Objectives: Health-care technology reviews now increasingly include outcome costs as well as clinical effects. This study reports the findings and implications of a survey to explore the usefulness of the National Health Service Economic Evaluation Database (NHS EED) within this process.

Methods: Postal survey of lead authors of Technology Assessment Reviews (TARs) commissioned by the United Kingdom's National Institute for Clinical Excellence (NICE). The questionnaire investigated the usefulness of NHS EED in terms of (a) search strategy, (b) data extraction, (c) quality assessment, and (d) determining requirements for new modeling studies. Qualitative data were requested, including opinions regarding NHS EED.

Results: NHS EED was used in 90 percent of all identified reviews (n=46). The questionnaire response rate was 63 percent. The percentage of scores 3 or above (most useful), 2 or below (least useful), or N/A were, respectively, (a) search strategy=62 percent, 23 percent, 15 percent; (b) data extraction=23 percent, 27 percent, 50 percent; (c) quality assessment=38 percent, 19 percent, 42 percent; (d) determining requirements for new modeling studies=27 percent, 23 percent, 50 percent. The results were expanded further in the qualitative data from the respondents.

Conclusions: NHS EED is a useful tool for a variety of tasks in the NICE/TAR process but not, unsurprisingly, as a replacement for understanding primary studies. There is, however, a need to reduce the impact of time lags between the publication of economic evaluations and the appearance of abstracts relating to them on NHS EED. The results will inform future developments of the NHS EED database, which should increase its usefulness to researchers.

Background: Home hemodialysis offers potential advantages over hospital hemodialysis, including the opportunity for more frequent and/or longer dialysis sessions. Expanding home hemodialysis services may help cope with the increasing numbers of people requiring hemodialysis.

Methods: We sought comparative studies or systematic reviews of home versus hospital/satellite unit hemodialysis for people with end-stage renal failure (ESRF). Outcomes included quality of life and survival. We searched MEDLINE, EMBASE, HealthSTAR, CINAHL, PREMEDLINE, and BIOSIS. Two reviewers independently extracted data and assessed the quality of the studies included.

Results: Twenty-seven studies of variable quality were included. People on home hemodialysis generally experienced a better quality of life and lived longer than those on hospital hemodialysis. Their partners, however, found home hemodialysis more stressful. Four studies using a Cox proportional hazards model to compare home with hospital hemodialysis reported a lower mortality risk for home hemodialysis. Of two studies using a Cox model to compare home with satellite unit hemodialysis, one reported a similar mortality risk, whereas the other reported a lower mortality risk for home hemodialysis.

Conclusions: Home hemodialysis was generally associated with better outcomes than hospital hemodialysis and (more modestly so) satellite unit hemodialysis, in terms of quality of life, survival, and other measures of effectiveness. People on home hemodialysis, however, are a highly selected group. Home hemodialysis also provides the opportunity for more frequent and/or longer dialysis sessions than would otherwise be possible. It is difficult to disentangle the true effects of home hemodialysis from such influencing factors.

Objectives: The purpose of this study is to provide a comparison of the benefits of reduction mammaplasty (RM) for women with heavy breasts often termed macromastia or breast hypertrophy (BH) surgery. The rationale is to provide information to allow decision-makers to make judgments about the cost-effectiveness of this intervention and make comparisons with other interventions which are commonly undertaken within publicly financed health-care systems.

Methods: Data from a previous outcomes study in Sweden is re-analyzed to derive quality of life measures, from which a mean level of benefit outcome is derived and a cost per quality-adjusted life year is calculated (cost per QALY).

Results: The low Cost per QALY suggests that reduction mammaplasty is cost-effective when compared with other treatments which are commonly undertaken.

Conclusions: The authors suggest that the evidence in favor of funding reduction mammaplasty is strong and that decision-makers review their policy in light of this new evidence.

