Optimizing Diabetes Management Using a Low-Calorie Diet in Saudi Arabia: A Cost-Benefit Analysis.

BACKGROUND: Low-calorie diets, high in protein and low in carbohydrates, are commonly recommended for patients with pre-diabetes and type 2 diabetes. The objective of this study was to carry out a cost-benefit analysis (CBA) of a low-calorie versus a standard diet from the perspective of the Saudi Arabian health system. METHODS: The CBA compares costs and benefits of the two diet strategies over a 1-year time horizon. Costs included diet and diabetes treatment-related resources while benefits were measured in terms of the costs of diabetes complications avoided. Data on costs and benefits were collected from published literature and subject matter experts. Incremental costs were estimated as the cost difference between low-calorie and standard diet. Incremental benefits were estimated as cost difference from medical complications when following a low-calorie or standard diet. The incremental absolute cost-benefit ratio was calculated to show the difference between the costs and benefits of the low-calorie diet. Incremental relative cost-benefit ratio was calculated to show the cost per dollar of benefit obtained. Monte Carlo simulation modeled variability in outcomes due to variation in costs and uncertainty of diabetes complications. RESULTS: The 1 year cost of standard diet was US$2515 ± 156 compared to US$2469 ± 107 per patient for a low-calorie diet. Incremental benefit is estimated at US$21,438 ± 7367 per patient. The estimated incremental absolute cost-benefit ratio was US$ - 21,360 establishing that benefits are greater than costs, while the estimated incremental relative cost-benefit ratio is 0.0037, establishing that benefits are 270 times greater than costs. CONCLUSION: The low-calorie diet was the dominant strategy compared to the standard diet in modeled scenarios. These findings highlight the importance of a low-calorie diet as part of diabetes management programs for outpatients with type 2 diabetes.

A Markov-model simulation of IVF programs for PCOS patients indicates that coupling myo-Inositol with rFSH is cost-effective for the Italian Health System.

Accumulating evidence suggests that oral supplementation with myo-Inositol (myo-Ins) is able to reduce the amount of gonadotropins and days of controlled ovarian hyperstimulation (COS) necessary to achieve adequate oocyte maturation in assisted reproduction technology (ART) protocols, particularly in women affected by polycystic ovary syndrome (PCOS). We used computational calculations based on simulation modellings. We simulated in vitro fertilization (IVF) procedures-with or without intracytoplasmic sperm injection (ICSI)-with 100,000 virtual patients, accounting for all the stages of the entire IVF procedure. A Monte Carlo technique was used to account for data uncertainty and to generate the outcome distribution at each stage. We considered virtual patients with PCOS undergoing IVF cycles to achieve pregnancy. Computational data were retrieved from clinical experience and published data. We investigated three parameters related to ART protocols: cost of single procedure; efficacy to achieve ongoing pregnancy at 12 gestational weeks; overall cost per single pregnancy. The administration of oral myo-Ins during COH protocols, compared to the standard COH with recombinant Follicle Stimulating Hormone (rFSH) only, may be considered a potential strategy to reduce costs of ART for the Italian Health System.

The FLURESP European commission project: cost-effectiveness assessment of ten public health measures against influenza in Italy: is there an interest in COVID-19 pandemic?

BACKGROUND: The FLURESP project is a public health research funded by the European Commission, with the objective to design a methodological framework to assess the cost-effectiveness of existing public health measures against human influenza pandemics. A dataset has been specifically collected in the frame of the Italian health system. As most of interventions against human influenza are relavant against other respiratory diseases pandemics, potential interests in COVID-19 are discussed. METHODS: Ten public health measures against human influenza pandemics pandemic were selected to be also relevant to other respiratory virus pandemics such as COVID 19: individual (hand washing, using masks), border control (quarantine, fever screening, border closure), community infection (school closure, class dismissal, social distancing, limitation of public transport), reduction of secondary infections (implementation of antibiotic therapy guidelines), pneumococcal vaccination for at-risk people, development of Intensive Care Unit (ICU) capacity, implementation of life support equipments in ICU, screening interventions, vaccination programs targeting health professional and targeting general population. RESULTS: Using mortality reduction as effectiveness criteria, the most cost-effective strategies are "reduction of secondary infections" and "implementation of life support equipment in ICU". The least cost-effective option whatever the level of pandemic events are screening interventions and mass vaccination. CONCLUSIONS: A number of intervention strategies against human influenza pandemics appears relevant against every respiratory virus, including the COVID-19 event. Measures against pandemics should be considered according to their expected effectiveness but also their costs for the society because they impose substantial burden to the population, confirming the interest of considering cost-effectiveness of public health measures to enlighten decision making.

