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Rationale: Mechanical thrombectomy (MT) associated with the best medical treatment (BMT) has recently shown efficacy for the management of acute ischemic stroke (AIS) secondary to a large vessel occlusion. However, evidence is lacking regarding the benefit of MT for more distal occlusions. Aim: To evaluate the efficacy in terms of good clinical outcome at 3 months of MT associated with the BMT over the BMT alone in AIS related to a distal occlusion. Methods: The DISCOUNT trial is a multicenter open-label randomized controlled trial involving French University hospitals. Adult patients (⩾18 years) with an AIS involving the anterior or posterior circulation secondary to a distal vessel occlusion within 6 h of symptom onset or within 24 h if no hyperintense signal on fluid attenuation inversion recovery acquisition will be randomized 1:1 to receive either MT associated with the BMT (experimental group) or BMT alone (control group). The number of patients to be included is 488. Study outcomes: The primary outcome is the rate of good clinical outcome at 3 months defined as a modified Rankin scale (mRS) ⩽2 and evaluated by an independent assessor blinded to the intervention arm. Secondary outcomes include recanalization of the occluded vessel within 48 h, angiographic reperfusion in the experimental group, 3-month excellent clinical outcome (mRS ⩽ 1), all adverse events, and death. A cost utility analysis will estimate the incremental cost per quality-adjusted life year (QALY) gained. Discussion: If positive, this study will open new insights in the management of AISs. Trial registration: ClinicalTrials.gov: NCT05030142 registered on 1 September 2021. [ABSTRACT FROM AUTHOR]

Résumé: Objectifs. La supplémentation en fer par voie parentérale est l'option préférée pour traiter efficacement la carence martiale chez les patients présentant une anémie ferriprive. Le fer saccharose et similaires (FS) et le carboxymaltose ferrique (CMF) sont les principales préparations de fer injectable pour le traitement de l'anémie ferriprive. Le risque de réaction anaphylactique associé à ces produits limite l'administration du fer injectable au milieu hospitalier. Les objectifs de l'étude étaient, d'une part, d'évaluer les pratiques d'utilisation du fer par voie parentérale par les médecins anesthésistes chez des patients présentant une carence martiale et une anémie ferriprive en chirurgie orthopédique ou gynécologique dans des établissements de santé français et, d'autre part, d'en déduire l'impact sur les coûts de prise en charge par les établissements. Méthodes. Nous avons utilisé dans cette étude la méthodologie validée des vignettes cliniques représentant quatre situations cliniques fictives pour le FS ou le CMF. Les principaux paramètres d'intérêt étaient : patients d'orthopédie ou de gynécologie, hospitalisation complète/préopératoire ou hospitalisation de jour/postopératoire et documentation de la dose de fer par perfusion, de la dose totale de fer, du nombre de perfusions et de la durée des soins infirmiers associés. Résultats et discussion. Sur 94 médecins participants actifs, 40 indiquent utiliser plus fréquemment le FS dans leur pratique courante et 54 choisissent le CMF. Au vu des simulations, les choix thérapeutiques faits par les médecins ont conduit à 134 cas d'anémie ferriprive traités par FS et 242 cas traités par CMF. Le nombre moyen de perfusions était plus faible avec le CMF (1,3 ± 0,5) qu'avec le FS (2,1 ± 0,7). La durée de soins infirmiers estimée était de 72,5 ± 170,3 minutes et 73,4 ± 186,5 minutes avec le CMF et de 86,1 ± 51,5 minutes et 94,8 ± 65,6 minutes avec le FS pour l'hospitalisation complète et l'hospitalisation de jour respectivement. Conclusion. Par rapport au FS, l'utilisation du CMF pourrait simplifier l'organisation de la prise en charge des patients en raison du nombre plus faible de perfusions, des hospitalisations plus courtes et de la moindre mobilisation des professionnels de santé. En considérant les coûts de transport et les coûts administratifs, le coût total du traitement de l'anémie ferriprive par le CMF pourrait être inférieur à celui du FS. What is known? Parenteral iron-replacement therapy (IRT) is the preferred option to treat efficiently iron deficiency (ID) in ID anemia (IDA) patients. Iron sucrose (IS/ISS) and ferric carboxy maltose (FCM) are the main available injectable iron preparations for the treatment of IDA. Due to reports of anaphylactic reactions, the prescription and the administration of injectable irons is now restricted to the hospital to ensure safety monitoring. Study objectives were, on the one hand, to assess the use of parenteral IRT by anesthesiologists in orthopedic or gynecological surgery patients with ID/IDA, and, on the other hand, to deduce the impact of the costs of management in the French hospital setting. Methods. We used the validated methodology of case-vignettes with four different fictive clinical cases built by randomization of clinical parameters. Key parameters of interest were: context of orthopedics or gynecology and patient situation, either inpatient stay/pre-surgery or outpatient stay/post-surgery. The selection of either IS/ISS or FCM was left to participant's decision and the iron administration was documented: iron dose per infusion, the total iron dose, number of infusions and nursing time required for each infusion. Results. Of 94 active participating physicians, 40 use IS/ISS more frequently in their routine practice and 54 choose FCM. In view of the simulations, the therapeutic choices made by the doctors led to 134 cases of IDA treated with IS/ISS and 242 cases treated with FCM. Mean number of infusions was lower with FCM (1.3 ± 0.5) than with IS/ISS (2.1 ± 0.7). The estimated nursing time spent by infusion: 72.5 ± 170.3 minutes for an inpatient stay and 73.4 ± 186.5 minutes for an outpatient stay with FCM and 86.1 ± 51.5 minutes and 94.8 ± 65.6 minutes with IS/ISS for inpatient and outpatient stay, respectively. Conclusion. Compared to IS/ISS, the use of FCM could result in an organizational simplification patient management due to reduced number of infusions, hospitalization duration and health professionals mobilization. Considering transportation costs and administrative costs, the total cost of treating IDA with FCM might be lower than that of IS/ISS. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

Background: Thiopurine S-methyltransferase (TPMT) testing, either by genotyping or phenotyping, can reduce the incidence of adverse severe myelotoxicity episodes induced by azathioprine. The comparative cost-effectiveness of TPMT genotyping and phenotyping are not known. Objective: Our aim was to assess the cost-effectiveness of phenotyping-based dosing of TPMT activity, genotyping-based screening and no screening (reference) for patients treated with azathioprine. Methods: A decision tree was built to compare the conventional weight-based dosing strategy with phenotyping and with genotyping using a micro-simulation model of patients with inflammatory bowel disease from the perspective of the French health care system. The time horizon was set up as 1 year. Only direct medical costs were used. Data used were obtained from previous reports, except for screening test and admission costs, which were from real cases. The main outcome was the cost-effectiveness ratios, with an effectiveness criterion of one averted severe myelotoxicity episode. Results: The total expected cost of the no screening strategy was €409/patient, the total expected cost of the phenotyping strategy was €427/patient, and the total expected cost of the genotyping strategy was €476/patient. The incremental cost-effectiveness ratio was €2602/severe myelotoxicity averted in using the phenotyping strategy, and €11,244/severe myelotoxicity averted in the genotyping strategy compared to the no screening strategy. At prevalence rates of severe myelotoxicity > 1%, phenotyping dominated genotyping and conventional strategies. Conclusion: The phenotype-based strategy to screen for TPMT deficiency dominates (cheaper and more effective) the genotype-based screening strategy in France. Phenotype-based screening dominates no screening in populations with a prevalence of severe myelosuppression due to azathioprine of > 1%. [ABSTRACT FROM AUTHOR]

