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Mental disorders often begin early in life and constitute five of the top ten causes of disability. Their total cost across Europe is estimated at more than 4% of GDP (more than € 600 billion). The last study investigating the cost of mental disorders in France by our group was based on data from 2007 and yielded an estimated indirect and direct cost of € 109 billions. The objective of this study was thus to provide an overall updated cost of mental health in France ten years later and before the COVID-19 pandemic. We estimated the costs related to the direct healthcare and medico-social system, loss of productivity and loss of quality of life. We conducted a literature search to identify direct healthcare, medico-social, indirect (loss of productivity and income compensation) and loss of quality of life during 2018. We included costs related to major psychiatric disorders, including autism and intellectual disability, but excluded the costs related to dementia. Our estimate of the total cost of mental disorders in France, including medical (14%), social (8%), indirect (27%) and loss of quality of life (51%), was € 163 billions in 2018. This total cost includes money spend, forgone earnings and DALYs lost. We found a 50% increase in costs relative to our previous 2007 study. Large-scale cost-effective interventions such as specialized consultations or the development of ambulatory care could help decrease direct healthcare costs related to hospitalization and productivity loss while greatly improving the quality of life of patients., Competing Interests: Conflict of interest All authors (C.L., O.G., L.BS., I.D.Z., M.L., Ad.D.) declare no conflicts of interests., (Copyright © 2023 Elsevier B.V. and ECNP. All rights reserved.)

In the context of health technologies assessment, patient-reported outcome measures (PROMs) have become assessment criteria that are expected by evaluation agencies along with the other usual clinical criteria. PROMs instruments measure all aspects of patient experience in connection with their health: symptoms, activities of daily living (physical function, sleep, etc.), various aspects of health-related quality of life (QoL), compliance, global impression of change in wellbeing. PROMs are useful both as 1) a primary or secondary efficacy endpoints, and 2) a tolerability criterion to supplement vigilance data reported by clinicians. Measurement of PROMs must be subject to methodological rigor that is identical to that of other assessment criteria measured by an observer. Scales must be validated, suitable for the objective, and where possible specific to a disease. In addition to standard measures of quality of life, PROMs are taken into consideration in the assessments performed by the HAS, even if their impact on the conclusions is difficult to isolate, as assessments are multifactorial and take into account all data available with regard to the medical context. The CEPS will indirectly take into account PROMs in the fixing of the price or tariff only if they have contributed to the award of the ASA/ASMR by the ad hoc committee of the HAS. The working group has formulated three recommendations which aim to further the implementation of patient-reported outcome measures: (1) Better information for all parties involved in a dossier for technology assessment, (2) Systematization of the collection of PROMs for evaluation of health products, (3) Improved quality of dossiers thanks to the use of relevant and validated tools., (Copyright © 2022 Société française de pharmacologie et de thérapeutique. Published by Elsevier Masson SAS. All rights reserved.)

Background: this study aimed to estimate the economic impact and health-related quality of life (HRQOL) of patients with spinal muscular atrophy (SMA) in three European countries. It was used a cross-sectional study carried out in France, Germany, and the United Kingdom. Data were collected from July 2015 to November 2015. Healthcare costs (hospitalizations, emergencies, medical tests, drugs used, visits to general practitioners (GPs) and specialists, medical material and healthcare transport), and non-healthcare costs (social services and informal care) were identified and valued. EuroQol instruments, the Zarit interview, and the Barthel Index were also used to reflect the burden and the social impact of the disease beyond the cost of healthcare. Results: we included 86 children with SMA, 26.7% of them had Type I, and 73.3% Type II or III. The annual average cost associated with SMA reaches €54,295 in the UK, €32,042 in France and €51,983 in Germany. The direct non-healthcare costs ranged between 79-86% of the total cost and the informal care costs were the main component of these costs. Additionally, people suffering from this disease have a very low health-related quality of life, and there are large differences between countries. Conclusions: SMA has a high socioeconomic impact in terms of healthcare and social costs. It was also observed that the HRQOL of affected children was extremely reduced. The figures shown in this study may help to design more efficient and equitable policies, with special emphasis on the support provided to the families or on non-healthcare aid.

