Innovative Drugs Approved in China After Registration Classification Reform: Current Status, Disparities, and Challenges.

Since 2016, the China National Medical Products Administration (NMPA) has reformed its drug registration classification rules and defines the innovative drugs as new drugs that have not been marketed domestically and overseas. This study sought to systematically evaluate the characteristics of the innovative drugs approved in China. The cross-sectional study included all innovative small molecules that submitted a marketing application after 2016 and were approved by the NMPA before 2024, as well as all innovative biologics and Chinese medicines that submitted after 2020 and were approved before 2024. Based on the NMPA's open databases, information on each drug and its pivotal trial was collected. A total of 167 innovative drugs were approved, consisting of 58.1% small molecules, 31.1% biologics, and 10.8% Chinese medicines. The domestic drugs dominated (86.8%) and continued to grow from 2018 to 2024. The drugs were concentrated in the oncology area (43.7%), particularly in biologics (55.8%). Most oncology drugs (90.4%) were approved based on surrogate end-point evidence. Chinese medicines received much less regulatory flexibility than small molecules/biologics. The expedited programs helped accelerate the clinical development and review processes. However, the review time tended to exceed the stipulated timeframes. The great efforts of the Chinese regulator and industry in nurturing innovation are bearing fruit. More investments from the regulator and drug developers are needed to focus on R&D in differentiated therapies, financial incentives and regulatory priorities for high-quality innovations, along with review capacity improvements, in order to facilitate an innovation-driven and internationally competent pharmaceutical industry in China.