Objectives: Research has shown that effectiveness, cost-effectiveness, and severity of illness each play a role in drug reimbursement decisions. However, the role of budget impact in such decisions is less obvious. Policy makers almost always demand a budget impact estimate yet seem reluctant to formally include budget impact as a rationing criterion. Health economists even reject budget impact as a legitimate criterion. For these reasons, it is important to examine its use in rationing decisions, and rationales underlying its use.

Methods: We trace several rationales supporting the use of budget impact through a literature review, supplemented by semistructured interviews with eleven key stakeholders involved in drug reimbursement decisions in the Netherlands.

Results: Budget impact arguments are used in certain instances, although policy makers appear uncomfortable with its use because well described rationales still are lacking. In addition, we identify the following rationales to support budget impact as a rationing criterion: opportunity costs, loss aversion, uncertainty and equal opportunity.

Conclusions: Budget impact plays a role in drug reimbursement decisions and has rationales to support its use. However, policy makers do not easily admit that they consider budget impact and are even reluctant to explicitly use budget impact as a formal criterion. A debate would strengthen the theoretical foundation of budget impact as a legitimate criterion in the context of drug reimbursement decisions. Such discussion of budget impact's role will also enhance policy-makers' accountability.

Objectives: The aim of this study was to estimate direct and indirect excess costs attributable to stroke in Sweden in 2009 and to compare these with similar estimates from 1997.

Methods: Data on first-ever stoke admissions in the first half of 2009 from the Swedish national stroke register (RS) were used for cost calculations and compared with results from 1997 also using RS data. A societal perspective was taken including the acute and follow-up phase, rehabilitation, stroke re-admissions, drugs, home- and residential care services for activities of daily life (ADL) support, and indirect costs for premature death and productivity losses (2009 prices). Survival was extrapolated to estimate the lifetime present value cost of stroke.

Results: The societal lifetime present value cost for stroke in 2009 was €68,800 per patient (ADL support: 59 percent; productivity losses: 21 percent). Women had higher costs than men in all age groups as a result from greater need for ADL support. Patients treated at a stroke unit indicated low incremental cost per life-year gained compared with those who had not. The total lifetime cost increased between 1997 and 2009. Hospitalization costs per patient were stable, while long-term costs for home- and residential care services increased.

Conclusions: Changes in patient characteristics, longer expected survival, and possibly in the Swedish stroke care, have led to higher annual and lifetime costs per patient in 2009 compared with 1997. A comprehensive national stroke care performance register like RS may be suitable for health economic assessments.

Objectives: The frequency and consequences of major bleeding associated with anticoagulant prophylaxis for prevention of venous thromboembolism is examined.

Methods: We conducted a systematic review and meta-analysis of controlled trials that reported rates of major bleeding after pharmaceutical thromboprophylaxis in patients undergoing major orthopedic surgery. Thromboprophylactic agents were divided into four groups:warfarin/other coumarin derivatives (WARF), unfractionated heparin (UFH), low molecular weight heparin (LMWH), and pentasaccharide (PS). Meta-analysis was conducted comparing LMWH with each of WARF, UFH, and PS. The frequency of re-operation due to major bleeding was reviewed and combined with published costs to estimate the mean cost of managing major bleeding events in these patients.

Results: Twenty-one studies including 20,523 patients met inclusion criteria for the meta-analysis. No evidence of significant between-trial heterogeneity in risk ratios was found. Combined (fixed effects) relative risks (RR) of major bleeding compared with LMWH were WARF – RR 0.59 (95 percent confidence interval [CI], 0.44–0.80); UFH – RR 1.52 (95 percent CI, 1.04–2.23); PS – RR 1.52 (95 percent CI, 1.11–2.09). Seventy-one studies including 32,433 patients were included in the review of consequences of major bleeding. We estimated that the average cost of major bleeding is $113 per patient receiving thromboprophylaxis.

Conclusions: LMWH results in fewer major bleeding episodes than UFH and PS but more than WARF. These events are costly and clinically important.