Objectives: Infliximab is a costly therapy for active Crohn's disease resistant to corticosteroids and immunosuppressive medication. The purpose of this study was to examine whether a treatment including infliximab (episodic re-infusions for relapse or maintenance therapy every 8 weeks) was relevant compared with conventional management (surgery and medical treatment without infliximab) for nonfistulizing resistant Crohn's disease.

Methods: We performed a life-time cost-utility analysis with an analytic Markov decision model from the perspective of the third-party payer system. Utility measurement using Standard Gamble was used to adjust the survival time for each health state of the disease. Direct costs were estimated from standard management based on expert opinion. A sensitivity analysis was conducted to gauge the effects of uncertainty in the values assigned to variables.

Results: The incremental effectiveness with infliximab therapy is .761 Quality-Adjusted Life Years (QALYs) for an added cost ranging from 48,478.79 euros to 596,990.35 euros, depending on treatment procedure. The incremental cost utility ratio expressed in euros per QALYs saved varied from 63,700.82 euros (episodic re-infusions) to over 762,245.09 euros (maintenance therapy).

Conclusions: Infliximab therapy could be cost-effective in the case of relapse treatment only, whereas the marginal cost-utility ratio exceeds conventional benchmarks for maintenance therapy. This analysis will be supplemented by conducting further randomized controlled trials and prospective observational study, focused on the costs of illness (direct and indirect), patient preferences, the disease's clinical course, and infliximab safety.

Objectives: To review the existing health economic literature on meningococcal disease vaccination.

Methods: A Medline search for economic evaluations of vaccination programs for meningococcal disease in developed countries was conducted. All identified studies were reviewed.

Results: Nine published studies were identified examining either mass vaccination during outbreaks or routine vaccination. Although net expenses were estimated in almost all studies, the resulting cost-effectiveness ratios varied widely. Vaccination of college-age students was found to be potentially cost-effective in Australia but not in the United States. With one exception, routine vaccination of children and adolescents in Europe was predicted to be cost-effective. Many simplifying assumptions were made, and important elements were often left out, in particular the potential for reduced transmission of disease.

Conclusions: The methods used and the vaccination strategies vary widely, and results do not provide strong grounds for making conclusions as to whether vaccination is cost-effective. Furthermore, in all instances, transmission of disease, changes in population carriage rates, and outbreaks are either ignored, dealt with using very broad simplifying assumptions, or are not necessarily generalizable to other settings. The analyses provide some insight into the potential cost-effectiveness of vaccination, but more importantly, they highlight areas requiring further study. Economic evaluations based on observed outcomes from recently implemented strategies would be helpful, as would more sophisticated health economic models. The choice of vaccination strategies cannot be based on the results of existing economic analyses.

Objectives: Decision analysis is commonly used to perform economic evaluations of new pharmaceuticals. The outcomes of such studies are often reported as an incremental cost per quality-adjusted life year (QALY) gained with the new agent. Decision analysis can also be used in the context of estimating drug cost before market entry. The current study used neurokinin-1 (NK-1) receptor antagonists, a new class of antiemetics for cancer patients, as an example to illustrate the process using an incremental cost of $Can20,000 per QALY gained as the target threshold.

Methods: A decision model was developed to simulate the control of acute and delayed emesis after cisplatin-based chemotherapy. The model compared standard therapy with granisetron and dexamethasone to the same protocol with the addition of an NK-1 before chemotherapy and continued twice daily for five days. The rates of complete emesis control were abstracted from a double-blind randomized trial. Costs of standard antiemetics and therapy for breakthrough vomiting were obtained from hospital sources. Utility estimates characterized as quality-adjusted emesis-free days were determined by interviewing twenty-five oncology nurses and pharmacists by using the Time Trade-Off technique. These data were then used to estimate the unit cost of the new antiemetic using a target threshold of $Can20,000 per QALY gained.

Results: A cost of $Can6.60 per NK-1 dose would generate an incremental cost of $Can20,000 per QALY. The sensitivity analysis on the unit cost identified a range from $Can4.80 to $Can10.00 per dose. For the recommended five days of therapy, the total cost should be $Can66.00 ($Can48.00–$Can100.00) for optimal economic efficiency relative to Canada's publicly funded health-care system.

