Objectives: Ethical evaluation has become an important part of health technology assessment (HTA), but so far no generally accepted method for doing this exists. This article explains the eclectic approach developed at the Finnish HTA office.

Methods: Each HTA report is produced in cooperation with the methodological and clinical experts from various levels of healthcare organizations. An open framework for ethical evaluation when assessing different types of interventions is used to identify all possible stakeholders for each particular intervention. The ethical consequences for each party are identified during the entire process of the HTA project.

Results: The results of an ethical evaluation in four different HTA projects (two on screening, one on surgical intervention, and one in rehabilitation) show that an open framework is useful for opening discussion and understanding the scope of each ethical evaluation. Both content and methodological experts have found the process to be useful in capturing the broad consequences of implementing a new method.

Conclusions: Ethical evaluation is a continuous process that considers the prevalent morals, values, and behavioral models of the society. An in-depth ethical evaluation helps the decision-makers to realize the consequences that implementing a new method has on individual citizens, the healthcare system, and society.

Background: Since its inception, the field of health technology assessment (HTA) has stressed the need for consideration of ethical and social issues. However, few concepts or analytic tools have been developed, and because of the complexity of the endeavor and a lack of integration of work already produced, such concepts remain difficult to apply in HTA.

Objectives: Through a descriptive “map” of concepts, tools, and processes, we summarize the most tangible efforts on the part of HTA producers to address social and ethical issues.

Methods: A literature review and content analysis of HTA reports in the Centre for Reviews and Dissemination database enables a synthesis of the reflections on, initiatives around, and gaps in knowledge related to the integration of social and ethical issues in HTA.

Results: We examine: (i) the aim of integrating ethical and social issues in HTA, (ii) the theoretical approaches used, (iii) the methods and processes applied, and (iv) the implications for HTA producers. We highlight two levels at which social and ethical issues can be considered: throughout the production process of HTA reports and as part of the organizational structure of HTA agencies.

Conclusions: Given the profound societal changes that occur in relation to healthcare technology development, HTA producers have a responsibility to inform and enlighten technology-related public and policy debates. Fulfilling this role, though, requires that socioethical dimensions of technology and HTA are made explicit.

Objectives: To examine differences in the ethical judgments made by Research Ethics Committees (RECs) or Institutional Review Boards (IRBs).

Methods: We did a review of the literature and included any study that attempted to compare the ethical judgments made by different RECs or IRBs when reviewing one or more protocol.

Results: There were twenty-six articles reporting such discrepancies across Europe, within the United Kingdom, Spain, and United States. Of these studies, there were only five reports of some RECs approving while others rejecting the same protocol. All studies, however, reported differences in the clarifications and revisions asked of researchers regarding consent, recruitment, risks and benefits, compensation arrangements, and scientific issues.

Conclusions: The studies were generally anecdotal reports of researchers trying to do research. New rules requiring a single ethical opinion for multi-site research at least in European Member States may simply conceal problematic issues in REC decision making. In the last analysis, we should expect a certain degree of variation and differences if we are to keep a committee system of review, although there is a pressing need to investigate the way in which RECs make these judgments. In particular, we need to identify the source of any aberrations, distortions, or confusions that could arbitrarily affect these judgments. Furthermore, local conditions remain important ethical considerations and should not be sidelined in pursuit of greater “consistency.”

This study discusses the value of primary data collection as part of health technology assessment (HTA). Primary data collection can help reduce uncertainty in HTA and better inform evidence-based decision making. However, methodological issues such as choosing appropriate study design and practical concerns such as the value of collecting additional information need to be addressed. The authors emphasize the conditions required for successful primary data collection in HTA: experienced researchers, sufficient funding, and coordination among stakeholders, government, and researchers. The authors conclude that, under specific conditions, primary data collection is a worthwhile endeavor in the HTA process.

Background: In conjunction with other important movements in contemporary medicine, including evidence-based medicine (EBM), health technology assessment (HTA) has promoted a culture of critical evaluation. Despite this impact, institutional and methodological challenges are associated with HTA. For example, only in recent years has HTA attempted an open dialogue with patients; however, this is normally done by giving them a “seat” at the HTA decision-making table, rather than by more scientific means.

