This article presents the pharmaceutical industry's perspective on health technology assessment (HTA) with specific comments on the HTA systems in England and Wales, France, The Netherlands, and Sweden. The comments are focused on the following main themes: (i) The contributions of the HTA system to overall efficiency in the health-care system, (ii) HTA as a cost-driver for industry, patients, government, and society, and (iii) The various implementation barriers that currently exist for a successful implementation of HTA results.

The Spanish Constitution of 1978 established a healthcare system available to everyone and free at the point of service. The General Health Law of 1986 also established the framework for a National Health System (NHS). The Constitution and the law form the regulatory framework for the devolution of healthcare services to the Autonomous Regions. All the 17 Autonomous Regions have complete power regarding public health and planning. However, responsibilities on healthcare financing, organization, provision, and management have devolved to only seven Autonomous Regions. Financial support for health services comes mostly from taxes. Global budgets are a mechanism used by hospitals to control the acquisition of medium and low health technology. Major capital investments for health technology are controlled by the central government in 10 Autonomous Regions (population coverage of 38%) and by the Regional Health Services in the seven remaining Autonomous Regions. In 1995 a regulation for basing the introduction of new procedures and medical equipment on the assessment of safety, efficacy, and efficiency was issued. Health technology assessment (HTA) has a long history in Spain, beginning with the Advisory Board on High Technology in the government of Catalonia in 1984. This board evolved into the Catalan Agency for HTA (CAHTA) in 1994. The Basque Country established a unit for HTA in 1992 (Osteba) and the Andalusian government created an agency in 1996 (AETSA). A national agency for HTA (AETS) was established in 1994. These different programs coordinate their work and together act as an Advisory Committee of the Interregional Council of the NHS.

Changing compliance with medication directives can greatly affect health outcomes. To assess programs that influence compliance, accurate measurement of compliance is essential. All currently available methods are imperfect. This paper suggests a new method—the “randomized response interview”—along with a strategy for its implementation.

Background: We calculated the daily cost of implantable cardioverter-defibrillators (ICDs) based on their actual longevity to prove whether the up-front cost is a reliable parameter for the ICD purchasing-process.

Methods. Longevity of single chamber (SC), double chamber (DC), and biventricular (BiV) ICDs from Medtronic (MDT), Guidant (GDT), and St. Jude Medical (SJM) was measured in all the patients implanted in years 2000, 2001, 2002 who reached device replacement within December 31, 2009. The cost of each ICD (device + lead/s) was normalized for its own longevity. Data are expressed as median (25th–75th percentile).

Results: A total of 123/153 patients completed the study, 70 percent being alive 8 years after implantation. MDT devices had a superior longevity compared with GDT and SJM (p < .001). Fifty-eight percent of replaced ICDs had a service life at least 1 year shorter than the manufacturers' prediction. Longer-lasting devices had a significantly lower daily cost: €4.8 (4.6–5.7) versus €6.8 (6.2–9.2) and €6.9 (6.2–7.6) for SC (p < .001); €6.9 (6.8–7.7) versus €12.6 (11.8–13.3) and €13.4 (10.3–16.1) for DC; €8.5 (8.3–10.3) versus €15.4 (15.1–15.8) and €14.6 (14.1–14.9) for BiV (p < .005).

Conclusions: The true cost of ICD treatment is strictly dependent on device longevity, whereas device up-front cost is unreliable. This aspect should be valued in the technology purchasing process, and could set the basis for an outcome-based reimbursement system. Our observations may be the benchmark respectively for ICD longevity and daily ICD cost in future comparisons. Independent observations in the real-life scenario are needed to properly value newer technologic improvements.

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

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

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

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

Objectives: The objectives of this study are to review the financing and organization of the Argentine healthcare system, the licensing and drug price setting mechanisms, the benefit packages and coverage policies of pharmaceuticals and other medical technologies, as well as the development of HTA in Argentina, and the role of the Institute of Clinical Effectiveness and Health Policy (IECS) as an HTA agency. Finally, the perspectives and future of HTA as a tool to make resource-allocation decisions and priority setting in Argentina is discussed.

Methods: The study is a discussion/review based largely on the experiences of the authors, but supported by available literature.

Results: Argentina is an upper-middle income country with major healthcare problems related to both equity and efficiency. Its healthcare system consists of a multitier system divided in three large sectors: public, social security, and private, where the federal Ministry of Health has a rather limited role in national health policy stewardship. Many of Argentina's shortcomings are due in part to its pluralistic and fragmented healthcare system. In the past decade, Argentina, like many other Latin American countries, has undergone a profound reform of its healthcare system. Whereas some of the objectives of the reforms were specific to each country, a common issue among all of them was to establish a mechanism that ensured a more efficient allocation of scarce resources, and guaranteed a wider provision of healthcare services on the basis of the local population needs and equity. Although some signals from the national government and congress show that there are plans to formally incorporate HTA to inform reimbursement policies, these signals are still very weak. Paradoxically, even though Argentina was the first country in the region to require formal health economic evidence for the adoption of technologies into the mandatory benefit package of the social security, this “fourth hurdle” is no longer required. Nevertheless, there is an increasing interest and demand for a more explicit and transparent resource-allocation process that include HTA as a formal tool to inform decision making, in most of Argentine healthcare stakeholders.

