Purpose: To provide an overview of and discuss newly authorised medicines with an improved efficacy.
Methods: This analysis focussed on new medicines with an improved efficacy based on the results of randomised active control trials. Information on comparative efficacy was obtained from the European Medicines Agency European Public Assessment Reports.
Results: Between 1999 and 2005 we identified 122 new medicines with a new active substance. Of these, 13 (10%) were shown to be superior to already available medicines in terms a statistically significant difference in primary clinical endpoints.
Conclusions: A proven advantage in efficacy at an early stage of drug development is the exception rather than the rule. The absence of evidence demonstrating differences between medicines does not necessarily mean that there are no actual differences. Optimal pharmacotherapy would benefit from more comparative research in the development of new medicines. The results of comparative trials need to be critically evaluated for their specific value in clinical practice. To this end, prescription data may be helpful.