Assessing the impact of exposome on the course of chronic obstructive pulmonary disease and cystc fibrosis: The REMEDIA European Project Approach.

Because of the direct interaction of lungs with the environment, respiratory diseases are among the leading causes of environment-related deaths in the world. Chronic obstructive pulmonary disease (COPD) and cystic fibrosis (CF) are two highly debilitating diseases that are of particular interest in the context of environmental studies; they both are characterized by a similar progressive loss of lung function with small bronchi alterations, and a high phenotypic variability of unknown origin, which prevents a good therapeutic efficacy. In the last years, there has been an evolution in the apprehension of the study of diseases going from a restricted "one exposure, one disease" approach to a broader concept with other associating factors, the exposome. The overall objective of the REMEDIA project is to extend the understanding of the contribution of the exposome to COPD and CF diseases. To achieve our aim, we will (1) exploit data from existing cohorts and population registries to create a unified global database gathering phenotype and exposome information; (2) develop a flexible individual sensor device combining environmental and biomarker toolkits; (3) use a versatile atmospheric simulation chamber to simulate the health effects of complex exposomes; (4) use machine learning supervised analyses and causal inference models to identify relevant risk factors; and (5) develop econometric and cost-effectiveness models to assess the costs, performance, and cost-effectiveness of a selection of prevention strategies. The results will be used to develop guidelines to better predict disease risks and constitute the elements of the REMEDIA toolbox. The multidisciplinary approach carried out by the REMEDIA European project should represent a major breakthrough in reducing the morbidity and mortality associated with COPD and CF diseases.

Cost-effectiveness modelling of use of urea breath test for the management of Helicobacter pylori-related dyspepsia and peptic ulcer in the UK.

OBJECTIVE: Clinical data comparing diagnostic strategies in the management of Helicobacter pylori-associated diseases are limited. Invasive and noninvasive diagnostic tests for detecting H. pylori infection are used in the clinical care of patients with dyspeptic symptoms. Modelling studies might help to identify the most cost-effective strategies. The objective of the study is to assess the cost-effectiveness of a 'test-and-treat' strategy with the urea breath test (UBT) compared with other strategies, in managing patients with H. pylori-associated dyspepsia and preventing peptic ulcer in the UK. DESIGN: Cost-effectiveness models compared four strategies: 'test-and-treat' with either UBT or faecal antigen test (FAT), 'endoscopy-based strategy' and 'symptomatic treatment'. A probabilistic cost-effectiveness analysis was performed using a simulation model in order to identify probabilities and costs associated with relief of dyspepsia symptoms (over a 4-week time horizon) and with prevention of peptic ulcers (over a 10-year time horizon). Clinical and cost inputs to the model were derived from routine medical practice in the UK. RESULTS: For relief of dyspepsia symptoms, 'test-and-treat' strategies with either UBT (€526/success) and FAT (€518/success) were the most cost-effective strategies compared with 'endoscopy-based strategy' (€1317/success) and 'symptomatic treatment' (€1 029/success). For the prevention of peptic ulcers, 'test-and-treat' strategies with either UBT (€208/ulcer avoided/year) or FAT (€191/ulcer avoided/year) were the most cost-effective strategies compared with 'endoscopy-based strategy' (€717/ulcer avoided/year) and 'symptomatic treatment' (€651/ulcer avoided/year) (1 EUR=0,871487 GBP at the time of the study). CONCLUSION: 'Test-and-treat' strategies with either UBT or FAT are the most cost-effective medical approaches for the management of H. pylori-associated dyspepsia and the prevention of peptic ulcer in the UK. A 'test-and-treat' strategy with UBT has comparable cost-effectiveness outcomes to the current standard of care using FAT in the UK.

Early detection of accelerated aging and cellular decline (AACD): A consensus statement.

