Accelerated approvals: The reality behind confirmatory trial delays

By Dr Nicola Davies

The accelerated approval pathway (AAP), which was first introduced by the US Food and Drug Administration (FDA) in 1992 to tackle the HIV epidemic, enables expedited development and approval of new therapies for serious diseases that lack efficient treatment options.1,2 Such expedited approval is based on non-medical surrogate endpoints or intermediate measures that are expected to predict clinical outcomes. This pathway ensures patients do not lose precious time in gaining access to potentially life-saving medicines.

However, the benefits of these drugs must still be established through confirmatory clinical trials, which ideally need to be underway at the time of approval.3 Instead, from 1992 to 2021, 50 confirmatory trials (42% of confirmatory trials required under the AAP) did not even start within a year of approval; among these, 19 have not started over three years post-approval.4