Conclusions: The use of decision modeling for estimating drug cost before product launch is a powerful technique to ensure value for money. Such information can be of value to both drug manufacturers and formulary committees, because it would facilitate negotiations for optimal pricing in a given jurisdiction.

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.

Objectives: To compare methods and results among four health technology assessment organizations in different countries.

Methods: All assessment reports published between 1999 and 2001 by VATAP (United States), NICE (United Kingdom), CCOHTA (Canada), and AETS (Spain), were reviewed. Detailed information about the organization, the technology assessed, the methods used, and the recommendations made were collected. A descriptive analysis of the variables, as well as comparisons of means and proportions, was performed.

Results: Sixty-one reports assessing seventy-six technologies were published: nine (11.8 percent) by VATAP, thirty-nine (51.3 percent) by NICE, twenty (26.3 percent) by CCOHTA, and eight (10.5 percent) by AETS. A total of 64.5 percent of the technologies assessed were related to a high prevalence disease in the corresponding country. Most of the assessments addressed treatments (73.7 percent) and were mostly drugs (56.6 percent) and devices (23.7 percent). Most organizations used reviews of effectiveness and economic evaluations (64.5 percent), systematic reviews (21.1 percent), and original economic evaluations (36.7 percent). In 38.1 percent, the technology was recommended; the rest of the cases had no formal recommendations.

Conclusions: Critical issues for future technology assessment efforts are making assessment processes more consistent, transparent, and evidence-based; formalizing the inclusion of economic and ethical considerations; and making more explicit the prioritization process for selecting technologies for assessment and reassessment.

Objectives: This study reviewed the European guidelines of the Committee for Proprietary Medicinal Products (CPMP) on how health-related quality of life (HRQOL) research should be conducted in clinical trials. Published product-level information was also reviewed to investigate the actual role of HRQOL data in the European regulatory process.

Methods: All disease-specific notes for guidance and concept papers on clinical investigations, development and evaluation of human medicinal products, as well as the European Public Assessment Reports (EPAR) of all approved drugs published on the European Agency for the Evaluation of Medicinal Products (EMEA) Web site were evaluated for their HRQOL recommendations.

Results: Only twenty of the fifty CPMP guidance notes for clinical investigation of pharmaceutical products in specific disease areas included a reference to HRQOL. Most of the recommendations were generic and vague, and the terminology used was inconsistent across documents. The EPAR provided nonspecific information about HRQOL and contradictory conclusions on the effect of a drug on HRQOL sometimes occurred in different documents. The criteria used by the CPMP to assess the HRQOL data could not be identified due to an ad hoc approach to the inclusion of data in the EPAR.

Conclusions: A more systematic approach is needed on the way health outcomes data are considered, reviewed, and interpreted by the regulatory authorities. For this to be achieved, CPMP should develop general guidelines on the importance of HRQOL and how research should be conducted if data are to be included in the registration process.

Objectives: While strategies for enhancing the dissemination and impact of Health Technology Assessment (HTA) are now being increasingly examined, the characteristics of HTA production have received less attention.

Methods: This study presents the results of a content analysis of the HTA documents (n=187) produced by six Canadian agencies from 1995 to 2001, supplemented by interviews with chief executive officers and researchers (n=40). The goal of this analysis was to characterize the agencies' portfolios and to analyze the challenges these agencies face in responding to the increased demand for HTA.

Results: On average, thirty HTA products were issued annually by the agencies. While the bulk of documents produced were full HTA reports (76 percent), two agencies showed significant diversification in their products. Three agencies in particular actively supported the publication of results in scientific journals. Three agencies showed evidence of adapting to different institutional environments by specializing in certain areas (drugs, health services). Overall, a significant portion of the agencies' HTAs contained data on costs (37 percent) and effectiveness (48 percent), whereas ethical and social issues were rarely addressed (17 percent). Most agencies addressed issues and outcomes that did not strictly fall under the typical definition of HTA but that increased the “contextualization” of their findings.

