The Uniform Data System for Medical Rehabilitation (UDSmr) provides a method for uniform assessment of the severity of patient disability and the outcomes of medical rehabilitative care. The effectiveness and efficiency of medical rehabilitation services may be analyzed using the Functional Independence Measure (FIM), the functional assessment component of the UDS, and other data. Program evaluation models based on the UDSMR and the FIM are useful for measuring resource cost of disability.

Objectives: The aim of this study was to estimate the relationship between the financial impact of a new drug and the recommendation for reimbursement by the Australian Pharmaceutical Benefits Advisory Committee (PBAC).

Methods: Data in the PBAC summary database were abstracted for decisions made between July 2005 and November 2009. Financial impact—the upper bound of the values presented in the PBAC summary database—was categorized as ≤A$0, >A$0 up to A$10 million, A$10 million up to A$30 million, and >A$30 million per year. Descriptive, logistic, survival, and recursive partitioning decision analyses were used to estimate the relationship between the financial impact of a new drug indication and the recommendation for reimbursement. Multivariable analyses controlled for other clinical and economic variables, including cost per quality-adjusted life-year gained.

Results: Financial impact was a significant predictor of the recommendation for reimbursement. In the logistic analysis, the odds ratios of reimbursement for drug submissions with financial impacts ≥A$10 million to ≥A$30 million or >A$0 to <A$10 million compared with ≤A$0 were 0.12 (95 percent confidence interval [CI]: 0.03–0.51) and 0.16 (95 percent CI: 0.04–0.60), respectively. In the recursive partition decision analysis, the first split of the data was for submissions with a positive financial impact compared with those with a zero or negative financial impact.

Conclusions: In Australia, financial impact on the drug budget is an important determinant of whether a new drug is recommended for reimbursement when cost-effectiveness estimates and other clinical and economic variables are controlled.

Objectives: Use of complementary and alternative medicine (CAM) is growing in all Western countries. The goal of this study was to evaluate quality of randomized controlled trials (RCTs) of CAM interventions for specific diagnoses to inform clinical decision making.

Methods: MEDLINE and related databases were searched for CAM RCTs. Visual review was done of bibliographies, meta-analyses, and CAM journals. Inclusion criteria for review and scoring were blinded RCT, specified diagnosis and intervention, complete study published between January 1, 1966 and July 31, 1998 in an English-language, peer-reviewed journal. Two reviewers independently scored each study.

Results: More than 5,000 trials were found, but only 258 met all study inclusion criteria. The main cause for rejection (> 90%) was that the study was not an RCT or had no blinding. Mean score across 95 diagnosis/intervention categories was 44.7 (S.D. ± 14.3) on a 100-point scale. Ordinary least-squares regression found date of publication, biostatistician as author or consultant, published in one of five widely read English-language medical journals and diagnosis/intervention category of hypertension/relaxation as significant predictors of higher scores.

Conclusions: The overall quality of evidence for CAM RCTs is poor but improving slowly over time, about the same as that of biomedicine. Thus, most services are provided without good evidence of benefit.

Objectives: This study assesses whether pegylated interferon and ribavirin is cost-effective compared with no antiviral treatment provided in routine clinical practice, for different patient subgroups.

Methods: The cost-effectiveness analysis (CEA) uses a Markov decision model to estimate the lifetime cost per quality-adjusted life-year (QALY) of antiviral treatment compared with no treatment. The model is populated with data on sustained virological responses, costs, and transition probabilities all taken from a large representative sample of UK cases and centers (Trent HCV database).

Results: The CEA found that pegylated interferon and ribavirin was cost-effective for most patient subgroups. The CEA found that for patients with genotype non-1, the intervention led to cost reductions and gains of at least 0.5 QALYs. For genotype 1 cases with mild or moderate disease, and younger cirrhotic patients (aged 40 or less), costs per QALY remained below £20,000 ($40,000 or €29,000). For genotype 1 cases with cirrhosis aged 50, the mean cost per QALY rose to over £60,000 ($120,000 or €87,000).

Conclusions: The study concludes that, based on cost and effectiveness data collected from routine clinical practice, treatment with pegylated interferon and ribavirin is generally cost-effective. The study shows that there are variations according to patient subgroup and for older (aged 50 or over) genotype 1 patients with cirrhosis, antiviral treatment appears less cost-effective.

