In the wake of the UN Summit on non-communicable diseases (NCDs) (1), the subject has become a priority on most health agendas and scientific publications in this area have increased considerably over the past few years. NCDs are a threat, both for the sustainability of public health structures and for the wellbeing of the general population worldwide. Many risk factors have been identified as preventable and the determining role of lifestyle habits, such as physical exercise and food, in the pathogenesis of NCDs is now generally acknowledged.

Objectives: The aim of this study was to assess potential cost-effectiveness of using a prostate cancer specific functional imaging technology capable of identifying residual localized disease versus small volume metastatic disease for asymptomatic men with low but detectable prostate specific antigen (PSA) elevation following radical prostatectomy.

Methods: Markov modeling was used to estimate the incremental impact on healthcare system costs (2012 USD) and quality-adjusted life-years (QALYs) of two alternative strategies: (i) using the new diagnostic to guide therapy versus (ii) current usual care—using a combination of computed tomography, magnetic resonance imaging, and bone scan to guide therapy. Costs were based on estimates from literature and Medicare reimbursement. Prostate cancer progression, survival, utilities, and background risk of all-cause mortality were obtained from literature. Base-case diagnostic sensitivity (75 percent), specificity (90 percent), and cost (USD 2,500) were provided by our industry partner GE Healthcare.

Results: The new diagnostic strategy provided an average gain of 1.83 (95 percent uncertainty interval [UI]: 1.24–2.64) QALYs with added costs of USD 15,595 (95 percent UI: USD -6,330–44,402) over 35 years. The resulting incremental cost-effectiveness ratio was USD 8,516/QALY (95 percent UI: USD -2,947–22,372). Results were most influenced by the utility discounting rate and test performance characteristics; however, the new diagnostic provided clinical benefits over a wide range of sensitivity and specificity.

Conclusion: This analysis suggests a diagnostic technology capable of identifying whether men with biochemical recurrence after radical prostatectomy have localized versus metastatic disease would be a cost-effective alternative to current standard work-up. The results support additional investment in development and validation of such a diagnostic.

Objectives: The aim of this study was to estimate the cost-effectiveness of nebulized magnesium sulphate (MgSO4) in acute asthma in children from the perspective of the UK National Health Service and personal social services.

Methods: An economic evaluation was conducted based on evidence from a randomized placebo controlled multi-center trial of nebulized MgSO4 in severe acute asthma in children. Participants comprised 508 children aged 2–16 years presenting to an emergency department or a children's assessment unit with severe acute asthma across thirty hospitals in the United Kingdom. Children were randomly allocated to receive nebulized salbutamol and ipratropium bromide mixed with either 2.5 ml of isotonic MgSO4 or 2.5 ml of isotonic saline on three occasions at 20-min intervals. Cost-effectiveness outcomes were constructed around the Yung Asthma Severity Score (ASS) after 60 min of treatment; whilst cost-utility outcomes were constructed around the quality-adjusted life-year (QALY) metric. The nonparametric bootstrap method was used to present cost-effectiveness acceptability curves at alternative cost-effectiveness thresholds for either: (i) a unit reduction in ASS; or (ii) an additional QALY.

Results: MgSO4 had a 75.1 percent probability of being cost-effective at a GBP 1,000 (EUR 1,148) per unit decrement in ASS threshold, an 88.0 percent probability of being more effective (in terms of reducing the ASS) and a 36.6 percent probability of being less costly. MgSO4 also had a 67.6 percent probability of being cost-effective at a GBP 20,000 (EUR 22,957) per QALY gained threshold, an 8.5 percent probability of being more effective (in terms of generating increased QALYs) and a 69.1 percent probability of being less costly. Sensitivity analyses showed that the results of the economic evaluation were particularly sensitive to the methods used for QALY estimation.

Conclusions: The probability of cost-effectiveness of nebulized isotonic MgSO4, given as an adjuvant to standard treatment of severe acute asthma in children, is less than 70 percent across accepted cost-effectiveness thresholds for an additional QALY.

