血液肿瘤创新药物关键性注册研究设计要素的审评考虑

Considerations on pivotal clinical trial design of innovative new drugs for hematological malignancies

近年来,国内抗肿瘤创新药物的研发环境快速发生变化,给药物创新带来了各种挑战,如何在早期临床研究阶段探索出药物的临床优势,并制定出科学合理且高效的关键性注册研究方案即是挑战之一。文章以血液肿瘤创新药物为例,从临床审评人员的角度出发,对进入关键性注册研究的时机和方案要素的审评考虑进行阐述,为新药研发提供参考。

The rapid changes in the research and development environment of new anti-tumor drugs in China have brought various challenges to drug innovation.How to explore the clinical advantages of new drugs in the early phase,and design scientific,reasonable and efficient pivotal clinical trials for drug registration accordingly,is one of the key challenges.This article takes innovative new drugs for hematological malignancies as an example,comprehensively elaborates the considerations on the timing for entering the pivotal clinical trial and the key elements of the trial design from the perspective of clinical reviewers.