Objectives: The aim of this study was to examine the impact of the National Institute for Health and Clinical Excellence's (NICE's) negative and restricting technology appraisals on the number of prescription items dispensed and the corresponding total net ingredient costs for drugs from 2000 to 2004 in the ambulatory care of the National Health Service (NHS) in England and Wales. In addition, it is discussed whether the NICE approach could be a role model for Germany.

Methods: The number of prescription items dispensed and the net ingredient costs of thirty-one drugs reimbursed by the NHS were analyzed, thereof thirteen drugs descriptively and twenty-one drugs with regression analyses. Data were extracted from the “Prescription-Costs-Analysis-Statistics” for the ambulatory care of the British Department of Health (England 1993–2005). In the case of the twenty-one drugs analyzed by regression analyses, predictions were established how the prescribing and the costs would have developed without NICE's drug appraisal. Finally, conclusions were drawn whether NICE's negative and restricting drug appraisals had a decreasing effect or not.

Results: For 97 percent of the drugs analyzed in this study, the publication of NICE's fourteen negative and restricting technology appraisals of drugs between 2000 and 2004 did not reduce the number of prescription items dispensed and net ingredient costs in the ambulatory care of the NHS in England and Wales.

Conclusions: Cost-effectiveness appraisals as performed by NICE or the German Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, IQWiG) are a useful and important tool to enhance the discussion about methods and acceptance of evidence-based medicine in general.

Objectives: The aim of this study was to examine the availability and consistency of economic evidence for the detection, diagnosis, treatment, and follow-up of colorectal cancer.

Methods: A systematic review of UK economic evaluations of colorectal cancer interventions was undertaken. Searches were undertaken across ten electronic databases. Studies were critically appraised through reference to a conceptual model of UK colorectal cancer services.

Results: Forty-seven studies met the inclusion criteria. There is a substantial economic evidence base surrounding population-level colorectal screening, surgical procedures, and cytotoxic therapies for the adjuvant and palliative treatment of colorectal cancer. There is limited evidence concerning the diagnosis of suspected colorectal cancer, curative treatments for metastatic disease and follow-up regimens for nonmetastatic disease. No studies were identified relating to the economics of radiotherapy, surveillance of increased-risk groups, end-of-life care, or the management of hereditary colorectal cancer. Where evidence is available, studies are subject to important differences concerning treatment options, decision criteria, and incongruent assumptions concerning the disease and its management.

Conclusions: Across many aspects of the colorectal cancer service, current practice appears to have emerged without the consideration or support of economic evidence. There is a need to develop a common understanding how colorectal cancer models should be structured and implemented.

The 1970s and early 1980s was the period in which high technology medicine became dominant. It had begun in the late 1950s with the introduction of effective artificial respiratory technology. This gave physicians, patients, and families the hope that the threats to fundamental life processes such as breathing could be countered by technology, and the dilemma of both meeting its costs and the ethical challenges of how to remove it when its use no longer produced benefits.

Objectives: An important option for the medical treatment of obstructive sleep apnea-hypopnea syndrome (OSAHS) is continuous positive airway pressure (CPAP) during sleep. This study reports on the cost-effectiveness of CPAP compared with dental devices and lifestyle advice. The work was commissioned by the NHS HTA Programme to inform the National Institute of Health and Clinical Excellence's (NICE) appraisal of CPAP.

Methods: A Markov model compared the interventions over the expected patient lifetime. The primary measure of cost-effectiveness was the incremental cost per quality-adjusted life-year (QALY) gained. The QALY incorporated the impact of treatments on daytime sleepiness, blood pressure and health-related quality of life (HRQoL).

Results: On average, CPAP was associated with higher costs and QALYs compared with dental devices or lifestyle advice. In the base-case analysis, the incremental cost-effectiveness ratio (ICER) for CPAP compared with dental devices was around £4,000 per QALY (2005–06 prices). The probability that CPAP is more cost-effective than dental devices or lifestyle advice at a threshold value of £20,000 per QALY was 0.78 for men and 0.80 for women. Several sensitivity analyses were undertaken and it was found that the ICER for CPAP consistently fell below £20,000 per QALY gained, apart from in a subgroup with mild disease.

