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Background <p>China’s Expedited Approval (EA) pathways, launched in 2005, aim to accelerate patient access to novel cancer therapies. Yet the clinical value and regulatory efficiency of oncology drugs approved under EA have not been comprehensively evaluated in a Chinese context or compared against global benchmarks.</p> Methods <p>We conducted a cross-sectional analysis of all malignant hematology and oncology drugs granted EA by China’s National Medical Products Administration between 2005 and 2021. Publicly available CDE reports, clinical trial registries, and literature sources were used to extract indication characteristics, review durations, trial design features, and ESMO-MCBS scores. Descriptive statistics summarized drug and trial features. Kruskal-Wallis tests compared median review times across factors; multiregional trial design and clinical benefit associations were explored qualitatively.</p> Results <p>Ninety-nine drugs were granted expedited approval for 144 indications, 68% were small-molecule agents and 32% biologics; 86% underwent Priority Review, 28% Special Approval, and 24% Conditional Approval. Median review duration declined from over 1 000 days (2014&amp;2016) to ~ 300 days post-2015. Imported drugs (median 289 days) were reviewed faster than domestic ones (401 days). Of 137 pivotal trials supporting new indications, 70% were randomized and 66% parallel-controlled; primary endpoints were ORR (43%) and PFS (37%). Among 86 solid-tumor trials with ESMO-MCBS data, 34% achieved scores indicating meaningful clinical benefit (grades 4–5 or A–B).</p> Conclusion <p>China’s EA program has markedly accelerated oncology drug approval timelines to levels approaching the EMA, though still trailing the FDA. While most pivotal trials used surrogate endpoints, only one-third of solid-tumor indications demonstrated high clinical benefit per ESMO-MCBS. To balance expedited access with therapeutic value, future reforms should emphasize early benefit assessment, real-world outcomes, and alignment of surrogate endpoints with overall survival benefits.</p>

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Accelerating cancer therapy review: a cross-sectional analysis of expedited approval in China, 2005–2021

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年2月16日

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  • Yun Tian,
  • Xingyu Liu,
  • Xingchen Liu,
  • Xiaoyong Liu,
  • Shuchen Hu,
  • Xiaodong Liu,
  • Caijun Yang,
  • Yu Fang

摘要

Background

China’s Expedited Approval (EA) pathways, launched in 2005, aim to accelerate patient access to novel cancer therapies. Yet the clinical value and regulatory efficiency of oncology drugs approved under EA have not been comprehensively evaluated in a Chinese context or compared against global benchmarks.

Methods

We conducted a cross-sectional analysis of all malignant hematology and oncology drugs granted EA by China’s National Medical Products Administration between 2005 and 2021. Publicly available CDE reports, clinical trial registries, and literature sources were used to extract indication characteristics, review durations, trial design features, and ESMO-MCBS scores. Descriptive statistics summarized drug and trial features. Kruskal-Wallis tests compared median review times across factors; multiregional trial design and clinical benefit associations were explored qualitatively.

Results

Ninety-nine drugs were granted expedited approval for 144 indications, 68% were small-molecule agents and 32% biologics; 86% underwent Priority Review, 28% Special Approval, and 24% Conditional Approval. Median review duration declined from over 1 000 days (2014&2016) to ~ 300 days post-2015. Imported drugs (median 289 days) were reviewed faster than domestic ones (401 days). Of 137 pivotal trials supporting new indications, 70% were randomized and 66% parallel-controlled; primary endpoints were ORR (43%) and PFS (37%). Among 86 solid-tumor trials with ESMO-MCBS data, 34% achieved scores indicating meaningful clinical benefit (grades 4–5 or A–B).

Conclusion

China’s EA program has markedly accelerated oncology drug approval timelines to levels approaching the EMA, though still trailing the FDA. While most pivotal trials used surrogate endpoints, only one-third of solid-tumor indications demonstrated high clinical benefit per ESMO-MCBS. To balance expedited access with therapeutic value, future reforms should emphasize early benefit assessment, real-world outcomes, and alignment of surrogate endpoints with overall survival benefits.