Early Access Programs (EAPs) in France: Reasons for Refusal

Patients in France can get access early to drugs that are presumed innovative through EAPs. A new approval process was established on July 1, 2021 empowering the Haute Autorité de Santé (HAS) to grant EAPs. Drugs need to meet all 5 criteria set by law to get an authorization.

OBJECTIVES: The aim of this research is to list the reasons for refusal of EAPs in the first 2 years of the new process, which now includes review by the Transparency Commission of the HAS and final decision by the HAS College, and to identify the main and recurring reasons.

METHODS: Systematic analysis of the HAS opinions refusing the grant of EAPs between July 1, 2019 and June 30, 2023.

RESULTS: Among 164 initial applications for early access authorization, mainly in the field of oncology, the HAS College issued 27 refusals (16.5%).

Four of these drugs were refused on the presumption that efficacy and safety could not be established. One drug was refused on the basis that it did not treat a rare and serious disease, a basic requirement of EAP. Most refusals were due to existing treatment alternatives (17/23, 73.9%) and lack of comparative data to establish the added value of the drug candidate (19/23, 82.6%). Only 3 drugs were presumed innovative and these were refused EAP approval because treatment alternatives existed.

CONCLUSIONS: Refusals of EAPs by HAS in France are low/uncommon (16.5 %). The reasons for refusal are widely the same: alternative therapies exist in the majority of cases and comparative data are missing, thus, the added benefit and the “innovative” status, cannot be established. The criteria are very stringent, and manufacturers may be underestimating the existing treatment alternatives and the need for comparative data.