Regulatory and clinical outcomes of priority-reviewed innovative cancer drugs in China between 2015 and 2024: an observational study
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Qi Li1, Nan Peng2, Linfeng Jiang1
1School of Pharmacy, Nanjing Medical University, No.101, Longmian Avenue, Nanjing, Jiangsu, 211166, China.
Background:
China implemented the Priority Review Program (PRP) to accelerate the approval of innovative drugs with significant clinical value. This study explores whether drugs approved through the PRP have more significant clinical benefits by comparing differences in approval time, efficacy, and safety between drugs approved through the priority and non-priority review pathways.
Methods:
In this observational comparative analysis, we conducted an analysis of innovative cancer drugs approved by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) between January 1, 2015, and December 31, 2024. Data were systematically collected and evaluated on NDA/BLA review times, efficacy outcomes (overall survival [OS], progression-free survival [PFS], and overall response rate [ORR]), and safety outcomes (Grade ≥ 3 adverse events [AEs] and treatment-related serious adverse events [SAEs]). A meta-analysis was conducted to assess the hazard ratios for PFS, ORR, and the risk ratios for Grade ≥ 3 AEs and treatment-related SAEs. Additionally, subgroup analyses were conducted to assess the impact of the 2020 policy changes on the PRP.
Results:
The NMPA approved 49 innovative cancer drugs for 69 indications, of which 47 indications were subject to priority review and 22 to non-priority review. Priority-reviewed indications significantly shortened NDA/BLA review times (263.5 vs. 352 days, p < 0.001). However, no significant differences in efficacy were observed between priority and non-priority-reviewed indications in terms of median PFS (9.87 vs. 8.95 months, p = 0.875), median OS (10.85 vs. 20.85 months, p = 0.240), the hazard ratio of PFS (0.46 vs. 0.50, p = 0.608), and ORR (60.8% vs. 67.6%, p = 0.915; pooled ORR: 54% vs. 53.7%, p = 0.986). In contrast, priority-reviewed indications were associated with higher risks of Grade ≥ 3 AEs (56.33% vs. 57.35%, p = 0.732; risk ratio: 1.68 vs. 1.12, p = 0.017) and treatment-related SAEs (14.46% vs. 24.55%, p = 0.087; risk ratio: 1.82 vs. 1.39, p = 0.021) compared to non-priority-reviewed indications.
Conclusions:
Although the PRP expedites patient access to innovative drugs, evidence remains insufficient to demonstrate significant additional benefits in efficacy and safety.
Insights
China's Priority Review Program (PRP) speeds up drug approvals but doesn't show significant improvements in efficacy or safety for innovative cancer drugs. Further evidence is needed to confirm additional clinical benefits.
Area of Science:
- Pharmacoeconomics and Health Economics
- Drug Regulatory Affairs
- Oncology Drug Development
Background:
- China's Priority Review Program (PRP) aims to accelerate the approval of innovative drugs with significant clinical value.
- This study evaluates if PRP-approved drugs offer greater clinical benefits compared to non-priority reviewed drugs.
Purpose of the Study:
- To compare the approval times, efficacy, and safety of innovative cancer drugs approved via priority versus non-priority review pathways in China.
- To determine if the PRP leads to superior clinical outcomes for patients.
Main Methods:
- Observational comparative analysis of innovative cancer drugs approved by China's NMPA (2015-2024).
- Data collection on review times, efficacy (OS, PFS, ORR), and safety (Grade ≥3 AEs, SAEs).
- Meta-analysis and subgroup analyses were performed to assess outcomes and policy impacts.
Main Results:
- Priority review significantly shortened drug approval times (263.5 vs. 352 days).
- No significant differences in efficacy (PFS, OS, ORR) were found between priority and non-priority reviewed drugs.
- Priority-reviewed drugs showed increased risks of Grade ≥3 adverse events (AEs) and serious adverse events (SAEs).
Conclusions:
- The PRP effectively accelerates patient access to innovative cancer drugs.
- Current evidence does not support significant additional benefits in efficacy or safety for drugs approved through the PRP.
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