Severe combined immunodeficiency (SCID) refers to a group of genetic disorders characterized by greatly compromised cellular and humoral immunity. Children with SCID are asymptomatic at birth, but they die from infections within the first months of life if not treated. Quantification of T-cell receptor excision circles is an extremely sensitive screening method for detecting newborns who may have SCID.The goal of the DEPISTREC study was to evaluate the feasibility of nationwide newborn screening for severe T-cell lymphopenia in France as well as its economic and clinical utility. The test universally used for neonatal screening for SCID was the quantification of TRECs on Guthrie cards. We compared a group of 190,517 babies from 48 maternities across the country who underwent newborn SCID screening with a control group of 1.4 million babies out of whom 28 were diagnosed with SCID without such screening during the course of the study. Within the screening group, 62 babies were found to be lymphopenic, including three with SCID. The cost of screening ranged from 4.7€ to €8.15 per newborn. The average 18-month cost was €257,574 vs €204,697 in the control group. In this large-scale study, we demonstrate that routine SCID screening is feasible and effective. This screening offers the additional benefit of aiding in the diagnosis of non-SCID lymphopenia. Economic evaluation allowed us to calculate the cost per test. Newborn screening may also prevent death by SCID before any curative treatment can be administered. The difference in cost between screened and control children could not be ascertained because of the very low numbers and death of one of the children tested. [ABSTRACT FROM AUTHOR]

Background: Territorial differences in the access to innovative anticancer drugs have been reported from many countries. The objectives of this study were to evaluate access to innovative treatments for metastatic lung cancer in France, and to assess whether socioeconomic indicators were predictors of access at the level of the municipality of residence.Methods: All incident cases of metastatic lung cancer hospitalised for a chemotherapy in public hospitals in 2011 were identified from the French National Hospital discharge database. Information on prescription of innovative drugs from an associated database (FICHCOMP) was crossed with the population density of the municipality and a social deprivation index based on national census data.Results: Overall, 21,974 incident cases of metastatic lung cancer were identified, all of whom were followed for 2 years. Of the 11,486 analysable patients receiving chemotherapy in the public sector, 6959 were treated with a FICHCOMP drug at least once, principally pemetrexed. In multivariate analysis, prescription of FICHCOMP drugs was less frequent in patients ≥66 years compared to those ≤55 years (odds ratio: 0.49 [0.44-0.55]), in men compared to women (0.86 [0.79-0.94]) and in patients with renal insufficiency (0.55 [0.41-0.73]) and other comorbidities. Prescription rates were also associated with social deprivation, being lowest in the most deprived municipalities compared to the most privileged municipalities (odds ratio: 0.82 [0.72-0.92]). No association was observed between the population density of the municipality and access to innovative drugs.Conclusion: Although access to innovative medication in France seems to be relatively equitable, social deprivation is associated with poorer access. The reasons for this need to be investigated and addressed. [ABSTRACT FROM AUTHOR]

Purpose: Microbiological diagnosis (MD) of infections remains insufficient. The resulting empirical antimicrobial therapy leads to multidrug resistance and inappropriate treatments. We therefore evaluated the cost-effectiveness of direct molecular detection of pathogens in blood for patients with severe sepsis (SES), febrile neutropenia (FN) and suspected infective endocarditis (SIE).Methods: Patients were enrolled in a multicentre, open-label, cluster-randomised crossover trial conducted during two consecutive periods, randomly assigned as control period (CP; standard diagnostic workup) or intervention period (IP; additional testing with LightCycler®SeptiFast). Multilevel models used to account for clustering were stratified by clinical setting (SES, FN, SIE).Results: A total of 1416 patients (907 SES, 440 FN, 69 SIE) were evaluated for the primary endpoint (rate of blood MD). For SES patients, the MD rate was higher during IP than during CP [42.6% (198/465) vs. 28.1% (125/442), odds ratio (OR) 1.89, 95% confidence interval (CI) 1.43-2.50; P < 0.001], with an absolute increase of 14.5% (95% CI 8.4-20.7). A trend towards an association was observed for SIE [35.4% (17/48) vs. 9.5% (2/21); OR 6.22 (0.98-39.6)], but not for FN [32.1% (70/218) vs. 30.2% (67/222), P = 0.66]. Overall, turn-around time was shorter during IP than during CP (22.9 vs. 49.5 h, P < 0.001) and hospital costs were similar (median, mean ± SD: IP €14,826, €18,118 ± 17,775; CP €17,828, €18,653 ± 15,966). Bootstrap analysis of the incremental cost-effectiveness ratio showed weak dominance of intervention in SES patients.Conclusion: Addition of molecular detection to standard care improves MD and thus efficiency of healthcare resource usage in patients with SES. ClinicalTrials.gov registration number: NCT00709358. [ABSTRACT FROM AUTHOR]

Context: In France more than 70,000 deaths from diseases related to smoking are recorded each year, and since 2005 prevalence of tobacco has increased. Providing free access to smoking cessation treatment would reduce this burden. The aim of our study was to estimate the incremental cost-effectiveness ratios (ICER) of providing free access to cessation treatment taking into account the cost offsets associated with the reduction of the three main diseases related to smoking: lung cancer, chronic obstructive pulmonary disease (COPD) and cardiovascular disease (CVD). To measure the financial impact of such a measure we also conducted a probabilistic budget impact analysis. Methods and Findings: We performed a cost-effectiveness analysis using a Markov state-transition model that compared free access to cessation treatment to the existing coverage of €50 provided by the French statutory health insurance, taking into account the cost offsets among current French smokers aged 15–75 years. Our results were expressed by the incremental cost-effectiveness ratio in 2009 Euros per life year gained (LYG) at the lifetime horizon. We estimated a base case scenario and carried out a Monte Carlo sensitivity analysis to account for uncertainty. Assuming a participation rate of 7.3%, the ICER value for free access to cessation treatment was €3,868 per LYG in the base case. The variation of parameters provided a range of ICER values from -€736 to €15,715 per LYG. In 99% of cases, the ICER for full coverage was lower than €11,187 per LYG. The probabilistic budget impact analysis showed that the potential cost saving for lung cancer, COPD and CVD ranges from €15 million to €215 million at the five-year horizon for an initial cessation treatment cost of €125 million to €421 million. Conclusion: The results suggest that providing medical support to smokers in their attempts to quit is very cost-effective and may even result in cost savings. [ABSTRACT FROM AUTHOR]

Atherothrombotic disease is a growing health problem, and is increasingly more costly to manage. Clopidogrel is an advanced, specific adenosine diphosphate receptor antagonist, which has been shown to be a highly potent antiplatelet agent. Data from the Clopidogrel versus Aspirin in Patients at Risk of Ischaemic Events (CAPRIE) study have demonstrated the significantly superior clinical benefit of clopidogrel over aspirin for secondary prevention of atherothrombotic disease, with a relative risk reduction in myocardial infarction, stroke or vascular death of 8.7% (95% confidence interval 0.3, 16.5; P = 0.043). Moreover, clopidogrel demonstrated an amplified clinical benefit versus aspirin in patients at high risk of atherothrombotic events, such as those with a previous history of symptomatic atherothrombotic disease or with major risk factors such as diabetes mellitus or hypercholesterolaemia. On the basis of commonly accepted threshold criteria (e20 000 per life-year gained; LYG), clopidogrel in comparison with aspirin is cost-effective for the secondary prevention of atherothrombotic disease (cost per LYG ranging from e19 462 to e3256). Economic analyses have demonstrated consistent cost-effectiveness results with clopidogrel in different countries. Moreover, in high-risk patient subgroups the cost-effectiveness of clopidogrel in comparison with aspirin was even better (cost per LYG ranging from e5900 to e6310). Compared with other treatment strategies used for the prevention of ischaemic or atherothrombotic events, the cost-effectiveness of clopidogrel in comparison with aspirin based on CAPRIE is favourable, with most analyses in the intermediate range of cost-effectiveness. The available data thus support the use of clopidogrel as a clinically efficient and cost-effective option for secondary prevention of atherothrombotic disease, particularly in high-risk patients. [ABSTRACT FROM AUTHOR]

Presents a study to compare the costs and benefits of a methicillin-resistant Staphylococcus aureus (MRSA) infection control program in an endemic setting. Context; Design and setting; Patients; Main outcome measures; Results; Conclusions.

