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Resume Introduction La prise en charge des cancers pediatriques s’effectue au sein de centres hospitaliers referents pouvant deleguer des soins aux centres hospitaliers de proximite (CHP). Il n’existe pas de CHP en Corse, contraignant les enfants et leur famille a quitter l’ile pour la quasi-totalite des soins. L’objectif etait de faire un etat des lieux de la situation en Corse. Methodes Il s’agissait d’une etude descriptive s’appuyant sur des donnees du Registre national des cancers de l’enfant, du Reseau d’hematologie et d’oncologie pediatrique du Sud-Est et d’un questionnaire patient. Nous avons inclus tous les enfants atteints de cancer et residents en Corse sur une periode de 10 ans. Resultats Depuis 2005, 5 a 12 nouveaux cas par an etaient enregistres. Le centre hospitalier de la Timone (Marseille) etait le centre de reference pour 73 % des patients, suivi du CHU de Nice pour 14 % ; 90 % des parents interroges etaient favorables a la creation de CHP sur l’ile et la totalite mettait en avant de nombreuses difficultes qui, selon eux, pourraient etre ameliorees par la presence de CHP (organisation des deplacements, delai et distance depuis le domicile…). Toutefois, il semble exister, a l’heure actuelle, un manque de formation du personnel medical et paramedical pour assurer des soins de seconde ligne de qualite. Conclusion Le nombre annuel de nouveaux cas de cancers pediatriques en Corse peut justifier la creation d’un CHP sur l’ile. Des etudes de couts et de faisabilite sont necessaires, en vue d’optimiser la prise en charge et la qualite de vie des enfants et de leur famille.

Transthyretin (TTR) Amyloid Cardiomyopathy (ATTR-CM) is a rare, progressively debilitating, fatal disease with poor prognosis caused by amyloid deposition of fibrils derived from the serum protein TTR in the extracellular matrix of the heart. As amyloid infiltration in the heart progresses, atrial and ventricular walls thicken and become restrictive, resulting in diastolic dysfunction and further progression leads to heart failure, usually with preserved ejection fraction. Systolic dysfunction occurs in the late stages of the disease. Cardiac symptoms and conduction abnormalities also become increasingly clinically apparent (e.g. fatigue, shortness of breath, syncope or arrhythmias) with progression over time. Epidemiology of ATTR-CM is poorly understood as there are few existing studies that estimate its frequency in the general population. We aimed to estimate the prevalence and the incidence of ATTR-CM in France between 2011 and 2017, to describe demographic characteristics of incident cases and to assess patient survival. We used data from the SNDS database, which collects all national health insurance and hospital discharge data. As there is no specific ICD-10 marker code for ATTR -CM used in the SNDS, ATTR-CM diagnosis required both an amyloidosis and a cardiovascular condition, not necessarily reported at the same visit. Diagnostic date was defined when the features from amyloidosis and cardiovascular conditions were selected as events. Patients with a probable AL form of the disease were excluded. To remain conservative, patients younger than 50 yo were also excluded. Between 2011 and 2017, 4,815 patients with incident ATTR-CM were identified. Incidence rate was multiplied by more than 3 times, from 0.5 / 100,000 person-year in 2011 to 1.8 / 100,000 person-year in 2017, reaching 1,225 new cases in 2017. Sex-ratio remained stable (2:1). Most of the 4,815 identified patients were older than 70 yo. In the group ATTR CM >70 y.o, there were 3 times more men than women. Median age at diagnosis was 84.0 for women and 82.0 for men. The range of 80–89 yo represents about 50% of diagnose rate in overall population. Median survival was 33.7 months overall with minor differences between gender. Survival probability was 0.69 a year after diagnosis, 0.58 two years after, 0.48 three years after, and 0.41 four years after. Using exponential modeling the life expectancy of this population would be about 4 years for the 20 next years. This study is, to our knowledge, the first estimate of ATTR-CM incidence rates based upon an analysis of a national claims database. Our findings are consistent with existing data concerning the frequency of amyloidosis, even though were only identified the ICD-10 coded diagnosed cases, leading to possible underestimation of the full prevalence of the disease in France. SNDS demonstrated to be a scientifically valid data base to follow-up the standard of care of ATTR-CM. Funding Acknowledgement Type of funding sources: Private company. Main funding source(s): Pfizer France - SAS

