Conditional approval of medicines by the EMA

Like other expedited procedures for early marketing authorisation, conditional approval12 stems from the assumption that immediate availability of new drugs offers patients a benefit that outweighs the risks of limited clinical information. To some extent, this assumption applies to all licensed drugs, but early approval magnifies the need for “managing the unknown.”34

Earlier this year, the European Medicines Agency reported its experience so far with conditional approval of new medicines.5 The report is an important step towards greater transparency and contributes useful data to help reflections on two important questions: what is the minimum evidence acceptable at the time of early approval, and do postmarketing commitments improve the evidence in a meaningful way?

In response to the first question, between 2006 and 2016 the EMA granted conditional approval to 30 medicines on the basis of 58 pivotal studies. The regulator reports that over half of these studies were preliminary phase II studies (30, including phases I/II and IIb); …