Abstract: We prospectively identified 102 mechanically restrained hospital patients and determined their hospital course. The cohort was elderly, cognitively impaired with multiple chronic diseases. The in-hospital mortality was 21 percent. Nosocomial infection developed in 12 percent and new pressure sores in 22 percent. Prolonged use of restraints (>4 days) was the strongest independent predictor of nosocomial infection (relative risk 1.8, 95% CI = 1.2, 2.8) and new pressure sores (RR 1.4, 95% CI = 1.1, 1.8) as determined by multiple logistic regression analysis. Patients placed in mechanical restraints for longer than four days experience frequent morbid events and should be monitored carefully. [ABSTRACT FROM AUTHOR]

Examines the design of a request form to reduce the ordering of serum tumor marker tests. Matrix of boxes, with individual markers and body organs as rows and columns; Published data and local consensus as basis; 25 percent decrease in ordering of tumor markers; Low-cost intervention that combines economic and clinical requirements.

Examines the economic implications of hepatic arterial infusion chemotherapy in the treatment of nonresectable colorectal liver metastases. Comparison of hepatic arterial infusion and intravenous chemotherapy; Health-care costs; Mean gain in life expectancy from hepatic arterial infusion.

Describes and makes a preliminary assessment of cost containment programs introduced in France from 1994 onwards, which combines mandatory practice guidelines on procedures and drug prescribing with a system of fines for doctors who do not comply. Background; Medical practice guidelines; Evaluation; Issues for debate.

The article discusses the significance of the cost-of-illness studies to the Americans. The author claims that a cost-of-illness study provides information at the micro-and macroeconomic levels, to help estimate appropriate funding for health policies. Its audiences are public or private institutions that decide to help in treating specific disorders. However, cost-of-illness studies are still relatively scarce compared with economic evaluations in general and epidemiological studies.

The authors reflect on the monitoring of intensive care unit (ICU) performances on both clinical and economic dimensions. They assert that in assessment of the quality of care and performances in ICU, methods should combine indicators of both clinical outcomes and resource utilization and must be constructed and presented in a way that provides enough information to guide managers toward specific approaches to performance improvement.

Abstract: The cost of radiotherapy, which concerns 150,000 new patients yearly in France has been estimated 1700 euros per patients in this country, 50% lower than the European average. Economic evaluations of radiotherapy have related the cost of either curative or palliative treatment to the medical outcome. For lung, rectal, breast cancer and bone metastases of prostate cancer, radiation therapy prolongs life by an estimated 6 to 10 months. The cost effectiveness ranges from 5,000 to 25,000 euros per life year gained. [Copyright &y& Elsevier]

Aims: Evaluating the benefit of telemonitoring in heart failure (HF) management in real‐world settings is crucial for optimizing the healthcare pathway. The aim of this study was to assess the association between a 6‐month application of the telemonitoring solution Chronic Care Connect™ (CCC) and mortality, HF hospitalizations, and associated costs compared with standard of care (SOC) in patients with a diagnosis of HF. Methods and results: From February 2018 to March 2020, a retrospective cohort study was conducted using the largest healthcare insurance system claims database in France (Système National des Données de Santé) linked to the CCC telemonitoring database of adult patients with an ICD‐10‐coded diagnosis of HF. Patients from the telemonitoring group were matched with up to two patients from the SOC group based on their high‐dimensional propensity score, without replacement, using the nearest‐neighbour method. A total of 1358 telemonitored patients were matched to 2456 SOC patients. The cohorts consisted of high‐risk patients with median times from last HF hospitalization to index date of 17.0 (interquartile range: 7.0–66.0) days for the telemonitoring group and 27.0 (15.0–70.0) days for the SOC group. After 6 months, telemonitoring was associated with mortality risk reduction (hazard ratio [HR] 0.71, 95% confidence interval [CI] 0.56–0.89), a higher risk of first HF hospitalization (HR 1.81, 95% CI 1.55–2.13), and higher HF healthcare costs (relative cost 1.38, 95% CI 1.26–1.51). Compared with the SOC group, the telemonitoring group experienced a shorter average length of overnight HF hospitalization and fewer emergency visits preceding HF hospitalizations. Conclusion: The results of this nationwide cohort study highlight a valuable role for telemonitoring solutions such as CCC in the management of high‐risk HF patients. However, for telemonitoring solutions based on weight and symptoms, consideration should be given to implement additional methods of assessment to recognize imminent worsening of HF, such as impedance changes, as a way to reduce mortality risk and the need for HF hospitalizations. Further studies are warranted to refine selection of patients who could benefit from a telemonitoring system and to confirm long‐term benefits in high‐risk and stable HF patients. [ABSTRACT FROM AUTHOR]

Background: Macular edema (ME) results from hyperpermeability of retinal vessels, leading to chronic extravasation of plasma components into the retina and hence potentially severe visual acuity loss. Current standard of care consists in using intravitreal injections (IVI), which results in a significant medical and economic burden. During diabetic retinopathy (DR) or retinal vein occlusion (RVO), it has recently been shown that focal vascular anomalies (capillary macro-aneurysms, also termed TelCaps) for telangiectatic capillaries may play a central role in the onset, early recurrence, and/or persistence of ME. Since targeted photocoagulation of TelCaps may improve vision, identification, and photocoagulation of TelCaps, it may represent a way to improve management of ME. Objective: The Targeted Laser in (Diabetic) Macular Edema (TalaDME) study aims to evaluate whether ICG-guided targeted laser (IGTL), in association with standard of care by IVI, allows reducing the number of injections during the first year of treatment compared with IVI only, while remaining non-inferior for visual acuity. Methods: TalaDME is a French, multicentric, two-arms, randomized, sham laser-controlled, double-masked trial evaluating the effect of photocoagulation of TelCaps combined to IVI in patients with ME associated with TelCaps. Patients with vision loss related to center involved ME secondary to RVO or DR and presenting TelCaps are eligible. Two hundred and seventy eyes of 270 patients are randomized in a 1:1 ratio to standard care, i.e., IVI of anti-VEGF solely (control group) or combined with IGTL therapy (experimental group). Stratification is done on the cause of ME (i.e., RVO versus diabetes). Anti-VEGF IVI are administered to both groups monthly for 3 months (loading dose) and then with a pro re nata regimen with a monthly follow-up for 12 months. The primary endpoint will be the number of IVI and the change in visual acuity from baseline to 12 months. Secondary endpoints will be the changes in central macular thickness, impact on quality of life, cost of treatment, and incremental cost-utility ratio in each groups. Key safety: Rare but severe AE linked to the use of IVI and laser, and previously described, are expected. In the sham group, rescue laser photocoagulation may be administered by the unmasked investigator if deemed necessary at month 3. Discussion: The best management of ME associated with TelCaps is debated, and there have been no randomized study designed to answer this question. Given the fact that TelCaps may affect 30 to 60% of patients with chronic ME due to DR or RVO, a large number of patients could benefit from a specific management of TelCaps. TalaDME aims to establish the clinical and medico-economic benefits of additional targeted laser. The results of TalaDME may raise new recommendations for managing ME and impact healthcare costs. Trial registration: EudraCT: 2018-A00800-55/ NCT03751501. Registration date: Nov. 23, 2018. [ABSTRACT FROM AUTHOR]

