Objective: The objective was to develop an economic prioritization model to assist those involved in the selection and prioritization of health technology assessment topics and commissioning of HTA projects.

Methods: The model used decision analytic techniques to estimate the expected costs and benefits of the health care interventions that were the focus of the HTA question(s) considered by the NHS Health Technology Assessment Programme in England. Initial estimation of the value for money of HTA was conducted for several topics considered in 1997 and 1998.

Results: The results indicate that, using information routinely available in the literature and from the vignettes, it was not possible to estimate the absolute value of HTA with any certainty for this stage of the prioritization process. Overall, the results were uncertain for 65% of the HTA questions or topics analyzed. The relative costs of the interventions or technologies compared to existing costs of care and likely levels of utilization were critical factors in most of the analyses. The probability that the technology was effective with the HTA and the impact of the HTA on utilization rates were also key determinants of expected costs and benefits.

Conclusions: The main conclusion was that it is feasible to conduct ex ante assessments of the value for money of HTA for specific topics. However, substantial work is required to ensure that the methods used are valid, reliable, consistent, and an efficient use of valuable research time.

Deliberative processes are a well-established part of health technology assessment (HTA) programs in a number of high- and middle-income countries, and serve to combine complex sets of evidence, perspectives, and values to support open, transparent, and accountable decision making. Nevertheless, there is little documentation and research to inform the development of effective and efficient deliberative processes, and to evaluate their quality. This article summarizes the 2020 HTAi Global Policy Forum (GPF) discussion on deliberative processes in HTA.

Through a combination of small and large group discussion and successive rounds of polling, the GPF members reached strong agreement on three core principles for deliberative processes in HTA: transparency, inclusivity, and impartiality. In addition, discussions revealed other important principles, such as respect, reviewability, consistency, and reasonableness, that may supplement the core set. A number of associated supporting actions for each of the principles are also described in order to make each principle realizable in a given HTA setting. The relative importance of the principles and actions are context-sensitive and must be considered in light of the political, legislative, and operational factors that may influence the functioning of any particular HTA environment within which the deliberative process is situated. The paper ends with suggested concrete next steps that HTA agencies, researchers, and stakeholders might take to move the field forward. The proposed principles and actions, and the next steps, provide a springboard for further research and better documentation of important aspects of deliberation that have historically been infrequently studied.

Swedish antenatal care is staffed by midwives working within the primary health care system, but with close collaboration with the hospital. The compliance to the program both from staff and mothers is very high, with 13.2 visits on average. There is a regional variation both in the number of visits and in the use of routine tests.

Objectives: Based on a prevalent framework in the information systems field, this study proposes and describes an integrated model for evaluating picture archiving and communication system (PACS) success from multiple users' perspectives.

Methods: Our study details the validation process of the proposed model at a large tertiary-care teaching hospital in Canada. Both qualitative and quantitative data were collected to assess the psychometric properties of the measurement instrument and test the research hypotheses.

Results: Our findings clearly reveal that radiologists, technologists, and clinicians have different views regarding the factors influencing PACS success. For instance, the results for radiologists show that their concern with efficiency and productivity is best guaranteed by a system that is reliable and easy to use. Furthermore, that only perceived system usefulness influenced clinicians' satisfaction with PACS is a reflection of the primary impact that technology has on their work, namely, the ability to have instant access to images from any point in the hospital. Even though, overall, all three groups view the adoption of PACS positively, the mean scores indicate that radiologists and technologists seem to be more satisfied and their expectations to be met at a higher level than clinicians.

Conclusions: We believe the measurement instruments developed in this study can be used as a diagnostic tool by project managers interested in better understanding the extent to which different groups of stakeholders perceive the deployment of PACS as being successful and how factors influencing perceptions of PACS success vary across user types.

