摘要
自从《罕用药法案》（Orphan Drug Act．ODA）于1983年在美国通过以来，357种罕见病药物（俗称“孤儿药”）、2100多种相关产品获准上市。而在该法案通过之前，市面上只有10种这类药物。由于美国现有约7000种罕见病，受累人群高达2000万-3000万（中国的各类罕见病患者人数也有千万之多），
同被引文献30
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二级引证文献3
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1刘菲,周静,胡明.我国罕见病用药医疗保障政策及医保目录收载情况分析[J].中国卫生经济,2018,37(3):71-76. 被引量：9
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2李永红,毕华,史新昌,秦玺,饶春明.人用基因治疗制品生产和质量控制的通用性技术要求[J].中国新药杂志,2018,27(21):2482-2489. 被引量：4
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3张仪,王剑.基因组医学时代罕见病的诊治进展[J].中华内分泌代谢杂志,2019,35(9):813-817. 被引量：3
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3张旭.“孤儿药”不孤单——跨国药企争相发力罕见病药物开发[J].中国食品药品监管,2012(10):57-59. 被引量：3
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7无所不能的搜索引擎[J].投资与合作,2013(5):76-76.
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8杨露,陈大明,毛开云.罕见病治疗用药的发展态势分析[J].生物产业技术,2014(5):88-92.
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