Conclusions: The model suggests that CPAP is cost-effective compared with dental devices and lifestyle advice for adults with moderate or severe symptomatic OSAHS at the cost-effectiveness thresholds used by NICE. This finding is reflected in the NICE guidance.

Objectives: Clear, transparent, and sufficiently detailed abstracts of journal articles and conference abstracts are important because readers often base their assessment of a trial on such information. There are concerns over the reliability and quality of trials published only in the proceedings of scientific meetings. This study aims to assess the reporting quality of abstracts of randomized trials published in Chinese medical conference abstracts.

Methods: Conference abstracts reporting randomized trials included in the China National Knowledge Infrastructure (CNKI) in 2007 were identified. A revised checklist (based on the CONSORT extension for reporting randomized controlled trials in journal and conference abstracts) was used to assess the reporting quality of these conference abstracts.

Results: A total of 567 conference abstracts of randomized trials were identified. Some aspects were well reported, including 94 percent of authors contact details, 83 percent of trial interventions and 78 percent of control interventions, 62 percent of participant eligibility criteria, and 66 percent the number of participants randomized to each group. Other areas were very poorly reported: only 1 percent identified the study as randomized in the abstract title, 2 percent reported the trial design, and only 7 percent reported on blinding. No details of allocation concealment, trial registration, or funding were reported.

Conclusion: The information given for trials in conference proceedings in China is very poor, especially in some aspects of methodological quality, trial registration, and funding source. The quality of conference abstracts for trials should be improved to further facilitate understanding of their conduct and validity.

The International Society for Technology Assessment in Health Care (ISTAHC) was formed in 1985. It grew out of the increasing awareness of the international dimensions of health technology assessment (HTA) and the need for new communication methods at the international level. The main function of ISTAHC was to present an annual conference, which gradually grew in size, and also to generally improve in quality from to year. ISTAHC overextended itself financially early in the first decade of the 2000s and had to cease its existence. A new society, Health Technology Assessment international (HTAi), based on many of the same ideas and people, grew up beginning in the year 2003. The two societies have played a large role in making the field of HTA visible to people around the world and providing a forum for discussion on the methods and role of HTA.

Objectives: Adapting health technology assessment (HTA) reports for different contexts could reduce the need for multiple reports on the same health technology with resultant saving of time and resources. This article describes an instrument, the adaptation toolkit, which has been developed to aid in the process of adaptation of HTA reports.

Methods: The toolkit was developed by a partnership of HTA agencies and networks from across Europe. The role of the toolkit is to guide the user through the process of selecting possible relevant material from these report(s), assessing the relevance, reliability, and transferability of the material, and adapting it for the desired context.

Results: The adaptation toolkit has been developed, it comprises a collection of resources that help the user assess whether data and information in existing HTA reports should and could be adapted for their own setting. The toolkit contains two sections: a preliminary speedy sifting section and the main toolkit. The main toolkit includes five domains: (i) technology use and development, (ii) safety, (iii) effectiveness (including efficacy), (iv) economic evaluation, and (v) organizational aspects. Legal, ethical, and social aspects are beyond the scope of the toolkit. The toolkit is designed for the adaptation of evidence synthesis rather than primary research.

Conclusions: The completed current version of the toolkit contains checklists and resources to aid in the adaptation of HTA reports. This collection of resources is available for use by all HTA agencies and can be accessed at: http://www.eunethta.net/upload/WP5/EUnetHTA_HTA_Adaptation_Toolkit_October08.pdf..

Background: The concept of using public funds to pay for healthcare interventions only when provided in the context of ongoing research is receiving increasing attention worldwide. Nevertheless, these decisions are often controversial and implementation can be problematic.

Objectives: The aim of this study was to investigate the views of United Kingdom stakeholders on the current arrangements for implementing “only in research” (OIR) decisions and to investigate how improvements might be made.