Objectives: The aim of this study was to evaluate the effectiveness of bilateral cochlear implantation (CI) compared with unilateral CI for deaf children in the context of the Republic of Kazakhstan health system.

Methods. A literature search was conducted, using the PubMed, Cochrane, and Embase data bases for studies that compared the effectiveness of bilateral and unilateral CI in children. The search included English language, publications from 2002–2012. Two reviewers independently evaluated all relevant studies. Administrative data relevant to CI in Kazakhstan were obtained from the Ministry of Health.

Results: Three relevant systematic reviews and an health technology assessment report were found. There was evidence of incremental benefits from bilateral CI but the quality of the available studies was poor and there was little information on longer term outcomes. No conclusions could be drawn regarding later incremental improvements to speech perception, learning, and quality of life. To date, in the Republic of Kazakhstan there is not full coverage of audiological screening due to the lack of medical equipment. This leads to late detection of hearing-impaired children and a long rehabilitation period, requiring more resources. Age of implantation in children is late and only a small minority attend general schools.

Conclusions: The clinical effectiveness of bilateral CI, an expensive health technology, requires further study. Given the current situation in Kazakhstan with audiological screening and access to unilateral CI, there appeared to be other priorities for improving services for children with profound hearing impairment.

Objectives: The aim of this study was to evaluate cost effectiveness of coronary artery bypass graft (CABG) versus percutaneous coronary intervention (PCI) with stenting from Iran society perspective.

Methods: A retrospective study was carried out to estimate the annual cost and health related quality of life (HRQoL) of 109 patients who underwent coronary revascularization (PCI [n = 75] and CABG [n = 34]). A Markov model has been developed to determine the cost effectiveness of CABG compared with PCI. We used the model to calculate lifetime costs, life-years (LYs), and quality-adjusted life-years (QALYs) of each strategy. We also used probabilistic sensitivity analysis to test model robustness.

Results: We found that discounted QALY lived per person in CABG versus PCI group in 5 years, 10 years, and lifetime time horizon were (3.8 ± 0.13 versus 3.88 ± 0.14), (6.4 ± 0.23 versus 6.33 ± 0.22), and (8.74 ± 0.29 versus 8.33 ± 0.27), respectively. The estimated medical cost of CABG and PCI per patient in 5 years, 10 years, and lifetime time horizon were (USD 6,819 ± 765 versus 9,011 ± 1,816), (USD 8,852 ± 1,348 versus 12,034 ± 2,375), and (USD 14,037± 4,201 versus 18,798 ± 5,821), respectively. The incremental cost-effectiveness ratio results showed CABG is a dominate alternative in 10 years and lifetime time horizon.

Conclusions: This study demonstrated that despite higher initial cost and lower HRQoL, CABG is a cost-effective revascularization strategy compared with PCI for patients with multivessel coronary artery disease in long-term.

Objectives: The E Sibling Project aims to address the needs of siblings of individuals affected by psychosis through provision of a comprehensive online intervention. The online intervention comprises four core elements, including: information on psychosis; various coping and promoting well-being strategies; siblings' blogs and discussion forum with peers; and an “Ask the Experts” function. After the intervention-prototype was developed, we tested its feasibility, usability and acceptability by siblings.

Methods: We evaluated the usability of the intervention-prototype using a non-randomised usability study with siblings of individuals diagnosed with psychosis. The usability study adapted Poulson et al's framework to collect subjective feedback from participants on ease of use, perceived usefulness and acceptability, together with objective usage data on the intervention.

Results: Twenty siblings were recruited to the usability test; 19 tried out the resource-prototype over a 4-week period and 17 completed the online evaluation after using the intervention. In total, 906 page-views were made by the participants and each spent about two hours using the resource. Participants evaluated the intervention as helpful, relevant and useful in terms of content, design and usability. Developments are needed to improve the navigation and intuitiveness of the resource.