Background [18F]2-fluoro-2-deoxy- d -glucose positron emission tomography/computed tomography (18FDG-PET/CT) has high sensitivity for detecting recurrences of colorectal cancer (CRC). Our objective was to determine whether adding routine 6-monthly 18FDG-PET/CT to our usual monitoring strategy improved patient outcomes and to assess the effect on costs. Patients and methods In this open-label multicentre trial, patients in remission of CRC (stage II perforated, stage III, or stage IV) after curative surgery were randomly assigned (1 : 1) to usual monitoring alone (3-monthly physical and tumour marker assays, 6-monthly liver ultrasound and chest radiograph, and 6-monthly whole-body computed tomography) or with 6-monthly 18FDG-PET/CT, for 3 years. A multidisciplinary committee reviewed each patient’s data every 3 months and classified the recurrence status as yes/no/doubtful. Recurrences were treated with curative surgery alone if feasible and with chemotherapy otherwise. The primary end point was treatment failure defined as unresectable recurrence or death. Relative risks were estimated, and survival was analysed using the Kaplan–Meier method, log-rank test, and Cox models. Direct costs were compared. Results Of the 239 enrolled patients, 120 were in the intervention arm and 119 in the control arm. The failure rate was 29.2% (31 unresectable recurrences and 4 deaths) in the intervention group and 23.7% (27 unresectable recurrences and 1 death) in the control group (relative risk = 1.23; 95% confidence interval, 0.80–1.88; P = 0.34). The multivariate analysis also showed no significant difference (hazards ratio, 1.33; 95% confidence interval, 0.8–2.19; P = 0.27). Median time to diagnosis of unresectable recurrence (months) was significantly shorter in the intervention group [7 (3–20) versus 14.3 (7.3–27), P = 0.016]. Mean cost/patient was higher in the intervention group (18 192 ± 27 679 € versus 11 131 ± 13 €, P Conclusion 18FDG-PET/CT, when added every 6 months, increased costs without decreasing treatment failure rates in patients in remission of CRC. The control group had very close follow-up, and any additional improvement (if present) would be small and hard to detect. ClinicalTrials.gov identifier NCT00624260

Resume Objectifs L’un des objectifs majeurs de l’utilisation systematique de l’echographie obstetricale pendant la grossesse est le depistage des malformations fœtales. Notre objectif etait d’analyser l’efficience medico-economique de ce depistage. Methodes Nous avons realise une revue de la litterature a partir de Medline via PubMed, entre 1985 et 2015, sur l’aspect medico-economique du depistage par echographie des malformations fœtales. Resultats La litterature etait tres pauvre sur le sujet et nous n’avons pu retenir et analyser que 12 articles presentant des donnees interessantes pour notre revue, dont huit veritables etudes medico-economiques. Nous avons identifie des arguments en faveur du caractere economiquement pertinent d’un programme de depistage des malformations, ceci etant en grande partie lie a la pratique des interruptions medicales de grossesse et aux couts des handicaps « evites », mais sans pouvoir conclure d’un point de vue medico-economique. Nous avons surtout pu mettre en evidence plusieurs elements compliquant l’application de ces analyses dans ce domaine : le choix du type d’etude, la forte incertitude sur deux parametres essentiels (l’efficacite de l’echographie et le cout des procedures), la difficulte a comparer ou generaliser les resultats. Nous avons aussi constate l’heterogeneite methodologique des etudes et l’absence d’etude francaise. Conclusion Les donnees existantes de la litterature sont actuellement insuffisantes pour evaluer l’efficience medico-economique du depistage des malformations par echographie.

Background The benefits of single‐use ultrasonic scissors in thyroid surgery are still debated. Although this device has been shown to reduce operating time compared with conventional haemostasis, its cost‐effectiveness has never been demonstrated. The aim of this study was to evaluate the efficacy, cost‐effectiveness and safety of ultrasonic scissors for total thyroidectomy. Methods This was a prospective, randomized, multicentre trial conducted at 13 hospital sites. The primary endpoint was the percentage of patients with hypocalcaemia (serum calcium level below 2 mmol/l) on day 2. Secondary endpoints included postoperative complications and costs, with calculation of incremental cost differences and cost‐effectiveness ratios. Results In total, 1329 patients who underwent total thyroidectomy were included in the analysis: 670 were randomized to treatment with ultrasonic scissors and 659 to conventional haemostasis. There was no difference between groups in the rate of complications, including hypocalcaemia on day 2 (19.7 per cent in ultrasonic scissors group versus 20.3 per cent in conventional haemostasis group; P = 0·743). Median operating times were significantly shorter with ultrasonic scissors (90 versus 100 min with conventional haemostasis; P

