Challenges and progress in access to rare cancer drugs in China: Insights from clinical trials, availability and affordability.

This study evaluated the development and accessibility of targeted therapies for 23 rare cancers in China. Clinical trials increased between 2013 and 2025 but have recently plateaued, with 12% terminated or suspended. Domestic enterprises now dominate trial activity, while trials of imported drugs have declined. As of 31 December 2025, 63 targeted-therapy indications had been approved globally, compared with 40 indications for 15 rare cancers in China, and eight rare cancers still lacked targeted treatments. The median monthly treatment cost was US$2165, substantially exceeding monthly disposable income per capita. Costs were significantly lower for drugs included in the National Reimbursement Drug List or those with generic alternatives. Despite progress, important gaps remain in R&D incentives, treatment availability and affordability.