Background: During 2001, over 32,000 patients in the United Kingdom received renal replacement therapy (RRT). Approximately half had a functioning transplant, with the remainder receiving dialysis therapy. The main form of dialysis is hemodialysis (HD), which is provided to 37.1 percent of the RRT population. HD is provided in three main settings: hospital (24.5 percent), satellite (10.9 percent), or home (1.7 percent). The objective of this study is to explore the cost-effectiveness of these different modalities.

Methods: By using clinical and cost data from a systematic review, a Markov model was developed to assess the costs and benefits of the three different modalities. The model included direct health service costs and quality-adjusted life years (QALYs). Sensitivity analyses were performed to assess the robustness of the results.

Results: Satellite HD has lower costs £46,000 and £62,050 at 5 and 10 years than home HD £47,660 and £63,540. The total effectiveness of home HD was slightly greater than for satellite HD, so the incremental cost per QALY of home versus satellite HD was modest at £6,665 at 5 years and £3,943 at 10 years. Both modalities dominated hospital HD.

Conclusions: Results from the study reveal that satellite HD was less costly than home HD, and home HD was less costly than hospital HD. The lack of robust data on the effectiveness and new dialysis equipment, which were not included in this review, throws some caution on these results. Nonetheless, the results are supportive of a shift from hospital HD to satellite and home HD.

The costs, effects, and cost-effectiveness of the Dutch Lung Transplantation program were assessed. The results show that lung transplantation is a very costly intervention that improves survival and quality of life. Costs per life-year and per QALY gained were, respectively, US $90,000 and US $71,000.

The National Institutes of Health (NIH) is periodically asked to conduct assessments of new medical technologies to assist in coverage decisions made at the Office of Health Technology Assessment (OHTA) for the Health Care Financing Administration coverage policy. Analysis of NIH assessments indicates that even though most NIH assessments rely only on expert opinion, OHTA agreed with NIH recommendaations in over 90%.

Objectives: The headroom approach to medical device development relies on the estimation of a value-based price ceiling at different stages of the development cycle. Such price-ceilings delineate the commercial opportunities for new products in many healthcare systems. We apply a simple model to obtain critical business information as the product proceeds along a development pathway, and indicate some future directions for the development of the approach.

Methods: Health economic modelling in the supply-side development cycle for new products.

Results: The headroom can be used: initially as a ‘reality check’ on the viability of the device in the healthcare market; to support product development decisions using a real options approach; and to contribute to a pricing policy which respects uncertainties in the reimbursement outlook.

Conclusions: The headroom provides a unifying thread for business decisions along the development cycle for a new product. Over the course of the cycle attitudes to uncertainty will evolve, based on the timing and manner in which new information accrues. Within this framework the developmental value of new information can justify the costs of clinical trials and other evidence-gathering activities. Headroom can function as a simple shared tool to parties in commercial negotiations around individual products or groups of products. The development of similar approaches in other contexts holds promise for more rational planning of service provision.

Decisions relating to designing and implementing economic evaluations alongside clinical trials are dependent on a number of issues. Initial consideration should focus on whether an economic evaluation is appropriate, whether a randomized controlled trial (RCT) is a suitable vehicle, and whether the design of the specific RCT is appropriate for integration of economic evaluation. If an economic evaluation is appropriate, decisions are required on how economic data are to be collected and analyzed. This paper discusses these and other issues and, where appropriate, provides recommendations for future studies.

During the past two decades a great deal has been learned about the role of physicians in generating medical costs and about ways of changing physicians' behavior so as to reduce those costs (8;10;19;30;34). Unfortunately, very little is known about how cost reductions affect the quality of care.

Objective: Extracorporeal shock wave therapy (ESWT) as an emerging technology in orthopedics has been assessed in Austria with the objective to establish a scientific basis for pending and pressing health policy decisions. Despite encouraging results within some indications and the promising prospect of a noninvasive treatment for some orthopedic diseases, it seemed crucial to assess this new field of application in the light of evidence-based standards, without forgetting the reality of healthcare decisions. This article presents the results of the abovementioned assessment in the context of the policy-making process in Germany, Austria, and Switzerland.

Methods: The study is based on an overview of the already existing reviews on the effectiveness of ESWT as carried out by different actors (health insurances and orthopedists), and on an overview of additional literature covering all relevant aspects (mode of action, recommendations, economic estimates). Orthopedic societies, several institutions involved in health technology assessment, and health insurance agencies were contacted in search of further relevant literature, ongoing studies, and assessments.

