Health technology assessment (HTA) in France covers a variety of activities performed for different customers (e.g., health professionals in the field and policy makers in government) for the benefit of patients. To promote the improvement of quality in health care, France has set up a series of distinct agencies that report to the Ministry of Health but are also accountable to their other customers. We place particular emphasis on ANAES (National Agency for Accreditation and Evaluation in Health) whose main remit is HTA. We show how the diversity of HTA activities and their decentralization suggests tight collaboration among all the different bodies which perform HTA or are closely involved with HTA, and we provide examples of such collaboration.

Pharmaceutical manufacturers, regulators, patients, providers, and payers all have a shared interest in improving health outcomes for patients with cancer. Each plays an important role in helping to achieve this common goal. Pharmaceutical manufacturers seek to develop new medicines that are supported by robust and clinically meaningful evidence of their safety, efficacy, and effectiveness. Regulators authorize these medicines based on evaluations of their safety and efficacy. Patients and providers together make treatment decisions and desire access to the most effective treatment options. Payers appraise new medicines with the goal of ensuring access to those medicines that constitute efficient uses of healthcare expenditure. Profits generated from the sale of the medicines provide a return to the manufacturer, which helps to drive continued research and development in an effort to improve patient health outcomes and societal well-being. Many payers have initiated health technology assessment (HTA) activities to inform decision making in light of the rising costs of health care. Given the financial constraints imposed as a result of the global economic crisis, HTAs are likely to become increasingly important as payers seek value for money solutions to major health problems. Successful collaboration and aligning incentives across stakeholders is critical to ensuring that patients are able to access the most effective medicines.

The purpose of this paper is to discuss the potential feasibility and utility of evidence-informed deliberative processes (EPDs) in low income country (LIC) contexts. EDPs are implemented in high and middle income countries and thought to improve the quality, consistency, and transparency of decisions informed by health technology assessment (HTA). Together these would ultimately improve the legitimacy of any decision making process. We argue—based on our previous work and in light of the priority setting literature—that EDPs are relevant and feasible within LICs. The extreme lack of resources necessitates making tough decisions which may mean depriving populations of potentially valuable health technologies. It is critical that the decisions and the decision making bodies are perceived as fair and legitimate by the people that are most affected by the decisions. EDPs are well aligned with the political infrastructure in some LICs, which encourages public participation in decision making. Furthermore, many countries are committed to evidence-informed decision making. However, the application of EDPs may be hampered by the limited availability of evidence of good quality, lack of interest in transparency and accountability (in some LICs), limited capacity to conduct HTA, as well as limited time and financial resources to invest in a deliberative process. While EDPs would potentially benefit many LICs, mitigating the identified potential barriers would strengthen their applicability. We believe that implementation studies in LICs, documenting the contextualized enablers and barriers will facilitate the development of context specific improvement strategies for EDPs.

Objective: To determine if a pre-assessment can be used to establish whether cost-effectiveness results would meet the actual information needs of Dutch healthcare decision makers.

Methods: Two recent studies in rehabilitation medicine served as study material. Based on Wholey, a limited pre-assessment was performed in which the potential impact of cost-effectiveness analysis (CEA) results on intended users' decision making was assessed. Desk research and semi-structured interviews with several intended users of CEA results were performed. These included general practitioners, representatives of health insurance companies, the Health Care Insurance Board (CvZ), and medical guidelines committees.

Results: In day-to-day decision making of the interviewed decision makers, a cost-effectiveness criterion seemed to be of limited importance. Instead, results from clinical effectiveness studies and budget impact studies appeared to be sufficient. CvZ, however, preferred relative cost-effectiveness to be a criterion for inclusion in future reimbursement guidelines. In both cases the limited pre-assessments changed the expectations of the investigators regarding decision-making impact of an economic evaluation.

Conclusion: This study revealed that the use of CEA results for Dutch micro- and meso-level healthcare decision making is not self-evident. The main purpose of CEA results is to support health policy making and planning at a macroeconomic level. Pre-assessment can be a valuable tool in designing a CEA to support the actual information needs of the decision makers.

Thirty-five patients were randomized to extracorporeal shock-wave lithotripsy (ESWL) and 25 to laparoscopic cholecystectomy (LC). Stone disappearance occurred in only 12 of 32 ESWL patients [38% (95% Cl: 21–56%)] during a 15-month follow-up. Greater incremental gains in quality of life after 6 months were observed among LC patients (p <.01). Total duration of disability was 6.8 ± 8.5 days for ESWL, and 22.7 ± 16.6 days for LC (p <.01). Nine (28%) patients crossed over electively to the LC group, but only 44% of these underwent LC within the next 3 years. ESWL cost Can $58.9/ day of disability saved. ESWL is limited by its selective applicability and modest stone disappearance rate. Its cost-effectiveness is largely dependent on patient acceptance of recurrent episodes of biliary colic due to the persistence of stone fragments.

Objectives: Adjuvant trastuzumab is widely used in HER2-positive (HER2+) early breast cancer, and despite its cost-effectiveness, it causes substantial costs for health care. The purpose of the study was to develop a tool for estimating the budget impact of new cancer treatments. With this tool, we were able to estimate the budget impact of adjuvant trastuzumab, as well as the probability of staying within a given budget constraint.

