OBJECTIVE  To gain insight into the utilisation of this program by examining the attributes of the drugs included in the breakthrough therapy program in China from 2020 to 2024.

METHODS The drugs included in the breakthrough therapy program from January 2020 to July 2024 were determined based on the lists issued by the National Center of Drug Evaluation. The information of each drug's clinical trials was collected based on the China Drug Trials Platform, and the trial features were analyzed.

RESULTS A total of 296 drugs were included in the breakthrough therapy program, of which 39.4% employed non-randomised trials, 35.8% utilised single-arm designs, 69.1% conducted open-label trials, and 60.6% employed surrogate endpoints. Nevertheless, 47.5% of the drugs were in phase Ⅲ clinical trials, and 35.1% were designated as breakthrough therapy only after the commencement of clinical trials.

CONCLUSION The Chinese breakthrough therapy program offers flexibility in clinical trial design for drugs with significant clinical value, thereby facilitating the acceleration of the development and launch of medicines. However, there are still concerns that require attention, including the program's delayed involvement in research and development and the applicant's untimely requests for this program.