Objectives: The objective of this study is to describe how patient aspects are assessed in Danish HTA reports, thereby contributing to the ongoing international debates concerning patient aspects in HTAs.

Methods: Fifty-eight Danish HTA reports published from 1999 to 2010 were read systematically, focusing on the inclusion of patient aspects, the methods used to generate data, and if and how clinical recommendations concerning patient aspects were drawn.

Results: Most of the fifty-eight HTA reports had a separate chapter about patient aspects. All reports included literature reviews for patient aspects. There was, however, much variation in the ways in which the authors presented and discussed their review methods. The most frequently used databases for the literature search were medical and HTA databases. More than half of the reports included primary research, either quantitative or qualitative methods, a few used both kinds of methods. Whereas some mentioned the importance of including patient aspects in daily clinical practice, others provided ideas on how to improve patient information or recommended changes in healthcare practices.

Conclusions: Danish HTA reports do, to a large extent, include patient aspects in the assessment and in the final conclusions of the reports. If health policy and decision making is to be patient-focused, it is important that HTAs in the future integrate patient aspects in recommendations of HTA. Further improvement in assessments of patient aspects, in relation to use and description of methodologies and theoretical considerations, will assist producing the relevant evidence for these recommendations.

Objectives: Contextual data and local expertise are important sources of data that cannot be ignored in hospital-based health technology assessment (HTA) processes. Despite a lack of or unconvincing evidence in the scientific literature, technology can be recommended in a given context. We illustrate this using a case study regarding biplane angiography for vascular neurointervention.

Methods: A systematic literature review was conducted, along with an analysis of the context in our setting. The outcomes of interest were radiation doses, clinical complications, procedure times, purchase cost, impact on teaching program, the confidence of clinicians in the technology, quality of care, accessibility, and the volume of activity. A committee comprising managers, clinical experts, physicians, physicists and HTA experts was created to produce a recommendation regarding biplane technology acquisition to replace a monoplane device.

Results: The systematic literature review yielded nine eligible articles for analysis. Despite a very low level of evidence in the literature, the biplane system appears to reduce ionizing radiation and medical complications, as well as shorten procedure time. Contextual data indicated that the biplane system could improve operator confidence, which could translate into reduced risk, particularly for complex procedures. In addition, the biplane system can support our institution in its advanced procedures teaching program.

Conclusions: Given the advantages provided by the biplane technology in our setting, the committee has recommended its acquisition. Contextual data were of utmost importance in this recommendation. Moreover, this technology should be implemented alongside a responsibility to collect outcome data to optimize clinical protocol in the doses of ionizing delivered.

In this paper we describe the present range of organizations that have a role in the early warning of new and emerging health care technologies in the United Kingdom. We discuss in more detail the processes and prioritization criteria used by the U.K. horizon-scanning project for the NHS Health Technology Assessment Programme, and the principal methods of technology identification for the horizon-scanning project are outlined. The United Kingdom plans to develop an integrated system for the identification of technologies for commercial planning, health service research prioritization, financial planning, and provision of information to policy makers, purchasers, and providers of health care.

Eight hundred and six medical and surgical patients who were hospitalized via the emergency ward were followed over their entire inpatient stays and rated in anterograde, double-blind fashion for inpatient incidents (falls, medication errors, other). Injuries were minor but affected 2.2% of admissions, a figure which is strikingly similar to studies in other hospitals. There was a statistical trend toward a higher-than-normal risk of hazardous in-hospital incidents for males age 20 to 40 admitted because of injury and for medically ill females over 60 years old.

Objectives: The purpose of this study was to empirically explore whether individuals take their expected income into consideration when directly valuing predefined health states. This was intended to help determine how to handle productivity costs due to morbidity in a cost-effectiveness analysis.

Methods: Two hundred students each valued four hypothetical health states by using time trade-off (TTO) and a visual analogue scale (VAS). The students were randomly assigned to two groups. One group was simply asked, without mentioning income, to value the different health states (the non-income group). The other group was explicitly asked to consider their expected income in relation to the health states in their valuations (the income group).

