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.

Introduction: Evidence requirements may differ across HTA bodies, and so pharmaceutical companies must plan to synergize their evidence generation strategy, across global regulatory and HTA bodies. Until recently, companies had no official platform to discuss the clinical development of a drug with HTA bodies; however, this is changing.

Objectives: To achieve broad usage in the EU, products must achieve both regulatory and reimbursement approval, the latter of which is based on HTA appraisal in many markets. The objective of this study is to present and evaluate the different options available for early HTA consultation (during drug development/Phase III) in the major European markets from the industry perspective.

Methods: An exploratory (nonsystematic) literature review was performed to identify the European markets offering early HTA consultations, and each process was analyzed using a set of predefined metrics that are relevant to industry (the ability to consult with the regulatory body in parallel, consultation fees, length of consultation meeting, language of consultation meeting, maximum number of pharmaceutical company employees attending, procedural timelines, nature of data for which consultative advice can be sought, the output of the process, and the ability to involve external experts).

Results: Four different types of early HTA consultation processes were identified across the major European HTA markets. The nature of these processes varied in terms of the types and number of questions that can be addressed, the length of the meeting, the reporting output, and the ability to involve external experts.

Conclusions: The availability of various options for early HTA consultation may help to avoid a mismatch between the evidence generated by means of a product's clinical development program, and the evidence expected by HTA bodies and payers, which can facilitate the pricing and reimbursement process upon a product's market authorization.

Sweden has a welfare system that is based on the fundamental principle that all citizens are entitled to good health and medical care, regardless of where they live or what their economic circumstances are. Health and medical care are considered to be public sector responsibilities. However, there is growing interest in establishing more private alternatives to public care. An important characteristic of the Swedish healthcare system is its decentralization, with a major role for county councils. County councils are now merging into larger administrative units (region). The whole Swedish system is in the process of reform, mainly because of perceptions that it was too rigid and had insufficient patient orientation. An important factor in the reforms is that power in the system will be even more decentralized and will have greater public input. This change is seen as calling for increased central follow-up and evaluation of matters such as social, ethical, and economic aspects. Although the state has decentralized control, it still attempts to control the general direction of the system through regulation, subsidy, recommendations, and guidelines. An important actor in the system is the Swedish Council on Technology Assessment in Health Care (SBU). SBU began in 1987 with assessments of health technologies, but its success has recently led policy makers to extend its coverage to dental care. Health technology assessment is increasingly visible to policy makers, who find it useful in decision making.

Objectives: To compare outcome attributes of three available technologies for stem cell transplantation—bone marrow transplantation (BM), peripheral blood cell transplantation (PB), and cord blood transplantation (CB). To compare the cost per recipient for the three technologies, incorporating all donor and antigen matching functions, as well as the transplantation procedures.

Methods: Review of relevant literature using the CancerLit and HealthSTAR databases (plus earlier searches of MEDLINE and Embase) to summarize outcome differences. Estimations of cost per transplant using models drawing on Canadian experience.

Results: The literature review indicated that PB may have a modest advantage over BM in terms of outcomes. The literature indicated that CB has longer engraftment times (although this may be due to inadequate controls). Our modeling exercise indicated that costs for PB and BM are similar. CB has a higher cost per recipient, due in large part to the higher testing costs for every donated unit.

Conclusions: Current literature is inadequate to provide an outcomes comparison of CB, PB, and BM. Viewed from a system-wide perspective, CB is a higher cost technique. However, PB and CB are emerging as technologies, and so our results should be viewed alongside the notion that there may be a steep learning curve in these technologies.

