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Introduction <p>The introduction of Accelerated Drug Marketing Registration Procedures (ADMRPs) in China has improved patient access to innovative therapies, but concerns remain about the robustness and consistency of efficacy evidence.</p> Aim <p>This study aimed to compare the treatment efficacy of approved targeted therapies for non-small cell lung cancer (NSCLC) before and after the implementation of ADMRPs.</p> Method <p>In this retrospective study, we analyzed targeted therapies for NSCLC approved in China as of December 31, 2024. Approvals were categorized as pre-policy or post-policy groups based on the July 1, 2020 implementation of ADMRPs. Descriptive statistics were used to examine approved indications and pivotal trial designs. Meta-analysis and meta-regression compared pre-approval efficacy outcomes, including overall survival (OS), progression-free survival (PFS), disease-free survival, and objective response rate (ORR), between groups. Sensitivity analyses evaluated post-marketing OS data and the robustness of results.</p> Results <p>A total of 59 indications supported by 72 trials were approved. Post-policy approvals increased markedly (41 vs 18), with rises in domestic drugs (16.7% pre-policy vs 61.0% post-policy) and uncommon targets (5.6% pre-policy vs 43.9% post-policy) (both <i>P</i> &lt; 0.05). Pivotal trials shifted from primarily Phase III-IV randomized controlled trials (RCTs) (76.0%) pre-policy to Phase I–II single-arm designs (68.1%) post-policy, mainly supporting drugs for uncommon or resistance mutations, whereas approvals for classical targets remained predominantly based on RCTs (90.0% pre-policy vs 93.8% post-policy; <i>P</i> = 0.416). Meta-analyses found no significant differences in pre-approval efficacy between groups for PFS (HR: 0.52 [0.45–0.61] vs 0.44 [0.36–0.54]; <i>P</i> = 0.206) or ORR (0.63 [0.55–0.70] vs 0.65 [0.59–0.70]; <i>P</i> = 0.705). However, OS meta-analysis was only feasible in the pre-policy group, whereas OS data in the post-policy group remained immature, with a median follow-up of 17&#xa0;months (IQR 8–34) after approval.</p> Conclusion <p>After the introduction of ADMRPs, approvals of targeted therapies for NSCLC increased substantially, without significant loss of pre-approval treatment efficacy. However, due to the limited follow-up, definitive survival benefits for the post-policy group cannot yet be established. The increased reliance on single-arm trials underscores the necessity of rigorous confirmatory post-marketing studies.</p>

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Comparison of treatment efficacy of targeted therapies approved in China for non-small cell lung cancer before and after the introduction of accelerated drug marketing registration procedures

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年3月17日

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  • Chenning Liu,
  • Liyang Lyu,
  • Yufan Zhang,
  • Yingyi Tian,
  • Jing Yuan,
  • Chihua Li,
  • Yuanjia Hu

摘要

Introduction

The introduction of Accelerated Drug Marketing Registration Procedures (ADMRPs) in China has improved patient access to innovative therapies, but concerns remain about the robustness and consistency of efficacy evidence.

Aim

This study aimed to compare the treatment efficacy of approved targeted therapies for non-small cell lung cancer (NSCLC) before and after the implementation of ADMRPs.

Method

In this retrospective study, we analyzed targeted therapies for NSCLC approved in China as of December 31, 2024. Approvals were categorized as pre-policy or post-policy groups based on the July 1, 2020 implementation of ADMRPs. Descriptive statistics were used to examine approved indications and pivotal trial designs. Meta-analysis and meta-regression compared pre-approval efficacy outcomes, including overall survival (OS), progression-free survival (PFS), disease-free survival, and objective response rate (ORR), between groups. Sensitivity analyses evaluated post-marketing OS data and the robustness of results.

Results

A total of 59 indications supported by 72 trials were approved. Post-policy approvals increased markedly (41 vs 18), with rises in domestic drugs (16.7% pre-policy vs 61.0% post-policy) and uncommon targets (5.6% pre-policy vs 43.9% post-policy) (both P < 0.05). Pivotal trials shifted from primarily Phase III-IV randomized controlled trials (RCTs) (76.0%) pre-policy to Phase I–II single-arm designs (68.1%) post-policy, mainly supporting drugs for uncommon or resistance mutations, whereas approvals for classical targets remained predominantly based on RCTs (90.0% pre-policy vs 93.8% post-policy; P = 0.416). Meta-analyses found no significant differences in pre-approval efficacy between groups for PFS (HR: 0.52 [0.45–0.61] vs 0.44 [0.36–0.54]; P = 0.206) or ORR (0.63 [0.55–0.70] vs 0.65 [0.59–0.70]; P = 0.705). However, OS meta-analysis was only feasible in the pre-policy group, whereas OS data in the post-policy group remained immature, with a median follow-up of 17 months (IQR 8–34) after approval.

Conclusion

After the introduction of ADMRPs, approvals of targeted therapies for NSCLC increased substantially, without significant loss of pre-approval treatment efficacy. However, due to the limited follow-up, definitive survival benefits for the post-policy group cannot yet be established. The increased reliance on single-arm trials underscores the necessity of rigorous confirmatory post-marketing studies.