This article reviews the methods of economic evaluation of health technology. The different forms of evaluation are outlined and the importance of assessing costs and benefits at the margin stressed. The approaches used by economists to analyze priorities for health technologies are discussed and a number of key managerial and policy issues identified. It is concluded that, while further methodological improvements can be made, a clear methodology for economic evaluation of health technologies has been established. However, attention needs to be paid to the timeliness and relevance of economic evaluation in order to ensure its maximum impact

Objectives: Interferon beta-1b has recently become available for the treatment of secondary progressive multiple sclerosis (SPMS). This study aims at estimating the cost-effectiveness of this new treatment that has been shown in a clinical trial to reduce the progression of the disease. Effectiveness is measured as the number of quality-adjusted life-years (QALYs) gained from the reduction in progression. Because the clinical trial period will only capture part of the treatment's effect in terms of QALYs gained, since benefits achieved during the trial will have an effect beyond it, the cost-effectiveness analysis involves modeling over the longer term using complementary data.

Methods: A Markov model with states based on disability expressed by EDSS scores was used. Transition probabilities were calculated directly from clinical trial data for the first 3 years and then extrapolated to 10 years. Mean costs and utilities for each Markov state were calculated from a population-based cross-sectional study in Sweden.

Results: The incremental cost per QALY is SEK 342,700 (US $39,250;US $1 = SEK 8.73, March 10, 2000) when all costs (direct, informal care, and indirect) are included (discounted 3%). When indirect costs are excluded, the cost per QALY is SEK 542,000 ($62,100).

Conclusions: Cost-effectiveness analysis in SPMS requires that the effect of treatment beyond clinical trials be included. Also, analysis should be done from a societal perspective, since many of the costs occur outside the healthcare system. The cost-utility ratios estimated in this analysis are at or below the mean threshold value indicated in a recent survey of health economists ($60,000).

This article considers technology assessment (TA) to be a comprehensive form of policy research. Technology assessment must then have a relation to policy-making; in the area of health care, TA must relate to such political decisions as resource allocation. Since policies are determined politically, i.e., by factors such as power and influence, technology assessment is, or should be, part of a political process. Technology assessment seems to be developing predominantly as a technical and professional activity, carried out in centers with no relation to the policy-making process. While the impact of technology on health, as well as such broader impacts as those on financial costs, is a key concern, political considerations and political decision-making must always be an important dimension in health care TA.

Canada's health system is a unique combination of public financing and private provision. With the significant government role in financing health services, health technology assessment (HTA) has found a ready audience as a form of policy research. In addition, Canada has been a leader in HTA and is entering a phase of deepening and maturation of HTA activities. The relative absence of dramatic change in the overall health system, coupled with public faith in the Canadian approach has been favorable to HTA's development in Canada. Emerging issues, beyond the demographic and economic pressures facing all Organisation for Economic Co-operation and Development health systems, include the convergence of assessment modalities and organizations for drug and nondrug technologies, increasing public concerns about the viability of Canada's approach to healthcare services, and the transition of HTA from an activity targeting macro-level policy makers to a management tool for healthcare facilities and providers.

This paper recounts the origins of consensus development as a means of technology assessment and traces the evolution of the process from its rather amorphous and variable beginnings to the codified and consistent model of today. Where appropriate, evaluative findings and anecdotal observations will be recounted.

Objectives: This study tested the reliability and validity of the Western Canada Waiting List Project priority criteria score (PCS) for prioritizing patients waiting for hip and knee arthroplasty.

Methods: Sixteen orthopedic surgeons assessed 233 consecutive patients at consultation for hip or knee arthroplasty. Measures included the PCS, a visual analogue scale of urgency (VAS urgency), and maximum acceptable waiting time (MAWT). Patients completed a VAS urgency, an MAWT, the Western Ontario McMaster Osteoarthritis Index (WOMAC), and the EQ-5D. Using correlational analysis, convergent and discriminant validity was assessed between similar constructs in the priority criteria and WOMAC. Median MAWTs were determined for five levels of urgency based on PCS percentiles. Internal consistency reliability was assessed with Cronbach's alpha.

Results: The sample of 233 patients (62 percent female) ranged in age from 18 to 89 years (mean, 66.3 years). A total of 45 percent were booked for hip and 55 percent for knee arthroplasty. Correlations were strong between the PCS and surgeon VAS urgency (r=.79) and weaker between patient and surgeon measures of VAS urgency (r=.24) and MAWT (r=.44). Correlation coefficients between similar constructs in the priority criteria and WOMAC ranged from 0.24 to 0.32 and were higher than those measuring dissimilar constructs. For decreasing levels of urgency, the median MAWT ranged from 10 to 12 weeks for surgeons and 4 to 12 weeks for patients. Cronbach's alpha was 0.79.