Objectives: The purpose of this health technology assessment (HTA), commissioned by the German Federal Ministry of Health and Social Security, was to systematically review the evidence for the effectiveness and cost-effectiveness of antiviral treatment (AVT) with interferon (INF) or peginterferon (PegIFN) in combination with ribavirin (RBV) in treatment-naïve patients with chronic hepatitis C (CHC) and to apply these data in the context of the German health-care system.

Methods: We performed a systematic literature search on effectiveness and cost-effectiveness of AVT and summarized results using meta-analysis and evidence tables. We applied the German Hepatitis C Model (GEHMO), a decision-analytic Markov model, to determine long-term clinical effectiveness, costs, and incremental cost-effectiveness ratios (ICER) of the examined treatment strategies. Model parameters were derived from German databases, published international randomized clinical trials (RCT), and a Cochrane Review.

Results: Overall, nine RCTs, two HTA reports, one Cochrane review, two meta-analyses, and seven economic evaluations met the inclusion criteria. These studies indicate that PegIFN+RBV achieved the highest sustained virological response rates (SVR) (54–61 percent), followed by IFN+RBV (38–54 percent) and IFN monotherapy (11–21 percent). Based on our meta-analysis, PegIFN+RBV reduced cases without SVR by 17 percent compared with INF+RBV. International cost-effectiveness studies indicate that INF+RBV is cost-effective when compared with INF monotherapy. For PegIFN+RBV, our decision analysis yielded an ICER of €9,800 per quality-adjusted life-year gained.

Conclusions: This HTA suggests that initial combination therapy prolongs life, improves quality of life, and is cost-effective in patients with CHC. Peginterferon plus ribavirin is the most effective and efficient treatment among the examined options. However, because not all chronic hepatitis C patients will develop progressive liver disease, a thorough assessment of the eligibility and appropriateness of treatment with combination therapy must be performed in each individual patient.

Objectives: A comprehensive and systematic assessment (HTA) of early home-supported discharge by a multidisciplinary team that plans, coordinates, and delivers care at home (EHSD) was undertaken and the results were compared with that of conventional rehabilitation at stroke units.

Methods: A systematic literature search for randomized trials (RCTs) on “early supported discharge” was closed in April 2005. RCTs on EHSD without information on (i) death or institution at follow-up, (ii) change in Barthél Index, (iii) length of hospital stay, (iv) intensity of home rehabilitation, or (v) baseline data are excluded. Seven RCTs on EHSD with 1,108 patients followed 3–12 months after discharge are selected for statistical meta-analysis of outcomes. The costs are calculated as a function of the average number of home training sessions. Economic evaluation is organized as a test of dominance (both better outcomes and lower costs).

Results: The odds ratio (OR) for “Death or institution” is reduced significantly by EHSD: OR = .75 (confidence interval [CI], .46–.95), and number needed to treat (NNT) = 14. Referrals to institution have OR = .45 (CI, .31–.96) and NNT = 20. The reduction of the rate of death is not significant. Length of stay is significantly reduced by 10 days (CI, 2.6–18 days). All outcomes have a nonsignificant positive covariance. The median number of home sessions is eleven, and the average cost per EHSD is 1,340 USD. The “action mechanism” and financial barriers to EHSD are discussed.

Conclusions: EHSD is evidenced as a dominant health intervention. However, financial barriers between municipalities and health authorities have to be overcome. For qualitative reasons, a learning path of implementation is recommended where one stroke unit in a region initiates EHSD for dissemination of new experience to the other stroke units.

Objectives: To estimate willingness to pay (WTP) for effective treatment in a sample of obese individuals and to examine whether WTP is associated with factors reflecting the severity of obesity as well as a number of other variables such as age, sex, education, and income.

Methods: WTP and data on the severity of obesity were collected from the study, Swedish Obese Subjects. Associations between WTP, income, and obesity-related factors were analyzed by linear regression.

Results: The mean age was 47 years (range, 37 through 59 years) and the mean body mass index (BMI) was 39.6 kg/m2 (n = 3,549). Average personal monthly income was SEK 13,000 (≈ US $1,585), average WTP was SEK 26,900 (≈ US $3,280) and the median value was SEK 10,900 (≈ US $1,330). A high WTP was associated with high personal and household income, high weight, high education, female sex, poor perceived health, low current age, and low age at onset of obesity. Over 50% of the patients deemed it necessary to borrow money to cover their WTP. When adding a loan to the regression analysis, the associations between WTP and perceived health, age, and gender disappeared.

