Objectives: The aim of this study is to determine the relative value and contribution of searching different sources to identify adverse effects data.

Methods: The process of updating a systematic review and meta-analysis of thiazolidinedione-related fractures in patients with type 2 diabetes mellitus was used as a case study. For each source searched, a record was made for each relevant reference included in the review noting whether it was retrieved with the search strategy used and whether it was available but not retrieved. The sensitivity, precision, and number needed to read from searching each source and from different combinations of sources were also calculated.

Results: There were 58 relevant references which presented sufficient numerical data to be included in a meta-analysis of fractures and bone mineral density. The highest number of relevant references were retrieved from Science Citation Index (SCI) (35), followed by BIOSIS Previews (27) and EMBASE (24). The precision of the searches varied from 0.88% (Scirus) to 41.67% (CENTRAL). With the search strategies used, the minimum combination of sources required to retrieve all the relevant references was; the GlaxoSmithKline (GSK) website, Science Citation Index (SCI), EMBASE, BIOSIS Previews, British Library Direct, Medscape DrugInfo, handsearching and reference checking, AHFS First, and Thomson Reuters Integrity or Conference Papers Index (CPI).

Conclusions: In order to identify all the relevant references for this case study a number of different sources needed to be searched. The minimum combination of sources required to identify all the relevant references did not include MEDLINE

Objectives: This study reports the cost-effectiveness of interventions with nonsignificant differences in effect, and considers reporting of cost-effectiveness in situations where nonsignificant differences arise in some but not all end points.

Methods: Data on costs and effects associated with three end points (adequate assessment, risk factors, and life-years) were derived from a trial of methods to promote secondary prevention of coronary heart disease. Incremental cost per life-year gained figures were calculated, and the uncertainty around these was displayed on cost-effectiveness planes in the form of ellipses.

Results: There was a significant difference in one of the intermediate end points (adequate assessment) but nonsignificant differences in the other intermediate end point (risk factors) and the final end point (life-years). Estimation of cost per life-year figures revealed the cost-effectiveness of the interventions to be unfavorable.

Conclusions: Cost-effectiveness ratios based on final end points should be calculated even in situations where nonsignificant differences in life-years arise, to avoid publication bias and to provide decision makers with useful information. Uncertainty in the incremental cost-effectiveness ratios should be estimated and presented graphically.We thank Rebecca Turner for comments on an earlier draft; Professor Paul Fenn for permission to adapt an existing model; and Dr. Andy Briggs for advice on interpretation and presentation of results and comments on an earlier draft. In addition to the authors of the paper, the ASSIST trial collaborative group includes G. Fowler, E. Fullard, A. Fuller, M. Murphy, A. Neil, T. Schofield, R. Turner, S. Thompson, F. Wells, R. Wiles, and L. Youngman. This research was funded by a grant from the national research and development program grant of the NHS Executive.

Objectives: The aims of this study were to estimate preferences and willingness-to-pay (WTP) for genetic screening for CYP2D6 polymorphisms among a group of former and currently depressed patients.

Methods: A Web-based discrete choice questionnaire was sent to 89 respondents, age 18–65. Four attributes were included: (i) shifts in antidepressant medication before symptom relief, (ii) time with antidepressant medication without symptom relief, (iii) time with antidepressant medication without symptoms but with adverse side-effects, (iv) cost of genetic screening. We used a switching model with two scenarios, one representing patients’ own treatment history and the other a treatment scenario with genetic screening.

Results: In a main-effects model involving the four attributes all coefficients had the expected sign, indicating that as the number of shifts, price or time without symptom relief, and/or dosage-adjustments increased, the likelihood of choosing the screening test decreased. Price and number of shifts in medicine were significant. Marginal WTP for 5 percent probability of a reduction of one in antidepressant shifts was DKK2,599 (€350).

Conclusions: Patients value reductions in shifts in antidepressants and price when choosing between genetic screening and no screening. They do not focus on how the reductions are provided, nor do they value the genetic information the test provides irrespective of its effect on outcome. Given, that the test is able to provide a reduction of one shift in the number of antidepressant shifts with a probability of 5 percent, WTP for the test exceeds its cost.

