Abstract：
The U.S. Food and Drug Administration and European Commission have developed successful orphan drug legislation to promote the research, development, and marketing approval of drugs to treat rare diseases. Central to these regulations are the concepts of structural similarity and clinical superiority/significant benefit to achieve orphan drug exclusivity. However, differences in health authority expectations remain regarding the qualification for an orphan drug designation, defining structural similarity, and demonstrating clinical superiority/significant benefit. These differences can create sponsor company uncertainty regarding the approvability of products (e.g., blocking risk by an existing orphan product) and divergent orphan drug decisions among health authorities. A comprehensive assessment of current regulations, case studies in exclusivities, and recommendations for improvement are presented.
摘要：
美国食品和药物管理局和欧盟委员会已经制定了成功的孤儿药立法来促进这项研究，发展,以及治疗罕见疾病的药物的上市批准。这些法规的核心是结构相似性和临床优势/实现孤儿药排他性的显着益处的概念。然而,卫生当局对孤儿药资格的期望仍然存在差异，定义结构相似性，并显示出临床优势/显著获益。这些差异可能会给赞助商公司带来关于产品可批准性的不确定性(例如，阻止现有孤儿产品的风险)和卫生当局之间不同的孤儿药物决定。全面评估现行法规,排他性案例研究，并提出了改进建议。
