Over the last few years, the pharmaceutical industry has financed an increasing number of health economic evaluations. The purpose of this article is to describe how and why the pharmaceutical industry is applying health economic evaluations. Seven different fields of application are identified, and the varying reasons for use are discussed.

The NHS Health Technology Assessment (HTA) Programme runs an annual process of identifying suggestions for health technology assessment. The objective of this paper is to describe and evaluate the relative importance of the different sources used by the program in 1998 to identify potential priorities. There were four different sources: a) a widespread consultation of healthcare commissioners, providers and consumers; b) research recommendations from systematic reviews; c) reconsidering previous research priorities which had not been taken forward for funding; and d) horizon scanning. Collectively, the four sources generated just over 1,100 HTA suggestions. By far the largest source of suggestions and priorities was the widespread consultation. However, the success rate of this source, in terms of being commissioned, was low. Research recommendations from systematic reviews provided the second largest source of priorities and the best success rate of all sources. Value was found from different sources for different healthcare areas.

Objectives: Although endoscopic ultrasound (EUS) staging of esophageal cancer is established in clinical practice, high-quality evidence about its impact on patient outcomes is not available. This study aims to determine the impact of EUS for esophageal cancer staging on patient management and survival.

Methods: A systematic review was conducted using Medline, PreMedline, Embase, and The Cochrane Library. Included studies were (i) comparative studies reporting survival following EUS esophageal cancer staging, (ii) therapeutic impact studies reporting change in patient management following EUS. The quality of included studies was critically appraised.

Results: One systematic review, five studies reporting therapeutic impact, and two studies reporting patient survival were identified. The design and quality of the therapeutic impact studies varied widely. Management changed in 24–29 percent of patients following EUS staging of esophageal cancer (two studies). No studies provided data on the avoidance of surgery for this indication. One retrospective cohort study with historical control found EUS staging of esophageal cancer improved patient survival; a second study with similar design limitations did not find a survival benefit for EUS staging in patients undergoing resection. These studies had a high potential for bias, limiting the value of these findings.

Conclusions: Two studies provided evidence of a change in patient management following EUS for staging esophageal cancer, a higher level of evidence for a clinical benefit than can be obtained from accuracy studies alone. This evidence contributed to a recommendation for public funding of EUS in staging esophageal cancer in Australia.

Objectives: To catalogue and comparatively assess the quality of Clinical Practice Guidelines (CPG) for ischemic stroke taking into account format and development methodology.

Methods: We performed a comprehensive, systematic bibliographic search of CPGs addressing the management of ischemic stroke. We designed a sensitive strategy, using methodological filters in the following databases: Medline, IME and Lilacs, National Guidelines Clearinghouse, National electronic Library for Health, NICE, Guidelines International Network (GIN), Canadian Medical Association Infobase, development groups such as Scottish Intercollegiate Guidelines Network (SIGN), New Zealand Guidelines Group (NZGG), Agency for Healthcare Research and Quality (AHRQ), Ministry of Health Singapore, Institute for Clinical Systems Improvement (ICSI); and scientific societies: American Heart Association, American Medical Association, Royal College of Physicians London. We included all CPGs published in English, French, Italian, Portuguese, or Spanish from 1999 to 2005 and excluded those CPGs whose scope was primary prevention and rehabilitation from ischemic stroke. Four researchers independently assessed the structure and methodologies followed in drafting the CPGs using the Changing Professional Practice (CPP) and Appraisal of Guidelines Research & Evaluation (AGREE) instruments.

Results: We retrieved 117 documents; following application of exclusion criteria, twenty-seven CPGs were appraised. With regard to methodological quality (using the AGREE instrument), the domains that scored highest were “Scope and purpose” and “Clarity and presentation.” The lowest scoring domains were “Stakeholder involvement,” “Rigor of development,” and “Applicability.” Most guidelines received an overall score of “would not recommend” (77.8 percent). Finally, based on the CPP instrument, most of the CPGs evaluated were aimed at secondary care and did not provide updating procedures.

Conclusions: The overall quality of the CPGs published for ischemic stroke management did not have minimum methodological quality. Quality improvement has been observed in more recent CPGs and may be due to the publication of new tools such as the AGREE or CPP instruments, as well as international initiatives for CPG improvement.