Background: A utility value is a health-related quality of life metric (HRQoL) metric used in a cost-effectiveness analysis. While utilities as outcomes in the treatment of advanced Parkinson's disease (PD) with deep brain stimulation (DBS) are available, they do not currently exist for PD with early motor complications. The objectives of this study were to predict utilities from observed disease-specific HRQoL data using two mapping algorithms, and investigate their performance in terms of longitudinal changes within and between treatment groups, and distribution by PD severity., Methods: This is a post hoc analysis of data from the EARLYSTIM trial of DBS compared with best medical therapy (BMT) in PD patients with early motor complications We used two published algorithms comprising ordinal and multinomial regression models to map EQ-5D-3L utilities from observed PD-specific 39 item Questionnaire (PDQ-39) scores in EARLYSTIM. Utilities were calculated using the predicted functioning levels of EQ-5D-3L dimensions and the established EQ-5D-3L UK tariffs. Statistical analyses (analysis of variance, two-tailed Student's t-test) were used to test the change from baseline within groups and difference in change from baseline between groups in utilities. Boxplots were developed to investigate the distribution of predicted utilities by PD severity, measured using the Hoehn and Yahr scale., Results: The change from baseline in predicted mean utilities was statistically significant at all visits up to 24 months for the DBS group (p < 0.001) with both algorithms, and statistically significant at 12 months only (p = 0.04) for the BMT group with one algorithm. With both algorithms, the between-groups difference in change from baseline in predicted mean utilities favored DBS at all follow-up visits (p < 0.001). Based on the Hoehn and Yahr scale, predicted utilities deteriorated with increasing disease severity., Conclusions: Among PD patients with early motor complications, utilities predicted by both mapping algorithms using PDQ-39 data demonstrated a statistically and clinically meaningful improvement with DBS compared with BMT. It was not possible to conclude if one algorithm was more responsive than other. In the absence of utilities collected directly from patients, mapping is an acceptable option permitting economic evaluations to be undertaken.

Introduction: The use of two quality-of-life questionnaires in a single clinical trial with an economic component can be challenging due to the associated workload in terms of data collection and an increased risk of missing data. The aim of our study was to determine whether the questionnaire chosen to measure health status, the St. George's Respiratory Questionnaire (SGRQ), could be administered on its own without adding the EuroQol five dimensions questionnaire (EQ-5D) for economic evaluation in emphysema studies., Materials and Methods: Data were prospectively collected during the REVOLENS trial assessing endobronchial coil treatment in severe emphysema. To quantify the association between the two questionnaires, correlations between the EQ-5D and the SGRQ were first tested and the concordance was then studied in order to know whether the two questionnaires were interchangeable. Finally, the Starkie et al algorithm predicting EQ-5D utility values from the SGRQ was used on REVOLENS's individual patient data. The Student's t -test, correlation and concordance between EQ-5D individual value (from the REVOLENS study) and predicted value (from the Starkie et al algorithm) were studied to test this algorithm., Results: Results showed a strong correlation but no concordance between the EQ-5D and the SGRQ, demonstrating that the two questionnaires are not interchangeable. Moreover, the algorithm predicting EQ-5D utilities from the SGRQ did not provide utility values comparable to those observed in the REVOLENS study. Indeed, our study demonstrated a strong correlation between predicted and individual EQ-5D values but no concordance., Conclusion: The use of both the EQ-5D and the SGRQ in a clinical study with an economic component is justified. Based on our results, the SGRQ should not be used to obtain a utility score to calculate the incremental cost-effectiveness ratio and conclude on the efficiency of an intervention in emphysema patients., Competing Interests: Charles Hugo Marquette and Gaëtan Deslée have been involved as investigators in previous studies sponsored by BTG/PneumRx and received travel reimbursements and speaker fees for educational sessions and consulting from BTG/PneumRx. Gaëtan Deslee also reports grants and personal fees from BTG/PneumRx during the conduct of the study, and personal fees from Boehringer, AstraZeneca, Chiesi, Novartis, and Nuvaira, outside the submitted work. Julie Bulsei, Jeanne Pfister, and Isabelle Durand-Zaleski report grants from French Ministry of Health, during the conduct of the study. Sylvie Leroy, Jeanne Marie Perotin, and Coralie Barbe indicated no conflicts of interest in this work., (© 2020 Bulsei et al.)

