The law on financing of the social security system for 2021 changed considerably the early access authorization procedure and exceptional reimbursement of medicinal products in France by eliminating the temporary authorisation for use system (ATU), temporary reimbursement (PEC-T) and temporary recommendations for use (RTU) and creating:
- A Pre-MA early access programme (EA) (formally, a cohort ATU) and post-MA (formally post-ATU and PEC-T)
- The compassionate use access program (CA) incorporating, on one hand, compassionate use access authorisation (CUA) (formally, ATU-by named patient) and also via the framework of compassionate use prescription (formally, the RTU).
Decisions related to early access are taken by HAS, if applicable, after the opinion of ANSM, while decisions relating to compassionate use access are taken by ANSM.
Early access authorisation (AAP) involves an exceptional waiver procedure which enables access to certain medical products in one or more indications, with automatic reimbursement by national solidarity, upon request from a pharmaceutical company, whenever the following five conditions specified in article L.5121-12 of the PHC are satisfied:
- Efficacy and safety of this medicinal product are strongly presumed in light of results of therapeutic trials when the medicinal product does not have MA in the indication concerned;
- The medicinal product is indicated in a serious, rare or disabling disorder;
- No appropriate treatment exists;
- The implementation of treatment cannot be delayed;
- This medicinal product is assumed to be innovative with regard to a possible clinically relevant comparator.
An application for early access authorisation (AAP) must be requested whenever a medicinal product which has compassionate use access authorisation is the subject of a research study involving the human person (RIHP) for purposes of marketing in the indication considered, under conditions set out in II of article L. 5121-12 and in article R. 5121-74-1 II of line 5 of the PHC. In this case, the time period to request AAP involves a decision from the GD of ANSM and is framed by article D. 5121-74-1-1 of the PHC.
Compassionate access authorisation can co-exist with an application for pre-MA AAP in the same indication. The CUA defines early access authorisation granted in the indication considered. It should be noted that an CUA can involve only an indication without MA.
Article 78 of law no. 2020-1576 of 14 December 2020 on financing of the social security system for 2021 published in the Official Journal (OJ) of 15/Dec/2020.