Core Insights - Approximately 100 rare disease medications have been included in the medical insurance catalog, covering 42 types of rare diseases [1] - In 2024, the medical insurance fund will pay 8.6 billion yuan for rare disease medications, accounting for 7.7% of the total payment for medications during the agreement period [1] - The establishment of a multi-layered guarantee system for rare diseases is underway, combining basic medical insurance, major illness insurance, and medical assistance [1][2] Group 1 - The National Healthcare Security Administration is actively promoting the inclusion of medications in the insurance catalog and the construction of a "dual-channel" supply mechanism to improve drug accessibility [1][2] - The number of hospitals in the national rare disease diagnosis and treatment collaboration network has increased from 324 in 2019 to 419 currently [1][2] - The national rare disease direct reporting system has registered a total of 1.64 million cases, with 1.5 million confirmed cases [2] Group 2 - Future selection criteria for rare disease medications should consider severity, intervention methods, and social impact, rather than just incidence rates [3] - The entry of rare disease medications into the catalog must prioritize clinical and patient benefits, focusing on drugs with clear biomarkers and measurable efficacy [3] - There is a need to challenge the perception that rare disease drugs must be high-priced, as some production technologies are mature and costs are controllable [3] Group 3 - Emphasis on refined management of rare disease medications is crucial due to their high cost and long-term prescription nature [3] - Collaboration among over 400 institutions in the national rare disease diagnosis and treatment collaboration network is essential for effective management practices [3][4] - The 2025 China Rare Disease Conference was co-hosted by multiple organizations, highlighting the collective effort in addressing rare diseases [4]

Core Insights - Hainan Boao Lecheng International Medical Tourism Pilot Zone is becoming a crucial link between rare disease drug companies and patients through policy iterations, insurance collaboration, and multi-dimensional empowerment [1] Policy Iteration - The policy framework for rare disease drug assurance in Lecheng has evolved through three versions: - Version 1.0 focuses on introducing licensed drugs and devices to allow some patients to access urgently needed medications [2] - Version 2.0 allows the use of real-world data from rare disease drugs for expedited domestic approval [2] - Version 3.0 expands the coverage to include special medical foods, health products, and new biomedical technologies [2] Special Drug Insurance - Lecheng has launched a special drug insurance plan to significantly reduce the financial burden on patients, with premiums starting at 49 yuan and coverage up to 2 million yuan for 120 types of global special drugs and 10 types of imported devices [3] Future Initiatives - Lecheng aims to strengthen the connection between rare disease drug companies and patients by leveraging special drug policies and introducing more special medical foods [4] - The zone has received approval for biomedical new technology policies, allowing clinical research and application transformation for technologies that have completed Phase II trials [4] - The initiative includes collaboration with the National Medical Products Administration and the National Healthcare Security Administration to expedite drug registration and insurance access [4]

新京报讯（记者王卡拉）当创新药上市为罕见病患者带来希望的同时，可及性问题成为他们治疗道路上 最大的障碍。在9月19日-21日举行的第十四届中国罕见病高峰论坛上，针对罕见病创新药支付相关话 题，全国政协委员、对外经济贸易大学保险学院副院长孙洁呼吁，在地方试点基础上，在国家层面规划 探索建立国家级罕见病专项基金，出台相关的基本原则、发展目标等，积极探索与保障基金与国家基本 医保、大病保险、医疗救助、商业保险等补偿政策及相关资源的衔接，实现罕见病高额药保障一站式结 算，切实减轻罕见病患者及家庭经济负担。 孙洁认为，罕见病专项基金是目前比较理想的一种保障支付方式，有利于解决医保保障罕见病、创新药 带来的对公平和效率的质疑；能够有效实现医保的筹资路径，充分调动社会资源参与，保障罕见病用 药；还能进一步提高公众对罕见病的认知，有效助力创新药的本土研发。她呼吁，以建立罕见病专项基 金作为制度抓手，并强调立法保障的重要性，让支付机制走向规范和可持续。地方罕见病专项基金由省 级社保专户管理，基金来源既有财政专项拨款，也有民政部门引导的基金会定向捐赠、企业和社会捐 赠、福利彩票等。国家专项基金应更注重多元筹资、医保基金结余、财政 ...

