Assessing Outcomes Emerging After Conversion to Regular Approval for Cancer Drug Indications Granted Accelerated Approval, 1992-2021

Tibau Martorell A, Romano A, Liu ITT, Han J, Cliff ERS, Kesselheim AS - Journal of the National Cancer Institute

  • Regulation and Clinical Evidence

Summary:

Among all cancer drug indications granted accelerated approval by the FDA between 1992 and 2021 that were later converted to full approval, only 32% of confirmatory trials used to support full approval showed overall survival benefit and fewer than half (47%) met the ESMO-MCBS threshold for substantial benefit. These findings highlight the need for stronger approval standards for cancer therapies granted accelerated approval and the value of scales like ESMO-MCBS better guide treatment value for patients and clinicians.

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