Background: Understanding cost-drivers and estimating societal costs are important challenges for economic evaluation of health technologies in low- and middle-income countries (LMICs). This study assessed community experiences of health resource usage and perceived cost-drivers from a societal perspective to inform the design of an economic model for the Community Level Interventions for Pre-eclampsia (CLIP) trials.

Methods: Qualitative research was undertaken alongside the CLIP trial in two districts of Sindh province, Pakistan. Nine focus groups were conducted with a wide range of stakeholders, including pregnant women, mothers-in-law, husbands, fathers-in-law, healthcare providers at community and health facility-levels, and health decision/policy makers at district-level. The societal perspective included out-of-pocket (OOP), health system, and program implementation costs related to CLIP. Thematic analysis was performed using NVivo software.

Results: Most pregnant women and male decision makers reported a large burden of OOP costs for in- and out-patient care, informal care from traditional healers, self-medication, childbirth, newborn care, transport to health facility, and missed wages by caretakers. Many healthcare providers identified health system costs associated with human resources for hypertension risk assessment, transport, and communication about patient referrals. Health decision/policy makers recognized program implementation costs (such as the mobile health infrastructure, staff training, and monitoring/supervision) as major investments for the health system.

Conclusions: Our investigation of care-seeking practices revealed financial implications for families of pregnant women, and program implementation costs for the health system. The societal perspective provided comprehensive knowledge of cost drivers to guide an economic appraisal of the CLIP trial in Sindh, Pakistan.

A new definition of health technology assessment (HTA), developed by an International Joint Task Group claims to be a “milestone,” “an historic achievement,” and “a cornerstone reference”—claims that we think to be unjustified. We too favor clear definitions, especially when confusion abounds. However, the Task Group seems to have developed a definition without the help of usual conventions regarding definitions and, in our view, through an ill-described process. A definition ought to differentiate the entity defined from other entities. This one fails to do so. It states traits that are true of HTA (e.g., that is interdisciplinary) but HTA is not alone in this. There are other concerns: examples of HTA’s use are embodied in the definition, precluding other uses; the adjectives used, although generally true of HTA, are not differentiating features; and attributing to HTA specific purposes, thereby excluding other purposes. We have sympathy for these purposes but cannot consider them HTA’s only purposes or even, its main purpose. A newcomer to HTA, on reading this definition, will have no idea of HTA’s true potential. These numerous failings, we feel, send all the wrong signals, and could ultimately weaken, rather than strengthen perceptions of HTA’s legitimacy and objectivity. The production of a good definition remains, therefore, a work in progress.

Objective: In this article, three preventive strategies—mammography screening for breast cancer, PSA screening for prostate cancer, and routine ultrasound in normal pregnancy—are discussed in the context of German health care.

Methods: Epidemiologic data and German studies evaluating different aspects of these preventive measures were identified and analyzed.

Results: Only a few studies could be identified that investigate these preventive measures. Despite sufficient evidence, in part derived from a German study, there is not yet a mammography screening program. In contrast, ultrasound in pregnancy is offered routinely, although there are controversies regarding the benefit of this practice. PSA screening is not offered as part of the screening program for prostate cancer. However, PSA tests as well as mammographies are done in large numbers in German ambulatory care—a practice that could be considered wild or opportunistic screening.

Conclusions: These case studies show that preventive programs and practices in Germany are not sufficiently based on sound evidence. The paucity of evaluation activities related to prevention in Germany is probably due to the low threshold to introduce new preventive programs into the German healthcare system in the past.

Objectives: The objectives of the study were to identify the current standards of clinical practice regarding prostate cancer screening in western Europe, Canada, and the United States, and to highlight major characteristics of current prostate cancer screening programs or patterns of practice.

Methods: We performed a semi-structured interview by means of a self-administered questionnaire sent by fax to 26 institutes pertaining to the International Network of Agencies for Health Technology Assessment.

Results: None of the countries surveyed had a formal national screening policy. Despite that, all the countries answering the questionnaire had discretionary, public-financed screening practices. Moreover, some scientific and professional organizations recommended population screening for prostate cancer, and few of the surveyed countries offered it as experimental practice within a randomized controlled trial. Survey results showed variation regarding screening policies, in particular test of choice, age cut-off points, and treatment prescribed for positive test results.

Conclusions: Despite the lack of conclusive evidence on the benefits of prostate cancer screening, the availability of simple and easy-to-administer tests has lead to an enormous variation on screening policies around the world. Practice variations also affect prostate cancer therapy.

Objectives: Gender as a social construct is a recognized health determinant. Because best practice in reporting health technology assessment (HTA) clearly specifies the need to appraise a technology's social impact within the target population, the extent to which gender issues are taken into account in HTA production is of interest, not only in light of equitable practices but also for reasons of effectiveness. The aim of this study is to provide a first assessment of the degree of gender sensitivity shown by HTA agencies around the world today.

