Flexibility in the FDA approach to orphan drug development

Nature Reviews Drug Discovery, Published online: 1 September 2017; doi:10.1038/nrd.2017.151

Scientific advances, in combination with government incentives and commercial opportunity, have fuelled strong investment in orphan drugs, resulting in many innovative therapies. Here, we discuss the approach of the FDA to a range of issues that remain crucial to maintaining this momentum, such as the use of the totality of evidence in evaluating orphan drugs.

中文翻译：

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FDA孤儿药开发方法的灵活性

FDA孤儿药开发方法的灵活性

Nature Reviews Drug Discovery，在线发布：2017年9月1日；doi：10.1038 / nrd.2017.151

科学的进步，加上政府的鼓励措施和商业机会，推动了对孤儿药的大力投资，从而产生了许多创新疗法。在这里，我们讨论了FDA处理一系列问题的方法，这些问题对于保持这种势头仍然至关重要，例如在评估孤儿药物中使用全部证据。