Conclusion: Obese patients are willing to pay approximately twice their monthly salary for effective treatment and a higher WTP is associated with higher weight and poorer perceived health.

Objectives: To obtain further information from members of the International Network of Agencies for Health Technology Assessment (INAHTA) on the involvement of consumers in their programs.

Methods: A questionnaire for a survey was developed and sent to member agencies in November 2010. Survey responses were compared with those from an earlier survey conducted in 2005.

Results: Of the thirty-three agencies that provided responses, 67 percent involve consumers in some aspects of their health technology assessment (HTA) programs, compared with 57 percent in 2005. As in the earlier survey, most agencies reporting involvement have contact with consumer or patient organizations and a large minority also involve individual consumers. Summaries of HTA reports that are intended to be easily understood by consumers are prepared by 84 percent of the agencies, and 42 percent involve consumers in dissemination of HTA material. In both areas, there was some increase from the levels previously reported.

Conclusions: The survey results suggest that there is a trend to increased involvement of consumers by the INAHTA agencies in their programs but that the level of involvement remains relatively limited. The manner of consumer participation varies between agencies.

Objectives: High-quality clinical evidence is most often lacking when novel high-risk devices enter the European market. At the same time, a randomized controlled trial (RCT) is often initiated as a requirement for obtaining market access in the US. Should coverage in Europe be postponed until RCT data are available? We studied the premarket clinical evaluation of innovative high-risk medical devices in Europe compared with the US, and with medicines, where appropriate.

Methods: The literature and regulatory documents were checked. Representatives from industry, Competent Authorities, Notified Bodies, Ethics Committees, and HTA agencies were consulted. We also discuss patient safety and the transparency of information.

Results: In contrast to the US, there is no requirement in Europe to demonstrate the clinical efficacy of high-risk devices in the premarket phase. Patients in Europe can thus have earlier access to a potentially lifesaving device, but at the risk of insufficiently documented efficacy and safety. Variations in the stringency of clinical reviews, both at the level of Notified Bodies and Competent Authorities, do not guarantee patient safety. We tried to document the design of premarket trials in Europe and number of patients exposed, but failed as this information is not made public. Furthermore, the Helsinki Declaration is not followed with respect to the registration and publication of premarket trials.

Conclusions: For innovative high-risk devices, new EU legislation should require the premarket demonstration of clinical efficacy and safety, using an RCT if possible, and a transparent clinical review, preferably centralized.

Objectives: Mini-HTA (health technology assessment) is increasingly being applied in Denmark as an input for decisions on the use of health technologies. Mini-HTA is a form or check list with questions concerning the prerequisites for and consequences of health technologies. At the national level, the National Board of Health uses mini-HTA when hospitals apply for permission to introduce new treatments. Mini-HTA is also compulsory in Danish Regions' annual collection of early warnings. At the local level some hospitals have made mini-HTA compulsory when clinical departments apply for funding for new technologies. The objective of this study is to assess the quality of the information included in mini-HTA used at Danish hospitals and to discuss the consequences of this to decision making.

Methods: The quality of mini-HTA is assessed by use of an INATHA checklist for HTA reports. Data consists of reviews of the quality in fifty-two mini-HTAs produced by Danish hospitals in 2008.

Results: The mini-HTAs generally include descriptions of the assessed technology and the comparator, but information about the selection and interpretation of the clinical literature and other data is often missing. The level of evidence for the clinical effects and the main references are generally included. Only 25 percent of the mini-HTAs include a quantitative estimate of the size of the clinical effects. Organizational consequences inside the clinical department is described in 81percent of the cases and 92 percent includes a cost estimate.

Conclusions: The results show that the quality of the information in many cases is insufficient. There is a strong need for quality assurance of mini-HTAs to improve the accuracy of the information, however, without harming the timeliness and the limited use of resources in producing the reports.

