The cost-effectiveness of a medical intervention at the population level may deviate from that reported for evaluations at the patient level. It is important for researchers and decision makers to know about the relevance of externalities, phasing-in effects, treatment effectiveness in the community, capacity issues, and different time perspectives in the evaluation of an intervention.

Physician's Desk Reference and Database is a Finnish collection of computerized primary care guidelines. The program has been in clinical use since 1989. For this study, a function producing a log file of all searches was added to the program. Two hundred twenty-seven users returned log files that contained 15,267 searches. The users made 3.12 (range 1–10.4) searches per day. The average time needed to find and read an article was 4.9 minutes. Sufficient facts were found in 71% of the searches. Dermatology was the most popular field of interest, followed by infectious diseases and cardiology.

Objectives: The aim of this study was to support health technology assessment (HTA) capacity building in Member States of the European Union with limited experience or without institutionalized HTA. The main output is a Handbook on HTA Capacity Building.

Methods: The methods used were worldwide surveys of (i) HTA organizations, (ii) information management units, and (iii) HTA educational programs. The results of two surveys (i & ii) were combined with expert opinion to produce the Handbook on HTA Capacity Building.

Results: Survey of HTA organizations (n = 41, response rate 35 percent). Most of the organizations were established by the government (61 percent), and all were not-for-profit. Working on HTA (80.5 percent) and doing research (63.4 percent) were the main lines of activity. Survey on information management units (n = 23, response rate 23 percent). Most (74.2 percent) of the responding HTA agencies reported having personnel dedicated to HTA information services. Survey on HTA educational programs (n = 48, response rate 60 percent). In total, nine Master of Science (MSc) programs were identified (three MSc in HTA and six MSc in HTA-related areas). Handbook on HTA Capacity Building. A group of twenty experts from thirteen countries developed the handbook. It consists of nine chapters focusing on HTA institutional development (structural setup, work processes, and visibility).

Conclusions: Setting up organizational structures and establishing effective HTA programs that guide key policy decisions is a challenging task. There are no standard models or pathways. “One size fits all” is not a useful principle because of the wide systemic and cultural differences between countries. The Handbook on HTA Capacity Building includes approaches for overall institutional development, especially in formulating objectives, setting up structures, and defining work processes.

Objectives: This study aims to provide the first estimates of the costs and effects of the large scale introduction of autologous transfusion technologies into the United Kingdom National Health Service.

Methods: A model was constructed to allow disparate data sources to be combined to produce estimates of the scale, costs, and effects of introducing four interventions. The interventions considered were preparing patients for surgery (PPS) clinics, preoperative autologous donation (PAD), intraoperative cell salvage (ICS), and postoperative cell salvage (PoCS).

Results: The key determinants of cost per operation are the anticipated level of reductions in blood use, the mean level of blood use, mean length of stay, and the cost of the technology. The results show the potential for considerable reductions in blood use. The greatest reductions are anticipated to be through the use of PPS and ICS. Vascular surgery, transplant surgery, and cardiothoracic surgery appear to be the specialties that will benefit most from the technologies.

Conclusions: Several simplifications were used in the production of these estimates; consequently, caution should be used in their interpretation and use. Despite the drawbacks in the methods used in the study, the model shows the scale of the issue, the importance of gathering better data, and the form that data must take. Such preliminary modeling exercises are essential for rational policy development and to direct future research and discussion among stakeholders.

Objectives: Health needs has attracted the interest of policy-makers, health economists, and health professionals as modern health services try to satisfy individual and population health needs to optimize resource utilization. Health needs can be assessed by administering various types of survey or interview-based instruments. If health needs are to be satisfied in changing health agendas in developed and developing countries, it is essential to employ valid and reliable tools. Despite the importance of needs assessment, no comprehensive review of tools is currently available. We carried out a literature search to define and categorize existing health needs assessment tools.

Methods: We reviewed medical and social search engines for items containing specific health needs–related words to identify needs tools across a range of specialties. Papers were reviewed in terms of design, subject matter, psychometric features, and method of administration method.

Results: Thirty-one employed in 52 studies including cancer, mental health, palliative care, multiple sclerosis, and cardiovascular disease tools were identified.

Conclusions: This report summarizes available health needs instruments in a range of diseases to assist researchers in accessing health needs resources more easily and to encourage further research in this field.

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

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

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

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

Objectives: This study aimed to produce valid patient-based UK National Health Service (NHS) costs for adults with cystic fibrosis to identify differences between hospital- and home-based treatments for infections.