The cellular hallmarks of accelerated aging and their clinical expression may be grouped using the terms 'accelerated aging and cellular decline' (AACD) and/or 'age-associated cellular decline'. This construct is designed to capture the biological background predisposing the development of age-related conditions. By classifying risk factors, early indicators, and clinical differentiators of AACD through expert consensus, this study aimed to identify the signs, symptoms, and markers indicative of AACD. In doing so, this work paves the way for future implementation of the AACD concept in the clinical and research settings. An interdisciplinary panel of experts with clinical and research expertise was selected to participate in a virtual workshop to discuss AACD. A modified nominal group technique was used to establish consensus among the group. An extended group of international experts critically reviewed an early draft of the manuscript, and their feedback was then incorporated into the model. Experts identified 13 factors predisposing to or clinically manifesting AACD. Among these, chronic diseases, obesity, and unfavorable genetic background were considered as the most important. There was a consensus that a gradual and nonspecific development often characterizes AACD, making its clinical detection potentially challenging. In addition, signs and symptoms might have multifactorial causes and overlapping origins, such as genetic and epigenetic predispositions. As a result, an initial checklist was outlined, listing clinical factors of special relevance (e.g., fatigue, low quality of sleep, and low mood) to represent early manifestations of the organism's exhaustion, which are also frequently neglected in the clinical setting. Differentiating AACD from other conditions is essential. The use of a combination of biomarkers was proposed as a viable method in a two-step process of differentiation: 1) identification of early AACD clinical indicators, followed by 2) symptom and biomarker confirmation with a focus on system domains (to be potentially targeted by future specific interventions). Although the AACD construct is not yet ready for routine use in clinical practice, its operationalization may support the early identification of age-related conditions (when this might still be amenable to reversion) and also encourage preventative interventions. Further investigation is needed to establish specific biomarkers that confirm independent risk factors for AACD and provide a more definitive structure to the concept of AACD (and age-associated cellular decline).

Helicobacter pylori "Test-and-Treat" strategy with urea breath test: A cost-effective strategy for the management of dyspepsia and the prevention of ulcer and gastric cancer in Spain-Results of the Hp-Breath initiative.

BACKGROUND: Data from clinical trials comparing Helicobacter pylori (H. pylori) management strategies in patients with dyspepsia are limited. Cost-effectiveness simulation models might help to identify the optimal strategy. OBJECTIVE: To assess the cost-effectiveness of the H. pylori "Test and Treat" (T&T) strategy including the use of urea breath test (UBT) vs symptomatic treatment (ST) and vs upper gastrointestinal endoscopy (UGE) as a first procedure in patients with dyspepsia. METHODS: Three main strategies: "T&T" strategy including the use of UBT, "UGE" and "ST" have been compared using cost-effectiveness models developed in accordance with the Spanish medical practice. For the model simulations, a time horizon of 4 weeks was considered for the endpoint "Dyspepsia symptoms relief" and 10 years when using "Peptic ulcer avoided" and "Gastric cancer avoided" endpoints. RESULTS: For the endpoint "Dyspepsia symptoms relief", T&T strategy appears to be the most cost-effective (883€/success) compared to UGE strategy and to ST strategy (respectively 1628€ and 990€/success). For the endpoint "Probability of peptic ulcer", the T&T strategy appears to be the most cost-effective (421€/peptic ulcer avoided/y) compared to UGE strategy and ST strategy (respectively 728€ and 632€/peptic ulcer avoided/y). For the endpoint "Gastric cancer avoided", the T&T strategy appears to be the most cost-effective (524€/gastric cancer avoided/y) compared to UGE strategy and "ST" strategy (respectively 716€ and 696€/gastric cancer avoided/y). CONCLUSIONS: T&T strategy including the use of UBT is the most cost-effective medical approach for management of dyspepsia and for the prevention of ulcer and gastric cancer.

Cost-effectiveness of public health interventions against human influenza pandemics in France: a methodological contribution from the FLURESP European Commission project.

