Objectives: The objectives of this study were to identify, in a systematic literature review, published studies having used quality-adjusted life years (QALYs) based on actual measurements of patients' health-related quality of life (HRQoL) and to determine which HRQoL instruments have been used to calculate QALYs. Furthermore, the aims were to characterize studies with regard to medical specialty, intervention studied, results obtained, quality, country of origin, QALY gain observed, and interpretation of results regarding cost-effectiveness.

Methods: Systematic searches of the literature were made using the MEDLINE, Embase, CINAHL, SCI, and Cochrane Library electronic databases. Initial screening of identified articles was based on abstracts read independently by two of the authors; full-text articles were again evaluated by two authors, who made the final decision on which articles should be included.

Results: The search identified 3,882 articles; 624 were obtained for closer review. Of the reviewed full-text articles, seventy reported QALYs based on actual before–after measurements using a valid HRQoL instrument. The most frequently used instrument was EuroQol HRQoL instrument (EQ-5D, 47.5 percent). Other instruments used were Health Utilities Index (HUI, 8.8 percent), the Rosser–Kind Index (6.3 percent), Quality of Well-Being (QWB, 6.3 percent), Short Form-6D (SF-6D, 5.0 percent), and 15D (2.5 percent). The rest (23.8 percent) used a direct valuation method: Time Trade-Off (10.0 percent), Standard Gamble (5.0 percent), visual analogue scale (5.0 percent), or rating scale (3.8 percent). The most frequently studied medical specialties were orthopedics (15.5 percent), pulmonary diseases (12.7 percent), and cardiology (9.9 percent). Ninety percent of the studies came from four countries: United Kingdom, United States, Canada, the Netherlands. Approximately half of the papers were methodologically high quality randomized trials. Forty-nine percent of the studied interventions were viewed by the authors of the original studies as being cost-effective; only 13 percent of interventions were deemed not to be cost-effective.

Conclusions: Although QALYs gained are considered an important measure of effectiveness of health care, the number of studies in which QALYs are based on actual measurements of patients' HRQoL is still fairly limited.

Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions, specific legislation has been enacted to encourage the development of drugs for rare diseases (orphan drugs), which would otherwise not be commercially viable. However, because of the small market, these drugs are often very expensive. Under the standard methods of health technology assessment (HTA) incorporating economic evaluation, orphan drugs do not usually prove to be cost-effective and this, coupled with their high cost, means that funding and patient access may be limited. However, these restrictions may not be in line with societal preferences. Therefore, this study discusses whether the standard methods of HTA are adequate for assisting decisions on patient access to and funding of orphan drugs and outlines a research agenda to help understand the societal value of orphan drugs and issues surrounding their development, funding, and use.

Objectives: Health technology assessment (HTA) is increasingly an international activity, and HTA agencies collaborate to avoid unnecessary duplication of effort. However, the sharing of the results from HTAs raises questions about their generalizability; namely, are the results of an HTA undertaken in one country relevant to another?

Methods: This study presents recommendations for increasing the generalizability of economic evaluations. They represent an important component of HTAs and are commonly thought to have limited generalizability.

Results: Recommendations are given for studies using patient-level data (i.e., evaluations conducted alongside clinical trials) and for studies using decision analytic modeling.

Conclusions: If implemented, the recommendations would increase the value for investments in HTA.

There is increasing emphasis on providing patient-focused health care and ensuring patient involvement in the design of health services. As health technology assessment (HTA) is meant to be a multidisciplinary, wide-ranging policy analysis that informs decision making, it would be expected that patients’ views should be incorporated into the assessment. However, HTA is still driven by collection of quantitative evidence to determine the clinical and cost effectiveness of a health technology. Patients’ perspectives about their illness and the technology are rarely included, perhaps because they are seen as anecdotal, biased views. There are two distinct but complementary ways in which HTAs can be strengthened by: (i) gathering robust evidence about the patients’ perspectives, and (ii) ensuring effective engagement of patients in the HTA process from scoping, through evidence gathering, assessment of value, development of recommendations and dissemination of findings. Robust evidence eliciting patients’ perspectives can be obtained through social science research that is well conducted, critically appraised and carefully reported, either through meta-synthesis of existing studies or new primary research. Engagement with patients can occur at several levels and we propose that HTA should seek to support effective patient participation to create a fair deliberative process. This should allow two-way flow of information, so that the views of patients are obtained in a supportive way and fed into decision-making processes in a transparent manner.

Objectives: This study investigated the benefits of and barriers to user involvement in medical device technology development and evaluation.

Methods: A structured review of published literature in peer-reviewed journals was conducted.