Objectives: The aim of this study was to develop a working definition of patient-based HTA, to identify the current barriers to adopting a patient-based model, and to formulate a vision of how a patient-based HTA could be used to promote patient empowerment and patient-centered care.

Results: In the ideal setting, a patient-based HTA would promote patient knowledge by providing access to information and promoting an informed dialogue between patients and their healthcare professionals. To implement a patient-based HTA, the focus must turn to the patient's issues and incorporate each patient's unique perspective and preferences. Processes must change to increase patient participation in all levels of HTA and aim to promote empowered patients who can make informed decisions.

Conclusions: Present-day HTA is broad and has numerous stakeholders, with none so important as the patient. By asking patient-oriented questions in HTA and better involving patients throughout the entire process, we can easily promote patient empowerment, and as such make patients more capable to play a more active role in healthcare decision making.

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: 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: 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: 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: 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: Shoulder complaints (SCs) constitute the second largest group of musculoskeletal disorders after low back pain. The economic burden in terms of costs of healthcare use and costs due to work absenteeism underlines the need for a cost-effectiveness analysis of the interventions involved. The education and activation program (EAP) is a newly developed early intervention to prevent the development of chronic SCs. A cost-effectiveness analysis should provide more information on the effect of an EAP on total costs related to SCs.

Methods: We conducted a cost-effectiveness analysis alongside a randomized clinical trial comparing the effectiveness of the EAP in addition to usual care (EAP group) with that of usual care alone (UC group) in terms of preventing chronicity in patients with acute SCs. The aim of the cost-effectiveness analysis was to compare the observed difference in costs with the clinical effectiveness (i.e., patient-perceived recovery after 26 weeks), using bootstraps.

Results: The comparison of total costs between treatment groups showed no significant (p = .077) difference after 26 weeks. The majority (82 percent) of the cost-effect pairs after bootstrap analysis were located in the northeast quadrant, suggesting more effect but at higher costs.

Conclusions: In view of the clinical relevance of the clinical outcomes and the considerable costs needed to achieve this, it can be concluded that the EAP is currently not cost-effective.

Objectives: Surveillance programs are recommended to both families at high risk (Amsterdam-positive families with known- and unknown mutation) and moderate risk (families not fulfilling all Amsterdam criteria) of colorectal cancer (CRC). Cost-effectiveness has so far only been estimated for the group at high risk. The aim of the present study is to determine cost-effectiveness of surveillance programs where families at both high and moderate risk of HNPCC participate.

Methods: A decision analytic model (Markov model) is developed to assess surveillance programs where families at high and moderate risk of HNPCC are offered surveillance from age 25 and age 45, respectively. The model includes costs for all families referred to genetic counseling, including genetic risk assessment, mutation analysis, and surveillance in relevant families with or without known mutation, plus the costs related to any surgical treatment. The risk of metachronous CRC is also modeled.

Results: Incremental costs per life year gained are estimated to be €980 when families at both high and moderate risk of HNPCC undergo surveillance (€508 for high risk and €1600 for moderate risk) and €1947 when the moderate risk group is evaluated genetically but not offered surveillance. Sensitivity analysis showed these findings to be robust, although cost-effectiveness can be improved in cases of more conservative referrals to genetic counseling.

Conclusions: The result for high risk families confirms the findings in similar studies. Somewhat surprisingly, cost-effectiveness improves when surveillance of the moderate risk groups are included in the decision model.

Objectives: A recent study showed that estimates of cost-effectiveness submitted to National Institute for Health and Clinical Excellence (NICE) by manufacturers had significantly lower incremental cost-effectiveness ratios (ICERs) than those submitted by university-based Assessment Groups. This study extends that analysis.

Methods: Data were abstracted from relevant NICE documentation for thirty-two of eighty-two possible appraisals.

Results: The results from the analysis showed that sources of the difference in ICERs appear to be the effectiveness estimates relating to the comparator technology and the cost estimates relating to the technology under evaluation. That is, manufacturers estimated lower average benefits for the comparator technology and lower costs relating to the technology under evaluation compared with estimates submitted by the Assessment Groups.

Conclusions: These findings may be particularly important, given the introduction of the “Single Technology Appraisal.” Considerable difficulties were encountered when undertaking this study, highlighting, above all else, the complexity of explaining why results from economic evaluations purporting to answer the same question diverge.