Conclusions: Results support the validity of the PCS as a measure of surgeon-rated urgency. Patients might be ranked differently with different prioritization measures.

Objectives: The aim of this study was to determine the insulin-delivery system and the attributes of insulin therapy that best meet patients' preferences, and to estimate patients' willingness-to-pay (WTP) for them.

Methods: This was a cross-sectional discrete choice experiment (DCE) study involving 378 Canadian patients with type 1 or type 2 diabetes. Patients were asked to choose between two hypothetical insulin treatment options made up of different combinations of the attribute levels. Regression coefficients derived using conditional logit models were used to calculate patients' WTP. Stratification of the sample was performed to evaluate WTP by predefined subgroups.

Results: A total of 274 patients successfully completed the survey. Overall, patients were willing to pay the most for better blood glucose control followed by weight gain. Surprisingly, route of insulin administration was the least important attribute overall. Segmented models indicated that insulin naïve diabetics were willing to pay significantly more for both oral and inhaled short-acting insulin compared with insulin users. Surprisingly, type 1 diabetics were willing to pay $C11.53 for subcutaneous short-acting insulin, while type 2 diabetics were willing to pay $C47.23 to avoid subcutaneous short-acting insulin (p < .05). These findings support the hypothesis of a psychological barrier to initiating insulin therapy, but once that this barrier has been overcome, they accommodate and accept injectable therapy as a treatment option.

Conclusions: By understanding and addressing patients' preferences for insulin therapy, diabetes educators can use this information to find an optimal treatment approach for each individual patient, which may ultimately lead to improved control, through improved compliance, and better diabetes outcomes.

Several new topical agents have been introduced recently and it can be expected that the treatment of glaucoma will change, depending on how effectively these agents control intraocular pressure (IOP). IOP is considered the major risk factor in the development of glaucomatous damage. In order to estimate the impact of these new agents on the cost of treating glaucoma, a simulation model was created to estimate the cost of treating patients with a recent diagnosis of primary open-angle glaucoma or ocular hypertension in different countries. The Markov model is based on retrospective chart reviews in different countries and calculates only cost, not outcome. Results are presented for France and the United Kingdom, where current treatment appeared to be comparable. Average one-year costs per patient with current treatment were FF2,389 (US $389) and £380 (US $627), respectively. Costs with the new treatments were lower than with current therapy.

Objectives: Previous research has suggested people with impaired fasting glucose (IFG) are less likely to develop Type 2 diabetes (T2DM) if they receive prolonged structured diet and exercise advice. This study examined the within-trial cost-effectiveness of such lifestyle interventions.

Methods: Screen-detected participants with either newly diagnosed T2DM or IFG were randomized 2:1 to intervention versus control (usual care) between February and December 2009, in Norfolk (UK). The intervention consisted of group based education, physiotherapy and peer support sessions, plus telephone contacts from T2DM volunteers. We monitored healthcare resource use, intervention costs, and quality of life (EQ-5D). The incremental cost per quality-adjusted life-year (QALY) gain (incremental cost effectiveness ratio [ICER]), and cost effectiveness acceptability curves (CEAC) were estimated.

Results: In total, 177 participants were recruited (118 intervention, 59 controls), with a mean follow-up of 7 months. Excluding screening and recruitment costs, the mean cost was estimated to be £551 per participant in the intervention arm, compared with £325 in the control arm. The QALY gains were –0.001 and –0.004, respectively. The intervention was estimated to have an ICER of £67,184 per QALY (16 percent probability of being cost-effective at the £20,000/QALY threshold). Cost-effectiveness estimates were more favorable for IFG participants and those with longer follow-up (≥4 months) (ICERs of £20,620 and £17,075 per QALY, respectively).

Conclusions: Group sessions to prevent T2DM were not estimated to be within current limits of cost-effectiveness. However, there was a large degree of uncertainty surrounding these estimates, suggesting the need for further research.