Conclusions: Our discussion highlights four paradoxes and reflects further on challenges raised by the coordination of HTA within large countries and among European states. This study concludes that HTA is being redefined in Canada as HTA agencies offer a more contextualized informational basis, an approach that may prove more compatible with the increased demand for HTA.

Objectives: This study outlines the needs and current development of the “fourth hurdle” (i.e., requirement of effectiveness and cost-effectiveness data for drug coverage policy decisions) in Hungary, describes the legal background and seeks to address some of the most important questions in this field.

Methods: The study draws on international experiences and discusses five issues that a given jurisdiction needs to consider before introducing the “fourth hurdle” for pharmaceuticals.

Results: The “fourth hurdle” is very relevant in Hungary because many existing drugs are unevaluated and many new, expensive drugs are becoming available. On the other hand, the existing resources for health technology assessment, including economic evaluation, are quite limited. All the five issues are relevant in the Hungarian setting and were helpful in determining exactly how the “fourth hurdle” should be applied.

Conclusions: The most important issue seems to be that the implementation of the “fourth hurdle” needs to be achieved in a way consistent with the limited resources for HTA in Hungary. Specifically this means that, in setting priorities for drugs to evaluate, additional criteria need to be applied. In particular, priority should be given to assessing drugs that have been evaluated in other countries, because this affords the opportunity to adapt existing studies or models to the Hungarian situation.

Objectives: To explore and test methods for the operation of a national Early Warning System (EWS) in Denmark and to support decision making by the Danish Centre for Evaluation and Health Technology Assessment on this issue.

Methods: On the basis of literature reviews, information from members of EuroScan, and supported by clinical experts and stakeholders, existing methods were adapted and new methods were developed as part of a feasibility study.

Results: Approximately 200 technologies in 30 specialties were identified on the basis of information by EuroScan. A new instrument was developed to distinguish between important and unimportant technologies (filtering). Clinical experts in six specialties applied the instrument to sixty-two technologies in their respective fields, of which nine (15%) were judged potentially important for the Danish health care system. For priority setting, adapting a Dutch instrument to the Danish context was discussed. In principle, the instrument was acceptable, but several changes were proposed, for example, relating to the decentralized structure of the Danish health care system. For early assessment, the format and methods applied by SBU and Canadian Coordinating Office for Health Technology Assessment (CCOHTA) were compared and applied to pharmaceuticals (glitazones in treatment of type 2 diabetes mellitus) and a procedure (embolization of uterine fibromas). Given the main target group of the Danish EWS, local decision makers, the CCOHTA format was preferred.

Conclusions: The findings of the study have laid the foundation for an EWS using appropriate methods adapted to local circumstances. On the basis of the findings, a decision was made to start an EWS.

Objectives: Assess the degree to which research project findings were published and explore factors that influenced publication.

Methods: Questionnaire to project leaders. Classification of publications and findings. Chi-squared; univariate and multivariate Cox regression analyses.

Results: Forty percent of projects published in peer-reviewed journal; highly statistically significant relationships between publication in peer-reviewed journals and (1) projects in Responsive/Fellowships streams (p=.045); and (2) projects awarded >£22,713 (p=.02); influence of study findings not statistically significant.

Conclusions: Funders should consider the significant number of studies that did not result in publication and the higher rate of publication in peer-reviewed journals from some programs.

Objectives: To assess the impact of the availability of drug-eluting stents (DES) in April 2002 in Emilia-Romagna (a four-million resident Italian region), on the rate of use of revascularization procedures. DES are expected to influence the rate of use of coronary artery bypass graft (CABG) and percutaneous coronary interventions (PCI). However, to date, little empirical information is available on the actual impact of this innovation on clinical practice.

