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FDA proposes improvements for oncology clinical trials supporting accelerated approval

The draft guidance discusses the advantage of randomised clinical trials over single-arm studies


The US Food and Drug Administration (FDA) has issued new draft guidance to improve oncology clinical trials used to support accelerated approval applications.

Under the accelerated approval pathway, the US regulator may approve therapies for serious diseases like cancer earlier, on the condition that companies conduct studies to confirm the anticipated clinical benefit.

If a confirmatory trial shows that a drug provides a clinical benefit, then the FDA grants it traditional approval. However, there have been concerns for many years that these trials are not always conducted, and that some drugs later prove to be ineffective.

The guidance, Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics, discusses a potential advantage of randomised clinical trials over single-arm studies.

'When properly designed and executed, a randomised controlled trial provides a more robust efficacy and safety assessment and allows direct comparisons to a concurrent control arm,' the guidance states.

The FDA suggests two approaches: one conducting a single randomised controlled trial to support both an accelerated approval and to verify clinical benefit (the 'one-trial' approach), and another conducting a trial to achieve approval in addition to a confirmatory trial.

The FDA adds that the ‘one-trial’ approach has the advantage of providing quicker verification of the benefits, stating: ‘The one-trial approach maintains efficiency in drug development by providing early access to an investigational drug using the accelerated approval pathway, while ensuring that a post marketing trial is fully accrued and well underway to verify longer term benefit in a timely fashion.’

Richard Pazdur, director of the FDA’s Oncology Center of Excellence, said: “The FDA’s accelerated approval programme has provided patients with cancer earlier access to novel treatments that can be practice changing.

“Today’s draft guidance provides recommendations to sponsors for designing clinical trials to support accelerated approval. Building quality and efficiency into the design of oncology clinical trials is a crucial component in providing maximum benefit to those living with cancer.”

The FDA’s Oncology Center of Excellence has also launched ‘Project Confirm’, an initiative aimed at promoting the transparency of outcomes related to accelerated approval for oncology indications.

The project has so far developed a searchable database with information on the status of all oncology accelerated approvals, a model that was adopted by the FDA’s Center for Drug Evaluation and Research for all accelerated approvals.

Article by
Emily Kimber

27th March 2023

From: Regulatory



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