Conclusions: Using an internet-based information-giving and peer support intervention to promote wellbeing and coping is feasible and acceptable to siblings of people with psychosis.

Objectives: The aim of this study was to examine the empirical and methodological cost-effectiveness evidence of surgical interventions for breast, colorectal, or prostate cancer.

Methods: A systematic search of seven databases including MEDLINE, EMBASE, and NHSEED, research registers, the NICE Web site and conference proceedings was conducted in April 2012. Study quality was assessed in terms of meeting essential, preferred and UK NICE specific requirements for economic evaluations.

Results: The seventeen (breast = 3, colorectal = 7, prostate = 7) included studies covered a broad range of settings (nine European; eight non-European) and six were published over 10 years ago. The populations, interventions and comparators were generally well defined. Very few studies were informed by literature reviews and few used synthesized clinical evidence. Although the interventions had potential differential effects on recurrence and mortality rates, some studies used relatively short time horizons. Univariate sensitivity analyses were reported in all studies but less than a third characterized all uncertainty with a probabilistic sensitivity analysis. Although a third of studies incorporated patients’ health-related quality of life data, only four studies used social tariff values.

Conclusions: There is a dearth of recent robust evidence describing the cost-effectiveness of surgical interventions in the management of breast, colorectal and prostate cancers. Many of the recent publications did not satisfy essential methodological requirements such as using clinical evidence informed by a systematic review and synthesis. Given the ratio of potential benefit and harms associated with cancer surgery and the volume of resources consumed by these, there is an urgent need to increase economic evaluations of these technologies.

Objectives: Vaccinating healthy children is proposed as a strategy to produce a herd effect and protect vulnerable groups. The Hutterite Influenza Prevention Study investigated this strategy, comparing communities with or without childhood influenza immunization programs. There are costs associated with vaccination therefore there may be a trade-off between these costs and the benefits of avoiding influenza cases. This evaluation estimates the cost-effectiveness of immunizing only healthy children in preventing cases of influenza within entire communities.

Methods: Effect data and resource utilization were collected during the trial. Cost data were collected from payer, literature and Internet sources. A two-stage bootstrap (TSB) with shrinkage correction was used to estimate average costs and effects. The incremental cost effectiveness ratio (ICER) and sample uncertainty around this estimate were calculated from the TSB results.

Results: Mean costs per patient for the treatment and control arms were $69.07 and $32.66 (difference $36.41). Mean number of influenza cases for the treatment and control arms were 0.04 and 0.27 (difference 0.23). ICER was $164.12 ($28.38, $2767.75) per case of influenza averted.

Conclusions: Immunizing healthy children for influenza is more costly, yet more effective than no immunization in preventing cases in the sample. At a cost of $164.12 to prevent a case of influenza, immunizing healthy children to protect all community members may be considered costeffective. Estimated results are conservative as the influenza season was mild and the sample population was healthy. In a more severe season with a less healthy population the ICER is expected to decrease.

Objectives: Real-time virtual sonography (RVS) is a promising navigation technique for percutaneous radiofrequency ablation (RFA) treatment, especially in ablating nodules poorly visualized on conventional ultrasonography (US). However, its cost-effectiveness has not been established. The purpose of this study is to evaluate the cost-effectiveness of RVS navigated RFA (RVS-RFA) relative to US guided RFA (US-RFA) in patients with small hepatocellular carcinoma (HCC) in China, from the modified societal perspective.

Methods: A state-transition Markov model was created using TreeAge Pro™ 2012. The parameters used in the model, including natural history of HCC patients, procedure efficacy and related costs, were obtained from a systematic search of literature through PubMed, EMBASE, and Science Citation Index databases. The simulated cohort was patients with solitary, small HCC (<3 cm in diameter) and Child-Pugh class A or B, whose tumors are poorly visualized in B-mode US but clearly detectable by CT or MRI.

