Objectives: The UK National Institute for Health and Clinical Excellence (NICE) has been widely hailed as an international leader in health technology assessment. The objectives of its guidance are to ensure uniformity in healthcare provision, and promote a systematic and accountable way to allocate resources. However, research relating to the implementation of guidance is limited, and little is known about how it influences decision making at the consultation level or how useful it is to individual patients.

Methods: In-depth interviews were undertaken with professionals involved in healthcare provision at the community level, and with clinical professionals and patients providing or receiving care for morbid obesity and breast cancer (n = 52).

Results: Although NICE guidance was generally well-regarded, in practice it was of more importance and usefulness to managerial than clinical professionals. Clinicians used a patchy approach to implementation depending on whether recommendations accorded with their personal interpretation of the evidence available, and whether funding was available locally. Many patients had not heard of NICE, and clinical professionals did not alert them to its existence. Even where patients knew about relevant guidance, they were rarely able to use it to assert their right to treatment.

Conclusions: These findings challenge the perception that NICE guidance results in consistent and accountable decision making, and the limited accessibility and usefulness of guidance to patients suggests more information and support is needed at the point of provision if these objectives are to be achieved.

Objectives: The aim of this study was to analyze the EuroScan Database and to describe and compare the characteristics of the included technologies and participating agencies.

Methods: Data of interest were exported from the EuroScan Database to Excel and to SPSS. A descriptive analysis depending on the agency, type of technology, stage of diffusion, and technology purpose was conducted. A frequency distribution analysis of the diffusion stage for different technology types and assigned purposes was made with the EpiCalc 2000 statistical calculator. A p value of less than .05 was considered to be statistically significant.

Results: Four agencies introduced the great majority of the technologies (81 percent), with drugs representing the 46.26 percent of the total, followed by devices (21.21 percent). The purpose of 24.45 percent of the identified technologies was not specified, and 34.58 percent of them were identified at the investigational or phase III stage. The frequency distribution of diffusion stage at identification was found to be similar for devices and diagnostics (p = .543), whereas drugs were identified earlier than devices (p <.001). Some agencies were found to focus their work on drugs, whereas others focused mainly on devices. Interagency differences were also observed with regard to the stage of diffusion at which technologies were identified.

Conclusions: This is the first analysis of one of the most important databases on new and emerging health technologies. Our study suggests that more active strategies should be designed to provide an earlier identification, mainly in the case of devices.

Objectives: The increasing availability of information about health care suggests an expanding role for consumers to exercise their preferences in health-care decision-making. Numerous methods are available to assess consumer preferences in health care. We conducted a systematic review to characterize the study of women's preferences about health care

Methods: A MEDLINE search from 1965 to July 1999 was conducted as well as hand searches of the itshape Medical Decision Making Journal (1981–1999) and references from retrieved articles. Only original articles on women's health issues were selected. Information on thirty-one variables related to study characteristics and preferences were extracted by two independent investigators. A third investigator resolved disagreements. Qualitative and quantitative analyses were conducted to synthesize the data.

Results: Four hundred eighty-three studies were identified in the initial search. Seventy articles were selected for review based on title, abstract, and inclusion criteria. There was an increase in published articles and number of methods used to elicit preferences. White women were studied more than black women (p<.001). Preferences were mainly studied in outpatient settings (p<.005) and in the United States, United Kingdom, and Canada (83 percent). Preferences related to participation in decision-making were the most common (21 percent). Only 4 percent of the studies were performed to inform the debate for public policy questions. Willingness to pay was the method most used (11 percent), followed by category scaling (10 percent), rating scale (9 percent), standard-gamble (6 percent). Preferences for individual particular (opposed to sequential and health states) outcomes (68 percent), different treatments/tests (47 percent), and related to a treatment episode (31 percent) were addressed. Information regarding diseases, conditions, or procedures was given in 57 percent of studies. Information provided was mainly written (37 percent) and included positive and negative potential outcomes (67 percent). There is no relationship between the method or tool used for delivery information and the choice performed.

Conclusions: The literature on preferences in women's health care is limited to a fairly homogeneous population (white women from the United States, United Kingdom, and Canada). Additionally, use of utility-based measures to capture preferences has decreased over time while others methods (e.g., time trade-off [TTO], contingent valuation) have increased. Women's preferences are not necessarily uniform even when asked similar questions using similar tools. Little information on women's preferences exists to inform policy-makers about women's health care.

Failures are inevitable in health care. When failure occurs as a result of practitioner error, quality in patient care may be compromised. This article proposes that analyzing clinical errors may contribute to quality assurance. Examples from physical therapy illustrate how information gained from analyzing errors can enhance patient care.