BACKGROUND: The FLURESP project is a public health project funded by the European Commission with the objective to design a methodological approach in order to compare the cost-effectiveness of existing public health measures against human influenza pandemics in four target countries: France, Italy, Poland and Romania. This article presents the results relevant to the French health system using a data set specifically collected for this purpose. METHODS: Eighteen public health interventions against human influenza pandemics were selected. Additionally, two public-health criteria were considered: 'achieving mortality reduction ≥40%' and 'achieving morbidity reduction ≥30%'. Costs and effectiveness data sources include existing reports, publications and expert opinions. Cost distributions were taken into account using a uniform distribution, according to the French health system. RESULTS: Using reduction of mortality as an effectiveness criterion, the most cost-effective options was 'implementation of new equipment of Extracorporeal membrane oxygenation (ECMO) equipment'. Targeting vaccination to health professionals appeared more cost-effective than vaccination programs targeting at risk populations. Concerning antiviral distribution programs, curative programs appeared more cost-effective than preventive programs. Using reduction of morbidity as effectiveness criterion, the most cost-effective option was 'implementation of new equipment ECMO'. Vaccination programs targeting the general population appeared more cost-effective than both vaccination programs of health professionals or at-risk populations. Curative antiviral programs appeared more cost-effective than preventive distribution programs, whatever the pandemic scenario. CONCLUSION: Intervention strategies against human influenza pandemics impose a substantial economic burden, suggesting a need to develop public-health cost-effectiveness assessments across countries.

Ophthalmic Insert versus Eye Drops for Mydriasis in Neonates: A Randomized Clinical Trial.

BACKGROUND: Eye drop treatment routinely used to obtain mydriasis prior to fundoscopy in neonates requires repeated instillations of mydriatic agents that can lead to systemic side effects. OBJECTIVES: The aim of this study was to compare the mydriatic efficacy and tolerance of administration of phenylephrine and tropicamide via the ophthalmic insert Mydriasert® versus standard eye drop treatment in neonates. METHODS: In this prospective, single-blinded, noninferiority study, 80 hospitalized neonates (age < 18 months) requiring bilateral fundus examination were randomized to receive eye drops (repeated instillations of 2.5% phenylephrine and 0.5% tropicamide) or the insert. The primary outcome was mydriasis suitable for fundoscopy 75 min after mydriatic agent introduction (T75). RESULTS: Mydriasis was successfully achieved in both eyes at T75 in 97.5% (n = 39/40) of the insert group patients versus 90% (n = 36/40) receiving eye drops (between-group difference 7.5%, demonstrating noninferiority). Mydriasis remained stable in 60.0% of patients (n = 24/40) in the insert group versus 15% (n = 6/40) in the eye drop group (p < 0.0001). The insert group required fewer nursing interventions than the eye drop group (p = 0.0001). Mean blood pressure were significantly higher in the insert versus the eye drop group (p < 0.0001 and p = 0.0003, respectively); mean heart rate was not significantly different between the groups (p = 0.37). In the insert group, 2 patients reported an adverse event (bradycardia, n = 1, and gastroesophageal reflux, n = 1; both appeared to be related to neonate pathology). CONCLUSIONS: The degree of mydriasis achieved with Mydriasert® was noninferior to that obtained with eye drops. The insert appears to be safe to use in neonates without a history of increased vagal tone or gastrointestinal reflux.

[Value of hospital electronic health records for clinical research: contribution of the European project EHR4CR]. / Intérêt des dossiers de santé électroniques hospitaliers pour la recherche clinique - Le projet européen EHR4CR.

Electronic health records in hospitals contribute to improving the quality of care by enabling better management of clinical information. The databases thus constituted facilitate the exchange of health information with healthcare providers and optimize multidisciplinary coordination for better therapeutic results. The EHR4CR (Electronic Health Records for Clinical Research) European project has developed an innovative pilot platform enabling the reuse of this digital information for clinical research. By enhancing and speeding up clinical research procedures, this innovative approach makes it possible to conduct clinical trials more efficiently, faster, and more economically.

Business analysis for a sustainable, multi-stakeholder ecosystem for leveraging the Electronic Health Records for Clinical Research (EHR4CR) platform in Europe.

