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KCE Reports 133 Special Solidarity Fund 65
7.1.1.1 Authorisation for temporary use in France
The French ATU system allows early access to new promising drugs that are not
covered by a MA in France. However, an ATU may concern medicinal products that
already have a MA abroad or drugs under development. In most cases, however, an
application procedure is pending at EMEA. The ATU procedure does not apply to the
use of a medicinal product having a MA in France for an indication other than that
envisaged in its MA : This off label use is the unique responsibility of the prescriber.
The following general conditions have to be fulfilled:
1. the drugs are used for the treatment of serious or rare diseases;
2. there is no other treatment available;
3. cost-benefit analysis of the treatment was conducted, with a positive result.
The drug which has been authorised for temporary usage needs to be used only for
therapeutic purposes and is not expected to hinder any further clinical trials.
There are two types of temporary use authorisation:
• nominated ATUs, provided for a single patient not taking part in a clinical
trial, under the responsibility of the prescribing doctor;
• and cohort ATUs for groups of patients, established at the request of the
holder of distribution rights.
A cohort ATU must be accompanied by a simultaneous demand for a MA or an
intention to introduce a MA (marketing authorization) dossier in the near future.
Moreover a cohort ATU is subordinated to the implementation of a protocol for
therapeutic use and information collection, established by AFSSAPS in collaboration
with the manufacturer. AFSSAPS may consider it as necessary that such a protocol is
also set up for certain drugs products made available within the framework of
nominative ATU.
The use of medicinal products subject to ATU cannot replace a clinical trial and the aim
is not one of investigation. The decision for ATU must not slow down the
implementation or the continuation of clinical trials, alone intended to determine
precise and essential elements concerning the benefit/risk ratio of a medicinal product.
Indeed, only clinical trials make it possible to collect reliable data, in particular in terms
of efficacy, safety of use, medicinal product interactions and therapeutic strategies, while
authorizing the access to medicinal products without MA. Making medicinal products
available according to the ATU procedure or for clinical trials depends in particular on
the level on information available on the medicinal product in question. Generally, in the
early stages of the development of the medicinal product, clinical trials must be
favoured.
Evaluation
NOMINATIVE ATU
The evaluation criteria concern the pharmaceutical quality (viral safety if applicable) of
the drug, its safety and efficacy in the indication claimed in the ATU and the absence of
any therapeutic alternative.
Each named ATU application is studied by the AFSSAPS, assisted by experts. There are
5 evaluators and 1 chief of the ATU unit. Three of the members are pharmacists, the
others are physicians. Given the large number of requests (+/- 20.000/year) the less
complicated cases are handled by one of the members of the unit. It mostly concerns
cases that are similar to earlier cases and/or renewals. More complicated problems are
dealt with collectively. For very rare diseases an appeal is made to external experts.
The agency notably relies on a dossier on the medicinal product supplied by the holder
of distribution rights at its request and, if necessary, by the ATU applicant and including,
in particular:
• a copy of the authorisation obtained abroad, if applicable;