Methods: The created model-based evaluation tool was used to explore the budget impact of trastuzumab in early breast cancer in a single Finnish hospital district with 250,000 inhabitants. The used model took into account the number of patients, HER2+ prevalence, length and cost of treatment, and the effectiveness of the therapy. Probabilistic sensitivity analysis and alternative case scenarios were performed to ensure the robustness of the results.

Results: Introduction of adjuvant trastuzumab caused substantial costs for a relatively small hospital district. In base-case analysis the 4-year net budget impact was €1.3 million. The trastuzumab acquisition costs were partially offset by the reduction in costs associated with the treatment of cancer recurrence and metastatic disease.

Conclusions: Budget impact analyses provide important information about the overall economic impact of new treatments, and thus offer complementary information to cost-effectiveness analyses. Inclusion of treatment outcomes and probabilistic sensitivity analysis provides more realistic estimates of the net budget impact. The length of trastuzumab treatment has a strong effect on the budget impact.

Objectives: To conduct a systematic review of selected health determinants, including gender, and their impact on hospitalization rates for depression. Depression includes both depressive and bipolar disorders. Selected health determinants were gender, age, sex, family structure, education, and socioeconomic status.

Methods: Systematic search of conventional and fugitive literature sources. All reports of primary data, systematic reviews, and meta-analysis of primary data were included if they focused on hospitalization for depression and reported data by one or more of the selected health determinants. Two researchers independently evaluated each citation for inclusion and extracted data from the included studies.

Results: There is an important underreporting of health determinants data in studies of hospitalization for depression. No studies examined the role of gender. Age and sex were reported in 83 percent and 80 percent of the 110 included studies. Women showed a higher rate of hospitalization for depression than men (p<.05). Age and diagnosis had different effects in men and women. Adult women were significantly more likely than men to report a depressive disorder, whereas men were more likely to report a bipolar disorder (p<.05). Little can be concluded on the other health determinants.

Conclusions: The importance of reporting hospitalization data and conducting hospital utilization analysis by sex and health determinants, including gender, must be emphasized.

Objectives: Type 2 diabetes is the main cause of end-stage renal disease in Europe and the United States. Angiotensin-converting enzyme (ACE) inhibitors slow down the progression of renal disease and, therefore, provide a renal-protective effect. The aim of this study was to assess the most cost-effective time to start an ACE inhibitor (or an angiotensin II receptor blocker in the event of cough) in patients with type 2 diabetes in Germany.

Methods: Three strategies were compared: treating all patients at the time of diagnosing type 2 diabetes, screening for microalbuminuria, and screening for macroalbuminuria. A lifetime Markov decision model with simulated 50-year-old patients with newly diagnosed diabetes mellitus was developed using published data on costs and health outcomes and simulating the progression of renal disease. A statutory health insurance perspective was adopted.

Results: In the base-case analysis, the treat-all strategy is associated with the lowest costs and highest benefit and, therefore, dominates screening both for macroalbuminuria and microalbuminuria. A multivariate sensitivity analysis shows that the probability of savings is 89 percent.

Conclusions: Patients with type 2 diabetes should receive an ACE inhibitor immediately after diagnosis if they do not have contraindications. The potential for cost savings would be even larger if the prevention of cardiovascular events were considered.

Technology assessment is becoming increasingly important in the area of critical care due both to the explosion of technology associated with this discipline and to the realization that future demand for these health care resources will undoubtedly exceed the ability to pay. Technology assessment remains both confusing and controversial to many physicians. This review tries to address some of the confusion by reviewing the basic strategies involved in this process. From there, problems and prospects for the evaluation of critical care as a program are presented, followed by the assessment of components within the area of critical care. Finally, recommendations are made on how technology assessment could proceed in the future to best achieve the efficient provision of this service.

Objectives: The aim of this study was to evaluate the cost-effectiveness of neonatal screening for phenylketonuria (PKU) and medium-chain acyl-coA dehydrogenase (MCAD) deficiency using tandem mass spectrometry (tandem MS).

Methods: A systematic review of clinical efficacy evidence and cost-effectiveness modeling of screening in newborn infants within a UK National Health Service perspective was performed. Marginal costs, life-years gained, and cost-effectiveness acceptability curves are presented.

Results: Substituting the use of tandem MS for existing technologies for the screening of PKU increases costs with no increase in health outcomes. However, the addition of screening for MCAD deficiency as part of a neonatal screening program for PKU using tandem MS, with an operational range of 50,000 to 60,000 specimens per system per year, would result in a mean incremental cost of −£17,298 (−£129,174, £66,434) for each cohort of 100,000 neonates screened. This cost saving is associated with a mean incremental gain of 57.3 (28.0, 91.4) life-years.

Conclusions: Cost-effectiveness analysis using economic modeling indicates that substituting the use of tandem MS for existing technologies for the screening of PKU alone is not economically justified. However, the addition of screening for MCAD deficiency as part of a neonatal screening program for PKU using tandem MS would be economically attractive.

