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基于《第一批罕见病目录》的我国罕见病药物上市情况分析
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| 篇名： | 基于《第一批罕见病目录》的我国罕见病药物上市情况分析 |
| TITLE： | Analysis of the marketing of rare disease drugs in China based on the First Batch of Rare Disease Catalog |
| 摘要： | 目的 考察我国罕见病药物的上市现状、一般特征以及时间发展趋势。方法基于《第一批罕见病目录》收纳的121种罕见病，从国家药品监督管理局药品审评中心和药智网数据库中获取相应适应证药物的名称和批准上市信息，提取相关特征变量进行描述性统计分析。结果截至2022年12月31日，该目录收纳的121种罕见病中，有治疗药物上市的仅有32种，共涉及79个罕见病药物。其中，46.84%的药物是国产药，有88.61%的药物被批准成人和儿童都可用；化药占比为67.09%，注射剂占比为59.49%；按解剖学、治疗学及化学分类系统（ATC）分类，A类（消化系统用药）是最多的，占比为20.25%。每年上市的罕见病药物数量在2021年最多，2018－2021年总体呈上升趋势，2022年呈下降趋势。每年上市的罕见病药物中，按ATC分类，L类（抗肿瘤药及免疫用药）在2021年上市的数量最多，为5个；按剂型分类，口服制剂在2022年上市的数量最多，注射剂在2021年上市的数量最多。结论近年来我国罕见病药物上市数量不断增长，但是还远不能满足患者需求，并且上市的国产罕见病药物还非常缺乏。应进一步加快罕见病药物的研发，促进罕见病药物的进口与仿制。 |
| ABSTRACT： | OBJECTIVE To investigate the marketing status， general characteristics， and time trends of rare disease drugs in China. METHODS Based on 121 kinds of rare diseases included in the First Batch of Rare Disease Catalog， the names and marketing approval information of corresponding drugs with indications were obtained from the databases of the Center for Drug Evaluation， National Medical Products Administration and Yaozhi.com， and the relevant characteristic variables were extracted for descriptive statistical analysis. RESULTS As of December 31， 2022， only 32 of 121 rare diseases have therapeutic drugs available for treatment on the market in China， and 79 rare disease drugs have been approved. Among them， 46.84% of the drugs are domestic drugs， 88.61% of the drugs are approved for use in both adults and children； 67.09% are chemicals and 59.49% are injections. According to the ATC classification， Category A （digestive system drugs） is the most， accounting for 20.25%. The number of rare disease drugs on the market each year is the highest in 2021， with an overall upward trend from 2018 to 2021 and a downward trend in 2022. Among rare disease drugs on the market each year， according to the ATC classification， the number of Category L （antineoplastics and immune inhibitors） will be the largest in 2021， being 5. By dosage form， oral medicines were marketed in the largest number in 2022， and injectable medicines in 2021. CONCLUSIONS In recent years， the number of approved rare disease drugs in China has been continuously increasing， but it is still far from meeting the needs of patients， and there is still a lack of domestically approved rare disease drugs. We should further accelerate the research and development of rare disease drugs， and promote the import and replication of rare disease drugs. |
| 期刊： | 2024年第35卷第11期 |
| 作者： | 彭菲菲；蒋君好；鲍宇健；郑航 |
| AUTHORS： | PENG Feifei，JIANG Junhao，BAO Yujian，ZHENG Hang |
| 关键字： | 罕见病；罕见病药物；第一批罕见病目录；上市情况；发展趋势 |
| KEYWORDS： | rare disease； rare disease drugs； the first batch of rare disease catalog； marketing； trend |
| 阅读数： | 4 次 |
| 本月下载数： | 0 次 |
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