Methods: After an internal review of previous OIR decisions issued by the National Institute for Health and Clinical Excellence (NICE), deliberations by NICE's Citizens Council, and an international workshop convened by NICE and the United States Agency for Healthcare Research and Quality, thirteen key stakeholders and experts from academia, industry, government, and the National Health Service (NHS) were interviewed using a semistructured interview guide. Interview transcripts were subjected to a framework-based analysis using computer-assisted qualitative data analysis software.

Results: All interviewees endorsed the use of the OIR option. There was a high degree of consensus for several suggestions regarding how the use of the OIR option might be improved. For example, there was universal agreement that a formal process should be established to prioritize research needs arising from OIR decisions and that funds for publicly funded research projects should be channeled in a manner that would better motivate healthcare providers to participate in OIR-related research.

Conclusions: The findings of this study suggest several potential modifications of the OIR pathway in the United Kingdom and may also be helpful to health technology assessment agencies in other countries that already use or are considering using an OIR-like option to reduce the uncertainty inherent in health technology assessment.

Objectives: Schizophrenia imposes a great burden on society, and while evaluation should play an important role in informing society's efforts to alleviate these burdens, it is unclear what “endpoints” should be chosen as the objective of such analyses. The objectives of the study were to elicit endpoints directly from patients with schizophrenia, to ascertain whether patients are sufficiently cognoscente to express what endpoints are and are not important to them and to rank the relevant endpoints.

Methods: We applied principles of patient-centered health technology assessment to identify and value endpoints from the patient's perspective. Focus groups were conducted to elicit endpoints, using interpretive phenomalogical analysis (IPA) to guide the collection, analysis and interpretation of data. Patient interviews were subsequently used to elicit patient preference over endpoints. Respondents were presented with cards outlining the endpoints and asked to remove irrelevant cards. They where then asked to identify and rank their five most relevant endpoints in order of importance. Interviews were recorded for the purposed of triangulation, and data was analyzed using descriptive statistics. Patients were recruited from five geographically diverse cities in Germany. Eligibility required a diagnosis of schizophrenia by a physician and treatment with an antipsychotic medication for at least one year. Respondents were excluded if they were experiencing an acute episode.

Results: Thirteen endpoints emerged as important from the focus groups spanning side-effects, functional status, processes of care and clinical outcomes. Respondents could clearly identify relevant and irrelevant endpoints, and rank which factors were important to them. Triangulation between field notes of the ranking exercise and recordings confirmed that rankings were not arbitrary, but justified from the respondents' point of view.

Conclusions: Patients with schizophrenia can express preferences over endpoints. Our results show that qualitative methods such as IPA can be used to identify factors, but ranking exercises provide a more robust method for ranking the importance of endpoints. Future research involving patients with schizophrenia ranking outcomes is needed to identify variations across patients and methods such as conjoint analysis could prove beneficial in identifying acceptable tradeoffs across endpoints.

Objectives: Implantable cardioverter defibrillators (ICDs) are an effective but expensive treatment for the prevention of sudden cardiac deaths in patients with severe left-ventricular dysfunction. Recent studies suggest that microvolt T-wave alternans (MTWA) predicts mortality and severe arrhythmic events in this population. However, the impact of MTWA on ICD cost-effectiveness is unknown.

Methods: A Markov decision-analysis model evaluated three treatment strategies for primary prevention in patients with severe left-ventricular dysfunction: (i) medical therapy for all; (ii) ICD therapy for all; and (iii) selective ICD therapy based on non-negative (positive or indeterminate) MTWA test results. Incremental cost-effectiveness ratios (ICER) were calculated from the perspective of a third party payer using a 10-year time horizon. Sensitivity analyses examined the robustness of the estimates.

Results: A treatment strategy involving ICD therapy in all patients was associated with an ICER of $121,800/quality-adjusted life-year (QALY) compared with medical therapy, whereas a treatment strategy involving the selective use of ICDs based on MTWA test results was associated with an ICER of $108,900/QALY compared with medical therapy. Sensitivity analyses suggest that, under most scenarios, the selective use of ICDs based on MTWA results does not decrease the ICER to below $100,000/QALY.

