What is a PAES imposed in accordance with the EMA?

PAES imposed in accordance with the Commission Delegated Regulation (EU) No 357/2014 it is meant an efficacy study which is requested by a Competent Authority pursuant to at least one of the situations set out in this said regulation. The data resulting from such a PAES conducted within an authorised therapeutic indication are required to be submitted as they are considered important for complementing available efficacy data in the light of well-reasoned scientific uncertainties on aspects of the evidence of benefits that is to be, or can only be, addressed post-authorisation. The results of the PAES have the potential to impact on the benefit-risk of the medicinal product or product information.

Such efficacy study conducted post-authorisation can be imposed either:

at the time of granting the initial marketing authorisation (MA) where concerns relating to some aspects of the efficacy of the medicinal product are identified and can be resolved only after the medicinal product has been marketed; or

after granting of a MA where the understanding of the disease or the clinical methodology or the use of the medicinal product under real-life conditions indicate that previous efficacy evaluations might have to be revised significantly.

It is also possible to impose the conduct of post-authorisation efficacy studies in the specific situations of a conditional MA, a MA granted in exceptional circumstances, a MA granted to an advanced therapy medicinal product, the paediatric use of a medicinal product, a referral procedure initiated under Article 31 or Article 107i of Directive 2001/83/EC or Article 20 of Regulation (EC) No 726/2004, however these fall outside the scope of the Delegated Regulation.