To analyze the status quo of clinical trials of rare disease drugs in China and abroad, to provide reference for further promoting the development of clinical research of rare disease drugs in China.

By extracting the data of clinical trials of rare disease drugs in China's "drug clinical trial registration and information publicity platform", weanalyzedthe characteristics of clinical trials of rare disease drugs in China from the aspects of project registration, test type and stage, involved diseases, and scheme design, and comparedthem with the registration data of foreign clinical trials of rare disease drugs.

As of March 31, 2022, there were a total of 337 clinical trials of rare disease drugs in China's "drug clinical trial registration and information publicity platform", involving 37 diseases and 125 experimental drugs. Among them, there were 127 bioequivalence tests, accountingfor 37.6%; 278 domestic projects, accountingfor 82.4%; and 103 projects involving children, accountingfor about 37.0%.Compared with those in other countries, clinical trials of rare disease drugs in China had the following characteristics: (1) the number of clinical trialsshowedan obviously increasing trend; (2) there were few clinical trials of innovative drugs but many bioequivalence tests of generic drugs; and (3) the proportion of trials involving children was high.

The clinical research on innovative drugs for rare diseases is still a weak link in China, and we need to improve legislation, increase financial support, enhance personnel training, and improve innovative research paths and methods.