Objectives: The association between type and amount of clinical evidence and type of National Institute for Health and Clinical Excellence recommendations for interventional procedures was examined.

Methods: The evidence about 736 studies (including 183,729 patients) relating to 130 different interventional procedures and about relevant recommendations was analyzed. Associations were examined between type of recommendation (“normal arrangements” or “cautionary guidance”) and evidence type, total number of treated patients, and mean follow-up length. Evidence type was categorized as (a) randomized, (b) nonrandomized controlled, and (c) case series/reports. The main outcome measures were frequency of evidence type, total number of patients treated, and mean follow-up length, by type of recommendation.

Results: “Normal arrangements” recommendations were made for 70 (54 percent) procedures and “cautionary guidance” was issued for 60 (46 percent) procedures. Procedures supported by at least one randomized study (34 percent, n = 44) were more likely to receive a “normal arrangements” recommendation (relative risk 1.38, p = .063). Overall, there were 85 (12 percent), 135 (18 percent), and 516 (70 percent) studies in categories a–c, respectively. The number of treated patients was significantly larger among procedures with “normal arrangements” (median, 605; range, 26–6,842) than among those with “cautionary guidance” (median, 240; range, 1–3,261; p < .001) recommendation. Mean follow-up length was longer in studies relating to procedures with “normal arrangements” recommendation (median, 16.7; range, 0–84 months) compared with those with “cautionary guidance” (median, 14.6; range, 0–67 months; p = .160).

Conclusions: Procedures supported by randomized studies, and with larger numbers of patients and longer follow-up length, were more likely to receive positive guidance. Future research and development on interventional procedures should aim to produce better and more relevant evidence to optimize the possibility of such procedures being accepted by policy makers.

Objectives: The aim of this study was to examine the influence of participants' characteristics on the results produced by formal consensus methods.

Methods: The approach was an experimental study of 346 participants in 20 groups rating the appropriateness of four mental health interventions for the treatment of chronic fatigue syndrome, irritable bowel syndrome, and chronic back pain. There were four factors in the design: systematic literature review provided or not, decisions made under realistic or “ideal” resource assumptions, clinically mixed (general practitioners and mental health professionals) or homogenous group (general practitioners only), convened or mail-only group. A group's rating was defined as the median of participants' ratings. The influence of participants' characteristics (age, sex, and specialty) was examined using multilevel models.

Results: The largest differences were between the GPs and mental health professionals, both in their initial ratings of the different interventions, and in how much they altered their ratings between rounds. There were smaller but statistically significant (p<.05) differences between specialty and age groups in initial ratings for the treatment (by whatever means) of different conditions, and for certain conditions women increased their ratings more than men. Women rated intervention more favorably when assuming “ideal” rather than realistic levels of resources, but men did not.

Conclusions: Our findings support the practice of treating professional specialty as an important determinant of the results in consensus panels.

Objectives: Pediatric cardiology has an expanding role in fetal and pediatric screening. The aims of this study were to observe how district hospitals use a pediatric telecardiology service, and to compare the costs and outcomes of patients referred to specialists by means of this service or conventionally.

Methods: A telemedicine service was set up between a pediatric cardiac center in London and four district hospitals for referrals of second trimester women, newborn babies, and older children. Clinicians in each hospital decided on the role for their service. Clinical events were audited prospectively and costed, and patient surveys were conducted.

Results: The hospitals differed in their selection of patient groups for the service. In all, 117 telemedicine patients were compared with 387 patients seen in London or in outreach clinics. Patients selected for telemedicine were generally healthier. For all patients, the mean cost for the initial consultation was £411 for tele-referrals and £277 for conventional referrals, a nonsignificant difference. Teleconsultations for women and children were significantly more expensive because of technology costs, whereas for babies, ambulance transfers were much more costly. After 6-months follow-up, the difference between referral methods for all patients was nonsignificant (telemedicine, £3,350; conventional referrals, £2,172), and nonsignificant within the patient groups.

Conclusions: Telemedicine was perceived by cardiologists, district clinicians, and families as reliable and efficient. The equivocal 6-month cost results indicate that investment in the technology is warranted to enhance pediatric and perinatal cardiology services.

