The deregionalization of neonatal intensive care in the United States has shifted the site of care for many newborn infants away from academic medical centers where subspecialty support is available.

Objective: To investigate the effect of immediate echocardiogram interpretation via telemedicine on rates of neonatal transfer to academic medical centers.

Methods: A logit model was developed to predict the probability of transfer from two regional level 3 neonatal intensive care units to academic medical centers. One of these units implemented a telecardiology program and the other acted as a comparison institution with on-site cardiology expertise. The telecardiology intervention began 18 months into the 36-month study period.

Subjects: Infants (n = 2,142) admitted to neonatal intensive care at either of the two institutions during calendar years 1994 through 1996.

Results: A statistically significant reduction in the rate of transfer to academic medical centers was observed. Telecardiology was associated with a 58% reduction of such transfers (p = .001, 95% CI = 30%, 75%). No such reduction was noted at the comparison institution. It is estimated that approximately 30 transfers were eliminated during the study period, resulting in the elimination of approximately $150,000 in hospital charges. In addition, the infants that were transferred after the adoption of telemedicine were more often transferred to their telemedicine partner institution (p < .02).

Objectives: To review the economic evaluations (EEs) done in Italy by Italian authors, following a common scheme to allow some comparisons of the studies selected and with the international reviews.

Methods: We selected all the original studies published by Italian authors (in Italian or English) in national and international journals. The period considered was January 1994 to December 2001. Both full and partial economic evaluations were included. Three international databases were interrogated: MEDLINE, Embase, and HealthStar; further articles were added from the internal database of our center (CESAV), which also classifies Italian local publications and journals specialized in health economics.

Results: A total of ninety-nine studies were reviewed. More than half of the fifty-seven full EEs focused on drugs as type of intervention (n=38), followed by diagnostic screening (n=7). The NHS viewpoint was the most used (n=55 studies), followed by that of society (n=27) and hospitals (n=12). Sixty-eight studies only analyzed direct costs and twenty-nine included both direct and indirect costs. Twenty-five of the thirty-eight pharmacoeconomic full EEs were sponsored by companies. In sixteen of the twenty-five sponsored studies, the sponsor's products were the dominant alternative.

Conclusions: The review showed that, in Italy, like elsewhere, there is a gap between theory and practice in EEs, and sponsors can considerably affect the results of EEs.

This paper critically evaluates methods for the preliminary economic evaluation of health technologies and the prioritization of health technology assessment projects. It reports on the literature, and considers methods currently employed and the purposes of preliminary appraisal. It concludes that a preliminary economic appraisal needs to be applied to the two main stages of the prioritization process; to have transparent criteria; to allow for an appropriate range of potential outcomes; to be practicable, flexible, and efficient; and to be relevant to the assessment of different research projects.

Health technology assessment (HTA) is commonly used to guide evidence-informed decisions to optimize resource use, prioritize policies, and support countries to achieve universal health coverage. Producing HTAs requires time, scientific expertise, and political commitment, but these are not available in all settings – especially in low- and middle-income countries (LMIC) where HTA processes may be less institutionalized. Transferring and adapting existing HTAs to local settings may offer a solution while reducing duplication efforts. This scoping review aims to provide an overview of tools, methods, approaches, and considerations which can aid HTA transfers. We systematically searched (from 2005 to 2020) six databases and, using predefined inclusion criteria, included twenty-two studies. Data extraction followed a structured process, while synthesis was more iterative. We identified a common approach for HTA transfers. It follows the de novo process of undertaking original HTAs, but with additional steps to assess relevance (applicability), quality, and transferability, as well as steps to adapt parameters where necessary. The EUnetHTA Adaptation Toolkit was the only tool that provided guidance for adapting multiple HTA domains. Other tools were specific to systematic reviews (n = 1) or economic evaluations (n = 12), where one provided guidance for systematic reviews of economic evaluations. Eight papers reported transferring an HTA, with only one transferring to an LMIC. Finally, we reported issues that may facilitate or hinder transferability. In conclusion, we identified fourteen transfer approaches in the form of guidance or checklists, but harmonized and pragmatic guidance for HTA transfers to suit settings with limited HTA capacity seems warranted.

Objectives: Clinical endpoints are defined as valid measures of clinical benefit or harm due to treatment, that describe the impact of treatment on how a patient feels, functions, and survives. The choice of endpoints and the manner in which they are reported have a major impact on the relative effectiveness assessment (REA) of pharmaceuticals. The aim of this article is to describe the guideline development process and the key findings that set a framework for appropriate use of endpoints in REAs in Europe.

Methods: A multi-health technology assessment (HTA)-agency collaborative process in EUnetHTA JA1 was used to scope, draft, and finalize methodological guidelines for REA in Europe.

Results: Patient-relevant clinical endpoints can be broadly categorized into: mortality, morbidity and health-related quality of life. A clinical endpoint is a main symptom or sign of a disease that is clinically relevant, valid, reproducible and responsive to change. Preference is for long-term or final endpoints whenever possible. Surrogate endpoints may be used when there is compelling evidence of a clear and consistent correlation of treatment effects on the surrogate and final outcome of interest.

