Health technology assessment (HTA) currently focuses on efficiency, rather than equity, on the basis that its primary objective is to maximize population health. Yet a strict cost-effectiveness approach sometimes conflicts with important equity concerns, such as the reduction of socioeconomic health inequalities. Managing such equity–efficiency trade-offs on the basis of intuition is unsatisfactory in a democracy, as it arouses suspicions of special pleading and favoritism toward vested interests. Over the next few decades, therefore, decision making may progress through up to three further stages of development observed historically in other areas of resource allocation. Stage two involves case law, limited to principles distilled from precedent. Stage three involves codification, seeking to generalize these principles without specifying their relative weights. Finally, at stage four, quantitative trade-offs are incorporated into a formula. At stage four, deliberation centers on adjustments to the formula, which would then be applied impartially, transparently, and fair-mindedly to all future decisions. Methods already exist for valuing equity–efficiency trade-offs, based on established methodological principles for valuing trade-offs between different dimensions of health. Early findings indicate that the general public thinks that social class inequalities are more inequitable than those by smoking status, with inequalities between the sexes somewhere in between. Relative weights can be calculated from these data, although the data are not yet comprehensive enough to do this credibly for current policy purposes. In the mean time, the equity–efficiency trade-offs suggested by current decisions can be estimated using standard cost-effectiveness analysis. This is because every departure from a strict cost-effectiveness approach has an opportunity cost. The size of that opportunity cost is a test of how much weight a particular equity concern is deemed to merit.

Objectives: Australia, Canada, and many European countries now use various forms of health technology assessment (HTA) in decision making regarding the reimbursement of drugs and other health technologies. To achieve a better understanding of the potential for use of HTA in this context, an analytical framework was developed to describe and classify existing fourth hurdle systems.

Methods: Based on a review of published literature, and official documentation, the key aspects of a fourth hurdle system were identified at two levels: policy implementation and individual technology decision. Characteristics of the systems were grouped under four main headings: constitution and governance, objectives, use of evidence and decision processes, and accountability. The comprehensiveness and relevance of this framework was assessed by an independent group of experts in HTA. A pilot study was undertaken, using only published sources, to test the feasibility of obtaining the information needed to complete the framework.

Results: The framework was found to be sufficiently broad to encompass all the issues of interest regarding the systems, but the proportion of information available from published sources was variable between sections of the framework and between countries, with average availability of 45 percent.

Conclusions: The analytical framework will help researchers and policy-makers in individual countries to understand their own systems and will allow some preliminary sharing of experience between countries. More experience of its application is needed to judge whether it will provide the basis for more formal comparison of systems and whether it will determine the appropriateness for particular decision contexts.

Objectives: Health technology assessment (HTA) has been used as input in decision making worldwide for more than 25 years. However, no uniform definition of HTA or agreement on assessment methods exists, leaving open the question of what influences the choice of assessment methods in HTAs. The objective of this study is to analyze statistically a possible relationship between methods of assessment used in practical HTAs, type of assessed technology, type of assessors, and year of publication.

Methods: A sample of 433 HTAs published by eleven leading institutions or agencies in nine countries was reviewed and analyzed by multiple logistic regression.

Results: The study shows that outsourcing of HTA reports to external partners is associated with a higher likelihood of using assessment methods, such as meta-analysis, surveys, economic evaluations, and randomized controlled trials; and with a lower likelihood of using assessment methods, such as literature reviews and “other methods.” The year of publication was statistically related to the inclusion of economic evaluations and shows a decreasing likelihood during the year span. The type of assessed technology was related to economic evaluations with a decreasing likelihood, to surveys, and to “other methods” with a decreasing likelihood when pharmaceuticals were the assessed type of technology.

Conclusions: During the period from 1989 to 2002, no major developments in assessment methods used in practical HTAs were shown statistically in a sample of 433 HTAs worldwide. Outsourcing to external assessors has a statistically significant influence on choice of assessment methods.

Objectives: To apply the Theory of Constraints (TOC) to the Evidence-based Practice Center (EPC) process.