Background: Decision analytic models, as used in economic evaluations, require data on several clinical parameters. The gold standard approach is to conduct a systematic review of the relevant clinical literature, although reviews of economic evaluations indicate that this is rarely done. Technology appraisals for the National Institute for Health and Clinical Excellence (NICE), which are fully funded, represent the best case scenario for the close integration of economic evaluations and systematic reviews. The objective of this study was to assess the extent to which the systematic review of the clinical literature informs the economic evaluation in NICE technology appraisals.

Methods: All NICE technology assessment reports (TARs) published between January 2003 and July 2006 were considered. Data were abstracted on the TAR topics, the primary measure of clinical effectiveness, the approach to pooling in the clinical review, the measure of economic benefit and the use, or non-use, of the systematic review in the economic evaluation.

Results: Forty-one TARs were published in the period studied, all of which contained a systematic review. Most of the economic evaluations (85 percent) were cost-utility analyses, reflecting NICE's guidelines for economic evaluation. In seventeen cases, the clinical data were not pooled in the review, owing to heterogeneity in the clinical data or the limited number of studies. In these cases, the economists used alternative approaches for estimating the key effectiveness parameter in the model. The results of the review (when pooled) were always used when the primary clinical effectiveness measure corresponded with the measure of economic benefit (e.g., survival). However, because preference-based quality of life measures are rarely included in clinical trials, the results of the systematic review were never directly used in the cost-utility analyses. Nevertheless, the outputs of the systematic review were used when the data were useful in estimating components of the quality-adjusted life-year (QALY) (e.g., the life-years gained, or the frequencies of health states to which QALYs could be assigned). Problems occurred mainly when the clinical data were not pooled, or when the measure of clinical benefit could not be converted into health states to which QALYs could be assigned.

Conclusions: Economic evaluations can benefit from systematic reviews of the clinical literature. However, such reviews are not a panacea for conducting a good economic evaluation. Much of the relevant data for estimating QALYs are not contained in such reviews and the chosen method for summarizing the clinical data may inhibit the assessment of economic benefit. Problems would be reduced if those undertaking the technology assessments discussed the data requirements for the economic model at an early stage.

It is difficult to study the epidemiology of ICUs, as they lack a uniform nomenclature and/or classification. The organization and distribution of intensive care medicine depend on the size and function of the hospital. The patients in ICUs are predominantly men, with a high proportion of elderly patients (≥70 years) constituting 25–30% of the total. Case-mix, severity of illness and outcome differ from one unit to another, and can be compared only if the patients are classified with a common classification system. Most survivors of intensive care seem to return to normal or near normal functional level within one year. Compared to Western Europe, the United States has more ICU beds and a nearly ten times higher admission rate to intensive care. These variations can be seen as a result of a fundamental difference in the attitudes toward withdrawing or withholding life support.

We analyze what is known and unknown about the contribution of the pharmacist as patient educator, physician consultant, and agent to affect patient outcomes in ambulatory settings. The need for pharmacist services is discussed, as are the theoretical underpinnings and quality of the scientific evidence to support their efficacy. The analysis is conducted in the context of a shift in pharmacists' roles from product to patient orientation as well as recent U.S. legislation mandating enhanced pharmacists' roles via drug utilization review for all Medicaid patients. We conclude with a research and action agenda, calling for stronger research designs in evaluating pharmacists' interventions. The shifting paradigm in the pharmacy profession, coupled with the implementation of the Omnibus Budget Reconciliation Act of 1990, provide unique opportunities for rigorous evaluations of pharmacists as agents of change for rational drug therapy.

Objectives: An important option for the medical treatment of obstructive sleep apnea-hypopnea syndrome (OSAHS) is continuous positive airway pressure (CPAP) during sleep. This study reports on the cost-effectiveness of CPAP compared with dental devices and lifestyle advice. The work was commissioned by the NHS HTA Programme to inform the National Institute of Health and Clinical Excellence's (NICE) appraisal of CPAP.

Methods: A Markov model compared the interventions over the expected patient lifetime. The primary measure of cost-effectiveness was the incremental cost per quality-adjusted life-year (QALY) gained. The QALY incorporated the impact of treatments on daytime sleepiness, blood pressure and health-related quality of life (HRQoL).