Conclusions: The relevance and hierarchy of the different types of clinical endpoints depend on the research question, disease, and the treatment investigated. Not only the primary endpoint, but also other relevant endpoints are assessed in comparison to adequate comparator(s). This simultaneous assessment of all relevant endpoints is a hallmark of REA.

In vitro fertilization (IVF) stands out as one of the contemporary period's most extraordinary technologies, and its social and ethical consequences among the most far reaching. Despite its uncertain effectiveness and medical consequences, IVF has contributed significantly to the medicalization of infertility and the increasingly imperative character of reproductive technology. New developments in IVF, particularly oocyte donation, have created new definitions of treatable infertility and new social needs for IVF; when the technology does not result in pregnancy or healthy babies, these developments have created profound new disappointments. IVF and the commodification of the extracorporeal embryo have also confused the social meaning and legal definition of parenthood. Ultimately the relationship between prospective parents, infertility specialists, and the embryos that they create is a highly ambiguous one. This ambiguity is likely to be a long-term characteristic of efforts to develop, use, and assess assisted reproductive technologies.

Objectives: The aim of this study was to develop a breast cancer Patient Decision Aid (PDA), using a Health Technology Assessment (HTA) process, to assist patients in their choice of therapeutic options, and to promote shared decision making among patients, healthcare professionals, and other interested parties.

Methods: A systematic review (SR) was conducted of existing breast cancer patient Decision Aids encountered in the main scientific journal databases and on institutional Web sites that create PDAs, together with a Qualitative Research (QR) study, using semi-structured interviews and focus group with stakeholders (patients, family members, and health professionals), with the aim of developing a PDA for breast cancer.

Results: The SR shows that PDAs in breast cancer not only increase patient knowledge of the illness, leading to more realistic expectations of treatment outcomes, but also reduce passivity in the decision-making process and facilitate the appropriate choice of treatment options in accordance with patient medical and personal preferences. The analysis of QR shows that both breast cancer patients and healthcare professionals agree that surgery, adjuvant treatments, and breast reconstruction represent the most important decisions to be made. Worry, anxiety, optimism, and trust in healthcare professionals were determined as factors that most affected patients subjective experiences of the illness. This HTA was used as the basis for developing a PDA software program.

Conclusions: The SR and QR used in the development of this PDA for breast cancer allowed patients to access information, gain additional knowledge of their illness, make shared treatment decisions, and gave healthcare professionals a deeper insight into patient experiences of the disease.

Magnetic resonance imaging (MRI) is a high-cost, new technology with great potential for improving patient care. The lack of a coherent public policy for MRI, or its predecessor computed tomography (CT), has caused considerable problems. Lack of an enunciated public policy has led to inconsistent reimbursement and reimbursement levels that develop in haphazard ways. Furthermore, diffusion has been unpredictable and has led to geographical excesses and deficiencies.

Technology assessments of MRI, although numerous, have used inconsistent criteria to rate MRI's clinical efficacy. The lack of methodologically sound studies of MRI severely hampered early evaluation. This article examines these problems and suggests that the medical profession take a stronger leadership role in developing policies for expensive, promising new diagnostic technologies. The profession should promote, demand, and perform rigorous clinical evaluations of new technologies, and help develop a consensus regarding the criteria for what constitutes a clinically valuable advance in diagnostic 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 objective of this study was to develop a method to allocate treatment effects when patients switch medication frequently in longitudinal observational studies and apply the approach to assess the cost-effectiveness of treatments in the Schizophrenia Outpatient Health Outcomes (SOHO) study.

Methods: Data were collected on patients at entry to the SOHO study at 3, 6, and 12 months. The 12-month follow-up period was considered as three epochs: 0–3 months, 3–6 months, and 6–12 months. Patients who switched treatment at 3 months had their new treatment considered as a new baseline observation, as these two 3-month observations provide two sets of information on the cost-effectiveness of a drug in the first 3 months after initiation. Multivariate regression analysis was used to adjust for baseline covariates. The model allowed for flexible functional forms, and the cost data were modeled using an exponential mean function. Bootstrapping assessed the uncertainty of the estimated parameters and incremental cost-effectiveness analysis decision rule.

Results and Conclusions: We show the feasibility of the epoch analysis approach using data from the SOHO study comparing two antipsychotics. Estimates for the incremental cost and effectiveness per epoch over the full 12-month period are presented. Using the estimates of 200 bootstrap samples, we demonstrate how one drug is cost-effective compared with another.

Objectives: A cost-effectiveness analysis was conducted comparing diagnostic strategies for determining the HER2 status of invasive breast carcinomas, as an indication for trastuzumab at metastatic relapse.

Methods: A decision tree compared five strategies distinguished by (i) the use of immunohistochemical (IHC) and/or fluorescent in situ hybridization (FISH) techniques, and (ii) the test schedule (at initial diagnosis or metastatic relapse). Most cost and effectiveness data came from a French multicentric study of 2,045 patients from eight hospitals. We were not able to select final criteria for trastuzumab effectiveness, because published data rely on IHC techniques not used in France (i.e., HercepTest). We, therefore, selected two intermediate criteria for inappropriate treatment at relapse, that is, patients with HER2-amplified tumors not receiving trastuzumab (Criterion 1) and HER2-nonamplified tumors improperly treated with trastuzumab (Criterion 2). Sensitivity analyses were then performed to assess the robustness of the results to (i) discount rate, (ii) cost of FISH, and (iii) tissue fixation technique.

