OBJECTIVES:

In July 2021, the temporary authorisation for use (Autorisation Temporaire d’Utilisation, ATU) system was the subject of a major reform, initially published within the 2021 healthcare plan in December 2020. The presumption of innovation for the products compared with the most clinically relevant comparator was introduced among other evaluation criteria. What are the implications of this reform and what does this mean for the pharma industry?

METHODS:

This study scrutinizes the application of all non-COVID related medications submitted for an Autorisation d’accès précoce (AAP, which includes the previous cohort ATU) between July 2021 and July 2022. The three evaluation criteria of the presumption of innovation (i.e. new treatment approach with an appropriate development plan and that addresses an unmet medical need) were analysed and linked to the decision outcomes of the Haute Autorité de Santé (HAS) for each product.

RESULTS:

This study shows that most products assessed post-ATU reform met all three evaluation criteria and were granted AAP approval. In their evaluation, consideration of unmet medical need was particularly important for oncology products for HAS. The HAS showed to accept the absence of comparative data in the case of concurrent development of comparative studies vs. standard of care and with additional input from external experts. However, the absence of novel mode of action or treatment approach and / or the lack evidence of comparative clinical efficacy data without appropriate clinical development plans were key factors leading to product rejection.

CONCLUSIONS:

This study shows that the majority of non-COVID related products were granted AAP approval in the first months post-reform. Although this remains an initial observation, this research suggests that the reforms to the French early access programme, whilst more restrictive, have not significantly impacted patient access.