Objectives: The aim of this study was to demonstrate how value of information analysis can measure the upper limit on returns to future research and identify the research priorities for computer-assisted total knee replacement (CAS-TKR).

Methods: Using a previous economic analysis of CAS-TKR compared with conventional TKR, the population expected value of perfect information (EVPI) was calculated using Monte Carlo simulation to provide an estimate of the upper limit on returns to future research. The population expected value of partial perfect information (EVPPI) for both individual parameters and groups of parameters was estimated to inform specific future research priorities.

Results: The UK individual EVPI would be £21.4 if the willingness to pay for one QALY (quality-adjusted life-year) were £30,000. The population EVPPI would be £8.3 million, assuming a 10-year time horizon for CAS-TKR. In this instance, the expected value of information is positively related to willingness to pay for one QALY for the range of £0 to £50,000. Although each individual parameter had an EVPPI of £0, groups of utility parameters had positive EVPPI. Population EVPPI was £5.6 million for utility parameters, £20,000 for transition probabilities relating to CAS-TKR, and £5,000 for transition probabilities related to conventional TKR.

Conclusions: The study provides evidence on which parameters further information may be of most value. Focusing research on the utility values associated with health states relating to TKR would be of greatest value.

Objectives: The aim of this study was to estimate the impact of new medical technologies on public healthcare expenditures in Israel over the period 2000–07.

Methods: For each year, government estimates for the costs of new technologies recommended as high-priority for public funding were summarized. The ratio of projected costs of these technologies to total public healthcare expenditures was calculated and compared with actual governmental budget allocations for new technologies.

Results: Funding all new high-priority medical technologies would have increased healthcare expenditures by 2.1 percent per year. Government allocations for new technologies raised expenditures by 1.0 percent per year.

Conclusions: New medical technologies significantly increase healthcare expenditures in Israel. Budgetary constraints have reduced their actual impact by 52 percent. This study indicates the need for an annual addition of 2 percent to public healthcare budget for funding new high-priority technologies.

Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions, specific legislation has been enacted to encourage the development of drugs for rare diseases (orphan drugs), which would otherwise not be commercially viable. However, because of the small market, these drugs are often very expensive. Under the standard methods of health technology assessment (HTA) incorporating economic evaluation, orphan drugs do not usually prove to be cost-effective and this, coupled with their high cost, means that funding and patient access may be limited. However, these restrictions may not be in line with societal preferences. Therefore, this study discusses whether the standard methods of HTA are adequate for assisting decisions on patient access to and funding of orphan drugs and outlines a research agenda to help understand the societal value of orphan drugs and issues surrounding their development, funding, and use.

Objectives: Congenital heart defects (CHD) are an important cause of death and morbidity in early childhood, but the effectiveness of alternative newborn screening strategies in preventing the collapse or death—before diagnosis—of infants with treatable but life-threatening defects is uncertain. We assessed their effectiveness and efficiency to inform policy and research priorities.

Methods: We compared the effectiveness of clinical examination alone and clinical examination with either pulse oximetry or screening echocardiography in making a timely diagnosis of life-threatening CHD or in diagnosing clinically significant CHD. We contrasted their cost-effectiveness, using a decision-analytic model based on 100,000 live births, and assessed future research priorities using value of information analysis.

Results: Clinical examination alone, pulse oximetry, and screening echocardiography achieved 34.0, 70.6, and 71.3 timely diagnoses per 100,000 live births, respectively. This finding represents an additional cost per additional timely diagnosis of £4,894 and £4,496,666 for pulse oximetry and for screening echocardiography. The equivalent costs for clinically significant CHD are £1,489 and £36,013, respectively. Key determinants of cost-effectiveness are detection rates and screening test costs. The false-positive rate is very high with screening echocardiography (5.4 percent), but lower with pulse oximetry (1.3 percent) or clinical examination alone (.5 percent).

Conclusions: Adding pulse oximetry to clinical examination is likely to be a cost-effective newborn screening strategy for CHD, but further research is required before this policy can be recommended. Screening echocardiography is unlikely to be cost-effective, unless the detection of all clinically significant CHD is considered beneficial and a 5 percent false-positive rate acceptable.