We conducted a retrospective cohort study based on a case note review to determine whether there are differences in the treatment pathways followed for men and women admitted with acute myocardial ischemia and infarction after adjusting for differences in case mix. Women were as likely as men to receive thrombolysis, but were less likely subsequently to undergo exercise testing (adjusted odds ratio, 0.58; 95% CI, 0.40–0.84) or angiography (adjusted odds ratio, 0.62; 95% CI, 0.39–0.99). Coronary anatomy was the strongest predictor of revascularization regardless of sex. Women with diagnosed cardiac pain are less likely than men to be placed on the investigative pathways that lead to revascularization. Those women who are investigated are as likely as men to undergo revascularization. These findings are independent of the effects of age, angina grade, comorbidity, or cardiac risk factors. Clinicians' and patients' beliefs and preferences about treatment require investigation.

The objectives of this paper are to describe the status and development of health technology regulation with regard to coverage decisions and utilization, and to analyze the current situation of health technology assessment (HTA) in Germany. The relevant literature for controlling health technologies was identified by searching the literature and databases and through personal contacts. The literature was analyzed with regard to the different sectors in the healthcare system. For the analysis of the current state of HTA in Germany, a national survey was carried out. In addition, the names of topics under assessment were collected. The results show that coverage decisions in the ambulatory sector appear to be much more regulated than those in the inpatient sector. The same is true for diffusion and usage of technologies. The strict separation of the hospital and the ambulatory care sector in Germany constitutes a barrier to regulation and to making HTA an effective instrument in Germany. Until recently, HTA in Germany focused on biotechnology, such as gene technology. Recently the German Scientific Working Group of Technology Assessment in Health Care has adopted a systematic methodology to undertake HTAs. It can be concluded that regulation of health technologies in Germany is characterized by inconsistencies ranging from strict regulation in the ambulatory sector to almost no regulation in the rehabilitation sector. Increasing interest in HTA, in conjunction with a high priority assigned to evaluation of health technologies by the newly elected government, is likely to improve this situation in the future.

Greater access to web-based information on health-care interventions might result in greater participation by patients in care and self-care decisions, but only improve health outcomes if the indicated actions produce the intended benefits. Unbiased research on benefits and harms of health information can provide a basis for evidence-based patient information systems.

Objectives: To evaluate the quality of the information content on bone-mineral density (BMD) testing posted on consumer health websites (CHWS).

Methods: Five popular engines (Yahoo, MSN, AOL, Lycos, and Go.com) were used to search for patient information on bone densitometry. The fifteen websites that supplied relevant content and were identified by three of the five search engines were selected in order of popularity of the search engine and primacy of placement. Six BMD reports from health technology assessment (HTA) organizations were used as a standard of scientific quality. These were identified from the HTA Database at York University United Kingdom and published between 1996 and 2001. Content was extracted from both document types, and these sets were compared independently by two reviewers.

Results: The majority of CHWS identified by popular search engines do not disclose the limited capacity of BMD to discriminate between low-risk individuals and those who will suffer future fractures. CHWS generally present BMD testing as quick, painless, noninvasive, and as being recommended, based on risk factors that are widespread among the general public. BMD testing information is prominently paired on CHWS sites with information on osteoporosis, with an emphasis on “silent disease” and the devastating consequences of advanced disease. Sponsors of CHWS sites are frequently either providers of BMD testing or companion drugs, and consequently in a position of conflict of interest with regard to decisions to undergo BMD testing. HTA organizations have no documented conflict of interest, nor do they invoke emotional arguments. Their approach is to emphasize the effects of testing on populations, on the basis of referenced research findings.

Conclusions: Content analysis demonstrates the omissions and divergence of information on BMD testing available to consumers on the Internet, as compared with HTA reports. The content of HTA reports has undergone rigorous systematic and peer review; therefore, their findings may be useful to consumers. This information is not generally accessible to patients using the most popular Internet search engines. Inaccurate and incomplete information may cause harm by deflecting patients from optimal decisions.

Objectives: The purpose of this study is to describe the situation with the distribution and utilization of prenatal diagnosis technology in China, to identify some important barriers to prenatal diagnosis use, and to suggest changes to improve the present situation.