Conclusion: MTWA only marginally improves the cost-effectiveness of ICDs for primary prevention in patients with severe left-ventricular dysfunction. There remains a need for improved means to effectively identify which patients will derive the greatest benefit from ICD implantation.

Objectives: Mini-HTA (health technology assessment) is increasingly being applied in Denmark as an input for decisions on the use of health technologies. Mini-HTA is a form or check list with questions concerning the prerequisites for and consequences of health technologies. At the national level, the National Board of Health uses mini-HTA when hospitals apply for permission to introduce new treatments. Mini-HTA is also compulsory in Danish Regions' annual collection of early warnings. At the local level some hospitals have made mini-HTA compulsory when clinical departments apply for funding for new technologies. The objective of this study is to assess the quality of the information included in mini-HTA used at Danish hospitals and to discuss the consequences of this to decision making.

Methods: The quality of mini-HTA is assessed by use of an INATHA checklist for HTA reports. Data consists of reviews of the quality in fifty-two mini-HTAs produced by Danish hospitals in 2008.

Results: The mini-HTAs generally include descriptions of the assessed technology and the comparator, but information about the selection and interpretation of the clinical literature and other data is often missing. The level of evidence for the clinical effects and the main references are generally included. Only 25 percent of the mini-HTAs include a quantitative estimate of the size of the clinical effects. Organizational consequences inside the clinical department is described in 81percent of the cases and 92 percent includes a cost estimate.

Conclusions: The results show that the quality of the information in many cases is insufficient. There is a strong need for quality assurance of mini-HTAs to improve the accuracy of the information, however, without harming the timeliness and the limited use of resources in producing the reports.

Objectives: The cost-effectiveness of adding a human papillomavirus (HPV) vaccination program in 12-year-old females to the recommended cervical cancer screening in Belgium is examined. Moreover, the health and economic consequences of a potential decline in screening uptake after initiation of a HPV vaccination program are investigated.

Methods: A static Markov model is developed to estimate the direct effect of vaccination on precancerous lesions and cervical cancers.

Results: Vaccination is estimated to avoid 20 percent of the cervical cancers occurring in a 12-year-old girls' cohort and to cost €32,665 per quality-adjusted life-year (QALY) gained (95 percent credibility interval [CrI]: €17,447 to €68,078), assuming a booster injection after 10 years, a limited duration of protection and discounting costs and effects at 3 percent and 1.5 percent, respectively. Assuming lifelong protection, HPV vaccination is estimated to cost €14,382 (95 percent CrI: €9,238 to €25,644) per QALY gained, while avoiding 50 percent of the cervical cancer cases. In the base-case, a 10 percent reduction in screening compliance after vaccination obliterates the effect of vaccination on cervical cancer cases avoided, whereas further declines in the level of screening compliance even turned out to be detrimental for the cohort's health, inducing a mean loss in QALYs and life-year gained compared with the situation prevaccination.

Conclusions: An HPV vaccination program should only be considered if the level of screening after vaccination can be maintained.

Objectives: To describe the development of the International Network of Agencies for Health Technology Assessment (INAHTA) and its activities.

Methods: Review of literature material and other documents produced by or relating to INAHTA.

Results: INAHTA includes organizations that provide health technology assessment (HTA) advice to governments and receive most of their funding from public sources. In early 2009, there were forty-six members from twenty-seven countries, including both national and regional agencies. Interaction with other organizations includes links to Health Technology Assessment International (HTAi), the European Union Network for Health Technology Assessment (EUnetHTA), PAHO (the Pan American Health Organization, and the World Health Organization (WHO). An important feature of INAHTA is the routine interaction of its members with public sector decision makers. Output from the network has included guidelines and frameworks on HTA, reports of surveys, and joint projects.

Conclusions: INAHTA has developed as a global point of contact and information for those with interests in HTA.

Objectives: The aim of this study was to assess current evidence supporting the use of percutaneous heart valves (PHV) in degenerative aortic valve and congenital pulmonary outflow tract disease, as compared to conservative medical therapy or traditional surgical valve replacement.

Methods: A systematic review of the literature on PHV was performed.