Results: For health states that are usually assumed to have a large effect on income, the valuations made by the income group seemed to be lower than the valuations made by the non-income group. Among the students in the non-income group, 96 percent stated that they had not thought about their expected income when they valued the health states. In the income group, 40 percent believed that their expected income had affected their valuations of the health states.

Conclusion: The results show that, as long as income is not mentioned, most individuals do not seem to consider their expected income when they value health states. This indicates that productivity costs due to morbidity are not captured within individuals' health state valuations. These findings, therefore, suggest that productivity costs due to morbidity should be included as a cost in cost-effectiveness analyses.

Objectives: To examine quantitatively the effects of an increase in patient copayments from 10% to 20% on the demand for medical services in Japan.

Methods: The subjects of the study were the employees insured by the 1,797 health insurance societies, belonging to the National Federation of Health Insurance Societies, in 1996 and 1998. Indicators of medical service demands analyzed include the inpatient, outpatient, and dental case rates, the number of serviced days per case, the medical cost per day and the medical cost per insured.

Results: When the effects of an increase in patient copayments from 10% to 20% were evaluated, taking into account the average age, the average monthly salary, the total number, the gender (male-to-female) ratio and the dependent ratio of the insured, the estimated change in the case rate was −6.96% for inpatient, −4.79% for outpatient, and −5.77% for dental care. The estimated change in the number of serviced day per case was −4.66% for inpatient, −5.67% for outpatient, and −1.82% for dental care. The estimated change in the medical cost per day was −3.15% for inpatient, −13.00% for outpatient, and −11.48% for dental care. The estimated change in the medical cost per insured was −14.08% for inpatient, −21.54% for outpatient, and −18.11% for dental care.

Conclusions: The increase in patient copayments from 10% to 20% enabled insurers to substantially reduce medical costs by cost shifting from the insurer to the insured, with resultant changes in the case rate and the number of service days per case.We thank Ms. Danita Joell for administrative assistance. This study was founded by a grant-in-aid for scientific research (c) from the Japan Ministry of Education, Science, and Culture (grant number 11670373).

The FDA recently approved Ceredase™, a new treatment for Gaucher's disease, under the provisions of the Orphan Drug Act. Ceredase™ is unusually expensive, but there are no satisfactory alternative therapies. It appears likely that Ceredase™would not have become available without the protection of the Orphan Drug Act, but its expense and the lack of information about its long-term effects on health raise questions about whether the ODA provides appropriate incentives to develop cost-effective technologies.

Objectives: The mainly tax-paid healthcare system in Denmark is decentralized with three defined policy and management levels. Health technology assessment (HTA) as a concept was introduced in the beginning of the 1980s. Significant implementation only happened when the first national strategy for HTA was developed by relevant stakeholders and issued as an official document in 1996. The introduction and the further development are described.

Methods: The Danish Institute for HTA was established in the National Board of Health in 1997 with responsibility for coordination and production of HTA. A local government reform from 2007 provides several new challenges to HTA and its coordination.

Results: An external evaluation in 2003 indicated that HTA was widely disseminated and that HTA results were benefitting political, administrative, and clinical decision making at all levels of the healthcare system.

Conclusions: The first national strategy for HTA, the broad HTA model covering four elements: Technology (clinical aspects), Patient, Organization, and Economy, and the development and introduction of mini-HTA as a tool for HTA-related activities in institutions and municipalities are major contributions to international HTA.

Background: The Institute for Quality and Efficiency in Health Care (IQWiG) was established in 2003 by the German parliament. Its legislative responsibilities are health technology assessment, mostly to support policy making and reimbursement decisions. It also has a mandate to serve patients’ interests directly, by assessing and communicating evidence for the general public.

Objectives: To develop a priority-setting framework based on the interests of patients and the general public.

Methods: A theoretical framework for priority setting from a patient/consumer perspective was developed. The process of development began with a poll to determine level of lay and health professional interest in the conclusions of 124 systematic reviews (194 responses). Data sources to identify patients’ and consumers’ information needs and interests were identified.

Results: IQWiG's theoretical framework encompasses criteria for quality of evidence and interest, as well as being explicit about editorial considerations, including potential for harm. Dimensions of “patient interest” were identified, such as patients’ concerns, information seeking, and use. Rather than being a single item capable of measurement by one means, the concept of “patients’ interests” requires consideration of data and opinions from various sources.