Objectives: A systematic review of the cost-effectiveness of abdominal aortic aneurysm (AAA) repair was conducted. Although open surgery has been considered the gold standard for prevention of AAA rupture, emerging less-invasive endovascular treatments have led to increased interest in evaluating the cost and cost-effectiveness of treatment options.

Methods: A systematic review of studies published in MEDLINE between 1999 and 2005 reporting the cost and/or cost-effectiveness of endovascular and/or open surgical repair of nonruptured AAAs was conducted. Case series studies with less than fifty patients per treatment were excluded.

Results: Of twenty eligible articles, three were randomized controlled trials, twelve case series, four Markov models, and one systematic review. Regardless of time frame, all studies found that endovascular repair costs more than open surgery. Although the high cost of the endovascular prosthesis was partially offset by reduced intensive care, hospital length of stay, operating time, blood transfusions, and perioperative complications, hospital costs were still greater for endovascular than open surgical repair. For patients medically fit for open surgery, mid-term costs were greater for endovascular repair with no difference in overall survival or quality of life. For patients medically unfit for open surgery, endovascular repair costs more than no intervention with no difference in survival.

Conclusions: Although conclusions regarding the cost-effectiveness of AAA treatment options are time dependent and vary by institutional perspective, from a societal perspective, endovascular repair is not currently cost-effective for patients with large AAA regardless of medical fitness.

When new medical technologies enter the market, there is often uncertainty about the added value for the patient and for society, hampering well-considered decision making about reimbursement. Current Belgian legislation already offered opportunities for the managed uptake of possibly innovative emerging implants. However, it has also some shortcomings such as the lack of a clear research design, rendering the scientific evaluation of clinical effectiveness, cost-effectiveness, and patient or organizational issues more difficult. Against this background, a new procedure was elaborated by the Belgian health insurance institute and the Belgian Health Care Knowledge Centre.

Objectives: Leukemia, together with lymphoma and multiple myeloma, are hematological malignancies, malignancies of the blood-forming organs. There are four major types of leukemia: acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and chronic lymphocytic leukemia (CLL). There is a growing amount of literature of the health economic aspects of leukemia. However, no comprehensive review is yet performed on the health economic evidence for the disease. Hence, our aim was to review and analyze the existing literature on economic evaluations of the different types of leukemia.

Methods: A systematic literature search used electronic databases to identify published cost analyses and economic evaluations of leukemia treatments. After reviewing all identified studies, sixty studies were considered relevant for the purpose of the review.

Results: The identified studies were published after 1990, with a few exceptions. Many of the identified economic evaluations in leukemia, particularly for ALL and AML, may be defined as cost-minimization analyses, where only the costs of different treatment strategies are compared. In CML, a new treatment, imatinib, was introduced in 2001 and several cost-effectiveness analyses have since then been conducted comparing imatinib with previous first line treatments.

Conclusions: This review indicates that there is a shortage of cost-effectiveness information in leukemia. The introduction of new therapies will stress the need for new economic evaluations in this group of diseases. More information about the total costs, that is, including indirect costs, and quality of life effects would be valuable in future evaluations in leukemia.

Objectives: The National Institutes of Health (NIH) Consensus Development Conference (CDC) was instituted to provide evidence-based guidance on controversial medical issues to researchers, health practitioners, and the public; however, the degree of impact this activity has on stimulating relevant research is unclear. This study examines the impact of CDC statements on the initiation of related NIH-funded research projects.

Methods: Six CDCs from 1998 to 2001 were examined. Research initiatives related to the Conferences' topics were collected through two discrete methods: (i) the overall number of relevant pre- and postconference research activities was compiled using NIH's Information for Management, Planning, Analysis, and Coordination II (IMPAC II) and the Department of Health and Human Services' (DHHS) Computer Retrieval of Information on Scientific Projects (CRISP) grant application and award databases; (ii) for each CDC, the sponsoring institute's conference coordinator and other identified Program Directors were queried for their knowledge of new conference-specific research initiatives sponsored by their institute. The main outcome measure was the total number of requests for applications, requests for proposals, program announcements, broad agency announcements, notices, and funded investigator-initiated research program grants (RO1s) for a given Consensus topic in the 3 years before (baseline measure) and following (measure of impact) a CDC.

