Challenge

To authorise the exceptional use of a medicinal product that has not yet been granted marketing authorisation (MA). Such option is covered at European Community level in Article 5(1) of Directive 2001/83/EC, duly amended. 

In France, the exceptional use of pharmaceutical products that do not have a MA and are not used in clinical trials is covered by obtaining a Temporary Authorisation for Use (ATU). Such authorisation is delivered by the French National Agency for Medicines and Health Products Safety (ANSM). It is aimed at medicinal products that are intended to treat serious or rare diseases, when no appropriate treatment exists and the initiation of treatment cannot be deferred (Article L.5121-12 of the French Code of Public Health).

EUDRAC was tasked to support one of its client with the approval of a cohort ATU (for a group of patients), within France, for a new medicinal product under assessment by the EMA.

Approach

EUDRAC acted as the French local regulatory contact on behalf of the company for the preparation, submission and approval of the cohort ATU. EUDRAC was involved in the company strategic discussions, provided guidance on the dossier requirements and registration process, coordinated the ATU dossier preparation with the different team members involved, submitted the request to the ANSM and acted as the main contact with the ANSM. Due to the importance of such a treatment, the ANSM was very keen to speed up the registration process and agreed to present the product to the appropriate Working Group and then to the Commission in very short timings.

A close relationship with the client and the ANSM including follow-ups, ad-hoc discussions, responses to complementary information and great responsiveness with the authority were key to a successful approval. 

Conclusion

The ANSM recognised the benefit of the product in urgently treating patients with a life-threatening disease for which no appropriate treatment was available on the market.

The client obtained a fast approval of the cohort ATU in the required indication, which allowed the patients to receive the treatment ahead of the MA approval.