Methods: A costing study was carried out in adults with cystic fibrosis (CF) in the United Kingdom, who required intravenous antibiotic treatments for respiratory infections, administered either at home or in the hospital. The perspective was that of the NHS hospital trust. Data were collected retrospectively for each patient for 1 year using clinical records. Data were collected for 116 adults with CF between 2000 and 2001, when 42,382 treatment days (454 courses) of intravenous antibiotics were administered; 213 courses with intention-to-treat at home and 241 courses with intention-to-treat in the hospital. The mean length of a course was 15.3 days.

Results: Patients who had >60 percent of courses at home over 1 year had a mean cost of £13,528, compared with £22,609 for patients who had >60 percent of courses in the hospital, and a mean cost of £19,927 for patients who had an equal mix of home and hospital care (p = .0001).

Conclusions: The key cost-generating events in CF respiratory infections are hospital admissions. Future studies assessing costs should concentrate on factors affecting admissions, length of stay, staff input, and alternative methods of home-care provision, rather than marginal effects, such as using different antibiotics.

Objectives: The aim of this study was to present the updated methodological guidelines for economic evaluations of healthcare interventions (drugs, medical devices, and other interventions) in Belgium.

Methods: The update of the guidelines was performed by three Belgian health economists following feedback from users of the former guidelines and personal experience. The updated guidelines were discussed with a multidisciplinary team consisting of other health economists, assessors of reimbursement request files, representatives of Belgian databases and representatives of the drugs and medical devices industry. The final document was validated by three external validators that were not involved in the previous discussions.

Results: The guidelines give methodological guidance for the following components of an economic evaluation: literature review, perspective of the evaluation, definition of the target population, choice of the comparator, analytic technique and study design, calculation of costs, valuation of outcomes, definition of the time horizon, modeling, handling uncertainty and discounting. We present a reference case that can be considered as the minimal requirement for Belgian economic evaluations of health interventions.

Conclusions: These guidelines will improve the methodological quality, transparency and uniformity of the economic evaluations performed in Belgium. The guidelines will also provide support to the researchers and assessors performing or evaluating economic evaluations.

Objectives: The English National Horizon Scanning Centre routinely scans thirty-five sources to identify new and emerging health technologies. The aim of the study was to develop and apply evaluation criteria and scores to assess the value of sources, and to identify a cutoff score below which sources would be recommended for removal from routine horizon scanning. Criteria to evaluate each source scanned could result in a more efficient approach in the selection process.

Methods: Evaluation criteria were developed following a review of the literature and discussions with horizon analysts. Proposed criteria were piloted on a random selection of six sources, and then applied to all thirty-five sources. The criteria were assessed using the Analytic Hierarchy Process.

Results: Eight criteria were identified as being most relevant for assessing the value of scanning sources. The three most important (primary) criteria were coverage (approximate percentage of relevant information), quality (reliable, accurate, objective), and efficiency (estimated time to identify one potentially significant health technology or other relevant information). Seven sources fell beneath the cutoff score and were recommended for removal from routine scanning.

Conclusions: The criteria were considered useful in the assessment of current sources, and have the potential to be used to assess new ones. These criteria may be useful for other horizon scanning centers to pilot and validate.

Objective: To evaluate the appropriateness of the use of hip joint replacements (HJRs) using explicit criteria developed by an expert panel.

Methods: Observational study. Nine hundred ninety-seven patients from five hospitals with a diagnosis of osteoarthritis, avascular necrosis, hip fracture, or revision who were undergoing HJR were consecutively included in the study during a 1-year period. The appropriateness of the indication was judged by explicit criteria. Complications were recorded at the time of the intervention and 3 months postoperatively.

Results: Of the 1,030 interventions, 604 were for osteoarthritis, 31 avascular necrosis, 191 fractures, and 204 revisions. No differences were found among the hospitals for the main clinical and patient variables. Indications for surgery were considered appropriate in 59% of cases, uncertain in 32%, and inappropriate in 8%, mainly in the osteoarthritis group. Differences were found in the rates of appropriateness among some centers. The complication rate did not differ among the groups based on the level of appropriateness of the procedure.

Conclusions: The appropriate use of HJR, as measured by the criteria established by the panel, identified a moderate percentage of inappropriate indications. Those equivocal and inappropriate cases demand further studies to identify patients with an adequate risk-to-benefit ratio from this procedure.

Health care providers are increasingly concerned about the rising costs of the total health care system, which is now approaching 10–12% of the gross national product (GNP). The long-term decrease in birth rates and the prolonged mean length of life of the population shifts the age pyramid to older ages, bringing a higher demand for health care at increasing costs. Another explanation for the rising health care expenditure is the application of modern medical technologies in the treatment of the terminally or critically ill at any age. It has been estimated that 1 of every 7 health care dollars is now consumed by patients who are within 6 months of the end of their lives (5).

