中国罕见病多重保障机制的现状与对策  

The Current Status and Policy Recommendations of Multi-Security Mechanism for Rare Diseases in China

作　　者：张连雪 刘远立[1] ZHANG Lianxue;LIU Yuanli(School of Health Policy and Management,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100193,China)

机构地区：[1]中国医学科学院北京协和医学院卫生健康管理政策学院,北京100193

出　　处：《罕见病研究》2025年第1期7-13,共7页Journal of Rare Diseases

摘　　要：罕见病患者是医疗卫生体系中的弱势群体,亟需政府和社会的广泛关注。尽管罕见病的患者总数相对较少,但他们面临诸多挑战,包括诊断和治疗手段的有限性、药物可及性的不足及沉重的经济负担。因此,为罕见病患者提供更完善的医疗保障不仅关涉公共福祉,也反映社会进步的水平。本文系统分析了中国罕见病保障的现状及面临的挑战,重点探讨诊疗体系建设、多层次社会保障体系优化以及药物可及性提升等关键议题。研究表明,当前健康保险覆盖范围仍存在缺口,医疗资源配置不均衡,药物研发激励机制不足,严重影响罕见病患者的医疗可及性。针对这些问题,本文提出以下对策:加强政策支持,健全多层次社会保障体系,鼓励社会力量深度参与,并推动医疗技术与药物研发创新,以期构建更加完善的罕见病医疗保障体系,为患者提供更为全面和可持续的社会支持。Patients with rare disease are a vulnerable group in healthcare who urgently need more attention from the government and society.Despite their overall number,they face challenges such as limited diagnosis and treatment options,poor access to medications,and heavy financial burdens.Providing better protection for this group is not only essential for public well-being,but also an important indicator of societal progress.This paper analyzes the current status and challenges of rare disease protection in China,with a focus on building diagnostic and treatment networks,optimizing the multitiered social security system,and improving pharmaceutical access mechanisms.It highlights issues such as incomplete health insurance coverage,unequal resource allocation,and insufficient incentives for drug development.To address these challenges,we suggest strengthening policies,improving the multitiered social security system,encouraging social support,and promoting medical and pharmaceutical innovation to provide comprehensive care for rare disease patients.

关 键 词：罕见病 医疗保障 制度供给 对策建议 

分 类 号：R-012[医药卫生]