The use of new therapeutic and diagnostic technologies has become commonplace in modern medical practice. To avoid both clinical disappointment and the waste of money, health, and lives, the introduction of these technologies will have to be based on evidence that these technologies will do more good than harm. The evidence supporting their use should be derived using research methods designed to deal with placebo effects, confounders, and biases. Some of these methods, and the rationale for their use, are discussed in this article. Although the value of evidence derived from randomized controlled trials is stressed, the importance of reviewing critically the methodological details of such trials and interpreting their results with caution is emphasized. The benefits and risks of relying on case-control and cohort studies are reviewed.

Objectives: Patient preferences should be a central consideration in healthcare decision making. However, stories of patients challenging regulatory and reimbursement decisions has led to questions on whether patient voices are being considered sufficiently during those decision making processes. This has led some to argue that it is necessary to quantify patient preferences before they can be adequately considered.

Methods: This study considers the lessons from the use of multi-criteria decision analysis (MCDA) for efforts to quantify patient preferences. It defines MCDA and summarizes the benefits it can provide to decision makers, identifies examples of MCDAs that have involved patients, and summarizes good practice guidelines as they relate to quantifying patient preferences.

Results: The guidance developed to support the use of MCDA in healthcare provide some useful considerations for the quantification of patient preferences, namely that researchers should give appropriate consideration to: the heterogeneity of patient preferences, and its relevance to decision makers; the cognitive challenges posed by different elicitation methods; and validity of the results they produce. Furthermore, it is important to consider how the relevance of these considerations varies with the decision being supported.

Conclusions: The MCDA literature holds important lessons for how patient preferences should be quantified to support healthcare decision making.

We examined the cost-effectiveness of screening for gastric cancer in hypothetical cohorts of asymptomatic Japanese ages 40, 50, 60, and 70, followed for 10 years. Current screening recommendations in Japan are not optimal. Although indirect x-ray is the correct method, screening for gastric cancer may not be cost-effective for Japanese under the age of 50. Policymakers worldwide should monitor the changing incidence of disease as they consider and recommend screening policies.

Objectives: Influenza (vernacular name, flu) is a viral infection that causes a high consumption of resources. Several studies have been carried out to provide an economic evaluation of the vaccination programs against influenza. Nevertheless, there is still a lack of evidence about the dynamic effects resulting from the reduction of the transmission power. This study considers the impact on contagiousness of alternative strategies against influenza in people aged 50–64 in Italy, France, Germany, and Spain.

Methods: By using the Influsim 2.0 dynamic model, we have determined the social benefits of different coverage levels in every country compared with the ones currently recommended. We have subsequently performed a Budget Impact Analysis to determine whether the currently recommended coverage results from an optimal budget allocation. A probabilistic sensitivity analysis was also conducted.

Results: We found that in Germany, the optimal coverage level is 38.5 percent, in France 32.4 percent, in Italy 32.75 percent, and 28.3 percent in Spain. By extending the coverage level, social saving tends to increase up to 100 percent for France and Italy and up to 80 percent for Germany and Spain.

Conclusions: Decision makers should allocate the budget for vaccination against influenza consistently with the estimation of the optimal coverage level and with the dynamic effects resulting from the reduction of the transmission power.

It should be pointed out that during the interviews most of the experts had positive expectations of ESM. The developers are more generally enthusiastic than the prospective users and affected parties who, especially in respect of the diffusion of ESM into practical application, only show a limited optimism.

However, the representatives of the medical profession and the health insurance industry were convinced that ESM might contribute to cost-neutral increases of quality in out-patient and in-patient medicine. But we also understood them to say that they consider other developments in medicine to be overriding, for example, a tendency of general medicine towards a more family-oriented medicine and a reduced emphasis on technology-oriented medicine. In respect of the conception shared by developers as well as potential users that over-enthusiastic expectations should rather be restrained, we consider such a balanced expectation of positive effects of ESM to be adequate to the actual knowledge of the subject.

This article describes a new group-judgment process, modified from the consensus development approach of the U.S. National Institutes of Health. It is called the “forum” method, a shorthand term for “technology assessment and practice guidelines forum.” This approach is aimed at (a) describing the current state of knowledge of the technology under study; (b) developing practice guidelines for the use of the technology; and (c) providing information for insurers, consumers, policymakers, and others. A pilot study was conducted focusing on total parenteral nutrition. The validity of the process and of the recommendations and their acceptability were evaluated in a survey of participants and a selected group of nonparticipants. Based on the survey results, the forum method appears to be a valuable method for assessing technologies, defining their clinical role, and developing practice guidelines.

Clinical judgment is increasingly being challenged by the need for randomized clinical trials. The 1987 National Cancer Institute mandate—that the Community Clinical Oncology Program (CCOP) accrue patients to cancer control protocols—provided an opportunity to examine the factors that affect accrual performance. An analysis of 52 CCOPs and their research bases participating in the program found that the availability of protocols, involvement with research base activities, a demonstrated link to community physicians (particularly those physicians, such as surgeons, who had access to patients), and the use of personal contacts to inform non-CCOP physicians about CCOP activities were important facilitating factors for accruing patients to cancer prevention and control trials.