Methods: Participants in the EPC process were interviewed, and the technical contracting literature was reviewed. Undesirable effects (UDEs) that might occur in the EPC process were identified and, in a TOC Current Reality Tree (CRT), a fundamental UDE was selected and tracked back to potential root causes, focusing on the core “constraint.” The resulting draft CRT was presented at an EPC directors' meeting and finalized. From possible solutions obtained from interviews and literature, a Future Reality Tree (FRT) was constructed.

Results: Three UDEs were identified and that a report is not used was deemed fundamentally undesirable. Nine root causes were identified, and using the CRT, a core constraint emerged: EPC partner does not know how to conceptualize and articulate needs, objectives, and specifications. Two change strategies (“injections”) appeared to address the core constraint: establish an ongoing relationship between partners, EPC, and Agency for Healthcare Research and Quality early in the process, and use a changes clause; in a FRT the injections lead to the positive outcome—the production of a successful EPC report that meets partner needs and, therefore, will be used.

Conclusions: As with any complex enterprise, the EPC needs to engage in self-reflection and active improvement to maximize its value. The development of technical reports intended to inform decision making represents a complicated area, involving a network of interrelated processes. Using the TOC permitted us to understand process failures; results serve as a guide to improvement.

Objectives: The purpose of this study was to identify the Evidence-Based Practice Center (EPC) network participants' perceptions of the characteristics of the EPC process and the relationship of the process to the success of EPC reports.

Methods: Semistructured interviews were conducted with the three groups involved in the EPC: EPC staff, Agency for Healthcare Research and Quality (AHRQ) staff, and representatives of partner organizations.

Results: The analysis of the coded transcripts revealed three related major themes, which form the conceptual basis for the interpretation presented here: the definition of a successful report, the determinants of a successful report, and the role of AHRQ in the process.

Conclusions: A successful report is a report that is used. The ultimate success of the core health technology assessment objective, moving from research to policy, depends on balancing two values: excellence and relevance. Our findings are consistent with the “two communities thesis,” which postulates the existence of two camps that confer different values to excellence and relevance, with resulting tension. A promising model for approaching this tension is integration or collaboration, which requires linking researchers and policy makers, promoting productive dialogues about the formulation and timing of analysis, and early consideration of how the resulting analysis will be used. This effort suggests that actively blurring the frontiers between these two groups will enhance their interaction. Furthermore, enhancing the role of the AHRQ as scientific broker will maximize the potential of the EPC network.

Objectives: The aim of this study was to evaluate the success of search strategies in retrieving key documents for a technology assessment report (TAR) on a social care topic.

Methods: This study measured the differential yield of relevant studies from various information sources and evaluated strategies in different databases, with particular reference to capturing randomized controlled trials (RCTs) as a study design.

Results: A combination of four major databases would have found all thirty-two key references. One database alone would have found 78 percent, with another two each locating 59 percent. Sixteen percent of the trials were unique references. In non–health care databases, more sensitive search strategies would have resulted in a higher yield of relevant studies, in part due to inconsistent indexing and in part to attempts to restrict searches to RCTs. Although additional terms could be used to increase the sensitivity of the original strategies, this raises the question of trading off time against exhaustiveness, given the greater number of irrelevant references likely to be retrieved.

Conclusions: A successful search for evidence on this social care topic would be possible using a combination of MEDLINE, EMBASE, the Cochrane Library and PsycINFO, supplemented by only limited use of supplementary databases. In areas such as social care where evidence-based research is not yet well established, attempts to replicate searches based on study design do not seem to be advisable, although this may be an area for future research.

Objectives: An overview was produced of indicators currently used to assess disease management programs and, based on these findings, provide a framework regarding sets of indicators that should be used when taking the aims and types of disease management programs into account.

Methods: A systematic literature review was performed.

Results: Thirty-six studies met the inclusion criteria. It appeared that a link between aims of disease management and evaluated structure, process, as well as outcome indicators does not exist in a substantial part of published studies on disease management of diabetes and asthma/chronic obstructive pulmonary disease, especially when efficiency of care is concerned. Furthermore, structure indicators are largely missing from the evaluations, although these are of major importance for the interpretation of outcomes for purposes of decision-making. Efficiency of disease management is mainly evaluated by means of process indicators; the use of outcome indicators is less common. Within a framework, structure, process, and outcome indicators for effectiveness and efficiency are recommended for each type of disease management program.