Results: The strategy using IHC at diagnosis was dominated by the four other strategies. Among these approaches, the only efficient strategy for both criteria was IHC used alone at metastatic relapse; strategies using FISH, or IHC followed by FISH on IHC2+ cases were efficient for Criterion 1, whereas IHC followed by FISH on IHC2+ and 3+ cases was efficient for Criterion 2.

Conclusions: Determining HER2 status at diagnosis, as an indication for trastuzumab at metastatic relapse, incurs substantial incremental costs, which do not appear to be justified. No other strategy can be excluded at first.

Objectives: Our objectives were to assess the current implementation of health technology assessment (HTA) in Hungary and to identify country-specific patterns of challenges and potential improvements.

Methods: We applied a structure that can be used to create HTA implementation roadmaps to evaluate various issues regarding HTA implementation. A comprehensive description of the Hungarian HTA system is presented according to relevant literature and experiences of the authors.

Results: By investigating eight components of HTA implementation, we identified the most important strengths and weaknesses of the Hungarian system. More specifically, we were mainly focusing on the emergence of HTA capacity, the establishment and current role of Department of HTA, the complex process of decision making, the quality elements developed in the near past, and the activity of Hungarian experts at international collaborations.

Conclusions: We concluded that there is a sophisticated methodological and educational basis for HTA in Hungary. A permanent focus on capacity building and changes to the reimbursement procedure can further improve transparency and the scientific basis of decision making in the country.

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.

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: Although psychiatric comorbidity is relevant for a number of diseases, it is often ignored in technology assessment. This study examines the service use rate in mental healthcare facilities and related costs for stroke patients discharged from the University Hospital Maastricht between 1987 and 1995.

Methods: Through anonymous record linkage, the medical registration of the hospital and the registration of the Maastricht Mental Health Case Register were linked.

Results: Linkage succeeded for 16% of the 2,020 stroke patients, indicating that these patients used mental health services during a 10-year period around the stroke (±5 years). Of the users' group, 88% had a mental healthcare contact following stroke. Regression analysis shows that age, length of hospital stay, and mental healthcare contact before stroke are associated with mental healthcare use after stroke. It is remarkable in that there is already an increase in the consumption of mental health care in the prodromal phase just before the stroke occurred. When comparing costs before and after stroke, the outpatient costs increased on average by \epsfbox{cj108-4-euro.eps}42.64, semi-institutionalized costs increased on average by \epsfbox{cj108-4-euro.eps}208.10, and intramural costs by \epsfbox{cj108-4-euro.eps}1,189.21. The total increase in costs is \epsfbox{cj108-4-euro.eps}1,439.95. For all mental healthcare facilities, the increase in costs is significant.

Conclusions: No study so far has revealed the total costs of mental healthcare facilities following stroke. Extrapolating these costs to the Netherlands illustrates that stroke patients have a high psychiatric comorbidity, inducing about 1.3% of total mental healthcare costs.

Conceptual issues arise in translating the results of technology assessment into policy. A 3 × 3 typology suggests that tough decisions occur when interventions promise increased benefit for increased cost or less benefit for less cost. These decisions are value based; technology assessment alone cannot determine who should receive an intervention and under what circumstances.

In the last several years, the development of an effective breast cancer screening procedure has increased the possibility of the early detection of this cancer. We investigated the cost-effectiveness of a breast cancer screening program to screen 100,000 women 50–64 years of age in Catalonia (Spain). The cost-effectiveness ratio was measured in terms of the cost per cancer detected comparing program costs to the estimated number of cancers detected. We assumed a participation rate, sensitivity, and specificity of 70%, 92%, and 94%, respectively, and that 0.36 breast cancers could be detected per 100 women screened. We estimated a total cost of $2.1 million with $1.4 million for the mammographic stage and $0.7 million for the detection of the true positive mammographic results. The cost per woman screened was $30. We estimated that 252 cases of breast cancer could be detected with the program. The cost-effectiveness ratio obtained in this study was $8,424 per cancer detected. Sensitivity analysis has shown that cost-effectiveness results are sensitive to the variations in the following variables: specificity, cancer detection rate, and screening costs. In planning preventive programs, breast cancer screening should be considered one of the priorities.

It is now established that the mucosal immune system is a separate entity and is regulated in a different fashion than that in peripheral lymphoid tissues (the systemic immune system). In this brief review, five selected areas within the field of mucosal immunity are discussed in the context of the goals for vaccines for the Children's Vaccine Initiative.

Patterns of diffusion were analyzed for three pairs of technologies: one of each pair represents a very recent innovation, and the other is more established. Tremendous growth in utilization from 1985–89 was documented, primarily due to more patients obtaining access to the technologies. Nevertheless, disturbing differences in levels of use remain between black and white patients.