2. Post-Authorisation Efficacy Studies (PAES)

A PAES may be required to be carried out by the holder of a marketing authorisation by:

• The national competent authorities (NCAs)
• The European Medicines Agency (EMA)
• The European Commission (EC)

A PAES shall only be required if one or more of the following cases arise:

1. An initial efficacy assessment based on surrogate endpoints which requires verification.
2. In case of medicinal products that are used in combination with other medicinal products, the need for further efficacy data to clarify uncertainties.
3. Uncertainties with respect to the efficacy of a medicinal product in certain sub-populations that could not be resolved prior to marketing authorisation.
4. The potential lack of efficacy in the long term that raises concerns with respect to the maintenance of a positive benefit-risk balance of the medicinal product.
5. Benefits of a medicinal product demonstrated in clinical trials are significantly affected by the use of the medicinal product under real-life conditions or, as for example in the case of vaccines, protective efficacy studies have not been feasible.
6. A change in the understanding of the standard of care for a disease or the pharmacology of a medicinal product that requires additional evidence on its efficacy.
7. New concrete and objective scientific factors that may constitute a basis for finding that previous efficacy evaluations might have to be revised significantly.

Irrespective of the above points it is also possible to impose the conduct of PAESs in the specific situations of a MA granted:

• conditionally
• in exceptional circumstances
• to an advanced therapy medicinal product
• the pediatric use of a medicinal product
• a referral procedure initiated.

To note: Health Technology Assessment results cannot trigger a PAES.