State Key Laboratory of Quality Research in Chinese Medicine, Institute of Chinese Medical Sciences, University of Macau, Macao Special Administrative Region, China.
School of Chinese Materia Medica, Guangdong Pharmaceutical University, Guangzhou, China.
Cancer. 2024 Mar 1;130(5):671-682. doi: 10.1002/cncr.35106. Epub 2023 Nov 20.
Since reforms were introduced to incentivize drug innovation in 2015, the Chinese pharmaceutical market has experienced unprecedented prosperity, with more new drugs than ever before, especially anticancer treatments. In 2021, Chinese regulatory agencies issued the new guideline for clinical research and development of antitumor drugs, triggering a series of responses on the drug market. Limited research has outlined the nature of the original new drugs in China to understand the dynamic response of the market.
The objective of this article was to map the clinical development of approved new oncology drugs in China from 2015 to 2021 and differed from previous studies by focusing on original new drugs, using the United States as a benchmark, and elaborating the endogenous features of clinical trials.
Clinical trials conducted in China have risen to a level similar to that of the United States in many aspects of trial design, but there is still distance between the implementation and operational details of clinical trials. In the meantime, China has made significant breakthroughs in drug approval. Greater than 60% of novel anticancer drugs in China received accelerated approved for their first listing. Approximately 90% of the pivotal clinical trials supporting initial drug approval used surrogate measures as end points, and one half were nonrandomized or did not have a control group. However, duplicate development without evidence of a clinical advantage compared with current therapies was widely observed.
By presenting a multidimensional landscape of clinical trials and approvals in the real world, this review allows interested researchers, developers, and even regulators to understand what has been done and what should be done next in anticancer drug development in China.
自 2015 年推出激励药物创新的改革以来，中国医药市场经历了前所未有的繁荣，新药数量前所未有，尤其是抗癌治疗药物。2021 年，中国监管机构发布了抗肿瘤药物临床研究和开发的新指南，引发了药物市场的一系列反应。有限的研究概述了中国原创新药的性质，以了解市场的动态反应。
本文的目的是绘制 2015 年至 2021 年在中国批准的新型肿瘤药物的临床开发情况，并与之前的研究有所不同，重点关注原创新药，以美国为基准，并详细阐述临床试验的内在特征。
中国开展的临床试验在试验设计的许多方面已达到与美国相似的水平，但临床试验的实施和操作细节仍存在差距。与此同时，中国在药物批准方面取得了重大突破。在中国，超过 60%的新型抗癌药物因其首次上市而获得加速批准。大约 90%支持初始药物批准的关键性临床试验使用替代指标作为终点，其中一半是非随机的或没有对照组。然而，广泛观察到与当前疗法相比没有临床优势的重复开发。
通过呈现真实世界临床试验和批准的多维景观，本综述使感兴趣的研究人员、开发者甚至监管机构能够了解在中国抗癌药物开发方面已经做了什么，以及下一步应该做什么。