Results: As identified through NIH's IMPAC II and DHHS' CRISP grants and announcements databases, the total number of relevant postconference research initiatives increased for five of six CDCs when compared with baseline activity levels; research activities remained constant for the sixth. When inclusion criteria were restricted to institute-identified research initiatives, two of six CDC topics had overall increases in relevant research activity in the postconference period.

Conclusions: CDCs appear to have a positive impact on the stimulation of related NIH-funded research initiatives. Future outcomes evaluations using prospective data collection methods and more robust participation by sponsoring and cosponsoring institutes should strengthen the reliability of the association between new research initiatives on a given topic and their causal relationship to a given CDC.

Objectives: The University of Minnesota has maintained a home monitoring program for over 10 years for lung and heart–lung transplant patients. A cost analysis was completed to assess the impact of home monitoring on the cost of post-transplant medical care.

Methods: Clinical information gathered with the monitoring system includes spirometry, vital signs, and symptom data. To estimate the impact of this system on medical costs, we completed a retrospective analysis of the effects of home monitoring on the cost of post-lung transplant medical care. The cost analysis used multivariate linear regression with inpatient, outpatient, and total medical care costs as the dependent variables. The independent variables for the regression include home monitoring adherence, underlying disease, ambulatory diagnostic group mapping variables, transplant type, and patient demographics.

Results: The multivariate regression of the overall cost results predicts a 52.4 percent reduction in total costs with 100 percent patient adherence; this rate includes a 72.24 percent reduction in inpatient costs and a 46.6 percent increase in outpatient costs. The actual first year average patient adherence was 74 percent.

Conclusions: Adherence to home monitoring increases outpatient costs and reduces inpatient costs and provides an overall cost savings. The break-even point for patient adherence was 25.28 percent, where the net savings covered the cost of home monitoring. This is well within the actual first year adherence rates (74 percent) for subjects in the lung transplant home monitoring program, providing a net savings with adherence to home monitoring.

Objectives: U.S. expenditures on medical devices ($70 billion in 2003) are one of the fastest growing components of hospital costs. Physicians’ selection of medical devices lacks an evidence base on the comparative clinical effectiveness of these products. Comparative studies (e.g., vendor 1 versus vendor 2, technology A versus technology B) are increasingly promoted in the public sector as a means of cost containment, value-based purchasing, and quality improvement. This study illustrates how hospitals and physicians can conduct comparative technology assessments of product performance.

Methods: Surgeons evaluated comparable medical devices manufactured by eight different vendors in standardized surgical procedures. Devices included sutures and endomechanical products, which account for $2.5 billion of total device spending. Evaluations covered multiple performance dimensions, including ergonomics, functionality, clinical acceptability, and vendor preference.

Results: One vendor's products garnered consistently high ratings from surgeons, while two other vendors garnered consistently low ratings. Differences in ratings were statistically significant and persist when controlling for physician background characteristics and prior experience. Study results were used by a large hospital group purchasing organization to select which vendors to contract with for these products.

Conclusions: Comparative technology evaluations assist physicians and hospitals in making cost-effective purchases of devices. These evaluations provide robust information on the performance of products routinely used by clinicians. Such evaluations can be carefully designed to have scientific rigor and clinical credibility.

Objectives: Despite controversies, endoscopic thoracic sympathectomy (ETS) has been used as a treatment for excessive sweating of hands and face and for facial blushing. This study aims to evaluate the effectiveness of ETS for the current indications in a systematic review.

Methods: Controlled clinical trials and cohort studies with more than 100 patients were included. Abstracts were searched from MEDLINE and CCTR from 1966 to June 2004. Two reviewers extracted the data and assessed study quality. Data on effectiveness and safety were synthesized qualitatively.

Results: We did not find any controlled clinical trials. Fifteen prospective studies were included. The internal and external quality of these studies were poor overall. Follow-up was commonly less than 2 years, during which time excessive sweating and facial blushing seemed to decrease among most patients. Immediate complications related to thoracoscopy occurred in up to 10 percent of patients. Compensatory sweating below breast level was reported in up to 90 percent of the patients. Other common side effects included dryness of face and hands, gustatory sweating, and neuralgic pain. Several other less common side effects were reported.

