French Public Health Code
In forceI. - The application for authorisation of an individually prepared advanced therapy medicinal product is accompanied by a dossier, the content of which is laid down by decision of the Director General of the Agence nationale de sécurité du médicament et des produits de santé and which includes :
1° The name of the medicinal product and its composition ;
2° A summary of the information relating to the product, the draft labelling and package leaflet;
3° The expected number of patients to be treated with the medicinal product;
4° Information concerning the tissues and cells removed and the products and materials coming into contact with them;
5° Data relating to the quality of the medicinal product concerning the active substance and the finished product, including the controls implemented;
6° The results of pre-clinical trials;
7° The proposed therapeutic indications and, where appropriate, the results of clinical trials justifying the therapeutic use of the medicinal product;
8° The posology, pharmaceutical form and methods and routes of administration;
9° A description of the pharmacovigilance system based on the pharmacovigilance provisions laid down for this category of medicinal product;
10° Details of the plan for monitoring efficacy and patient safety;
11° The public or private health establishments in which the medicinal product may be administered;
12° Where the medicinal product is prepared, distributed and administered in accordance with II of Article L. 4211-9-1, information intended to establish the necessity and safety of administering this medicinal product as part of the same medical intervention as that for the removal of the autologous tissues or cells used in its composition. This information shall relate in particular to the clinical benefit for the patient and to the control of the risks associated with the preparation in this context.
II - When the application concerns a combined advanced therapy medicinal product prepared on an ad hoc basis incorporating one or more devices mentioned in Article L. 5211-1, the dossier also includes :
1° The intended purpose of the device(s) as defined in 1° of Article R. 5211-4 ;
2° The design specifications, including the standards applied and the results of the risk analysis;
3° Where the standards referred to in Article R. 5211-18 are not applied in full, a description of the solutions adopted to meet the essential requirements referred to in Articles R. 5211-21 et seq, which apply to the product.
III - Where it has not been possible to carry out clinical trials, the applicant must also provide the following justification:
1° The reasons why the clinical trials could not be carried out ;
2° At the time of the application for authorisation, there is no appropriate treatment to improve the patient's condition and the treatment in question appears to be the only chance of avoiding a fatal outcome in the short term;
3° The patient, his legal representative if he is a minor, the person in charge of the representation measure relating to the person if he is an adult subject to such a measure if he is a minor, as well as the person in charge of the legal protection measure with representation relating to the person or the trusted support person that he has designated in application of article L. 1111-6 has received from the prescribing doctor information appropriate to his/her situation concerning the absence of therapeutic alternatives, the risks involved, the constraints and the benefits likely to be derived from the medicinal product. The procedure followed is recorded in the medical record;
4° The medicinal product is likely to be of benefit to the patient and the state of scientific knowledge allows its efficacy and safety to be prejudged;
5° Any safety and efficacy data available.
IV - The application for authorisation is sent to the Director General of the Agence nationale de sécurité du médicament et des produits de santé (French National Agency for the Safety of Medicines and Health Products) by any means which allows the application to be dated, by the establishments or organisations authorised in application of articles L. 4211-9-1, L. 5124-3 or L. 5124-9-1.
V. - The provisions of Section 2 of Chapter III of Title III of Book V of the Environmental Code are applicable to medicinal products prepared on an ad hoc basis when they contain all or part of genetically modified organisms.