Objective: To determine the incremental cost-effectiveness of magnetic resonance imaging (MRI) and computed tomography (CT) in young adults presenting with equivocal neurological signs and symptoms. Designs and methods: A decision analysis of long-term survival using accuracy data from a diagnostic technology assessment of MRI and CT in patients with suspected multiple sclerosis, information from the medical literature, and clinical assumptions. Main results: In the baseline analysis, at 30% likelihood of an underlying neurologic disease, MRI use has an incremental cost of $101,670 for each additional quality-adjusted life-year saved compared with $20,290 for CT use. As the probability of disease increases, further MRI use becomes a cost-effective alternative costing $30,000 for each quality-adjusted life-year saved. If a negative MRI result provides reassurance, the incremental costs of immediate MRI use decreases and falls below $25,000 for each quality-adjusted life-year saved no matter the likelihood of disease. Conclusions: For most individuals with neurological symptoms or signs, CT imaging is cost-effective while MR imaging is not. The cost-effectiveness of MRI use, however, improves as the likelihood of an underlying neurological disease increases. For selected patients who highly value diagnostic information, MRI is a reasonable and cost-effective use of medical resources when even the likelihood of disease is quite low (5%).

Objectives: To compare four contingent valuation elicitation methods as a means to estimate the value of a pneumococcal vaccine in Bangladesh and to test if the elicitation methods are subject to bias and if they produce valid responses.

Methods: Three hundred sixty-one households with at least one child under 5 years of age were recruited in Dhaka, Bangladesh. Subjects were cluster-randomized to various elicitation methods: open-ended, dichotomous choice (at one of two asking prices), payment card (one of two cards with differing ranges). The dichotomous choice method was then followed up with a bidding game methodology, with the dichotomous choice price acting as the starting price for the bidding game. Analysis focused on summary statistics, demand curve estimation and multivariate regression analysis to test for validity and bias.

Results: Thirty-one households refused to participate, leaving a total of 330 participating households (a 91.4 percent response rate). Willingness to pay estimates varied significantly across the methods (p < .001), with average estimates varying between $2.34 and $18 (US). The open-ended elicitation method was found to produce highly inflated values that were insensitive to construct validity tests. The dichotomous choice method produced quantity (demand) estimates rather than value estimates, and there was some evidence of yea saying. The payment card elicitation method was found to be affected by range bias. The bidding game elicitation method was found to be less sensitive to starting point bias and yea-saying.

Conclusions: Different elicitation format do give rise to different demand curves; however, this may be partially due to the fact that they do not measure the same outcome. For example, the dichotomous choice format produces a demand curve, while the payment card, open-ended and bidding game produce inverse demand curves. All formats are prone to multiple biases. When choosing an elicitation format, it is important to first consider the purpose and use of the data. Each elicitation method has strengths and weaknesses and can be used for different purposes in technology assessment.

This article considers the distinction between “explanatory” and “pragmatic” aims in clinical trials—the distinction between testing a biological hypothesis and providing evidence to permit a choice between alternative treatment policies. The choice of treatments to compare, the selection of patients for the trial, the study size, and how the treatment comparison should be made are among the matters discussed. In general, where explanatory and pragmatic aims conflict, the pragmatic aim will often take priority.

Objectives: To determine whether unpublished dissertations are an important source of trials for systematic reviews.

Methods: In a review of infant massage, we identified 17 dissertations. We recorded whether each dissertation was included in the review and, if so, whether it contributed data to any analyses.

Results: Thirteen dissertations were excluded, 11 because of poor reporting of methodology. Three dissertations were also published as journal papers. The data from the remaining dissertation were not included in any analysis. Reviewing data on the Cochrane Library, only one of 878 reviews included data from a dissertation that might have changed a review's conclusions.

Conclusions: Searching for and retrieving unpublished dissertations involves considerable time and effort and appears to influence the conclusions of a review only rarely.

Healthcare technology assessment (HTA) aims to support decisions as to which technologies should be used in which situations to optimize value. Because such decisions will create winners and losers, they are bound to be controversial. HTA, then, faces a dilemma: should it stay away from such controversies, remaining a source of incomplete advice and risking an important kind of marginalization, or should it enter the controversy? The question is a challenging one, because we lack agreement on principles that are fine grained enough to tell us what choices we should make. In this study, we will argue that HTA should take a stand on ethical issues raised by the technology that is being investigated. To do so, we propose adding a form of procedural justice to HTA to arrive at decisions that the public can regard as legitimate and fair. A fair process involves deliberation about the reasons, evidence, and rationales that are considered relevant to meeting population-health needs fairly. One important way to make sure that there is real deliberation about relevant reasons is to include a range of stakeholders in the deliberative process. To illustrate how such deliberation might work, we use the case of cochlear implants for deaf children.

Objectives: Some countries make considerable effort to involve patients and patient groups in their health technology assessment (HTA) processes; others are only just considering or are yet to consider patient involvement in HTA.

