通讯作者: 邵蓉, shaorong118@163.com

DOI：10.12201/bmr.202302.00010

Comparison and enlightenment of orphan drug identification standards between America and Europe

• 摘要：目的 比较分析美国和欧盟的孤儿药认定审批标准，为完善我国罕见病及孤儿药认定标准提供建议。方法 通过检索FDA官网、EMA官网及相关文献比较分析欧美美国和欧盟孤儿药的认定标准。结果：美国和欧盟对于孤儿药认定均存在流行病学标准、预期成本-收益标准、精准医学标准以及同种药物的临床优势要求。但是欧盟在孤儿药资格认定标准的方面相较于美国还有疾病附加危重程度标准和显著收益这两条标准。而美国和欧盟在实操层面上对精准医学下孤儿亚组的评估态度也较不同。此外，美国和欧盟孤儿药认定制度除其积极意义之外还存在疾病细分致孤儿药政策滥用、市场独占致高价孤儿药垄断市场等负面影响。结论 建议我国罕见病及孤儿药认定标准可借鉴美国和欧盟的孤儿药认定经验，从流行病学、预期成本-收益、附加危重程度及精准医学这四个标准展开。

  关键词： 罕见病，孤儿药认定，孤儿药审批; ; 

   

  Abstract: Objective: Comparative analysis of orphan drug designation and approval standards in Europe and the US, to provide suggestions for improving the designation standards for rare diseases and orphan drugs in China. Methods:. By searching the official websites of FDA, EMA and related literature to compare and analyze the designation standards for orphan drugs in Europe and the US. Results: Epidemiological criteria, expected cost-benefit criteria, precision medicine criteria, and clinical advantages for the same drug exist in both Europe and the United States for orphan drug designation standards. However, the EU has two additional criteria for orphan drug designation compared to the US, namely the additional criticality of the disease and the significant benefit. The attitude towards the assessment of orphan subgroups under precision medicine at the practical level is also more different in Europe and the US. In addition, the orphan drug designation system in Europe and the US, in addition to its positive significance, also has the negative impact of disease segmentation leading to orphan drug policy abuse, market exclusivity leading to high-priced orphan drugs to monopolize the market. Conclusion: It is suggested that the designation standards for rare diseases and orphan drugs in China can be borrowed from the experience of orphan drug identification in Europe and the US, starting from the four criteria of epidemiology, expected cost-benefit, additional criticality and precision medicine.

  Key words: rare disease; orphan drug designations; orphan drug approvals

  提交时间：2023-02-28

  版权声明：作者本人独立拥有该论文的版权，预印本系统仅拥有论文的永久保存权利。任何人未经允许不得重复使用。
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• 序号	提交日期	编号	操作
  1	2022-08-11	bmr.202302.00010V1	下载

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