On 10 April 2014, the European Commission (“Commission”) adopted Commission Delegated Regulation (EU) No 357/2014 as regards situations in which post-authorisation efficacy studies may be required (“Delegated Regulation”). The Delegated Regulation will enter into force on 30 April 2014.

According to the Community Code on Medicinal Products, the European Medicines Agency (“EMA”), the Commission or the competent authorities of the EU Member States may request the marketing authorisation holder of a medicinal product to conduct a post-authorisation efficacy study in certain circumstances. These circumstances include concerns relating to the efficacy of the medicinal product which can only be resolved after the medicinal product has been authorised; or if the further understanding of the disease, the clinical methodology or the post-authorisation use of the medicinal product indicate that previous efficacy evaluations would require significant revisions.

To supplement the provisions of the Community Code on Medicinal Products, the Commission adopted the Delegated Regulation to determine the circumstances in which these post-authorisation efficacy studies may be required.

In accordance with the Delegated Regulation, the EMA, the Commission or the competent authorities of the EU Member States may request pharmaceutical companies to conduct post-authorisation efficacy studies in a number of circumstances which are as follows:

  • If the initial efficacy assessment was based on surrogate endpoints.
  • To resolve uncertainties about combination medicinal products.
  • To resolve uncertainties about the efficacy of a medicinal product in certain sub-populations that could not be resolved prior to marketing authorisation.
  • If a potential lack of long-term efficacy raises concerns about a positive benefit-risk balance of the medicinal product.
  • If the benefits of the medicinal product are significantly affected by real-life conditions.
  • A change in the standard of care for a disease or the pharmacology of a medicinal product that requires additional evidence on its efficacy.
  • If new concrete and objective scientific factors may require previous efficacy evaluations to be revised significantly.

The EMA, in collaboration with the EU Member States, will publish scientific guidance governing the design of the post-authorisation efficacy studies which are provided for in the Delegated Regulation.