Are you ready for the regulatory changes to early access in France?

Changes to the existing ATU include two distinct pathways for drugs/therapies in clinical development and off-label including use in rare disease, that now include a commitment to collection of data. 

By Heather Manna, Director of Regulatory Affairs, RareiTi

The most recent French Social Security law of Decembe14, 2020 (Article 78- La Loi de financement de la sécurité sociale, FSSL) established changes to the existing ATU into two distinct pathways: 

  • early access authorization programme (Accès précoce aux médicaments, EAP) which includes the cohort ATU (cATU) and 
  • compassionate access programme (Accès compassionnel aux médicaments, CAP) which includes the nominative ATU (nATU). 

 The EAP route is intended to be used for access to treatments that are in clinical development, where the manufacturer plans to submit a Marketing Authorization Application (MAA).  The CAP route is intended for off-label use of approved treatments where the manufacturer has no intention to seek marketing authorization for the unapproved indication and relevant for use in rare disease.  The manufacturer must commit to funding for the collection of data in both the EAP and CAP routes, specifically ensuring data collection by the prescriber while being funded by the manufacturer in CAP.   

The French National Authority for Health (HAS) is now included as an early access decision maker, working together with ANSM.  The addition of HAS allows the introduction of the HTA perspective on innovation as a prerequisite. 

The new process requires drug manufacturers to submit requests to the Haute Autorité de Santé (HAS), not to ANSM. While the ANSM will still be consulted, the decision to approve requests will sit with HAS.  Data collected in ATU programs should be collected using a standardized approach, making it possible that the HAS will consider these data in clinical assessments of a drug/therapy, which may lead to faster HTA reviews by the (TC) and quicker time to reimbursement. 

The final decree was issued in France and the new ATU system was effective July 1, 2021.  For further information or expert advice on the new ATU in France contact me, Heather Manna RareiTiMind Early Access Consulting. RareiTiCare Patient Access Platform. RareiTi Data. Insights and Data for pre- approval programs. 

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