Outcomes In Economic Evaluations Research Articles

Economic analysis of a cosmetic initiative addressing stochastic aspects and risk quantification

This paper evaluates the economic feasibility of a small/medium-scale cosmetic initiative in a typical Brazilian city. The Brazilian cosmetic sector is very promising for investors, particularly when e-commerce platforms are designed, as virtual sales are the new trend in the sector. Therefore, the required costs to implement an e-commerce platform are considered to guarantee demand for the factory’s productive capacity. While deterministic analysis might provide initial overviews of economic feasibility, there are inherent risks associated with manufacturing. These risks are likely to influence considerably the outcome of economic evaluation, hence, stochastic analysis is performed based on the software Oracle Crystal Ball®. The proposed approach guarantees an accurate and detailed evaluation, as well as solid conclusions regarding the economic feasibility of cosmetic initiatives in Brazil. Results show that such initiatives are highly profitable.

Societal benefits of surrogate outcomes to support reimbursement decisions: The case of prostate cancer.

372 Background: Incorporating surrogate outcomes for survival into economic evaluations for localized prostate cancer (LPC) has the potential to reduce the time needed to subsidise new therapies but may increase uncertainty regarding true benefit. The ICECaP study demonstrated that metastasis-free survival (MFS) is a strong surrogate of overall survival (OS) in LPC. We sought to model the benefits to society of earlier funding decisions, and to understand the context of societal trade-offs between aspects of drug value and time to reimbursement decision-making in LPC. Methods: Societal benefits were modelled using a 4-health state (biochemical recurrence (BCR), BCR-free, metastasis and death) partitioned survival model to evaluate the cost-effectiveness of abiraterone plus prednisolone (AAP) plus standard of care (SoC) compared to SoC alone in LPC. Survival outcomes were dependent on the ICECaP surrogacy relationship. Treatment pathways and costs reflected Australian practice and quality of life was informed by the literature. The model evaluated the difference between deciding to reimburse AAP now, based on MFS, versus wait for OS 3 years. Societal trade-offs were captured using a population based discrete choice experiment exploring the influence on preference for drug reimbursement of cost-effectiveness, cost to Government, adverse events, proportions expected to benefit, nature of benefits, the completeness of data and the time of follow-up. Preferences were analysed using mixed logit analyses. Results: The analysis of societal benefits showed that using MFS as a surrogate for OS could prevent 117 patients from recurrence, 30 from metastases and save 63 lives in the short-term when AAP is compared with SoC for LPC patients in Australia. Delaying the reimbursement decision for 3 years results in a loss of 4.48 years, 0.47 quality adjusted life years, and increase the cost to $11,558 per patient over the lifetime. The analysis of societal trade-offs reflected results from 1,003 community members and showed an inclination to favour reimbursing interventions that benefit a high proportion of patients (OR=2.10 CI 95%=1.91, 2.32), have a lower cost-effectiveness ratio (OR=0.81 CI 95%= 0.78, 0.88) and an aversion for evidence from ongoing studies (OR= 0.83 CI 95%= 0.78, 0.88). Conclusions: Using MFS as surrogate for funding decision in LPC is likely to produce benefits to society in terms of improved short and long-term outcomes. This contrasts with societal preferences to delay reimbursement decisions to increase certainty. This suggests, improved cost-effectiveness could be used to compensate for the uncertainty arising from the use of surrogate outcomes in reimbursement decisions. The use of evidence-based surrogate outcomes in economic evaluations to support reimbursement decisions can reduce the time it takes new interventions to become available improving the benefits to society.

Economic models of community-based falls prevention: a systematic review with subsequent commissioning and methodological recommendations

