EMA leaders say single pivotal trial should not be default, decision must match evidence needed

EMA chief Emer Cooke and other officials weighed in on the ongoing debate about the number of pivotal trials required for drug approval.

They argued that requiring a single pivotal trial as a rigid default is inappropriate, emphasizing that the choice should be driven by the specific evidence needed to demonstrate a positive benefit-risk balance.

The officials suggested that sponsors and regulators should assess each therapy individually, deciding whether one or multiple trials best support the assessment.

This stance reflects EMA’s broader push for flexibility in trial design, aiming to align regulatory requirements with scientific justification rather than a one-size-fits-all rule.