Methods: The Web sites of sixty HTA agencies were analyzed. The consideration of gender aspects was specifically looked for in each agency's general mission statement, its priority setting process, and its methodological approach. Additionally, specific gender-oriented initiatives not belonging to any of the aforementioned categories were identified.

Results: Of the sixty agencies, less than half mention a commitment to addressing the social implication of health technologies. Only fifteen institutions make information on their priority setting principles available on their Web sites and gender was an issue in two of those cases. Data on methodology were obtainable online from18 agencies, two of which mentioned gender issues explicitly. Finally, gender-oriented initiatives were identified by thirteen agencies.

Conclusions: A gender-sensitive approach is apparently rarely adopted in current HTA production. Exceptional practices and relevant tools do exist and could serve as examples to be promoted by international collaborative networks.

Background: The matching of favorable human leukocyte antigen (HLA) combinations is rarely performed in penetrating keratoplasty procedures for primary prophylaxis of immune reactions. However, clinical studies suggest that the incidence of graft rejection decreases substantially when patients receive favorably matched grafts.

Objective: The aim of this study was to assess the cost-effectiveness of HLA matching for patients undergoing penetrating keratoplasty in everyday clinical practice.

Methods: In the absence of a randomized controlled clinical trial, we used administrative data from the Freiburg University Eye Hospital in Germany. Our study population consisted of all patients who underwent their first keratoplasty between 11/2003 and 01/2010 and for whom information on HLA histocompatibility was available. We used propensity score matching to estimate a causal effect of favorable HLA matching, parametric survival regression techniques to predict graft survival and expert opinion to model incremental cost for HLA matching. Because the availability of favorable HLA histocompatibility ultimately depends on the patients’ HLA phenotype, we modeled the incremental cost-effectiveness ratio (ICER) as a function of the probability that a patient will receive a favorably matched HLA, and used expert opinion to set a point estimate.

Results: We predicted that corneal grafts with favorable HLA matching were associated with improved rejection-free graft survival time (more than 1,000 days). We estimated the incremental cost of HLA matching at EUR 1,200 and the ICER at EUR 4.62 per additional day of graft survival.

Conclusions: The ICER of HLA matching is acceptable, given the high cost of alternative treatment and the shortage of corneal donors in Germany.

Objective: A United Kingdom Department of Health directive (EL[94]72) asked Health Authorities to manage the entry of new drugs into practice. There seem to be costs associated with the decision-making process of managed entry, but no clear evidence of benefit to patient populations. The objective of this study was to assess the potential costs and outcomes of different models of managed entry, using the example of donepezil in the North West Health Region of the U.K. National Health Service. This is a preliminary study designed to identify the key pieces of information required to evaluate the value of managed entry.

Methods: Decision analytic models of three Health Authorities' approaches to manage the entry of donepezil were used to estimate the expected costs and effectiveness of the process. Resource use data were obtained from published sources and the relevant Health Authority. Probabilistic sensitivity analysis was used to determine the robustness of the results.

Results: The process of managed entry of donepezil was associated with higher expected costs and higher expected outcome than no managed entry. The 95% confidence intervals for the net expected costs and net expected outcomes were relatively narrow and did not cross zero, which suggests a statistical difference between managed entry and no managed entry for donepezil. The incremental cost-effectiveness ratios for managed entry of donepezil indicate that, compared with no managed entry, there were substantial differences between the different models used in the three study sites. The expected cost per unit of cognitive function gained was between £18,000 in study site 001 to £28,000 in study site 010. The expected cost per person with a clinically significant improvement was between £140,000 and £230,000. The expected cost per QALY ranged from £470,000 to £19.3 million.

Conclusions: Managed entry does not appear to be a worthwhile mechanism to introduce drugs into practice. However, poor accessibility and availability of data means that the results are highly uncertain. The lack of data presents serious obstacles for both researchers and policy makers wishing to develop evidence-based policy and practice.

The number of publications on health technology assessment (HTA) from Central, Eastern, and Southern Europe (CESE countries) is still low compared with the north and west of Europe. It is not surprising, as the idea of HTA originated from high-income Western economies and was afterward adopted by the south-eastern part of Europe, which mostly consists of middle-income countries. These CESE countries, with less capacity and experience with HTA processes, must deal with even tougher decisions on financing health technologies than north-western Europe. There may even be a lack of confidence to open discussions on their specific needs for HTA.

Background: Health-related quality of life (HRQoL) is an important endpoint of many healthcare interventions. This study develops guidance on how to select appropriate HRQoL measures for inclusion in a clinical trial, given the purposes of the HRQoL measurement.

Methods: The guidance is based on a systematic literature review, discussions with members of the European Network for Health Technology Assessment (EUnetHTA) and two rounds of public consultation.