Background The benefits of single-use ultrasonic scissors in thyroid surgery are still debated. Although this device has been shown to reduce operating time compared with conventional haemostasis, its cost-effectiveness has never been demonstrated. The aim of this study was to evaluate the efficacy, cost-effectiveness and safety of ultrasonic scissors for total thyroidectomy. Methods This was a prospective, randomized, multicentre trial conducted at 13 hospital sites. The primary endpoint was the percentage of patients with hypocalcaemia (serum calcium level below 2 mmol/l) on day 2. Secondary endpoints included postoperative complications and costs, with calculation of incremental cost differences and cost-effectiveness ratios. Results In total, 1329 patients who underwent total thyroidectomy were included in the analysis: 670 were randomized to treatment with ultrasonic scissors and 659 to conventional haemostasis. There was no difference between groups in the rate of complications, including hypocalcaemia on day 2 (19.7 per cent in ultrasonic scissors group versus 20.3 per cent in conventional haemostasis group; P = 0·743). Median operating times were significantly shorter with ultrasonic scissors (90 versus 100 min with conventional haemostasis; P

Our primary objective was to compare the grading of the value of cancer drugs ('Amélioration du Service Médical Rendu' [ASMR] level) by the French health technology assessment authority ('Haute Autorité de santé' [HAS]) with that by the American Society of Clinical Oncology Value Framework (ASCO-VF) and the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Our secondary objective was to study the drivers of the French grading system.We included new drugs for solid tumors assessed by the HAS between 2010 and 2016 and compared their ASMR level to scores calculated by the 2016-updated ASCO-VF and 2015 ESMO-MCBS.We investigated 27 new cancer drugs assessed by the French HAS between 2010 and 2016. Among the 17 drugs eligible for comparison, the correlation between ASMR levels and ASCO and ESMO scores was weak (r = 0.34 and r = 0.27, respectively). The agreement between the HAS and ESMO regarding the level of meaningful additional benefit was moderate (kappa = 0.43). We found no significant association between 12 potential variables and ASMR level of additional benefit of drugs.Our findings show inconsistencies in cancer drug appraisals among the three appraisers.

In France, the management of pediatric cancers is carried out in reference hospitals that can delegate care to local health centres (LHC), forming "care networks". There is no LHC in Corsica, forcing children and their families to leave the island for all care in the reference centre. The aim of this study was to describe the situation in Corsica and to consider this organisation.This is a descriptive preliminary study based on databases from the National Childhood Cancer Registry, "RHeOP" network and a patient questionnaire. We included over a period of 10 years all children with onco-hematological disease who resided in Corsica.The incidence of pediatric cancers since 2005 ranged from 5 to 12 new cases per year. The hospital centre of Timone (Marseille) was the reference centre for 73% of patients, followed by Nice University Hospital for 14%. Almost all the parents interviewed (90%) were in favour of creating an LHC and all of them highlighted many difficulties that, according to them, could be improved by the presence of a LHC in Corsica (organisation of travel, delay and distance from home…). However, there currently appears to be a lack of training for medical and paramedical staff to provide quality second-line care.The rates of pediatric onco-hematological diseases in Corsica may warrant the creation of an LHC on the island. Additional cost studies on the feasibility of an LHC in Corsica are needed to optimise the care and quality of life of these children and their families.

Additional file 1. Nine Dimensions for Assessing the Level of Pragmatism in a Trial, as Proposed in the Pragmatic–Explanatory Continuum Indicator Summary 2 (PRECIS-2) Tool (Ford I, Norrie J. 2016) [31].

Today by the e-health and the telemedicine, many people are more and more interested by the improvement of disease knowledge on cardiovascular diseases and associated risk factors, personalized self management support follow-up and e-Health monitoring. MGEN is a not-for-profit complementary health insurance gave itself the ways to use the new digital tools in health. MGEN developed an original and personalized program VIVOPTIM for the primary prevention of the cardiovascular risks for their members. The VIVOPTIM Pilot program is based upon digital services and was experimented by November 2015 to December, 2017 with 8000 members of the MGEN, from 30 to 70 years old and resident in two French areas (Occitanie and Bourgogne Franche-Comté). The assessment of the experiment VIVOPTIM e -health program was positive for the personalized cardiovascular support and for their health. Therefore, the MGEN generalized the VIVOPTIM program of cardiovascular prevention, to the whole France on July 11th, 2018.