Background: Using multiples of India's per capita gross domestic product (GDP) as the threshold for economic value as suggested by the World Health Organization (WHO), decision analysis modeling was used to estimate a more affordable monthly cost in India for a hypothetical new cancer drug that provides a 3-month survival benefit to Indian patients with metastatic colorectal cancer (mCRC).

Methods: A decision model was developed to simulate progression-free and overall survival in mCRC patients receiving chemotherapy with and without the new drug. Costs for chemotherapy and side-effects management were obtained from both public and private hospitals in India. Utility estimates measured as quality-adjusted life-years (QALY) were determined by interviewing twenty-four oncology nurses using the Time Trade-Off technique. The monthly cost of the new drug was then estimated using a target threshold of US$9,300 per QALY gained, which is three times the Indian per capita GDP.

Results: The base-case analysis suggested that a price of US$98.00 per dose would be considered cost-effective from the Indian public healthcare perspective. If the drug were able to improve patient quality of life above the standard of care or survival from 3 to 6 months, the price per dose could increase to US$170 and US$253 and offer the same value.

Conclusions: The use of the WHO criteria for estimating the cost of a new drug based on economic value for a developing country like India is feasible and can be used to estimate a more affordable cost based on societal value thresholds.

Objective: The series of papers in this issue was developed to examine the use of health technology assessment in policies toward prevention—specifically toward mass screening—in European countries. The papers actually examined three screening strategies: mammography screening for breast cancer, prostate-specific antigen screening for prostate cancer, and routine ultrasound in normal pregnancy.

Methods: Papers were sought from the member states of the European Union, plus Switzerland. Ultimately, nine acceptable papers were received, and were reviewed, revised, and edited.

Results: Screening is an accepted strategy in many countries for reducing the burden of disease through early detection and intervention. In part, this is because of successful screening programs that have been evaluated and implemented in many countries. At the same time, unevaluated and even useless and harmful screening programs—unjustified medically or economically—are widespread. Health technology assessment could help assure that only effective and cost-effective screening programs are implemented.

Conclusion: The main conclusion is that screening is an important preventive strategy. Any screening program, however, should be carefully assessed before implementation.

Purpose: A peripherally inserted central catheter (PICC) is a useful option in providing secure venous access, which enables patients to be discharged earlier with the provision of home care. The objective was to identify the costs associated with having a PICC from a societal perspective, and to identify factors that are associated with total PICC costs.

Methods: Data were obtained from a retrospective cohort of 469 hospitalized pediatric patients with PICCs inserted. Both direct and indirect costs were estimated from a societal perspective. Insertion costs, complication costs, nurse and physician assessment costs, inpatient ward costs, catheter removal costs, home care costs, travel costs, and the cost associated with productivity losses incurred by parents were included in this study.

Results: Based on catheter dwell time, the median total cost associated with a PICC per patient per day (including inpatient hospital costs) was $3,133.5 ($2,210.7–$9,627.0) in 2017 Canadian dollars ($1.00USD = $1.25CAD in 2017). The adjusted mean cost per patient per day was $2,648.2 ($2,402.4–$2,920.4). Excluding inpatient ward costs, the median total and adjusted costs per patient per day were $198.8 ($91.8–$2,475.8) and $362.7($341.0–$386.0), respectively. Younger age, occurrence of complications, more catheter dwell days, wards with more intensive care, and the absence of home care were significant factors associated with higher total PICC costs.

Conclusions: This study has demonstrated the costs associated with PICCs. This information may be helpful for healthcare providers to understand PICC related cost in children and resource implications.

A clinical and economic evaluation of red blood cell (RBC) utilization in cancer patients during chemotherapy is described. Using a randomized sampling process, 100 patients who had received chemotherapy with or without cisplatin were selected (50 in each group). Multiple logistic regression was then used to identify risks factors for transfusion requirements. Twenty-five percent of patients in the cisplatin and 12% in the noncisplatin group received at least one blood transfusion during chemotherapy (p =.09). Depressed hemoglobin levels and cisplatin dosage were identified as risk factors for transfusion requirements. Combining all transfused patients revealed an overall cost of Can $599 (95% Cl: $513–$683) per transfusion. The results of the current study indicated that anemia is a common complication of cancer chemotherapy that can be costly to manage.