Conclusions: The link between aims of disease management and evaluated structure, process, and outcome indicators does not exist in a substantial part of published studies on disease management. The added value of this study mainly lies in the development of a framework to guide the choice of indicators for health technology assessment of disease management.

Objectives: The overall objective of this article was to review the theoretical and conceptual dimensions of how the implementation of clinical practice guidelines (CPGs) is likely to affect treatment costs.

Methods: An important limitation of the extant literature on the cost effects of CPGs is that the main focus has been on clinical adaptation. We submit that the process innovation aspects of CPGs require changes in both clinical and organizational dimensions. We identify five organizational factors that are likely to affect the relationship between CPGs and total treatment costs: implementation, coordination, learning, human resources, and information. We review the literature supporting each of these factors.

Results: The net organizational effects of CPGs on costs depends on whether the cost-reducing properties of coordination, learning, and human resource management offset potential cost increases due to implementation and information management.

Conclusions: Studies of the cost effects of clinical practice guidelines should attempt to measure, to the extent possible, the effects of each of these clinical and organizational factors.

Objectives: To assess the cost-effectiveness of brief physiotherapy intervention versus usual physiotherapy management in patients with neck pain of musculoskeletal origin in the community setting.

Methods: A cost-effectiveness analysis was conducted alongside a multicenter pragmatic randomized controlled clinical trial. Individuals 18 years of age and older with neck pain of more than 2 weeks were recruited from physiotherapy departments with referrals from general practitioners (GPs) in the East Yorkshire and North Lincolnshire regions in the United Kingdom. A total of 139 patients were allocated to the brief intervention, and 129 to the usual physiotherapy. Resource use data were prospectively collected on the number of physiotherapy sessions, hospital stay, specialist, and GP visits. Quality-adjusted life years (QALYs) were estimated using EQ-5D data collected at baseline, 3 and 12 months from the start of the treatment. The economic evaluation was conducted from the U.K. National Health System perspective.

Results: On average, brief intervention produced lower costs (£−68; 95 percent confidence interval [CI], £−103 to £−35) and marginally lower QALYs (−0.001; 95 percent CI, −0.030 to 0.028) compared with usual physiotherapy, resulting in an incremental cost per QALY of £68,000 for usual physiotherapy. These results are sensitive to patients' treatment preferences.

Conclusions: Usual physiotherapy may not be good value for money for the average individual in this trial but could be a cost-effective strategy for those who are indifferent toward which treatment they receive.

Objectives: The present study evaluated the cost-effectiveness of a behavioral graded exercise therapy (GET) program compared with usual care (UC) in terms of the performance of daily activities by patients with chronic shoulder complaints in primary care.

Methods: A total of 176 patients were randomly assigned either to GET (n=87) or to UC (n=89). Clinical outcomes (main complaints, shoulder disability [SDQ] and generic health-related quality of life [EQ-5D], and costs [intervention costs, direct health care costs, direct non–health-related costs, and indirect costs]) were assessed during the 12-week treatment period and at 52 weeks of follow-up.

Results: Results showed that GET was more effective than UC in restoring daily activities as assessed by the main complaints instrument after the 12-week treatment period (p=.049; mean difference, 7.5; confidence interval [CI], 0.0–15.0). These effects lasted for at least 52 weeks (p=.025; mean difference 9.2; CI, 1.2–17.3). No statistically significant differences were found on the SDQ or EQ5D. GET significantly reduced direct health care costs (p=.000) and direct non–health care costs (p=.029). Nevertheless, total costs during the 1-year follow-up period were significantly higher (p=.001; GET=€530 versus UC=€377) due to the higher costs of the intervention. Incremental cost-effectiveness ratios for the main complaints (0–100), SDQ (0–100), and EQ-5D (−1.0–1.0) were €17, €74, and €5,278 per unit of improvement, respectively.

Conclusions: GET proved to be more effective in the short- and long-term and reduces direct health care costs and direct non–health care costs but is associated with higher costs of the intervention itself.