Resume L’efficience peut etre recherchee a toutes les etapes de la transfusion : le recrutement et la selection des donneurs, la selection des dons, le choix du type de don, pour des interventions programmees, la reduction des pertes sanguines ou la recuperation durant les interventions, l’utilisation d’erythropoietine. Nous avons cherche dans la litterature internationale, les donnees sur l’efficience des differentes approches qui permettent une meilleure utilisation des dons. Les donnees disponibles indiquent un contraste entre les preferences des responsables des politiques de sante transfusionnelle et les resultats des etudes. Les strategies de tres loin les moins efficientes sont celles qui consistent a augmenter le nombre de recherches de marqueurs biologiques sur les dons, alors que les strategies qui consistent a reduire les besoins transfusionnels ou a mieux gerer ceux-ci semblent preferables economiquement.

The systematic use of ultrasound during pregnancy aims at birth defect detection. Our objective was to assess the economic efficiency of prenatal ultrasound screening for fetal malformations.We carried out a literature review on Medline via PubMed between 1985 and 2015, from the economic perspective of the prenatal ultrasound screening for fetal malformations.The literature on this subject was sparse and we selected only twelve articles presenting relevant economic data, of which only eight were proper medico-economic studies. We found arguments for the economic effectiveness of ultrasound screening for fetal malformation detection, which is largely linked to the terminations of pregnancies and to the cost of the handicaps "avoided". However, none of the reviewed articles could reach medico-economic conclusions. Additionally, we highlighted various elements making economic analyses more complex in this field: the choice of the method, the uncertainty around two essential parameters (the efficiency of ultrasound and the costs of procedures) and the difficulties to compare or to generalize results. We also noticed important methodological heterogeneity among the studies and the absence of French study.Previously published data are insufficient to assess the economic efficiency of prenatal ultrasound screening for fetal malformations.

Introduction Selon les recommandations de l’European Medical Agency (EMA), les immunoglobulines polyvalentes sont indiquees dans le traitement substitutif des deficits immunitaires secondaires (DIS) dans des indications precises : les hypogammaglobulinemies severes consecutives a l’allogreffe de cellules souches hematopoietiques (HSCT) ou myelome multiple (MM) et leucemie lymphoide chronique (LLC) avec infections recurrentes et echec d’antibiotherapie. A notre connaissance aucune etude n’a evalue l’adequation des prescriptions a ces recommandations. Objectif Evaluer l’adequation des prescriptions d’Ig au cours des DIS entre la pratique courante et les recommandations de l’EMA. Les objectifs secondaires sont l’evaluation de l’efficacite des IgIV et IgSC dans la prevention d’infection pour l’ensemble de la population des patients traites, la correlation de l’efficacite des IgIV ou IgSC avec la dose, le niveau residuel d’IgG et le traitement antibiotique pour l’ensemble de la population, l’evaluation de la tolerance des IgIV et IgSC pour l’ensemble de la population. Patients et methodes Il s’agit d’une etude de cohorte retrospective, multicentrique s’adressant a l’ensemble des malades ayant recu, durant l’annee 2012, une preparation d’immunoglobulines par voie intraveineuse (IgIV) ou sous-cutanee (IgSC) dans le cadre du traitement de leur DIS. Les donnees ont ete recueillies pendant un an a partir de la premiere prise de traitement par immunoglobuline. L’etude a porte sur seize hopitaux d’Ile de France. Les donnees ont ete recueillies de maniere retrospective concernant les criteres definis par les recommandations, les donnees d’efficacite et de tolerance des IgIV ou IgSC. Resultats Trois cent soixante-huit patients ont ete analyses dans l’etude. Les pathologies motivant la prescriptions d’IgIV ou IgSC etaient : les lymphomes non-Hodgkiniens (LNH), 82 (22,3 %), les MM : 76 (20,7 %), les LLC : 64 (17,4 %) et autres indications pour 79 (21,5 %) patients. Ces autres indications sont majoritairement representees par la transplantation d’organe solide, les maladies inflammatoires/auto-immunes et l’asthme. Seuls 140 patients (38 %) ont beneficie d’une supplementation IgIV ou IgSC respectant les recommandations de l’EMA, 196 (53,3 %) et 262 (76,2 %) patients respectivement ont beneficies d’une antibioprophylaxie ou avaient un taux d’IgG Conclusion Dans cette etude retrospective multicentrique portant sur 368 patients recevant une supplementation par IgIV ou IgSC, seules 38 % des prescriptions respectaient recommandations de l’EMA. Cette discordance entre la pratique quotidienne et les recommandations souligne l’importance du mesusage des IgIV et IgSC dans les DIS.