Results: This literature review revealed that the main benefits of user involvement were an increased access to user needs, experiences, and ideas; improvements in medical device designs and user interfaces; and an increase in the functionality, usability, and quality of the devices. On the other hand, resource issues, particularly those of time and money were found the key impediments to involving users in the development and evaluation of medical device technologies. This study also has categorized both the benefits of and barriers to user involvement.

Conclusions: The involvement of users in medical device technology development and evaluation requires resources, which are limited; however, this involvement is essential from both users and manufacturers perspectives.

Medical noncompliance has been identified as a major public health problem that imposes a considerable financial burden upon modern health care systems. There is a large research record focusing on the understanding, measurement, and resolution of noncompliance, but it is consistently found that between one third and one half of patients fail to comply with medical advice and prescriptions. Critically absent from this research record has been the patient's role in medical decision making. For patients, particularly those with chronic illnesses, compliance is not an issue: they make their own reasoned decisions about treatments based on their own beliefs, personal circumstances, and the information available to them. The traditional concept of compliance is thus outmoded in modern health care systems, where chronic illness and questioning patients predominate.

Objectives: The aim of this study was to assess the test performance and clinical effectiveness of photodynamic diagnosis (PDD) compared with white light cystoscopy (WLC) in people suspected of new or recurrent bladder cancer.

Methods: A systematic review was conducted of randomized controlled trials (RCTs), nonrandomized comparative studies, or diagnostic cross-sectional studies comparing PDD with WLC. Fifteen electronic databases and Web sites were searched (last searches April 2008). For clinical effectiveness, only RCTs were considered.

Results: Twenty-seven studies (2,949 participants) assessed test performance. PDD had higher sensitivity than WLC (92 percent, 95 percent confidence interval [CI], 80–100 percent versus 71 percent, 95 percent CI, 49–93 percent) but lower specificity (57 percent, 95 percent CI, 36–79 percent versus 72 percent, 95 percent CI, 47–96 percent). For detecting higher risk tumors, median range sensitivity of PDD (89 percent [6–100 percent]) was higher than WLC (56 percent [0–100 percent]) whereas for lower risk tumors it was broadly similar (92 percent [20–95 percent] versus 95 percent [8–100 percent]). Four RCTs (709 participants) using 5-aminolaevulinic acid (5-ALA) as the photosensitising agent reported clinical effectiveness. Using PDD at transurethral resection of bladder tumor (TURBT) resulted in fewer residual tumors at check cystoscopy (relative risk [RR], 0.37, 95 percent CI, 0.20–0.69) and longer recurrence-free survival (RR, 1.37, 95 percent CI, 1.18–1.59), compared with WLC.

Conclusions: PDD detects more bladder tumors than WLC, including more high-risk tumors. Based on four RCTs reporting clinical effectiveness, 5-aminolaevulinic acid–mediated PDD at TURBT facilitates a more complete resection and prolongs recurrence-free survival.

There has been an exponential growth in the literature on economic evaluation in health care. As the range and quality of analytical work has improved, economic studies are becoming more influential with health care decision makers. The development of standards for economic evaluation methods would help maintain the scientific quality of studies, facilitate the comparison of economic evaluation results for different health care interventions, and assist in the interpretation of results from setting to setting. However, standardization might unnecessarily stifle methodological developments. This paper reviews the arguments for and against standardization, assesses attempts to date, outlines the main areas of agreement and disagreement on methods for economic evaluation, and makes recommendations for further work.

Objectives: The aim of this study was to suggest an acceptable and generic theoretical framework for involving various types of users in the medical device technology (MDT) development process (MDTDP).

Methods: The authors propose a theoretical framework suggesting different routes, methods and stages through which various types of medical device users can be involved in the MDTDP.

Results: The suggested framework comprises two streams of users’ involvement in MDT development, that is, what might be called the end users’ stream and the professional users’ stream for involving these two groups respectively in the process of developing both simple and more complex and innovative medical devices from conceptualization through to the market deployment. This framework suggests various methods that can be used for users’ involvement at different stages of the MDT lifecycle. To illustrate the application of the framework, several MDT development scenarios and device exemplars are presented.

Conclusions: Development of medical devices from users’ perspectives requires not only the involvement of healthcare professionals but also that of the ultimate end users, that is, patients, people with disabilities and/or special needs, and their caregivers. The evidence shows that such end users quickly discard devices that do not fulfill their personal expectations, even though both manufacturers and healthcare professionals may consider those end users’ requirements met. Developers and manufacturers need to recognize this potent potential discrepancy between the parties involved, and involve end users and professional healthcare staff directly in the MDTDP. The framework, the authors contend, is a step forward in helping medical device manufacturers plan and make decisions about users’ involvement at different stages of the MDTDP.