INTRODUCTION: The Electronic Health Records for Clinical Research (EHR4CR) technological platform has been developed to enable the trustworthy reuse of hospital electronic health records data for clinical research. The EHR4CR platform can enhance and speed up clinical research scenarios: protocol feasibility assessment, patient identification for recruitment in clinical trials, and clinical data exchange, including for reporting serious adverse events. Our objective was to seed a multi-stakeholder ecosystem to enable the scalable exploitation of the EHR4CR platform in Europe, and to assess its economic sustainability. MATERIALS AND METHODS: Market analyses were conducted by a multidisciplinary task force to define an EHR4CR emerging ecosystem and multi-stakeholder value chain. This involved mapping stakeholder groups and defining their unmet needs, incentives, potential barriers for adopting innovative solutions, roles and interdependencies. A comprehensive business model, value propositions, and sustainability strategies were developed accordingly. Using simulation modelling (including Monte Carlo simulations) and a 5-year horizon, the potential financial outcomes of the business model were forecasted from the perspective of an EHR4CR service provider. RESULTS: A business ecosystem was defined to leverage the EHR4CR multi-stakeholder value chain. Value propositions were developed describing the expected benefits of EHR4CR solutions for all stakeholders. From an EHR4CR service provider's viewpoint, the business model simulation estimated that a profitability ratio of up to 1.8 could be achieved at year 1, with potential for growth in subsequent years depending on projected market uptake. CONCLUSIONS: By enhancing and speeding up existing processes, EHR4CR solutions promise to transform the clinical research landscape. The ecosystem defined provides the organisational framework for optimising the value and benefits for all stakeholders involved, in a sustainable manner. Our study suggests that the exploitation of EHR4CR solutions appears profitable and sustainable in Europe, with a growth potential depending on the rates of market and hospital adoption.

Is there an alternative to quality-adjusted life years for supporting healthcare decision making?

INTRODUCTION: Over the years, a number of criticisms have been raised about the robustness of the Quality-Adjusted Life Years (QALY) indicator and its use in cost-utility analyses; however, costs/QALY are still nowadays recommended as reference case by several Health Technology Assessment (HTA) agencies from some Commonwealth countries, such as the National Institute for Health and Care Excellence (NICE) in the UK, claiming that no alternatives exist to allocate health care resources. AREAS COVERED: This review presents a selection of robust alternative methodologies that could be used to support HTA decisions more accurately and more fairly than using the QALYs, including for determining the level of patient access and reimbursement coverage to healthcare interventions. Expert commentary: Because of the scientific complexity of the situations raised by existing and innovative health technologies and interventions, there is currently no single alternative paradigm to propose at this time, but a spectrum of additional analytical techniques which could handle various outcomes including costs and health consequences, and which are not based on a simple multiplicative formula.

An inception cohort study assessing the role of pneumococcal and other bacterial pathogens in children with influenza and ILI and a clinical decision model for stringent antibiotic use.

BACKGROUND: Influenza-like illness (ILI) is a common reason for paediatric consultations. Viral causes predominate, but antibiotics are used frequently. With regard to influenza, pneumococcal coinfections are considered major contributors to morbidity/mortality. METHODS: In the context of a perennial quality management (QM) programme at the Charité Departments of Paediatrics and Microbiology in collaboration with the Robert Koch Institute, children aged 0-18 years presenting with signs and symptoms of ILI were followed from the time of initial presentation until hospital discharge (Charité Influenza-Like Disease = ChILD Cohort). An independent QM team performed highly standardized clinical assessments using a disease severity score based on World Health Organization criteria for uncomplicated and complicated/progressive disease. Nasopharyngeal and pharyngeal samples were collected for viral reverse transcription polymerase chain reaction and bacterial culture/sensitivity and MaldiTOF analyses. The term 'detection' was used to denote any evidence of viral or bacterial pathogens in the (naso)pharyngeal cavity. With the ChILD Cohort data collected, a standard operating procedure (SOP) was created as a model system to reduce the inappropriate use of antibiotics in children with ILI. Monte Carlo simulations were performed to assess cost-effectiveness. RESULTS: Among 2,569 ChILD Cohort patients enrolled from 12/2010 to 04/2013 (55% male, mean age 3.2 years, range 0-18, 19% >5 years), 411 patients showed laboratory-confirmed influenza, with bacterial co-detection in 35%. Influenza and pneumococcus were detected simultaneously in 12/2,569 patients, with disease severity clearly below average. Pneumococcal vaccination rates were close to 90%. Nonetheless, every fifth patient was already on antibiotics upon presentation; new antibiotic prescriptions were issued in an additional 20%. Simulation of the model SOP in the same dataset revealed that the proposed decision model could have reduced the inappropriate use of antibiotics significantly (P<0.01) with an incremental cost-effectiveness ratio of -99.55€. CONCLUSIONS: Physicians should be made aware that in times of pneumococcal vaccination the prevalence and severity of influenza infections complicated by pneumococci may decline. Microbiological testing in combination with standardized disease severity assessments and review of vaccination records could be cost-effective, as well as promoting stringent use of antibiotics and a personalized approach to managing children with ILI.

