Clinical benefit and regulatory outcomes of cancer drugs receiving accelerated approval

The FDA accelerated approval pathway allows approval of investigational drugs treating unmet medical needs based on changes to surrogate measures considered ‘reasonably likely’ to predict clinical benefit. Postapproval clinical trials are then required to confirm whether these drugs offer clinical benefit. In a cohort study of 46 cancer drugs granted accelerated approval from 2013 to 2017, 41% did not improve overall survival or quality of life in confirmatory trial after more than 5 years of follow-up, with results not yet available for another 15%. Among drugs converted to regular approval, 60% of conversions relied on surrogate measures. The authors conclude that although accelerated approval can be useful, some cancer drugs do not end up demonstrating benefit in extending patients’ lives or improving their quality of life.