Zhang Yumeng, Yang Minghao, Su Qiang, Sui Yuanhao, Sun Lihua
Department of Pharmacy Administration, School of Business Administration, Shenyang Pharmaceutical University, 103 Wenhua Road, Shenyang 110016, China.
Healthcare (Basel). 2025 Aug 31;13(17):2178. doi: 10.3390/healthcare13172178.
: Patients with rare diseases in China face extremely high medical expenses. The current coverage framework remains inadequate in terms of coverage depth and proactive risk control, underscoring an urgent need for institutional reform. : This study employs a policy content analysis approach to review the current landscape of rare disease protection in China. Drawing on risk management theory and the health capital model, it constructs an analytical framework to examine potential institutional reforms through the lens of risk response pathways and the efficiency of health investment. : The findings reveal that basic medical insurance (BMI) provides limited financial protection for patients with rare diseases. Among China's 31 provincial-level administrative centers, 24 have set general outpatient reimbursement ceilings under the urban and rural resident basic medical insurance (URRBMI) at 1000 RMB or less. In comparison, 24 cities have set outpatient reimbursement limits under the urban employee basic medical insurance (UEBMI) at 6000 RMB or less. The security system relies predominantly on the BMI, while supplementary mechanisms have failed to provide effective support or continuity in coverage. Current policies are generally reactive, with coverage typically triggered only after a confirmed diagnosis and often lacking early intervention or preventive strategies. : China's rare disease security system urgently requires structural improvements in coverage depth and proactive risk management. The proposed Dedicated Insurance Scheme for Rare Diseases (DISRD) presents a feasible and sustainable model for China's multi-tiered system of securing rare diseases. It provides valuable institutional insights for other countries and regions seeking to build public health systems with proactive risk control capabilities.
中国罕见病患者面临极高的医疗费用。当前的保障框架在保障深度和主动风险控制方面仍存在不足，凸显了进行制度改革的迫切需求。
本研究采用政策内容分析法审视中国罕见病保障的现状。借鉴风险管理理论和健康资本模型，构建分析框架，从风险应对路径和健康投资效率的角度审视潜在的制度改革。
研究结果显示，基本医疗保险为罕见病患者提供的经济保障有限。在中国31个省级行政中心中，有24个将城乡居民基本医疗保险的普通门诊报销上限设定为1000元及以下。相比之下，有24个城市将城镇职工基本医疗保险的门诊报销限额设定为6000元及以下。保障体系主要依赖基本医疗保险，而补充机制未能提供有效的支持或持续的保障。当前政策普遍具有滞后性，通常只有在确诊后才提供保障，且往往缺乏早期干预或预防策略。
中国的罕见病保障体系迫切需要在保障深度和主动风险管理方面进行结构性改进。拟议的罕见病专项保险计划为中国多层次的罕见病保障体系提供了一个可行且可持续的模式。它为其他寻求建立具有主动风险控制能力的公共卫生体系的国家和地区提供了宝贵的制度借鉴。
