罕见病医疗保障机制的完善是中国特色多层次医疗保障体系高质量发展的重要组成部分。本文以血友病患者及其医疗保障情况作为研究对象,通过实地调查、个案访谈等研究方法,探讨罕见病患者治疗情况、用药方式、医疗机构选择和基本医保参保状况等。同时,综合分析我国血友病患者的医疗保障政策,包括国家医保药品目录准入情况、城乡居民大病保险制度、重特大疾病医疗保险和救助制度,以及地方血友病医疗保障政策等。基于这些分析,本文提出支持罕见病用药创新发展、完善医保药品目录准入机制、建立健全罕见病保障体系,以及加强罕见病诊疗能力建设的政策建议,以此推动罕见病医疗保障机制的完善。

The improvement of the medical security mechanism for rare diseases is an important part of the high-quality development of a multi-level medical security system with Chinese characteristics. In this paper, hemophilia patients and their medical security were the research objects. Through field investigation, case interview and other research methods, the paper discussed the treatments, medication methods, medical institution selection and basic medical insurance participation of rare disease patients. At the same time, it comprehensively analyzed the medical security policies of hemophilia patients in China, including the access to the national medical insurance drug list, the serious illness insurance system for urban and rural residents, the medical insurance and assistance system for major and very serious diseases, and the local hemophilia medical security policies. Based on these analyses, policy suggestions were put forward to support the innovation and development of rare disease drugs, improve the access mechanism of medical insurance drug catalogue, establish and improve a sound rare disease security system, and strengthen the construction of diagnosis and treatment capacity, so as to promote the improvement of the rare disease medical security mechanism.