Objectives: In this study, we explored crucial factors that explain a person's attitude toward and his or her assessment of telemedical systems. Special focus lies on the link between the perspective of physicians (telemedicine users) and technicians (telemedicine designers) to find potential barriers hindering the broad application of telemedical systems in hospitals and doctors’ offices.

Methods: A survey among medical professionals (n = 34), technical professionals (n = 39), and a control group (n = 44) was conducted. The collected data were assessed in terms of domain knowledge, attitudes toward telemedicine, and potential implementation barriers.

Results: Participants favored the conventional method over telemedical monitoring in regards to privacy, security, and time efficiency. In contrast, telemedicine was preferred with reference to efficiency of data analysis, long-term care, and emergency adequacy. Significant differences between the professional groups were found regarding perceived cost effectiveness, patients’ compliance, privacy protection, and false alarm sensitivity. Medical professionals exhibited the most reluctance toward using telemedical treatments.

Conclusions: The perceived drawbacks are attributed to a general uncertainty about the reliability of telemedical systems, in combination with concerns about personal data privacy, security, and loss of control. The reported fear of not being able to correctly use and handle the systems assumes a low usability of devices. To acquaint medical professionals with the benefits and limitations of telemedical systems, telemonitoring and tele-treatment should be included in the education of medical personnel at an early stage.

Objectives: Major depression is common in elderly patients. Interpersonal psychotherapy (IPT) is a potentially effective treatment for depressed elderly patients. The objective of this study was to evaluate the cost-effectiveness of IPT delivered by mental health workers in primary care practices, for depressed patients 55 years of age and older identified by screening, in comparison with care as usual (CAU).

Methods: We conducted a full economic evaluation alongside a randomized controlled trial comparing IPT with CAU. Outcome measures were depressive symptoms, presence of major depression, and quality of life. Resource use was measured from a societal perspective over a 12-month period by cost diaries. Multiple imputation and bootstrapping were used to analyze the data.

Results: At 6 and 12 months, the differences in clinical outcomes between IPT and CAU were small and nonsignificant. Total costs at 12 months were €5,753 in the IPT group and €4,984 in the CAU group (mean difference, €769; 95 percent confidence interval, −2,459 – 3,433). Cost-effectiveness planes indicated that there was much uncertainty around the cost-effectiveness ratios.

Conclusions: Based on these results, provision of IPT in primary care to elderly depressed patients was not cost-effective in comparison to CAU. Future research should focus on improvement of patient selection and treatments that have more robust effects in the acute and maintenance phase of treatment.

Objectives: This study aims to compare the impact of two different sources of resource use, self-report versus care provider registrations, on cost and cost utility.

Methods: Data were gathered for a cost-effectiveness study performed alongside a 2-year randomized controlled trial evaluating the effect of an INTERdisciplinary COMmunity-based management program (INTERCOM) for patients with chronic obstructive pulmonary disease (COPD). The program was offered by physiotherapists, dieticians and respiratory nurses. During the 2-year period, patients reported all resource use in a cost booklet. In addition, data on hospital admissions and outpatient visits, visits to the physiotherapist, dietician or respiratory nurse, diet nutrition, and outpatient medication were obtained from administrative records. The cost per quality-adjusted life-year (QALY) was calculated in two ways, using data from the cost booklet or registrations.

Results: In total, 175 patients were included in the study. Agreement between self-report and registrations was almost perfect for hospitalizations (rho = 0.93) and physiotherapist visits (rho = 0.86), but above 0.55, moderate, for all other types of care. The total cost difference between the registrations and the cost booklet was 464 euros with the highest difference for hospitalizations 386 euro. Based on the cost booklet the cost difference between the treatment group and usual care was 2,444 euros (95 percent confidence interval [CI], −819 to 5,950), which resulted in a cost-utility of 29,100 euro/QALY. For the registrations, the results were 2,498 euros (95 percent CI, −88 to 6,084) and 29,390 euro/QALY, respectively.

Conclusions: This study showed that the use of self-reported data or data from registrations effected within-group costs, but not between-group costs or the cost utility.

Governments may perform health technology assessment (HTA) in their roles as a regulator in the public interest, as a source of information for decision makers in the public and private sectors, and/or as a purchaser or provider of healthcare services. The U.S. government's roles in the health sector as a regulator, source of information, and purchaser and provider of services are influenced both by characteristics of that sector and by its stakeholders' effectiveness in influencing national health policy debates.

