Objectives: The aim of this study was to establish a list of priority topics for methods research to support decision making at the National Institute for Health and Clinical Excellence (NICE).

Methods: Potential priorities for methods research topics were identified through a focused literature review, interviews, an email survey, a workshop and a Web-based feedback exercise. Participants were members of the NICE secretariat and its advisory bodies, representatives from academia, industry, and other organizations working closely with NICE. The Web exercise was open to anyone to complete but publicized among the above groups.

Results: A list of potential topics was collated. Priorities for further research differed according to the type of respondent and the extent to which they work directly with NICE. Priorities emerging from the group closest to NICE included: methodology for indirect and mixed treatment comparisons; synthesis of qualitative evidence; research relating to the use of quality-adjusted life-years (QALYs) in decision making; methods and empirical research for establishing the cost-effectiveness threshold; and determining how data on the uncertainty of effectiveness and cost-effectiveness data should be taken into account in the decision-making process. Priorities emerging from the broadest group of respondents (through the Web exercise) included: methods for extrapolating beyond evidence observed in trials, methods for capturing benefits not included in the QALY and methods to assess when technologies should be recommended in the context of further evidence gathering.

Conclusions: Consideration needs to be given to the needs of those who use the outputs of research for decision making when determining priorities for future methods research.

Objectives: The aim of this study was to evaluate the extent of reporting necessary information in published health economic research in World Health Organization Eastern Mediterranean Countries (WHO EMC).

Methods: A systematic literature search was conducted using PubMed and Google Scholar to identify pharmacoeconomic studies conducted in WHO EMC. The inclusion criteria for the studies were: (i) original studies, (ii) compared pharmaceutical services or drugs, (iii) conducted in WHO EMC, (iv) manuscript published in English. The articles were reviewed by two independent reviewers using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist.

Results: A total of seventeen studies were included, each of which were published in seventeen different journals. The mean CHEERS checklist score was 16 ± 4. Most studies were published in journals outside WHO EMC (n = 12; 71 percent). Cost-effectiveness (n = 5; 29 percent) and cost-utility analyses (n = 5; 29 percent) were the most frequently used methods of economic evaluation.

Conclusions: Pharmacoeconomic studies in WHO EMC are limited and sometimes incomplete. Economic evaluation of pharmaceuticals should be encouraged in WHO EMC to ensure the appropriate allocation of healthcare resources.

Objectives: Evidence-based guideline committees are multidisciplinary and explicitly consider the existing evidence. They are thus in an ideal position to identify research gaps. However, gaps have not been systematically identified through guidelines. We pilot tested a method to systematically identify and classify gaps from evidence-based guidelines.

Methods: We reviewed all evidence-based guidelines published by the Cystic Fibrosis Foundation. We identified research gaps as topics for which there was insufficient evidence (recommendations were not made or consensus recommendations were made) and topics specified as needing further research. We characterized gaps using a standard framework and classified them by type of management issue, specificity of target population, and age of target population.

Results: We identified sixty-two research gaps in five guidelines (mean = 12.4/guidelines document). While thirteen gaps were topics specified as needing further research, most (n = 49) were topics with insufficient evidence. Of these forty-nine, recommendations were not made for twenty-two topics while consensus recommendations were made for twenty-seven topics. Most gaps were issues of comparative effectiveness (44/62), addressed the general cystic fibrosis population (40/62), and were specific to infants (33/62). Relevant comparisons and outcomes were explicitly stated for only 7 percent and 16 percent of gaps respectively.

Conclusions: Almost 80 percent of the gaps were not topics identified as future research needs in the guidelines documents but rather were topics with insufficient evidence for making recommendations. Although we used cystic fibrosis in the United States as an example, the method we developed could be applied in other settings, including other countries and for different diseases.

An increasing number of economic evaluations are being conducted alongside clinical trials. While this practice offers the prospect of collecting comprehensive and accurate cost data, it requires considerable time and effort. In the case of clinical data, key analytic decisions such as which data to collect and sample size are often made with reference to smaller (pilot) trials. However, this approach is not normally followed in the case of economic evaluation.

