Hwang TJ, et al. - Researchers assessed the efficacy, safety, times to approval of breakthrough-designated vs non–breakthrough-designated cancer drugs approved by the US Food and Drug Administration (FDA). Relative to non–breakthrough-designated drugs, breakthrough-designated cancer drugs were related to faster times to approval, but offered no evident improvements in safety or novelty, with no statistically significant efficacy advantage.

Methods

• Researchers studied all new cancer drugs approved by the FDA between January 2012 and December 2017.
• Breakthrough-designated and non–breakthrough-designated cancer drugs were compared with respect to time to FDA approval, pivotal trial efficacy end point, and novelty of mechanism of action.
• Using random-effects meta-regression, they evaluated the link between breakthrough therapy designation and hazard ratios for progression-free survival (PFS), response rates (RRs) for solid tumors, serious adverse events, and deaths not attributed to disease progression.

Results

• Data showed that 58 new cancer drugs were approved by the FDA between 2012 and 2017, 25 (43%) of which received breakthrough therapy designation.
• Median time to first FDA approval was 5.2 years vs 7.1 years for breakthrough-designated drugs and non–breakthrough-designated drugs, respectively (difference, 1.9 years; P=.01).
• Median PFS gains (8.6 v 4.0 months; P=.11), hazard ratios for PFS (0.43 v 0.51; P=.28), or RRs for solid tumors (37% v 39%; P=.74) were not significantly dissimilar between breakthrough-designated and non–breakthrough-designated drugs.
• Findings demonstrated that breakthrough therapy–designated drugs were not more likely to act via an innovative mechanism of action (36% v 39%; P=1.00).
• In breakthrough-designated and non–breakthrough-designated drugs, similar rates of deaths (6% v 4%; P=.99) and serious adverse events (38% v 36%; P=0.93) were noted.