Objective: We reviewed and appraised the methods by which the issue of the learning curve has been addressed during health technology assessment in the past.

Method: We performed a systematic review of papers in clinical databases (BIOSIS, CINAHL, Cochrane Library, EMBASE, HealthSTAR, MEDLINE, Science Citation Index, and Social Science Citation Index) using the search term “learning curve.”

Results: The clinical search retrieved 4,571 abstracts for assessment, of which 559 (12%) published articles were eligible for review. Of these, 272 were judged to have formally assessed a learning curve. The procedures assessed were minimal access (51%), other surgical (41%), and diagnostic (8%). The majority of the studies were case series (95%). Some 47% of studies addressed only individual operator performance and 52% addressed institutional performance. The data were collected prospectively in 40%, retrospectively in 26%, and the method was unclear for 31%. The statistical methods used were simple graphs (44%), splitting the data chronologically and performing a t test or chi-squared test (60%), curve fitting (12%), and other model fitting (5%).

Conclusions: Learning curves are rarely considered formally in health technology assessment. Where they are, the reporting of the studies and the statistical methods used are weak. As a minimum, reporting of learning should include the number and experience of the operators and a detailed description of data collection. Improved statistical methods would enhance the assessment of health technologies that require learning.

Objectives: This review assessed current practice in the preparation of rapid reviews by health technology assessment (HTA) organizations, both internationally and in the Australian context, and evaluated the available peer-reviewed literature pertaining to the methodology used in the preparation of these reviews.

Methods: A survey tool was developed and distributed to a total of fifty International Network of Agencies for Health Technology Assessment (INAHTA) members and other selected HTA organizations. Data on a broad range of themes related to the conduct of rapid reviews were collated, discussed narratively, and subjected to simple statistical analysis where appropriate. Systematic searches of the Cochrane Library, EMBASE, MEDLINE, and the Australian Medical Index were undertaken in March 2007 to identify literature pertaining to rapid review methodology. Comparative studies, guidelines, program evaluations, methods studies, commentaries, and surveys were considered for inclusion.

Results: Twenty-three surveys were returned (46 percent), with eighteen agencies reporting on thirty-six rapid review products. Axiomatic trends were identified, but there was little cohesion between organizations regarding the contents, methods, and definition of a rapid review. The twelve studies identified by the systematic literature search did not specifically address the methodology underpinning rapid review; rather, many highlighted the complexity of the area. Authors suggested restricted research questions and truncated search strategies as methods to limit the time taken to complete a review.

Conclusions: Rather than developing a formalized methodology by which to conduct rapid reviews, agencies should work toward increasing the transparency of the methods used for each review. It is perhaps the appropriate use, not the appropriate methodology, of a rapid review that requires future consideration.

The aim of Working Group 4 has been to develop and disseminate best practice in undertaking and reporting assessments, and to identify needs for methodologic development.

Cost-effectiveness analysis (CEA) is a method of economic evaluation that can be used to assess the efficiency with which health care technologies use limited resources to produce health outputs. However, inconsistencies in the way that such ratios are constructed often lead to misleading conclusions when CEAs are compared. Some of these inconsistencies, such as failure to discount or to calculate incremental ratios correctly, reflect analytical errors that, if corrected, would resolve the inconsistencies. Others reflect fundamental differences in the viewpoint of the analysis. The perspectives of different decision-making entities can properly lead to different items in the numerator and denominator of the cost-effectiveness (C/E) ratio. Producers and consumers of CEA need to be more conscious of the perspectives of analysis, so that C/E comparisons from a given perspective are based upon a common understanding of the elements that are properly included.

To practice evidence-based medicine, physicians need data on the clinical effectiveness, toxicity, convenience, and cost of new drugs compared with available alternatives. We give examples of published drug studies that are defective, sometimes because pharmaceutical industry funding has affected their content and quality. We make recommendations on how to avoid these defects.

Objectives: In 2000, the first “Dutch Manual for Costing: Methods and Reference Prices for Economic Evaluations in Healthcare” was published, followed by an updated version in 2004. The purpose of the Manual is to facilitate the implementation and assessment of costing studies in economic evaluations. New developments necessitated the publication of a thoroughly updated version of the Manual in 2010. The present study aims to describe the main changes of the 2010 Manual compared with earlier editions of the Manual.

Methods: New and updated topics of the Manual were identified. The recommendations of the Manual were compared with the health economic guidelines of other countries, eliciting strengths and limitations of alternative methods.