Results: Until now, clear evidence has not been available on the effectiveness of ESWT in four currently recommended indications (tendinosis calcarea of the shoulder, epicondylopathia humeri radialis, calcaneal spur, and pseudarthrosis), although a substantial body of literature has been produced. Despite encouraging results in some indications, the lack of coherent therapy schemes and the poor quality in the design of clinical studies has been serious enough to hinder the drawing of reasonable conclusions.

Conclusions: Due to the rapid diffusion of ESWT and the pressure on reimbursement agencies to prove the ineffectiveness of ESWT, the policy option of a pragmatic approach is recommended to controll the diffusion of an uncertain health technology, alongside active research that would allow a more comprehensive appraisal of this potentially interesting treatment strategy in orthopedics.

Objectives: Health technology assessment (HTA) has to innovate to best support changing health system environments and to help provide access to valuable innovation under fiscal constraint.

Methods: Issues associated with changing HTA paradigms were identified through scoping and explored through deliberation at a meeting of industry and HTA leaders.

Results: Five broad areas of change (engagement, scientific dialogue, research prioritization, adaptive approaches, and real world data) were identified. The meeting focused on two themes derived from these: re-thinking scientific dialogue and multi-stakeholder engagement, and re-thinking value, affordability, and access. Earlier and ongoing engagement to steer the innovation process and help achieve appropriate use across the technology lifecycle was perceived as important but would be resource intensive and would require priority setting. Patients need to be involved throughout, and particularly at the early stages. Further discussion is needed on the type of body best suited to convening the dialogue required. There was agreement that HTA must continue to assess value, but views differed on the role that HTA should play in assessing affordability and on appropriate responses to challenges around affordability. Enhanced horizon scanning could play an important role in preparing for significant future investments.

Conclusions: Early and ongoing multi-stakeholder engagement and revisiting approaches to valuing innovation are required. Questions remain as to the most appropriate role for HTA bodies. Changing HTA paradigms extend HTA's traditional remit of being responsive to decision-makers demands to being more proactive and considering whole system value.

Objectives: Chondral defects of the knee cartilage are prevalent. Autologous chondrocyte implantation (ACI) and mosaicplasty are increasingly used to treat symptomatic knee defects. This study assessed the costs and health status outcomes after ACI and mosaicplasty.

Methods: Patients were eligible to participate in this cross-sectional study if they received ACI or mosaicplasty at the Royal National Orthopaedic Hospital between 1997 and 2001 or were on a waiting list for ACI. Secondary-care resource use was collected to 2 years postoperatively using a resource collection proforma. Participants responded to postal questions about sociodemographic characteristics and knee-related (Modified Cincinnati Knee Rating System) and general health status (EQ-5D).

Results: Fifty-three ACI, twenty mosaicplasty, and twenty-two patients waiting for ACI participated. The average cost per patient was higher for ACI (£10,600: 95 percent confidence interval [CI], £10,036–£11,214) than mosaicplasty (£7,948: 95 percent CI, £6,957–£9,243). Postoperatively, ACI and mosaicplasty patients (combined) experienced better health status than those waiting for ACI. ACI patients tended to have better health status outcomes than mosaicplasty patients (not statistically significant). Estimated average EQ-5D social tariff improvements for quality-adjusted life year (QALY) calculations were 0.23 (ACI) and 0.06 (mosaicplasty). Average costs per QALY were £23,043 (ACI) and £66,233 (mosaicplasty). The incremental cost effectiveness ratio (ICER) for providing ACI over mosaicplasty was £16,349.

Conclusions: Average costs were higher for ACI than mosaicplasty. However, both the estimated cost per QALY and ICER for providing ACI over mosaicplasty fell beneath an implicit English funding threshold of £30,000 per QALY. Prospective studies should include measures of utility to confirm the estimated cost utility ratios of ACI and mosaicplasty.

Objectives: Differential pricing, based on countries’ purchasing power, is recommended by the World Health Organization to secure affordable medicines. However, in developing countries innovative drugs often have similar or even higher prices than in high-income countries. We evaluated the potential implications of trastuzumab global pricing policies in terms of cost-effectiveness (CE), coverage, and accessibility for patients with breast cancer in Latin America (LA).