Results: In this cohort of difficult cases, RVS-RFA was a preferred strategy saving 2,467 CNY ($392) throughout the patient's life while gaining additional 1.4 QALYs compared with conventional US guidance. The results were sensitive to the efficacy of US-RFA and RVS-RFA including complete ablation rate and local recurrence rate, the median survival for patients with progressive HCC, the probability of performing RFA for recurrent HCC, and the cost of RVS navigation, disposable needle or hospitalization.

Conclusions: RVS-RFA is a dominant strategy for patients with small HCC unidentifiable in B-mode US, in terms of cost savings and QALYs gained, relative to the conventional US-guided method.

Objectives: Mobile systems are widely adopted in healthcare services. Mobile reporting systems have been recently introduced for social home care by municipalities in Sweden. The study aims to assess the benefits of using these systems.

Methods: We followed an expert survey approach. Data were collected by means of telephone interviews with the experts in charge of managing and implementing the systems at the municipalities. In addition, several workshops were organized for assessing the economic value of the systems at one municipality. We performed thematic analysis and cost-benefit analysis of the data.

Results: The thematic analysis showed the three main benefits of using the mobile reporting systems in social home care: municipal benefits, care providers’ benefits, and care recipients’ benefits. The cost-benefit analysis indicated that the systems could bring substantial long-term economic value for municipalities. The results also revealed the difficulties encountered at the early stage of the deployment and implementation of the systems.

Conclusions: The mobile systems yield benefits for all the actors, that is, municipalities, care recipients and care providers. These enhance the public-private coordination and cooperation in social home care in Sweden. The municipalities are called upon to address change management and technical challenges in the implementation.

Objectives: Rare diseases are often heterogeneous in their progression and response to treatment, with only a small population for study. This provides challenges for evidence generation to support HTA, so novel research methods are required.

Methods: Discussion with an expert panel was augmented with references and case studies to explore robust approaches for HTA evidence generation for rare disease treatments.

Results: Traditional RCTs can be modified using sequential, three-stage or adaptive designs to gain more power from a small patient population or to focus trial design. However, such designs need to maintain important design aspects such as randomization and blinding and be analyzed to take account of the multiple analyses performed. N-of-1 trials use within-patient randomization to test repeat periods of treatment and control until a response is clear. Such trials could be particularly valuable for rare diseases and when prospectively planned across several patients and analyzed using Bayesian techniques, a population effect can be estimated that might be of value to HTA. When the optimal outcome is unclear in a rare disease, disease specific patient reported outcomes can elucidate impacts on patients’ functioning and wellbeing. Likewise, qualitative research can be used to elicit patients’ perspectives, with just a small number of patients.

Conclusions: International consensus is needed on ways to improve evidence collection and assessment of technologies for rare diseases, which recognize the value of novel study designs and analyses in a setting where the outcomes and effects of importance are yet to be agreed.

Background: The most effective sources to search to identify adverse effects data for medical devices are currently unknown.

Methods: The included studies from a systematic review of the safety of recombinant human bone morphogenetic protein-2 (rhBMP-2) for spinal fusion were used for analysis. For each source searched, a record was made for each relevant publication of whether it was retrieved by the search strategy used and whether it was available in the database but not retrieved. To account for multiple publications of the same study, a record was made of the relevant studies identified. The sensitivity, precision, and number needed to read were calculated as well as the minimum combination of sources to identify all the publications or studies.

Results: There were eighty-two publications (forty-nine studies) included in the systematic review. Only one article was available in a database searched but not retrieved by our search strategy. Science Citation Index (SCI) and EMBASE both achieved the highest sensitivity (62 percent), followed closely by MEDLINE/PubMED (56 percent). With the search strategies used, the minimum combination of sources needed to identify all the publications was SCI, EMBASE, CENTRAL, and either MEDLINE or PubMED, in addition to reference checking, contacting authors and an automated current awareness service. In relation to identifying all the relevant studies, the minimum combination of studies was similar with the exclusion of CENTRAL.

