OBJECTIVES: We evaluated the efficacy, safety, and regulatory approval between priority and non-priority reviewed innovative cancer drugs approved by China’s National Medical Products Administration (NMPA) from 2015 to 2023.

METHODS: Using public databases, innovative cancer drugs from January 1, 2015, to December 31, 2023 were included. We compared drug development time and New Drug Application (NDA)/Biologics License Application (BLA) approval time between priority and non-priority reviewed drugs. Efficacy and safety information supporting marketing were identified. Efficacy of randomized controlled trials (RCTs) was measured using overall survival (OS) and progression-free survival (PFS), while single-arm trials used response rate (RR). Safety was measured by treatment-related serious adverse events and grade ≥3 adverse events.

RESULTS: Out of 40 new cancer drugs corresponding to 54 indications approved during the study period, 34 drugs with 39 indications were granted priority review. There was no significant difference in drug development time between priority and non-priority reviewed drugs (3.08 vs. 2.99 years; p=0.596), but the NDA/BLA approval time was significantly shorter for priority reviewed drugs (378 vs. 459 days; p=0.025). In terms of efficacy, no significant differences were found between priority and non-priority reviewed drugs in median OS (9.46 vs. 15.3 months), median PFS (13.6 vs. 8.9 months, p=0.305), and median RR (56% vs. 67.6%, p=0.856). In RCTs, the rate of grade ≥3 adverse events were higher for priority reviewed drugs (28.84% vs. 24.15%, p=0.813), while treatment-emergent serious adverse events rate was significantly lower for non-priority reviewed drugs (5.10% vs. 10.20%, p=0.017). In single-arm trials, priority reviewed drugs showed higher rates in both indicators (40.38% vs. 22.46%, p=0.028, and 20.3% vs. 8.91%, p=0.055).

CONCLUSIONS: The priority review and approval process significantly reduced NDA approval time, but no evidence showed drugs approved through this process differ significantly in efficacy. In addition, priority reviewed drugs may be associated with higher risk adverse events.