Cost-benefit assessment of using electronic health records data for clinical research versus current practices: Contribution of the Electronic Health Records for Clinical Research (EHR4CR) European Project.

INTRODUCTION: The widespread adoption of electronic health records (EHR) provides a new opportunity to improve the efficiency of clinical research. The European EHR4CR (Electronic Health Records for Clinical Research) 4-year project has developed an innovative technological platform to enable the re-use of EHR data for clinical research. The objective of this cost-benefit assessment (CBA) is to assess the value of EHR4CR solutions compared to current practices, from the perspective of sponsors of clinical trials. MATERIALS AND METHODS: A CBA model was developed using an advanced modeling approach. The costs of performing three clinical research scenarios (S) applied to a hypothetical Phase II or III oncology clinical trial workflow (reference case) were estimated under current and EHR4CR conditions, namely protocol feasibility assessment (S1), patient identification for recruitment (S2), and clinical study execution (S3). The potential benefits were calculated considering that the estimated reduction in actual person-time and costs for performing EHR4CR S1, S2, and S3 would accelerate time to market (TTM). Probabilistic sensitivity analyses using Monte Carlo simulations were conducted to manage uncertainty. RESULTS: Should the estimated efficiency gains achieved with the EHR4CR platform translate into faster TTM, the expected benefits for the global pharmaceutical oncology sector were estimated at €161.5m (S1), €45.7m (S2), €204.5m (S1+S2), €1906m (S3), and up to €2121.8m (S1+S2+S3) when the scenarios were used sequentially. CONCLUSIONS: The results suggest that optimizing clinical trial design and execution with the EHR4CR platform would generate substantial added value for pharmaceutical industry, as main sponsors of clinical trials in Europe, and beyond.

Cost-effectiveness modelling of percutaneous coronary interventions in stable coronary artery disease.

The objective of this study is to develop a cost-effectiveness model comparing drug eluting stents (DES) vs bare metal stent (BMS) in patients suffering of stable coronary artery disease. Using a 2-years time horizon, two simulation models have been developed: BMS first line strategy and DES first line strategy. Direct medical costs were estimated considering ambulatory and hospital costs. The effectiveness endpoint was defined as treatment success, which is the absence of major adverse cardiac events. Probabilistic sensitivity analyses were carried out using 10000 Monte-Carlo simulations. DES appeared slightly more efficacious over 2 years (60% of success) when compared to BMS (58% of success). Total costs over 2 years were estimated at 9303 € for the DES and at 8926 € for bare metal stent. Hence, corresponding mean cost-effectiveness ratios showed slightly lower costs (P < 0.05) per success for the BMS strategy (15520 €/success), as compared to the DES strategy (15588 €/success). Incremental cost-effectiveness ratio is 18850 € for one additional percent of success. The sequential strategy including BMS as the first option appears to be slightly less efficacious but more cost-effective compared to the strategy including DES as first option. Future modelling approaches should confirm these results as further comparative data in stable coronary artery disease and long-term evidence become available.

Quality of life assessment in cosmetics: specificity and interest of the international BeautyQol instrument.

The wide use of cosmetics and their perceived benefits upon well-being imply objective descriptions of their effects upon the different dimensions contributing to the quality of life (QoL). Such a goal pleas for using relevant and validated scientific instruments with robust measurement methods. This paper discusses the interest of the new validated questionnaire BeautyQoL specifically designed to assess the effect of cosmetic products on physical appearance and QoL. After conducting a review of skin appearance and QoL, three phases of the international codevelopment have been carried out in the following sequence: semi-directed interviews (Phase 1), acceptability study (Phase 2), and validation study (Phase 3). Data collection and validation process have been carried out in 16 languages. This review confirms that QoL instruments developed in dermatology are not suitable to assess cosmetic products, mainly because of their lack of sensitivity. General acceptability of BeautyQol was very good. Forty-two questions have been structured in five dimensions that explained 76.7% of the total variance: Social Life, Self-confidence, Mood, Vitality, and Attractiveness. Cronbach's alpha coefficients are between 0.932 and 0.978, confirming the good internal consistency of the results. The BeautyQol questionnaire is the first international instrument specific to cosmetic products and physical appearance that has been validated in 16 languages and could be used in a number of clinical trials and descriptive studies to demonstrate the added value of these products on the QoL.

