Ethical considerations in orphan drug approval and use.
Ethical considerations in orphan drug approval and use.
复制标题
孤儿药审批和使用中的伦理考虑。
DOI：
10.1038/clpt.2012.92
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发表时间：
2012
影响因子：
6.7
通讯作者：
Kesselheim,AS
中科院分区：
文献类型：
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作者：
Kesselheim,AS
《孤儿药法案》旨在实现为罕见疾病患者提供治疗选择的功利目标。然而，数据显示，与非孤儿药相比，孤儿药通常在上市前测试中进行了更有限的批准，因此使患者面临更大的风险和更不确定的疗效。因此，正义和仁慈的伦理原则可能需要关注接受药物的患者的知情同意，并加大对上市后监测和确认测试的投资。临床药理学与治疗(2012);922, 153–155。 doi:10.1038/clpt.2012.92
The Orphan Drug Act seeks to meet a utilitarian goal of advancing therapeutic options for patients with rare diseases. However, data show that orphan drugs are often approved with more limited premarket testing than that carried out for nonorphan drugs and consequently expose patients to more risk and less certain efficacy. Therefore, the ethical principles of justice and beneficence may require attention to informed consent among patients receiving the drugs and greater investment in postmarket surveillance and confirmational testing.Clinical Pharmacology & Therapeutics(2012);922, 153–155. doi:10.1038/clpt.2012.92