Objectives: The aim of this study was to assess the cost-effectiveness of a class-based exercise program supplementing a home-based program when compared with a home-based program alone. In addition, we estimated the probability that the supplementary class program is cost-effective over a range of values of a decision maker's willingness to pay for an additional quality-adjusted life-year (QALY).

Methods: The resource use and effectiveness data were collected as part of the clinical trial detailed elsewhere. Unit costs were estimated from published sources. The net benefit approach to cost-effectiveness analysis is used to estimate the probability of the intervention being cost-effective.

Results: The addition of a supplementary class-based group results in an increase in QALYs and lower costs. For all plausible values of a decision maker's willingness to pay for a QALY, the supplementary class group is likely to be cost-effective.

Conclusions: The addition of a class-based exercise program is likely to be cost-effective and, on current evidence, should be implemented.

Objectives: The aim of this study was to assess the cost-effectiveness of drotrecogin alfa (activated) compared with best supportive care in a UK cohort of adult intensive-care patients with severe sepsis.

Methods: A systematic review of evidence on the clinical- and cost-effectiveness of drotrecogin alfa (activated) was undertaken, and a decision-analytic model was developed to estimate the cost-effectiveness of treatment in the United Kingdom. Trial data from the Recombinant Human Activated Protein C Worldwide Evaluation in Severe Sepsis (PROWESS) study have been synthesized with other data, including UK data on severe sepsis, to estimate the costs and consequences of treatment over time.

Results: For patients with severe sepsis and multiple organ dysfunction, the estimates of cost per life year and cost per quality-adjusted life year (QALY) are £4,931 and £8,228, respectively. For patients with severe sepsis alone, the cost per life-year and cost per QALY are £5,495 and £9,161, respectively.

Conclusions: Whereas the therapeutic cost for drotrecogin alfa (activated) appears high (at around £5,000 per patient) and the potential impact on the provider budget is considerable, drotrecogin alfa (activated) is clinically effective, represents a cost-effective use of resources, and is a significant advance in the treatment of severe sepsis in patients requiring intensive care.

Objectives: The aim of this study was to estimate the expected cost and clinical benefits associated with the use of drotrecogin alfa (activated) (Xigris; Eli Lilly and Company; Indianapolis, IN) in the French hospital setting.

Methods: The recombinant human activated PROtein C Worldwide Evaluation in Severe Sepsis (PROWESS) study results (1,271 patients with multiple organ failure) were adjusted to 9,948 hospital stays from a database of Parisian area intensive-care units (ICUs)—the CubRea (Intensive Care Database User Group) database. The analysis features a decision tree with a probabilistic sensitivity analysis.

Results: The cost per life year gained (LYG) of drotrecogin treatment for severe sepsis with multiple organ failure (European indication) was estimated to be $11,812. At the hospital level, the drug is expected to induce an additional cost of $7,545 per treated patient. The incremental cost-effectiveness ratio ranges from $7,873 per LYG for patients receiving three organ supports during ICU stay to $17,704 per LYG for patients receiving less than two organ supports.

Conclusions: Drotrecogin alfa (activated) is cost-effective in the treatment of severe sepsis with multiple organ failure when added to best standard care. The cost-effectiveness of the drug increases with baseline disease severity, but it remains cost-effective for all patients when used in compliance with the European approved indication.

Background: Endoscopic retrograde cholangiopancreatography (ERCP) is considered the gold standard for imaging of the biliary tract but is associated with complications. Less invasive imaging techniques, such as magnetic resonance cholangiopancreatography (MRCP), have a much lower complication rate. The accuracy of MRCP is comparable to that of ERCP, and MRCP may be more effective and cost-effective, particularly in cases for which the suspected prevalence of disease is low and further intervention can be avoided. A model was constructed to compare the effectiveness and cost-effectiveness of MRCP and ERCP in patients with a previous history of cholecystectomy, presenting with abdominal pain and/or abnormal liver function tests.

Methods: Diagnostic accuracy estimates came from a systematic review of MRCP. A decision analytic model was constructed to represent the diagnostic and treatment pathway of this patient group. The model compared the following two diagnostic strategies: (i) MRCP followed with ERCP if positive, and then management based on ERCP; and (ii) ERCP only. Deterministic and probabilistic analyses were used to assess the likelihood of MRCP being cost-effective. Sensitivity analyses examined the impact of prior probabilities of common bile duct stones (CBDS) and test performance characteristics. The outcomes considered were costs, quality-adjusted life years (QALYs), and cost per additional QALY.