Conclusions: The evidence of the effectiveness of ETS is weak due to a lack of randomized trials. The intervention leads to severe immediate complications in some of the patients, and to persistent side-effects for many of the patients.

Objectives: To determine the relative extent to which the National Institute for Health and Clinical Excellence (NICE) appraises new versus existing technologies, and pharmaceutical versus nonpharmaceutical health technologies.

Methods: We categorized technologies within NICE appraisals published between March 2000 and June 2006 by type and classified them as new or existing using the timeline between launch in the United Kingdom and referral to NICE. We used a 3-year postlaunch cutoff to determine whether a technology was new, with a sensitivity analysis of 1 and 5 years.

Results: We reviewed 159 technologies from 88 appraisals. Of these, 84 (53 percent) were new (sensitivity analysis 36 to 67 percent) and 75 (47 percent) were existing technologies. A total of 119 (75 percent) were pharmaceuticals, 22 (14 percent) were devices, 14 (9 percent) were procedures, and 4 (3 percent) were categorized as miscellaneous. Classification according to newness and technology type showed that 62 percent (42 to 75 percent) of the pharmaceuticals appraised were new.

Conclusions: By developing and applying a definition of new, we have found that the criticism of the bias toward new technologies is unfounded when applied to the appraisal program overall. At the same time, new pharmaceuticals are over-represented in the program compared with devices and procedures. This domination may cause inflationary pressures on the health service, but any wholesale move away from the technological frontier may be more costly.

Objectives: The aim of this study was to assess the cost-effectiveness of laparoscopic surgery compared with open surgery for the treatment of colorectal cancer.

Methods: A Markov model was developed to model cost-effectiveness over 25 years. Data on the clinical effectiveness of laparoscopic and open surgery for colorectal cancer were obtained from a systematic review of the literature. Data on costs came from a systematic review of economic evaluations and from published sources. The outcomes of the model were presented as the incremental cost per life-year gained and using cost-effectiveness acceptability curves to illustrate the likelihood that a treatment was cost-effective at various threshold values for society's willingness to pay for an additional life-year.

Results: Laparoscopic surgery was on average £300 more costly and slightly less effective than open surgery and had a 30 percent chance of being cost-effective if society is willing to pay £30,000 for a life-year. One interpretation of the available data suggests equal survival and disease-free survival. Making this assumption, laparoscopic surgery had a greater chance of being considered cost-effective. Presenting the results as incremental cost per quality-adjusted life-year (QALY) made no difference to the results, as utility data were poor. Evidence suggests short-term benefits after laparoscopic repair. This benefit would have to be at least 0.01 of a QALY for laparoscopic surgery to be considered cost-effective.

Conclusions: Laparoscopic surgery is likely to be associated with short-term quality of life benefits, similar long-term outcomes, and an additional £300 per patient. A judgment is required as to whether the short-term benefits are worth this extra cost.

Objectives: The clinical effectiveness and cost-effectiveness of implantable cardioverter defibrillators (ICD) for arrhythmias was assessed.

Methods: A systematic review of the literature of systematic reviews and randomized controlled trials that reported mortality outcomes associated with implantable cardioverter defibrillators compared with antiarrhythmic drug therapy in people at risk of sudden cardiac death due to arrhythmias was undertaken. Economic evaluations were also sought. Inclusion criteria, data extraction, and quality assessment were undertaken by standard methodology. A decision analytic model was constructed using best available evidence to determine cost-effectiveness in a UK setting.

Results: Eight randomized controlled trials, two systematic reviews, and a meta-analysis met the inclusion criteria and were of variable quality. Evidence suggests that ICDs reduce mortality in both secondary and primary prevention, although the magnitude of benefit depends on baseline risk for sudden cardiac death. Incremental cost per quality-adjusted life year ranged from £52,000 ($98,000) to over £200,000 ($379,000), depending on mortality risk and assumptions made.

Conclusions: Evidence suggests that ICDs reduce total mortality but may be cost-effective only in some subgroups of patients at high risk of ventricular arrhythmias. Further research is needed on risk stratification of patients in whom ICDs are most likely to be clinically and cost-effective.