Resume Le diabete est une maladie chronique et une Affection de longue duree (ALD) qui touche 2,5 millions de personnes traitees en France. Les couts annuels de prise en charge (ALD et hors ALD) sont estimes, en France, a 18 milliards d’euros. Une evaluation economique appliquee a la maladie a recherche si une action globale de depistage et de prevention des complications serait efficiente. Le depistage est efficient pour toute la population entre 40 et 70 ans, meme en supposant que seuls 50 % de la population seront testes ; il est d’autant plus efficient que l’on s’adresse aux tranches d’âge elevees. Les evaluations economiques des strategies de prise en charge du diabete s’orientent maintenant vers les changements organisationnels, plus que vers les innovations therapeutiques. La pertinence d’une approche par pathologie (le diabete, l’hypertension arterielle, l’obesite) ou d’une approche populationnelle, compte tenu de la frequente intrication des pathologies, reste un debat d’actualite dans les differents pays europeens.

Background: The DENERHTN trial (Renal Denervation for Hypertension) confirmed the blood pressure–lowering efficacy of renal denervation added to a standardized stepped-care antihypertensive treatment for resistant hypertension at 6 months. We report the influence of adherence to antihypertensive treatment on blood pressure control. Methods: One hundred six patients with hypertension resistant to 4 weeks of treatment with indapamide 1.5 mg/d, ramipril 10 mg/d (or irbesartan 300 mg/d), and amlodipine 10 mg/d were randomly assigned to renal denervation plus standardized stepped-care antihypertensive treatment, or the same antihypertensive treatment alone. For standardized stepped-care antihypertensive treatment, spironolactone 25 mg/d, bisoprolol 10 mg/d, prazosin 5 mg/d, and rilmenidine 1 mg/d were sequentially added at monthly visits if home blood pressure was ≥135/85 mm Hg after randomization. We assessed adherence to antihypertensive treatment at 6 months by drug screening in urine/plasma samples from 85 patients. Results: The numbers of fully adherent (20/40 versus 21/45), partially nonadherent (13/40 versus 20/45), or completely nonadherent patients (7/40 versus 4/45) to antihypertensive treatment were not different in the renal denervation and the control groups, respectively ( P =0.3605). The difference in the change in daytime ambulatory systolic blood pressure from baseline to 6 months between the 2 groups was –6.7 mm Hg ( P =0.0461) in fully adherent and –7.8 mm Hg ( P =0.0996) in nonadherent (partially nonadherent plus completely nonadherent) patients. The between-patient variability of daytime ambulatory systolic blood pressure was greater for nonadherent than for fully adherent patients. Conclusions: In the DENERHTN trial, the prevalence of nonadherence to antihypertensive drugs at 6 months was high (≈50%) but not different in the renal denervation and control groups. Regardless of adherence to treatment, renal denervation plus standardized stepped-care antihypertensive treatment resulted in a greater decrease in blood pressure than standardized stepped-care antihypertensive treatment alone. Clinical Trial Registration: URL: http://www.clinicaltrials.gov . Unique identifier: NCT01570777.

Drugs used in oncology represent more than half of the innovative and costly drugs which are not covered by a Group Homogene de Soins (DRG type classification) within the context of the case-mix based payment system (termed T2A). For these drugs, good practice reference guidelines have been drawn up by scientific societies and registration agencies. Recognised indications, relevant indications and situations where the treatment should not be prescribed are defined by the National Institute of Cancer. The reference guidelines should lead towards the good use of these drugs and allow the sick funds to control prescriptions. They should evolve with time, which means that bibliographic monitoring and independent expert opinion is necessary to update them as science provides new data. Manufacturers are involved in this process which in no case should undermine developmental efforts leading to registration. The objective of this protocolisation is to allow all patients early and legitimate access to drugs representing real therapeutic progress. These reference guidelines should be integrated into the life-cycle of a drug and should give rise to new developments allowing the good use of cancer products in situations which have been properly validated.

