Authorisation under exceptional circumstances

An authorisation under exceptional circumstances is a marketing authorisation granted where the applicant shows that it is unable to provide comprehensive data on the efficacy and safety of the medicinal product under normal conditions of use. It is provided for by Regulation (EC) No 726/2004 for centralised authorisations and by Directive 2001/83/EC for national authorisations. Unlike a conditional authorisation, it does not assume that the missing data will one day be obtained.

Grounds for the authorisation

Three situations justify this regime, provided the applicant establishes them. The indications concerned may be so rare that comprehensive data cannot reasonably be expected. The present state of scientific knowledge may not allow such data to be gathered. Finally, generally accepted principles of medical ethics may prohibit their collection, for example where it would be unethical to expose subjects to a pathogen in order to test a vaccine or an antidote. The regime often concerns very rare diseases, without being the same as orphan medicinal product status, which has its own criteria.

Obligations of the holder

The authorisation is subject to specific conditions, in particular regarding safety, reporting of incidents and information for healthcare professionals. It may require that the medicinal product be supplied only on medical prescription or under strict supervision. The benefit/risk balance is reassessed annually, in the light of pharmacovigilance data and of any studies requested. The package leaflet and the medical information state that the available data on the medicinal product remain inadequate in certain respects.

Duration and effects of the authorisation

As regards its duration, an authorisation under exceptional circumstances follows the ordinary regime of validity and renewal, subject to the annual reassessment, the outcome of which may lead to its suspension or withdrawal. Unlike a conditional authorisation, it is not normally intended to become a standard authorisation. Granted under the usual procedures, it triggers regulatory protection and may found a supplementary protection certificate.

Key points

An authorisation under exceptional circumstances addresses the lasting impossibility of gathering comprehensive data, owing to the rarity of the disease, the state of science or medical ethics. It imposes enhanced obligations and an annual reassessment of the benefit/risk balance. It must not be confused with a conditional authorisation, which on the contrary assumes that the data will be completed. The ground of impossibility should be documented precisely from the application stage, as the authority focuses its examination on that point.

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This glossary entry is general in scope and does not constitute legal advice.

Dhenne Avocats acts in pharmaceutical patent disputes and on the medicines regulatory questions that accompany them, from the marketing authorisation to the supplementary protection certificate.

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