French Public Health Code
In forceI.-The application for authorisation to obtain the early access provided for in Article L. 5121-12 is sent by electronic means, by the holder of the exploitation rights or his authorised representative, simultaneously to the Haute Autorité de Santé and to the Ministers for Health and Social Security and, where the application concerns a medicinal product mentioned in 1° of II of the aforementioned Article, to the Agence nationale de sécurité du médicament et des produits de santé.
II.- The application referred to in I is accompanied by a file including: 1° The name and address of the holder of the exploitation rights or his authorised representative and, in this case, the representative on whose behalf the authorised representative is acting.The application referred to in I must be accompanied by a dossier comprising:
1° The name and address of the holder of the exploitation rights or of his authorised representative and, in this case, the representative on whose behalf the authorised representative is making the application;
2° Information and documents relating to the medicinal product concerned and its use in the therapeutic indication applied for, including :
a) The draft summary of product characteristics or, where applicable, the marketing authorisation and its annexes;
b) The titles and objectives of any research involving the human person for commercial purposes that is in progress or has been finalised, together with the status of any planned research, the identity of the principal investigator for these trials and, for research conducted in France, the identity of all the investigators and the location(s) concerned;
c) Where applicable, the documents mentioned in articles R. 5121-21 and R. 5121-25 or, failing these, the brochure for the investigator mentioned in article R. 1123-20 and the technical dossier mentioned in 3° of article R. 1123-30;
3° The reasons for the application, including the information needed to establish, for the therapeutic indication in question :
a) that there is no appropriate treatment for the serious, rare or debilitating disease in question;
b) that it is impossible to defer the implementation of the treatment;
c) that the efficacy and safety of this medicinal product are strongly presumed in the light of the results of the research involving the human body for commercial purposes which was carried out with a view to the application for marketing authorisation;
d) The presumed innovative nature of this medicinal product, in particular with regard to any clinically relevant comparator;
4° Where applicable, the proposed labelling drawn up in accordance with the provisions of article R. 5121-144 and the draft package leaflet, drawn up in accordance with the draft summary of product characteristics;
5° Where the application relates to a medicinal product authorised in another Member State of the European Union or party to the Agreement on the European Economic Area or in a third country, a copy of the marketing authorisation and the summary of product characteristics as well as the last updated periodic safety report or equivalent documents;
6° Where applicable, a copy of any decisions to refuse or withdraw marketing authorisation for this medicinal product taken in a Member State of the European Union or party to the Agreement on the European Economic Area or in a third country for each of the indications of the medicinal product which is the subject of the application for early access authorisation and a summary table for each of the authorised indications of the medicinal product;
7° Where applicable, a copy of the designation of the medicinal product as an orphan medicinal product pursuant to Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999;
8° Any information relating to exceptional and early use in the indication applied for, in another Member State of the European Union or party to the Agreement on the European Economic Area or in a third country as well as, where applicable, a copy of any authorisation or scientific opinion relating to such use issued by the competent authority of a Member State or third country, or by the European Medicines Agency;
9° The draft therapeutic use protocol and data collection defined in article R. 5121-70;
10° An estimate of the target population of patients likely to benefit from the speciality throughout the period of the early access authorisation, in the indication in question, taking into account the medical and epidemiological data available;
11° An estimate of the number of patients likely to be treated each year until a decision is taken on its inclusion on one of the lists referred to in Article L. 162-17 of the Social Security Code and Article L. 5123-2 of this Code, in the indication in question, under its marketing authorisation;
Where applicable, the number of patients treated under authorisations granted for compassionate access pursuant to II of Article L. 5121-12-1;
12° The undertaking by the holder of the exploitation rights or its authorised representative to make the medicinal product available within two months of the granting of the early access authorisation;
13° The undertaking by the holder of the exploitation rights or its authorised representative to finance the collection of data under the conditions mentioned in IV of Article L. 5121-12;