Methods: A time series regression analysis of the monthly number of procedures (PCI and CABG) performed between January 1998 and March 2003.

Results: DES availability was associated with statistically significant changes in the use of revascularization procedures, although the size of the effect was always small or moderate. PCI increased only by 0.36 more procedures per month (approximately four per year), whereas more evident was the concurrent reduction of isolated CABG (4.15 fewer per month, that is fifty fewer per year). Overall, considering all the surgical revascularizations (i.e., CAGB both isolated and associated with other interventions), there was a reduction of 2.52 procedures per month (thirty less per year).

Conclusions: Despite DES being at a very early stage of their diffusion process, our results indicate that they are already having an impact, although moderate, on the use of revascularization procedures. If these findings will be confirmed, they will have a substantial influence on the patterns of care for patients with coronary artery disease and relevant policy implications for health services.

Objectives: To study the impact on public health in terms of utility of various proportions of first-eye and second-eye cataract surgery.

Methods: A model was used to study the impact on a population of a fixed cataract surgical rate (9,250 operations/1,000,000 people) with varying proportions of first-eye and second-eye cataract operations. The study population was the County of Blekinge with a known incidence of previous cataract surgery. The prevalence of cataract, the estimated need for cataract surgery, and the utility values were taken from the literature. The population was grouped by disability stage of cataract and previous cataract surgery in accordance with prevalence studies and data from a large national database on cataract surgery and patients' self-assessed visual function. The mortality rate was taken from real data for the study population.

Results: Given a fixed cataract surgical rate over a period of five years, a high percentage of second-eye cataract surgery (42 percent) resulted in a mean utility of 0.82239 in the population forty years of age and older and the corresponding number for a low percentage of second-eye cataract surgery (25 percent) was 0.82253. A high percentage of second-eye surgeries resulted in 421 more individuals who were well compared with a low percentage of second-eye surgeries. On the other hand, a low percentage of second-eye surgeries resulted in 152 fewer individuals with disability and 118 fewer individuals with dependence compared with a high percentage of second-eye surgeries.

Conclusions: A high frequency of first-eye cataract surgeries instead of second-eye surgeries affects more individuals and means an optimized improvement of utility in a population. This should be recommended if the cataract surgical rate is very insufficient. If the cataract surgical rate is high, more second-eye surgeries should be performed to optimize quality of life to as many as possible.

Objectives: The objective of the study was to estimate the costs and health benefits of a public awareness campaign aimed at shortening the delay for thrombolytic therapy in patients with acute myocardial infarction (AMI) and to estimate the incremental costs and benefits of an additional telemedicine program.

Methods and Results: By using trial data on the impact of a Swedish campaign, a model was developed to simulate the current distribution of thrombolytic delay in Denmark and the delay after a campaign. The reduction in delay was translated into reduced fatality assuming reductions from the campaign and additional effects of a telemedicine program. The costs of the campaign were based on trial data and Danish unit costs while telemedicine costs were taken from a Danish demonstration program. The analyses indicate that the awareness campaign will translate into five fewer fatal AMIs (sixty-two life years gained) and a cost per life year of DKK283,300, with both costs and benefits discounted at 5 percent. When combining the public campaign with prehospital telemedicine diagnostics, the incremental cost per life year gained was DKK854.700.

Conclusions: Programs aimed at reducing delay of thrombolysis in patients with AMI are likely to have a limited impact on AMI fatality. Information campaigns may have acceptable cost-effectiveness ratios, while telemedicine programs lead to threefold greater ratios. Whether such programs can be considered cost-effective will depend on how life year gains are valued by society.

Objectives: To estimate the excess cost of stroke in Sweden and the potential costs that could be avoided by preventing first-ever strokes.