This study was based on a recently completed health technology assessment comparing conventional radiotherapy with continuous hyperfractionated accelerated radiotherapy (CHART) for patients with head and neck cancer or carcinoma of the bronchus. In the full health technology assessment, cost data were available for 526 head and neck patients (314 CHART and 212 conventional therapy) and 286 bronchus patients (175 CHART and 109 conventional therapy). In order to simulate a pilot study, data were extracted for the patients recruited to both trials in the first 3 months. These were then compared with the full data set in order to assess whether such a pilot study would have given useful guidance on: a) the usefulness of undertaking a full study; b) the sample size required; and c) the important resource items for which comprehensive data collection would be required.

Pilot studies can be helpful in determining the likely advantages of undertaking full economic evaluations and in identifying important resource items. Therefore, it is important that clinical researchers and research funding bodies create the necessary time window to enable such studies to take place. However, formal sample size calculations are more difficult to perform on limited data, since they also require knowledge of the unit cost (or prices) to be attached to the resource items and the correlation between costs and clinical effects.

Objectives: The objective of this study was to analyze preferences for activities comprised in comprehensive cardiac rehabilitation programs among former cardiac patients from three different hospitals in Copenhagen County, Denmark.

Methods: A discrete choice experiment was applied to elicit the preferences for the offer of participation in various cardiac rehabilitation program activities: smoking cessation course, physical exercise program, personal meetings with cardiac nurse, group meetings managed by cardiac nurses, and nutritional counseling guidance. The questionnaire was sent to 742 former cardiac patients. We had a response rate of 69 percent.

Results: We found that preferences differed with respect to gender and age and that the offer of participation in cardiac rehabilitation activities was not highly valued by older patients, in particular among older men.

Conclusions: The discrete choice experiment proved a valuable instrument for the measurement of preferences for cardiac rehabilitation. The study provides important information on patients' preferences for cardiac rehabilitation for healthcare professionals and decision makers.

A cost-effectiveness analysis is part of the U.K. ECMO Trial. In preparation for this analysis, existing evidence on the costs and effectiveness of neonatal ECMO was evaluated. ECMO appears to be more costly but may be more or less effective than conventional treatment. No case can be made for introducing ECMO before trial results are available.

Objectives: Most studies typically measure health preferences excluding health states perceived as worse than death. The objective of this study is to test the impact of including (versus excluding) health states perceived to be worse than death on utility measurementusing standard gamble (SG) and visual analogue scale (VAS) methods.

Methods: By means of a cross-sectional descriptive study design, women were asked to rate the utility of three hypothetical breast cancer health states: cure, treatment, and recurrence (n=119). Preference weights were estimated, allowing for negative utilities with death (perfect health) scaled at zero (1.0).

Results: Unpaired t-test analysis showed significantly greater change in SG and VAS weights for individuals perceiving cancer recurrence as worse than death than those perceiving death as least desirable state. Excluding negative utilities from the study resulted in significantly smaller changes in utility. Study results show that preference elicitation methods can be successfully adapted to acquire negative utilities.

Conclusions: Changes in utility were greater when negative preferences were permitted. Addressing negative preference scores could significantly affect quality adjusted life year estimates in economic analyses.

Objectives: To propose a new method for comparing and integrating original qualitative data with systematic reviews of quantitative and qualitative studies, demonstrated by a study of the psychosocial needs of chronic fatigue syndrome (CFS) sufferers in Québec.

Methods: A systematic literature review was performed across various databases for English and French language studies, on the psychosocial aspects of CFS. Qualitative, quantitative, and mixed method studies published between January 1994 and July 2008 were included. Unpublished literature and reference lists of included studies were also searched. Themes identified in the literature were used to guide semi-structured interviews with seventeen CFS-sufferers, mostly recruited from a large specialist practice in Montreal. Interviews were transcribed verbatim and validated by a research assistant. Transcripts were coded using the identified themes. New codes were created when new issues arose. All themes were subsequently synthesized into overall categories using a constant comparative method.