Results: On average, CPAP was associated with higher costs and QALYs compared with dental devices or lifestyle advice. In the base-case analysis, the incremental cost-effectiveness ratio (ICER) for CPAP compared with dental devices was around £4,000 per QALY (2005–06 prices). The probability that CPAP is more cost-effective than dental devices or lifestyle advice at a threshold value of £20,000 per QALY was 0.78 for men and 0.80 for women. Several sensitivity analyses were undertaken and it was found that the ICER for CPAP consistently fell below £20,000 per QALY gained, apart from in a subgroup with mild disease.

Conclusions: The model suggests that CPAP is cost-effective compared with dental devices and lifestyle advice for adults with moderate or severe symptomatic OSAHS at the cost-effectiveness thresholds used by NICE. This finding is reflected in the NICE guidance.

Despite the perception of many people that lasers represent the cutting edge of high-technology medicine, this form of medical technology has been subject to relatively little rigorous evaluation. This dearth of research relates particularly to economic evaluation, where there have been few attempts to justify the high cost of laser equipment. This paper details an economic evaluation of the use of laser technology as a secondary adjunct to angioplasty to treat peripheral arterial occlusions. Using data from a range of sources, including a published randomized trial, a cost-utility model is developed to estimate the costs and benefits of the laser, relative to standard angioplasty. The best available data indicate a cost-effective role for the laser, but important areas of uncertainty exist, including the laser's secondary recanalization rate, which has been estimated on the basis of limited numbers of patients. This uncertainty suggests that further research is required before widespread diffusion of the laser for use in this clinical context.

Objectives: A set of fifteen key principles (KP) has been recently proposed to guide decisions on the structure of HTA programs, the methods of HTA, the processes for conducting HTA and the use of HTA findings in decision-making. The objective of this research is to explore whether these KPs are relevant and useful in Latin America (LA), and to what extent they are being applied.

Methods: A Web-based survey was sent to 11,792 HTA researchers and users in LA to explore the perceived relevance of each KP, its current level of application and the gap between these two.

Results: We received 1,142 responses from nineteen LA countries (9.7 percent response rate). The subgroup of KP related to Methods and to the Use of HTA received the higher mean scores in the relevance scale (9.00 and 8.94). Level of current application scored low in all KP (3.2 to 4.9). Higher gaps were observed in principles related to the use of HTA in decision making and to the processes for conducting HTA. Countries with more developed HTA showed higher scores in the degree of current application (5.3 versus 3.4, p < .01) and lower gaps (3.84 versus 5.21, p < .01). Researchers, compared with research users, scored the relevance of the KPs higher.

Conclusions: KPs seem to be very relevant to most HTA researchers and users in LA. However, the current level of application was considered uniformly poor. Higher gaps were observed in KPs related to the link between HTA and decision making, highlighting one of the major challenges for the countries in the region.

Objectives: In recent years, there has been growth in the use of health technology assessment (HTA) for making decisions about the reimbursement, coverage, or guidance on the use of health technologies. Given this greater emphasis on the use of HTA, it is important to develop standards of good practice and to benchmark the various HTA organizations against these standards.

Methods: This study discusses the conceptual and methodological challenges associated with benchmarking HTA organizations and proposes a series of audit questions based on a previously published set of principles of good practice.

Results and Conclusions: It is concluded that a benchmarking exercise would be feasible and useful, although the question of who should do the benchmarking requires further discussion. Key issues for further research are the alternative methods for weighting the various principles and for generating an overall score, or summary statement of adherence to the principles. Any weighting system, if developed, would need to be explored in different jurisdictions to assess the extent to which the relative importance of the principles is perceived to vary. Finally, the development and precise wording of the audit questions requires further study, with a view to making the questions as unambiguous as possible, and the reproducibility of the assessments as high as possible.

Objectives: Rare diseases are often heterogeneous in their progression and response to treatment, with only a small population for study. This provides challenges for evidence generation to support HTA, so novel research methods are required.

