Wei Yuxuan, Zhang Yichen, Xu Ziyue, Wang Guoan, Zhou Yue, Li Huangqianyu, Shi Luwen, Naci Huseyin, Wagner Anita K, Guan Xiaodong
International Research Centre for Medicinal Administration, Peking University, Beijing, China.
Department of Pharmacy Administration and Clinical Pharmacy, School of Pharmaceutical Sciences, Peking University, Beijing, China.
Lancet Reg Health West Pac. 2024 Apr 2;45:101055. doi: 10.1016/j.lanwpc.2024.101055. eCollection 2024 Apr.
Perceived delays in cancer drug approvals have been a major concern for policymakers in China. Policies have been implemented to accelerate the launch of new cancer drugs and indications. This study aimed to assess similarities and differences between China and the United States in the approvals, timing, and clinical benefit evidence of cancer drug indications between 2001 and 2020.
This study retrospectively identified all cancer drugs and indications approved in both China and the United States from January 1st, 2001 to December 31, 2020, and described differences in approval times as well as in submission and review times. Information on the availability of overall survival benefit evidence by December 31, 2020, was collected. Univariate and multiple logistic regression analyses were used to assess whether evidence of benefit and other factors affected the propensity and timing of approvals of cancer drug indications in China.
Between 2001 and 2020, 229 indications corresponding to 145 cancer drugs approved in the United States were identified. Of those, 80 indications (34.9%) were also approved in China by the end of 2020. Cancer drug indications were approved in China at a median of 1273.5 days after approval in the United States. The median submission and review time differences for cancer drug indications in China were 1198.0 days and 180.0 days respectively. Submission time differences accounted for most of the approval time differences (p < 0.001). Indications supported by overall survival benefit evidence had shorter median review time differences (145.0 days) than those without such evidence (235.0 days, p = 0.008). Indications with overall survival benefit evidence were 3.94 times more likely to be approved in China compared to those without such evidence (p = 0.001), controlling for approval year, cancer type, and the prevalence of cancer by site.
FDA-approved cancer drug indications demonstrating a survival benefit were more likely to receive approvals in China with shorter regulatory review times compared to indications without such evidence. Given that manufacturer submission times were the main driver of cancer drug approval times in China, factors influencing submission timing should be explored.
No funding.
癌症药物获批的延迟问题一直是中国政策制定者的主要担忧。中国已实施相关政策以加速新型癌症药物及适应证的上市。本研究旨在评估2001年至2020年间中美两国在癌症药物适应证的获批情况、时间以及临床获益证据方面的异同。
本研究回顾性确定了2001年1月1日至2020年12月31日期间中美两国均获批的所有癌症药物及适应证，并描述了获批时间以及提交和审评时间的差异。收集了截至2020年12月31日总生存获益证据的可得情况。采用单因素和多因素逻辑回归分析来评估获益证据及其他因素是否影响中国癌症药物适应证的获批倾向和时间。
2001年至2020年间，共确定了美国获批的145种癌症药物对应的229个适应证。其中，截至2020年底，80个适应证（34.9%）在中国也获得了批准。中国癌症药物适应证在美国获批后的中位获批时间为1273.5天。中国癌症药物适应证的中位提交和审评时间差异分别为1198.0天和180.0天。提交时间差异占获批时间差异的大部分（p<0.001）。有总生存获益证据支持的适应证的中位审评时间差异（145.0天）比无此类证据的适应证（235.0天，p=0.008）更短。在控制获批年份、癌症类型和各部位癌症患病率的情况下，有总生存获益证据的适应证在中国获批的可能性是无此类证据适应证的3.94倍（p=0.001）。
与无生存获益证据的适应证相比，美国食品药品监督管理局（FDA）批准的显示有生存获益的癌症药物适应证在中国更有可能获批，且监管审评时间更短。鉴于在中国制造商的提交时间是癌症药物获批时间的主要驱动因素，应探索影响提交时间的因素。
无资助。