Conclusions: To identify all the relevant publications or studies included in this case study systematic review, several different sources needed to be searched.

Objectives: A mapping exercise was undertaken to determine how HTA is being described and conducted across the International Network of Agencies for Health Technology Assessment (INAHTA), with the aim of harmonizing terminologies and approaches.

Methods: Three progressive surveys were undertaken. In 2010, INAHTA agencies were asked to provide details on all of their HTA products. In 2013, additional information was sought on key methodological characteristics of five of the most common HTA product types. Subsequently, final agreement was sought on three proposed product types.

Results: Forty-five HTA agencies responded to at least one of the surveys. In 2010, twenty-one agencies reported publishing over seventy named HTA products. Core domains associated with full HTA reports were reported by a third of agencies but were labeled differently, so products were classified according to product type (n = 17). Agencies producing short, tailored products increased between 2010 and 2013, with the publication of rapid reviews doubling from 33 percent to 66 percent. In 2013, half of the agencies adapted their common HTA products from documents produced by other agencies. A consensus (>70 percent) was achieved on definitions for HTA reports, mini-HTAs, and rapid reviews.

Conclusions: The product label for an HTA is not always indicative of its content. Terminology has, therefore, been agreed to make explicit the trade-off between rigor and timeliness in three common HTA product types. An INAHTA Product Type (IPT) Mark has been created to identify each of these. It is hoped this will further facilitate HTA adaptation between agencies and reduce duplication of effort.

Objectives: The aim of this study was to compare Bayesian methods with the standard methods that are used for evidence-based policy making.

Methods: We performed a Bayesian reanalysis of the data underlying a reimbursement advice by the Dutch National Health Insurance Board (CVZ) regarding the anti-diabetic drug exenatide (an alternative to insulin). We synthesized evidence from various sources that was available when the CVZ advice was drafted: expert opinion (as elicited from internists), experimental data (from direct comparison studies), and observational data. Subsequently, the original frequentist results and the results from the Bayesian reanalysis were compared in terms of outcomes and interpretations. These results were presented in a meeting with staff from CVZ, whose opinions about the usefulness of a Bayesian approach were assessed using a questionnaire.

Results: The Bayesian approach yields outcomes that summarize different pieces of evidence, which would have been difficult to obtain otherwise. Moreover, there are conceptual differences, and the Bayesian approach allows for determining probabilities of clinically relevant differences. The staff at CVZ were fairly positive with respect to the use of Bayesian methods, although practical barriers were also seen as important.

Conclusions: The Bayesian outcomes are different and could be more suited to the informational needs of policy makers. The response from staff at CVZ provides some support for this statement, but more research at the interface of science and policy is needed.

Rationale: Bayesian methods provide an interesting approach to assessing an implantable medical device (IMD) that has evolved through successive versions because they allow for explicit incorporation of prior knowledge into the analysis. However, the literature is sparse on the feasibility and reliability of elicitation in cases where expert beliefs are used to form priors.

Objectives: To develop an Internet-based method for eliciting experts’ beliefs about the success rate of an intracranial stenting procedure and to assess their impact on the estimated benefit of the latest version.

Study Design and Setting: The elicitation questionnaire was administered to a group of nineteen experts. Elicited experts’ beliefs were used to inform the prior distributions of a Bayesian hierarchical meta-analysis model, allowing for the estimation of the success rate of each version. RESULTS: Experts believed that the success rate of the latest version was slightly higher than that of the previous one (median: 80.8 percent versus 75.9 percent). When using noninformative priors in the model, the latest version was found to have a lower success rate (median: 83.1 percent versus 86.0 percent), while no difference between the two versions was detected with informative priors (median: 85.3 percent versus 85.6 percent).