This review assesses the evidence regarding the effectiveness of bone density measurement (BDM) screening and related interventions—hormone replacement therapy (HRT) and intranasal salmon calcitonin (SCT[N])—in menopausal women to prevent fractures in later life. Data sources included systematic reviews of evidence and relevant primary studies identified through literature searches on MEDLINE and EMBASE. Study selection included trials of BDM screening programs, prospective studies exam-ining the predictive value of BDM, randomized controlled trials, cohort studies, and case-controlstudies of HRT and SCT(N). The evidence was evaluated using a classification system incorporatingstudy design and quality. Outcomes were measured in terms of relative risk of fracture for a 1 SDdecrease in bone mineral density below the age-adjusted mean, relative risks or odds ratios forfractures associated with treatments, and proportion of hip fractures potentially prevented by BDMscreening linked to treatments. Fair evidence from prospective cohort studies suggests that BDMcan predict the risk of fractures, but not with high accuracy. Fair evidence from low-quality randomizedcontrolled trials and observational studies suggests that HRT and SCT(N) are efficacious in preventingfractures. Good evidence supports the efficacy of these treatments in preserving bone mass, butthere is fair evidence that the effect wears off after cessation of therapy. There is little evidence onthe impact of screening menopausal women with BDM in association with HRT or SCT(N) treatment.Estimates based on combining existing evidence regarding the predictive value of BDM and efficacyof HRT suggests that 1–7% of hip fractures might be prevented.

Objectives: The Weight Loss Maintenance Trial (WLM) was a multicenter, randomized trial comparing two weight loss maintenance interventions, a personal contact (PC) program with primarily telephone-based monthly contacts, and an Internet-based program (interactive technology, IT), to a self-directed control group, among overweight or obese individuals at high cardiovascular risk. This study describes implementation costs of both interventions as well as IT development costs.

Methods: Resources were micro-costed in 2006 dollars from the primary perspective of a sponsoring healthcare system considering adopting an extant intervention, rather than developing its own. Costs were discounted at 3 percent annually. Length of trial participation was 30 months (randomization during February–November 2004). IT development costs were assessed over 36 months. Univariate and multivariate, including probabilistic, sensitivity analyses were performed.

Results: Total discounted IT development costs over 36 months were $839,949 ($2,414 per IT participant). Discounted 30-month implementation costs for 342 PC participants were $537,242 ($1,571 per participant), and for 348 IT participants, were $214,879 ($617 per participant). Under all plausible scenarios, PC implementation costs exceeded IT implementation costs.

Conclusions: Costs of implementing and operating an Internet-based intervention for weight loss maintenance were substantially less than analogous costs of an intervention using standard phone and in-person contacts and are of a magnitude that would be attractive to many health systems, subject to demonstration of cost-effectiveness.

Although medical malpractice litigation in the United States has generated extensive professional and scholarly attention, few analyses of the issue have explored its underlying causes. This essay develops and employs an historical framework to explain the late 20th century phenomenon and concludes that widespread medical malpractice suits are the result of a combination of short-term topical causes and long-term cultural changes that are ignored or left untouched by most reform efforts. Most importantly, however, the development and proliferation of new and improved medical technologies has played a pivotal role throughout the entire history of the litigation, an effect that has become most prominent and important in the last third of the 20th century.

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

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

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

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

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

Objectives: There is evidence that breastmilk feeding reduces mortality and short and long-term morbidity among infants born too soon or too small. The aim of this study was to evaluate the cost-effectiveness of enhanced staff contact for mothers with infants in a neonatal unit with a birth weight of 500–2,500 g from the perspective of the UK National Health Service.

Methods: A decision-tree model linked clinical outcomes with long-term health outcomes. The study population was divided into three weight bands: 500–999 g, 1000–1,749 g, and 1,750–2,500 g. Clinical and resource use data were obtained from literature reviews. The measure of benefit was quality-adjusted life-years. Uncertainty was evaluated using cost-effectiveness acceptability curves and sensitivity analyses.

Results: The intervention was less costly and more effective than the comparator in the base–case analysis for each birth weight group. The results were quite robust to the sensitivity analyses performed.