Conclusions: The best evidence to communicate to patients/consumers is right, relevant and likely to be considered interesting and/or important to the people affected. What is likely to be interesting for the community generally is sufficient evidence for a concrete conclusion, in a common condition. More research is needed on characteristics of information that interest patients and consumers, methods of evaluating the effectiveness of priority setting, and methods to determine priorities for disinvestment.

Objectives: Many innovative health technologies do not have a sufficient evidence-base to allow for adequate assessment of their benefits. Funders in several countries have been exploring arrangements that allow for temporary or partial coverage of these technologies, but only as part of a further evaluation. The public's support of arrangements that restrict access to innovative technology until sufficient evidence is available is crucial if these arrangements are going to remain viable. The project's other objective is to examine the lay public's views on a case in which patients’ publicly funded access to an innovative health technology is being delayed until there is sufficient evidence to justify a coverage decision. The case considered is the Ontario (Canada) government's decision to restrict access to positron emission tomography (PET) scans until further evidence becomes available.

Methods: The case was deliberated on by twenty-six members of the Toronto Health Policy Citizens' Council, with a follow-up survey administered to individual council members.

Results: The majority of council members agreed that the approach taken by the government was reasonable and in the best interests of its citizens. The council did express concerns regarding certain aspects of the case, including about the length of time it is taking to obtain further evidence.

Conclusions: Public support for arrangements that limit access to new technologies will likely vary depending on the details of the specific arrangement being proposed. Deliberative public dialogue can be effectively used to identify cases the general public is most likely to support.

Any evaluation of intensive care must include an ethical assessment of that technology. This allows us to consider the use of technologyin light of the ends that we desire. The most pressing ethical issues in intensive care are: forgoing life-sustaining treatment, dehumanization of patients and staff within the technological environment, and the allocation of the technology that is integral to intensive care.

Objectives: To identify and examine the methodologic issues related to evaluating the effectiveness of treatment adherence to clinical guidelines. The example of antiretroviral therapy guidelines for human immunodeficiency virus (HIV) disease is used to illustrate the points.

Methods: Regression analysis was applied to observational HIV clinic data for patients with CD4+ cell counts less than 500 per μL and greater than 50 per μL at baseline (n = 704),using Cox proportional hazards time-varying covariates models controlling for baseline risk. The results are compared with simpler models (Cox model [without time-varying covariates] and logistic regression). In addition, the effect of including a measure of exposure to antiretroviral guidelines in the model is explored.

Results: This study has three implications for modeling clinical guideline effectiveness. To capture events that are time-sensitive, a duration model should be used, and covariates that are time-varying should be modeled as time-varying. Thirdly, incorporating a threshold measure of exposure to reflect the minimum period of time for guideline adherence required for a measurable effect on patient outcome should be considered.

Conclusions: The methods proposed in this paper are important to consider if guidelines are to evolve from being a tool for summarizing and transferring the results of research from the literature to clinicians into a practical tool that influences clinical practice patterns. However, the methodology tested in this study needs to be validated using additional data on similar patients and using data on patients with other diseases.

Objectives: To examine the growing evidence and the consensus in the medical community concerning the efficacy and cost-effectiveness of hyperbaric oxygen therapy (HBO2T) and to suggest future research areas to ensure the appropriate use of this technology.

Methods: A literature search of articles published between 1985 and 2000 was conducted using PubMed to describe the growth of HBO2T-related articles published over the past fifteen years. In addition, articles involving the qualitative synthesis of the efficacy and cost-effectiveness of HBO2T in thirteen major application areas were identified and compared with the changing view of the medical community toward the evidence of HBO2T.

Results: The total number of HBO2T-related articles published annually has steadily increased over the past fifteen years. This increase has gradually contributed to a consensus in the medical community concerning the evidence of efficacy in its major application areas. However, information regarding the cost-effectiveness of HBO2T is still extremely limited.

Conclusions: Although evidence concerning the efficacy of HBO2T has been growing in the past, more evidence is still needed for some of its major application areas. Moreover, there is an urgent need to shift part of the research focus on HBO2T to its cost-effectiveness to provide decision makers with relevant information to evaluate this technology objectively.