BackgroundFalls impose significant health and economic burdens among older populations, making their prevention a priority. Health economic models can inform whether the falls prevention intervention represents a cost-effective use of resources and/or meet additional objectives such as reducing social inequities of health. This study aims to conduct a systematic review (SR) of community-based falls prevention economic models to: (i) systematically identify such models; (ii) synthesise and critically appraise modelling methods/results; and (iii) formulate methodological and commissioning recommendations.MethodsThe SR followed PRISMA 2021 guideline, covering the period 2003–2020, 12 academic databases and grey literature. A study was included if it: targeted community-dwelling persons aged 60 and over and/or aged 50–59 at high falls risk; evaluated intervention(s) designed to reduce falls or fall-related injuries; against any comparator(s); reported outcomes of economic evaluation; used decision modelling; and had English full text. Extracted data fields were grouped by: (A) model and evaluation overview; (B) falls epidemiology features; (C) falls prevention intervention features; and (D) evaluation methods and outcomes. A checklist for falls prevention economic evaluations was used to assess reporting/methodological quality. Extracted fields were narratively synthesised and critically appraised to inform methodological and commissioning recommendations. The SR protocol is registered in the Prospective Register of Systematic Reviews (CRD42021232147).ResultsForty-six models were identified. The most prevalent issue according to the checklist was non-incorporation of all-cause care costs. Based on general population, lifetime models conducting cost-utility analyses, seven interventions produced favourable ICERs relative to no intervention under the cost-effectiveness threshold of US$41,900 (£30,000) per QALY gained; of these, results for (1) combined multifactorial and environmental intervention, (2) physical activity promotion for women, and (3) targeted vitamin D supplementation were from validated models. Decision-makers should explore the transferability and reaches of interventions in their local settings. There was some evidence that exercise and home modification exacerbate existing social inequities of health. Sixteen methodological recommendations were formulated.ConclusionThere is significant methodological heterogeneity across falls prevention models. This SR’s appraisals of modelling methods should facilitate the conceptualisation of future falls prevention models. Its synthesis of evaluation outcomes, though limited to published evidence, could inform commissioning.

Estimating and Comparing Health and Financial Risk Protection Outcomes in Economic Evaluations

ObjectivesImproving health and financial risk protection (FRP, the prevention of medical impoverishment) and their distributions is a major objective of national health systems. Explicitly describing FRP and disaggregated (eg, across socioeconomic groups) impact of health interventions in economic evaluations can provide decision makers with a broader set of health and financial outcomes to compare and prioritize interventions against each other. MethodsWe propose methods to synthesize such a broader set of outcomes by estimating and comparing the distributions in both health and FRP benefits procured by health interventions. We build on benefit-cost analysis frameworks and utility-based models, and we illustrate our methods with the case study of universal public finance (financing by government regardless of whom an intervention is targeting) of disease treatment in a low- and middle-income country setting. ResultsTwo key findings seem to emerge: FRP is critical when diseases are less lethal (eg, case fatality rates <1% or so), and quantitative valuation of inequality aversion across income groups matters greatly. We recommend the use of numerous sensitivity analyses and that all distributional health and financial outcomes be first presented in a disaggregated form (before potential subsequent aggregation). ConclusionsEstimation approaches such as the one we propose provide explicit disaggregated considerations of equity, FRP, and poverty impact for the development of health sector policies, with high relevance for population-based preventive measures.

Monetary Valuation of Children's Cognitive Outcomes in Economic Evaluations from a Societal Perspective: A Review.

Cognitive ability in childhood is positively associated with economic productivity in adulthood. Expected gains in economic output from interventions that protect cognitive function can be incorporated in benefit–cost and cost-effectiveness analyses conducted from a societal perspective. This review summarizes estimates from high-income countries of the association of general cognitive ability, standardized as intelligence quotient (IQ), with annual and lifetime earnings among adults. Estimates of the association of adult earnings with cognitive ability assessed in childhood or adolescence vary from 0.5% to 2.5% per IQ point. That range reflects differences in data sources and analytic methods. We take a conservative published estimate of a 1.4% difference in market productivity per IQ point in the United States from a recent study that controlled for confounding by family background and behavioral attributes. Using that estimate and the present value of lifetime earnings calculated using a 3% discount rate, the implied lifetime monetary valuation of an IQ point in the United States is USD 10,600–13,100. Despite uncertainty and the exclusion of non-market productivity, incorporation of such estimates could lead to a fuller assessment of the benefits of public health and clinical interventions that protect the developing brains of fetuses, infants, and young children.

