The temporary authorisation for use (Autorisation Temporaire d’Utilisation, ATU) programme in France is intended to provide early access to medicines for patients with a severe or rare disease with high unmet need (with no authorised therapeutic alternatives available).
While the ATU system was initially designed to ensure access to new therapies for human immunodeficiency viruses (HIV), it has progressively expanded to cover all therapy areas, including oncology, haematology and rare diseases.
As a result of expanded use of cATUs over recent years, funding of new products through the system has grown to over €1 billion a year . In 2020, the French ministry of health proposed several reforms to the ATU system, the full implications of which remain unclear. But it is important to question if the reforms will improve early access for patients or make the situation more challenging.