More than 10 years ago, the Australian government took the bold and unprecedented step of announcing that it would request evidence on comparative cost-effectiveness of new pharmaceutical products, assess this evidence, and then use it to guide systematically the decisions as to whether to subsidize these new products. This paper summarizes the Australian drug subsidy scheme and the activities involved in making this announcement work in practice, assesses the impact these activities have had on broader policy actions, and examines whether these have resulted in any worthwhile achievements.

Background: Oncotype DX (ODX), a tumor gene profiling test, has been incorporated into clinical guidelines to aid in adjuvant chemotherapy decision making for early-stage, hormone receptor positive breast cancer patients. Despite United States (U.S.) guidelines, less than half of eligible women receive testing. Reasons for low usage are unclear: Our objective was to better understand U.S. oncologists’ ODX uptake and how they use ODX during adjuvant chemotherapy decision making.

Methods: We conducted semi-structured, ~30-minute phone interviews with medical and surgical oncologists in one U.S. State using purposive sampling. Oncologists were included if they saw greater than or equal to five breast cancer patients per week. Recruitment ended upon thematic saturation. Interviews were recorded, transcribed, and double-coded using template analysis.

Results: During analysis, themes emerged across three domains. First, organizational factors (i.e., departmental structure, ODX marketing, and medical/insurance guidelines) influenced ease of ODX use. Second, oncologists referenced the influence of interpersonal factors (e.g., normative beliefs and peer use of ODX) over their own practices and recommendations. Third, intrapersonal factors (e.g., oncologist attitudes, perceived barriers, and research gaps) were discussed: although oncologists largely held positive attitudes about ODX, they reported challenges with interpreting intermediate scores for treatment decisions and explaining test results to patients. Finally, oncologists identified several research gaps.

Conclusions: As more tumor gene profiling tests are incorporated into cancer care for treatment decision making, it is important to understand their use in clinical practice. This study identified multi-level factors that influence ODX uptake into clinical practice, providing insights into facilitators and modifiable barriers that can be leveraged for improving ODX uptake to aid treatment decision making.

This study deals with the question of how to model health effects after the cessation of a randomized controlled trial (RCT). By using clinical trial data on severe congestive heart failure patients, we illustrate how survival beyond the cessation of an RCT can be predicted based on parametric survival models. In the analysis, we compare predicted survival and the resulting incremental cost-effectiveness ratio (ICER) of different survival models with actual survival/ICER. Our main finding is that the results are sensitive to the choice of survival model and that an extensive sensitivity analysis in the CE analysis is required.We thank John Kjekshus for providing us with data from the consensus and a ten-year follow-up study. Comments from Magnus Johannesson on a previous version of the article are also highly appreciated.

Objectives: The aim of this study was to review 5 years of activity from a new system devised by the National Institute for Health and Care Excellence (NICE), for assessing medical devices and diagnostics aimed at identifying and speeding adoption of technologies with clinical and cost advantages, compared with current practice in the United Kingdom healthcare system.

Methods: All eligible notified technologies were classified using the Food and Drug Administration and Global Medical Device Nomenclature nomenclatures. Decisions about selecting technologies for full assessment to produce NICE recommendations were reviewed, along with the reasons given to companies for not selecting products.

Results: Between 2009 and 2014, 186 technologies were notified (46 percent therapeutic and 54 percent diagnostic). Thirty-nine were judged ineligible (no regulatory approval), and 147 were considered by an independent committee. Of these, eighty (54 percent) were not selected for full assessment, most commonly because of insufficient evidence (86 percent): there were uncertainties specifically about benefits to the health service (54 percent), to patients (39 percent), and about cost (24 percent). The remaining 67 were selected and assessed for Medical Technology guidance (52 percent) (noninferior and/or lower cost consequences than current practice), for Diagnostics guidance (43 percent) or other NICE recommendations about adoption and use. Classifying technologies by two different systems showed no selection bias for any technology type or disease area.

Conclusions: Identifying new or under-used devices and diagnostics with potential benefits and promoting their adoption is important to health services in the United Kingdom and worldwide. This new system offers a means of fostering both uptake and further research. Lack of research data on new products is a major obstacle to evaluation.