An overview of published studies on three common cancers in childhood was conducted to estimate the effectiveness and consequences of treatment. Of 1,016 separate citations, 316 were reviewed in detail but only 162 were deemed to be “useful.” An approach to a structured appraisal of the literature is presented.

Objectives: Examine the validity of using a self-completed Stated Preference Discrete Choice Modeling (SPDCM) questionnaire to measure parents' preferences for vaccinating their children against varicella.

Methods: A qualitative approach was used to assess the way parents understood the technical information in the questionnaire and the factors they considered to be important to the immunization decision. After completion of the SPDCM questionnaire, thirty-four participants completed a semistructured interview by telephone. Interview transcripts were analyzed by using content analysis. Comparisons were then made with the SPDCM questionnaire results.

Results: The technical information used to describe the program attributes appeared to be used appropriately by participants, although their explanations indicated that their understanding did not always come from the questionnaire information. Only one participant appeared to misunderstand the stated preference task, and a small number thought that the complexity and length should be reduced. The results of analysis of the questionnaire data were supported by the qualitative study, with the notable features of the model being reflected in the views commonly expressed about the immunization decision. Several additional factors were identified as important to the choice, including beliefs about vaccination generally and perceptions of the seriousness of varicella.

Conclusions: Although more research is required to investigate the validity of SPDCM for the measurement of preferences in health care, this study supports the validity of its use in childhood immunization where parents are familiar with the decision context.

By introducing discounting in the DEALE method, we reanalyze a recent publication on the cost-effectiveness of early thrombolytic therapy with intracoronary streptokinase after acute myocardial infarction. We show that failure to discount gains in life expectancy results in a serious overestimation of the cost-effectiveness of this technology.

We consider the nature of technology assessment and then briefly summarize technology assessment activities in five countries: Canada, France, the Netherlands, the United Kingdom, and the United States. Drawing from these examples, we then identify determinants of the emergence and impact of technology assessment.

Health technology assessment (HTA) aims, through empirical analysis, to shed light on the value of health technologies (O’Rourke et al. [2020, International Journal of Technology Assessment in Health Care 36, 187–90]). HTA is, then, where facts and values meet. But how, where, and when do facts and values meet in HTA? Currently, HTA is usually portrayed as a sequential process, starting with empirical analysis (assessment), followed by a deliberation on the implications of the findings for a judgment of a health technology’s value (appraisal). In this paper, we will argue that in HTA, empirical analysis and normative inquiry are much more closely entwined. In fact, as we hope to show, normative commitments act as an indispensable guide for the collection and interpretation of empirical evidence. Drawing on policy sciences, we will suggest a concrete methodology that can help HTA practitioners to integrate empirical analysis and normative inquiry in a transparent way. The proposed methodology can be conceived as a concrete means for conducting a scoping exercise in HTA. Moreover, it offers a distinct way of giving stakeholders a structural and constructive role in HTA. This paper outlines the approach developed by the values in doing assessments of health technologies project, a project funded by the Erasmus+ program (contract number 2018-1-NL01-KA203-038960), which is the European Union’s program to support education, training, youth, and sport in Europe. The project has resulted in an E-learning course, an accompanying handbook, and a consensus statement, all freely available from the project’s website www.validatehta.eu.

Background: In August 2002, an application for the listing on the Medicare Benefits Schedule (MBS) of PillCam® Capsule Endoscopy (formally M2A®) as a diagnostic procedure for obscure gastrointestinal bleeding (OGIB) was made to the Medical Services Advisory Committee (MSAC). As a result of this application, in May 2004 PillCam® Capsule Endoscopy was approved with interim funding until April 2007. This funding was conditional on the collection of Australian data on the long-term safety, effectiveness, and cost-effectiveness of capsule endoscopy.

Methods: A review was conducted of how the data were collected, the methodological difficulties associated with the collection and analysis of the data, and the outcomes of the data.

Results: The PillCam® Capsule Endoscopy Register ran from 2004 to 2007 and amassed data on 4,099 patients forming the largest database on PillCam® in the world. Based on these data, in November 2007, MSAC recommended that full public funding be supported under the current MBS Item Number 11820 as capsule endoscopy is as safe as and more effective than comparable diagnostic tests. It is the preferred choice of patients and has the potential to reduce the number and cost of previous investigations.