Conclusions: This is the first economic evaluation in this complex field and offers a model to be developed in future research. The results provide preliminary indications that enhanced staff contact may be cost-effective. However, the limited evidence available, and the limited UK data in particular, suggest that further research is required to provide results with confidence.

Objectives: After 4 years of deepening recession, Argentina's economy plummeted after default in 2002. This crisis critically affected health expenditures and triggered acute rationing. Our objective was to explore health decision-makers' knowledge and attitudes about economic evaluations (EE) and whether health technology assessment (HTA) were increasingly used for decision making.

Methods: A qualitative design based on semistructured interviews and focus groups was used to explore how decision makers belonging to different health sectors implement resource allocation decisions.

Results: Informants were mostly unaware of EE. The most important criteria mentioned to adopt a treatment were evidence of effectiveness, social/stakeholder demand, or resource availability. Despite general positive attitudes about EE, knowledge was rather limited. Although cost considerations were widely accepted by purchasers and managers, clinicians argued about these issues as interfering with the doctor–patient relationship. Other important perceived barriers to HTA use were lack of confidence in the transferability of studies conducted in developed countries and institutional fragmentation of the Argentine healthcare system. The new macroeconomic context was cited as a justification of implicit rationing measures. Although explicit priority setting was implemented by many purchasers and managers, HTA was not used to improve technical and/or allocative efficiency.

Conclusions: The crisis seems to be a strong incentive to extend the use of HTA in Argentina, provided decision makers are aware as well as involved in the generation of local studies.

Objectives: The objective of this study was to conduct a cost-effectiveness analysis of Herceptin® from the hospital's point of view. This new biotechnological pharmaceutical is a humanized monoclonal antibody that targets the HER2 receptor, an important anti-cancer target.

Methods: A cost model with standard diagnostic and treatment options for breast cancer was set up for a Belgian university hospital in close collaboration with its specialists. Direct and indirect costs were calculated for each diagnostic and treatment option using the micro-costing method. Effectiveness was estimated through a literature study. The model allowed us to take cost consequences in other stages of the model into account and to calculate changes in monthly treatment costs from different “starting points.” With an incremental cost-effectiveness analysis, differences in costs and effectiveness with and without Herceptin® were compared.

Results: Over the complete treatment period from diagnosis until the metastatic phase, monthly costs for the hospital rose from €85.07 to €90.35 for stage I diagnosed breast cancer when adding Herceptin® to the model. In the metastatic phase alone, these costs rose from €1,132.33 to €1,256.23. With Herceptin®, we found an extra cost of €3,981.44 per extra life-month.

Conclusions: This cost-effectiveness ratio was rather high, because Herceptin® was quite expensive and the product was additive in its current use and did not replace existing treatments. Future research will concentrate on alternative applications of Herceptin® based on ongoing Herceptin® trials and expert opinions.

There has been an increased focus on the relationship between health technology assessment (HTA) and regulatory assessments and how regulatory, HTA and coverage bodies, and industry can work better together to improve efficiency and alignment of processes. There is increasingly agreement across sectors that improved communication and coordination could contribute to facilitating timely patient access to effective, affordable treatments that offer value to the health system. Discussions on aspects of this relationship are being held in different forums and various forms of coordination and collaboration are being developed or piloted within several jurisdictions. It is therefore both timely and of value to stakeholders to describe and reflect on current initiatives intended to improve interactions between regulatory, HTA and coverage bodies, and industry. Drawing on 2011 meetings of the HTAi Policy Forum and the Center for Innovation in Regulatory Science (CIRS), this study aims to describe and compare initiatives, and point to success factors and challenges that are likely to inform future work and collaboration.

Background: Despite the growing medical and public interest in reducing exposure to allogeneic blood, little is known about the use of alternatives to allogeneic transfusion. This study was conducted to determine the availability of these technologies in Canadian hospitals and was undertaken under the auspices of the International Study of Peri-Operative Transfusion (ISPOT), a 10-country study of the effectiveness of, attitudes toward, and practices related to the use of alternatives to allogeneic transfusion.

Methods: A cross-sectional national mail survey of Canadian hospitals with greater than 50 medical/surgical beds. Chiefs of anesthesia, surgery, and the divisions of cardiac, orthopedic, vascular, and urology were initially mailed a brief postcard asking which of seven technologies were used in their center. This was then followed up with a one-page questionnaire asking how frequently the technologies were used, their thoughts on the appropriateness of the use of the technologies, barriers to their greater use, and reasons for nonuse of the technologies.