Objectives: This study investigated the direct medical resource use and cost, informal care cost, and indirect cost associated with breast cancer in different states of the disease in normal clinical practice.

Methods: A retrospective database analysis was used to estimate direct medical resource use and cost, and a patient questionnaire was used to evaluate informal care and work capacity in different states of breast cancer.

Results: For patients younger than 65 years of age, the first year after a primary diagnosis total cost amounted to 280,000 SEK ($39,000) and the first year after a local or contralateral recurrence total cost was 351,000 SEK ($48,900). The second and following years after primary breast cancer or recurrence had substantially lower total cost, amounting to 94,000 SEK ($13,000). For patients with metastatic disease, the annual total cost was estimated to 334,000 SEK ($46,500). For patients older than 65 years of age, the total cost for the first year after a primary diagnosis amounted to 80,000 SEK ($11,200) and the total cost for the first year after a local or contralateral recurrence was 92,000 SEK ($12,900). The total cost for the second and following years after primary breast cancer or recurrence was estimated to 18,000 SEK ($2,600), and the total annual cost for patients with metastatic was 122,000 SEK ($17,000).

Conclusions: Both direct medical costs and indirect cost vary substantially between disease states. For patients under 65 year of age, indirect costs accounted for more than 50 percent of the total cost.

Objectives: Implantable cardioverter defibrillator (ICD) therapy reduces the risk of sudden death in patients with ischemic cardiomyopathy, but their novelty and cost may represent barriers to utilization. The objective of this study was to examine the influence of age, gender, place of residence, and socioeconomic status on rates of ICD implantation for the primary prevention of death.

Methods: We conducted a population-based retrospective cohort study involving the entire province of Ontario, Canada. Patients were eligible if they had survived following hospitalization for heart failure from 1 January 1993, to 31 March 2004, and previously sustained an acute coronary syndrome within 5 years. Patients with an existing ICD or a documented history of cardiac arrest were excluded, as were patients who died in the hospital. Primary outcome was ICD implantation.

Results: We identified 48,426 patients hospitalized for heart failure who survived to hospital discharge. Of these, 440 received an ICD, with a gradual 30-fold increase in implantation rates over the study period (.12–3.9 percent). ICD recipients were more likely to be men (odds ratio [OR] = 4.14; 95 percent confidence interval [CI], 3.24–5.30), younger than 75 years of age (OR = 3.19; 95 percent CI, 2.57–3.96), reside in a metropolitan area (OR = 1.42; 95 percent CI, 1.04–1.9), and live in a higher socioeconomic neighborhood (OR = 1.32; 95 percent CI, 1.08–1.61).

Conclusions: Among patients with heart failure and a previous myocardial infarction, ICD use is increasing in Ontario. However, the application of this technology is characterized by major sociodemographic inequities. The causes and consequences of the pronounced age and gender discrepancies, in particular, warrant further investigation.

Objectives: The aim of this study was to systematically review economic analyses comparing drug-eluting stents (DES) to bare metal stents (BMS) in patients who undergo percutaneous coronary intervention to form an overall view about cost-effectiveness of DES and to construct a simple decision analysis model to evaluate the cost–utility of DES.

Methods: Electronic databases searched from January 2004 to January 2006 were Cochrane Database of Systematic Reviews; DARE, HTA, EED (NHS CRD); MEDLINE(R) In-Process, Other Non-Indexed Citations, MEDLINE(R). References of the papers identified were checked. We included randomized controlled trials (RCT) or model-based cost-effectiveness analyses comparing DES to BMS in patients with coronary artery disease. The methodological quality of the papers was assessed by Drummond's criteria. Baseline characteristics and results of the studies were extracted and data synthesized descriptively. A decision tree model was constructed to evaluate the cost–utility of DES in comparison to BMS, where health-related quality of life was measured by the 15D.

Results: We identified thirteen good-quality economic evaluations. In two of these based on RCTs, DES was found cost-effective. In six studies, it was concluded that DES might probably be a cost-effective strategy in some circumstances, but not as a single strategy, and four studies concluded that DES is not cost-effective. One study did not draw a clear conclusion. In our analysis, the overall incremental cost-effectiveness ratio was €98,827 per quality-adjusted life-years gained. Avoiding one revascularization with DES would cost €4,794, when revascularization with BMS costs €3,260.