Impact of pigmentary disorders on quality of life in Japan: Interest of the BeautyQoL instrument.

Skin pigmentary disorders and uneven skin tone represent common cosmetic concerns in Japan where fairer skin is culturally desirable. As the demographics of Asian countries continue to evolve, there is a need to understand the impact of cosmetic skin concerns on quality of life (QoL). 199 Japanese women self-claiming facial skin pigmentation disorders were asked to complete the BeautyQoL questionnaire, and the results were compared with those of a control group of 200 women. Of the five dimensions of the BeautyQoL questionnaire, the dimension "mood" appeared to be significantly lower in the group presenting facial dark spots, as compared with the control group (p < 0.05). In the group presenting facial dark spots, the five dimensions and the global score showed that subjects concerned had lower scores than subjects less concerned, even if statistical significance was not reached. This study confirms that common pigmentary disorders such as facial black spots may negatively impact QoL. Further comparative studies with a controlled randomized design would be necessary to confirm these findings.

Economic burden of functional and organic mitral valve regurgitation.

BACKGROUND: Very little is known about the costs of mitral regurgitation (MR) in Europe. AIM: To evaluate the cost of MR from a French National Payer perspective, based on annual costs of surgical and non-surgical patients. METHODS: A 12-month retrospective population-based analysis of patient demographics, outcomes and acute hospital and post-discharge resource utilizations, extracted from the 2009 French Medical Information System. RESULTS: A total of 19,868 patients with MR were identified. Surgical group (n=4099): index hospitalization length of stay (LOS), 17±14.7 days; patients discharged to rehabilitation, 72% (LOS 23±16 days); 12-month rehospitalization rate, 25%; total cost per surgical patient, €24,871±13,940 (ranging from €21,970±11,787 for mitral valve repair [n=2567, 62.6%] to €29,732±15,796 for mitral valve replacement). Non-surgical group (n=15,769): number of hospitalizations over 12 months, 3.1±1.5 (LOS 23.5±20.4 days); admitted to rehabilitation, 24% (LOS 38.8±37.6 days); total cost per patient, €12,177±10,913 (varying between €9957±9080 and €13,538±11,692 for those without and with heart failure [HF], respectively). The total observed cost for 19,868 MR patients over 12 months was €292.8 million: surgical group, €100.8 million; medical group €192.0 million. Patients with MR and HF who were managed medically consumed 45% (€132.3 million) of the overall annual cost of MR. CONCLUSION: The costs of care associated with MR are highly heterogeneous. There are significant differences in costs and resources used between the surgical and medical MR subgroups, with further differences depending on type of surgery and presence of HF.

Validation of the underlying assumptions of the quality-adjusted life-years outcome: results from the ECHOUTCOME European project.

BACKGROUND: Quality-adjusted life-years (QALYs) have been used since the 1980s as a standard health outcome measure for conducting cost-utility analyses, which are often inadequately labeled as 'cost-effectiveness analyses'. This synthetic outcome, which combines the quantity of life lived with its quality expressed as a preference score, is currently recommended as reference case by some health technology assessment (HTA) agencies. While critics of the QALY approach have expressed concerns about equity and ethical issues, surprisingly, very few have tested the basic methodological assumptions supporting the QALY equation so as to establish its scientific validity. OBJECTIVES: The main objective of the ECHOUTCOME European project was to test the validity of the underlying assumptions of the QALY outcome and its relevance in health decision making. METHODS: An experiment has been conducted with 1,361 subjects from Belgium, France, Italy, and the UK. The subjects were asked to express their preferences regarding various hypothetical health states derived from combining different health states with time durations in order to compare observed utility values of the couples (health state, time) and calculated utility values using the QALY formula. RESULTS: Observed and calculated utility values of the couples (health state, time) were significantly different, confirming that preferences expressed by the respondents were not consistent with the QALY theoretical assumptions. CONCLUSIONS: This European study contributes to establishing that the QALY multiplicative model is an invalid measure. This explains why costs/QALY estimates may vary greatly, leading to inconsistent recommendations relevant to providing access to innovative medicines and health technologies. HTA agencies should consider other more robust methodological approaches to guide reimbursement decisions.