Clinical trials of orphan drugs in China over the decade 2012–2022: Opportunities and challenges

Rare diseases refer to diseases with very low prevalence. Along with the support of national policies and improvement of research capability, a new landscape for orphan drug is emerging in China. To identity unmet clinical needs and provide insight on the development of orphan drugs, we reviewed the changes over time of orphan drug clinical trials in China from 2012 to 2022. A total of 261 trials of 40 drugs were initiated, of which 66.3% trials were sponsored by Chinese local pharmaceutical enterprises. Among the 261 trials, chemical drugs (about 63.6%) and biological products (35.6%) account for the high proportions, and traditional Chinese medicine (0.8%) was the least; the indications mainly focused on homozygous hypercholesterolemia, hemophilia, multiple sclerosis and idiopathic pulmonary fibrosis; single-arm study design was applied to 50% of the clinical trials, with an average sample size of 52 participants. Additionally, totally 122 trials were completed by January 2022, of which the average duration time was 15.7 months for new drug and 3.5 months for generic drug, respectively. The trends over time illustrated that remarkable progress has been achieved in development of orphan drugs in China since 2012. Given the large patient pool and the rising capability of innovation, it is believed that China will contribute more to the global drug pipelines for rare diseases.