Methods: The study uses cross-sectional surveys to capture quantitative data from both providers and consumers. Qualitative information based on focus group discussions is also presented.

Results: A mail survey of the provincial Bureaus of Health (BOHs) reveals that sixteen provincial prenatal diagnosis centers and twelve city level centers were accredited by the BOHs by July of 2001. These centers were located in thirteen provinces, of thirty in all of China. Of 147 selected institutions surveyed separately, 90.5 ercent offer ultrasound examination, 72.1 percent provide pathogen tests (mainly Toxoplasma, rubella virus, cytomegalovirus, and herpes simplex or TORCH), 57.1 percent do biochemical tests, 21.8 percent have genetic counseling, 13.6 percent do karyotype testing, 7.5 percent do enzymology testing, and 5.4 percent carry out molecular genetic testing. Chromosome diseases, congenital diseases, and several gene diseases are the target diseases. According to qualitative data, macromanagement for prenatal diagnosis, supplier provision of tests, and population demand are the main influences on prenatal diagnosis use.

Conclusions: From the quantitative and qualitative analysis, it is clear that the technology of prenatal diagnosis is not diffusing well throughout China and is apparently not appropriately used. The situation of prenatal diagnosis has implications for policy-makers, including identification of priorities, regulation of prenatal diagnosis, strategic planning, development of guidelines based on health technology assessment, and consumer orientation.

Since the 1990s, health policy makers in Finland have been supportive of evidence-based medicine and approaches to implement its results. The Finnish Office for Health Technology Assessment (Finohta) has grown from a small start in 1995 to a medium-sized health technology assessment (HTA) agency, with special responsibility in providing assessments to underpin national policies in screening. External evaluations enhanced the rapid growth. In the Finnish environment, decision making on health technologies is extremely decentralized, so Finohta has developed some practical tools for implementing HTA findings. The Managed Uptake of Medical Methods program links the hospital districts to agree on introduction of technologies. The Ohtanen database provides Finnish-language summaries of major assessments made in other countries.

Objectives: Peer-review publication is a critical step to the translation and dissemination of research results into clinical practice guidelines, health technology assessment (HTA) and payment policies, and clinical care. The objective of this study was to examine current views of journal editors regarding: (i) The value of real-world evidence (RWE) and how it compares with other types of studies; (ii) Education and/or resources journal editors provide to their peer reviewers or perceive as needed for authors, reviewers, and editors related to RWE.

Methods: Journal editors’ views on the value of RWE and editorial procedures for RWE manuscripts were obtained through telephone interviews, a survey, and in-person, roundtable discussion.

Results: In total, seventy-nine journals were approached, resulting in fifteen telephone interviews, seventeen survey responses and eight roundtable participants. RWE was considered valuable by all interviewed editors (n = 15). Characteristics of high-quality RWE manuscripts included: novelty/relevance, rigorous methodology, and alignment of data to research question. Editors experience challenges finding peer reviewers; however, these challenges persist across all study designs. Journals generally do not provide guidance, assistance, or training for reviewers, including for RWE studies. Health policy/health services research (HSR) editors were more likely than specialty or general medicine editors to participate in this study, potentially indicating that HSR researchers are more comfortable/interested in RWE.

Conclusions: Editors report favorable views of RWE studies provided studies examine important questions and are methodologically rigorous. Improving peer-review processes across all study designs, has the potential to improve the evidence base for decision making, including HTA.

Introduction: Hospital-based health technology assessment (HB-HTA) has been introduced to help hospital management in decision making about the adoption of new health technologies (HTs). We reviewed the accuracy of the expected medical impact of HTs assessed at our hospital, as well as the acceptance of this process by clinicians.

Methods: For each HT adopted between 2002 and 2011, a semi-structured interview with the involved clinician was conducted, assessing (i) the perceived utility of the HB-HTA process, (ii) the accuracy of the new HT's expected medical impact as compared with observed patient data from the year 2012, and (iii) the compliance with the indications of the HB-HTA report.

