Early Access Program for drugs in France

Promoting rapid, wide and framed access to innovation for concerned patients in France

ATU (Temporary Use Authorizations)

In France, access to medicinal products not benefiting from a Marketing Authorisation and not used in clinical trials is conditioned by obtaining an ATU by the French Competent Authority (ANSM).

 

This provision allows access to new promising drugs not covered by a MA in France (approved abroad or being developed) when there is an unmet medical need.

 

ATUs are granted on a purely derogatory, exceptional and temporary basis, when the following conditions are met (article L5121-12 from the French Public Health Code):

– Treating, preventing or diagnosing serious or rare diseases,

– In the absence of a suitable therapeutic alternative (medicinal product or other) available in France,

– When the benefit /risk ratio of the medicinal product is presumed positive.

 

Medicines without a marketing authorization (MA) in France can be in several situations: either authorized abroad, under development or MA being evaluated. There is also the case of drugs whose MA has been withdrawn, suspended or refused, or when the marketing of the drug is stopped, but there remains a need for some patients.

 

 

Two types of ATUs

Nominative ATU (ATUn)

  • Delivered for one named patient (if she/he cannot enter a clinical study).
  • On the request and responsibility of a prescribing physician.
  • ATU granted by ANSM for the duration of the patient treatment.
  • The medicinal product is likely to present a benefit for this patient.

 

 

 

 

  • Follow up of patients and data collection can be performed according to a protocol for therapeutic use (PTU) – ANSM decision

Cohort ATU (ATUc)

  • Delivered for a group of patients, for a specific indication.
  • ATUc application filed to ANSM by the pharmaceutical company, with the commitment to submit a MAA within a stated time.
  • ATU for one-year duration, renewal possible.
  • Safety and efficacy of the product are highly presumed.
  • SmPC, patient information leaflet, Labelling.

 

  • Follow up of all patients and data collection are always performed according to a protocol for therapeutic use (PTU)
  • Regular data reporting to ANSM (periodic reports)

PTU: Protocol for therapeutic use and data collection

A PTU is established between the ANSM and the applicant (Article R5121-70).

 

The aim of this PTU is to:

– Provide physicians and pharmacists with information on the product, conditions for using the product and monitoring patients:

– Organize the pharmacovigilance

– Organize data collection and analysis 

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