Objectives: This study is to review health technology assessment (HTA) development in China during the past two decades, and to facilitate further development of HTA and its integration into policy making.

Methods: The study depends very much on the extensive experiences of the authors in involvement of HTA in China. In addition, literature review and Web site searching have been used to trace the process of growth of HTA in China.

Results: With its rapid economic development, the Chinese health system has had many achievements in the past 30 years. However, there are some weaknesses that have developed during this period. HTA is traced back to the early 1990s in China. First, HTA knowledge transfer and establishment of HTA units were effective ways to develop HTA in China. By the end of 1990s, the policy makers of the Ministry of Health (MoH) made efforts to merge HTA with policy making to improve the quality and efficiency of health care. The main government authorities related to health technology are the State Food and Drug Administration, the Ministry of Labor and Social Security, and the MoH. Their involvements in HTA are varied.

Conclusion: A technology licensure mechanism based on HTA, including technology permission for use, institution licensure, and workforce licensure, is being gradually carried out by the MoH in China. Moreover, HTA can play an important role in technology market entry, insurance benefit coverage, formulary, clinical pathway, reimbursement, and so on. There is a great opportunity for HTA to be an important part of health reform, especially to help policy makers within the health sector to make difficult decisions.

Objective: To explore the impact of methodologic issues on the results of meta-analyses. The following issues were examined: the type of literature search strategy used; inclusion or exclusion of non–peer-reviewed studies; the inclusion or exclusion of non-English language publications; the effect of trial quality; and the inclusion or exclusion of non–placebo-controlled studies.

Methods: The International Study of Perioperative Transfusion (ISPOT) meta-analyses were used to evaluate each of the methodologic issues. The 10 meta-analyses consisted of technologies to reduce the need for perioperative red blood cell transfusion. The number of trials for each of the meta-analyses varied from 2 to 45. Both EMBASE and MEDLINE searches were conducted, including the use of systematic search strategies.

Results: MEDLINE identified the vast majority of trials. Alone, MEDLINE would have missed 8 studies compared to 10 for EMBASE. Use of the systematic search strategies greatly reduced the number of articles to be reviewed compared to open searches. Type of publication, country of study origin, inclusion of non-English publications, and trial quality had very little impact on the estimates of effect. The use of placebo versus open-label control affected the magnitude of the odds ratio for two of the meta-analyses. The results of the two meta-analyses were not statistically significant if only placebo-controlled trials were included.

Conclusions: While methodologic issues had very little impact on the ISPOT meta-analyses, further studies are needed in a variety of other clinical settings. Because MEDLINE, coupled with a review of the references in the identified trials, identified the vast majority of trials, one needs to consider the costs and benefits of searching EMBASE and the pursuance of unpublished and unindexed trials.

Objectives: The mode of contact and response levels of authors who had been asked to provide missing or incomplete data for a systematic review on diet and exercise interventions for weight loss was examined.

Methods: We contacted authors by electronic mail, letter, or both. Survival analyses were performed with the Kaplan–Meier method to determine differences in the proportion of responders over time among the different modes of contact and to determine whether response rates differed between authors from the United States and those from other countries. Logistic regression was used to determine whether the number of items requested and publication date influenced the likelihood of response.

Results: Two hundred forty-one (39.9 percent) studies had missing or incomplete data (e.g., sample size, age, caloric restriction, exercise amount, and so on). We were unable to locate ninety-five authors (39.4 percent). Of the remaining authors, forty-six authors (31.5 percent) responded to information requests. Time to respond differed by contact method (p<.05): e-mail (3 ± 3 days), letter (27 ± 30 days), and both (13 ±12 days). Response rates from U.S. authors did not differ from those of other countries.

Conclusions: Our study suggests poor success in the acquisition of essential information. Given considerable time and resources, weight loss studies require improved reporting standards to minimize the relatively unsuccessful attempt to contact authors for important and necessary information.

Data relevant to economic evaluation are being collected alongside clinical trials with increasing frequency. When these trials involve health outcomes and resource utilization in many different countries, numerous methodologic problems arise. This paper describes and discusses the methodology for economic evaluation that will be used in the Economic Substudy of GUSTO llb, as an example of how to address the problems that arise in economic evaluations linked to international trials.

