OBJECTIVES: France has a long lasting experience in early-entry program (ATU). About 30,000 patients get access through ATU every year. Eligible medicines need to target severe conditions with no therapeutic alternative, and strong signal of favorable benefit risk ratio. This study objective was to provide a description of cohort ATU (ATUc) in France and provide relevant characteristics of involved products.

METHODS: We identified ATUc granted between 2014 and 2018 on medical agency (ANSM) website, collecting dates of ATUc start and end (when applicable) and of marketing authorization. HTA assessment, ASMR, SMR and size of targeted populations were extracted from HTA body (HAS) website. Data were computed to assess ATUc duration pre and post approval and post-ATU duration (extension of the patient access to treatment until price is settled).

RESULTS: ATUc was granted for 67 products. Oncology, hematology and neurology were the most represented therapeutic groups (respectively 27%, 15% and 15%). Targeted indications were usually rare: 31% was targeting less than 500 patients per year and 35% between 1,000 and 5,000. The great majority of involved indications obtained an ASMR IV (40%) or V (42%), while 18% obtain I-III contrasting with a range of 3 to 10% according to HAS statistics during 2012-2015. ATU duration was in average 340 days followed by 452 days in post-ATU until price is settled. 9% were considered not reimbursable.

CONCLUSIONS: This descriptive study supports that ATUc enhance chances of higher HTA scoring, while it may contribute to delay pricing negotiations. However products remain accessible during price negotiation but manufacturer cannot retail the product. The lack of reimbursement for 9% and the lack of acknowledged benefit for 82% contrast with ATU eligibility criteria. French health system remains the most generous in granting early-entry to innovation, but needs to use advantage of granted access to strengthen price negotiation.