Methods: Discussion with an expert panel was augmented with references and case studies to explore robust approaches for HTA evidence generation for rare disease treatments.

Results: Traditional RCTs can be modified using sequential, three-stage or adaptive designs to gain more power from a small patient population or to focus trial design. However, such designs need to maintain important design aspects such as randomization and blinding and be analyzed to take account of the multiple analyses performed. N-of-1 trials use within-patient randomization to test repeat periods of treatment and control until a response is clear. Such trials could be particularly valuable for rare diseases and when prospectively planned across several patients and analyzed using Bayesian techniques, a population effect can be estimated that might be of value to HTA. When the optimal outcome is unclear in a rare disease, disease specific patient reported outcomes can elucidate impacts on patients’ functioning and wellbeing. Likewise, qualitative research can be used to elicit patients’ perspectives, with just a small number of patients.

Conclusions: International consensus is needed on ways to improve evidence collection and assessment of technologies for rare diseases, which recognize the value of novel study designs and analyses in a setting where the outcomes and effects of importance are yet to be agreed.

Based on an American multicenter study, an economic evaluation of prophylactic misoprostol was undertaken in Sweden. The study included 420 patients with osteoarthritis and nonsteroidal anti-inflammatory drug (NSAID)-associated abdominal pain, but no gastric ulcer at inclusion. The frequency of ulcer development with and without prophylactic misoprostol was assessed at 21.7% and 5.6%, respectively, for a 3-month period. All costs for drugs, ambulatory care, hospital care, loss of production, as well as other factors such as dosage and compliance, were transferred to Swedish conditions. It was concluded that in patients with osteoarthritis and NSAID-induced abdominal pain, prophylaxis with misoprostol is cost-effective in Sweden, which is similar to what is found for other countries. A prerequisite for this result is a frequency of ulcer development of 15%. A patient compliance to prophylactic treatment of more than 60% is also presupposed (79% was observed in the above study). Due to the high age of the osteoarthritis patient population, the cost-effectiveness is influenced to only a minor extent by whether indirect costs are included in the calculation.

Cost-effectiveness analysis compared four treatments of end-stage renal disease in Brazil: continuous ambulatory peritoneal dialysis (CAPD), in-center hemodialysis (HD), cadaver donor transplantation (CD-Tx), and living related donor transplantation (LR-Tx). After 2 years, the costs per year of survival were CAPD, $12,134; HO, $10,065; CO-Tx, $6,978; and LR-Tx, $3,022. The HD cost was lower than CAPO partially because of the reuse of hemodialyzers in Brazil. Although less cost-effective, both dialysis treatments yielded more years of survival after 2 years. This analysis reveals a trade-off between cost per year of survival and years of survival.

Objective: To review published data regarding the accuracy and effectiveness of three screening tests: mammography, prostate-specific antigen (PSA), and prenatal ultrasound.

Methods: Published evidence regarding the accuracy and effectiveness of the three tests was collected by computerized literature search and supplemented by manual review of relevant bibliographies.

Results: Screening mammograms lower breast cancer mortality by about 20%. Most data come from women aged 50–64 years; women aged 40–49 years may also benefit, but the absolute risk reduction is lower. Up to 1,500 to 2,500 women must undergo screening to prevent one death from breast cancer. Mammograms miss approximately 12% to 37% of cancers, generate false-positive results, and cause anxiety while abnormal results are evaluated. PSA screening can detect 80% to 85% of prostate cancers but has a high false-positive rate. There is little direct evidence that early detection reduces morbidity or mortality. Indirect evidence includes a trend toward earlier stage tumors and steadily declining mortality rates in geographic areas where PSA screening has become common. Potential harms include the morbidity associated with evaluating abnormal results, and complications from treatment (e.g., impotence, incontinence). The overall balance of benefits and harms remains uncertain in the absence of better evidence. Prenatal ultrasound may reduce perinatal mortality, primarily through elective abortions for congenital anomalies, but does not appear to lower live birth rates. Although ultrasound has no proven effect on neonatal morbidity, it provides more accurate estimates of gestational age that prevent unnecessary inductions for post-term pregnancy. Screening detects multiple gestations, congenital anomalies, and intrauterine growth retardation, but direct health benefits from having this knowledge are unproved. Ultrasound has both positive and negative psychological effects on parents. The scans do not appear to harm childhood development.