We examined the use of percutaneous transluminal coronary angioplasty, kidney stone lithotripsy, and bone marrow transplant among patients with different health insurance plans in California. HMO enrollees were less likely to receive these procedures compared with fee-for-service patients. Our results have implications for the inflationary effects of technology under managed care.

Objectives: This paper gives an overview of health technology assessment (HTA) in Belgium.

Methods: The information included in the overview is based on legal documents and publicly available year reports of the Belgian Health Care Knowledge Centre (KCE).

Results: Belgium has a relatively young history in HTA. The principle of evidence-based medicine (EBM) was introduced in the drug reimbursement procedure in 2001, with the establishment of the Drug Reimbursement Committee (DRC). The DRC assesses the efficacy, safety, convenience, applicability, and effectiveness of a drug relative to existing treatment alternatives. For some drugs, relative cost-effectiveness is also evaluated. The activities of the DRC can, therefore, be considered to be the first official HTA activities in Belgium. Later, in 2003, KCE was established. Its mission was to perform policy preparing research in the healthcare and health insurance sector and to give advice to policy makers about how they can obtain an efficient allocation of limited healthcare resources that optimizes the quality and accessibility of health care. This broad mission has been operationalized by activities in three domains of research: HTA, health services research, and good clinical practice. KCE is independent from the policy maker. Its HTAs contain policy recommendations that may inform policy decisions but are not binding.

Conclusions: Although the Belgian history of HTA is relatively short, its foundations are strong and the impact of HTA increasing. Nevertheless KCE has many challenges for the future, including continued quality assurance, further development of international collaboration, and further development of methodological guidance for HTA.

Objectives: The aim of this study is to examine key informants’ perceptions of how the New Zealand Pharmaceutical Management Agency (PHARMAC) operates in New Zealand.

Methods: We carried out qualitative analysis of semi-structured interviews with key informants. We obtained ethics approval from the University of Otago School of Pharmacy, and all participants gave informed consent. We digitally recorded the interviews, which were then transcribed, and coded in NVivo. The data were analyzed by theme using constant comparison methods. Twenty informants who had previously published research or commentary on New Zealand's access to medicines, acted as spokespersons for interest groups, or held positions that gave them key insights into New Zealand's medicines system agreed to participate. Informants were purposefully selected to ensure a wide range of views, including five people working in medicine, four in pharmacy, three Members of Parliament from different parties, and two each from PHARMAC and the pharmaceutical industry.

Results: Respondents saw PHARMAC as an organization that contained medicine costs effectively, was politically neutral, and resistant to lobbying. It enjoyed broad political support and, with extremely rare exceptions, had been allowed to carry out its functions independently regardless of who was in government. As a result of this political stability, the relationship between PHARMAC and the pharmaceutical industry has been improving.

Conclusion: PHARMAC's longevity and increasing influence are largely due to political choices made to prioritize containing pharmaceutical expenditure and to respecting PHARMAC's independence. This may be difficult to replicate in other countries.

This paper reviews the evolution of U.S. policy toward medical technology in areas such as cost containment, regulation of devices and drugs, and third party reimbursement. In addition the authors chronicle the diffusion of major medical technologies, procedures, and organizational innovations in the United States. Finally, the article provides tentative observations on the effect of recent policy changes and concludes with some recommendations for the future.

When it is well conducted, a randomized clinical provides the strongest evidence available for evaluating the comparative effectiveness of the interventions tested. Over the last two generations, we have learned much about various devices for strengthening them and about methods of avoiding between in their design, execution, analysis, and reporting. In a trial, we seek evidence for a causal link between treatment and observed outcomes. Becaues the controlled trial depends on an argument based on exculsion (i.e., no other causes or differences affected the experimental groups), we strengthen its inference by taking steps to exclude any such differences.

This article discusses a number of issues that deserve consideration: problems of multiplicity and generalizability, devices for strengthening trials, issues of power and sample size, the relationship between study design and reported gains, when to undertake a trial, the role of collaborative trials, and ways to make trials more feasible in clinical settings.

Although there have been numerous technological devices introduced for the diagnosis and treatment of temporomandibular disorders, many are either ineffective or are research tools without direct clinical application. This article reviews the various modalities and makes recommendations regarding their effectiveness based on the available clinical and scientific evidence.

Commodification of low-cost diagnostic technologies (LCDTs) carries the risk of an unintended and potentially worrisome shift in how we conceive of health assessment. Optimizing practice requires a balanced understanding of both the beneficial and harmful impact of the use of LCDTs and the development of system incentives for appropriate use.

