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Background and importance In order to ensure the sustainability of the French healthcare system, the government launched two incentives to increase biosimilar use in August 2018, within the framework of the social security funding law. The first redirected 20% of the price difference between the reference product and its biosimilar to the hospital, for every biosimilar prescription from the hospital, and provided it in retail pharmacies. The second (called article 51) was an experiment where 40 hospitals were selected after a call for a proposal. The clinical units of these 40 hospitals received 30% of the price difference between the reference product and its biosimilar for every biosimilar prescription from the hospital. Aim and objectives The aim of this study was to compare the efficacy of both incentives 10 months after implementation in October 2018. Material and methods IQVIA Xponent data were used to evaluate public hospital prescriptions of etanercept. These are based on 14 000 retail pharmacy panels (60% of the French retail pharmacies) and allows observation of the number of boxes delivered in retail pharmacies linked to the initial hospital prescription. Data from the 40 hospitals selected in the experiment were compared with hospitals not in the experiment. We assessed savings that could be made if the experiment was extended to every hospital after 10 months Results In July 2019, the average use of etanercept biosimilar reached 44.2% (+19.5 points compared with October 2018) in the 40 hospitals selected in the experiment whereas it increased by 10.5 points in the other hospitals. After 10 months of the experiment, there was a difference of 12.3 points between the groups. The government expected to reach a difference of 15 points to prove the efficacy of this measure after 3 years. The 40 selected hospitals represent about 46% of potential etanercept prescriptions. If all hospitals reach 44.2% biosimilar use, the savings could be doubled, from 650k€ to 1.4M€. Conclusion and relevance The first results of this experiment show that incentives to prescribe etanercept biosimilars seem to have an impact on biosimilar use in France. References and/or acknowledgements Acknowledgements to Iqvia and Biogen France. Conflict of interest Corporate sponsored research or other substantive relationships: Iqvia and Biogen France.

Resume Objectifs L’hemophilie est une maladie hemorragique rare. Elle se traduit cliniquement par des hemorragies spontanees ou provoquees. Les facteurs antihemophiliques constituent le principal traitement. Ce sont cependant des medicaments tres onereux. Les objectifs de cette etude etaient de determiner le cout de ces traitements en hospitalisation ainsi que les facteurs l’influencant. Methode Une etude retrospective monocentrique a ete menee sur les patients atteints d’hemophilie congenitale hospitalises a l’hopital Cochin entre le 1er janvier 2014 et le 31 decembre 2015 et ayant recu au moins une injection de facteur antihemophilique pendant leur hospitalisation. Un recueil des donnees cliniques et des traitements recus pendant l’hospitalisation a ete realise pour chaque patient. Resultats Cinquante et un patients ont ete inclus dans l’etude avec un total de 68 hospitalisations. Le cout median des facteurs antihemophiliques par hospitalisation etait de 16 908 €. La part mediane des facteurs anthemophiliques sur le cout total de l’hospitalisation etait de 68,2 %. Le cout des facteurs par sejour etait plus eleve pour les hemophiles severes (p = 0,015) et pour les chirurgies majeures (p Conclusions Les facteurs antihemophiliques representent la part la plus important du cout d’hospitalisation d’un hemophile. Leur cout en hospitalisation varie en fonction de nombreux facteurs et est susceptible d’evoluer avec l’arrivee des facteurs antihemophiliques a longue duree d’action.

Resume Objectif Determiner la frequence du suivi plaquettaire et les risques hemorragiques associes a l’usage des anticoagulants injectables en vie reelle et estimer les couts associes. Methode Une analyse des donnees 2013 de l’echantillon generaliste des beneficiaires a ete realisee pour identifier les dosages plaquettaires effectues sur les periodes d’exposition aux anticoagulants injectables et les interruptions therapeutiques du fait d’une thrombopenie ou d’une transfusion. Ces evenements ont ete valorises et un differentiel de cout etabli. Resultats L’etude a porte sur 15 985 patients adultes representant 12 264 mois d’exposition aux anticoagulants injectables ; 2,8 % des sequences concernaient une heparine non fractionnee, 86,9 % une heparine de bas poids moleculaire et 13,1 % le fondaparinux. Les patients sous heparine non fractionnee etaient plus âges (77 vs 57 et 59 ans) avec des durees de traitement superieures (32,6 vs 25,1 et 21 jours). Le nombre mensuel moyen de dosages plaquettaires etait, apres ajustement, 1,36 fois inferieur chez les patients traites par fondaparinux par rapport aux heparines de bas poids moleculaire (p Conclusion Les patients sous fondaparinux effectuent significativement moins de dosages plaquettaires que les patients sous heparines de bas poids moleculaire sans risque hemorragique supplementaire. Cet aspect doit etre pris en compte dans l’evaluation des couts de ces traitements.