Conclusions: Even for the most established screening tests, the appropriateness of routine testing depends on subjective value judgments about the quality of supporting evidence and about the trade-offs between benefits and harms. Individuals, clinicians, policy makers, and governments must weigh the evidence in light of these values and the constraints imposed by available resources.

Objectives: New medical technologies have been identified as the leading cause of increasing health-care expenditures. Adoption of a new technology is one of the most important decisions in medical centers. The objectives of this study were to map and describe the function of hospital decision-makers within the area of new technology assessment and adoption, and to examine relevant considerations, sources of information, and decision-making processes in the adoption of a new technology.

Methods: A questionnaire was mailed to hospital executives and referred to (i) the considerations for and against adoption of a new technology, (ii) the decision-making process, (iii) information sources used in the decision-making process.

Results: The most frequent criteria favoring adoption included increased cost-effectiveness, increased efficacy, and decrease in complication rates. An increase in complication rates or side effects and decreased efficacy were the top ranked criteria against adoption. The final decision-making responsibility varied among technologies; the medical director frequently made the final decision when a new device was involved, but this responsibility decreased when a new drug or a new procedure was considered. Participation in scientific meetings, opinions of local experts, medical journals, and Food and Drug Administration clearance documents were the most important information sources used in the decision-making process. However, these were not necessarily the optimal sources of information. Significant barriers in adoption decision-making are lack of timely data regarding the safety of the new technology, its cost-effectiveness, and efficacy.

Conclusion: To improve the adoption decisions, hospitals must develop criteria upon which the decision-making will be based.

Objectives: There is widespread commitment—at least in principle—to “evidence-informed” clinical practice and policy development in health care. The intention is that only “appropriate” care ought to be delivered at public expense. Although the rationale for an appropriateness agenda is widely endorsed, and methods have been proposed for addressing it, few published studies exist of contemporary policy initiatives which have actually led to successful disinvestment. Our objective was to explore whether the direct involvement of policy stakeholders could advance appropriateness and disinvestment.

Methods: Several collaborative engagements with policy stakeholders were undertaken to adapt and combine conceptual and empirical material related to appropriateness and disinvestment from the literature to create tools and processes for use in Canada and the province of Ontario in particular.

Results: By combining inputs from the literature with colloquial evidence from policy stakeholders, a definition of appropriateness was developed and, importantly, endorsed by all the provincial and territorial ministers of health in Canada. Second, a reassessment framework was successfully implemented for identifying priorities for selective disinvestment.

Conclusions: When scientific evidence was combined with colloquial evidence from policy stakeholders, progress was made on the design and successful implementation of policies for appropriateness and disinvestment.

Objectives: The aim of this study was to describe the developing health technology assessment (HTA) activities in Brazil, both historically and in the present day.

Methods: This report is a descriptive analysis based on personal experiences of the authors and on selected literature.

Results: Interest in HTA in Brazil began in the mid-1980s. Several seminars and consultations were held, often with invited foreign participants. A cadre of people with knowledge and expertise in HTA was gradually developed. In 2003, several policies were developed by the federal government of Brazil to encourage HTA and base clinical, management, and policy decisions on HTA. During the past 5 years, institutional development has been rapid in government, private companies (mainly prepaid health plans), academia, and research institutes. Further policy changes are needed to maximize the impact of these developments. Nevertheless, although the growing network of HTA programs will have a considerable impact on Brazilian health care, further institutional development could stimulate this change.

Conclusions: It would be desirable if the Federal Ministry of Health of Brazil were to proceed to develop a national agency for HTA.