罕见病药品贝林妥欧单抗临床研发和风险获益评估及启示

贝林妥欧单抗（通用名blinatumomab、中文商品名贝利妥、英文商品名Blincyto）于2014年获美国食品药品管理局（FDA）批准上市,是全球首个治疗急性淋巴细胞白血病（ALL）的双靶点抗体药物,持证商为美国安进公司（Amgen）。作为创新生物制品,其上市申请、审评和批准过程分别经历了FDA孤儿药（orphan drug）资格认定、突破性治疗药品（breakthrough therapy）资格认定、优先审评（priority review）资格认定、附条件批准（accelerated approval）等创新药的优先和特殊政策。通过文献研究法,收集、整理、分析了FDA首次批准贝林妥欧单抗的审评报告及相关文献,在全面了解该品种特点及审评审批过程的基础上,以有效性研究评价模式为切入点,尝试总结“以患者需求为核心,以临床价值为导向”的创新药物审评审批理念的具体实践经验,以期为我国新药研发和审评提供借鉴。

Clinical development and risk benefit assessment of orphan drug blinatumomab and its implications

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.