Results: No randomized controlled trials (RCT) on PHV have been published so far. Only observational data from series and data presented at cardiology meetings are available. Both percutaneous aortic valve (PAV) and percutaneous pulmonary valve (PPV) seem feasible in the hands of an experienced team. Safety, however, seems to be a problem in PAV, as shown by the high 30-day and 6-month mortality rates.

Conclusions: Due to safety concerns, PAV reimbursement is not recommended and patients should only be subjected to PAV insertion within the boundaries of an RCT. In contrast, PPV implantation seems to be a safe and promising technology for which reimbursement under strict conditions may be recommended.

Background: The classic cut and sew surgical Maze procedure has been shown to reduce atrial fibrillation (AF), and recently newer surgical ablation techniques with reduced technical complexity have been introduced. This study sought to systematically review the efficacy and safety of these newer techniques and to evaluate their long-term cost-effectiveness at the time of scheduled mitral valve (MV) surgery.

Methods: A systematic literature search and meta-analysis was performed to generate the most reliable efficacy and safety parameter estimates for a Markov decision analysis model comparing MV surgery alone to MV surgery plus an ablation Maze procedure. Both basic and probabilistic sensitivity analyses were conducted.

Results: Based on the six randomized controlled trials (RCTs) identified, the pooled 1-year estimate of AF after surgery alone was 71 percent (95 percent confidence interval [CI], 64 percent to 78 percent). The pooled risk ratio of AF after surgical ablation treatment at the time of mitral valve surgery relative to valve surgery alone was 0.33 (95 percent CI, 0.19 to 0.55). The pooled analyses showed that no statistical significant increases in operative mortality, permanent pacemaker implantation, and postoperative bleeding with the ablative Maze procedures. An ablation-based Maze procedure at the time of mitral valve surgery had an incremental cost-effectiveness ratio (ICER) of $4,446CAD ($3,850US) per quality-adjusted life-year (QALY) compared with valve surgery alone. Specifically costs were an extra $900CAD but with improved clinical outcomes (0.20 QALYs), including a prolonged life expectancy of 0.10 life-years. In one-way sensitivity analyses, survival time after MV surgery had the largest impact on ICER. Other variables influencing the ICER included the risk ratio of AF, utility, and cost estimates. Probabilistic sensitivity analysis suggests that 58.1 percent, 73.9 percent, and 89.3 percent of the simulations of the Maze strategy are cost-effective at willingness to pay thresholds of $20,000, $50,000, and $100,000CAD per QALY gained, respectively.

Conclusions: Our meta-analysis suggests that a Maze surgical ablation procedure at the time of MV surgery is associated with a reduced postoperative AF risk. Our economic model further suggests that the surgical ablation strategy at the time of mitral valve surgery is likely a cost-effective intervention, provided patients have a good long-term postsurgical prognosis.

Background: The way people use health technology assessment (HTA) terms varies considerably across Europe. Such variation can lead to misunderstandings when reading HTA reports from different contexts. This work is one of the outputs of the EUnetHTA Project and was undertaken between 2006 and 2008.

Objectives: The aim of this study was to develop a glossary of HTA adaptation terms to help reduce the misunderstandings of terms used in HTA reports from contexts other than the reader's own.

Methods: Several HTA glossaries were examined to identify ways in which an additional glossary could offer readers something new and to identify adaptation terms for inclusion. Twenty-eight European HTA organizations provided terms for the glossary and drafted descriptions and examples of how each specific term was used in their particular setting. The organizations then commented on the descriptions provided by the other groups and worked together to draft a single description for certain terms.

Results: A glossary of HTA adaptation terms was developed. It provides a comprehensive range of descriptions, examples, and comments for forty-two potentially confusing HTA terms related to adaptation.

Conclusions: This glossary will be a valuable resource for European HTA agencies when reading HTA reports produced in different contexts and for adapting HTA reports produced in other countries. The glossary will help improve understanding and help facilitate the adaptation process.

Objectives: EUnetHTA WP 7 (Strand B) aimed to promote sharing information on new and emerging technologies. The task was to develop a prototype of a newsletter and pilot the processes of production.

