Ther Innov Regul Sci. 2026 Feb 16. doi: 10.1007/s43441-026-00927-x. Online ahead of print.
ABSTRACT
BACKGROUND: Rare diseases present urgent public health challenges requiring coordinated global efforts. This study analyzes China’s orphan drug innovation landscape through patent applications (1995-2023) to identify R&D patterns and policy implications.
METHODS: We analyzed 323 Patent Cooperation Treaty applications from Chinese entities using IPC classification and bipartite network modeling. Data from World Intellectual Property Organization were retrieved via PatSnap, with diseases mapped to China’s Rare Disease Catalogues (207 conditions). Network metrics quantified disease-technology linkages.
RESULTS: Three key findings emerged: (1) Patent filings surged 58.2% (188 patents) during 2018-2023, coinciding with China’s regulatory reforms; (2) Innovation concentrated in oncology/neurology (34.3% patents cover 2 diseases), with 66.2% of catalogued diseases lacking patents; (3) Dominant technologies are small molecules and genetic therapies. Network analysis revealed an R&D ecosystem without a single dominant entity, alongside robust collaborative ties between Chinese and U.S. organizations.
CONCLUSIONS: China’s orphan drug innovation is expanding rapidly but remains uneven, concentrated in specific diseases and technological platforms. Policy interventions are needed to address therapeutic neglect through tiered incentives, dedicated funding for neglected diseases, and global partnerships to accelerate equitable treatment access.
PMID:41697637 | DOI:10.1007/s43441-026-00927-x
