Background: Two general alternative approaches, cost-effectiveness analysis and the therapeutic added value approach, link the pricing and approval of drugs to value. Value as assessed by payers is a function of: benefit less cost, willingness to pay for benefit, and how they handle uncertainty.

Methods: This study uses international examples to explore the elements of value that can be included in the assessment of health technologies, approaches to scoring the elements of value and how they can be combined to make a decision.

Results: A range of value elements, measures, and approaches to aggregation are identified across different HTA systems. We show that seemingly arbitrary differences in measurement and aggregation can lead to significantly different outcomes, and argue that the choice of values, measures, and decision-making processes should be informed by the societal values that underpin a health system.

Conclusions: We identify three areas for further research to improve both health system and industry R&D decision making: (i) whether more consistency could be achieved across health systems on the elements of value that matter; (ii) the relative merits of discrete versus continuous measures of value; and (iii) how structured decision making (to aggregate the elements of value) could or should become.

Total treatment costs by stage at diagnosis are estimated for a sample of breast cancer patients. At 4 years, stage 4 cancers emerge as being more expensive to treat than those at earlier stages, although this difference fails to achieve significance when expected lifetime costs are considered. The inclusion of treatment cost estimates in a screening model indicates that screening may increase expected treatment costs by a marginal amount, although the model also suggests that the cost effectiveness ratio of breast cancer screening might be better than had originally been thought.

Objectives: The aim of the study is to make an international comparison of Health Technology Assessment (HTA) Agencies, to show their similarities and differences.

Methods: An e-mail questionnaire was sent to thirty HTA agencies internationally. Questions related to the structure of the agency, the relationship with health-related institutions, the prescriptiveness of the decisions taken, the main core and the modalities to spread the assessment, and the type of funding.

Results: Twenty-four HTA Agencies answered the questionnaire: 25 percent in America, 4.2 percent in Australia, and 70.8 percent in Europe. Fifty-four percent of HTA Agencies are governmental institutions (83.3 percent have central government funding), while 62.5 percent have relationships with health-related governmental institutions. Of the agencies, 87 percent reported that their decisions are not prescriptive, while for 20.8 percent and 8.3 percent of them stated that this was the case totally or partially, respectively, especially for the governmental and American Agencies. Seventeen agencies (70.8 percent) declared their work on multiannual programs (77 percent of the governmental HTA Agencies and 100 percent of the American ones). The assessments mainly addressed diagnostic procedures (85.7 percent) and pharmaceuticals (25 percent). The most common way to disseminate results is by means of paper report (91.7 percent), followed by the Internet (16.7 percent), and seminars to expert audiences (12.5 percent).

Conclusions: The comparative analysis of HTA Agencies showed that governmental and American Agencies have a profound impact on the prescriptiveness of their assessment, and this could be linked to the fact that these types of Agencies work on multiannual programs. European and American HTA Agencies have many similarities in terms of type of assessment, funding, and dissemination of results.

Background: Information retrieval (IR) in health technology assessment (HTA) calls for transparency and reproducibility, but common practice in the documentation and presentation of this process is inadequate in fulfilling this demand.

Objectives: Our objective is to promote good IR practice by presenting the conceptualization of retrieval and transcription readable to non-information specialists, and reporting of effectively processed search strategies.

Methods: We performed a comprehensive database search (04/2010) to synthesize the current state-of-the-art. We then developed graphical and tabular presentation methods and tested their feasibility on existing research questions and defined recommendations.

Results: No generally accepted standard of reporting of IR in HTA exists. We, therefore, developed templates for presenting the retrieval conceptualization, database selection, and additional hand-searching as well as for presenting search histories of complex and lengthy search strategies. No single template fits all conceptualizations, but some can be applied to most processes. Database interface providers report queries as entered, not as they are actually processed. In PubMed®, the huge difference between entered and processed query is shown in “Details.” Quality control and evaluation of search strategies using a validated tool such as the PRESS checklist is suboptimal when only entry-query based search histories are applied.

Conclusions: Moving toward an internationally accepted IR reporting standard calls for advances in common reporting practices. Comprehensive, process-based reporting and presentation would make IR more understandable to others than information specialists and facilitate quality control.

The principle that infants can be protected from invasive diseases caused by encapsulated organisms has been proved with the introduction of Haemophilus influenzae type b conjugate vaccines. The use of glycoconjugates to implement some of the goals of the Children's Vaccine Initiative requires a clear delineation of the chemical and immunological specifications for optimal vaccines.

The utility weights that are used in published scientific articles for assessing benefits in terms of quality adjusted life-years gained often have a weak theoretical and empirical basis. To a large extent, the weights seem to be used without critical thought and sensible discussion. In a majority of cases, the sensitivity of the results to the weights that have been chosen is not demonstrated, and readers are not provided with information in a way that facilitates independent calculations. This paper calls for a standard for future publications.

