OBJECTIVES: France has a long lasting experience in early-entry program (ATU). About 30,000 patients get access to innovative therapies through an ATU every year. Eligible medicines need to target a severe condition with no therapeutic alternative and have strong signal of favorable benefit risk ratio. Free pricing is applied during ATU period and once the negotiated price is settled, companies have to reimburse the difference. This study objective was to provide an assessment of the price discount achieved after ATU to access reimbursement.

METHODS: We identified cohort ATU (ATUc) granted between 2014 and 2018 on medical agency (ANSM) website, and collected related information regarding ATU period and HTA assessment on HAS website. We collected prices for ATU and hospital-restricted products through internal proprietary sources as they are not publicly available, and ex-factory prices for retail products from AMELI database. Difference in price between the ATU one and the reimbursed one was expressed in % of reduction.

RESULTS: During the last 4 years, ATUc was granted for 67 products. ATU and reimbursed prices were both available for 22. On average, the price discount between ATU price and reimbursed price was 13%. However, looking more closely at this discount, we noticed it was highly variable among the 22 products with a wide range of 0% to 39%. Comparing various characteristics of each product (therapeutic class, HTA assessment, target population, product listed on top of DRG or not), no logical correlation has been established between the applied discount and these features.

CONCLUSIONS: This descriptive study shows that reimbursed prices for ATUc products are almost systematically discounted. But the lack of consistent correlation between the discount and product features also suggests that the ability of pharmaceutical companies to set a fair ATU price and to negotiate reimbursed price have a strong impact on the discount.