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。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。
Objective: To compare and analyze the orphan drug identification and approval standards in the United States(US) and the European Union(EU), so as to provide suggestions for improving the identification standards for rare diseases and orphan drugs in China. Methods: By searching the official websites of FDA, EMA and related literature, the identification standards for orphan drugs in the EU and the US were compared and analyzed. Results: Both the EU and the US include epidemiological criteria, expected cost-benefit criteria, precision medicine criteria, and clinical advantage over existing drugs of the same type in their orphan drug identification standards. However, the EU has two additional criteria compared to the US, namely, the additional disease criticality criteria and the significant benefit criteria. In practice, the attitude of the EU and the US towards the assessment of orphan subgroups under precision medicine is also different. In addition, the orphan drug identification systems in the EU and the US, in addition to their positive significance, also has negative impacts like causing disease over-categorization leading to orphan drug policy abuse, and market exclusivity leading to market monopoly of high-priced orphan drugs. Conclusion: It is suggested that China refer to the experience of the EU and the US in orphan drug identification to design its own identification standards for rare diseases and orphan drugs, which may be expanded from the following four criteria: epidemiology, expected cost-benefit, additional criticality and precision medicine.
作者
翁婷婷
谢金平
闫丽羽
杨伊凡
邵蓉
WENG Ting-ting;XIE Jin-ping;YAN Li-yu;YANG Yi-fan;SHAO Rong(The Research Center of National Drug Policy&Ecosystem,China Pharmaceutical University,Nanjing Jiangsu 211198,China)
出处
《中国卫生政策研究》
CSCD
北大核心
2023年第1期74-79,共6页
Chinese Journal of Health Policy
关键词
罕见病
孤儿药认定
孤儿药审批
Rare disease
Orphan drug identification
Orphan drug approval
