The European Medicines Agency has published a summary of a “Workshop on methods for efficacy studies in everyday practice”, which was held in October. The meeting took place as a preparatory step to the development of scientific guidance on post-authorisation efficacy studies (PAES), which the Agency will develop according to a mandate set out in the new pharmacovigilance legislation and subsequent to any outputs of the European Commission on the situations in which such studies may be required.
The objectives of the workshop were to understand strengths and weaknesses of different design options to study efficacy in the conditions of the everyday medical practice, to issue recommendations on best use of methods to account for bias and confounding and to identify needs for the improvement of methods in the field of efficacy studies.
Five main topics were addressed by invited experts in working groups:
- pragmatic trials
- observational studies
- the use of electronic health records for pragmatic trials, and
- methods to control for confounding.