Conclusions: In conclusion, what is needed in Argentina is a clear political will to push forward for a national agency of HTA that, similar to other developed countries, advance the regulation on the adoption of new health technologies to improve not only technical or allocative efficiency, but also health equity. Until this milestone is accomplished, the HTA production and use to inform healthcare coverage policies will continue to mirror the current fragmented healthcare system.

On the basis of two case histories of medical device innovation, two aspects of demand are identified: (a) the prospect of new markets and (b) information about user needs. Conditions for direct interaction between the user and producer in the innovation process are identified. The article concludes with some normative implications for the publicly designed generation of new medical technology.

Interest in Bayesian analyses has increased recently, in part as a response to policy makers wanting sound scientific bases for health technology assessments, and associated healthcare funding decisions. This paper provides a brief and simplified description of Bayesian reasoning. Bayes is illustrated in a clinical setting of an expert helping a woman understand the potential risk of passing on an inheritable disease (hemophilia) to her next child, based on disease occurrence in two living children. The illustration describes fundamental concepts and derivations, such as Bayes theorem, likelihood functions, prior probability, and posterior probability. A second illustration shows the use of Bayes for interpreting clinical trial results. The uncertainty in the clinical effect before and after the trial analyses has been completed is characterized by the Bayes prior and posterior probabilities, respectively. Techniques are also shown for estimating the potential loss (e.g., in lives lost) for making the wrong decision with and without knowledge of the trial results, an estimation that cannot be carried out using techniques of hypotheses testing associated with the frequentist school of statistics. Information from Bayes analysis then may be used to help policy makers decide, or justify, whether the analyses provides a sufficient basis for making a treatment recommendation, or whether there remains a need to request more information. Subsequent papers in this volume offer additional examples and clarification of the use of Bayes in clinical practice and in interpretation of clinical studies.

Objectives: The aim of this study was to examine the impact of the National Institute for Health and Clinical Excellence's (NICE's) negative and restricting technology appraisals on the number of prescription items dispensed and the corresponding total net ingredient costs for drugs from 2000 to 2004 in the ambulatory care of the National Health Service (NHS) in England and Wales. In addition, it is discussed whether the NICE approach could be a role model for Germany.

Methods: The number of prescription items dispensed and the net ingredient costs of thirty-one drugs reimbursed by the NHS were analyzed, thereof thirteen drugs descriptively and twenty-one drugs with regression analyses. Data were extracted from the “Prescription-Costs-Analysis-Statistics” for the ambulatory care of the British Department of Health (England 1993–2005). In the case of the twenty-one drugs analyzed by regression analyses, predictions were established how the prescribing and the costs would have developed without NICE's drug appraisal. Finally, conclusions were drawn whether NICE's negative and restricting drug appraisals had a decreasing effect or not.

Results: For 97 percent of the drugs analyzed in this study, the publication of NICE's fourteen negative and restricting technology appraisals of drugs between 2000 and 2004 did not reduce the number of prescription items dispensed and net ingredient costs in the ambulatory care of the NHS in England and Wales.

Conclusions: Cost-effectiveness appraisals as performed by NICE or the German Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, IQWiG) are a useful and important tool to enhance the discussion about methods and acceptance of evidence-based medicine in general.

Objectives: The aim of our study was to describe approaches to health technology assessment (HTA) for medicines in the Slovak healthcare system and the related decision-making processes concerning reimbursement for medicines.

Methods: Analysis of the Slovak legislative framework related to HTA and the reimbursement process for medicines was performed. Additionally, current practices of the Working Group for Pharmacoeconomics, Clinical Outcomes and Health Technology Assessment of the Slovak Ministry of Health were evaluated.

Results: In Slovakia, there is always at least one treatment available in each determined therapeutic class with no co-payment. HTA is becoming an established method for the evaluation of cost-effectiveness of medicines in Slovak healthcare policy. The majority of decision makers within Slovakia support the idea of increased use of and the quality and efficiency of HTA methods. However, it is crucial to overcome several practical barriers to facilitate progress in the field of HTA in the Slovak Republic.

Conclusions: It can be seen that participation within the European Network for Health Technology Assessment (EUnetHTA JA 2 and EUnetHTA JA 3 projects) has significantly improved the quality of the process of HTA in Slovakia. Further legislative activities in this field are required due to the approved strategy for European Union cooperation on HTA.

Initial functional ability (Barthel Index, mean 57) was found to be an important predictor of functional ability 1 year after stroke (mean 80) and for costs during the period. On average the total cost for a stroke patient was about SEK 200,000; the main expense, accommodation, averaged about SEK 140,000, while assistive devices amounted to SEK 2,600. Those who use assistive devices, although having achieved a high functional ability, perceive and rate their life situation (Nottingham Health Profile) considerably more impaired than those without assistive devices.