Objective: To determine the impact of endobronchial coils on health-related quality-of-life (HRQoL). This paper utilizes trial data to identify the predictors of HRQoL in patients with severe emphysema, and subsequently estimates the impact of a new treatment on HRQoL (measured by utilities). These utility estimates are used to generate indicative long-term QALY estimates for a range of clinically plausible scenarios as a precursor to cost-effectiveness analyses., Methods: Patient level HRQoL data from RENEW and the National Emphysema Treatment Trial (NETT) were combined and mapped to generic EuroQol 5-dimension health utility questionnaire (EQ-5D) values using a published algorithm. Multilevel statistical models were developed using treatment, time, response, and baseline characteristics (EQ-5D, age, gender, FEV1, lung RV) to predict EQ-5D over time. Lifetime QALY estimates were generated using published survival data from NETT (assuming no impact of treatment on mortality) and four clinically plausible response profiles. Each response profile was combined with assumptions around treatment impact (constant or time varying)., Results: After controlling for baseline characteristics, both treatment and response had a statistically significant impact (p < .001) on utility (+0.101 and +0.061, respectively). When combined with selected baseline characteristics and time, Coils and Standard of Care (SoC) generated more QALYs than SoC alone in all scenarios, with incremental lifetime benefit ranging from 0.29-0.55 QALYs., Conclusions: Coils and SoC resulted in statistically significant improvements in HRQoL compared to SoC alone in patients with severe emphysema.

Background: Prior clinical trials have suggested that home-based Ig treatment in multifocal motor neuropathy (MMN) and chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and its variant Lewis-Sumner syndrome (LSS) is safe and effective and is less costly than hospital-administered intravenous immunoglobulin (IVIg)., Methods: A French prospective, dual-center, cost minimization analysis was carried out to evaluate IVIg administration (5% concentrated) at home versus in hospital with regard to costs, patients' autonomy, and patients' quality of life. The primary endpoint was the overall cost of treatment, and we adopted the perspective of the payer (French Social Health Insurance)., Results: Twenty-four patients aged 52.3 (12.2) years were analyzed: nine patients with MMN, eight with CIDP, and seven with LSS. IVIg (g/kg) dosage was 1.51 ± 0.43 in hospital and 1.52 ± 0.4 at home. Nine-month total costs per patient extrapolated to 1 year of treatment were €48,189 ± 26,105 versus €91,798 ± 51,125 in the home and hospital groups, respectively ( p  <   .0001). The most frequently reported factors for choosing home treatment were the good tolerance and absence of side effects of IVIg administration, as well as a good understanding of the advantages and drawbacks of home treatment (75% of respondents). The mRankin scores before and after switch to home treatment were 1.61 ± 0.72 and 1.36 ± 0.76, respectively ( p  =   .027)., Discussion: The switch from hospital-based to home-based IVIg treatment for patients with immune neuropathy represents potentially significant savings in the management of the disease.

There is a lack of comparable health-related quality of life (HRQoL) and utility data across all mental disorders and all inpatient and outpatient settings. Our objective was to investigate the HRQoL and utility scores of people with mental disorders in France, treated in outpatient and inpatient settings, and to identify the HRQoL and utility score losses attributable to mental disorders compared to the non-mentally ill general population. A cross-sectional survey was conducted to assess HRQoL (SF-12) and utility scores of patients with mental disorders and followed in four psychiatric sectors in France. Scores were described by demographic and clinical characteristics and were then adjusted on age and gender and compared with those of the non-mentally ill general population. Median HRQoL and utility scores were significantly lower in patients with mental disorders than in the non-mentally ill general population; median differences amounted to 5.4 for the HRQoL physical score, to 11.8 for the HRQoL mental score and to 0.125 for the utility score. Our findings underscore the negative impact of mental disorders on HRQoL in France and provide a baseline to assess the global impact of current and future organizational changes in the mental health care system.

