1. Non-standard marketing authorisations

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1. Non-standard marketing authorisations

1.2. Marketing Authorisation under exceptional circumstances

An authorisation under exceptional circumstances is possible for products for which the applicant can demonstrate in the application that it is not possible to provide comprehensive data on the efficacy and safety under normal conditions of use for objective, verifiable reasons. Consequently, it will normally hardly ever be possible to assemble a full dossier in respect of a marketing authorisation granted under exceptional circumstances.

Eligibility

Depending on the application the different options are:

  • the indications for which the product in question is intended are encountered so rarely that the applicant cannot reasonably be expected to provide comprehensive evidence; or
  • in the present state of scientific knowledge, comprehensive information cannot be provided; or
  • it would be contrary to generally accepted principles of medical ethics to collect such information.

Conditions

A marketing authorisation may be granted subject to certain specific obligations. These obligations may include the following:

  • completion of an identified programme of studies within a time period specified by the competent authority, the results of which will form the basis of a reassessment of the benefit/risk profile,
  • supply of the medicine on prescription only and in certain cases administration only under strict medical supervision, possibly in a hospital,
  • the package leaflet and any medical information has to mention that a marketing authorisation has been granted subject to certain specific obligations to be reviewed annually,
  • a requirement for the applicant to introduce specific procedures, in particular concerning the safety of the medicine,
  • notification to the competent authorities of any incident relating to the use of the medicine, and action to be taken.

The fulfilment of specific procedures/obligations is aimed at the provision of information on the safe and effective use of the product.

This type of authorisation is reviewed annually to re-assess the benefit-risk profile.

The renewal of the marketing authorisation of a medicine under exceptional circumstances follows the same rules as a “normal” marketing authorisation. After 5 years, the marketing authorisation will then be renewed under exceptional circumstances for an unlimited period, unless the competent authority decides, on justified grounds relating to pharmacovigilance, to proceed with one additional five-year renewal.

Legal basis: 

Article 14 (8) of Regulation (EC) No 726/2004
Guideline on procedures for the granting of a marketing authorisation under exceptional circumstances, pursuant to Article 14 (8) of Regulation (EC) No 726/2004.
Article 22, Directive 2001/83/EC, as amended
Annex I, Part II, documentation for applications in exceptional circumstances, Directive 2001/83/EC, as amended.