Results: New topics in the Manual concern medical costs in life-years gained, the database of the Diagnosis Treatment Combination (DBC) casemix System, reference prices for the mental healthcare sector and the costs borne by informal care-givers. Updated topics relate to the friction cost method, discounting future effects and options for transferring cost results from international studies to the Dutch situation.

Conclusions: The Action Plan is quite similar to many health economic guidelines in healthcare. However, the recommendations on particular aspects may differ between national guidelines in some respects. Although the Manual may serve as an example to countries intending to develop a manual of this kind, it should always be kept in mind that preferred methods predominantly depend on a country's specific context.

We assessed the predictive validity of an expert panel's ratings of the appropriateness of carotid endarterectomy by comparing ratings to the results of subsequent randomized clinical trials. We found the trials confirmed the ratings for 44 indications (covering almost 30% of operations performed in 1981) and refuted the ratings for none.

Evidence-informed patient choice involves providing people with research-based information about the effectiveness of health care options and promoting their involvement in decisions about their treatment. Although the concept seems desirable, the processes and outcomes of evidence-informed patient choice are poorly understood, and it should be carefully evaluated.

Objectives: The research objectives were two-fold: first, to systematically review the literature on the cost-effectiveness of home telehealth for chronic diseases, and second to develop a framework for the conduct of economic evaluation of home telehealth projects for patients with chronic diseases.

Methods: A comprehensive literature search identified twenty-two studies (n = 4,871 patients) on home telehealth for chronic diseases published between 1998 and 2008. Studies were reviewed in terms of their methodological quality and their conclusions.

Results: Home telehealth was found to be cost saving from the healthcare system and insurance provider perspectives in all but two studies, but the quality of the studies was generally low. An evaluative framework was developed which provides a basis to improve the quality of future studies to facilitate improved healthcare decision making, and an application of the framework is illustrated using data from an existing program evaluation of a home telehealth program.

Conclusions: Current evidence suggests that home telehealth has the potential to reduce costs, but its impact from a societal perspective remains uncertain until higher quality studies become available.

A framework is presented that distinguishes the conceptually separate decisions of which treatment strategy is optimal from the question of whether more information is required to inform this choice in the future. The authors argue that the choice of treatment strategy should be based on expected utility, and the only valid reason to characterize the uncertainty surrounding outcomes of interest is to establish the value of acquiring additional information. A Bayesian decision theoretic approach is demonstrated through a probabilistic analysis of a published policy model of Alzheimer's disease. The expected value of perfect information is estimated for the decision to adopt a new pharmaceutical for the population of patients with Alzheimer's disease in the United States. This provides an upper bound on the value of additional research. The value of information is also estimated for each of the model inputs. This analysis can focus future research by identifying those parameters where more precise estimates would be most valuable and indicating whether an experimental design would be required. We also discuss how this type of analysis can also be used to design experimental research efficiently (identifying optimal sample size and optimal sample allocation) based on the marginal cost and marginal benefit of sample information. Value-of-information analysis can provide a measure of the expected payoff from proposed research, which can be used to set priorities in research and development. It can also inform an efficient regulatory framework for new healthcare technologies: an analysis of the value of information would define when a claim for a new technology should be deemed substantiated and when evidence should be considered competent and reliable when it is not cost-effective to gather any more information.

Background: Patient involvement is widely acknowledged to be a valuable component in health technology assessment (HTA) and healthcare decision making. However, quantitative approaches to ascertain patients' preferences for treatment endpoints are not yet established. The objective of this study is to introduce the analytic hierarchy process (AHP) as a preference elicitation method in HTA. Based on a systematic literature review on the use of AHP in health care in 2009, the German Institute for Quality and Efficiency in Health Care (IQWiG) initiated an AHP study related to its HTA work in 2010.

Methods: The AHP study included two AHP workshops, one with twelve patients and one with seven healthcare professionals. In these workshops, both patients and professionals rated their preferences with respect to the importance of different endpoints of antidepressant treatment by a pairwise comparison of individual endpoints. These comparisons were performed and evaluated by the AHP method and relative weights were generated for each endpoint.

Results: The AHP study indicates that AHP is a well-structured technique whose cognitive demands were well handled by patients and professionals. The two groups rated some of the included endpoints of antidepressant treatment differently. For both groups, however, the same six of the eleven endpoints analyzed accounted for more than 80 percent of the total weight.

Conclusions: AHP can be used in HTA to give a quantitative dimension to patients' preferences for treatment endpoints. Preference elicitation could provide important information at various stages of HTA and challenge opinions on the importance of endpoints.