In light of growing demands for public accountability, the broadening scope of health technology assessment organizations (HTAOs) activities and their increasing role in decision-making underscore the importance for them to demonstrate their performance. Based on Parson's social action theory, we propose a conceptual model that includes four functions an organization needs to balance to perform well: (i) goal attainment, (ii) production, (iii) adaptation to the environment, and (iv) culture and values maintenance. From a review of the HTA literature, we identify specific dimensions pertaining to the four functions and show how they relate to performance. We compare our model with evaluations reported in the scientific and gray literature to confirm its capacity to accommodate various evaluation designs, contexts of evaluation, and organizational models and perspectives. Our findings reveal the dimensions of performance most often assessed and other important ones that, hitherto, remain unexplored. The model provides a flexible and theoretically grounded tool to assess the performance of HTAOs.

Health technology assessment (HTA) seeks to inform health policy makers by using the best scientific evidence on the medical, social, economic, and ethical implications of investments in health care. Technology is broadly defined to include the drugs, devices, medical, and surgical procedures used in health care, as well as measures for prevention and rehabilitation of disease, and the organizational and support systems in which health care is provided.

The issue of evidence for decision-making, including formal policy-making and public health policy, has increased rapidly in prominence during the past few years. The term “evidence-based” was first used in health care in 1992, when Sackett and others coined the term “evidence-based medicine” (42). More recently, many have asked if public health activities are based on evidence (2;8;13;19;30).

Objectives: The cost-effectiveness of adding a human papillomavirus (HPV) vaccination program in 12-year-old females to the recommended cervical cancer screening in Belgium is examined. Moreover, the health and economic consequences of a potential decline in screening uptake after initiation of a HPV vaccination program are investigated.

Methods: A static Markov model is developed to estimate the direct effect of vaccination on precancerous lesions and cervical cancers.

Results: Vaccination is estimated to avoid 20 percent of the cervical cancers occurring in a 12-year-old girls' cohort and to cost €32,665 per quality-adjusted life-year (QALY) gained (95 percent credibility interval [CrI]: €17,447 to €68,078), assuming a booster injection after 10 years, a limited duration of protection and discounting costs and effects at 3 percent and 1.5 percent, respectively. Assuming lifelong protection, HPV vaccination is estimated to cost €14,382 (95 percent CrI: €9,238 to €25,644) per QALY gained, while avoiding 50 percent of the cervical cancer cases. In the base-case, a 10 percent reduction in screening compliance after vaccination obliterates the effect of vaccination on cervical cancer cases avoided, whereas further declines in the level of screening compliance even turned out to be detrimental for the cohort's health, inducing a mean loss in QALYs and life-year gained compared with the situation prevaccination.

Conclusions: An HPV vaccination program should only be considered if the level of screening after vaccination can be maintained.

Objectives: Evaluating the impact of recommendations based upon health technology assessment (HTA) represents a challenge for both HTA agencies and healthcare policy makers. This research sought to understand factors affecting the uptake of HTA recommendations to support decision making with respect to the introduction of three health technologies.

Methods: Using a multidimensional framework, based upon a combination of theoretical models, a case study was conducted. A total of twenty-eight semistructured interviews were done with physicians from fifteen hospitals and other stakeholders in Catalonia. Interview content was analyzed iteratively and classified according to theoretical dimensions and contextual factors.

Results: At the sociopolitical level, factors related to the organization and financing of the health system were found to affect the utilization of HTA recommendations. At the healthcare organization level, existing collaborations between the hospital and the HTA agency favored the integration of recommendations into practices. Formalism in the organization also influenced the utilization of HTA recommendations. At the professional level, the high degree of autonomy of specialists, the importance of peers and collegial control, and the definition of professional roles and responsibilities influenced physicians' willingness to integrate HTA recommendations into their practice.

Conclusions: This study offers a comprehensive framework to understand the complex dynamics that affect adoption of health technologies in organizational and professional practices. The findings suggest some avenues to promote the integration of HTA recommendations into practices and, thus, increase the utilization of scientific evidence to support decision making in health care.

This paper investigates the theoretical properties of healthy-years equivalents (HYEs) and quality-adjusted life-years (QALYs). A distinction is made between ex ante HYEs (EA-HYEs) and expected HYEs (EXP-HYEs) and between risk-neutral quality-adjusted life-years (RN-QALYs) and risk-adjusted quality adjusted life-years (RA-QALYs). In the case of certainty, HYEs always rank health profiles according to individual preferences, whereas QALYs only rank health profiles according to individual preferences if constant proportional trade-off holds for all health states and if additive independence of quality in different periods holds. In the case of uncertainty, EA-HYEs always rank risky health profiles the same way as expected utility. The assumptions needed for the other measures to rank risky health profiles the same way as expected utility are: risk neutrality with respect to healthy time for EXP-HYEs; risk neutrality with respect to time in all health states and additive independence of quality in different periods for RN-HYEs; and constant proportional risk posture with respect to time in all health states and additive independence of quality in different periods for RA-QALYs.