Conclusions: This form of CED proved to be ideally suited to PillCam® Capsule Endoscopy. The PillCam® Capsule Endoscopy Register provided data that made it possible to validate assumptions used in the economic modeling in the assessment carried out for MSAC in response to the application for funding.

Discussion: The use of interim funding requires both risk and cost sharing among the key players: industry, government, the medical profession, and the hospitals. Although the characteristics of PillCam® Capsule Endoscopy proved to be suited to data collection, this may not be the case with other emerging health technologies. If interim funding coupled with data collection is to become an effective mechanism for bridging the evidence gap, work needs to be carried out by health technology assessment agencies to provide guidance on the design of registers so that they cater for the unique characteristics of individual procedures.

Evaluating the use of resources as well as outcomes in cancer therapy is increasingly becoming recognized by both clinicians and others as a legitimate and indeed even desirable activity. While this trend is to be welcomed if it facilitates the efficient use of resources for cancer care, there are dangers in applying estimates of unit costs, in particular average costs to the evaluation of trends in practice in cancer therapies. This article examines the use of appropriate measures of average and marginal cost in the economic evaluation of developments in cancer therapy, taking illustrations from radiotherapy and chemotherapy.

The treatment of prostate cancer was reviewed at a U.S. National Institutes of Health Consensus Development Conference in June 1987. Data from the U.S. National Cancer Institute's Surveillance, Epidemiology, and End Results tumor registries were analyzed and showed that the proportion of eligible prostate cancer patients receiving the recommended therapies did not increase at a faster rate after the conference than before.

Objectives: We compared Polish and Scottish Health Technology Assessment (HTA) process in order to elicit recommendations for future development of HTA methodological guidelines in Poland.

Methods: We studied the differences between Polish and Scottish HTA methodological guidelines. HTA recommendations issued by Polish HTA agency (AHTAPol) in the period January 1 through December 31, 2008, were benchmarked to HTA guidance published by Scottish Medical Consortium (SMC) for the same drug technology.

Results: The Scottish HTA methodological guidelines were more instructive in terms of clinical and economic evaluations than Polish guidelines. SMC evaluated forty-eight of sixty-eight drug technologies appraised by AHTAPoL. There were thirty drug technologies that received similar guidance in both countries and eighteen with contradictory HTA recommendations. In Scotland, there were more positive HTA recommendations than there were in Poland. While comments about efficacy or safety were commonplace among reasons for negative recommendations in Poland, insufficient justification of treatment's cost in relation to benefits was the most often cited reason for rejection in Scotland. SMC tended to recommend restricted use to specific sub-populations for several drug technologies negatively appraised by AHTAPoL.

Conclusions: The comparison between SMC and AHTAPoL suggests that there is potential room of improvement of the Polish HTA methodological guidelines. Comparative effectiveness and safety, subgroup analysis, and adaptation of models to local settings were identified as key areas for further development of Polish HTA methodological guidelines.

Objectives: The objective of this study was to explore barriers and facilitators influencing the integration of ethical considerations in health technology assessment (HTA).

Methods: The study consisted of two complementary approaches: (a) a systematic review of the literature; and (b) an eighteen-item online survey that was distributed to fifty-six HTA agencies affiliated with the International Network of Agencies for Health Technology Assessment.

Results: The review identified twenty-six relevant articles. The most often cited barriers in the literature were: scarcity, heterogeneity and complexity of ethical analysis methods; challenges in translating ethical analysis results into knowledge that is useful for decision makers; and lack of organizational support in terms of required expertise, time and financial resources. The most frequently cited facilitators included: usage of value-based appraisal methods, stakeholder and public engagement, enhancement of practice guidelines, ethical expertise, and educational interventions.

Representatives of twenty-six (46.5 percent) agencies from nineteen countries completed the survey. A median of 10 percent (interquartile range, 5 percent to 50 percent) of the HTA products produced by the agencies was reported to include an assessment of ethical aspects. The most commonly perceived barriers were: limited ethical knowledge and expertise, insufficient time and resources, and difficulties in finding ethical evidence or using ethical guidelines. Educational interventions, demand by policy makers, and involvement of ethicists in HTA were the most commonly perceived facilitators.

