· Jul 16, 2021

New compassionate access and early access procedures for drugs in France

Since the beginning of July 2021, a new reform has replaced the ATU and RTU by two other procedures: compassionate access and early access. This reform has also modulated the missions of the HAS and ANSM in granting these procedures.

As a reminder, compassionate access (replaces the ATUn and RTU) is granted to drugs not intended to be marketed in the targeted indication (no development underway, no marketing authorization procedure planned). Early access (replaces the ATUc, ATUei, post-ATU and PECT) is granted for presumed innovative drugs, intended to be marketed and for which clinical data are available or being collected.

4 conditions are essential to access these procedures:

  • The drug must be intended to treat serious, rare, or disabling diseases.
  • There is no appropriate treatment available.
  • The implementation of the treatment cannot be delayed.
  • The drug is presumed to be innovative, compared to a possible clinically relevant comparator.

Among the new features of this regulation, the collection of real-life data is strengthened, through the establishment of a protocol for therapeutic use and the collection of patient follow-up data. All this data collection must be funded by the manufacturers and submitted to the HAS and the ANSM.

We remain of course at your disposal to advise and support you in such procedures.