Accès au marché précoce des médicaments : comparaison France/Canada de deux systèmes d'accès à des médicaments non approuvés pour des patients en impasse thérapeutique

Some patients need treatments not yet approved by health authorities. The aim of this study was to describe and compare two special access programs, the autorisation temporaire d'utilisation (ATU) in France, and special access program (SAP) in Canada, and their consequences on patients' care. We compared data obtained from ANSM and health ministry in France and Health-Canada in Canada, as well as ANSM's nominative ATU (ATUn) and cohort ATU (ATUc) lists and Ontario Pharmacists Association's SAP drugs list in 2014. In France, after ANSM authorization, the drug can be obtained and is reimbursed, whereas in Canada the manufacturer can decide after Health-Canada authorization to deliver or not the drug, without reimbursement. The implication of hospital pharmacist is higher in France (co-responsibility of drug use with the prescriber). In 2014, 41 drugs were authorized for use within ATUc (median duration 4.5 months) and 231 within ATUn (25,521 requests). In Canada, a total of 166 drugs were on Ontario Pharmacists Association's SAP drugs list (only available data on SAP). The ATU system in France seems to be more advantageous for patients than SAP. However, it is important to conduct additional studies to confirm that these expanded accesses do not represent a way of bypassing health authorities' evaluations.