The pharmaceutical companies that have carried out the clinical trials from phases 1 to 3 and obtained positive results will aim to produce the compound industrially and market it in the form of a drug. To do so, they must gather all the data of their trials in an application for Marketing Authorisation (MA).
The MA is issued by the competent European authorities (the European Commission, in line with the recommendation of the European Medicine Agency - EMA) or national authorities (the National Drug Safety Agency - ANSM in France).
The MA file has an internationally uniform structure to facilitate its evaluation by the authorities. It describes three key aspects:
- quality: data linked to the industrial fabrication of the drug (production of raw materials and the finished product, fabrication process reproducibility controls).
- safety: data of the study performed during preclinical development (toxicology, pharmacokinetics, etc.);
- efficacy: results of clinical studies on healthy humans and patients, with a positive benefit/risk ratio.
The MA is also associated with the Summary of Product Characteristics (SPC), the patient information leaflet and information on labelling.
Different marketing authorisation procedures are possible:
- centralised: the file is registered at the head office of the EMA for an authorisation valid for all European countries;
- mutual recognition: the file is registered with the competent authority of a Member State and can then be extended to other countries;
- decentralised: the file is registered at the same time in all the Member States and the authorisation is issued by a reference State.
Temporary Authorisation for Use (TAU)
Exceptionally, a drug intended to treat a serious or rare disease can be made available in large hospitals before its MA, in the case of emergency or in the absence of any other appropriate treatment.
In France, the MA file is submitted to the Commission on Transparency of the Executive Health Authority, which issues its opinion on the therapeutic value and on the improvement of therapeutic value that the drug offers to patients with the disease targeted. The price of the drug is set by the Economic Commission on Health Products (CEPS) and its rate of reimbursement, by the National Health Insurance Fund (UNCAM).