Methods: This commentary offers four arguments why patient involvement should be prioritized by those HTA agencies that do not yet involve patients: (1) from a patients’ rights perspective, (2) based on patient and community values, (3) centering on evidentiary contributions, and (4) from a methodological perspective.

Results: The first argument builds on the Alma-Ata Declaration, which holds that patients have a right and duty to have a say in the planning and delivery of their health care, individually and collectively. Where HTA is used to determine access to technologies and services, we argue that patients have a right to be heard. The second argues that decisions about treatments and services need to be aligned with the core values and morals of the patients whom the health system serves. The third argues that patients have unique knowledge and insights about living with a health condition and their needs for services and treatments regarding that condition, which can add to the knowledge base and value of the HTA process. The fourth argues that involvement of patients can facilitate methodological advancement of HTA, in areas such as early scientific advice and managed entry with evidence development.

Conclusions: An HTA process that includes patient perspectives can, therefore, provide added value to patients, policy makers and healthcare professionals alike.

Background: Ethics has been part of health technology assessment (HTA) from its beginning in the 1970s, and is currently part of HTA definitions. Several methods in ethics have been used in HTA. Some approaches have been developed especially for HTA, such as the Socratic approach, which has been used for a wide range of health technologies. The Socratic approach is used in several ways, and there is a need for harmonization to promote its usability and the transferability of its results. Accordingly, the objective of this study was to stimulate experts in ethics and HTA to revise the Socratic approach.

Methods: Based on the current literature and experiences in applying methods in ethics, a panel of ethics experts involved in HTA critically analyzed the limitations of the Socratic approach during a face-to-face workshop. On the basis of this analysis a revision of the Socratic approach was agreed on after deliberation in several rounds through e-mail correspondence.

Results: Several limitations with the Socratic approach are identified and addressed in the revised version which consists of a procedure of six steps, 7 main questions and thirty-three explanatory and guiding questions. The revised approach has a broader scope and provides more guidance than its predecessor. Methods for information retrieval have been elaborated.

Conclusion: The presented revision of the Socratic approach is the result of a joint effort of experts in the field of ethics and HTA. Consensus is reached in the expert panel on an approach that is considered to be more clear, comprehensive, and applicable for addressing ethical issues in HTA.

Background: Although value issues are increasingly addressed in health technology assessment (HTA) reports, HTA is still seen as a scientific endeavor and sometimes contrasted with value judgments, which are considered arbitrary and unscientific. This article aims at illustrating how numerous value judgments are at play in the HTA process, and why it is important to acknowledge and address value judgments.

Methods: A panel of experts involved in HTA, including ethicists, scrutinized the HTA process with regard to implicit value judgments. It was analyzed whether these value judgments undermine the accountability of HTA results. The final results were obtained after several rounds of deliberation.

Results: Value judgments are identified before the assessment when identifying and selecting health technologies to assess, and as part of assessment. They are at play in the processes of deciding on how to select, frame, present, summarize or synthesize information in systematic reviews. Also, in economic analysis, value judgments are ubiquitous. Addressing the ethical, legal, and social issues of a given health technology involves moral, legal, and social value judgments by definition. So do the appraisal and the decision-making process.

Conclusions: HTA by and large is a process of value judgments. However, the preponderance of value judgments does not render HTA biased or flawed. On the contrary they are basic elements of the HTA process. Acknowledging and explicitly addressing value judgments may improve the accountability of HTA.

Objectives: To study the socioeconomic impact of rheumatic illness in Sweden and to discuss the consequences for technology assessment studies.

Methods: A cost-of-illness study based on data from official statistics and treatment studies.

Results: The total socioeconomic cost was 52 billion Swedish kronor (SEK) in 1994. The imbalance between direct (10% of total) and indirect costs (90effectiveness of the healthcare sector, the need for new treatment methods, appropriate information systems, and technology assessment studies as well as the institutional arrangements for rehabilitation and basic medical research.

Conclusions: A discussion of solutions for financial cooperation between county councils and regional social insurance offices should be considered. The new biotechnological pharmaceuticals will increase the cost for drugs in health care about 20 times, but the total socioeconomic cost for society may remain at the same level due to a decrease of inpatient costs and indirect costs for loss of production as well as a decrease of transfer payments from social insurance. It is unavoidable that the new pharmaceuticals require priority discussions and active resource allocation in health care and in other sectors of society.