Objectives: Health technology assessment (HTA) programs influence practice on a broad scale through reimbursement decisions or national guidelines. Hospital-based HTA programs inform clinical decisions at the local level. Typically, they do this by adapting general HTA to their local setting, or by creating new HTA. However, unlike payer-based HTA organizations, hospital-based HTA organizations can also integrate local data into their reports.

Methods: We describe two examples of local data integrated into hospital-based HTA. In the first, qualitative data were used to select a new cardiac catheterization lab. In the second, quantitative data was used to inform a decision on whether to continue telemedicine services to critical care units. Local evidence sources included equipment service records, and interviews with physicians, technicians, and administrative staff in the first example, and the hospital's administrative and claims databases in the second example.

Results: In each case, there was little evidence from the peer-reviewed literature that could be applied to the decision. In the first example, staffing patterns and local preferences had considerable bearing on technology choices. In the second example, local outcomes data from administrative records were decisive.

Conclusions: Hospital-based HTA using local data can fill gaps in the published evidence, and also improve the generalizability of evidence to the local setting. To take advantage of local evidence, health systems should encourage the development of hospital-based HTA centers, seek out local preference data, and maintain databases of patient outcomes and utilization of services.

This paper examines the determinants of hip fracture costs and further evaluates potential savings in costs when the occurrence of hip fracture is prevented. The costs of hip fracture are comprised of direct costs from health care and the social welfare system. Data were collected for 1,080 postmenopausal women admitted from private residence for primary hip fracture surgery during the year of 1992 in the city of Stockholm, Sweden. It was found that the cost of hip fracture is significantly related to age, mortality the year after a fracture, type of fracture, costs 1 year before a fracture, and hospital admission. The savings in direct costs for an average woman surviving the year after a fracture amount to SEK 210,000.

Objectives: In quality-adjusted life-years (QALY) models, it is customary to weigh life-years with quality of life via multiplication. As a consequence, for positive health states a longer duration has more QALYs than a shorter duration (i.e., longer is better). However, we have found that for poor health states, many prefer to live only a limited amount of time (i.e., longer is worse). Such preferences are said to be maximum endurable time (MET). In the present contribution, the following questions are asked: a) How low does the utility have to be in order for a MET to arise? and b) Do MET preferences occur when patients judge hypothetical health states?

Methods and Results: We reanalyzed data from 176 students for the hypothetical health states of “living with migraines” and “living with metastasized cancer.” For utilities smaller than 0.7 (ranging from 0 to 1), the MET preference rate was larger than 50%. High MET preference rates were also found in two new studies on migraine and esophageal cancer patients, who evaluated hypothetical health states related to their disease.

Conclusions: We discuss the interpretation of the MET preferences and the preference reversal phenomenon. Standard QALY models imply that longer is better. However, we find that more often, longer is worse for poorly evaluated health states. Consider the following question: are 3 years with a weight of 0.3 equally as valuable as 1 year with a weight of 0.9? Our results suggest that the 3-year period may be less valuable because for poor health, many will prefer a 1-year over a 3-year period.

Objectives: This article explains how the issue of stakeholder involvement was addressed in the European network for Health Technology Assessment (EUnetHTA) Project and describes the structures of future stakeholder involvement in the EUnetHTA Collaboration.

Methods: Initiatives led to a dialogue with stakeholders and exchanging views and expectations on health technology assessment (HTA) processes and the future development of EUnetHTA. The methods of involving different stakeholder groups in EUnetHTA included general information to stakeholders about EUnetHTA, targeted information on a Web site, analysis of stakeholder opinions on HTA and EUnetHTA, and development of a draft stakeholder policy.

Results: First steps were taken to organize processes to consolidate the legitimacy of EUnetHTA and its products and encourage the representation of interests, thus contributing to promoting the utilization of HTA in national/regional policy making. A stakeholder Web site, analyses of stakeholder opinions on HTA and EUnetHTA in a discussion topic catalog, and a draft stakeholder policy resulted from the work.

Conclusions: Stakeholder involvement in EUnetHTA is necessary to ensure the legitimacy and prospects for utilization of EUnetHTA and its products. The described activities and results create the foundation for a continued dialogue with, and involvement of, stakeholders. The EUnetHTA stakeholder meeting can be considered as a successful experience of dialogue between EUnetHTA and stakeholders, which should be continued. Our experience shows the challenge of obtaining balanced stakeholder representation across the identified stakeholder groups. Continued attention should be given to achieving balanced stakeholder representation.