Conclusions: Our results emphasize the importance of simplification of ethics methodology and development of good practice guidelines in HTA, as well as capacity building for engaging HTA practitioners in ethical analyses.

Objectives: The aim of this study is to analyze the quality and transferability issues reported in published peer-reviewed English-language economic evaluations based in healthcare settings of the Central and Eastern European (CEE) and former Soviet countries.

Methods: A systematic search of economic evaluations of healthcare interventions was performed for Armenia, Azerbaijan, Belarus, Bulgaria, Estonia, Georgia, Turkmenistan, Kazakhstan, Lithuania, the former Yugoslav Republic of Macedonia, Republic of Moldova, Romania, the Russian Federation, Serbia, Slovenia, and Ukraine. The included studies were assessed according to their characteristics, quality (using Drummond's checklist), use of local data, and the transferability of inputs and results, if addressed.

Results: Most of the thirty-four economic evaluations identified were conducted from a healthcare or payer perspective (74 percent), with 47 percent of studies focusing on infectious diseases. The least frequently and transparently addressed parameters were the items’ stated perspectives, relevant costs included, accurately measured costs in appropriate units, outcomes and costs credibly valued, and uncertainties addressed. Local data were often used to assess unit costs, baseline risk, and resource usage, while jurisdiction-specific utilities were included in only one study. Only 32 percent of relevant studies discussed the limitations of using foreign data, and 36 percent of studies discussed the transferability of their own study results to other jurisdictions.

Conclusions: Transferability of the results is not sufficiently discussed in published economic evaluations. To simplify the transferability of studies to other jurisdictions, the following should be comprehensively addressed: uncertainty, impact of influential parameters, and data transferability. The transparency of reporting should be improved.

Objectives: To describe the first hospital-based health technology assessment (HTA) program in a public hospital in Argentina, and report some clinical, educational, economic and organizational results after 10 years of its implementation.

Methods: A hospital-based HTA program was created in March 2001 at Hospital Garrahan (Buenos Aires, Argentina), a national pediatric facility with a self-managed budget. Its main goal is to promote a rational and evidence-based technologic development. The program consists of HTA reports for technology acquisition, clinical practice guidelines (CPG), capacity building in research and management, and technical support for health services research (HSR). The evaluation cycle comprises: prioritization, evidence synthesis, dissemination and monitoring. We report program performance, comment educational and organizational effects, and discuss unresolved issues and future challenges.

Results: During the first 10 years the program produced 18 HTA reports on drugs (6 = 33 percent), therapeutic (6 = 33 percent), preventive (2 = 11 percent) or diagnostic (2 = 11 percent) procedures and institutional programs (3 = 17 percent). The scope covered effectiveness (12 = 67 percent), safety (10 = 56 percent), budget impact (6 = 33 percent), cost-effectiveness (2 = 11 percent) and organizational impact (3 = 17 percent). Mean time from request to report was 10 months. Eleven pediatric CPGs were submitted to expert consensus and disseminated for full-text Web access. A 1-year course on research and management was completed by 225 professionals in 6 years, and twenty-two projects for HSR were coached.

Conclusions: Our experience shows that an HTA program is both feasible and useful in a public hospital of a developing country. Promotion of hospital-based HTA, professional integration in HTA activities and network collaboration to discuss unresolved issues with colleagues can multiply the benefits and optimize the use of hospital budgets.

The importance of health technology assessment for developing countries is unquestionable. Although these countries are still plagues by tropical diseases and primary health problems, they have been forced to import modern medical technologies because of internal and international pressures. Recognition of the potentoial of health technology assessment for improving the management of technology and health care delivery in developing countries leads to the critical question of how to promote the development of assessment activities there. But as technology assessment is itself also a technology, it can be assuned that its direct transfer to developing countries in the form that it has unfolded in the richer nations is doomed to failure. How successful transfer of health technology assessment can be achieved is outlined.

Objective: To analyze the rates and influences on the adoption of three selected health technologies: statins, coronary stents, and magnetic resonance imaging (MRI).Methods:A retrospective diffusion study using primary care prescribing data and questionnaire responses from acute hospital trusts in the West Midlands region (population 5.3 million or 10% of England).

