The US Food and Drug Administration (FDA) issued draft guidance on the design of oncology trials for accelerated approval, calling randomized controlled trials (RCTs) – rather than single-arm studies — the “preferred approach” to support accelerated approval.
Sponsors can conduct a single RCT to support accelerated approval and verify clinical benefit or run two trials, one that supports accelerated approval through the use of an early endpoint and one confirmatory trial that is powered to assess a longer-term clinical endpoint, according to the draft guidance, which was published in the Federal Register on 27 March.
“This ‘one-trial’ approach maintains efficiency in drug development and can provide early access to a drug using the accelerated approval pathway, while ensuring that a postmarketing trial is fully accrued and well underway to verify longer term benefit in a timely fashion,” the agency wrote in the draft guidance…