Background: In 1994/95, two genes, BRCA1/2, associated with a predisposition to breast or ovarian cancer were identified. Genetic testing of deleterious BRCA1/2 mutations consequently can be proposed to individuals with a family history of breast or ovarian cancer to identify who is at risk. The granting of U.S. patents on BRCA1/2 to a privately owned company has led to the monopoly use of a unique technique (Direct Sequencing of the gene, DS) for BRCA1/2 testing in this country. Alternative strategies using prescreening techniques, however, have been experienced worldwide.

Methods: On the basis of data collected at three laboratories of French public hospitals, we carried out a cost-effectiveness study comparing DS to 19 alternative strategies with the number of deleterious BRCA1 mutations detected as the outcome.

Results: Results show that the DS strategy presents the highest average cost per mutation detected (9,882.5 °) and that there exist strategies using prescreening techniques that can reach similar effectiveness while reducing total costs. Moreover, other strategies can obtain a four- to sevenfold reduction in the average cost per mutation detected as soon as some rates of false negatives (2% to 13%) are deemed to be acceptable.

Conclusions: Results suggest that gene patents with a very broad scope, covering all potential medical applications, may prevent health care systems from identifying and adopting the most efficient genetic testing strategies due to the monopoly granted for the exploitation of the gene. Policy implications for regulatory authorities, in the current context of the extension of BRCA1/2 patents in other countries, are discussed.

Four studies, each on health technology assessment (HTA) in a different country, are presented in this volume. Conveying differing levels of sensitivity to political aspects of HTA, their storylines are similar in terms of the importance of the institutional structures that produce HTA and mediate its influence on health policy decision making. Regarding the internal politics of HTA, the latter appears to have developed in a relatively depoliticized environment, supported by a dense and varied web of institutional sites for funding, production, and consumption of HTA, buffered from the capricious impacts of electoral politics. Regarding external politics, HTA in all the countries began with relatively politically innocuous studies of technologies recognized to be of major import to national health systems or researcher-initiated studies. However, with increased focus in health systems on explicit determination of health benefits baskets, the role of HTA has become more high profile. This means that political accountability for the entire HTA process will increase. The implication is that future management of HTA programs will require self-conscious attention to the building of institutions capable of handling the delicate process of integrating science and politics in health policy.

Objective: There is increasing empirical evidence for the existence of bias in the publication of primary clinical research, with statistically significant results being published more readily, more quickly, and in higher impact journals. Meta-analysis of individual patient data (IPD) may represent a gold standard of “secondary” clinical research, giving the best possible summary of current evidence for a particular question, but publication of these may also be subject to bias. This study aimed to explore which factors might be associated with publication of IPD meta-analyses and to identify potential sources of bias.

Methods: For all known IPD meta-analysis projects in cancer, the responsible investigator was surveyed by means of a questionnaire to determine descriptive characteristics of the meta-analysis, the nature of the results, and details of the publication history.

Results: There is no good evidence that overall publication status of meta-analyses in cancer is dependent on the statistical or clinical significance of the results. However, those meta-analyses with nonsignificant results did seem to take longer to publish and were published in lower impact journals compared with those with more striking results.

Conclusions: Based on the current data, there seems to be no strong association between the results of IPD meta-analyses in cancer and publication.

Objectives: Pediatric cardiology has an expanding role in fetal and pediatric screening. The aims of this study were to observe how district hospitals use a pediatric telecardiology service, and to compare the costs and outcomes of patients referred to specialists by means of this service or conventionally.

Methods: A telemedicine service was set up between a pediatric cardiac center in London and four district hospitals for referrals of second trimester women, newborn babies, and older children. Clinicians in each hospital decided on the role for their service. Clinical events were audited prospectively and costed, and patient surveys were conducted.

Results: The hospitals differed in their selection of patient groups for the service. In all, 117 telemedicine patients were compared with 387 patients seen in London or in outreach clinics. Patients selected for telemedicine were generally healthier. For all patients, the mean cost for the initial consultation was £411 for tele-referrals and £277 for conventional referrals, a nonsignificant difference. Teleconsultations for women and children were significantly more expensive because of technology costs, whereas for babies, ambulance transfers were much more costly. After 6-months follow-up, the difference between referral methods for all patients was nonsignificant (telemedicine, £3,350; conventional referrals, £2,172), and nonsignificant within the patient groups.