This study examined the conclusions of published reports that review the literature and make recommendations about appropriate applications of bone density measurement. It is based on a survey of 22 organizations producing such reports between 1986 and Spring 1995. Overall, the application of bone density measurement for the diagnosis of osteoporosis was supported by 65% of reports, by 44% for the monitoring and follow-up of patients with previously diagnosed disease, and by 59% for monitoring and follow-up of patients receiving treatment that may affect their bone density. A smaller proportion of reports from government and public organizations compared with other types of organizations and a smaller proportion of reports using more rigorous methods supported the applications for bone density measurement identified in the survey. A larger proportion of the reports published after 1990 compared with those published between 1986 and 1990 support the applications of bone density measurement, with the exception of population screening. The results of this survey suggest that there is considerable disagreement about the potential applications of bone density measurement. Publicly funded organisations and those using more rigorous methods tended to be more conservative in their conclusions.

Objectives: Value assessment frameworks have gained prominence recently in the context of U.S. healthcare. Such frameworks set out a series of factors that are considered in funding decisions. The UK's National Institute of Health and Care Excellence (NICE) is an established health technology assessment (HTA) agency. We present a novel application of text analysis that characterizes NICE's Technology Appraisals in the context of the newer assessment frameworks and present the results in a visual way.

Methods: A total of 243 documents of NICE's medicines guidance from 2007 to 2016 were analyzed. Text analysis was used to identify a hierarchical set of decision factors considered in the assessments. The frequency of decision factors stated in the documents was determined and their association with terms related to uncertainty. The results were incorporated into visual representations of hierarchical factors.

Results: We identified 125 decision factors, and hierarchically grouped these into eight domains: Clinical Effectiveness, Cost Effectiveness, Condition, Current Practice, Clinical Need, New Treatment, Studies, and Other Factors. Textual analysis showed all domains appeared consistently in the guidance documents. Many factors were commonly associated with terms relating to uncertainty. A series of visual representations was created.

Conclusions: This study reveals the complexity and consistency of NICE's decision-making processes and demonstrates that cost effectiveness is not the only decision-criteria. The study highlights the importance of processes and methodology that can take both quantitative and qualitative information into account. Visualizations can help effectively communicate this complex information during the decision-making process and subsequently to stakeholders.

With an increasing awareness that active engagement between policy decision makers, HTA agencies, regulators and payers with industry in the premarket space is needed, a disruptive comprehensive approach is described which moves the evidentiary process exclusively into this space. Single harmonized studies pre-market to address regulatory and coverage needs and expectations are more likely to be efficient and less costly and position evidence to drive rather than test innovation. An example of such a process through the MaRS EXCITE program in Ontario, Canada, now undergoing proof of concept, is briefly discussed. Other examples of dialogue between decision makers and industry pre-market are provided though these are less robust than a comprehensive evidentiary approach.

Objectives: Japanese authorities have announced a plan to introduce a health technology assessment (HTA) system in 2016. This study assessed the potential impact of such a policy on the price of the antivirologic drug simeprevir.

Methods: Taking the antivirologic drug simeprevir as an example, we compared the current Japanese price with hypothetical prices that might result if a U.K. (cost-utility) or German (efficiency frontier) style HTA assessment was in place.

Results: The simeprevir unit price under the current Japanese pricing scheme is 13,122 Japanese yen (equivalent to 109.35 U.S. dollars as of April 2015). Depending on the selection of comparators and the pricing method, and assuming that HTA will be used as a basis for price setting, the estimated prices of simeprevir vary up to four times higher than under the current Japanese pricing scheme.

Conclusions: Although the analysis is based on only one drug, it cannot be taken for granted that a new HTA system would reduce public healthcare expenditure in Japan.

Health technology assessment (HTA) conducted to inform developers of health technologies (development-focused HTA, DF-HTA) has a number of distinct features when compared to HTA conducted to inform usage decisions (use-focused HTA). To conduct effective DF-HTA, it is important that analysts are aware of its distinct features as analyses are often not published. We set out a framework of ten features, drawn from the literature and our own experience: a target audience of developers and investors; an underlying user objective to maximize return on investment; a broad range of decisions to inform; wide decision space; reduced evidence available; earlier timing of analysis; fluid business model; constrained resources for analysis; a positive stance of analysis; and a “consumer”-specific burden of proof. This paper presents a framework of ten features of DF-HTA intended to initiate debate as well as provide an introduction for analysts unfamiliar with the field.