| 王敏,范平安,王志远,钱兵,聂小燕,管晓东,史录文,陈敬.美国孤儿药资格认定及批准上市情况分析[J].中国药事,2021,35(12):1406-1413 |
| 美国孤儿药资格认定及批准上市情况分析 |
| Analysis of Orphan Drug Designation and Marketing Approval in the United States |
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| DOI：10.16153/j.1002-7777.2021.12.012 |
| 中文关键词: 美国食品药品监督管理局 孤儿药 审批 资格认定 |
| 英文关键词: FDA orphan drugs approval designation |
| 基金项目:国家自然科学基金面上项目(编号 71874006) |
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| 中文摘要: |
| 目的： 系统了解美国孤儿药批准情况，分析资格认定和审批趋势。方法：以美国食品药品监督管理局(FDA)数据库Drugs@ FDA和孤儿药资格认定和批准在线数据库为主要数据来源，系统收集美国FDA自1983至2020年认定和批准的孤儿药信息，从审批数量、审批时间、治疗领域等方面进行统计分析。结果：1983-2020年FDA共认定孤儿药资格5757个，批准孤儿药适应症943个。从孤儿药认定身份到获批上市平均所需的时间为5.14年。批准的孤儿药适应症中抗肿瘤和免疫机能调节相关适应症最多，为 408个，占比为43.27%。结论：自1983年《孤儿药法案》实施以来，FDA授予的孤儿药资格数量和批准的孤儿药适应症数量呈现明显的增长趋势。 |
| 英文摘要: |
| Objective: To systematically understand the approval status of orphan drugs, and analyze the trend of designations and approvals in the United States. Methods: Taking the Drugs@ FDA database and FDA Orphan Drug Designations and Approvals online databases as the main data sources, and the information of orphan drugs designated and approved by FDA from 1983 to 2020 was systematically collected. The statistical analysis was performed in terms of the number of approved orphan drugs, approval time and therapeutic areas. Results: From 1983 to 2020, FDA designated 5757 orphan drugs and approved 943 orphan drug indications. The average time required from orphan drug designation to market approval was 5.14 years. Among the approved indications of orphan drugs, FDA approved 408 indications for antineoplastic and immunomodulatory therapy, accounting for 43.27%. Conclusion: Since the implementation of the Orphan Drug Act, both the number of designated orphan drugs and the number of approved orphan drug indications have shown a significant growth trend. |
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