ArticleFrontiers in pharmacology2026
The impact of China's drug regulatory reform on access to orphan drugs: a cross-sectional study of diseases listed in the rare disease catalog.
Article in Frontiers in pharmacology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Objective: China released two batches of the Rare Disease Catalog in 2018 and 2023, respectively, listing a total of 207 diseases. This study aims to analyze the accessibility of medications for diseases included in the catalog and assess the impact of drug regulatory reforms on the supply of medications for rare diseases. Methods: This study compiled five categories of information on drugs for 207 rare diseases: basic information on drug availability, the number of drug approvals and production capacity, drug development trends, marketing approval efficiency, and medical insurance coverage. Descriptive statistical analysis was employed. Results: By the end of 2025, out of 207 rare diseases, 98 (47.3%) had drugs approved in China, with a single company being the exclusive supplier for 37 of these diseases. Between 2015 and 2025, the NMPA approved a total of 2,007 Investigational New Drug (IND) applications and 1,134 New Drug Applications (NDAs) for rare diseases, showing an overall upward trend. Following the publication of the catalog, the median review time for NDA applications for rare disease drugs was reduced by 79 days compared to pre-publication (516 vs. 595, P < 0.001); for drugs eligible for Priority Review and Approval (PRA), the median review time was reduced by 73 days compared to non-PRA drugs (493 vs. 566, P < 0.001). The time difference between the market launches of the same drug in China and the United States has been reduced by 1.52 years (5.53 vs. 4.01, P < 0.001). Of the 263 drugs used to treat 98 rare diseases, 181 were included in the medical insurance coverage, and the time to inclusion in the insurance coverage was reduced by 1.01 years following the publication of the directory (2.09 vs. 1.07, P < 0.001). Conclusion: With the release of the rare disease catalog and the advancement of drug regulatory reforms, access to orphan drugs in China has improved. However, more than half of all rare diseases still face the dilemma of having no available treatments. It is recommended that the coordination mechanism between drug regulation and medical insurance be maintained to better meet patients' clinical medication needs.
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