Conclusions: The evidence is inconsistent of whether DES would be a cost-effective treatment compared with BMS in any healthcare system where evaluated. A marked restenosis risk reduction should be achieved before use of DES is justifiable at present prices. When considering adoption of a new health technology with a high incremental cost within a fixed budget, opportunity cost in terms of untreated patients should be seriously considered as a question of collective ethics.

Objectives: The aim of this study was to evaluate the cost-effectiveness of four risk-lowering interventions (smoking cessation, antihypertensives, aspirin, and statins) in primary prevention of cardiovascular disease.

Methods: Using data from the Framingham Heart Study and the Framingham Offspring study, we built life tables to model the benefits of the selected interventions. Participants were classified by age and level of risk of coronary heart disease. The effects of risk reduction are obtained as numbers of death averted and life-years saved within a 10-year period. Estimates of risk reduction by the interventions were obtained from meta-analyses and costs from Dutch sources.

Results: The most cost-effective is smoking cessation therapy, representing savings in all situations. Aspirin is the second most cost-effective (€2,263 to €16,949 per year of life saved) followed by antihypertensives. Statins are the least cost-effective (€73,971 to €190,276 per year of life saved).

Conclusions: A cost-effective strategy should offer smoking cessation for smokers and aspirin for moderate and high levels of risk among men 45 years of age and older. Statin therapy is the most expensive option in primary prevention at levels of 10-year coronary heart disease risk below 30 percent and should not constitute the first choice of treatment in these populations.

Objectives: The aim of this study was to evaluate the cost-effectiveness of on-site automated external defibrillators (AEDs) in the initial management of cardiac arrest in Ontario.

Methods: This was a cost-effectiveness analysis based on published literature and data from the Canadian Institute of Health Information. The participants were fictitious male and female cardiac arrest patients who were initially managed with on-site AEDs, compared with similar patients managed without on-site AEDs. This group included a subgroup of high-risk patients (i.e., heart failure and left ventricular ejection fraction <35 percent). The analysis was conducted in a variety of settings including hospitals and homes in Ontario, Canada. The main outcome evaluated was cost per quality-adjusted life-year (QALY) gained from a payer's perspective.

Results: Cost per QALY (all costs reported in Canadian dollars) was $12,768 when AEDs were deployed in hospitals, $511,766 when deployed in office buildings, $2,360,023 when deployed in apartment buildings, $87,569 when deployed in homes of high-risk patients, and $1,529,371 when deployed in homes of people older than 55 years of age.

Conclusions: Indiscriminate deployment of AEDs is not a cost-effective means of improving health outcomes of cardiac arrest. Their use should be restricted to emergency response programs, high-risk sites (such as hospitals), and high-risk patients.

Objectives: The aim of this study was to estimate the time and travel costs generated by women when attending for Papanicolaou (Pap) smear tests or colposcopy appointments in the United Kingdom, both absolutely and relative to the health service cost of the national cervical cancer screening programs.

Methods: Data were obtained from questionnaires completed by two samples of women participating in a three-center trial of management of low-grade abnormalities detected by screening (n = 1,106 for Pap smears and n = 1,203 for colposcopy appointments). Women were 20 to 59 years of age and resident in Grampian or Tayside, Scotland, or Nottingham, England. Questionnaire data were supplemented with sociodemographic information previously collected at the time of recruitment to the trial.

Results: The mean total time and travel costs per attendance at a smear test and at a colposcopy appointment were estimated to be £9.2 and £27.4, respectively, averaged across the three trial areas (valued at 2002 prices). Statistically significant intercenter disparities in time and travel costs were identified, particularly with respect to colposcopy appointments. For these, time and travel costs in Nottingham were substantially less than those in Grampian and Tayside (£22.9, £30.2, and £32.1, respectively). Time and travel costs amount to 26 and 33 percent, approximately, over and above the direct health service costs of the English and Scottish screening programs, respectively.

Conclusions: The time and travel costs associated with participation in the UK cervical cancer screening programs are substantial and are not spatially uniform across the country.