Isakov Leah, Jin Bo, Jacobs Ira Allen
1Biotechnology Clinical Development Statistics, Pfizer Inc, Cambridge, MA; and 2Global Established Pharma Medicines Development Group, Pfizer Inc, New York, NY.
Am J Ther. 2016 Nov/Dec;23(6):e1903-e1910. doi: 10.1097/MJT.0000000000000391.
A biosimilar is highly similar to a licensed biological product and has no clinically meaningful differences between the biological product and the reference (originator) product in terms of safety, purity, and potency and is approved under specific regulatory approval processes. Because both the originator and the potential biosimilar are large and structurally complex proteins, biosimilars are not generic equivalents of the originator. Thus, the regulatory approach for a small-molecule generic is not appropriate for a potential biosimilar. As a result, different study designs and statistical approaches are used in the assessment of a potential biosimilar. This review covers concepts and terminology used in statistical analyses in the clinical development of biosimilars so that clinicians can understand how similarity is evaluated. This should allow the clinician to understand the statistical considerations in biosimilar clinical trials and make informed prescribing decisions when an approved biosimilar is available.
生物类似药与已获许可的生物制品高度相似，在安全性、纯度和效力方面，生物制品与参比（原研）产品之间不存在具有临床意义的差异，并且是根据特定的监管审批程序获得批准的。由于原研产品和潜在的生物类似药都是大型且结构复杂的蛋白质，因此生物类似药并非原研产品的仿制药等效物。因此，小分子仿制药的监管方法不适用于潜在的生物类似药。结果，在评估潜在生物类似药时会使用不同的研究设计和统计方法。本综述涵盖了生物类似药临床开发中统计分析所使用的概念和术语，以便临床医生能够理解相似性是如何评估的。这应能使临床医生了解生物类似药临床试验中的统计考量因素，并在有获批的生物类似药可用时做出明智的处方决策。