Given the treatment landscape evolution for aRCC, real-world data are needed. All French patients newly treated for aRCC initiated in 2016 were included. In first-line, patients were mainly treated with sunitinib, and in secondline half were treated with nivolumab. The highest OS was observed for patients treated with sunitinib in firstline and for patients treated with nivolumab in second-line. Background: Treatment landscape for advanced renal cell carcinoma (aRCC) has evolved quickly and few data about the real-world treatment patterns are available. This study aimed at describing the real-world treatment patterns and effectiveness of all systemic treatments available for aRCC in first and second-line treatment. Materials and Methods: A cohort of patients initiating a first-line systemic treatment for aRCC in 2016 was extracted from the French nationwide healthcare insurance system database (SNDS). The first-line treatment initiation date constituted the index date and patients were followed until death, loss to follow-up, or December 31, 2019, whichever occurred first. aRCC was identified using hospital diagnosis, long-term disease, or renal biopsy before index date. All analyses were performed for first and second-line treatment. Overall survival (OS) and time-to-next treatment or death (TNT-D) were estimated using Kaplan-Meier approach. Results: In 2016, 1629 patients initiated a first-line treatment for aRCC. Most of them were male (75.9%) and the median age was 67 years. Most of patients (91.7%) had received a tyrosine kinase inhibitor as first-line treatment, mainly sunitinib (64.4%), and 53.5% received a second-line, among which 43.7% nivolumab. Median OS (95% confidence interval [CI]) was 20.7 (95% CI:18.2-22.4) months from first-line treatment initiation and 15.4 (13.9-17.5) months from second-line treatment initiation. Median TNT-D were respectively 9.3 (9.7-12.1) months and 6.9 (5.9-7.7) months. Conclusion: This study highlights the limited survival of aRCC patients These results provide a valuable baseline and highlight the need for innovation, such as immune checkpoint inhibitor-based combinations that have recently became first-line standard of care. [ABSTRACT FROM AUTHOR]

The NICO (non-invasive airway management in comatose patients) randomized controlled trial found that withholding intubation in acute poisoned comatose patients with a Glasgow Coma Scale < 9 led to significant clinical improvement in terms of in-hospital death, length of stay in the intensive care unit (ICU), and length of hospital stay compared to routine practice. The trial also conducted an economic evaluation, which showed that withholding intubation was both cost-saving and event-reducing. The study suggests that policymakers should consider utilizing these monetary benefits in critical care. [Extracted from the article]

Assesses the impact of a 1989 consensus conference about the treatment of hypovolemia on prescribing practice at a hospital. Conclusion that consensus conferences can be a useful means of improving medical practice; Features that ensured success in this particular program; Consensus conferences organized by the French Society of Intensive Care Medicine; More.

Background: With 11,558 deaths and 200,000 suicide attempts in 2019, France is among the European countries most affected. The aim of this study was to determine the costs and burden of suicides and suicide attempts in France (population 67 million). Methods: We estimated direct costs, comprising healthcare, as well as post-mortem costs including autopsy, body removal, funeral expenses, police intervention and support groups; indirect costs comprised lost productivity, daily allowances; the burden of disease calculations used a monetary value for death and disability based on incidence data. Data was obtained from the national statistics, health and social care database, registries, global burden of disease, supplemented by expert opinion. We combined top down and bottom up approaches. Results: The total costs and burden of suicides and suicide attempts was estimated at €18.5 billion and €5.4 billion, respectively. Direct costs were €566 million and €75 million; indirect costs were €3.8 billion and €3.5 billion; monetary value for death and disability was €14.6 billion and €1.3. The monetary value for death and disability represented 79.1% and 24.8% of total costs for suicide and suicide attempt respectively. Some costs were based upon expert opinion, caregivers' burden was not counted and pre COVID data only is reported. Conclusions: In France, the total cost and burden of suicides and suicide attempts was several billion €, suggesting major potential savings from public health interventions. [ABSTRACT FROM AUTHOR]

Background: The systematic use of susceptibility testing and tailored first‐line treatment for Helicobacter pylori eradication has yet to be established. Aim: To compare 14‐day tailored PCR‐guided triple therapy to 14‐day non‐Bismuth concomitant quadruple therapy for first‐line Helicobacter pylori eradication. Patients and Methods: We performed a multicenter, parallel‐group, randomized noninferiority controlled trial. Naive adult patients with Helicobacter pylori infection were treated with 14‐day tailored PCR‐guided triple therapy (esomeprazole 40 mg and amoxicillin 1000 mg b.d. plus clarithromycin 500 mg or levofloxacin 500 mg b.d. according to clarithromycin susceptibility) or 14‐day non‐Bismuth concomitant quadruple therapy (esomeprazole 40 mg, amoxicillin 1000 mg, clarithromycin 500 mg, and metronidazole 500 mg b.d.). The primary endpoint was H. pylori eradication. Results: We screened 991 patients for eligibility and randomized 241 patients. The first‐line eradication rate was 99.2% in the tailored PCR‐guided group and 95.9% in the control group (ITT population; absolute difference of +3.30%, with a lower bound of CI at −0.68%). Both first‐line therapies were well tolerated, with a formally significant difference in favor of the tailored PCR‐guided group (61.4% vs. 41.2%, p = 0.003). Economic analyses revealed a lower cost of the tailored PCR‐guided arm, with a 92% chance of being jointly more effective and less expensive than the control arm in the ITT population. Conclusion: In a country with a high level of clarithromycin resistance, the results of our study demonstrated the noninferiority of 14‐day tailored PCR‐guided triple therapy as a first‐line H. pylori eradication therapy compared to 14‐day non‐Bismuth quadruple therapy (ClinicalTrials.gov NCT02576236). [ABSTRACT FROM AUTHOR]

Background: Rivaroxaban 2.5 mg twice daily with aspirin 100 mg daily was shown to be better than aspirin 100 mg daily for preventing cardiovascular (CV) death, stroke or myocardial infarction in patients with either stable coronary artery disease (CAD) or peripheral artery disease (PAD). The cost-effectiveness of this regimen in this population is essential for decision-makers to know. Methods: US direct healthcare system costs (in USD) were applied to hospitalized events, procedures and study drugs utilized by all patients. We determined the mean cost per participant for the full duration of the trial (mean follow-up of 23 months) plus quality-adjusted life years (QALYs) and the incremental cost-effectiveness ratio (ICER) over a lifetime using a two-state Markov model with 1-year cycle length. Sensitivity analyses were performed on the price of rivaroxaban and the annual discontinuation rate. Results: The costs of events and procedures were reduced for Cardiovascular Outcomes for People Using Anticoagulation Strategies (COMPASS) patients who received rivaroxaban 2.5 mg orally (BID) plus acetylsalicylic acid (ASA) compared with ASA alone. Total costs were higher for the combination group ($7426 versus $4173) after considering acquisition costs of the study drug. Over a lifetime, patients receiving rivaroxaban plus ASA incurred $27,255 more and gained 1.17 QALYs compared with those receiving ASA alone resulting in an ICER of $23,295/QALY. ICERs for PAD only and polyvascular disease subgroups were lower. Conclusion: Rivaroxaban 2.5 mg BID plus ASA compared with ASA alone was cost-effective (high value) in the USA. COMPASS ClinicalTrials.gov identifier: NCT01776424. [ABSTRACT FROM AUTHOR]

The Clinical Trials Subcommittee of the International Headache Society presents the first Health Technology Assessment for the Acute Treatment of Migraine Attacks and Prevention of Migraine. Health technology assessments are systematic evaluations of the properties, effects, and consequences of healthcare technologies; this position statement is designed to inform decision makers about access to and reimbursement for medications and devices for the acute and preventive treatment of migraine. This position statement extends beyond the already available guidelines on randomized controlled trials for migraine to incorporate real-world evidence and a synthetic approach for considering multiple data sources and modelling methods when assessing the value of migraine treatments. [ABSTRACT FROM AUTHOR]

