French Public Health Code
In forceI. - Articles L. 5121-8 and L. 5121-9-1 do not preclude the exceptional use of certain medicinal products for specific therapeutic indications under compassionate access when the following conditions are met:
1° The medicinal product is not the subject of research involving the human person for commercial purposes;
2° No appropriate treatment exists;
3° The efficacy and safety of the medicinal product are presumed on the basis of available clinical data and, where the indication concerns a rare disease, on the basis of work and data collected by healthcare professionals under conditions defined by decree by the Conseil d'Etat.
This exceptional use is carried out in application of either an authorisation as defined in II of this article, or a compassionate prescription framework as defined in III.
II. - At the request of a prescribing doctor, with a view to treating a serious, rare or incapacitating disease, the Agence nationale de sécurité du médicament et des produits de santé may authorise, under the compassionate access defined in I and for a maximum period of one year, renewable, the use for a named patient of a medicinal product which does not have, whatever the therapeutic indication, of a marketing authorisation issued by the Agence nationale de sécurité du médicament et des produits de santé or by the European Commission, or of a medicinal product whose marketing has been discontinued and whose marketing authorisation does not cover the therapeutic indication requested.
By way of derogation from 1° of the same I, a medicinal product which is the subject, at a very early stage, of research involving the human person in the indication in question may be the subject of an authorisation under this II when the implementation of the treatment cannot be deferred and the patient cannot participate in this research, provided that the holder of the exploitation rights for the medicinal product undertakes to submit, within a period , defined by decree, starting from the granting of the first authorisation issued by the Agency in this indication, an application for early access as defined in Article L. 5121-12 in this indication.
Medicinal products authorised in application of this II are imported, where applicable, by in-house pharmacies.
III. - On its own initiative or at the request of the Minister for Health or the Minister for Social Security, the Agence nationale de sécurité du médicament et des produits de santé may establish, under the compassionate access defined in I and for a renewable period of three years, a prescription framework relating to a medicinal product which is the subject, for other indications, of a marketing authorisation issued by the Agency or by the European Commission, in order to ensure the safety of a prescription which does not comply with this authorisation.
By way of derogation from 2° of the same I, the existence, in the indication concerned, of a treatment with a marketing authorisation does not prevent the establishment of a compassionate prescription framework for a medicinal product when it is not a medicinal product with the same active principle, the same strength and the same pharmaceutical form. This medicinal product may be prescribed in accordance with the framework thus established, provided that the prescriber considers that it meets the patient's needs at least as well as the medicinal product which has been granted marketing authorisation for this indication.
IV. - Before issuing an authorisation or establishing a framework for prescribing under compassionate access, the Agence nationale de sécurité du médicament et des produits de santé informs the marketing authorisation holder or, where applicable, the holder of the exploitation rights for the medicinal product concerned.
V. - Compassionate access authorisations and prescription frameworks are accompanied by a therapeutic use and patient monitoring protocol which specifies the conditions for gathering information concerning the efficacy, adverse effects and actual conditions of use of the medicinal product and, where applicable, the characteristics of the population benefiting from the medicinal product authorised or supervised in this way. When it is linked to an authorisation granted under the second paragraph of II or to a compassionate prescription framework, this data collection is financed by the holder of the rights to use the medicinal product or the company which uses it. The requirement for a therapeutic use and patient monitoring protocol provided for in this paragraph may be waived where there is sufficient experience of the conditions of use of the medicinal product in the indication covered by the compassionate prescribing framework or where there is another comparable medicinal product with a marketing authorisation in this indication.
For medicinal products authorised or covered by a compassionate access prescribing framework, prescribers ensure, where applicable, the collection and transmission of data on the follow-up of patients treated as required by the Agence nationale de sécurité du médicament et des produits de santé (French National Agency for the Safety of Medicines and Health Products) in accordance with the procedures laid down by the Agency for each medicinal product concerned. These procedures ensure that medical confidentiality is respected.
VI. - The prescriber informs the patient, his/her legal representative if he/she is a minor, as well as the person in charge of the protection measure if he/she is an adult subject to a legal protection measure with representation relating to the person, or the trusted support person that he/she has designated in application of article L. 1111-6, that the medicinal product is not being prescribed under a marketing authorisation but under compassionate access pursuant to II or III of this article, where applicable, the absence of a therapeutic alternative, the risks incurred and the constraints and benefits likely to be provided by the medicinal product. He will write on the prescription: "Prescription under compassionate access outside the framework of a marketing authorisation".
The prescriber also informs the patient of the conditions for reimbursement by the health insurance scheme for the medicinal product prescribed for the indication in question.
The procedure followed is recorded in the medical record. In the case of a medicinal product covered by a compassionate prescribing framework, the reasons for prescribing the medicinal product are recorded in this file, except where there is sufficient experience of the conditions of use of the medicinal product in the indication covered by the compassionate prescribing framework or where there is another comparable medicinal product with a marketing authorisation in this indication.
VII. - The authorisation or compassionate prescribing framework may be suspended or withdrawn by the Agence nationale de sécurité du médicament et des produits de santé if the conditions defined in this article are no longer met or for public health reasons.
VIII.However, and where applicable, by way of derogation from I and II:
1° Failure to submit an application for early access authorisation under Article L. 5121-12 within the period mentioned in the second paragraph of II of this article, or the refusal of such an application, shall not prevent the maintenance of a currently valid compassionate access authorisation or its renewal to take account of the particular situation of a given patient, for a maximum period laid down by decree;
2° The implementation of research involving the human person for commercial purposes in a given indication shall not prevent the maintenance of a previously established compassionate prescription framework in the same indication or its renewal on public health grounds.