Economic evaluations of psychological treatments for common mental disorders in low- and middle-income countries: protocol for a systematic review

ABSTRACT Background Common mental disorders (CMDs) are highly prevalent conditions that constitute a major public health and economic burden on society in low- and middle-income countries (LMICs). Despite the increased demand for economic evidence to support resource allocation for scaled-up implementation of mental health services in these contexts, economic evaluations of psychological treatments for CMDs remain scarce. Objective The proposed systematic review aims to synthesize findings on methods and outcomes of economic evaluations of psychological treatments for CMDs in LMICs and appraise quality. Methods We will identify, select, and extract data from published economic evaluations of psychological interventions for CMDs conducted in LMICs. We will search bibliographic databases (PubMed, EMBASE, CINAHL, Web of Science, EconLit, PsycINFO, Africa-Wide Information, Cochrane library, Centre for Reviews and Dissemination (CRD), Cost Effectiveness Analysis (CEA) Registry), and the African Journals Online (AJOL) and Google Scholar platforms. Only full economic evaluations (Cost-Effectiveness Analysis (CEA), Cost-Utility Analysis (CUA), Cost-Consequence Analysis (CCA), or Cost-Benefit Analysis (CBA)) of psychological treatments for CMDs (defined as depressive, anxiety, and substance use disorders) conducted in LMICs will be included. There will be no restrictions based on date of publication, perspective, follow-up duration or sample size. Data extraction will be guided by the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. Results The results presented will be examined using a narrative synthesis approach. The quality of included studies will be assessed using the Drummond & Jefferson checklist. Conclusion The fledgling evidence base in this area provides an opportunity to promote improved economic evaluation methods in line with repeated calls for economic evidence alongside effectiveness evidence in these settings. A rigorously developed economic evaluation evidence base will support resource allocation decisions for scaled up implementation of psychological interventions in LMIC settings. Systematic review registration PROSPERO CRD42020185277.

Exploring the efficacy and safety of herbal medicine on Korean obese women with or without metabolic syndrome risk factors: A study protocol for a double-blind, randomized, multi-center, placebo-controlled clinical trial.

The prevalence of obesity among women is increasing. Obesity is associated with various metabolic syndromes; conventional treatments are limited and may induce serious adverse events due to polytherapy regimens. Currently, demands for complementary and alternative medicine that has a proven safety profile for the treatment of obesity with or without metabolic risk factors are increasing.Our team of preclinical experts reported a significant anti-obesity effect of the Korean herbal medicine, Galgeun-tang (GGT). Thus, we designed this trial to explore the effects of GGT among obese women to accumulate optimal clinical evidence.Obesity is not only a component of metabolic syndrome and a factor associated with an increased risk of cardiovascular disease but is also related to insulin resistance. Previous research has confirmed that an increasing body mass index is highly related with increased risk of metabolic syndrome among overweight and obese individuals. The effectiveness of the Korean medicine herbal formula, GGT on obesity has been previously reported. The objective of this study is to assess the efficacy and safety of GGT for weight loss among obese Korean women with or without high risk for metabolic syndrome. This study is a randomized, double-blinded, placebo-controlled, multi-center clinical trial. A total of 160 participants will be randomly distributed in 2 groups, the GGT group or the placebo group in a 1:1 ratio using a web-based randomization system. Each group will be administered GGT or placebo 3 times a day for 12 weeks. The primary endpoint is to assess the change in weight from baseline. The secondary endpoints are the following: the changes in body composition measurements, anthropomorphic measurements, obesity screening Laboratory tests, patient self-reported questionnaires, and economic evaluation outcomes. Adverse events will also be reported. The findings of this study will confirm methodologies regarding the efficacy and safety of GGT for weight loss among obese Korean women with or without metabolic risk factors.