Background: The aim of this trial-based economic evaluation was to assess the incremental costs and cost-effectiveness of the modified diagnostic strategy combining the YEARS rule and age-adjusted D-dimer threshold compared with the control (which used the age-adjusted D-dimer threshold only) for the diagnosis of pulmonary embolism (PE) in the Emergency Department (ED). Methods: Economic evaluation from a healthcare system perspective alongside a non-inferiority, crossover, and cluster-randomized trial conducted in 16 EDs in France and two in Spain with three months of follow-up. The primary endpoint was the additional cost of a patient without failure of the diagnostic strategy, defined as venous thromboembolism (VTE) diagnosis at 3months after exclusion of PE during the initial ED visit. Mean differences in 3-month failure and costs were estimated using separate generalized linear-regression mixed models, adjusted for strategy type, period, and the interaction between strategy and period as fixed effects and the hospital as a random effect. The incremental cost-effectiveness ratio (ICER) was obtained by dividing the incremental costs by the incremental frequency of VTE. Results: Of the 1,414 included patients, 1,217 (86%) were analyzed in the per-protocol analysis (648 in the intervention group and 623 in the control group). At three months, there were no statistically significant differences in total costs (€-46; 95% CI: €-93 to €0.2), and the failure rate was non inferior in the intervention group (-0.64%, one-sided 97.5% CI: -∞ to 0.21%, non-inferiority margin 1.5%) between groups. The point estimate of the incremental cost-effectiveness ratio (ICER) indicating that each undetected VTE averted in the intervention group is associated with cost savings of €7,142 in comparison with the control group. There was a 93% probability that the intervention was dominant. Similar results were found in the as randomized population. Conclusions: Given the observed cost decrease of borderline significance, and according to the 95% confidence ellipses, the intervention strategy has a potential to lead to cost savings as a result of a reduction in the use of chest imaging and of the number of undetected VTE averted. Policy-makers should investigate how these monetary benefits can be distributed across stakeholders. Clinicaltrials: Trial registration number ClinicalTrials.gov Identifier: NCT04032769; July 25, 2019. [ABSTRACT FROM AUTHOR]

Aims: The Cardiovascular Outcomes for People Using Anticoagulation Strategies (COMPASS) trial demonstrated that rivaroxaban 2.5 mg BID with aspirin 100 mg was more effective than aspirin 100 mg daily alone for the prevention of cardiovascular (CV) death, stroke, or myocardial infarction in patients with stable coronary artery disease (CAD) or peripheral artery disease (PAD). We aimed to examine the cost-effectiveness of rivaroxaban using patient-level data from the COMPASS trial. Methods and results: We performed an in-trial analysis and extrapolated our results for 33 years using a two-state Markov model with a 1-year cycle length. Hospitalization events, procedures, and study drugs were documented for patients. We applied countryspecific (Canada, France, and Germany) direct healthcare system costs (in USD) to healthcare resources consumed by patients. Average cost per patient during the trial (mean follow-up of 23 months), quality-adjusted life years (QALYs), and lifetime cost-effectiveness were calculated. Costs of events and procedures were reduced with rivaroxaban 2.5 mg BID with aspirin. The addition of rivaroxaban 2.5 mg BID increased total costs for the combination group. Over a lifetime horizon (in trial +33 years), rivaroxaban plus aspirin was associated with 1.17 QALYs gained, yielding an incremental cost-effectiveness ratio (ICER) of $3946/QALY, $9962/QALY, and $10 264/QALY in Canada, France, and Germany, respectively. PAD and polyvascular disease subgroups had lower ICERs. Conclusion: Rivaroxaban 2.5 mg twice daily plus aspirin compared with aspirin alone reduces direct healthcare costs. After acquisition costs of rivaroxaban, the lifetime cost-effectiveness of 2.5 mg twice daily plus aspirin is highly cost-effective in Canada, France, and Germany. [ABSTRACT FROM AUTHOR]

Whilst much uncertainty exists as to the efficacy of renal denervation (RDN), the positive results of the DENERHTN study in France confirmed the interest of an economic evaluation in order to assess efficiency of RDN and inform local decision makers about the costs and benefits of this intervention. The uncertainty surrounding both the outcomes and the costs can be described using health economic methods such as the non-parametric bootstrap. Internationally, numerous health economic studies using a cost-effectiveness model to assess the impact of RDN in terms of cost and effectiveness compared to antihypertensive medical treatment have been conducted. The DENERHTN cost-effectiveness study was the first health economic evaluation specifically designed to assess the cost-effectiveness of RDN using individual data. Using the DENERHTN results as an example, we provide here a summary of the principle methods used to perform a cost-effectiveness analysis. [ABSTRACT FROM PUBLISHER]

The COVID-19 pandemic highlighted the fragmented nature of governmental policy decisions in Europe. However, the extent to which COVID-19 vaccination policies differed between European countries remains unclear. Here, we mapped the COVID-19 vaccination policies that were in effect in January 2022 as well as booster regulations in April 2022 in Austria, Denmark, England, France, Germany, Ireland, Italy, the Netherlands, Poland, and Spain. National public health and health policy experts from these ten European nations developed and completed an electronic questionnaire. The questionnaire included a series of questions that addressed six critical components of vaccine implementation, including (1) authorization, (2) prioritization, (3) procurement and distribution, (4) data collection, (5) administration, and (6) mandate requirements. Our findings revealed significant variations in COVID-19 vaccination policies across Europe. We observed critical differences in COVID-19 vaccine formulations authorized for use, as well as the specific groups that were provided with priority access. We also identified discrepancies in how vaccination-related data were recorded in each country and what vaccination requirements were implemented. Each of the ten European nations surveyed in this study reported different COVID-19 vaccination policies. These differences complicated efforts to provide a coordinated pandemic response. These findings might alert policymakers in Europe of the need to coordinate their efforts to avoid fostering divergent and socially disruptive policies. [ABSTRACT FROM AUTHOR]

• Our estimate of the global burden of mental health was € 163 bn in 2018, in France. • We found a relative 50% increase of costs compared to our previous study, published 10 years ago. • Medical, social, indirect and intangible costs represented respectively 14%, 8%, 27% and 51% of the global burden of mental health. 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. [ABSTRACT FROM AUTHOR]

The French healthcare system is a universal healthcare system with no financial barrier to access to health services and cancer drugs. The objective of the study is to investigate associations between, on the one hand, incidence and survival of patients diagnosed with lung cancer in France and, on the other, the socioeconomic deprivation and population density of their municipality of residence. A national, longitudinal analysis using data from the French National Hospital database crossed with the population density of the municipality and a social deprivation index based on census data aggregated at the municipality level. For lung cancer diagnosed at the metastatic stage, one-year and two-year survival was not associated with the population density of the municipality of residence. In contrast, mortality was higher for people living in very deprived, deprived and privileged areas compared to very privileged areas (hazard ratios at two years: 1.19 [1.13–1.25], 1.14 [1.08–1.20] and 1.10 [1.04–1.16] respectively). Similar associations are also observed in patients diagnosed with non-metastatic disease (hazard ratios at two years: 1.21 [1.13–1.30], 1.15 [1.08–1.23] and 1.10 [1.03–1.18] for people living in very deprived, deprived and privileged areas compared to very privileged areas). Despite a universal healthcare coverage, survival inequalities in patients with lung cancer can be observed in France with respect to certain socioeconomic indicators. [ABSTRACT FROM AUTHOR]

