Trend of clinical trials of new drugs for rare diseases in China in recent 10 years

Abstract

Abstract Background: Rare disease is a general term for a disease that affects a small number of people but be recognized as a global public health priority. Governments worldwide are paying more and more attention to the academical research and drug investment of rare diseases. The conduct of rare disease clinical trials is still difficult, despite the promotion of government policies and the awakening of social consciousness. In this article, we outlined the characteristics and obstacles of clinical trials of rare diseases in China and expected to provide reference for subsequent clinical trials in this field. Results: In the recent years, China has made some progress in clinical trials of rare diseases in the past 10 years. There are a total of 481 clinical trials on rare diseases, covering more than 10 rare diseases with high incidence. Clinical trial applications on rare diseases for a total of 481 were submitted and with an average annual growth rate of 28.2% from 2013 to 2022. The number of clinical trial application for rare diseases in 2016 dramatically increased by 80% compared to 2015 due to the policy document issued by China for clinical research in rare diseases in 2015. Besides, about 70% of applications registering for clinical trials could recruit subjects as expected. Despite this, the number of clinical trials of rare diseases in China is smaller than that of the United States, Europe and Japan, and the types of infant drugs are limited to biological products and chemical drugs lacking other new treatments. Conclusions: Efforts have been made in recent years to develop clinical research on rare diseases in China. The number of clinical trials for rare diseases in China is growing steadily every year, which is inseparable from the support of the country, society and rare disease patients. Still, there is a large gap between China and other developed countries in this field and this merit further investigation.