PMU81 WHAT IS THE VALUE OF DIGITAL THERAPEUTICS? A REVIEW OF HEALTH ECONOMIC EVIDENCE

Digital therapeutics (DTx), a subset of digital health, deliver software-generated therapeutic interventions directly to patients to prevent, manage, or treat a disease. While the role of DTx in mainstream healthcare is evolving, there is little information on whether such technologies efficiently use resources or improve patient outcomes. To better understand the methodological challenges in assessing the value of DTx, we reviewed the current landscape of health economic studies on DTx. A targeted review of literature published between January 2011 and December 2019 was conducted using prespecified search terms in multiple scientific literature databases. Studies that employed economic methods in evaluation of DTx were included, such as cost-effectiveness, cost-consequence, or cost-benefit analysis. For each included study, information on costs, quality-adjusted life years (QALYs), perspective, time horizon and method of economic evaluation was assessed. Our initial search identified 243 studies, and 235 were excluded based on a review of their titles or abstracts. Among the economic studies meeting the pre-defined inclusion criteria (n=8), only 2 studies were conducted in the United States. The most common medical condition studied was cardiovascular diseases (n=3), followed by physical activity (n=2), asthma (n=1), diabetes (n=1), and stress urinary incontinence (n=1). Notably, 5 of the 8 studies concluded DTx was cost-effective. This review found that time horizons used in these economic studies were relatively short in duration (1-3 years). Lastly, these analyses did not capture the QALYs or costs associated with program attrition, any harmful results, or the impact of DTx on indirect measures such as employment or caregiver burden. Although clinical evidence for DTx is growing, this study demonstrates health economic evidence associated with DTx is inadequate. Future research should prioritize generation of real-world evidence and facilitate improved measurement of patient-centered and value-based outcomes in economic evaluations of DTx.

Exploring the cost-effectiveness of child dental caries prevention programmes. Are we comparing apples and oranges?

Exploring the cost-effectiveness of child dental caries prevention programmes. Are we comparing apples and oranges?

Is the whole larger than the sum of its parts? Impact of missing data imputation in economic evaluation conducted alongside randomized controlled trials

Outcomes in economic evaluations, such as health utilities and costs, are products of multiple variables, often requiring complete item responses to questionnaires. Therefore, missing data are very common in cost-effectiveness analyses. Multiple imputations (MI) are predominately recommended and could be made either for individual items or at the aggregate level. We, therefore, aimed to assess the precision of both MI approaches (the item imputation vs. aggregate imputation) on the cost-effectiveness results. The original data set came from a cluster-randomized, controlled trial and was used to describe the missing data pattern and compare the differences in the cost-effectiveness results between the two imputation approaches. A simulation study with different missing data scenarios generated based on a complete data set was used to assess the precision of both imputation approaches. For health utility and cost, patients more often had a partial (9% vs. 23%, respectively) rather than complete missing (4% vs. 0%). The imputation approaches differed in the cost-effectiveness results (the item imputation: − 61,079€/QALY vs. the aggregate imputation: 15,399€/QALY). Within the simulation study mean relative bias (< 5% vs. < 10%) and range of bias (< 38% vs. < 83%) to the true incremental cost and incremental QALYs were lower for the item imputation compared to the aggregate imputation. Even when 40% of data were missing, relative bias to true cost-effectiveness curves was less than 16% using the item imputation, but up to 39% for the aggregate imputation. Thus, the imputation strategies could have a significant impact on the cost-effectiveness conclusions when more than 20% of data are missing. The item imputation approach has better precision than the imputation at the aggregate level.

PNS24 NORDIC COLLABORATIONS ON PROCUREMENT AND HEALTH TECHNOLOGY ASSESSMENT: CONCEPTS, COMPONENTS, AND OUTLOOK

Over the past two years, cross-border collaborations in HTA and procurement have been implemented in the Nordics. These are the joint procurement of hospital pharmaceuticals on behalf of the Norwegian, Icelandic and Danish Authorities, and FINOSE, a HTA collaboration network between Fimea (Finland), NoMA (Norway) and TLV (Sweden). The aims of joint procurement are to ensure better security of the supply of medicines, and to optimise pricing opportunities. The FINOSE collaboration aims to ensure earlier access to drugs through cooperation on assessment of relative efficacy and effectiveness outcomes in economic evaluations. These collaborations have the opportunity to provide both financial and administrative benefits to both pharmaceutical companies and appraisers. The FINOSE collaboration can reduce the burden on pharmaceutical companies and their affiliates by limiting the number of submissions, with country-specific components relating to costs in economic models. Individual agencies are still responsible for reimbursement decisions, however it is not yet clear exactly what country-specific documentation will be required alongside the FINOSE submission. The procurement procedure is largely aligned with current tendering processes in Denmark and Norway, with the opportunities for multiple winners and rankings. The joint procurement process can provide some financial stability to manufacturers, with prices being fixed in Euros and converted to local currencies once annually, although revenues in local currencies may vary year-on-year. Additionally, security against weaknesses in the exchange rate of local currencies is provided by the minimum price being the lowest of the conversion or an existing price set locally. Prices will also remain confidential in the cross-border agreements. Looking forwards, Joint Nordic Procurement aims to include novel pharmaceuticals, and FINOSE agencies are keen to explore options for outcomes-based contracting and integrate real-world evidence into appraisals. How Nordic HTA processes and Nordic procurement will align with each other is yet to be seen.

