The US Food and Drug Administration (FDA) has suggested that cancer drug developers perform more rigorous trials in order to obtain accelerated clearance for their candidates.
The FDA’s proposed recommendation comes after the controversial approval of Biogen Inc’s Alzheimer’s treatment Aduhelm, as well as an independent federal review of the accelerated approval pathway.
Approvals enable the agency to bring therapies that treat serious and life-threatening conditions to market more rapidly, but they have been criticised because some drugs have been shown to be ineffective in the past.
The FDA suggested that companies conduct controlled trials in which patients receive either a therapy or another alternative treatment rather than single-arm studies in which the drug is tested without a comparator.
Single-arm studies can be conducted in some instances, but they must be discussed with the agency first, according to the statement.
“Given the limitations of single-arm trials, a randomized controlled trial is the preferred approach to support an application for accelerated approval,” the FDA said.
Drug developers who submit single-arm studies for accelerated clearance usually follow up with a separate trial to validate clinical benefits.
The regulator proposed two approaches: one was to perform a randomised clinical trial before receiving the accelerated approval and then again to confirm the benefits, and the other was to hold only one trial to seek the approval with long-term follow-up.
According to the FDA, conducting a single long randomised trial has the advantage of giving faster verification of the benefits.