To draw on the useful experience of the breakthrough therapy designation (BTD) system in the United States (US) to provide suggestions and references for improving the BTD system in China.

Through a comparative analysis of the implementation effects of the BTD systems in China and the US, the implementation effects and gaps of the BTD systems in the two countries were revealed.

The numbers of applications for BTD recognition in China and the US were generally on the rise, but the rates of BTD recognition and approval in China were significantly lower than those in the US, which may be related to the uneven quality of the applications and the lack of policy refinement. China's BTD drugs had a shorter pre-market clinical development time, but a longer review time, reflecting the difference in the strength of clinical trial guidance between the two countries' drug regulatory agencies. In terms of clinical trial design for antitumour drugs, China preferred single-arm, non-randomised design with objective remission rate as the primary endpoint; whereas the trial designs in the US were richer and more diverse. In addition, no BTD Chinese medicine has been approved for marketing. It is recommended that China's drug regulatory agencies learn from the US experience, formulate BTD review and management standards, strengthen the leading role of reviewers, set up interdisciplinary teams, strengthen the guidance of clinical trial development and improve the efficiency of research and development and review; at the same time, strengthen the supervision of BTD-recognised medicines after superimposed conditional approval for listing, and promote the declaration and approval of BTD traditional Chinese medicines. Pharmaceutical companies should strengthen the training of R&D teams on BTD regulations and skills, improve the quality of BTD drug applications, and promote the accelerated marketing of products to meet patients' clinical needs.