Early access to medicinal products
The Transparency Committee (TC) of the French National Authority for Health (HAS) is responsible for the scientific and medical assessment of medicinal products when pharmaceuticals companies submit applications to the French Minister of Health for registration on the lists of medicinal products reimbursed by National Health Insurance.
The remit of the HAS was extended with the publication of the French Social Security Financing Act for 2021¹ which completely reformed the authorising procedure for early and derogatory access to medicinal products by creating:
- Pre-marketing authorisation (formerly cohort Temporary Authorisation for Use - ATUc) and postmarketing authorisation (formerly Temporary Funding Scheme - PECT) early access (AP, accès précoce)
- Compassionate access (AC, accès compassionel) incorporating compassionate use authorisations (AAC, formerly ATUn) and compassionate prescription schemes (formerly RTU).
The HAS is now responsible for early access authorisation decisions. These decisions must be issued within short and regulated timeframes, allowing prompt access for patients to presumptively innovative medicinal products for indications with an unmet medical need. Authorisations for compassionate access are under the scope of competency of the ANSM.
The reform did not change the key role of the ANSM in establishing the presumption of efficacy and safety of an indication for which a marketing authorisation (MA) has not been granted. However, the reform gives the HAS the decision-making role in respect of early access authorisations and their public funding cover. This structure ensures that assessments and decisions are consistent by creating a continuum of access between derogation-based schemes and the common law funding scheme. The reform allows both institutions to collaborate within their respective area of expertise (registration in the list of treatments eligible for reimbursement as regards the HAS).
The doctrine enables the HAS to describe its methods in close collaboration with the ANSM: transparency, patient involvement, and observational/real-word data collection reinforcement.
By adopting an assessment doctrine that is specific to early access, the HAS has sought to provide useful information for stakeholders on how it will issue its authorisations because, beyond its transparency obligation, the HAS is very committed to giving visibility to its assessment methods and thus ensuring consistency in its decisions.
- Autorisation d’accès précoce aux médicaments - Doctrine d’évaluation de la HAS
- Accès précoce des médicaments : accompagnement des laboratoires - Guide
- Matrice de dossier type - Accès précoce
- Modèle de Protocole d’utilisation thérapeutique et de recueil de données (PUT-RD)_01102021
- Modèle rapport de synthèse AP
- Modèle de résumé de rapport de synthèse AP
- Calendrier des rendez-vous pré-dépôts pour les demandes d’accès précoce 2022
- Acces precoces - Infographie de la reforme