Results: Response rates to the postcard survey ranged from 70%–98%, depending on the technology and type of surgery, and ranged from 27%–53% for the follow-up questionnaire. All technologies were used most frequently in cardiac surgery. Aprotinin, tranexamic acid, aminocaproic acid, desmopressin, and cell salvage were reported used in over 70% of cardiac surgery centers. Of these, tranexamic acid and cell salvage were the only ones used routinely in some centers. Acute normovolemic hemodilution and erythropoietin were used in 45% and 20% of cardiac centers, respectively. The drugs were used in less than 15% of orthopedic, vascular, and urologic divisions, with the exception of desmopressin in urologic and vascular surgery and aminocaproic acid in urologic surgery. The techniques of cell salvage and acute normovolemic hemodilution were used in 30%–45% of these divisions, with the exception of cell salvage, which was used in less than 15% of urology units. In more than 60% of cases, the technologies were considered to be used “about right,” although an important minority felt that they were underused.

Conclusions: In general, alternatives to perioperative allogeneic transfusion were rarely used except in cardiac surgery.

Leukoreduction, filtering white blood cells from transfusion blood, effectively avoids leukocyte-related complications of blood transfusion. The technology has proven its relative cost-effectiveness for specific patient populations. With the advent of variant Creutzfeldt–Jakob disease, a transmittable spongiform encephalopathy caused by mad cow disease (bovine spongiform encephalopathy), the hard hit United Kingdom introduced universal leukoreduction for all patients as a precaution for transmission of prions in 1999. This costly policy was followed by many other countries, in the absence of much evidence of an actual health problem or of a more than presumed effectiveness of leukoreduction in preventing prion transmission. The core problem proved to be legal. The blood banks are legally accountable for blood safety. This accountability is absolute, based on avoidance of all possible risks, regardless of costs. This strategy leads to inefficiencies in health care: (i) blood safety management is guided by available rather than cost-effective technology, and (ii) private insurance premiums for civil liability are sharply increasing, while they are in no way related to the expected returns and the high and increasing blood safety. A rational safety policy is to be optimal, taking into account costs and effects of the safety procedures. This issue will need an open discussion with the general public of the real risks and a clear and unambiguous definition of proportionality in the precautionary principle, based on the European law.

The issue of health benefits coverage—and its relation to health technology assessment (HTA)—has gained increasing attention in recent years. Economic constraints on health care, as well as the rapid pace of technological change, have forced European countries to face difficult choices in providing such care. The active use of coverage decision making has been proposed as a tool to help rationalize health care, and HTA has been advocated as a necessary activity to improve coverage decisions.

Objectives: In 2012, Colombia experienced an important institutional transformation after the establishment of the Health Technology Assessment Institute (IETS), the disbandment of the Regulatory Commission for Health and the reassignment of reimbursement decision-making powers to the Ministry of Health and Social Protection (MoHSP). These dynamic changes provided the opportunity to test Multi-Criteria Decision Analysis (MCDA) for systematic and more transparent resource-allocation decision-making.

Methods: During 2012 and 2013, the MCDA framework Evidence and Value: Impact on Decision Making (EVIDEM) was tested in Colombia. This consisted of a preparatory stage in which the investigators conducted literature searches and produced HTA reports for four interventions of interest, followed by a panel session with decision makers. This method was contrasted with a current approach used in Colombia for updating the publicly financed benefits package (POS), where narrative health technology assessment (HTA) reports are presented alongside comprehensive budget impact analyses (BIAs).

Results: Disease severity, size of population, and efficacy ranked at the top among fifteen preselected relevant criteria. MCDA estimates of technologies of interest ranged between 71 to 90 percent of maximum value. The ranking of technologies was sensitive to the methods used. Participants considered that a two-step approach including an MCDA template, complemented by a detailed BIA would be the best approach to assist decision-making in this context. Participants agreed that systematic priority setting should take place in Colombia.

Conclusions: This work may serve as the basis to the MoHSP on its interest of setting up a systematic and more transparent process for resource-allocation decision-making.