Safety population size and duration of exposure prior to approval of new medicines: A database analysis of medicines centralised approved in the European Union between 2011 and 2023

Orphan medicines and "precision" medicines tend to have less clinical data available at the time of marketing authorisation. The median of the total number of subjects studied prior to approval has been decreasing overtime. Additional pharmacovigilance activities play, in these cases, a key role in the safety monitoring and assessment of the benefit-risk profile during the medicine's lifecycle.