Objectives: To better understand the reasons for differences in reimbursement decisions for orphan drugs in four European countries that were not readily apparent from health technology assessment (HTA) reports and operating procedures.Methods: Semistructured interviews with representatives of HTA bodies in England, Scotland, Sweden, and France were conducted. An interview topic guide was developed on the basis of findings from a systematic comparison of HTA decisions for 10 orphan drugs. Qualitative thematic data analysis was applied to the interview transcripts using the framework approach.Results: Eight representatives from the four HTA bodies were interviewed between March and June 2015. Evidentiary requirements and approaches to dealing with imperfect or incomplete evidence were explored, including trial design and duration, study population and subgroups, comparators, and end points. Interviewees agreed that decisions regarding orphan drugs are made in a context of lower quality evidence, and the threshold of acceptable uncertainty varied by country. Some countries imposed higher evidentiary standards for greater clinical claims, which may be more challenging for orphan diseases. The acceptability of surrogate end points was not consistent across countries nor were the validation requirements. The most common social value judgments identified related to innovation, disease severity, and unmet need. Differences were seen in the way these concepts were defined and accounted for across countries.Conclusions: Although agreement was seen in evidentiary requirements or preferences, there were subtle differences in the circumstances in which uncertain evidence may be considered acceptable, possibly explaining differences in HTA recommendations across countries. [ABSTRACT FROM AUTHOR]

Objective: To evaluate a saliva diagnostic test (Endotest®) for endometriosis compared with the conventional algorithm. Design: A cost‐effectiveness analysis with a decision‐tree model based on literature data. Setting: France. Population: Women with chronic pelvic pain. Methods: Strategy I is the French algorithm, representing the comparator. For strategy II, all patients have an Endotest®. For strategy III, patients undergo ultrasonography to detect endometrioma and patients with no endometrioma detected have an Endotest®. For strategy IV, patients with no endometrioma detected on ultrasonography undergo pelvic magnetic resonance imaging (MRI) to detect endometrioma and/or deep endometriosis. An Endotest® is then performed for patients with a negative result on MRI. Main outcomes measures: Costs and accuracy rates and incremental cost‐effectiveness ratios (ICERs). Three analyses were performed with an Endotest® priced at €500, €750, and €1000. Probabilistic sensitivity analysis was conducted with Monte Carlo simulations. Results: With an Endotest® priced at €750, the cost per correctly diagnosed case was €1542, €990, €919 and €1000, respectively, for strategies I, II, III and IV. Strategy I was dominated by all other strategies. Strategies IV, III and II were, respectively, preferred for a willingness‐to‐pay threshold below €473, between €473 and €4670, and beyond €4670 per correctly diagnosed case. At a price of €500 per Endotest®, strategy I was dominated by all other strategies. At €1000, the ICERs of strategies II and III were €724 and €387 per correctly diagnosed case, respectively, compared with strategy I. Conclusion: The present study demonstrates the value of the Endotest® from an economic perspective. [ABSTRACT FROM AUTHOR]

Objective: To evaluate a saliva diagnostic test (Endotest®) for endometriosis compared with the conventional algorithm. Design: A cost‐effectiveness analysis with a decision‐tree model based on literature data. Setting: France. Population: Women with chronic pelvic pain. Methods: Strategy I is the French algorithm, representing the comparator. For strategy II, all patients have an Endotest®. For strategy III, patients undergo ultrasonography to detect endometrioma and patients with no endometrioma detected have an Endotest®. For strategy IV, patients with no endometrioma detected on ultrasonography undergo pelvic magnetic resonance imaging (MRI) to detect endometrioma and/or deep endometriosis. An Endotest® is then performed for patients with a negative result on MRI. Main outcomes measures: Costs and accuracy rates and incremental cost‐effectiveness ratios (ICERs). Three analyses were performed with an Endotest® priced at €500, €750, and €1000. Probabilistic sensitivity analysis was conducted with Monte Carlo simulations. Results: With an Endotest® priced at €750, the cost per correctly diagnosed case was €1542, €990, €919 and €1000, respectively, for strategies I, II, III and IV. Strategy I was dominated by all other strategies. Strategies IV, III and II were, respectively, preferred for a willingness‐to‐pay threshold below €473, between €473 and €4670, and beyond €4670 per correctly diagnosed case. At a price of €500 per Endotest®, strategy I was dominated by all other strategies. At €1000, the ICERs of strategies II and III were €724 and €387 per correctly diagnosed case, respectively, compared with strategy I. Conclusion: The present study demonstrates the value of the Endotest® from an economic perspective. [ABSTRACT FROM AUTHOR]

Background: 82-Rubidium-Positron emission tomography myocardial perfusion imaging (Rb-PET-MPI) offers higher diagnostic performance for the detection of myocardial ischemia compared to Tc-SPECT-MPI. The aim of this economic evaluation was to perform a cost-effectiveness analysis of Rb-PET-MPI versus Tc-SPECT-MPI in patients with suspected myocardial ischemia according to pretest probabilities (PTP) of obstructive coronary artery disease based on the results of the RUBIS Trial. Methods: Costs and effectiveness were calculated for all patients over 1 year and an incremental analysis of differences in costs and effectiveness in terms of diagnostic accuracy was performed. The uncertainty of the results was estimated using bootstrap. The analysis was conducted from the perspective of the French health care system with a time horizon of 12 months. Results: The average cost of a Rb-PET-MPI-based strategy for the detection of myocardial ischemia was €219 lower than a SPECT-MPI-based strategy (€1192 (± 1834) vs €973 (± 1939), p < 0.01). The one-year incremental cost-effectiveness ratio was negative: − €2730 (money saved per additional accurate diagnosis) in patients presenting PTP > 15% for the Rb-PET-MPI vs. Tc-SPECT-MPI strategy. Analysis of the joint distribution of costs and outcomes found that the Rb-PET-MPI strategy had a 92% probability to be dominant (cost-saving and outcome-improving). Conclusions: Rb-PET-MPI is cost-effective compared to Tc-SPECT-MPI for the detection of myocardial ischemia in patients with PTP > 15% of obstructive coronary artery disease. Trial registration: RUBIS Trial registration: NCT01679886, Registered 03 September 2012, https://clinicaltrials.gov/ct2/show/NCT01679886. [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]

Summary: Obtaining informed consent is a requirement, which can result in better patient management and outcome. Trial results indicate thatpatients prefer to receive information orally and sequentially from different professionals and later to read documents or access informations via the Internet. In patients suffering from low back pain, providing detailed information results in a moderately better functional outcome and performance for patients and significantly better satisfaction with care. A newer extension of the informed consent process is shared decision making by which patients'' preferences with regards to treatment patterns and outcomes are explicitly used to decide on the treatment choice [Copyright &y& Elsevier]

Simple Summary: There is a need to better understand the effectiveness of new treatments, such as the recently approved nivolumab, in patients with locally advanced or metastatic non-small cell lung cancer in clinical practice. This study aims to report the characteristics and outcomes of 2784 patients with locally advanced or metastatic non-small cell lung cancer receiving nivolumab in second-line or later in France (ESME-AMLC) and Germany (CRISP) between 2015 and 2020. Two-year survival rates were 26.7% in patients with tumors with squamous histology and 32.8% in patients with non-squamous/others histologies in ESME-AMLC, and 20.9% and 18.9%, respectively, in CRISP. Poorer performance score and shorter duration from the previous line of therapy initiation were significantly associated with shorter treatment duration with nivolumab and overall survival. These real-world data provide insight into the characteristics of patients receiving nivolumab in France and Germany and confirm the efficacy of nivolumab previously observed in clinical trials. This study reports characteristics and outcomes in patients with locally advanced or metastatic non-small cell lung cancer (aNSCLC) receiving nivolumab in second-line or later (2L+) in France and Germany between 2015 and 2020. Patients with aNSCLC (stage IIIB–C/IV) receiving nivolumab in 2L+ were included from the retrospective Epidemiological Strategy and Medical Economics of Advanced and Metastatic Lung Cancer cohort (ESME-AMLC, France; 2015–2019) and Clinical Research platform Into molecular testing, treatment and outcome of non-Small cell lung carcinoma Patients (CRISP, Germany; 2016–2020). Overall, 2262 ESME-AMLC and 522 CRISP patients were included. Median treatment duration (95% confidence intervals) was 2.8 months (2.5–3.2) in squamous and 2.5 months (2.3–2.8) in non-squamous/others patients in ESME-AMLC, and 2.3 months (1.4–3.1) and 2.3 months (2.0–2.8), respectively in CRISP. One-year and two-year overall survival (OS) were 47.2% and 26.7% in squamous and 50.8% and 32.8% in non-squamous/others patients in ESME-AMLC, and 43.1% and 20.9%, and 37.7% and 18.9%, respectively in CRISP. Poorer performance score and shorter time from start of previous line of therapy initiation were significantly associated with shorter treatment duration and OS. This study confirms, in real-world clinical databases, the efficacy of nivolumab previously observed in clinical trials. [ABSTRACT FROM AUTHOR]