Results: The literature search yielded thirty-one papers: twenty-eight primary studies and three systematic reviews. Twelve themes were identified and synthesized into four overall problem categories, such as “Lack of professional recognition.” Interviews confirmed findings from the literature, but also revealed unidentified needs specific to CFS-sufferers in Québec. Policy recommendations were provided to address these needs.

Conclusions: Multi-Source Synthesis provides a systematic method for synthesizing data from original studies with literature findings, thereby broadening the knowledge base and the local relevance of decisions concerning specific patient populations.

Objective: The aim of this paper is to examine the costs of introducing a nursing-led ward program together with examining the impact this may have on patients' outcomes.

Methods: The study had a sample size of 177 patients with a mean age of 77, and randomized to either a treatment group (care on a nursing-led ward, n = 97) or a control group (standard care usually on a consultant-led acute ward, n = 80). Resource use data including length of stay, tests and investigations performed, and multidisciplinary involvement in care were collected.

Results: There were no significant differences in outcome between the two groups. The inpatient costs for the treatment group were significantly higher, due to the longer length of stay in this group. However, the postdischarge costs were significantly lower for the treatment group.

Conclusions: The provision of nursing-led intermediate care units has been proposed as a solution to inappropriate use of acute medical wards by patients who require additional nursing rather than medical care. Whether the treatment group is ultimately cost-additive is dependent on how long reductions in postdischarge resource use are maintained.

Objectives: The aim of the study was to evaluate the validity of the systematic reviews as a source of best evidence and to present and interpret the evidence of the systematic reviews on effectiveness of surgery and percutaneous interventions for stable coronary artery disease.

Methods: Electronic databases were searched without language restriction from January 1966 to March 2004. The databases used included the Cochrane Database of Systematic Reviews, the Cochrane Central Register of Controlled Trials, DARE, the Health Technology Assessment Database, MEDLINE(R), MEDLINE(R) In-Process & Other Non-Indexed Citations. We included systematic reviews of randomized clinical trials on patients with stable coronary heart disease undergoing percutaneous coronary intervention or coronary artery bypass surgery in comparison with medical treatment or a comparison between invasive techniques. At least one of the following outcomes had to be reported: death, myocardial infarction, angina pectoris, revascularization. The methodological quality was assessed using a modified version of the scale devised by Oxman and Guyatt (1991). A standardized data-extraction form was used. The method used to evaluate clinical relevance was carried out with updated method guidelines from the Cochrane Back Research Group. Quantitative synthesis of the effectiveness data is presented.

Results: We found nineteen systematic reviews. The median score of validity was 13 points (range, 6–17 points), with a maximum of 18 points. vCoronary artery bypass surgery gives better relief of angina, and the need for repeated procedures is reduced after bypass surgery compared with percutaneous interventions. There is inconsistent evidence as to whether bypass surgery improves survival compared with percutaneous intervention. A smaller need for repeated procedures exists after bare metal stent and even more so after drug-eluting stent placement than after percutaneous intervention without stent placement. However, according to the current evidence, these treatment alternatives do not differ in terms of mortality or myocardial infarction.

Conclusions: We found some high-quality systematic reviews. There was evidence on the potential of invasive treatments to provide symptomatic relief. Surgery seems to provide a longer-lasting effect than percutaneous interventions with bare metal stents or without stents. Evidence in favor of drug-eluting stents so far is based on short-term follow-up and mostly on patients with single-vessel disease.

The purpose of this survey was to assess potential information sources for identifying new health care technologies. A three-round Delphi study was conducted, involving 38 selected experts who suggested and assessed potential sources by applying agreed criteria. Twenty-six potential information sources were considered. Timeliness, time efficiency, and sensitivity were important criteria in determining which were the most important sources. The eight recommended sources were: pharmaceutical journals, pharmaceutical and biotechnology companies, specialist medical journals, key medical journals, medical engineering companies, private health care providers, newsletters and bulletins from other health technology assessment agencies, and groups of expert health professionals. There is a need to use a combination of sources because the most useful sources will vary according to the type of technology under consideration.