PSU7 THE DUTCH REFERENCE PRICE FOR INPATIENT DAYS SHOULD BE USED WITH SOME CAUTION

The Dutch guideline for economic evaluations in healthcare provides a reference price for an inpatient day. However, this price is based on an unpublished micro-costing study taking into account the costs of only three hospital units, that is neurology-, paediatric- and surgery unit. We aim to construct a reference price based on all hospital units and assess the impact of this modified reference price on the incremental cost utility ratio (ICUR) of minimally invasive pancreatectomy. We used UK tariffs of inpatients days to compare the average price of an inpatient day at the three hospital units examined in the unpublished Dutch micro-costing study with the average price of an inpatient day at all hospital units. The deviation in inpatient day price of the three units relative to all hospital units was used to convert the Dutch reference price to a modified average overall price. The ICUR of minimally invasive pancreatectomy compared with open distal pancreatectomy was re-estimated using the modified reference price and a sensitivity analysis was performed using the bounds of the 95% CI. The average price of the inpatient day of the three units compared to the overall average showed a deviation of +34.09%. Adjustment of the current reference price would change the inpatient day price from €476 to €355 (95%CI: €275-€615). The modified reference price increases the ICUR from -€23,722 to €4,980 per QALY (sensitivity analysis: -€20,969 - €13,015 per QALY). Our study suggests that the current Dutch reference price is an overestimation that can have a substantial impact on outcomes of economic evaluations. In addition, when performing sensitivity analyses the range applied for the cost of the inpatient day should be set rather wide to account for the variation in the price of inpatient days across hospital units.

A Bayesian parametric approach to handle missing longitudinal outcome data in trial-based health economic evaluations.

Trial-based economic evaluations are typically performed on cross-sectional variables, derived from the responses for only the completers in the study, using methods that ignore the complexities of utility and cost data (e.g. skewness and spikes). We present an alternative and more efficient Bayesian parametric approach to handle missing longitudinal outcomes in economic evaluations, while accounting for the complexities of the data. We specify a flexible parametric model for the observed data and partially identify the distribution of the missing data with partial identifying restrictions and sensitivity parameters. We explore alternative non-ignorable missingness scenarios through different priors for the sensitivity parameters, calibrated on the observed data. Our approach is motivated by, and applied to, data from a trial assessing the cost-effectiveness of a new treatment for intellectual disability and challenging behaviour.

Effects of eating breakfast on children and adolescents: A systematic review of potentially relevant outcomes in economic evaluations.

BackgroundBreakfast is often described as the most important meal of the day. Several studies have focused on examining if breakfast habits have any short-term effects on school attendance, academic achievement, and general health in children and adolescents. Informed decisions of whether to promote eating breakfast or not require a more long-term perspective.ObjectiveThe aim of this study was to conduct a systematic review of scientific publications studying the effects identified as potentially relevant for the economic evaluation of eating breakfast in children and adolescents.DesignA systematic literature review was conducted. Studies were identified by searching the electronic databases PubMed, CINAHL, Web of Science, and PsycINFO between January 2000 and October 2017. The inclusion criteria applied were published articles from peer-reviewed journals with full text in English, quantitative studies collecting primary data with school-aged children, and adolescents aged from 6 to 18 years as participants, performed entirely or partly in countries with advanced economies, except Japan and Taiwan.ResultsTwenty-six studies fulfilled the inclusion criteria, and studies that were judged to be of at least moderate quality were included in the analysis. The results of the review of eating breakfast studies showed positive and conclusive effects on cognitive performance, academic achievement, quality of life, well-being and on morbidity risk factors.ConclusionsThe overall assessment of the studies indicated positive effects of eating breakfast. How the identified effects influence societal costs and an individual’s quality-adjusted life years require further research.

