貝林妥歐單抗（通用名blinatumomab、中文商品名貝利妥、英文商品名Blincyto）于2014年獲美國食品藥品管理局（FDA）批準上市，是全球首個治療急性淋巴細胞白血病（ALL）的雙靶點抗體藥物，持證商為美國安進公司（Amgen）。作為創(chuàng)新生物制品，其上市申請、審評和批準過程分別經(jīng)歷了FDA孤兒藥（orphan drug）資格認定、突破性治療藥品（breakthrough therapy）資格認定、優(yōu)先審評（priority review）資格認定、附條件批準（accelerated approval）等創(chuàng)新藥的優(yōu)先和特殊政策。通過文獻研究法，收集、整理、分析了FDA首次批準貝林妥歐單抗的審評報告及相關文獻，在全面了解該品種特點及審評審批過程的基礎上，以有效性研究評價模式為切入點，嘗試總結“以患者需求為核心，以臨床價值為導向”的創(chuàng)新藥物審評審批理念的具體實踐經(jīng)驗，以期為我國新藥研發(fā)和審評提供借鑒。

Blinatumomab (proprietary name is Blincyto) was approved by the US Food and Drug Administration (FDA) in 2014. It is the first bispecific antibody drug for the treatment of acute lymphoblastic leukemia (ALL), licensed by Amgen. As an innovative biological product, the marketing application, review and approval process of blinatumomab went through expedited procedures such as Orphan Drug designation, Breakthrough Therapy designation, Priority Review designation and Accelerated Approval. This study collected, sorted out and analyzed the review report and related literature of the initial approval of blinatumomab by FDA through literature research method. Based on a comprehensive understanding of the characteristics of this drug and its review and approval process, focusing on its efficacy evaluation model, the author tries to conclude the specific practical experience of the review and approval concept for new drugs, that is "needs of patients as the core and clinical value of drug as the orientation", which is expected to provide reference for the development and evaluation of new drugs in China.

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