Risk‐sharing approach for managing factor VIIa reimbursement in haemophilia patients with inhibitors

Managing orthopaedic surgery in haemophilia patients with inhibitors [1] raises formidable economic issues that are similar to those raised by the new targeted anticancer agents [2]. 1 The payment-by-results approach [3,4] has been designed to manage high-cost therapeutic problems in which an immediate decision is needed in terms of access to treatment and reimbursement, but effectiveness data and therapeutic guidelines are still preliminary. Thus far, this approach has been mainly used to handle innovative antineoplastic agents as well as high-cost medical devices [3–5]. At our institution, an experimental reimbursement model for managing peri-operative treatment with factor VIIa in haemophilia patients with inhibitors has been developed in which the economic risk of a more than average expenditure per patient is equally shared by the third payer (our regional health system) and the manufacturer. This model is conceptually very simple: in fact, all FVIIa doses exceeding the cumulative limit of 5 mg kg in a given patient are subjected to 50% discount; in addition, an appropriate registry is set up to document analytically the dosing history of all operated patients. According to this model, the so-called excess dose is first calculated as follows: