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 in France can be in several situations: either authorized abroad, under development or AMM being evaluated. There is also the case of drugs whose marketing authorization has been withdrawn, suspended or refused, or when the marketing of the drug is stopped, but there remains a need for some patients.