Methods: A Markov model was designed to estimate life-years (LYs), quality-adjusted life-years (QALYs), and costs from a healthcare perspective. To better fit local cancer prognosis, a base case scenario using transition probabilities from clinical trials was complemented with two alternative scenarios with transition probabilities adjusted to reflect breast cancer epidemiology in each country.

Results: Incremental discounted benefits ranged from 0.87 to 1.00 LY and 0.51 to 0.60 QALY and incremental CE ratios from USD 42,104 to USD 110,283 per QALY (2012 U.S. dollars), equivalent to 3.6 gross domestic product per capita (GDPPC) per QALY in Uruguay and to 35.5 GDPPC in Bolivia. Probabilistic sensitivity analysis showed 0 percent probability that trastuzumab is CE if the willingness-to-pay threshold is one GDPPC per QALY, and remained so at three GDPPC threshold except for Chile and Uruguay (4.3 percent and 26.6 percent, respectively). Trastuzumab price would need to decrease between 69.6 percent to 94.9 percent to became CE in LA.

Conclusions: Although CE in other settings, trastuzumab was not CE in LA. The use of health technology assessment to prioritize resource allocation and support price negotiations is critical to making innovative drugs available and affordable in developing countries.

Objectives: There is a growing need for efficient procedures for identification of emerging technologies by horizon scanning systems. We demonstrate the value of best-worst scaling (BWS) in exploring clinicians’ views on emerging technologies that will impact outcomes in hepatocellular carcinoma (HCC) in the next 5 to 10 years.

Methods: Clinicians in Asia, Europe, and the United States were surveyed and their views about eleven emerging technologies relevant to HCC were explored using BWS (case 1). This involved systematically presenting respondents with subsets of five technologies and asking them to identify those that will have the most and least impact on HCC within 5 to 10 years. Statistical analysis was based on sequential best-worst and analyzed using conditional logistic regression.

Results: A total of 120 clinicians uniformly distributed across ten countries completed the survey (37 percent response rate). Respondents were predominately hepatologist (41 percent) who focused on HCC (65 percent) and had national influence in this field (39 percent). Respondents viewed molecular targeted therapy (p < .001) and early detection of HCC (p < .001) as having most potential, while improved surgical techniques (p < .001) and biopsy free HCC diagnostics (p < .001) were viewed upon negatively.

Conclusions: We demonstrate that BWS could be an important research tool to facilitate horizon scanning and HTA more broadly. Our research demonstrates the value of including clinicians’ preferences as a source of data in horizon scanning, but such methods could be used to incorporate the opinions of a broad array of stakeholders, including those in advocacy and public policy.

Objectives: There is little specific guidance on performing an early cost-effectiveness analysis (CEA) of medical tests. We developed a framework with general steps and applied it to two cases.

Methods: Step 1 is to narrow down the scope of analysis by defining the test's application, target population, outcome measures, and investigating current test strategies and test strategies if the new test were available. Step 2 is to collect evidence on the current test strategy. Step 3 is to develop a conceptual model of the current and new test strategies. Step 4 is to conduct the early-CEA by evaluating the potential (cost-)effectiveness of the new test in clinical practice. Step 5 involves a decision about the further development of the test.

Results: The first case illustrated the impact of varying the test performance on the headroom (maximum possible price) of an add-on test for patients with an intermediate-risk of having rheumatoid arthritis. Analyses showed that the headroom is particularly dependent on test performance. The second case estimated the minimum performance of a confirmatory imaging test to predict individual stroke risk. Different combinations of sensitivity and specificity were found to be cost-effective; if these combinations are attainable, the medical test developer can feel more confident about the value of further development of the test.

Conclusions: A well-designed early-CEA methodology can improve the ability to develop (cost-)effective medical tests in an efficient manner. Early-CEAs should continuously integrate insights and evidence that arise through feedback, which may convince developers to return to earlier steps.

Objectives: This study explores the factors that enhance or reduce the prospects for public involvement in the activities of health technology assessment (HTA) agencies.

Methods: The analytical framework for this study is based on the work of John W. Kingdon, which provides a comprehensive synthesis of the factors influencing governments and public organizations' agenda. The study draws insights from forty-two semistructured telephone interviews with informants involved in international HTA networks and/or in HTA agencies in Canada, Denmark, and the United Kingdom.

