Division of Clinical Pharmacology-3, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
Clin Pharmacol Ther. 2016 Oct;100(4):327-9. doi: 10.1002/cpt.427. Epub 2016 Aug 26.
Orphan drugs or drugs for rare diseases represents a particular regulatory conundrum. There is a desperate need for effective therapies for these patients, who have been historically underserved by the drug development community. However, there is also a need to make sure these therapies are both safe and effective. In response, the US Food and Drug Administration (FDA) has evolved new approaches to facilitate drug development in this area.
孤儿药或罕见病药物代表了一个特殊的监管难题。这些患者非常需要有效的治疗方法，但他们在历史上一直得不到药物开发界的关注。然而，也有必要确保这些治疗方法既安全又有效。为此，美国食品和药物管理局（FDA）已经发展出了新的方法来促进这一领域的药物开发。