This article provides a critical analysis of the impact of health technology assessment (HTA) on priority setting in The Netherlands. It describes the limited steering powers of the Dutch government; its complex interactions with insurers, health-care providers, and patients; and the role of HTA in this context as an attempt to rationalize the debate about cost-effectiveness issues. HTA has been drawn upon for decision making on the health insurance package. Also, HTA findings have been linked to the national guideline development programs of the medical community. However, these impacts by no means have been straightforward. We argue that the political nature of the priority-setting debate asks for a broader approach to what constitutes HTA, and how it should be drawn upon in priority setting. Suggestions are made on how to do justice to the social dynamics of decision making and the behavior of stakeholders in health-care systems.

Objectives: The clinical and cost-effectiveness of left ventricular assist devices as destination therapy for people with end-stage heart failure is assessed through a systematic review and economic evaluation.

Methods: Systematic review was performed of randomized controlled trials, quasiexperimental studies, case series, and case studies identified through searching eighteen electronic databases, bibliographies, and consultation with experts and manufacturers. Studies assessed survival, functional capacity, and quality of life. Cost-effectiveness was assessed through a 5-year decision analytic model to estimate the incremental cost-effectiveness ratio for using left ventricular assist devices compared with usual care.

Results: Six studies met the inclusion criteria, showing that left ventricular assist devices appear beneficial, improving survival and quality of life. Adverse events are a serious concern. The economic evaluation showed that left ventricular assist devices had a cost per quality adjusted life year of £170,616. Sensitivity analysis showed that the cost-effectiveness was not sensitive to changes in costs or utility.

Conclusions: Although left ventricular assist devices appear clinically effective as destination therapy, it is unlikely they will be cost-effective unless costs decrease or the benefits of their use increase.

Objectives: There are no current established pathognomonic diagnostic features for uterine leiomyosarcomas in the pre- or perioperative setting. Recent inadvertent upstaging of this rare malignancy during laparoscopic morcellation of a presumed fibroid has prompted widespread debate among clinicians regarding the safety of current surgical techniques for management of fibroids. This study aims to conduct a systematic review investigating significant diagnostic features in magnetic resonance imaging (MRI) of uterine leiomyosarcomas.

Methods: A comprehensive database search was conducted guided by PRISMA recommendations for peer-reviewed publications to November 2017. Parameters available in MRI were compared for reliability and accuracy of diagnosis of leiomyosarcomas. A decision tree algorithm classifier model was constructed to investigate whether T1 and T2 MRI signal intensities are useful indicators.

Results: Nine eligible studies were identified for analysis. There appears to be a significant relationship between histopathological type and T1 and T2 intensity signals (p < .05). A decision tree model analyzing T1 and T2 signal intensity readings supports this trend, with a diagnostic specificity of 77.78 percent for uterine leiomyosarcomas. The apparent diffusion coefficient (ADC) values were not observed to have a significant relationship with tumor pathology (p = .18).

Conclusions: Various studies have investigated pre- and perioperative techniques in differentiating uterine leiomyosarcoma from benign fibroids. Given the rarity of the malignancy and lack of pathognomonic diagnostic parameters, there is difficulty in establishing definitive criteria. A decision tree model is proposed to aid diagnosis based on MRI signal intensities.

Objectives: The cost-effectiveness of fracture prevention treatments (vitamin D and calcium and hip protectors) in male and female populations older than seventy years of age in the United-Kingdom was investigated.

Methods: A Markov model was developed to follow up, over lifetime, a hypothetical cohort of males and females at high-risk and general risk of fracture. Patients could sustain hip, wrist, vertebral, and/or other fractures. Fracture rates were obtained from population surveys in the United Kingdom. Effectiveness and quality of life data were identified from the clinical literature. Costs were those incurred by the UK National Health Service, and were obtained from several published sources. Uncertainty was explored through probabilistic sensitivity analysis.

