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Background <p>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.</p> Methods <p>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.</p> Results <p>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, <i>p</i> &lt; 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, <i>p</i> = 0.875), median OS (10.85 vs. 20.85 months, <i>p</i> = 0.240), the hazard ratio of PFS (0.46 vs. 0.50, <i>p</i> = 0.608), and ORR (60.8% vs. 67.6%, <i>p</i> = 0.915; pooled ORR: 54% vs. 53.7%, <i>p</i> = 0.986). In contrast, priority-reviewed indications were associated with higher risks of Grade ≥ 3 AEs (56.33% vs. 57.35%, <i>p</i> = 0.732; risk ratio: 1.68 vs. 1.12, <i>p</i> = 0.017) and treatment-related SAEs (14.46% vs. 24.55%, <i>p</i> = 0.087; risk ratio: 1.82 vs. 1.39, <i>p</i> = 0.021) compared to non-priority-reviewed indications.</p> Conclusions <p>Although the PRP expedites patient access to innovative drugs, evidence remains insufficient to demonstrate significant additional benefits in efficacy and safety.</p>

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Regulatory and clinical outcomes of priority-reviewed innovative cancer drugs in China between 2015 and 2024: an observational study

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年7月15日

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  • Qi Li,
  • Nan Peng,
  • Linfeng Jiang,
  • Huting Zhou,
  • Pengcheng Liu,
  • Haijing Guan,
  • Dongning Yao

摘要

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.