[Assessment of the budgetary impact of an emicizumab therapy introduction for patients with severe haemophilia A without inhibitor]. / Évaluation de l'impact budgétaire de l'introduction d'un traitement par émicizumab chez des patients atteints d'hémophilie A sévère congénitale sans inhibiteur.

Since March 2019, émicizumab is indicated for the treatment of patients with severe haemophilia A without inhibitor. This therapy's price amounts approximately to €33 600 per 4 weeks for a 70kg patient which represents about two times more than a factor VIII concentrates treatment's price. This study aims to assess the budgetary impact for the French Health Insurance of an émicizumab therapy introduction for patients with severe haemophilia A without inhibitor. It was an observational, retrospective, and monocentric study. Every severe haemophilia A without inhibitor patient over 18 years old followed at the Cochin Hospital haemophilia treatment centre who received émicizumab from June 2020 and for at least one year have been included. The budgetary impact was estimated by comparing the total costs of patient care the year before versus the year after émicizumab initiation. Total costs of patient care included prices of i) treatments consumed, ii) consultations with specialist physicians, iii) hospitalizations and iv) imaging procedures. Thirty-eight patients were included. The total cost of patient care increased significantly the year after émicizumab introduction (P < 0.0001). On average, this cost was estimated at €537 887 ± €137 139 per patient whereas it was at €151 442 ± €94 708 the year before. While costs of physician consultations increased, no significant difference has been reported about hospitalizations and imaging costs. Over a one-year period, émicizumab therapy significantly increased the total costs of patient care. It is mostly caused by the drug price itself.

[Market access agreements: Definition and features. Could MAA be defined as a solution for immunotherapy funding by French health system?] / Les contrats d'accès au marché : définition et caractéristiques. Une solution pour garantir la prise en charge de l'immunothérapie par le système de santé français ?

Over the last few years, many therapeutic innovations have been approved and marketed in France, within a strained financial setting. Legal dispositions allowed manufacturers (LEEM - les enterprises du medicament) and the economic committee for health products (CEPS) to contract various confidential market access agreements to contain health product expenses. The purposes of this article are to define and describe these different existing market access agreements and to open discussion on their applicability to the problematic of immune-oncology drugs financing. Financial agreements, which led to major savings (discounts refunded to the public payer), have not responded completely to the therapeutic innovations financing problems. Performance agreements (funding based on real-life data and effectiveness of the drug) constitute a hope for health products financing, but major methodological challenges for their use in routine restrict them to rare cases only today. Even though several financial agreements could partly respond to this problematic, use of performance agreements could really constitute an interesting track to tackle this issue.

Hospital reimbursement price cap for cancer drugs: the French experience in controlling hospital drug expenditures.

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. 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. 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. 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. 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.