Results: In the general-risk female (male) population, the incremental cost per Quality Adjusted Life Year (QALY) was $11,722 ($47,426) for hip protectors. In the male high-risk population, the incremental cost per QALY was $17,017 for hip protectors. In the female high-risk population, hip protectors were cost-saving. Vitamin D and calcium alone was dominated by hip protectors in all four subgroups.

Conclusions: Current information available on interventions to prevent fractures in the elderly in the United Kingdom, suggests that, at the decision-maker's ceiling ratio of $20,000 per QALY, hip protectors are cost-effective in the general female population and high-risk male population, and cost-saving in the high-risk female population, despite the low compliance rate with the treatment.

Objectives: The objective of this study is to assess the impact of CEDIT (French Committee for the Assessment and Dissemination of Technological Innovations) recommendations on the introduction of technological innovations within the AP-HP (Assistance Publique–Hôpitaux de Paris), the French hospital network to which this body is attached.

Methods: In 2002, a study based on semidirective interviews of fourteen people affected by these recommendations and a case study relating to thirteen recommendations issued between 1995 and 1998 were conducted.

Results: The CEDIT is very scientifically reputable among interviewees. There is generally widespread interest for the recommendations. They are used as decision-making tools by administrative staff and as negotiating instruments by doctors in their dealings with management. Based on the case study, ten of thirteen recommendations had an impact on the introduction of the technology in health establishments. One recommendation appears not to have had an impact. Furthermore, the impact of two technologies was impossible to assess.

Conclusions: This study highlights the significant impact of recommendations arising from a structure that is attached to a hospital network and the good match between CEDIT's objectives and its assignments.

Through the use of three scenarios, this paper presents the challenges for clinical practice guidelines in the 21st century. Such challenges relate to technological developments to improve the efficiency and pace of the development process, to ensure that clinical practice guidelines are kept up to date, and to facilitate implementation of guidelines in the clinical setting. To improve and ensure the validity of the content of clinical practice guidelines, we need to address the important problem of publication bias, for which researchers, granting agencies, industry, and journal editors share responsibility. This means insisting on registration of trials at their inception, and incentives backed up by rules for funding and peer review publication that would promote behaviors to avoid publication bias. The more difficult challenges for clinical practice guidelines relate to what are referred to as attitudinal factors. To achieve optimal efficiencies in development and maintenance of clinical practice guidelines, we need to promote cooperation among various information resource providers internationally and to stress partnership over leadership. Finally, there needs to be reconciliation of the different stakeholder perspectives of the value and purpose of clinical practice guidelines so that they are used appropriately as aids to decision making and are not abused as tools for controlling clinical practice.

Objectives: To explore the cost-effectiveness of early biologic treatment, followed by dose-reduction in the case of remission, of active rheumatoid arthritis (RA), compared with standard treatment with methotrexate (MTX) in Sweden.

Methods: Effectiveness (function, disease activity, erosions) in early RA for both alternatives was taken from a clinical trial comparing etanercept (ETA) combined with MTX to MTX alone. Patients discontinuing treatment can switch to another or their first biologic treatment. For patients in remission (Disease Activity Score [DAS28] < 2.6), ETA is reduced to half the dose. Return to full dose occurs when DAS28 reaches ≥ 3.2 again. Costs and utilities by level of functional capacity from an observational study are used. The model is analyzed as a micro-simulation and results are presented from the societal perspective for Sweden, for 10 years; costs (€2008) and effects are discounted at 3 percent. Sensitivity analysis was performed for the perspective, the time horizon, switching, and dose-reduction.

Results: The main analysis conservatively assumes 50 percent switching at discontinuation. The cost per quality-adjusted life-year (QALY) gained with early ETA/MTX treatment is €13,500 (societal perspective, incremental cost of €15,500 and incremental QALYs of 1.15). With 75 percent switching, the cost per QALY gained was €10,400. Over 20 years, the cost per QALY gained was €8,200. Results were further sensitive to the time patients remained on half dose and the perspective.

Conclusions and Policy Implications: This study combines clinical trial and clinical practice data to explore cost-effective treatment scenarios in early RA, including the use of biologics. Our results indicate that a situation where a considerable proportion of patients achieve remission, dose-adjustments will increase the cost-effectiveness of treatment.

Objectives: Although health technology assessment (HTA) has been well established in all developed countries, it has not found a firm footing in many developing countries. This is especially true of the Asia Pacific region, which has much of the world population.