Methods: We adopted the incidence approach for estimating the present value of both direct and indirect costs. Data on mortality, stroke recurrence, and inpatient care were estimated from a national register of patient data with a four-year follow-up period. To estimate costs for social services, we used survey data on living conditions before stroke onset and at three and at twenty-four months. Costs for outpatient visits, rehabilitation, drugs, and production losses due to premature death and early retirement were estimated on the basis of both published and nonpublished sources. Lifetime costs were based on life tables adjusted for excess mortality of stroke, and costs in year 4 were extrapolated to subsequent years.

Results: The present value direct cost for an average stroke patient is SEK513,800 (US$56,024 or Euro60,825). The corresponding indirect cost is SEK125,110 (US$13,640 or Euro14,810). Almost 45 percent of the direct costs were attributable to social services. Women had higher costs than men, and costs for survivors increased with age due to social services.

Conclusions: With an incidence of 213 first-ever strokes per 100,000 individuals, the total excess direct and indirect cost of stroke would be SEK12.3 billion (approximately US$1.3 billion or Euro1.5 billion). Hence, there are large potential cost offsets both in the health-care sector and in the social service sector if the incidence of first-ever stroke could be reduced.

Objectives: Early warning systems are an integral part of many health technology assessment programs. Despite this finding, to date, there have been no quantitative evaluations of the accuracy of predictions made by these systems. We report a study evaluating the accuracy of predictions made by the main United Kingdom early warning system.

Methods: As prediction of impact is analogous to diagnosis, a method normally applied to determine the accuracy of diagnostic tests was used. The sensitivity, specificity, and predictive values of the National Horizon Scanning Centre's prediction methods were estimated with reference to an (imperfect) gold standard, that is, expert opinion of impact 3 to 5 years after prediction.

Results: The sensitivity of predictions was 71 percent (95 percent confidence interval [CI], 0.36–0.92), and the specificity was 73 percent (95 percent CI, 0.64–0.8). The negative predictive value was 98 percent (95 percent CI, 0.92–0.99), and the positive predictive value was 14 percent (95 percent CI, 0.06–0.3).

Conclusions: Forecasting is difficult, but the results suggest that this early warning system's predictions have an acceptable level of accuracy. However, there are caveats. The first is that early warning systems may themselves reduce the impact of a technology, as helping to control adoption and diffusion is their main purpose. The second is that the use of an imperfect gold standard may bias the results. As early warning systems are viewed as an increasingly important component of health technology assessment and decision making, their outcomes must be evaluated. The method used here should be investigated further and the accuracy of other early warning systems explored.

Objectives: Hospital readmission rate is currently used as a quality of care indicator, although its validity has not been established. Our aims were to identify the frequency and characteristics of potential avoidable readmissions and to compare the assessment of quality of care derived from readmission rate with other measure of quality (judgment of experts).

Methods: Design: cross-sectional observational study; Setting: acute care hospital located in Marbella, South of Spain; Study participants: random sample of patients readmitted at the hospital within six months from discharge (n=363); Interventions: review of clinical records by a pair of observers to assess the causes of readmissions and their potential avoidability; Main measures: logistic regression analysis to identify the variables from the databases of hospital discharges which are related to avoidability of readmissions. Determination of sensitivity and specificity of different definitions of readmission rate to detect avoidable situations.

Results: Nineteen percent of readmissions were considered potentially avoidable. Variables related to readmission avoidability were (i) time elapsed between index admission and readmission and (ii) difference in diagnoses of both episodes. None of the definitions of readmission rate used in this study provided adequate values of sensitivity and specificity in the identification of potentially avoidable readmissions.

Conclusions: Most readmissions in our hospital were unavoidable. Thus, readmission rate might not be considered a valid indicator of quality of care.

Designing & Conducting Cost-Effectiveness Analyses in Medicine and Health Care by Muennig P and contributing editor Khan K. San Francisco: Jossey-Bass, John Wiley & Sons, Inc; 2002, 356 pp., UK £48.50, hardbound.

Reviewed by: Wija Oortwijn, PhD and Eddy Adang, PhD, University Medical Centre Nijmegen, Department of HTA, Nijmegen, The Netherlands.