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| “港澳药械通”政策保障罕见病用药可及性的实施成效与优化路径 |
| 李慧仪,刘天阳,魏媛怡,梁翠丽,刘丽,毛喆,余乔立,邓后亮,贾炜,张婧绮,周文浩,莫小兰 |
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| (广州医科大学附属妇女儿童医疗中心,广州 510623) |
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| 摘要: |
| 目的:评估“港澳药械通”政策提升罕见病用药可及性的成效。方法:基于我院“港澳药械通”实践经验与案例分析,结合政策实施过程中的挑战,采用描述性研究方法探讨关键问题,提出优化路径与建议。结果:截至2025 年12 月,“港澳药械通”累计批准的药品覆盖罕见病目录207 个病种中的10 种,部分药品滞后周期由12 年缩短至4 年。政策实施面临管理复杂、供应链稳定性不足、医患信息知晓度低、申报流程繁琐、患者经济负担重等多重挑战。结论:“港澳药械通” 政策的实施有效缩短了境外创新罕见病药品的市场准入时间,但在管理体系、供应链保障、支付机制等方面需系统性优化。建议积极推进科学管理工具的应用、构建区域协同保障机制、建立多层次支付体系,从而全面提升政策实施效能。 |
| 关键词: “港澳药械通” 罕见病 药品可及性 |
| DOI：doi:10.13407/j.cnki.jpp.1672-108X.2026.04.002 |
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| 基金项目:广东省药学会2025 年罕见病超说明书用药评价研究科研基金,编号2025HJB01008。 |
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| Implementation Effectiveness and Optimization Approach of the “Hong Kong and Macao Medicine and Equipment Connect” Policy in Ensuring Drug Availability of Rare Disease |
| Li Huiyi, Liu Tianyang, Wei Yuanyi, Liang Cuili, Liu Li, Mao Zhe, Yu Qiaoli, Deng Houliang, Jia Wei, ZhangJingqi, Zhou Wenhao, Mo Xiaolan |
| (Guangzhou Women and Children ’ s Medical Center, Guangzhou Medical University, Guangzhou 510623, China) |
| Abstract: |
| Objective: To evaluate the implementation effectiveness of the “Hong Kong and Macao Medicine and Equipment Connect” policy in improving drug availability of rare disease. Methods: Based on practice and challenges during the policy implementation in our hospital, a descriptive research approach was employed to delve into key issues in policy execution and propose corresponding optimization approaches and recommendations. Results: By the end of Dec. 2025, the drugs approved by the “Hong Kong and Macao Medicine and Equipment Connect” policy had accumulated to cover 10 of the 207 diseases in the list of rare diseases, significantly reducing the lag for availability of some drugs from the original 12 years to 4 years. However, the implementation process still had challenges, including complex management, unstable supply chain, low awareness among clinicians and patients, cumbersome application processes, and nonnegligible financial burdens on patients. Conclusion: The “ Hong Kong and Macao Medicine and Equipment Connect” policy effectively shortens the time for rare drugs approved into China from overseas. However, optimizations are still required in areas such as management systems, supply chain, and payment mechanisms. It is recommended to further advance the
application of scientific management tools, establish regional collaborative mechanism, and create wide-ranging payment system to comprehensively enhance policy implementation effectiveness. |
| Key words: “Hong Kong and Macao Medicine and Equipment Connect” rare diseases drug availability |