Objectives: Hemodialysis is a well-established treatment for 74 percent of end-stage renal disease (ESRD) patients in Greece. The purpose of this study is to provide an estimate of the direct cost of dialysis in a public hospital setting and an estimate of the loss of production for ESRD patients. The results will be useful for public health facility planning purposes.

Methods: A socioeconomic prevalence-based analysis was performed using micro-economic evaluation of health-care resources consumed to provide hemodialysis for ESRD patients in 2000. Lost productivity costs due to illness were estimated for the patient and family using the human capital approach and the friction method. Indirect morbidity costs due to absence from work and long-term were estimated, as well as mortality costs. Mean gross income was used for both patient and family.

Results: Total health-sector cost for hemodialysis in Greece exceeds €171 million, or €182 per session and €229 per inpatient day. There were 2,046 years lost due to mortality, and the potential productivity cost was estimated at €9.9 million, according to the human capital approach, and €303.000, according to the friction method. Total morbidity cost due to absence from work and early retirement was estimated at more than €273 million, according to the human capital approach, and €12.5, according to the friction method.

Conclusions: Providing hemodialysis care for 0.05 percent of the population suffering from ESRD absorbs approximately 2 percent of total health expenditure in Greece. In addition to the cost for the National Health System, production loss due to mortality and morbidity from the disease are also considerable. Promoting alternative technologies such as organ transplantation and home dialysis as well as improving hemodialysis efficiency through satellite units are strategies that may prove more cost-effective and psychologically advantageous for the patients.

Objective: This paper presents the method we used in programming research on the efficacy of therapeutic interventions for nine chronic benign pain disorders.

Methods: We started with an inventory to identify commonly applied interventions. For these interventions we searched the literature to identify gaps in evidence. First, we searched for recent reviews, of which we assessed the methodologic quality. If only reviews of poor or moderate quality were found, a new systematic review was recommended. When we found no recent reviews, we searched for randomized controlled trials (RCTs). If there was insufficient or inconsistent evidence, the performance of a new RCT was advised. This structured procedure resulted in a list with topics for which new systematic reviews were recommended and topics for which new RCTs were needed. These lists were the starting points for priority setting by four Centers for Pain Management and Research. All members of the centers were asked to state the priority of each topic. The resulting hierarchy of topics for their own center was discussed in a meeting in each center, giving participants the opportunity to elucidate their views and considerations.

Results: The final result was a robust priority list for the need of research (systematic reviews and RCTs) on chronic benign pain syndromes.

Discussion: The strength and weaknesses of this approach are discussed. This method of priority setting is by no means restricted to treatments for chronic pain; it is also a useful approach for programming research to enable evidence-based medicine in other fields of interest.

Objectives: The aim of this study was to define an effective early warning system, to identify and rank the characteristics of an effective early warning system for emerging health technologies, and to evaluate current early warning systems against these characteristics.

Methods: An iterative Delphi-type process with the thirteen members of the International Information Network on New and Changing Health Technologies (EuroScan). We synthesized key characteristics that network members had graded. Members were then asked whether these characteristics were present or fulfilled in their system.

Results: The definition of an effective early warning system developed was the following: a system that identifies innovations in the field of health technology likely to have a significant impact; and disseminates information relevant to the needs of the customer which is timely, so as to enable appropriate decision making (such as resource allocation), facilitate appropriate adoption, and identify further research requirements. Five primary and eleven secondary components of effective early warning systems were identified. The five primary characteristics concerned relevance, independence, resourcing, a clear pathway for the outputs to reach decision makers, and defined customers. Although the primary characteristics were present or fulfilled to some extent in the majority of evaluated early warning systems, there was considerable variability in the presence of the secondary characteristics in the evaluated systems.

Conclusions: Our study provides a definition for an effective early warning system and a shared understanding of the important characteristics and components of such systems. This work should provide guidance to those setting up new early warning systems as well as for those managing and reviewing current systems.

Objectives: To review and compare current Health Technology Assessment (HTA) activities for medical devices (MDs) across European HTA institutions.

Methods: A comprehensive approach was adopted to identify institutions involved in HTA in European countries. We systematically searched institutional Web sites and other online sources by using a structured tool to extract information on the role and link to decision making, structure, scope, process, methodological approach, and available HTA reports for each included institution.