Health technology assessment (HTA) in France covers a variety of activities performed for different customers (e.g., health professionals in the field and policy makers in government) for the benefit of patients. To promote the improvement of quality in health care, France has set up a series of distinct agencies that report to the Ministry of Health but are also accountable to their other customers. We place particular emphasis on ANAES (National Agency for Accreditation and Evaluation in Health) whose main remit is HTA. We show how the diversity of HTA activities and their decentralization suggests tight collaboration among all the different bodies which perform HTA or are closely involved with HTA, and we provide examples of such collaboration.

Objectives: Value assessments of marketed drug technologies have been developed through disinvestment frameworks. Components of these frameworks are varied and implementation challenges are prevalent. The objective of this systematic literature review was to describe disinvestment framework process components for drugs and to report on framework components, challenges, and solutions.

Methods: A systematic literature search was conducted using the terms: reassessment, reallocation, reinvestment, disinvestment, delist, decommission or obsolescence in MEDLINE, EMBASE, NLM PubMed, the Cochrane Library, and CINAHL from January 1, 2000, until November 14, 2015. Additional citations were identified through a gray literature search of Health Technology Assessment international (HTAi) and the International Network of Agencies for Health Technology Assessment (INAHTA) member Web sites and from bibliographies of full-text reviewed manuscripts.

Results: Sixty-three articles underwent full text review and forty were included in the qualitative analysis. Framework components including disinvestment terms and definitions, identification and prioritization criteria and methods, assessment processes, stakeholders and dissemination strategies, challenges, and solutions were compiled. This review finds that stakeholders lack the political, administrative, and clinical will to support disinvestment and that there is not one disinvestment framework that is considered best practice.

Conclusions: Drug technology disinvestment components and processes vary and challenges are numerous. Future research should focus on lessening value assessment challenges. This could include adopting more neutral framework terminology, setting fixed reassessment timelines, conducting therapeutic reviews, and modifying current qualitative decision-making assessment frameworks.

Until the mid-1980s, most economic analyses of healthcare technologies were based on decision theory and used decision-analytic models. The goal was to synthesize all relevant clinical and economic evidence for the purpose of assisting decision makers to efficiently allocate society's scarce resources. This was true of virtually all the early cost-effectiveness evaluations sponsored and/or published by the U.S. Congressional Office of Technology Assessment (OTA) (15), Centers of Disease Control and Prevention (CDC), the National Cancer Institute, other elements of the U.S. Public Health Service, and of healthcare technology assessors in Europe and elsewhere around the world. Methodologists routinely espoused, or at minimum assumed, that these economic analyses were based on decision theory (8;24;25). Since decision theory is rooted in—in fact, an informal application of—Bayesian statistical theory, these analysts were conducting studies to assist healthcare decision making by appealing to a Bayesian rather than a classical, or frequentist, inference approach. But their efforts were not so labeled. Oddly, the statistical training of these decision analysts was invariably classical, not Bayesian. Many were not—and still are not—conversant with Bayesian statistical approaches.

Objectives: The evaluation of public health interventions poses some challenges. As a consequence, health technology assessment (HTA) methods for public health interventions (PHI) have to be adapted. This study aimed to summarize the available guidance on methods for HTA of PHI.

Methods: We systematically searched for methodological guidance on HTA of PHIs. Our focus was on research synthesis methods to evaluate effectiveness. Relevant information was synthesized narratively in a standardized way.

Results: Only four guidance documents were identified specifically for HTAs of PHI. The approaches used for HTAs of PHIs are broader and more flexible than those for medical interventions. For this reason, there is a tendency to identify the intervention components and context factors that influence the effectiveness and transferability of an intervention rather than to assess its effectiveness in general. The details in the guidance vary without justification. Unjustified heterogeneity between the different guidance approaches is most pronounced for quality assessment, assessment of applicability, and methods to integrate qualitative and quantitative evidence. Descriptions for the assessment of integrity, heterogeneity, sustainability, context factors, and applicability are often vague.