Objectives: This study evaluates the cost of dialysis care in Brazil, including costs of ambulatory care and hospital admissions due to all causes and cardiovascular events.

Methods: Data were analyzed for 200 patients with end-stage renal disease (ESRD) on chronic hemodialysis in Brazil between 2001 and 2004. Main end points were all-cause mortality, all-cause hospital admissions, and cardiovascular events. Direct costs of dialysis treatment and complications were computed from the perspective of two payers, the Ministry of Health (MoH) and private health insurance (PHI).

Results: Mean number of days of hospitalization was 12 per patient-year. There were 105 cardiovascular events; the most frequent events were coronary disease (n = 59, 56 percent) and congestive heart failure (n = 26, 25 percent). The rate of cardiovascular events was 193 per 1,000 patient-years. There were 43 deaths, and the death rate was 79 per 1,000 patient-years. Median cost per hospital admission was US$ 675 and US$ 932 from the perspective of the MoH and PHI. For admissions due to cardiovascular causes, the corresponding costs were US$ 1,639 and US$ 4,499, respectively. Mean global cost per patient-year for chronic hemodialysis therapy was US$ 7,980 and US$ 13,428 from the perspective of the MoH and PHI, respectively.

Conclusions: Patients on chronic hemodialysis care incur significant healthcare resources due to the costs of dialysis and complications, notably cardiovascular disease. New disease management programs aimed at reducing cardiovascular morbidity and efficient use of resources are critical to ensuring the sustainability of treatments for ESRD in Brazil.

Objectives: This study investigated the benefits of and barriers to user involvement in medical device technology development and evaluation.

Methods: A structured review of published literature in peer-reviewed journals was conducted.

Results: This literature review revealed that the main benefits of user involvement were an increased access to user needs, experiences, and ideas; improvements in medical device designs and user interfaces; and an increase in the functionality, usability, and quality of the devices. On the other hand, resource issues, particularly those of time and money were found the key impediments to involving users in the development and evaluation of medical device technologies. This study also has categorized both the benefits of and barriers to user involvement.

Conclusions: The involvement of users in medical device technology development and evaluation requires resources, which are limited; however, this involvement is essential from both users and manufacturers perspectives.

Objectives: The cost-effectiveness of prenatal diagnosis intervention for Down's syndrome (DS) in China was assessed and evidence-based information for policy makers and providers is presented.

Methods: Based on field surveys in four selected cities in China and a literature review, the economic evaluation of prenatal diagnosis for DS from a societal perspective is conducted by cost-effectiveness analysis.

Results: In current clinical practice, for a cohort of 10,000 pregnant women, the strategy that delivers karyotyping by chorionic villus sampling (CVS) or amniocentesis (AC) only to those pregnant women 35 years of age and older (maternal age screening strategy) can detect .67 DS births. The strategy that offers the diagnostic test after maternal serum screening with α-fetoprotein and human chorionic gonadotrophin (maternal serum screening strategy) can detect 1.41 DS births. The cost per prevented DS birth by the maternal age screening strategy and maternal serum screening strategy is US$13,091 and US$56,048, respectively. Sensitivity analysis shows that the maternal serum screening strategy can be cost-effective if uptake rate of CVS or AC for patients with positive serum tests increase while the cost of serum screening decreases.

Conclusions: Although, in general, serum screening has been found to be more cost-effective than maternal age screening, this appears not to be the case in China. The reasons appear to be low uptake rate of the maternal serum strategy, low uptake rate of CVS or AC, and the high price of serum screening. Our findings are that health system factors concerning technology utilization are important determinants of the technology's efficiency.

This consensus statement was prepared by an independent panel of health professionals, academics, and public representatives based on (1) relevant published studies assembled by the Institute of Health Economics, (2) presentations by experts working in areas relevant to the conference questions, (3) presentations by people living with diabetes, (4) questions and comments from conference attendees during open discussion periods, and (5) closed deliberations by the panel. It is an independent report of the panel and is not a policy statement of the Institute of Health Economics or the Government of Alberta. This consensus statement reflects the panel's assessment of scientific knowledge available at the time the statement was written. The objectives of the panel are to develop a consensus statement on the benefits and costs of testing supplies for self-monitoring of blood glucose in diabetes, to inform patients, health policy, and practice.