Introduction Les paralysies recurrentielles (PR) sont retrouvees chez 3 % des patients apres thyroidectomie. L’utilisation du neuromonitoring (NIM) des nerfs recurrents se generalise. Le but de notre travail etait d’evaluer l’impact du NIM sur le taux de PR apres thyroidectomie. Methodes Cette etude prospective, multicentrique compare les taux de PR apres thyroidectomie totale avec ou sans NIM. Les patients ont eu une laryngoscopie postoperatoire systematique. Resultats Parmi les 1328 patients inclus, 807 (60,8 %) ont ete operes avec le NIM et 521 sans NIM. En postoperatoire, 131 (9,93 %) ont presente une PR. Dans le groupe NIM, 69 (8,6 %) avaient une PR ; 38 avaient une PR partielle (4,7 %) et 31 (3,8 %) une complete. Dans le groupe sans NIM, 62 (12,1 %) avaient une PR ; 38 (7,4 %) une PR partielle et 24 (4,7 %) une complete. Cette difference etait significative en analyse univariee (OR : 0,68, IC 95 % : 0,47–0,98, p = 0,003). La difference n’etait plus significative en analyse multivariee (OR : 0,74, IC 95 % : 0,47–1,17, p = 0,19). Les sensibilite, specificite, valeur predictives positive et negative etaient respectivement de 29 %, 98 %, 61 % et 94 %. Conclusion Dans notre etude, le NIM ne permettait pas de diminuer significativement le taux de PR. Sa bonne specificite permet de prevoir de facon fiable une bonne mobilite des cordes vocales.

Resume Contexte Des etudes recentes ont montre l'efficacite du depistage de la trisomie 21 par le test combine du premier trimestre reposant sur l'âge maternel, les marqueurs seriques (β-HCG libre, PAPP-A) et la mesure echographique de la clarte nucale. Cependant, ces etudes n'incorporaient pas les performances de l'echographie de routine de 20–22 semaines d'amenorrhee (SA), largement realisee. Methodes Nous avons realise une etude interventionelle, multicentrique, en population generale dans le departement des Yvelines dont le but etait d'evaluer la performance du test combine du premier trimestre, suivi par l'echographie du second trimestre et/ou le dosage des marqueurs seriques maternels (β-HCG libre et alpha-fœtoproteine ou HCG totale, alpha-fœtoproteine et œstriol non conjugue) quand ce depistage serique etait prescrit incidemment. Les taux de detection et de tests positifs ont ete estimes en utilisant une methode de correction pour les issues non verifiees. Une analyse des couts a ete egalement effectuee. Resultats Pendant la periode d'etude, 14 934 femmes ont ete incluses. Cinquante et un cas de trisomie 21 ont ete observes, donnant une prevalence de 3,4 pour 1000 grossesses. Parmi ceux-ci, 46 cas ont ete diagnostiques, dont 41 au premier trimestre et cinq au second trimestre. Parmi les cinq cas de trisomie 21 non depistes, tous ont ete diagnostiques en postnatal apres une grossesse non compliquee. Les taux de detection et de tests positifs du depistage combine du premier trimestre ont ete de 79,6 et 2,7 %, respectivement. Ces taux ont atteint 89,7 et 4,2 %, respectivement quand le test combine a ete complete par l'echographie du second trimestre. Le cout moyen de la procedure complete du depistage a ete de 108 € par femme et le cout par grossesse affectee de trisomie 21 diagnostiquee a ete de 7,118 €. Conclusion Nos resultats suggerent qu'une approche interventionelle pragmatique en deux etapes utilisant le test combine du premier trimestre suivi par l'echographie du second trimestre est une option souhaitable et acceptable pour le depistage de la trisomie 21 au cours de la grossesse.