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: Traditionally, patients with chronic hepatitis C virus (HCV) infection have not received treatment until their infection reaches the moderate to severe stage. The aim of this systematic review was to assess the clinical effectiveness of pegylated (PEG) and non-pegylated interferon (IFN) alfa and ribavirin (RBV) for the treatment of adults with histologically mild HCV.

Methods: We performed a sensitive search of fourteen electronic bibliographic databases for literature that met criteria defined in a research protocol. Two reviewers independently selected studies, extracted data and assessed methodological quality.

Results: Ten randomized, controlled trials (RCTs) were included. Treatment with PEG + RBV combination therapy resulted in significantly higher sustained virological response (SVR) rates than treatment with IFN + RBV combination therapy. Treatment for 48 weeks with PEG + RBV was significantly more effective than the same treatment for 24 weeks. Significantly higher SVR rates were seen with IFN + RBV compared with either IFN monotherapy or no treatment. In the meta-analysis (four IFN trials), the relative risk of not experiencing an SVR was 0.59 (95 percent CI, 0.51 – 0.69) and was statistically significant (p < .00001). SVRs were higher for patients with genotype non-1 compared with genotype 1 for both PEG + RBV and IFN + RBV treatments.

Conclusions: Patients with histologically mild HCV can be successfully treated with both PEG and IFN combination therapy, and response rates are broadly comparable with those achieved in patients with advanced disease. Treating patients in the early milder stages of HCV is, therefore, a clinically effective option.

Health technology assessment as a formalized set of activities has a relatively short history. At its current stage of development, it is clear that it has global dimensions and impact. In this paper we review the history of health technology assessment, its development as a form of health services research, and its “institutionalization.” We then identify the reasons for its internationalization, review current international initiatives, and propose actions to be taken to improve cooperation among countries.

Objectives: Case series constitute a weak form of evidence for effectiveness of health technologies. However, for a variety of reasons, such studies may be included in health technology assessments. There are no clear criteria for assessing the quality of case series. We carried out an empirical investigation of the association between outcome frequency and methodological characteristics in a sample of health technology assessments.

Methods: Systematic reviews of functional endoscopic sinus surgery for nasal polyps, spinal cord stimulation for chronic back pain, and percutaneous transluminal coronary angioplasty and coronary artery bypass grafting for chronic stable angina were identified as containing more than forty case series. Data were extracted by one reviewer and checked by a second on population characteristics, outcomes, and the following methodological features: sample size, prospective/retrospective approach, consecutive recruitment, multi- or single-center organization, length of follow-up, independence of outcome measurement, and date of publication. Association between methodological features and outcome were explored in univariate and multivariate analyses using parametric and nonparametric tests and robust regression or analysis of variance/analysis of covariance, as appropriate.

Results: Included reviews contained between forty-two and seventy-six case series studies, involving 5 to 172,283 participants. Reporting of methodological features was poor and limited the analyses. In general, we found little evidence of any association between methodological characteristics and outcome. Sample size is used as an inclusion criterion in many reviews of case series but was consistently shown to have no relationship to outcome in all analyses. A prospective approach was not associated with outcome. Insufficient data were available to explore consecutive recruitment. Mixed results were shown for length of follow-up, independence of outcome measurement, and publication date.

Conclusion: We found little evidence to support the use of many of the factors included in tools used for quality assessment of case series. Importantly, we found no relationship between study size and outcome across the four examples studied. Isolated examples of a potentially important relationship between other methodological factors and outcome were shown, for example, blinding of outcome measurement, but these examples were not shown consistently across the small number of examples studied. Further research into the determinants of quality in case series studies is required to support health technology assessment.

Planning for a cost-effective mix of providers is an important aspect of health care reform. Currently, there are too many specialists and insufficient numbers of primary care providers. Nurses in advanced practice roles are an important resource for expanding the nation's supply of primary care providers.

In the past decade the prevention and management of prematurity have begun to be addressed with more appropriate designs. A few strategies—very few—can now be recommended. A few, some widely implemented, can be abandoned. The risks and benefits of most interventions still require clarification.