Results: Over the 10-year period, forty HB-HTAs were carried out, of which thirty-four led to acceptance. Twenty-seven of the twenty-eight clinicians involved in these thirty-four HTs accepted the interview and 85 percent acknowledged the utility of the HB-HTA process. Five of the thirty-four HTs were no longer in use. For the twenty-nine remaining HTs, observed patients’ number was as expected in eight, higher in four, lower in fifteen, and not available in two cases. Available average length of stay was 61 percent longer than expected. Two HTs had a higher complication rate and three a lower success rate. Indications evolved in 55 percent of HTs after a few years (seven restrictions, six broadenings, and three other changes).

Conclusions: A HB-HTA process is useful to improve quality in decision making. Follow-up analysis should routinely be performed to adapt HB-HTA reports’ conclusions to practical experience and new scientific evidence.

This article describes the characteristics of the health system and reviews the history of health technology assessment (HTA) in Iran, including its inception, processes, challenges, and lessons learned. This study was conducted by analyzing existing documents, reports, and guidelines related to HTA and published articles in the field. HTA in Iran has been established since the late 2000s and was first introduced as a secretariat by the Deputy of Health at the Ministry of Health and Medical Education. The mission of the HTA office is to systematically assess technologies to improve evidence-informed decision making. Despite its 10 years of existence, HTA in Iran still faces some challenges. The most pressing problems currently facing HTA in Iran include conflicts of interest among researchers performing the HTAs, the absence of a systematic structure for identifying and introducing new technologies, the lack of interest in HTA results among high-level policy makers, and the lack of external oversight for HTA projects.

Objectives: U.S. expenditures on medical devices ($70 billion in 2003) are one of the fastest growing components of hospital costs. Physicians’ selection of medical devices lacks an evidence base on the comparative clinical effectiveness of these products. Comparative studies (e.g., vendor 1 versus vendor 2, technology A versus technology B) are increasingly promoted in the public sector as a means of cost containment, value-based purchasing, and quality improvement. This study illustrates how hospitals and physicians can conduct comparative technology assessments of product performance.

Methods: Surgeons evaluated comparable medical devices manufactured by eight different vendors in standardized surgical procedures. Devices included sutures and endomechanical products, which account for $2.5 billion of total device spending. Evaluations covered multiple performance dimensions, including ergonomics, functionality, clinical acceptability, and vendor preference.

Results: One vendor's products garnered consistently high ratings from surgeons, while two other vendors garnered consistently low ratings. Differences in ratings were statistically significant and persist when controlling for physician background characteristics and prior experience. Study results were used by a large hospital group purchasing organization to select which vendors to contract with for these products.

Conclusions: Comparative technology evaluations assist physicians and hospitals in making cost-effective purchases of devices. These evaluations provide robust information on the performance of products routinely used by clinicians. Such evaluations can be carefully designed to have scientific rigor and clinical credibility.

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: 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.

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.

We performed a cost-effectiveness analysis to examine the relative efficacy and costs of percutaneous ultrasonic lithotripsy (PUL), extracorporeal shock-wave lithotripsy (ESWL), and surgery for the treatment of upper urinary tract stones. We developed a Markov model with 35 states, cycles of 3 months, and a time frame of 5 years. Probability estimates were derived from a meta-analysis of the published literature. For stones less than or equal to 2 cm, ESWL is preferred to PUL, since it prevents 2 additional days of morbidity and saves $440. For larger stones, PUL is preferable to ESWL, avoiding 4 more days of morbidity, and saving $722. Both ESWL and PUL were superior to surgery. Sensitivity analysis showed that the results are sensitive to ESWL efficacy rates, the stone recurrence rate, and the hospital component of the ESWL cost. Our analysis suggests that although ESWL is preferable, relatively small changes in the efficacy and cost can shift the preferred strategy; in addition, these findings underscore the need for more reliable data.