Prostate cancer is the most frequent cancer and the third leading cause of cancer death in men in France. The development of treatment for prostate cancer is fast and sometimes relies on costly innovations. Medico-economic studies are however rare in this area. This literature review aims to summarize available medico-economic data on the initial management of localized prostate cancer and discuss the quality and usability of existing economic studies on the subject.Literature review was done using PubMed and Cochrane databases. Studies and articles were selected based on several criteria: population with initial treatment for localized prostate cancer (without metastasis), comparative studies with surgery as control treatment, studies in countries members of the OECD, articles in English or French published between 2004 and 2014.The surgical robot, one of the newest innovations, is more expensive than conventional open surgery or no robotic laparoscopy, even if it is associated with a reduction of the original period of stay. Radiation therapy seems more expensive than surgery as initial therapy of localized prostate cancer.Conclusions remain limited because of the rarity of reliable health economic studies on the subject.

The increasing pressure on healthcare resources affects blood donation and transfusion. We attempted a survey of the efficiency of different strategies, actual or proposed to improve the management of blood products. We found an important disconnect between the cost effectiveness ratio of strategies and their uptake by policy makers. In other words, the least efficient strategies are those which increase transfusion safety by increasing the number of biological markers and are those preferred by health authorities in developed countries. Other more efficient strategies are more slowly implemented and included a systematic use of transfusion guidelines, reducing blood losses or increasing pre operative blood levels in elective surgeries.

Les medicaments utilises en cancerologie representent plus de la moitie des medicaments innovants et couteux pris en charge hors d'un groupe homogene de soins dans le cadre de la tarification a l'activite. Pour ces medicaments, des referentiels de bon usage elabores par les societes savantes, les agences d'enregistrement et l'Institut National du Cancer (INCa) definissent les indications reconnues, les indications pertinentes et les situations ou le traitement ne doit pas etre prescrit. Ces referentiels devraient tendre a un bon usage de ces medicaments et permettre un controle des prescriptions par les organismes payeurs. Ces referentiels se doivent d'etre evolutifs, d'ou la necessite d'une veille bibliographique et de l'appel a des experts independants pour une actualisation en fonction des donnees nouvelles de la science. Les industriels sont impliques dans cette demarche qui ne doit en rien se substituer aux efforts des developpements pouvant conduire a un enregistrement. L'objectif de cette protocolisation est de permettre un acces precoce et legitime de tous les patients aux medicaments representant un reel progres therapeutique. Ces referentiels doivent s'integrer dans la vie du medicament et doivent permettre de nouveaux developpements permettant le bon usage des produits de cancerologie dans des situations correctement validees.

Resume La schizophrenie est une maladie chronique qui necessite un traitement au long cours. La continuite du traitement est un element cle de la prise en charge permettant une reduction du nombre de rechutes et du nombre de re-hospitalisations. Les antipsychotiques atypiques administres quotidiennement permettent un gain de tolerance, d’efficacite et une amelioration de l’observance par rapport aux neuroleptiques conventionnels. Objectifs L’objectif de cette etude medico-economique est d’evaluer si le benefice clinique attendu de risperidone injectable a liberation prolongee (LP), selon une hypothese de prix, s’accompagne ou non d’un surcout pour l’assurance maladie. La risperidone injectable LP a ete comparee a l’olanzapine et a l’haloperidol decanoate. Methode Une modelisation pharmaco-economique de type cout-efficacite comparant ces 3 alternatives therapeutiques, pendant 2 ans, a ete realisee. L’hypothese principale est qu’une amelioration de l’observance grâce a une forme injectable a liberation prolongee entraine une augmentation de l’efficacite. La perspective adoptee est celle de l’assurance maladie. Les probabilites de transition et les couts estimes ont ete determines a l’aide de donnees publiees et d’avis d’experts. Seuls les couts directs ont ete pris en compte. Le critere d’efficacite retenu est le taux de patients traites avec succes, celui-ci etant defini comme le maintien du traitement pendant 2 ans. Resultats Parmi les patients traites par risperidone injectable a LP, 82,7 % sont toujours traites avec succes a 2 ans 74,80 % et 57,3 % respectivement pour olanzapine et haloperidol decanoate. Le cout direct par patient traite pendant 2 ans avec risperidone injectable LP est de 14 055 r. Ce cout est de 14 351 r avec olanzapine et de 17 203 r avec haloperidol decanoate. Conclusion Les resultats observes dans cette modelisation montrent que le traitement par un antipsychotique atypique sous forme injectable a liberation prolongee permet une meilleure continuite du traitement, reduit les rechutes, le niveau des ressources consommees et par consequent permet une diminution des couts.