Results: This exploratory study suggests that the HTA community is moving toward greater public involvement. However, the HTA community remains cautious and ambivalent about the technical feasibility of public involvement, its acceptability to policy makers and practitioners, and its impacts on HTA agencies' resources and procedures.

Conclusions: The study stresses the importance of conducting rigorous and compelling evaluations to inform HTA agencies' decision to adopt, or reject, public involvement practices.

Objectives: The aim of this study was to demonstrate the best way of identifying all relevant published health economic evaluation studies, which have increased in number rapidly in the past few decades. Nevertheless, health technology assessment projects are often faced with a scarcity of relevant studies.

Methods: Six bibliographic databases were searched using various individually adapted strategies. The particular example involves the cost-effectiveness of diagnosing gastroesophageal reflux disease. Inclusion and exclusion criteria were formulated.

Results: After irrelevant studies and duplicates had been excluded, sixty-eight abstracts were reviewed. We chose forty-one of them as relevant for full-text review, which identified fourteen papers as having met the inclusion criteria. Most of the relevant studies were identified by searching the National Health Service Economic Evaluation Database (NHS EED) and PubMed databases.

Conclusions: A search in NHS EED, by means of the Cochrane Library or the Center for Reviews and Dissimination, along with a supplementary search in PubMed, is generally an appropriate, cost-effective strategy. However, because “cost-effectiveness” is not consistently indexed with Medical Subject Heading terms in PubMed, all economic search terms need to be used to fully identify the relevant references.

Objectives: The aim of this study was to review the history of health technology assessment (HTA) in Italy.

Methods: Founded in 1978, the Italian National Health Service (NHS) has been strongly regionalized mainly after a constitutional reform, which started a devolution process. HTA started in the 1980s at the National Institute of Health and in a few University Hospitals, with a focus on big ticket technology: that process was driven by clinical engineers.

Results: In recent years, HTA is becoming an important tool for decision-making processes at central, regional, and local levels. In particular, the National Agency for Regional Health Services (AGENAS) and five regions (of twenty-one) are strongly committed to develop HTA initiatives connected with the planning process.

Conclusions: At the local level, the hospital-based HTA activity is probably the most important peculiarity of the country and the real driver of the HTA movement.

Health technology assessment (HTA) has increasingly become a valuable tool to examine effectiveness, quality of care, patient outcomes, and cost-effectiveness of health care technologies. In 1994 and 1995, a survey was conducted to document the scope of worldwide HTA activities; 103 organizations from 24 countries responded. This paper summarizes the results of this international survey, including a description of each country's program, types of technologies assessed, methodologies used, and dissemination approaches. Results for Canada, the United States, and European and other countries are presented separately.

We describe an evaluation model to guide public coverage of new predictive genetic tests in Ontario, Canada. The model confronts common “gray zones” in evaluation and coverage policy for challenging new technologies. Analysis addresses three domains of the evaluation picture. The first specifies evaluative criteria (purpose, effectiveness, additional effects, unit cost, demand, cost-effectiveness). The second induces or deduces acceptable cutoffs for each criterion. The third domain addresses the need to make decisions under uncertainty and to respond to “gray” evaluations with conditional-coverage decisions. The evaluation criteria should be applied within sound decision-making processes.

Objectives: Several models of hospital-based health technology assessment (HTA) have been developed worldwide, for the introduction of innovative medical devices and support evidence-based decision making in hospitals. Two such models, the HTA unit and mini-HTA models, are widespread in university hospitals and involve various stakeholders. The purpose of this work was to highlight the potential role of hospital pharmacists in hospital-based HTA activities.

Methods: We searched for articles, reviews, and letters relating to hospital-based HTA, as defined by the Hospital-Based Health Technology Assessment Worldwide Surveypublished by the Health Technology Assessment International (HTAi) Society, in the Health Technology Assessment database, MEDLINE, EMBASE, and hospital pharmacyjournals.

Results: The number of university hospitals performing hospital-based HTA has increased since the 2008 Hospital-Based Health Technology Assessment Worldwide Survey. Our own experience and international findings show that hospital pharmacists already contribute to hospital-based HTA activities and have developed study interpretation skills and a knowledge of medical devices.

Conclusions: Promoting multidisciplinary approaches is one of the key success factors in hospital-based HTA. Hospital pharmacists occupy a position between hospital managers, clinicians, health economists, biomedical engineers, and patients and can provide a new perspective. In the future, hospital pharmacists are likely to become increasingly involved in hospital-based HTA activities.