EU workshop will inform post-authorisation efficacy assessment guidelines
Five main topics were addressed
A workshop on improving assessments of the efficacy of authorised medicines has been held by the European Medicines Agency (EMA), to help it draft scientific guidance on the issue.
EMA has a mandate under new European Union (EU) pharmacovigilance legislation to develop post-authorisation efficacy studies guidelines and held the workshop was held to help understand the strengths and weaknesses of different study design options.
A key issue, said EMA, was considering 'recommendations on best use of methods to account for bias and confounding [hidden impacts on drug effects] and to identify needs for the improvement of methods in the field of efficacy studies'. Five main topics were addressed by invited experts: pragmatic trials; observational studies; registries; using electronic health records for pragmatic trials; and methods to control for confounding.
A report on the workshop’s proceedings noted, for instance, concerns that 'for ethical reasons, pragmatic trials are less appropriate where efficacy is uncertain'.
EMA also stressed that observational studies were not be used to determine basic efficacy, but 'to study effect modifiers, namely variables that may influence the level of efficacy of the drug'.
As for registries, participants at the workshop said they 'are most appropriate, when information is not available in other settings, when long-term follow-up is available'.