Conclusions: We proposed a practical method to elicit experts’ beliefs on the success rates of successive IMD versions and to explicitly combine all available evidence in the evaluation of the latest one. Our results suggest that the experts were overoptimistic about this last version. Nevertheless, the proposed method should be simplified and assessed in larger, representative samples.

Objectives: The aim of this study was to analyze the experience of the National Institute for Health and Care Excellence (NICE) in identifying new procedures entering the United Kingdom (UK) healthcare system, for assessment and publication of recommendations on their use. This system is designed to provide guidance in an area where regulation is lacking worldwide.

Methods: Retrospective analysis of all procedures notified to the Interventional Procedures Programme (NICE) between 2002 and 2012. Notifications were analyzed year by year for their source (who notified them), clinical specialties involved, and whether guidance was subsequently published.

Results: A total of 1,094 procedures were notified by clinicians (51 percent), and by others, including hospitals (6 percent), horizon scanners (5 percent), patients (4 percent), private health insurers (4 percent), and medical device manufacturers (3 percent). Guidance was published on 44 percent of procedures notified to the program. There was a decrease in the numbers of procedures notified during 2003–2012 (p = .049). There were notifications across all specialties, with the largest numbers in general surgery (125), urology (104), orthopedics (99), interventional radiology (93), cardiology (82), and obstetrics and gynecology (82).

Conclusions: The “open” NICE Web portal allows anyone to notify new procedures, aiming to maximize the opportunity of identifying all those procedures entering clinical practice. This has resulted in identification of large numbers of procedures from across the whole range of medical specialties. The fact that similar proportions of procedures notified from diverse sources have been selected for assessment and publication of practice recommendations suggests that this inclusive approach is worthwhile.

Objectives: The aim of this study was to determine the prevalence and methods of expert knowledge elicitation (EKE) for specifying input parameters in health economic decision models (HEDM).

Methods: We created two samples using the National Health System Economic Evaluations Database: (1) 100 randomly selected HEDM studies to determine prevalence of EKE and (2) sixty studies using a formal EKE process to determine methods used.

Results: Fifty-seven (57 percent) of the random sample included at least one EKE-derived parameter. Of these, six (10 percent) used a formal expert process. Thirty-four studies from our second sample of sixty studies (57 percent) described at least one aspect of the process (e.g., elicitation method) with reasonable clarity. In approximately two-thirds of studies the external experts estimated parameters de novo; the remainder confirmed or modified initial estimates provided by authors, or the method was unclear. The majority of elicitations obtained point estimates only, although a few studies asked experts to estimate ranges of parameter values.

Conclusions: The use of EKE for parameter estimation is common in HEDMs, although there is room for improvement in the methods used.

Objectives: The aim of this study was to determine the economic costs and the level of catastrophic health expenditure (CHE) due to childhood epilepsy.

Methods: The study was conducted at the Paediatric Neurology Clinic of the University of Nigeria Teaching Hospital, Enugu. Data were collected using pre-tested questionnaires that were administered to caregivers of the children. The indirect and direct expenditure due to childhood epilepsy were computed. A 40 percent of monthly non-food expenditure was used to estimate CHE.

Results: The average annual direct and indirect expenditures were USD 162.6 and USD 82.3, respectively. Most of direct costs were drugs (25.4 percent versus 35.3 percent) and investigations (48.7 percent versus 61.3 percent) for out-patient and in-patient, respectively. CHE was 34.1 percent and 63.6 percent for out-patient and in-patient care, respectively. The total annual costs: (direct and indirect), for childhood epilepsy of USD244.9. Considering the estimated 190,000 epileptic children in Nigeria, it will amount to USD46.53 million annually, approximately 0.018 percent of Nigeria Gross Domestic Product (GDP). All payments were made out-of-pocket with no health insurance for financial risk protection.

Conclusions: The cost of treatment of childhood epilepsy is high and catastrophic for many households. There was lack of usage of health financial risk mechanisms. Scale-up use of health financial risk protection mechanisms such as health insurance can reduce the economic burden.