VP82 Impact of Evidence Synthesis Methods on Outcome of Economic Evaluation

IntroductionEvidence synthesis (ES) is often required for economic evaluation (EE) of pharmaceuticals. Commonly used methods are based on the assumption of proportional hazards in trial data, using the hazard ratio (HR). Alternative methods for ES are increasingly used in EE, in situations where the pattern of hazards in the trial data indicates that the proportional hazards assumption may be violated. The impact of these methodological choices on model outcomes is explored.MethodsA network of trials of BRAF-targeted treatments for advanced melanoma, derived using a systematic review of the literature, is chosen for the study. Guyot's method is used to create individual-patient Kaplan-Meier (K-M) data from published survival curves. Log-cumulative hazard plots and Schoenfeld residuals are derived to examine patterns in hazards within the trial data. All analyses are conducted in R version 3.5.0©. Three alternative methods for ES are tested: 1) Network meta-analysis (NMA) based on published HRs and the assumption of proportional hazards. 2) NMA using fractional polynomials (FP) based on digitised K-M data, allowing the relaxation of the proportional hazards assumption. 3) NMA using an accelerated failure time (AFT) model based on digitised K-M data, allowing the relaxation of the proportional hazards assumption. The derived estimates of relative efficacy from each method are applied in a partitioned survival cost-effectiveness model programmed in Microsoft Excel™.ResultsThe model outcomes predicted by each method (HR, FP and AFT) are presented and compared. Both deterministic and probabilistic results are presented, alongside a discussion around how the uncertainty in these structural assumptions may be captured in EE.ConclusionsStructural assumptions in ES may lead to differences in model outcomes. The impact of these differences may be important in situations where decision uncertainty is high. Methods should be chosen and justified based on patterns of hazard present in the trial data.

On the use of economic evaluation of new technology in helicopter emergency medical services

When evaluating new technology in helicopter emergency medical services (HEMS), economic evaluation is normally part of the assessment. The outcome of economic evaluation may provide fruitful insight for the decision-makers, but, due to the complexity of the HEMS system, it is challenging to perform such evaluations in this context. A change in the components affects the whole system, and a lack of consideration of system components and their interactions may lead to unintended economic consequences when implementing new technology. Ultimately, this may cause a negative contribution to overall patient safety. We propose a conceptual methodology, using a systems model as part of the economic evaluation. The article is a revised and expanded version of a conference article presented at ESREL 2017, Slovenia.

Discounting in Economic Evaluations.

Appropriate discounting rules in economic evaluations have received considerable attention in the literature and in national guidelines for economic evaluations. Rightfully so, as discounting can be quite influential on the outcomes of economic evaluations. The most prominent controversies regarding discounting involve the basis for and height of the discount rate, whether costs and effects should be discounted at the same rate, and whether discount rates should decline or stay constant over time. Moreover, the choice for discount rules depends on the decision context one adopts as the most relevant. In this article, we review these issues and debates, and describe and discuss the current discounting recommendations of the countries publishing their national guidelines. We finish the article by proposing a research agenda.

The insights of health and welfare professionals on hurdles that impede economic evaluations of welfare interventions

ABSTRACTBackground: Four hurdles associated with economic evaluations in welfare interventions were identified and discussed in a previous published literature review. These hurdles include (i) ‘Ignoring the impact of condition-specific outcomes’, (ii) ‘Ignoring the impact of QoL externalities’, (iii) ‘Calculation of costs from a too narrow perspective’ and (iv) ‘The lack of well-described & standardized interventions’. This study aims to determine how healthcare providers and social workers experience and deal with these hurdles in practice and what solutions or new insights they would suggest.Methods: Twenty-two professionals of welfare interventions carried out in Flanders, were interviewed about the four described hurdles using a semi-structured interview. A thematic framework was developed to enable the qualitative analysis. The analysis of the semi-structured interviews was facilitated through the use of the software program QRS NVivo 10.Results: The interviews revealed a clear need to tackle these hurdles. The interviewees confirmed that further study of condition-specific outcomes in economic evaluations are needed, especially in the field of mental health and stress. The proposed dimensions for the condition-specific questionnaires varied however between the groups of interviewees (i.e. general practitioners vs social workers). With respect to QoL externalities, the interviewees confirmed that welfare interventions have an impact on the social environment of the patient (friends and family). There was however no consensus on how this impact of QoL externalities should be taken into account in welfare interventions. Professionals also suggested that besides health care costs, the impact of welfare interventions on work productivity, the patients’ social life and other items should be incorporated. Standardization appears to be of limited added value for most of the interviewees because they need a certain degree of freedom to interpret the intervention. Furthermore, the target population of the interventions is diverse which requires a tailor-made approach.Conclusion: This qualitative research demonstrated that these hurdles occur in practice. The proposed solutions for these hurdles can contribute to the improvement of the methodological quality of economic evaluations of welfare interventions.