14° For applications relating to a medicinal product mentioned in 1° of II of Article L. 5121-12 :
a) A copy of the marketing authorisation application letter for the indication in question, or
b) Where such an application has not yet been filed, an undertaking by the holder of the exploitation rights or its authorised representative to file one, within a period determined by the Haute Autorité de santé which may not exceed a period set by decree, and the date on which the holder of the exploitation rights or its authorised representative intends to file such an application;
15° For applications relating to a medicinal product mentioned in 2° of II of Article L. 5121-12, the undertaking by the holder of the exploitation rights or its authorised representative to submit its application for registration, in the indication in question, on one of the lists mentioned in the first paragraph of Article L. 5123-2 of this Code or in the first paragraph of Article L. 162-17 of the Social Security Code in the month following the granting of the corresponding marketing authorisation or, where applicable, the date on which this application was submitted;
16° Economic data including :
a) Where applicable, the prices charged and sales volumes recorded in other Member States or parties to the Agreement on the European Economic Area and in the United States of America;
b) Forecast sales volumes in the countries mentioned in a) above, as well as the reimbursement status of the product in each country concerned;
c) Prices charged and sales volumes recorded in France when indications for the product are already covered by the health insurance scheme;
d) Three-year sales forecasts for the indication in question and, where applicable, for each of its indications already covered;
e) Where the medicinal product is not marketed in France, the compensation that will be claimed by the holder of the exploitation rights or its authorised representative under an early access authorisation;
f) The selling price to healthcare establishments where the medicinal product does not have a price or tariff in application of articles L. 162-16-4, L. 162-16-5 or L. 162-16-6 of the Social Security Code, is registered for other indications on the L. 5123-2 list of the Public Health Code without being subject to a maximum price for sale to healthcare establishments in application of article L. 162-16-4-3 of the French Social Security Code;
17° Extensions of indications likely to be the subject of a request for reimbursement within two years of the request being made, together with their provisional timetable for marketing;
18° Information on any ongoing research involving the human being for commercial purposes in other indications, in particular protocols, timetables, indications concerned, lists of co-investigating French centres and comparators;
19° An undertaking by the holder of the exploitation rights or its authorised representative to declare annually to the Ministers for Health and Social Security the information provided for in article R. 163-32 of the Social Security Code.
III.-The Haute Autorité de santé acknowledges receipt of the complete application and informs the ministers responsible for health and social security and, for the applications mentioned in 1° of II of article L. 5121-12 of the public health code, the Agence nationale de sécurité du médicament et des produits de santé.
In the event that the information provided by the holder of the exploitation rights or its authorised representative is insufficient, the Haute Autorité de santé, where appropriate on a proposal from the Agence nationale de sécurité du médicament et des produits de santé and the ministers responsible for health and social security, notifies the applicant of the list of additional information required by any means giving a certain date of receipt of this notification. If the company does not complete its file within twenty days of receiving this list, the request for early access is deemed to have been abandoned.
IV. -During the examination of the application, the applicant will immediately send by electronic means to the Haute Autorité de santé, the ministers responsible for health and social security and, for the applications mentioned in 1° of II of Article L. 5121-12, to the Agence nationale de sécurité du médicament et des produits de santé (French National Agency for the Safety of Medicines and Health Products), any additional information relating to the constituent elements of the dossier, in particular the submission of the marketing authorisation application, and any new data that it has or becomes aware of that may influence the assessment of the application for early access authorisation, with regard to the criteria mentioned in I of Article L. 5121-12.
In this case, the Haute Autorité de santé may suspend the period mentioned in I of Article R. 5121-69 to enable it to analyse this additional information. This suspension may not exceed 45 days.
V.-When the company wishes to withdraw its request for early access authorisation mentioned in I during the investigation, it must submit a request to the Haute Autorité de santé, which will acknowledge receipt and inform the ministers responsible for health and social security of the withdrawal of the request and, for the requests mentioned in 1° of II of Article L. 5121-12, the Agence nationale de sécurité du médicament et des produits de santé.