Results: The selected technologies had markedly different diffusion curves. Statins diffused rapidly soon after launch. Coronary stents were initially used 6 years after first availability, but within 2 years all responding hospitals reported using them. MRI scanners were initially purchased 6 years after first availability with a subsequently slow rate of diffusion, and are still absent from some hospitals. Influences on the adoption of each technology were different. Commercial marketing was reported as a major influence on the diffusion of statins but not at all on MRIs. Cost impact was a major negative influence on the diffusion of MRI scanners and statins, whereas enthusiastic individuals were key to the diffusion of stents.

Conclusions: Influences on adoption and consequent diffusion rates are very different for different health technologies. It is not at all clear that such diffusion patterns relate well to an optimum timing rate. This has important implications for technology gatekeepers in health care.

Decision making in health care has become increasingly reliant on information technology, evidence-based processes, and performance measurement. It is therefore a time at which it is of critical importance to make data and analyses more relevant to decision makers. Those who support Bayesian approaches contend that their analyses provide more relevant information for decision making than do classical or “frequentist” methods, and that a paradigm shift to the former is long overdue. While formal Bayesian analyses may eventually play an important role in decision making, there are several obstacles to overcome if these methods are to gain acceptance in an environment dominated by frequentist approaches. Supporters of Bayesian statistics must find more accommodating approaches to making their case, especially in finding ways to make these methods more transparent and accessible. Moreover, they must better understand the decision-making environment they hope to influence. This paper discusses these issues and provides some suggestions for overcoming some of these barriers to greater acceptance.

Objectives: The aim of this study was to present the experience of a Canadian hospital-based health technology assessment (HTA) unit that performed the traditional functions of the HTA process along with many other activities to facilitate the choice of smart pumps.

Methods: A rapid literature review was initiated, but little evidence was found. Moreover, the evidence provided was too far from our hospital context. To help our decision makers, we offered them a list of various services based on the skills of our HTA unit staff.

Results: To involve our HTA unit in the choice of the new smart pumps led to a strong collaboration between hospital services. After a rapid review on smart pumps, we proceeded to establish the clinical needs, followed by an evaluation of technical features. To ascertain clinical needs, we participated in the establishment of a conformity list for the tender, a failure and mode-effect analysis, an audit on the use of actual smart pumps, and simulation exercises with nurses and doctors to evaluate the ease of use and ergonomics. With regard to technical tests, these were mainly conducted to identify potential dysfunction and to assess the efficiency of the pump. This experience with smart pumps was useful for evidence-based procurement and led to the formulation of a nine-step process to guide future work.

Conclusions: HTA units and agencies are faced with rapid development of new technologies that may not be supported by sufficient amount of pertinent published evidence. Under these circumstances, approaches other than evidence-based selection might provide useful information. Because these activities may be different from those related to classic HTA, this widens the scope of what can be done in HTA to support decision making.

Objectives: The aim of this study was to assess systematically the scope of evidence and purposes for which evidence is used in decision-analytic models of cost-effectiveness and to assess the implications for search methods.

Methods: A content analysis of published reports of models was undertaken. Details of cited sources were extracted and categorized according to three dimensions; type of information provided by the evidence, type of source from which the evidence was drawn and type of modeling activity supported by the evidence. The analysis was used to generate a classification of evidence. Relationships within and between the categories within the classification were sought and the implications for searching considered.

Results: The classification generated fourteen types of information, seven types of sources of evidence and five modeling activities supported by evidence. A broad range of evidence was identified drawn from a diverse range of sources including both research-based and non–research-based sources. The use of evidence was not restricted to the population of model parameters but was used to inform the development of the modeling framework and to justify the analytical and methodological approach.

Conclusions: Decision-analytic models use evidence to support all aspects of model development. The classification of evidence defines in depth the role of evidence in modeling. It can be used to inform the systematic identification of evidence.

A literature review compared the results of different kidney function tests using the inulin clearance or similar methods as a gold standard. The methodological strength of the available studies was weak. For the time being, the calculated creatinine clearance using formula, is a first choice, especially in general practice.