Results: A set of twelve recommendations was developed, addressing the requirements for HRQoL data for relative effectiveness assessment, for cost-utility analyses and for informing clinical decision making. Recommendations relate to the choice of the type of measure as well as to aspects such as measurement frequency, target population and presentation.

Conclusions: The purpose and context of HRQoL measurement is crucial for the relevance of the data obtained with a specific HRQoL measure. It is recommended to always include a generic HRQoL instrument in clinical trials to cover a wide range of possible future uses of the HRQoL data.

Objectives: In the Netherlands, allocation decisions have not yet been explicitly based on the Value of Statistical Life. However, when policy makers decide whether or not to implement life saving interventions this trade-off is made implicitly. This study aimed to gain insights into this trade-off, hereafter referred to as Implicit Value of Statistical Life (IVSL), by means of a retrospective investment analysis of life saving interventions implemented in the Netherlands.

Methods: A literature search was conducted to find life saving intervention cases meeting the requirements for IVSL calculation. A final sample of ten cases was included in the study concerning interventions implemented in different societal sectors. For each case, an IVSL estimate was calculated according to a uniform method.

Results: IVSL estimates derived from the intervention cases differed considerably and ranged from €1 to almost €11 million. Differences were most extreme when comparing IVSL estimates concerning interventions implemented in different societal sectors. However, IVSL estimates also varied greatly between interventions in the same sector and even within the same interventions when critical assumptions were altered.

Conclusion: Our findings suggest that there are great imbalances between societal investments for preventing a statistical death. This highlights the need for further deliberation about how to improve transparency of policy decisions. An approach ex ante determining the Value of Statistical Life by means of empirical methods and based on societal preferences might circumvent the problems associated with the IVSL and needs further exploration.

Medical technology will contribute to improving the health status of people in developing countries only when it is carefully implemented after proper planning. Providers, users, health ministry employees, and industry all share responsibility for proper planning and careful implementation.

This paper describes the technical approach in the TUFFA (Technology Procurement for Disabled in Working Life) project and a model for cooperating areas of competence based on a holistic view of the individual's abilities, the environmental conditions at the work site, and technical solutions.

In the Central African Republic (CAR) malaria is a major public health problem and hampers socioeconomic development. It accounts for 40 percent of complaints and 10 percent of deaths in health facilities (15;17). Pregnant women, who make up 4 percent of the population, and children under 5 years of age, who represent 17.3 percent, are the groups most vulnerable to malaria owing to their low levels of immunity.

Objectives: Guidelines for economic evaluation studies recommend that modeling be undertaken to estimate long-term, downstream costs. In this study, we conduct a review of a sample of studies that estimated the lifetime medical care costs for a variety of conditions.

Methods: We developed a categorization of the elements for a lifetime-costing study and based on these elements, we abstracted information from a sample of 33 papers in the following areas: study subject, purpose, scope, methods (including time profile, utilization, and cost), and results.

Results: We analyzed papers that were observational, models or that combined the two approaches. The time profiles were estimated from registry and published data. Utilization data were obtained from administrative data, chart reviews, and professional opinion. Costs were obtained from administrative and financial records and were estimated using all charges, allocated costs, and provider payments. We noted wide variations in methods and reporting practices.

Conclusions: Following current guidelines (CCOHTA), lifetime models can be more easily interpreted and applied if investigators are more clear in their study aims, if they incorporate assumptions that are based on current data, if they follow current methodological practices (such as deflation, discounting, and sensitivity analyses), and if reporting is more transparent.

Objectives: A rapid scoping review was performed to support the development of a new clinical technology platform. An iterative sifting approach was adopted to address the challenges posed by the nature of the review question and the extremely large volume of search results to be sifted within the timescales of the review.

Methods: This study describes the iterative sifting approach applied in the scoping review and a preliminary validation of the methods applied.

Results: The searches performed for the rapid scoping review retrieved 27,198 records. This was the full set of records subjected to the staged, iterative sifting approach and the subsequent validation process. The iterative sifting approach involved the screening for relevance of 17,354 (i.e., 63.8 percent) of the 27,198 records. A list of fifty-three potential biomarker names was generated as a result of this iterative sifting method, of which nineteen were selected by clinical specialists for further scrutiny. The preliminary validation involved the exhaustive sifting of the remaining 9,844 previously unsifted records. The validation process identified sixteen additional potential biomarker names not identified by the iterative sifting process. The clinical specialists subsequently concluded that none were of further clinical interest.

Conclusions: This study describes an approach to the screening of search records that can be successfully applied in appropriate review and decision problems to allow the prioritization of the most relevant search records and achieve time savings. Following further refinement and standardization, this iterative sifting method may have potential for further applications in reviews and other decision problems.