Mental disorders are at-risk of severe COVID-19 outcomes. There is limited and heterogeneous national data in hospital settings evaluating the risks associated with any pre-existing mental disorder, and susceptible subgroups. Our study aimed to investigate the association between pre-existing psychiatric disorders and outcomes of adults hospitalised for COVID-19. We used data obtained from the French national hospital database linked to the state-level psychiatric registry. The primary outcome was 30-days in-hospital mortality. Secondary outcomes were to compare the length of hospital stay, Intensive Care Unit (ICU) admission and ICU length. Propensity score matching analysis was used to control for COVID-19 confounding factors between patients with or without mental disorder and stratified by psychiatric subgroups. Among 97 302 adults hospitalised for COVID-19 from March to September 2020, 10 083 (10.3%) had a pre-existing mental disorder, mainly dementia (3581 [35.5%]), mood disorders (1298 [12.9%]), anxiety disorders (995 [9.9%]), psychoactive substance use disorders (960 [9.5%]), and psychotic disorders (866 [8.6%]). In propensity-matched analysis, 30-days in-hospital mortality was increased among those with at least one pre-existing mental disorder (hazard ratio (HR) 1.15, 95% CI 1.08–1.23), psychotic disorder (1.90, 1.24–2.90), and psychoactive substance disorders (1.53, 1.10–2.14). The odds of ICU admission were consistently decreased for patients with any pre-existing mental disorder (OR 0.83, 95% CI 0.76–0.92) and for those with dementia (0.64, 0.53–0.76). Pre-existing mental disorders were independently associated with in-hospital mortality. These findings underscore the important need for adequate care and targeted interventions for at-risk individuals with severe mental illness. [ABSTRACT FROM AUTHOR]

Objectives: The purpose of this study was to compare the costs and organizational benefits of diagnostic workup without and with MRI dedicated to the ED. Methods: We conducted a prospective observational uncontrolled before-after study in one ED of a university hospital in France from July 1, 2018, and January 3, 2020. We included all consecutive patients presenting with dizziness or diplopia. The main outcomes were the clinical decision time of ED physicians and the total costs for each strategy. Outcomes were compared using propensity score with inverse probability weighting in the 2 arms and an incremental cost-effectiveness ratio (ICER) was calculated. Results: Among the 199 patients during the "before" period (average age: 60.4 years ± 17.6): 112 men (57%), and 181 during the "after" period (average age, 54.8 years ± 18.5): 107 men (59%), the average costs were €2701 (95% CI 1918; 3704) and €2389 (95% CI: €1627; 3280) per patient, respectively. The average time to clinical decision was 9.8 h (95% CI: 8.9 10.7) in the group "before" and 7.7 h (95% CI: 7.1; 8.4) in the group "after" (ICER: €151 saved for a reduction of 1 h in clinical decision time). The probabilistic sensitivity analysis estimated a 71% chance that the MRI dedicated to ED was dominant (less costly and more effective). Conclusion: Easy access to MRI in the ED for posterior circulation stroke-like symptoms must be considered a relevant approach to help physicians for an appropriate and rapid diagnostic with reduction of costs. Trial registration: NCT03660852 Key Points: • A dedicated MRI in the ED for diplopia or dizziness may be considered an efficient strategy improving diagnostic performance, reducing physicians' decision time, and decreasing hospital costs. • This strategy supports clinical decision-making with early treatment and management of patients with posterior circulation-like symptoms in the ED. • There is 71% chance that the MRI dedicated to ED was dominant (less costly and more effective) compared with a strategy without dedicated MRI. [ABSTRACT FROM AUTHOR]

• Treatments and outcomes in locally advanced/metastatic non-small cell lung cancer. • Real-world patients in France and Germany (2015–2018) without ALK/EGFR mutations. • In first-line-treated patients, >50 % received an immune checkpoint inhibitor (ICI) • Future analyses will allow evaluation of the impact of wider ICI use in first-line. To describe the impact of immune checkpoint inhibitors (ICIs) on treatment patterns and survival outcomes in patients with locally advanced or metastatic non-small cell lung cancer (aNSCLC) in France and Germany. Patients with aNSCLC without known ALK or EGFR mutations receiving first-line (1L) therapy were included from (i) the retrospective Epidemiological-Strategy and Medical Economics Advanced and Metastatic Lung Cancer cohort (ESME-AMLC, France; 2015–2018) and (ii) the prospective Clinical Research platform Into molecular testing, treatment and outcome of non-Small cell lung carcinoma Patients platform (CRISP, Germany; 2016–2018). Analyses were stratified according to histology. Survival outcomes were estimated using Kaplan–Meier methodology and stratified by year of 1L therapy. Data sources were analysed separately. In ESME-AMLC and CRISP, 8,046 and 2,359 patients were included in the study, respectively. In both countries, approximately 20 % of all patients received pembrolizumab monotherapy as 1L treatment in 2018. In ESME-AMLC, the proportion receiving an ICI over the course of treatment (any line) increased from 42.2 % (2015) to 56.1 % (2018) in patients with squamous histology, and 28.9 % to 51.9 % with non-squamous/other; in CRISP, it increased from 50.6 % (2016) to 65.2 % (2018) with squamous histology, and 40.8 % to 62.7 % with non-squamous/other. Two-year overall survival from 1L initiation was 36.8 % and 25.6 % in the squamous cohorts and 36.5 % and 30.8 % in the non-squamous/other cohorts in ESME-AMLC and CRISP, respectively. No significant change in overall survival was observed over time; however, the follow-up time available was limited in the later years of the analysis. The results of this joint research from two large clinical databases in France and Germany demonstrate the growing use of ICIs in the management of aNSCLC. Future analyses will allow for the evaluation of the impact of ICIs on long-term survival of patients with aNSCLC. [ABSTRACT FROM AUTHOR]

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]

