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Cancer Therapy Review Speed and Clinical Trials Evidence in Expedited Approval in China, 2005-2021
Background: Cancer poses a significant global health burden, with China experiencing the highest incidence and mortality rates. To facilitate timely patient access to novel therapies, China implemented Expedited Approval (EA) pathways starting in 2005. This study analyzes the clinical trials evidence and evaluates the impact of EA pathways on drug review speed for oncology drugs in China.
Methods: We conducted a cross-sectional analysis of cancer drugs approved under EA pathways in China between 2005 and 2021, using data from CDE reports, clinical trial websites, and literature. Key metrics included indication characteristics, trial information, and clinical benefits assessed using the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS). Descriptive statistics characterized indication features and clinical trial data, and factors influencing review durations were examined.
Findings: From 2005 to 2021, 99 oncology drugs received EA for 144 indications. The median approval time was 333.5 days, with faster review for foreign drugs and biologics. The approval times significantly decreased in 2015 and showed a downward trend since then. Priority review was predominantly used. Most pivotal clinical trials reported overall response rate as endpoints. Approximately one in third of trials with ESMO-MCBS scores showed clinically meaningful benefit.
Interpretation: While EA has improved drug accessibility in China, review times still lag behind those of the U.S. Food and Drug Administration (FDA). Many drugs showed moderate benefits, necessitating enhanced evaluation frameworks to balance innovation and efficacy. Drugs with significant clinical benefits exhibited shorter review periods, highlighting the EA program's focus on high-impact therapies.
Keywords: Cancer, expedited approval, pivotal clinical trials, China
Tian, Yun and Liu, Xingyu and Liu, Xingchen and Liu, Xiaoyong and Hu, Shuchen and Liu, Xiaodong and Yang, Caijun and Fang, Yu, Cancer Therapy Review Speed and Clinical Trials Evidence in Expedited Approval in China, 2005-2021. Available at SSRN: https://ssrn.com/abstract=5199083 or http://dx.doi.org/10.2139/ssrn.5199083
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