经济观察报记者张铃 "我们需要总结地方试点的经验，从下而上推动国家级罕见病专项基金的建立。"在9月20日举行的第十 四届中国罕见病高峰论坛上，全国政协委员、对外经济贸易大学教授孙洁说。 孙洁认为，对于罕见病用药保障而言，支付是一个尤其重要的难题。 再比如，江苏省在医疗保障条例中精准、明确地提出建立罕见病用药保障机制，用药保障基金实行省级 统筹、单独筹资，建立由政府主导、市场主体、社会慈善组织等参与的多渠道筹资机制。罕见病用药保 障资金纳入省财政社保专户管理，专款专用，独立核算。在用药保障范围，对药品进行有效性评估、专 家论证、价格谈判。 孙洁认为，尽管地方取得了一些经验，但分散化探索模式也暴露出固有局限性，成为构建全国保障性体 系的瓶颈。目前，需要总结地方试点的经验，从下而上推动国家级罕见病专项基金的建立。她建议，采 用以罕见病专项基金为核心，补充医疗保险、医疗救助、政策性的商业医疗保险、慈善基金等多方共付 的模式减轻财政压力和患者经济负担。 孙洁列举了罕见病专项基金的三个主要意义： 一是有利于解决医保保障罕见病、创新药带来的对公平和效率的质疑。针对罕见病设立专项基金，一方 面可以跳出现有的医保制度，通过专门 ...

王雪静 图片来源：主办方供图 近年来，我国对罕见病重视度提升，为罕见病用药保障带来机遇。王雪静表示，医保层支持举措包括优化准入程序（如2024年医保目录调整对罕见病用药 放宽上市时间限制），开展医保谈判降价，通过"双通道"提升保障水平，加强门诊保障（开展慢病和特病单独支付、建立普通门诊统筹）等。 每经记者｜甄素静 每经编辑｜陈柯名 董兴生 向江林 9月20日，在第十四届中国罕见病高峰论坛上，全国人大代表、湖北省咸宁市中心医院院感办主任王雪静以《关于加强罕见病用药保障》为题发表了主题 演讲。 王雪静表示，当前罕见病用药存在药物可及性低、负担重，诊疗体系和支持能力不足，地区间保障不平衡，支付体系与高价药矛盾，药物研发与市场激励 不足等困境，严重影响用药保障。 记者|甄素静 编辑|陈柯名 董兴生 向江林 校对|董兴生 ｜每日经济新闻 nbdnews 原创文章｜ 未经许可禁止转载、摘编、复制及镜像等使用 王雪静认为，应对罕见病用药难题，需依靠政府主导的多层次医疗保障和综合救助体系。为此，她提出三项建议：第一，创新罕见病医保准入模式，借鉴 国际风险共担协议经验，结合我国国情，对严重威胁生命且无等效治疗药物的罕见病实行风 ...

近年来，我国对罕见病重视度提升，为罕见病用药保障带来机遇。王雪静表示，医保层支持举措包括优化准入程序（如2024年医保目录调整对罕见病用药放 宽上市时间限制），开展医保谈判降价，通过"双通道"提升保障水平，加强门诊保障（开展慢病和特病单独支付、建立普通门诊统筹）等。 王雪静认为，应对罕见病用药难题，需依靠政府主导的多层次医疗保障和综合救助体系。为此，她提出三项建议：第一，创新罕见病医保准入模式，借鉴国 际风险共担协议经验，结合我国国情，对严重威胁生命且无等效治疗药物的罕见病实行风险共担，建立第三方效果评估机制。 每经记者｜甄素静 每经编辑｜董兴生 9月20日，在第十四届中国罕见病高峰论坛上，全国人大代表、湖北省咸宁市中心医院院感办主任王雪静以《关于加强罕见病用药保障》为题发表了主题演 讲。 王雪静表示，当前罕见病用药存在药物可及性低、负担重，诊疗体系和支持能力不足，地区间保障不平衡，支付体系与高价药矛盾，药物研发与市场激励不 足等困境，严重影响用药保障。 第三，建立罕见病用药专项保障基金，实行省级统筹、分账管理、独立核算、专项专用。资金来源需政府（医保结余、定向拨款）与社会（民政引导社会力 量、彩票公益金、税 ...