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出自识林

2014-04-27 Lachman CONSULTANTS

你是否在考虑生产单抗，并希望你的产品与其它已经获得7年罕见病用药专营权（Orphan Drug Exclusivity，ODE）的产品相比，足够与众不同呢？在阅读过本周早些时候FDA发布的关于“根据罕见病用药法规解读单克隆抗体产品的相同性”最终指南，你或许会期望有新的思考。

单克隆抗体可能具有相似的功能和特点，但也可能有所不同。上面提到的这份指南针对具体案例给出了具体的建议，在罕见药法案和其具体法规的规定下，FDA将如何决定一个单克隆抗体是否与另外一个相同。该指南的目的是建立一套批准，来判断是否应该授予第二阶段ODE，还是（如果认为两个产品是相同的）决定第一阶段ODE阻止“类似”单克隆抗体的批准。

决定背后的科学考虑超出了我们这篇文章的考虑范围，但请回顾指南文件以了解FDA在作出决定时考虑的各种因素。这些信息无疑将会回答很多用于ODE目的的相同性问题。但由于FDA将根据每个产品自身的价值作出决定，我肯定这种情况下将会有一些小动作颇为活跃。如果从结构角度将产品视为相同，那么最终决定就是看新产品是否在临床上更胜一筹。

Lachman CONSULTANTS - Bob Pollock先生 2014-04-24
编译：识林-椒 2014-04-26

Orphan Drug Exclusivity and Monoclonal Antibodies
Written by Bob Pollock • April 24, 2014

Are you thinking of making a monoclonal antibody and hoping that your product is different enough than another already approved to gain 7 years of Orphan Drug Exclusivity (ODE)? Think again – or better yet just read FDA’s final guidance on the issue released earlier this week. The full guidance, "Interpreting Sameness of Monoclonal Antibody Products Under the Orphan Drug Regulations", may be found here.

Monoclonal antibodies may have similar functions and features, but could also appear somewhat different. The above-referenced guidance gives specific advice on how FDA will, on a case-by-case basis, determine whether one monoclonal antibody is the same as another, under the provisions of the Orphan Drug Act and its implementing regulations. The purpose of this is to establish whether a second period of ODE could be awarded, or (if the products are considered to be the same) to determine that the first period of ODE blocks approval of the “similar” monoclonal antibody.

The science behind the determination is beyond the scope of this blog, but please review the guidance document for an understanding of the various factors FDA will use in making its decision. The information will certainly answer many questions relative to sameness for ODE purposes, but since FDA will decide each case on its own merit, I am certain that the gamesmanship will be alive and well on this stage. The ultimate determination if the products are deemed the same from a structural standpoint will be if the new product is clinically superior.