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Design Characteristics and Survival Outcomes of Local Trials Supporting the Approval of Targeted Anticancer Drugs in

Leyuan Qi1, Xiaoyan Chang2, Zixuan Zhuang3

  • 1Sixth Clinical School, Capital Medical University, Beijing, China.

Cancer Medicine
|August 19, 2026
PubMed
Abstract

Insights

Targeted anticancer drugs significantly benefit Chinese patients, improving survival outcomes. However, more local clinical trials are needed to fully understand their impact and guide treatment decisions.

Area of Science:

  • Oncology
  • Clinical Trials
  • Drug Development

Background:

  • China's rapid progress in precision oncology is marked by numerous locally approved targeted anticancer drugs.
  • The design and outcomes of pivotal trials supporting these approvals in Chinese populations remain underexplored.

Purpose of the Study:

  • To analyze the characteristics and therapeutic outcomes of pivotal trials for targeted anticancer drugs approved in China.
  • To evaluate the effectiveness of these drugs in the Chinese population based on local trial data.

Main Methods:

  • Systematic identification of targeted anticancer drugs approved in China by December 31, 2022.
  • Collection and analysis of therapeutic outcomes from trials involving Chinese patients using public data and regulatory comments.

Main Results:

  • Out of 300 oncology indications, 201 were supported by randomized controlled trials (RCTs), with 36 local RCTs specifically on Chinese populations.
  • Targeted anticancer drugs demonstrated significant benefits, reducing the risk of death (Overall Survival HR: 0.68) and progression (Progression-Free Survival HR: 0.47).
  • Median survival gains were modest (OS: 2.90 months, PFS: 2.95 months), with first-in-class drugs showing superior Overall Survival improvement.

Conclusions:

  • Targeted anticancer drugs offer significant survival benefits for Chinese patients, though the magnitude of Overall Survival gain is moderate.
  • There is a need to increase the number of local clinical trials involving Chinese patients.
  • This study provides valuable insights into local trial evidence, aiding clinical decision-making and trial design for targeted therapies in China.
Keywords:
Chinese participantsrandomized controlled trialstargeted anticancer drugstherapeutic outcometrial design

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