EMA releases guidance on methods to be used in the design and conduct of post authorisation efficacy studies
The European Medicines Agency (EMA) has released a draft scientific guideline that outlines how post-authorisation efficacy studies (PAES) should be designed by companies to support regulatory decision making in the European Union (EU). In addition, a guidance that describes the regulatory aspects for the fulfilment of imposed PAES is also published.
These studies are conducted within the authorised indication after a medicine has been granted a marketing authorisation, to collect data on aspects of its benefits that can only be or need to be explored once the medicine is marketed.
In particular, PAES can address questions related to the benefits of a medicine stemming from the way it is used in everyday medical practice, including in specific populations, in relation to its use with other medicines or over time, and when there are changes in the understanding of a disease or the medicine’s mechanism of action.
The knowledge generated by these studies complements the information about the medicine’s benefits that was assessed during its approval process.
These studies can be imposed by regulators or may be carried out voluntarily by companies. The situations where a PAES can be required by medicines regulatory authorities in the EU were specified by the European Commission in April 2014. Prior to that date, regulators could request these types of studies in certain cases such as in the context of conditional marketing authorisations, authorisations under exceptional circumstances, paediatric use or referral procedures.
PAES can now be required for medicines with a standard marketing authorisation:
- at the time a marketing authorisation is recommended, when there are questions about the efficacy of the medicine that can only be answered once the medicine has been marketed;
- after a marketing authorisation has been granted, if new data indicate that the benefits of the medicine should be further studied.
The results of these studies should translate into better labelling and better use of medicines by patients and prescribers in clinical practice.
The draft scientific guideline applies to imposed and voluntary PAES. It has been developed in collaboration with the EU Member States and other interested parties and is released for a three-month public consultation.
Comments on the scientific guideline are invited until 31 January 2016; they should be sent to firstname.lastname@example.org using the form provided.
Additional regulatory and procedural guidance is being released together with the scientific guidance to clarify aspects in relation to the imposition of PAES, including the submission of study protocols by companies and their assessment by EMA, the assessment of the study results, and the possible regulatory outcomes following the conduct of an imposed PAES.
Posted on the EMA website on 6 November 2015