Results: Information was obtained from eighty-four institutions, forty-seven of which were analyzed. Fifty-four methodological documents from twenty-three agencies in eighteen countries were identified. Only five agencies had separate documents for the assessment of MDs. A few agencies made separate provisions for the assessment of MDs in their general methods. The amount of publicly available HTA reports on MDs varied by device category and agency remit.

Conclusions: Despite growing consensus on their importance and international initiatives, such as the EUnetHTA Core Model®, specific tools for the assessment of MDs are rarely developed and implemented at the national level. Separate additional signposts incorporated in existing general methods guides may be sufficient for the evaluation of MDs.

Objectives: Industrial management principles could be used to improve the quality and efficiency of health care. In this study, we have evaluated the effects of a process management approach to trauma patient care. The major objective was to reduce the waiting times and increase the efficiency of the hospital.

Methods: Urgent surgery care was analyzed as an overall process. The process development followed the Plan-Do-Check-Act (PDCA) cycle and was based on statistical analysis of certain performance metrics. Data were collected from hospital databases and by personnel interviews. To develop the process, the anesthesia induction was performed outside the operating room, better process guidance was developed, and patient flow was reorganized. The transition time for these changes was 1 year (2002 to 2003).

Results: Waiting times decreased by 20.5 percent (p<.05), nonoperative times in the operating room were reduced by 23.1 percent (p<.001), and efficiency was increased by 9.7 percent (p<.001) after reengineering of the care process. Overtime hours decreased by 30.9 percent.

Conclusions: Managing urgent surgical care as a process can improve the productivity and quality of care without a need to increase personnel resources. The focus should be on reducing waiting times and waste times.

Background: There is a considerable gap between randomized clinical trials and implementing the results into practice. This is particularly relevant in the use of broad-spectrum antibiotics in hospitals. Hospital pharmacists can be effective vehicles for bridging this gap and promoting evidence-based medicine. To determine the most effective way of using the pharmacist in this role, a prospective cefotaxime intervention study was conducted with randomization incorporated into the design as well as patient-related therapeutic outcomes.

Methods: A total of 323 patients who were prescribed cefotaxime were randomized into an intervention or nonintervention group where only the former was challenged by pharmacists for inappropriate cefotaxime usage relative to hospital guidelines. The primary outcome was the appropriateness of cefotaxime prescribing between groups. Logistic regression analysis was then used to identify factors that were associated with successful clinical response.

Results: Overall, 94% of orders in the intervention group met cefotaxime dosage criteria compared with 86% in the control group (p = .018). However, there was no impact with respect to promoting cefotaxime use for an appropriate indication (81% vs. 80%; p = .67). There was a trend for improved clinical outcomes in patients who received cefotaxime within hospital guidelines (OR = 1.73; p = .31).

Conclusions: The pharmacist as a vehicle for promoting the appropriate use of broad-spectrum antibiotics in the acute care hospital setting can improve the dosing of such agents. However, several barriers to optimizing the impact of the pharmacist were implied by the data. Removing these barriers could increase the pharmacists' utility as an agent for improved patient care.

Objectives: Assessment of the appropriateness of screening programs involves consideration of the harms as well as the benefits. These harms include the risk of false-negative results, the consequences of which have remained underinvestigated. This paper reports the results of a systematic literature review that aimed to assess the medical psychological, economic, and legal consequences of false-negative results in national screening programs.

Methods: The review included a comprehensive literature search and contact with experts to identify relevant literature. Most studies that were identified presented only anecdotal evidence. However, thirteen studies presented quantitative information on medical consequences of false negatives, eight studies presented information on psychological consequences, and two studies presented information on economic consequences.

Results: The strength of evidence from most of the primary studies was low. There is some evidence, however, that false-negative results may have a large legal impact. There is also a consensus in the literature that false negatives may have a negative impact on public confidence on screening; evidence is however limited.

Conclusions: False negatives are evident even in high-quality screening programs. They may have the potential to delay the detection of breast and cervical cancer, but there is little evidence to help in assessing their psychological consequences. They also may lead to legal action being taken by those affected and may reduce public confidence in screening. Their impact may be reduced by provision of full information to participants about the benefits and limitations of screening programs and by increasing public education on these issues.