E-Health/m-Health technologies are increasingly present in the field of HTA. Yet, our review identified several missing elements. Most of the reports failed to respond to relevant assessment components, especially ethical, social and organizational implications.