Technology assessment involves application of the scientific method to the practice of medicine. Finding all of the assessment reports in a given field is not an easy task. Proper evaluation of those assessments requires the conduct of a prospective experiment in which the sources and results are blinded when the choice is made of papers to exclude and to include, and the process should be carried in duplicate. There are several available data bases for carrying out the search, but because of problems they should be supplemented by reference to the bibliographies of pertinent published articles. Clinical trials included in meta-analyses should be graded by quality and thus facilitate sensitivity analyses. Attention must be paid to the possibility of publication bias. Finally, the advent of meta-analysis makes it desirable to begin randomized controlled trials in areas of uncertainty, even when there is no possibility that individual investigators will encounter enough patients to draw valid conclusions.

Pharmaceutical regulators and healthcare reimbursement authorities operate in different intellectual paradigms and adopt very different decision rules. As a result, drugs that have been licensed are often not available to all patients who could benefit because reimbursement authorities judge that the cost of therapies is greater than the health produced. This finding creates uncertainty for pharmaceutical companies planning their research and development investment, as licensing is no longer a guarantee of market access. In this study, we propose that it would be consistent with the objectives of pharmaceutical regulators to use the Net Benefit Framework of reimbursement authorities to identify those therapies that should be subject to priority review, that it is feasible to do so and that this would have several positive effects for patients, industry, and healthcare systems.

Objectives: Pharmaceutical reimbursement agencies’ processes and methods of appraisal vary across countries. The objective of this study was to examine the contribution of formal health economic analysis in a process using such analysis in Scotland in comparison to a process not routinely using such analysis in France.

Methods: A framework for classifying reimbursement systems was used to analyze the two systems. A typology of recommendation was defined and a qualitative analysis of decisions on a sample of medicines appraised by both reimbursement agencies was conducted. Reasons for differences in recommendations were analyzed and case studies selected to illustrate the common reasons.

Results: Thirty-nine common medicines appraised by both agencies were identified between 2005 and 2010, treating a variety of diseases for which the Scottish Medicines Consortium tended to provide more restrictive, or did not recommend, listing. Similarities in clinical evidence submitted to the respective reimbursement committees were observed. Differences in recommendation can be explained by a combination of the manufacturer's freedom to set price and the incentives provided by the consideration of health economic analysis and quality of life, alongside differences in relevant comparators, relevant outcomes, treatment guidelines, and the propensity to use network meta-analysis, in decision making.

Conclusions: This study provides some explanations and hypotheses for the differences observed in recommendations for a selected sample of medicines with regards to differences in appraisal processes and methods adopted. Further research using larger datasets may allow stakeholders to assess the impact of such differences on the efficient use of health resources.

Japanese surgeons have performed only one heart transplant in the quarter century since the procedure was developed. Possessing the requisite training and technology, transplant surgeons have been stymied by several factors that elude professional and political solution. Most critically, the lack of a brain death standard limits the availability of transplantable organs. Mistrust of the medical profession, traditional outlooks on death, and the primacy placed on consensual decision making have fueled debate about brain death and transplantation. Volatile and value laden, these issues have overwhelmed the discussion of health care resources, equal access to high-technology medical procedures, and insurance coverage for transplantation.

The direct and indirect costs of acute myeloid leukemia were estimated for Sweden in 1989. The calculated total cost was SEK 460 million. Nearly half of the costs, or 1.7 million per patient diagnosed, were indirect costs due to premature mortality. Direct costs of relapses and indirect costs of mortality represent costs due to the absence of completely curable therapy. They also represent potential cost savings that could be ootained after introduction of new treatment options in the future.

New models for determining the cost of care and length of stay in the neonatal intensive care unit (NICU) were developed using financial and clinical data from 588 admissions to our NICU. The model for determining costs explained 71% of the variability in total hospital costs. Models such as the ones developed in this study can be used to compare costs in different institutions, determine temporal trends in costs, and examine the financial impact of using new technologies. Such models can also be useful components of a rational prospective pricing system for the NICU.

Objectives: This work has investigated under what conditions cost-effectiveness data from a cluster randomized trial (CRT) are suitable for analysis using a cluster-adjusted nonparametric bootstrap. The bootstrap's main advantages are in dealing with skewed data and its ability to take correlations between costs and effects into account. However, there are known theoretical problems with a commonly used cluster bootstrap procedure, and the practical implications of these require investigation.

Methods: Simulations were used to estimate the coverage of confidence intervals around incremental cost-effectiveness ratios from CRTs using two bootstrap methods.

Results: The bootstrap gave excessively narrow confidence intervals, but there was evidence to suggest that, when the number of clusters per treatment arm exceeded 24, it might give acceptable results. The method that resampled individuals as well as clusters did not perform well when cost and effectiveness data were correlated.

Conclusions: If economic data from such trials are to be analyzed adequately, then there is a need for further investigations of more complex bootstrap procedures. Similarly, further research is required on methods such as the net benefit approach.