OBJECTIVES: In France, pricing of drugs is set during a negotiation with Authorities. Drugs without alternative could benefit from an early access program (ATU), and in that case companies are allowed to temporary charge with a free price. The Ministry of Health (MoH) publishes since 2018 the ATU amount per drug. We investigate whether ATU price is a predictive factor of the final negotiated list price.

METHODS: We matched the amount of the list of ATU products, from the MoH website, with their respective final negotiated price, from the official journal. Other criteria were also considered, such as the HTA appraisal and rating of medical value (SMR, ASMR).

RESULTS: Among 113 products, 23 drugs (20%) with early access had finalized the pricing negotiation. The mean difference between the early access price and the final list price was a discount of -18% (min-max: +26%;-45%). The final price was, in comparison to the early access amount: higher in only 1 product (innovative drug with a high value recognized: ASMR II), similar for 2 products (both with also a good value recognized: ASMR III), and discounted for the majority (87%). The level of discount varied from -7% to -45%. Highest discount was seen in ASMR IV products. We noticed that products with no improvement in value (ASMR V) have not yet a published price. Then, the early access amount was predictive of the final price in 8.7% of the cases.

CONCLUSIONS: Only a limited number of products with an early access price finalized the pricing negotiation within a 2 years-timeframe. The early access amount does not automatically lead to the final price, value is driving the outcome. Pricing transparency may be developed in the near future to overcome the uncertainty of temporary price and to allow predictive pricing scheme.