| 引用本文：徐陆欣怡, 王妮, 尹适成, 滕彩霞, 崔丹.我国罕见病药品的可及性及采购使用现状研究[J].中国卫生政策研究,2022,15(2):60-64 |
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| 我国罕见病药品的可及性及采购使用现状研究 |
| 投稿时间：2021-12-07 修订日期：2022-02-04 PDF全文浏览 HTML全文浏览 |
| 徐陆欣怡1,2, 王妮3, 尹适成1,2, 滕彩霞1,2, 崔丹1,2 |
1. 武汉大学公共卫生学院 湖北武汉 430071;
2. 武汉大学全球健康研究中心 湖北武汉 430072;
3. 南京大学医学院附属鼓楼医院 江苏南京 210008 |
| 摘要:目的：分析我国罕见病药品的可及性和采购使用现状，为完善我国罕见病用药保障政策提供参考。方法：采用文献分析法，从可获得和可负担角度评价药品可及性；从采用率、构成比等分析罕见病药品采购数量、金额等现状。结果：从可获得看，国内上市药品仅占全球上市药品的58.9%，16种罕见病的患者面临“境外有药，境内无药”，24.2%的国内上市药品未注册罕见病适应症，20种罕见病的所有药品均为超适应症用药；从可负担看，仅43.4%的国内上市药品实际得到了医保覆盖，76.0%的医保目录外药品属于高值药品。2018和2019年全国医疗机构罕见病药品采购DDDs同比增长571.56%和13.80%，采购金额同比增长45.22%和25.40%。部分高值罕见病药品的覆盖患者率仅为0.02%~3.06%。结论：我国罕见病患者的药品可获得性不及境外，应完善、落实药品审评审批制度改革，推动仿制药研发上市。目录准入、药品集采改善了罕见病药品的可负担性，但罕见病患者尤其是高值罕见病药品所涉患者的受益仍然有限；建立多层次的罕见病用药保障体系，重点加强高值罕见病药品的兜底保障。 |
| 关键词:罕见病 孤儿药 可及性 疾病负担 |
| 基金项目:国家卫生健康委员会委托课题“药品使用数据宏观分析指标研究”(09202004);湖北省罕见病用药医疗保障政策体系研究 |
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| Research on accessibility and procurement of orphan drugs in China |
| XU Lu-xin-yi1,2, WANG Ni3, YIN Shi-cheng1,2, TENG Cai-xia1,2, CUI Dan1,2 |
1. School of Public Health, Wuhan University, Wuhan Hubei 430071, China;
2. Wuhan University Global Health Institute, Wuhan Hubei 430072, China;
3. The Affiliated Hospital of Nanjing University Medical School, Nanjing Jiangsu 210008, China |
| Abstract:Objective: To provide a reference for improving drug security policies by analyzing the accessibility, procurement and application of orphan drugs in China. Methods: Literature analysis was used to evaluate the accessibility of drugs from two aspects of availability and affordability. Adoption rate, constituent ratio, etc. was used to analyze the current status of purchasing quantity and amount of drugs for rare diseases. Results: From the perspective of availability, domestic listed drugs only account for 58.9% of global listed drugs, and 16 rare diseases have no drugs to treat in China. 24.2% of domestic listed drugs have not registered for rare diseases, and all drugs of 20 rare diseases are off-label. From the perspective of affordability, only 43.4% of domestic listed drugs are truly covered by medical insurance, and 76.0% of drugs outside the medical insurance list are high-value drugs. DDDs of orphan drugs purchased by medical institutions increased with year-on-year growth of 571.56% in 2018 and 13.80% in 2019, and the procurement amount increased with year-on-year growth of 45.22% in 2018 and 25.40% in 2019. The coverage rate of some high-value orphan drugs was only 0.02%~3.06%. Conclusions: The availability of orphan drugs in China is not as good as that in foreign countries. The drug evaluation and approval system should be improved to promote the R&D and marketing of generic drugs. Catalog access and pharmaceutical centralized purchase have improved the affordability of orphan drugs, but the benefits of rare disease patients, especially those involved in high-value drugs, are still limited. A multi-level medical insurance policy for orphan drugs should be established, especially for high-value drugs. |
| Key words:Rare diseases Orphan drug Accessibility Disease burden |
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