International audience; The development of new anticancer drugs has strongly contributed to improve patients’ outcomes. However, the question can be raised whether the sometimes minor benefits are worth the cost to both the individuals and the society. The aim of this medico-economic study was to evaluate trabectedin cost-effectiveness compared with end-stage care in the treatment of soft-tissue sarcoma. We retrospectively analyzed data from 45 patients treated with trabectedin for soft tissue sarcoma. Real-life data of clinical benefits and costs of trabectedin were compared with simulated end-stage treatment. Univariate and probabilistic sensitivity analyses identified key drivers of the incremental cost-effectiveness ratio. Trabectedin was associated with longer progression-free survival (4.0 months versus 1.6 months) and higher costs (€24,780 versus €15,490) compared with end-stage treatment. The incremental cost per year of progression-free survival gained with trabectedin was €46,104 (95% confidence interval; €45,039–€47,169). The results were relatively insensitive to changes. Trabectedin was cost-effective in the context of an orphan disease, when used to treat patients with a short life expectancy and few therapeutic options.

Background:Background: In 2005, the French Government implemented a new way of financing high-cost drugs for hospitals in order to promote innovation. Such drugs are gathered on a positive list, established by the Ministry of Health, with a reimbursement price cap. Hospitals still negotiate with pharmaceutical firms, who set their prices freely, and then charge the national health insurance according to their consumption, without budgetary constraints, but on the condition of good use of care. They are not allowed to charge a price higher than this ceiling price, which is called the 'responsibility tariff' (RT). This measure is included in another, larger reform, which concerns hospital financing through allotted amounts at a specific diagnosis-based level. The purpose of this add-on payment on top of the health funds is firstly to avoid heterogeneity in costs per diagnostic-related group and secondly to avoid an uncontrolled increase of prices due to a lack of interest in negotiation from hospitals, as supplementary funding could reduce hospital price sensitivity. Abstract: Objectives:Objectives: The aim of this work was to assess the bargaining power of hospitals with the pharmaceutical firms in the monopoly market of innovative cancer drugs since the implementation of this reimbursement price cap. Abstract: Methods:Methods: This study used data from the French Technical Agency of Information on Hospitals (ATIH; Agence Technique de l'Information sur l'Hospitalisation) and included 487 hospitals, which were public and non-profit private. The analysis was conducted on the cancer drugs of the regulated list. An index representing the ratio of the purchase prices to the RT was built from 2004 to 2007 in order to make a 'before-and-after' comparison. Abstract: Results:Results: Results showed a transient price decrease in 2005 before an alignment of patented drugs with regulated prices in the context of a dynamic market with a 22.5% yearly growth rate in value between 2004 and 2007. Abstract: Conclusion:Conclusion: Hospitals are able to impose the RT for single-brand drugs. However, they are no longer able to negotiate below the RT except for generic drugs. Negotiations take place upstream for setting the RT between the public authorities and the firms.

Resume Dans le service de psychopathologie de l’Enfant et de l’Adolescent du centre hospitalier Sainte-Anne a Paris, les patients presentant un trouble autistique beneficient d’une prise en charge pluridisciplinaire, avec parfois la prescription de psychotropes. Pour l’un d’entre eux, une instabilite majeure, une hetero et autoagressivite severes ont conduit a la prescription successive de psychotropes sans amelioration clinique. Un traitement par naltrexone a ete instaure en reference a de nombreuses publications presentant l’interet de ce medicament dans le traitement de l’autisme. Pour obtenir le produit sous forme de sirop adapte a la prise chez un enfant, nous avons fait une demande d’autorisation temporaire d’utilisation aupres de l’Agence francaise de securite sanitaire des produits de sante (Afssaps). L’evolution des symptomes comportementaux a ete suivie a l’aide de l’echelle de Conners et l’echelle de Nisonger. Les questionnaires ont ete remplis une fois par semaine par les parents et le patient a ete revu tous les 15 jours. Le suivi a dure 12 semaines. Le debut de traitement et la posologie de 1 mg/kg par jour ont ete a l’origine d’une recrudescence transitoire des comportements a problemes. C’est a la posologie de 0,75 mg/kg par jour que des ameliorations significatives ont ete observees sur les deux echelles de Conners et Nisonger. Les automutilations ont cesse. Les effets secondaires observes ont ete une sedation transitoire a l’initiation du traitement puis l’apparition d’une constipation moderee. Ce cas clinique confirme que le traitement par naltrexone en suspension buvable dans le cadre d’un trouble autistique severe de l’enfant offre une alternative therapeutique interessante en cas de troubles du comportement resistants a une therapeutique medicamenteuse classique.

International audience; Over the last 20 years, melatonin, a pineal hormone synthesized from serotonin, has been implicated in various studies on the autism spectrum disorder (ASD) and altered melatonin levels were detected in subgroups of subjects with ASD. Its effect on sleep disturbances got the attention of clinicians and several investigations were carried out to determine the usefulness and safety of melatonin administration in this disorder. Hypotheses were also raised regarding the possibility that the dysfunctional synthesis and secretion of melatonin detected in subgroups of subjects with ASD may increase the risk as well the severity of ASD. The purpose of this paper is to review our pharmacokinetic knowledge on melatonin and present results from recent studies on sleep disorders in autism, their treatment with melatonin and the impact of melatonin prescription in children with ASD evaluated in a Diagnostic Center for Autism Spectrum Disorder in Paris, France.

ISPOR 14th Annual European congress : "Rational Health Care Decision Making in Challenging Economic Times", 11-2011, Madrid, Espagne