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Assessment of the clinical benefit of cancer drugs receiving accelerated approval

JAMA Jun 04, 2019

Gyawali B, et al. - In this updated review of 93 cancer drug indications granted accelerated approval by the FDA from December 11, 1992, through May 31, 2017, researchers compared the endpoints used in pre-approval studies resulting in accelerated approval with endpoints used in the required confirmatory studies verifying clinical benefit and updating the results of accelerated approvals with confirmatory studies ongoing at the time of the FDA review. Confirmatory trials in this updated review reported an improvement in overall survival for 19 of 93 (20%) cancer drug approvals, 19 (20%) reported improvement in the same surrogate used in the pre-approval trial, and 20 (21%) reported improvement in another surrogate. According to findings, confirmatory studies for one-fifth (n = 19 of 93) indications of cancer drugs approved through the accelerated approval pathway of the FDA showed improvements in overall patient survival. Reassessment of confirmatory testing requirements may be necessary to obtain more information that is clinically meaningful.
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