Challenges for Chinese innovative cancer drugs in going global: insights from multiregional clinical trials

Abstract

Since the reform of China's drug review and approval in 2015, it has successfully stimulated the enthusiasm of domestic pharmaceutical companies to carry out innovative drug research and development, especially in oncology. Chinese pharmaceutical companies that developed cancer drugs have begun shifting from an in China, for China model to an in China, for global model given the constrained market share. However, many challenges are confronting Chinese pharmaceutical companies when entering overseas markets. Conducting multiregional clinical trials (MRCTs) has been widely regarded by the industry as a crucial step for innovative drugs to achieve overseas commercialisation. The number of MRCTs conducted by Chinese pharmaceutical companies in the cancer field has substantially increased, but it remains scarce compared with multinational pharmaceutical companies. The populations enrolled in these trials conducted by Chinese pharmaceutical companies were most frequently from China, followed by the USA and Australia. The success rate of MRCTs conducted for cancer drugs was substantially lower in the USA than in China. As of May 25, 2025, Chinese pharmaceutical companies obtained approval from the US Food and Drug Administration and European Medicines Agency for 14 cancer indications each. Most of the pivotal trials supporting the US Food and Drug Administration and European Medicines Agency approval of these cancer drugs were conducted as MRCTs. The main challenges for these cancer drugs when listed overseas include little diversity in clinical trial populations and manufacturing issues. Geopolitical shifts have accelerated Chinese pharmaceutical companies to expansion into so-called Belt and Road markets (eg, southeast Asia and Africa). China should actively support the implementation of MRCTs, enhance the diversity of trial populations, and deepen collaboration with international regulatory agencies to accelerate the global commercialisation of domestic novel cancer drugs.