Results: The deterministic analysis indicated that MRCP was dominant over ERCP. At prior probabilities of CBDS, less than 60 percent MRCP was the less costly initial diagnostic test; above this threshold, ERCP was less costly. Similarly, at probabilities of CBDS less than 68 percent, MRCP was also the more effective strategy (generated more QALYs). Above this threshold, ERCP became the more effective strategy. Probabilistic sensitivity analyses indicated that, in this patient group for which there is a low to moderate probability of CBDS, there was a 59 percent likelihood that MRCP was cost-saving, an 83 percent chance that MRCP was more effective with a higher quality adjusted survival, and an 83 percent chance that MRCP had a cost-effectiveness ratio more favorable than $50,000 per QALY gained.

Conclusions: Costs and cost-effectiveness are dependent upon the prior probability of CBDS. However, probabilistic analysis indicated that, with a high degree of certainty, MRCP was the more effective and cost-effective initial test in postcholecystectomy patients with a low to moderate probability of CBDS.

Objectives: This study sought to examine the safety, efficacy, and economic implications of the use of cerebral protection devices during carotid artery angioplasty and stenting (CAS) in high-risk patients with severe carotid artery disease (CAD).

Methods: A comprehensive search for peer- and non–peer-reviewed studies that compared carotid endarterectomy (CEA) or CAS without cerebral protection to CAS with cerebral protection and appeared in the English language literature between January 1990 and January 2005 was completed. Information from studies identified was extracted using a common data abstraction form and then critically appraised against published quality assessment criteria.

Results: Of the eight studies found, six provided information on technical or procedural success rates, with values ranging from 95.6 percent to 100 percent. Three of the four studies comparing groups of patients who received CAS with cerebral protection with those who received only CAS reported a non-statistically significantly higher 30-day incidence of death and stroke (major or minor) in the latter group. None of the three studies comparing CAS with cerebral protection to CEA demonstrated a statistically significant difference in the 30-day incidence of death, major stroke, or myocardial infarction between treatment groups. No economic analyses were found.

Conclusions: In high-risk patients with severe CAD, the evidence suggests that CAS with cerebral protection may offer a safe and efficacious alternative to CEA, reducing the risk of embolic peri-procedural complications associated with CAS to acceptable levels.

Objectives: The objective is to describe the principles for the design of the economic evaluation of COLOR II, a randomized, multi-country study comparing laparoscopic and open surgery for rectal cancer.

Methods: By using the experiences gained in a recent economic evaluation in colon cancer, where the same surgical techniques were compared, we could improve the method for identifying and measuring resource use items and also accommodate the use of data from the global study population.

Results: In the design of the study, the uncertainty in the resource-use variables was reduced by considering (i) what aspects drive each variable, (ii) what resource use is related to the intervention, (iii) how data from different countries affects the variable.

Conclusions: The aim was to refine the data collection so that the economic research question could be answered in the best possible way, given the circumstances in the clinical study. Thus, (i) some variables were treated as stochastic variables and others as deterministic variables, (ii) aggregate key cost-driving resource items were developed that corresponded to clinical events, and (iii) a surrogate variable was selected, instead of the “obvious variable”, to reduce the impact of confounding factors for one particular resource unit.

Evaluations of telemedicine have sought to assess various measures of effectiveness (e.g., diagnostic accuracy), efficiency (e.g., cost), and engagement (e.g., patient satisfaction) to determine its success. Few studies, however, have looked at evaluating the organizational impact of telemedicine, which involves technology and process changes that affect the way that it is used and accepted by patients and clinicians alike. This study reviews and discusses the conceptual issues in telemedicine research and proposes a fresh approach for evaluating telemedicine. First, we advance a patient pathway perspective, as most of the existing studies view telemedicine as a support to a singular rather than multiple aspects of a health care process. Second, to conceptualize patient pathways and understand how telemedicine impacts upon them, we propose simulation as a tool to enhance understanding of the traditional and telemedicine patient pathway.