Quality of life (QoL) assessment is increasingly used in mental health. Multiple instruments exist, but the conditions for choosing one instrument over another for purposes of a specific study are not clear. We performed a systematic review to identify the QoL instruments used in mental health. The instruments were systematically described regarding their intrinsic properties (e.g., generic v. disease-specific) and their characteristics of utilization in studies (e.g., study objectives). Using cluster analyses, we investigated the existence of similar instruments with respect to each of these sets of characteristics and studied potential links between instruments' intrinsic properties and their characteristics of utilization. We included 149 studies in which 56 distinct instruments were used. Similarities were found among instruments in terms of their intrinsic properties as well as their characteristics of utilization, leading to the construction of four clusters of instruments in each case. However, no relevant links were identified between instruments' intrinsic properties and their characteristics of utilization, suggesting that the choice of QoL instruments did not depend on their properties. A consensus about common QoL instruments must be reached to facilitate the choice of instruments, the comparison of results and thus to have an impact on clinical and policy decision-making., (© 2013 Published by Elsevier Ireland Ltd.)

Background: Despite the availability of guidelines for the specific treatment of hereditary angioedema (HAE) attacks, HAE morbidity and mortality rates remain substantial. HAE attacks are a major medical issue requiring specific treatment as well as a considerable socio-economic burden. We report a protocol designed to test whether a dedicated call centre is more effective than usual practice in the management of patients experiencing an HAE attack. Methods/design: This prospective, cluster-randomised, single-blind, parallel-group, multicentre trial evaluates the morbidity and consequent socio-economic costs of the management of patients experiencing an HAE attack by a dedicated call centre as compared to usual practice. The trial aims to recruit 200 patients. Patients in the intervention arm are provided with an SOS-HAE card with the call centre's freephone number that they can access in the case of an attack. The centre's mission is to provide recommended expert advice on early home treatment. The centre can route the call to a local emergency medical service with competency in HAE management or even arrange for the drugs needed for the specific treatment of an HAE attack to be sent to the emergency department of the local hospital. The primary outcome measure is the number of hospital admissions for an HAE attack. Each patient will be followed up every 2 months for 2 years. The study has been approved by the ethics committee (Comité de Protection des Personnes d'Ile de France 10; registration number: 2012-A00044-39; date of approval: 19 January 2012). Discussion: The SOS-HAE protocol has been designed to address the handling of attacks experienced by patients with HAE in the home. The proposed trial will determine whether the setting up of a dedicated call centre is more effective than usual practice in terms of reducing morbidity as given by the numbers of hospital admissions. The results are also anticipated to have important implications in terms of socio-economic costs for both healthcare services and patients. Trial Registration: ClinicalTrials.gov NCT01679912 . [ABSTRACT FROM AUTHOR]

Background: With the development of information and communication technologies, telemedicine has been proposed as a way to improve patient management by facilitating access to appropriate diagnosis and treatment. The Paris Ile de France Regional Health Agency is currently funding a comprehensive program of telemedicine experiments. This article describes the protocols for the evaluation of the implementation of telemedicine in the Paris region.Methods/design: Over 2,500 patients have been included in eight studies addressing the use of telemedicine in the context of specific diseases or settings. Two projects are randomized controlled trials, while the six other projects are based on before-after designs (differences in differences studies). Based on the MAST model and the French national framework, we identified endpoints to assess the impact of telemedicine on five dimensions: clinical effectiveness, cost-effectiveness, security of the application, patient satisfaction and quality of life and perception of professionals.Discussion: Telemedicine encompasses a wide range of services and stakeholders, and thus study protocols must be tailored to the specific constraints and interests of the users.Trial Registration: NCT02110433 (03/07/2014), NCT02157740 (05/27/2014), NCT02374697 (02/05/2015), NCT02157727 (05/27/2014), NCT02229279 (08/28/2014), NCT02368769 (02/05/2015), NCT02164747 (NCT02164747), NCT02309905 (11/27/2014). [ABSTRACT FROM AUTHOR]

In the context of health-care costs, containment and economic downturn, there is a question as to whether the high cost of growth hormone (GH) therapy for patients with idiopathic short stature (ISS) is worth the health benefit provided. The economic evaluations of GH for the treatment of patients with ISS have considered gain in height as the major clinical endpoint. The incremental cost of each centimetre of adult height gained due to GH treatment has been estimated to be between £13,500 and £27,200 (€16,400 to €33,100), but could range from £4,300 to £272,000 (€5,200 to €330,900) depending on height gain, GH dose and unit cost, as well as discount rate chosen. Evidence regarding a potential benefit on health-related quality of life is lacking. Copyright © 2011 S. Karger AG, Basel [ABSTRACT FROM AUTHOR]