Methods: The beginnings of HTA in this region go back to the work of Dr. David Banta in China and the establishment of the Asian HTA Network. The Network embarked on increasing awareness and building capacity among personnel from the region. Attempts were made to introduce HTA in countries where it did not exist, as well as to strengthen existing HTA programs. The Network had regular meetings, and also carried out a study on the diffusion and utilization of magnetic resonance imaging in the region. In an attempt to boost the efforts of the Network, the author spearheaded the organization of the Asian Regional HTA Conferences in Kuala Lumpur in 2000 and 2003, and in Manila, Philippines, in 2004. In addition, the author carried out a survey on HTA in the region. There are two broad categories of countries in the region: Australia, Malaysia, Singapore, New Zealand, China, Philippines, Korea, Thailand, and Taiwan, which have formal HTA programs, and others for which informal mechanisms or related activities exist, which include Bangladesh, Bhutan, Brunei, Cambodia, India, Indonesia, Laos, Maldives, Mongolia, Nepal, Pakistan, Sri Lanka, and Vietnam.

Results: It is important that once HTA is established, it is used effectively. Perseverance and dedication is needed to ensure the success of an HTA program.

Conclusion: Some countries in the region have effective HTA programs, whereas in some, efforts are being made to establish HTA, or HTA-related activities are being carried out in the absence of a formal HTA program.

Objectives: Health services research is a typical instance of applied research, meaning that its research findings should contribute to a more evidence-based health policy. A basic assumption for the usefulness of policy research is that it is of good scientific quality, but evaluation of health services research would be incomplete without an assessment of its societal relevance. However, there is no generally accepted framework in which criteria to assess societal relevance are defined.

Methods: In this study, we made an effort to specify criteria and indicators for policy relevance.

Results and Conclusions: In general, we concluded that it is important to define from the start of a research project, what kind of answers are being sought to the questions at hand, who the intended users of these answers will be, and how they can be reached. Furthermore, a productive dialogue between researchers and policy makers is indispensable for remaining in tune with policy thinking. In addition, we specified domains and a list of potential criteria for assessing policy relevance. For the purpose of quality assurance, these criteria have to be integrated into a quality improvement cycle. This means that the societal output of health services research should be related to the aims and missions at the level of projects, programs, and institutes. Furthermore, it is important that consensus is reached about (the weighting of) relevant indicators and that routines are developed for collecting information on these indicators.

Health technology assessment (HTA) has become increasingly important in the European Union as an aid to decision making. As agencies and programs have been established, there is increasing attention to coordination of HTA at the European level, especially considering the growing role of the European Union in public health in Europe. This series of papers describes and analyzes the situation with regard to HTA in the 15 members of the European Union, plus Switzerland. The final paper draws some conclusions, especially concerning the future involvement of the European Commission in HTA.

Objectives: Traditional economic evaluations for most health technology assessments (HTAs) have previously not included environmental outcomes. With the growing interest in reducing the environmental impact of human activities, the need to consider how to include environmental outcomes into HTAs has increased. We present a simple method of doing so.

Methods: We adapted an existing clinical-economic model to include environmental outcomes (carbon dioxide [CO2] emissions) to predict the consequences of adding insulin to an oral antidiabetic (OAD) regimen for patients with type 2 diabetes mellitus (T2DM) over 30 years, from the United Kingdom payer perspective. Epidemiological, efficacy, healthcare costs, utility, and carbon emissions data were derived from published literature. A scenario analysis was performed to explore the impact of parameter uncertainty.

Results: The addition of insulin to an OAD regimen increases costs by 2,668 British pounds per patient and is associated with 0.36 additional quality-adjusted life-years per patient. The insulin-OAD combination regimen generates more treatment and disease management-related CO2 emissions per patient (1,686 kg) than the OAD-only regimen (310 kg), but generates fewer emissions associated with treating complications (3,019 kg versus 3,337 kg). Overall, adding insulin to OAD therapy generates an extra 1,057 kg of CO2 emissions per patient over 30 years.

Conclusions: The model offers a simple approach for incorporating environmental outcomes into health economic analyses, to support a decision-maker's objective of reducing the environmental impact of health care. Further work is required to improve the accuracy of the approach; in particular, the generation of resource-specific environmental impacts.