Background: Spread of resistant bacteria causes severe morbidity and mortality. Stringent control measures can be expensive and disrupt hospital organization. In the present study, we assessed the effectiveness and cost-effectiveness of control strategies to prevent the spread of Carbapenemase-producing Enterobacterales (CPE) in a general hospital ward (GW). Methods: A dynamic, stochastic model simulated the transmission of CPE by the hands of healthcare workers (HCWs) and the environment in a hypothetical 25-bed GW. Input parameters were based on published data; we assumed the prevalence at admission of 0.1%. 12 strategies were compared to the baseline (no control) and combined different prevention and control interventions: targeted or universal screening at admission (TS or US), contact precautions (CP), isolation in a single room, dedicated nursing staff (DNS) for carriers and weekly screening of contact patients (WSC). Time horizon was one year. Outcomes were the number of CPE acquisitions, costs, and incremental cost-effectiveness ratios (ICER). A hospital perspective was adopted to estimate costs, which included laboratory costs, single room, contact precautions, staff time, i.e. infection control nurse and/or dedicated nursing staff, and lost bed-days due to prolonged hospital stay of identified carriers. The model was calibrated on actual datasets. Sensitivity analyses were performed. Results: The baseline scenario resulted in 0.93 CPE acquisitions/1000 admissions and costs 32,050 €/1000 admissions. All control strategies increased costs and improved the outcome. The efficiency frontier was represented by: (1) TS with DNS at a 17,407 €/avoided CPE case, (2) TS + DNS + WSC at a 30,700 €/avoided CPE case and (3) US + DNS + WSC at 181,472 €/avoided CPE case. Other strategies were dominated. Sensitivity analyses showed that TS + CP might be cost-effective if CPE carriers are identified upon admission or if the cases have a short hospital stay. However, CP were effective only when high level of compliance with hand hygiene was obtained. Conclusions: Targeted screening at admission combined with DNS for identified CPE carriers with or without weekly screening were the most cost-effective options to limit the spread of CPE. These results support current recommendations from several high-income countries. [ABSTRACT FROM AUTHOR]

Simple Summary: The optimal management of non-metastatic hepatocellular carcinoma (HCC) remains debated. The association between HCC and cirrhosis influences prognosis and therapeutic choices between curative and palliative treatments. The goal of our retrospective study was to evaluate the cost-effectiveness of the extended use of ablation for the treatment of HCC with cirrhosis in an expert ablation center when compared to the non-extended use of ablation in equivalent tertiary care centers. In a propensity-score matched cohort of 532 patients with naïve HCC, the extended use of ablation led to better compliance with the Barcelona Clinic Liver Classification (BCLC) guidelines (80% vs. 67%) and was more effective and less expensive than the non-extended use of ablation strategy, particularly at an earlier stage of the disease. The shift from curative to palliative treatments was noted in a considerable percentage of patients; therefore, this needs to be redefined as the wide choice of ablation techniques and technical advances in imaging guidance increase the curative options available to treat a maximum of patients with HCC. Background: To evaluate the cost-effectiveness of the extended use of ablation for the treatment of hepatocellular carcinoma (HCC) with cirrhosis in an expert ablation center when compared to the non-extended use of ablation in equivalent tertiary care centers. Methods: Consecutive cirrhotic patients with non-metastatic HCC, no prior treatment, and referred to three tertiary care centers between 2012 and 2016 were retrospectively identified. The Bondy group, including all of the patients treated at Jean Verdier Hospital, where the extended use of ablation is routinely performed, was compared to the standard of care (SOC) group, including all of the patients treated at the Beaujon and Mondor Hospitals, using propensity score matching. A cost-effectiveness analysis was carried out from the perspective of French health insurance using a Markov model on a lifetime horizon. Results: 532 patients were matched. The Bondy group led to incremental discounted lifetime effects of 0.8 life-years gained (LYG) (95% confidence interval: 0.4, 1.3) and a decrease in lifetime costs of EUR 7288 (USD 8016) (95% confidence interval: EUR 5730 [USD 6303], EUR 10,620 [USD 11,682]) per patient, compared with the SOC group, resulting in a dominant mean incremental cost-effectiveness ratio (ICER). A compliance with the Barcelona Clinic Liver Classification (BCLC) guidelines for earlier stage contributed to the greater part of the ICER. Conclusion: The extended use of ablation in cirrhotic patients with HCC was more effective and less expensive than the non-extended use of the ablation strategy. [ABSTRACT FROM AUTHOR]

Background: Bipolar disorder (BD) is a severe chronic psychiatric disorder affecting 0.5 to 1% of the population worldwide. To date, most studies have estimated the cost of BD via information sourced from insurance claims with limited information on clinical characteristics and course of BD. The aims of this study are (i) to estimate the direct healthcare cost associated with BD and to identify contributing factors and (ii) to study the evolution of cost during a two-year follow-up period.Method: We analyzed a sample of 1116 individuals with BD included in the Advanced Centers of Expertise in Bipolar Disorder cohort. We estimated the direct healthcare cost per year and per patient, and we identified the clinical features of patients with BD associated with higher direct healthcare costs. In a subsample of patients followed up for two years centers of expertise for BD, we studied the evolution of direct healthcare cost.Results: The average cost of bipolar disorder was € 6910 per year and per patient. Clinical features of BD, sociodemographic characteristics, and associated addiction were associated with higher direct healthcare costs. In the subsample of patients followed-up for two years, direct healthcare cost dropped by more than 50%, strongly suggesting the beneficial effect of specialized care organization.Limitation: We did not estimate indirect healthcare and intangible costs.Conclusion: Our study investigates the cost of BD and its evolution in a deeply phenotyped longitudinal sample. Cost-utility and cost-effectiveness analyses are required to inform resource allocation decisions and to promote innovative healthcare organizations. [ABSTRACT FROM AUTHOR]

Objective To estimate the incremental cost effectiveness of full coverage of the medical management of smoking cessation from the perspective of statutory health insurance (SHI) in France. Design and population Cost-effectiveness analysis based on a Markov state-transition decision analytic model was used to compare full SHI coverage of smoking cessation and actual coverage based on an annual €50 lump sum per insured person among current French smokers aged 15–75 years. We used a scenario approach to take into account the many different behaviours of smokers and the likely variability of SHI policy choices in terms of participation rate and number and frequency of attempts covered. Interventions Drug treatments for smoking cessation combined with six medical consultations including individual counselling. Main outcomes measures The cost effectiveness of full coverage was expressed by the incremental costeffectiveness ratio (ICER) in 2009 euros per life-year gained (LYG) at the lifetime horizon. Results The cost effectiveness per LYG for smokers ranged from €1786 to €2012, with an average value of €1911. The minimum value was very close to the maximum value with a difference of only €226. The cost-effectiveness ratio was only minimally sensitive to the participation rate, the number of attempts covered and the cessation rate. Conclusions Compared to other health measures in primary and secondary prevention of cardiovascular disease already covered by SHI, full coverage of smoking cessation is the most cost-effective approach. [ABSTRACT FROM AUTHOR]

Abstract: 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. [Copyright &y& Elsevier]

Abstract: Purpose: FRAX™ is a fracture prediction algorithm to determine a patient''s absolute fracture risk. There is a growing consensus that osteoporosis treatment should be based on individual 10-year fracture probability, as calculated in the FRAX™ algorithm, rather than on T-scores alone. Objective: Our objective was to evaluate the cost-effectiveness of five years of branded alendronate therapy in postmenopausal French women with a known FRAX™ score. Method: A Markov cohort state transition model using FRAX™ values and whenever possible population-specific data and probabilities. We estimated the incremental cost-effectiveness ratio (ICER) of alendronate versus no treatment in postmenopausal women with FRAX™ ranging from 10 to 3%. Outcomes: Number of women to treat (NNT) for preventing hip fracture, costs, quality-adjusted life-years, incremental cost-effectiveness ratios. Results: The incremental cost-effectiveness ratios (ICER) compared to no treatment at age 70 ranged from €104,183 to €413,473 per QALY when FRAX™ decreased from 10 to 3%. The NNTs for preventing one hip fracture ranged from 97 to 388 according to age (50–80 years) and FRAX™. Sensitivity analyses showed that the main determinants of cost-effectiveness were adherence to therapy and cost of treatment. Conclusion: Using French costs of branded drug and current estimates of treatment efficacy, alendronate therapy for 70-year-old women with 10-year probability of hip fracture of 10% just meets the accepted cost-effectiveness threshold. Improving treatment adherence and/or decreasing treatment cost lowers the ICER. The model however underestimates the potential benefit by excluding other fractures. [Copyright &y& Elsevier]