Health technology assessment (HTA) (1), also known as healthcare technology assessment or medical technology assessment, is a form of policy research that systematically examines short- and long-term consequences of the application of a health technology, a set of related technologies, or an issue related to technology. The goal of HTA is to provide input to decision making in policy and practice. The essential properties of HTA are this orientation to decision making and its multidisciplinary and comprehensive nature.

The purpose of this study was to identify the determinants of choice of surgical procedure (anterior colporrhaphy, colposuspension, or needle suspension) to treat stress incontinence in women. We used multilevel modeling of data on 271 patients in 18 hospitals in England in 1993–94. Patient-related factors included sociodemographic details, anatomical diagnosis, symptom severity, symptom impact, previous treatment, parity, comorbidity, and general health status. Surgeon-related factors were specialty, grade, and annual volume of procedures undertaken. Hospital teaching status was considered. Some patient-related factors were associated with choice of procedure: women with a concomitant genital prolapse, with a history of high parity, and with no previous nonsurgical treatment were more likely to undergo an anterior colporrhaphy than a colposuspension or needle suspension (although this finding could be confounded by surgical specialty). In addition, women were more likely to be treated by colposuspension if their surgeon specialized in incontinence surgery (measured by annual volume of cases). Finally, being treated by needle suspension depended on there being a consultant surgeon familiar with the procedure at the hospital attended. While choice of surgical procedure depends partly on the patient's anatomical diagnosis, it is also dependent on the specialty of the surgeon whom she consults and the hospital that she attends. This variability, in turn, could have implications for the patient (as the relative effectiveness of the different procedures is unknown) and for the purchasers of care (as the relative cost-effectiveness of procedures is also unknown).

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: The objective of this study is to illustrate and provide a better understanding of the role of health technology assessment (HTA) processes in decision making for drug reimbursement in Poland and how this approach could be considered by other countries of limited resources.

Methods: We analyzed the evolution of the HTA system and processes in Poland over the past decade and current developments based on publicly available information.

Results: The role of HTA in drug-reimbursement process in Poland has increased substantially over the recent decade, starting in 2005 with the formation the Agency for Health Technology Assessment and Tariff System (AOTMiT). The key success factors in this development were effective capacity building based on the use of international expertise, the implementation of transparent criteria into the drug reimbursement processes, and the selective approach to the adoption of innovative medicines based on the cost-effectiveness threshold among other criteria.

Conclusions: While Poland is regarded as a leader in Central and Eastern Europe, there is room for improvement, especially with regard to the quality of HTA processes and the consistency of HTA guidelines with reimbursement law. In the “pragmatic” HTA model use by AOTMiT, the pharmaceutical company is responsible for the preparation of a reimbursement dossier of good quality in line with HTA guidelines while the assessment team in AOTMiT is responsible for critical review of that dossier. Adoption of this model may be considered by other countries with limited resources to balance differing priorities and ensure transparent and objective access to medicines for patients who need them.

Objectives: Reducing sexually transmitted infections (STI) and teenage pregnancy through effective health education is a high priority for health policy. Behavioral interventions which teach skills to practice safer sex may reduce the incidence of STIs. We evaluated the cost-effectiveness of school-based behavioral interventions in young people.

Methods: We developed an economic model to estimate the total number of STI cases averted, consequent gain in health related quality of life (HRQoL) and savings in medical costs, based on changes in sexual behavior. The parameters for the model were derived from a systematic literature search on the intervention effectiveness, epidemiology of STIs, sexual behavior and lifestyles, HRQoL and health service costs.

Results: The costs of providing teacher-led and peer-led behavioral interventions were €5.16 and €18 per pupil, respectively. For a cohort of 1000 boys and 1000 girls aged 15 years, the model estimated that the behavioral interventions would avert two STI cases and save 0.35 Quality Adjusted Life Years (QALYs). Compared to standard education, the incremental cost-effectiveness of the teacher-led and peer-led interventions was €24,268 and €96,938 per QALY gained, respectively.

Conclusions: School-based behavioral interventions which provide information and teach young people sexual health skills can bring about improvements in knowledge and increased self-efficacy, though these may be limited in terms of impact on sexual behavior. There was uncertainty around the results due to the limited effect of the intervention on behavioral outcomes and paucity of data for other input parameters.

Objectives: The objective is to describe the principles for the design of the economic evaluation of COLOR II, a randomized, multi-country study comparing laparoscopic and open surgery for rectal cancer.

Methods: By using the experiences gained in a recent economic evaluation in colon cancer, where the same surgical techniques were compared, we could improve the method for identifying and measuring resource use items and also accommodate the use of data from the global study population.

Results: In the design of the study, the uncertainty in the resource-use variables was reduced by considering (i) what aspects drive each variable, (ii) what resource use is related to the intervention, (iii) how data from different countries affects the variable.

Conclusions: The aim was to refine the data collection so that the economic research question could be answered in the best possible way, given the circumstances in the clinical study. Thus, (i) some variables were treated as stochastic variables and others as deterministic variables, (ii) aggregate key cost-driving resource items were developed that corresponded to clinical events, and (iii) a surrogate variable was selected, instead of the “obvious variable”, to reduce the impact of confounding factors for one particular resource unit.