Nie Ruifang, Zhao Zhen, Zhang Yahui, Xu Bo, Zhang Wen
Department of Pharmacy, Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jinan, China.
Finance Department, Shandong Provincial Public Resources Trading Center, Jinan, China.
Front Pharmacol. 2025 Mar 31;16:1476910. doi: 10.3389/fphar.2025.1476910. eCollection 2025.
China attaches great importance to the prevention and treatment of rare diseases. The government has successively formulated two rare disease catalogs, and approved a variety of rare diseases treatment drugs. However, the actual supply and utilization of these drugs post-marketing remains unclear.
Based on the first and second list of national rare disease catalog in China, this study sort out the specific therapeutic drugs and extract procurement data from the provincial platform over the past 3 years. Subsequently, the drug allocation, shortages, delivery rate, temporal changes, and spatial distribution were analyzed to gain a comprehensive understanding of the local drug supply situation.
In the first catalog of 121 rare diseases, China has listed 54 specific drugs; in the second catalog of 86 rare diseases, 35 specific therapeutic drugs have been identified. Among these drugs, Shandong Province has access to 42 and 28, respectively. Spesolimab, Sodium Phenylbutyrate, Nitisinone and Emapalumab are currently in short supply, and the delivery rate of 16 drugs such as Selumetinib, Sirolimus (tablet), Octreotide, Dimethyl Fumarate and Lanreotide is below 80%. The number of available drugs increased year by year. The allocation of 19 drugs increased significantly, and 19 drugs were newly developed. The overall procurement cost of drugs increased and then decreased, which may be related to national policies. Additionally, there are significant regional disparities in drug cost, with Jinan, the provincial capital, leading at 770 million RMB.
The number of specific drugs for rare diseases has steadily increased, with the drug availability rate in Shandong Province reaching 80%. This indicates a generally high level of accessibility to drugs for rare diseases in China. However, attention should be given to improving the supply capacity for drugs that are in short supply and have a low delivery rate.
中国高度重视罕见病的防治工作。政府先后制定了两版罕见病目录，并批准了多种罕见病治疗药物。然而，这些药物上市后的实际供应和使用情况仍不明确。
基于中国国家罕见病目录的第一版和第二版清单，本研究梳理了具体的治疗药物，并提取了过去3年省级平台的采购数据。随后，对药物分配、短缺情况、配送率、时间变化和空间分布进行了分析，以全面了解当地药物供应情况。
在121种罕见病的第一版目录中，中国列出了54种具体药物；在86种罕见病的第二版目录中，确定了35种具体治疗药物。在这些药物中，山东省分别有42种和28种可供使用。斯佩索利单抗、苯丁酸钠、尼替西农和依帕卢单抗目前供应短缺，而司美替尼、西罗莫司（片剂）、奥曲肽、富马酸二甲酯和兰瑞肽等16种药物的配送率低于80%。可用药物数量逐年增加。19种药物的分配量显著增加，19种药物为新开发药物。药物总体采购成本先上升后下降，这可能与国家政策有关。此外，药物成本存在显著的地区差异，省会济南以7.7亿元领先。
罕见病的具体药物数量稳步增加，山东省的药物可及率达到80%。这表明中国罕见病药物的总体可及性水平较高。然而，应注意提高短缺和配送率低的药物的供应能力。
