Assessment of Efficacy Evidence for Approvals of Novel Drugs in China Between 2018 and 2022

After the reform of China's drug review and approval system, there has been a notable increase in the number of novel drugs approved. This cross-sectional study aimed to assess the characteristics of efficacy evidence for novel drugs approved in China. This study included 240 novel drugs approved by the NMPA for 256 indications between 2018 and 2022. Compared with imported original drugs, the trials of innovative drugs were less frequently subjected to be randomized (67.7% vs. 80.3%; P = 0.01) and placebo (32.3% vs. 45.5%; P = 0.02), and more likely to use external control and surrogate endpoint. There was a significant difference in all features between pivotal trials of drugs with conditional approval and regular approval. Among imported original drugs, the drug for urgent clinical needs used placebo control (58.1% vs. 39.5%; P < 0.001) and scale endpoint more frequently, whereas rare disease drugs seldom utilized active control. The median number of subjects across all pivotal trial intervention groups was 197 (IQR, 86-379), with a median trial duration of 6.0 months (IQR, 3.0-12.7). The median number of pivotal trials per indication for imported original drugs was higher than that of innovative drugs (2.0 [IQR, 1.0-3.0] vs. 1.0 [IQR, 1.0-1.0]; P < 0.001). The innovative drugs typically had either one pivotal trial or one trial plus supportive evidence, both of which accounted for 79.8% of all indications of innovative drugs. The most commonly applied types of supportive evidence across all indications were additional studies and mechanistic evidence. This study illustrated the characteristics of the quality and quantity of efficacy evidence. Special regulatory programs and special drug catalogs offered the agency regulatory flexibility with respect to evidentiary requirements.