Conclusions: The heterogeneity in approaches indicates that there is currently no consensus on methods to deal with the challenges of the PHI evaluations. A possible explanation for this may be that the methods are not sufficiently developed, and advantages and disadvantages of a certain method in relation to the research question (e.g., broad/focused) have not yet been sufficiently evaluated.

Objectives: We examined the impact of household income on the use of medical services in Japan, where there is a “health care for all” policy, with important, centralized influence by the national government designed to ensure universal access.

Methods and Subjects: All healthcare societies operating in 2003 were included in the study, representing 14,776,193 insured adults and 15,496,752 insured dependents. The mean case rate (the average number of monthly bills per patient), the mean number of service days per person, and the mean medical cost per person served as indicators of medical service use. Multiple regression analysis was performed by the forced entry method using case rate, the number of service days, and medical cost as outcome variables, and average monthly salary, dependent ratio, average age, and premium rate as the explanatory variables.

Results: In the multiple regression analyses, average monthly salary showed a high positive correlation of outpatient and dental indicators, including case rate, the number of service days, and medical cost. If the average monthly salary were reduced 20 percent lower than the mean, the estimated changes (95 percent CI) in case rate for the insured were −7.49 (−8.14 ∼ −6.84) percent for outpatient visits and −8.16 (−8.77 ∼ −7.56) percent for dental services.

Conclusions: Average monthly salary intensifies the effects of copayments on the case rate, the number of service days, and medical cost in the “Employees Health Insurance” in Japan. Thus, a low salary appears to discourage patients from seeking medical and dental services.

We evaluated trends in the use of echocardiography among residents of Olmsted County, Minnesota, from January 1, 1975, through June 30, 1987. Annual examinations per 100,000 population among Olmsted County residents increased 6-fold from 264 in 1975 to 1,644 in 1986. Given the pattern of echo use in Olmsted County, these population-based data project a potential annual utilization of 4 million echo exams in the United States. Marked increases over time in labeling of patients with regurgitant valvular heart disease were noted following the introduction of Doppler echocardiography. The diagnostic labeling associated with this evolving technology and the resultant apparent epidemic of valvular regurgitation present a significant clinical technology assessment dilemma.

Objective: To evaluate the long-term impact of an intervention designed to reduce the ordering of three tumor markers frequently prescribed for gastroenterologic diseases (carcinoembryonic antigen, alpha-fetoprotein, carbohydrate antigen 19-9).

Methods: A prospective study with time series analysis in a teaching hospital. Local clinical guidelines were developed and implemented through a new order form, designed as a reminder to the physician, restricting the ordering of laboratory tests. Ratios between the number of markers ordered and number of admissions were recorded during a 3-month period before and after intervention in the whole hospital and monthly on a 4-year period in two wards of the hospital (Department of Gastroenterology and Department of Internal Medicine). To evaluate the appropriateness of tumor marker orders, audits were performed on a sample of order forms, before and after (1 month and 2 years after) the implementation of the new order form.

Results: The analysis of covariance showed a significant effect of the intervention in the hospital (p < .01), and in the Departments of Gastroenterology (p < .01) and Internal Medicine (p < .007). The decrease of tumor marker orders ranged from 25% (Internal Medicine Department) to 55% (whole hospital). A similar decrease was observed for the three studied markers. The appropriateness of prescriptions increased from 54.6% before to 73.6% after the implementation of the new order form, but decreased to 52.9% 2 years after intervention.

Conclusions: Providing a reminder to clinicians through a specific order form represents an inexpensive and easy way to implement guidelines on use of laboratory tests.

This paper describes the evolution of written patient information on prescription drugs, FDA's patient package insert proposal and its revocation before its implementation in the early 1980s, the status of voluntary and government efforts since that time, and a review of current trends in this area. The burgeoning practice of direct-to-consumer advertising of prescription drugs in the lay press is viewed within the context of these efforts. This paper also reviews the current developments in certain FDA regulated consumer products—over-the-counter drug labeling and nutrition labeling. The current state of patient information in the United States is reviewed in light of programs to improve prescription drug counseling, and current research in this area is discussed.