Methods: The EuroScan database served as information source on pertinent technologies. To prioritize, a set of criteria for scoring the potential impact and for selecting the technologies for articles was applied and a pilot newsletter was produced.

Results: Being objective and transparent about the content of a newsletter required a method for prioritizing health technologies. Using significance criteria, members of the prioritization panel selected twelve technologies for articles of different length and depth. Potential recipients, surveyed on relevance, content, timeliness, and readability responded mostly positive, but requested more information on cost effectiveness and criticized timeliness.

Conclusions: Dissemination of an EU-wide newsletter would be feasible, but time-consuming. Although a newsletter appears to fulfill a need for information on emerging and new health technologies, it is not considered the right tool to avoid duplication of effort in the present international constellation of horizon scanning for new health technologies. Other options will be pursued as part of future collaborative actions, for example, a core set of early awareness information, or an on-demand electronic information system.

Objectives: Depression is associated with high healthcare utilization and related costs. Effective treatments might reduce the economic burden. The objective of this study was to establish the cost-utility of a minimal psychological intervention (MPI) aimed at reducing depression and improving quality of life in elderly persons with diabetes or chronic obstructive pulmonary disease and co-occurring minor, mild, or moderate depression.

Methods: Trial-based cost-utility analysis was used to compare the MPI with usual care. Annual costs and quality-adjusted life-years (QALYs) based on the Euroqol (EQ5D) and on depression-free days were calculated.

Results: Annual costs and effects were not significantly different for the MPI group and care as usual. Bootstrap analysis indicated a dominant intervention, with a probability of 63 percent that the MPI is less costly and more effective than usual care.

Conclusions: The cost-effectiveness analysis does not support dissemination of the MPI in its current form. The economic evaluation study showed limited probability that MPI is cost-effective over usual care. Further adjustments to the MPI are needed to make the intervention suitable for dissemination in regular care. Trial registration: isrctn.org, identifier: ISRCTN92331982.

The U.S. Congressional Office of Technology Assessment (OTA) established a health program in 1975. During the next few years, OTA's health program published a series of reports dealing with different aspects of health technology assessment (HTA) in some depth. The key report in this series concerned the efficacy and safety of health technology, which in many ways played a ground-breaking role. It pointed out the pervasive lack of accessible information on efficacy and safety, despite more-than-adequate methods of assessment. It also pointed to many problems that resulted from this lack, and the limited use of such information in clinical practice and policy making. It promoted synthesis of existing literature as a practical method of assessment. Other key reports developed other aspects of HTA, including cost-effectiveness. These reports are generally considered to have shaped the field of HTA at least into the 1990s. OTA also pioneered the use of HTA in determining what preventive services to cover in public healthcare programs.

Objectives: This study assesses whether pegylated interferon and ribavirin is cost-effective compared with no antiviral treatment provided in routine clinical practice, for different patient subgroups.

Methods: The cost-effectiveness analysis (CEA) uses a Markov decision model to estimate the lifetime cost per quality-adjusted life-year (QALY) of antiviral treatment compared with no treatment. The model is populated with data on sustained virological responses, costs, and transition probabilities all taken from a large representative sample of UK cases and centers (Trent HCV database).

Results: The CEA found that pegylated interferon and ribavirin was cost-effective for most patient subgroups. The CEA found that for patients with genotype non-1, the intervention led to cost reductions and gains of at least 0.5 QALYs. For genotype 1 cases with mild or moderate disease, and younger cirrhotic patients (aged 40 or less), costs per QALY remained below £20,000 ($40,000 or €29,000). For genotype 1 cases with cirrhosis aged 50, the mean cost per QALY rose to over £60,000 ($120,000 or €87,000).

Conclusions: The study concludes that, based on cost and effectiveness data collected from routine clinical practice, treatment with pegylated interferon and ribavirin is generally cost-effective. The study shows that there are variations according to patient subgroup and for older (aged 50 or over) genotype 1 patients with cirrhosis, antiviral treatment appears less cost-effective.