OBJECTIVES: To provide advices for Chinese pharmaceutical companies by analyzing the current situation of international and Chinese orphan drugs market and getting insight into Chinese market prospect. METHODS: The data of R&D and post-marketing orphan drugs were collected from WHO and database (China National Knowledge Infrastructure, CNKI). The data was divided into group USA (group_USA), group EU (group_EU) and group China (group_CHN). Descriptive analysis was applied by Excel. RESULTS: There are more than 6,000 kinds of rare diseases were confirmed by WHO and they accounted for about 10% of human diseases. However, only less than 1% of rare diseases have effective treatment of drugs. According to the world average incidence rate, there were nearly 10 million rare diseases patients in China in 2013. But lack of attention and policy support, the shortage of orphan drugs for rare disease become more and more serious in China. By the end of 2013, group_USA had approved 373 orphan drugs post-marketing by FDA and 9 kinds were approved in the first half of 2014. There were about 452 new drugs in R&D. Group_EU had 67 kinds of orphan drugs were approved. But in China, there were few kinds of orphan drugs approved. Group_CHN had 61 kinds of imported orphan drugs post-marketing and 84 kinds in R&D by Chinese companies in 2013. Most of the species were in the field of malignant tumor. So rare diseases market was not competition very heated for Chinese pharmaceutical companies. CONCLUSIONS: There was a large number of patients and an increasing prevalence rate of rare diseases in China. It would be prospective to develop orphan drugs for Chinese pharmaceutical companies, especially in malignant tumor disease.