Cost-utility analyses of drug therapies in breast cancer: a systematic review.

The economic evaluation (EE) of health care products has become a necessity. Their quality must be high in order to trust the results and make informed decisions. While cost-utility analyses (CUAs) should be preferred to cost-effectiveness analyses in the oncology area, the quality of breast cancer (BC)-related CUA has been given little attention so far. Thus, firstly, a systematic review of published CUA related to drug therapies for BC, gene expression profiling, and HER2 status testing was performed. Secondly, the quality of selected CUA was assessed and the factors associated with a high-quality CUA identified. The systematic literature search was conducted in PubMed, MEDLINE/EMBASE, and Cochrane to identify published CUA between 2000 and 2014. After screening and data extraction, the quality of each selected CUA was assessed by two independent reviewers, using the checklist proposed by Drummond et al. The analysis of factors associated with a high-quality CUA (defined as a Drummond score ≥7) was performed using a two-step approach. Our systematic review was based on 140 CUAs and showed a wide variety of methodological approaches, including differences in the perspective adopted, the time horizon, measurement of cost and effectiveness, and more specially health-state utility values (HSUVs). The median Drummond score was 7 [range 3-10]. Only one in two of the CUA (n=74) had a Drummond score ≥7, synonymous of "high quality." The statistically significant predictors of a high-quality CUA were article with "gene expression profiling" topic (p=0.001), consulting or pharmaceutical company as main location of first author (p=0.004), and articles with both incremental cost-utility ratio and incremental cost-effectiveness ratio as outcomes of EE (p=0.02). Our systematic review identified only 140 CUAs published over the past 15 years with one in two of high quality. It showed a wide variety of methodological approaches, especially focused on HSUVs. A critical appraisal of utility values is necessary to better understand one of the main difficulties encountered by authors and propose areas for improvement to increase the quality of CUA. Since the last 5years, there is a tendency toward an improvement in the quality of these studies, probably coupled with economic context, a better and widely spreading of recommendations and thus appropriation by medical practitioners. That being said, there is an urgent need for mandatory use of European and international recommendations to ensure quality of such approaches and to allow easy comparison.

Measuring Health and Broader Well-Being Benefits in the Context of Opiate Dependence: The Psychometric Performance of the ICECAP-A and the EQ-5D-5L

BackgroundMeasuring outcomes in economic evaluations of social care interventions is challenging because both health and well-being benefits are evident. The ICEpop CAPability instrument for adults (ICECAP-A) and the five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) are measures potentially suitable for the economic evaluation of treatments for substance use disorders. Evidence for their validity in this context is, however, lacking. ObjectivesTo assess the construct validity of the ICECAP-A and the EQ-5D-5L in terms of convergent and discriminative validity and sensitivity to change on the basis of standard clinical measures (Clinical Outcomes in Routine Evaluation-Outcome Measure, Treatment Outcomes Profile, Interpersonal Support Evaluation List, Leeds Dependence Questionnaire, and Social Satisfaction Questionnaire). MethodsA secondary analysis of pilot trial data for heroin users in opiate substitution treatment was conducted. Baseline convergence with clinical measures was assessed using the Pearson correlation coefficient. Discriminative validity was assessed using one-way analysis of variance and stepwise regressions. Sensitivity to changes in clinical indicators was assessed at 3 and 12 months using the standardized response mean statistic and parametric and nonparametric testing. ResultsBoth measures had the same level of construct validity, except for clinical indicators of well-being, for which the ICECAP-A performed better. The ICECAP-A was sensitive to changes in both health and well-being indicators. The EQ-5D-5L had lower levels of sensitivity to change, and a ceiling effect (27%), particularly evident in the dimensions of self-care (89%), mobility (75%), and usual activities (72%). ConclusionsThe findings support the construct validity of both measures, but the ICECAP-A gives more attention to broader impacts and is more sensitive to change. The ICECAP-A shows promise in evaluating treatments for substance use disorders for which recovery is the desired outcome.