Conclusions: Telemedicine was perceived by cardiologists, district clinicians, and families as reliable and efficient. The equivocal 6-month cost results indicate that investment in the technology is warranted to enhance pediatric and perinatal cardiology services.

Objectives: The objective of this study is to profile the health technology assessments (HTAs) produced in Canada and other selected countries and assess their potential to inform policy making about health systems in jurisdictions other than the ones for which they were produced, and to develop and pilot test prototypes for packaging and assessing the relevance of HTAs for health system managers and policy makers.

Methods: We compiled an inventory of all HTAs that were produced by nine HTA agencies between September 2003 and August 2006; coded the title and abstract of each HTA according to the technologies assessed, methods used, and whether or not context-specific actionable messages were provided; developed a prototype for a structured, decision-relevant HTA summary and for a relevance-assessment form; and pilot-tested the prototypes using semistructured telephone interviews with a purposive sample of Canadian healthcare managers and policy makers.

Results: Our review of the 223 HTAs identified that: (i) 44 HTAs addressed health system arrangements (20 percent); (ii) 205 incorporated a systematic review (92 percent), whereas only 12 incorporated a sociopolitical assessment using explicit methods (5 percent); and (iii) 50 contained context-specific actionable messages (22 percent). Our interviews identified significant support for both the general idea of an HTA summary and the prototype's specific elements, but mixed views about using peer assessments of relevance.

Conclusions: Those involved in supporting the use of HTAs in policy making about health systems may wish to produce structured decision-relevant summaries for their systematic review-containing HTAs to increase the prospects for their HTAs being used outside the jurisdiction for which they were produced.

Objectives: The aim of this study was to assess whether a multicomponent cognitive behavioral group intervention is preferable to usual care in terms of (healthcare) costs and effects on fear of falling and activity avoidance.

Methods: This economic evaluation was embedded in a randomized controlled trial among 540 community-living adults in the Netherlands, aged 70 years and older who reported fear of falling and fear-induced activity avoidance. The participants allocated to the intervention group received a multicomponent cognitive behavioral group intervention consisting of eight weekly sessions and a booster session. The sessions were aimed at instilling realistic views of falls, reducing fall risk, and increasing activity and safe behavior. Participants in the control group received usual care. Cost outcome measures were healthcare costs, and patient and family costs. Clinical outcomes were fear of falling and activity avoidance. All outcomes were assessed at baseline and at 2, 8, and 14 months by means of registration forms, self-administered questionnaires, and interviews by telephone.

Results: Participants were randomly allocated to intervention (n = 280) and control groups (n = 260). Costs for the intervention program were on average €276 per person. Total costs per person were comparable (€4,925 in intervention group and €4,828 in control group). Furthermore, favorable effects of the program were observed for fear of falling and activity avoidance.

Conclusions: This study showed that the intervention program is preferable to usual care in terms of costs and effects. The program had comparable costs and significantly reduced fear of falling and associated activity avoidance among older community-living persons.

Objectives: Depression is common in diabetes and linked to a wide range of adverse outcomes. UK policy indicates that depression should be treated using conventional psychological treatments in a stepped care framework. This review aimed to identify current economic evidence of psychological treatments for depression among people with diabetes.

Method: Electronic search strategies (conducted in MEDLINE, EMBASE, PsycINFO, CINAHL, NHS EED) combined clinical and economic search terms to identify full economic evaluations of the relevant interventions. Prespecified screening and inclusion criteria were used. Standardized data extraction and critical appraisal were conducted and the results summarized qualitatively.

Results: Excluding duplicates, 1,516 studies for co-morbid depression and diabetes were screened. Four economic evaluations were identified. The studies found that the interventions improved health status, reduced depression and were cost-effective compared with usual care. The studies were all U.S.-based and evaluated collaborative care programs that included psychological therapies. Critical appraisal indicated limitations with the study designs, analysis and results for all studies.

Conclusions: The review highlighted the paucity of evidence in this area. The four studies indicated the potential of interventions to reduce depression and be cost-effective compared with usual care. Two studies reported costs per QALY gained of USD 267 to USD 4,317, whilst two studies reported the intervention dominated usual care, with net savings of USD 440 to USD 612 and net gains in patient free days or QALYs.

This pilot study is the first known attempt to test the willingness-to-pay or willingness-to-accept (WTP/WTA) method of assessment on a community-based primary prevention program against cardiovas cular disease. The results indicate that the population's perceived WTA outweighs the program's costs by at least three to one. However, the methodological problems of using the WTP/WTA method in community-based programs are far from solved, and further research in this area is required.