新药早期临床研究的推进思路——从机制验证到概念验证  

Strategies for pushing forward the early phase clinical trials:from proof of mechanism into proof of concept

作　　者：魏敏吉[1] 赵德恒[2] 

机构地区：[1]北京大学第一医院临床药理研究所,北京100191 [2]国家食品药品监督管理总局药品审评中心,北京100038

出　　处：《中国新药杂志》2015年第11期1269-1274,共6页Chinese Journal of New Drugs

摘　　要：新药研究项目需要面对Ⅱ期临床研究阶段因有效性原因所致的高失败率。目前受到推崇的解决方案是在早期临床研究中采用机制验证和概念验证的策略。本文介绍了机制验证和概念验证的概念、试验设计要求、重要性等,并给出了研究示例加以说明。期望这种设计思路能够尽快整合到我国自主开发的新药早期临床研究中,为加快新药临床研究项目的推进以及为关键性决策提供依据。Projects of new drug development are required to face the higher attrition rates of drug candidates in the phase II clinical trials. Current appraised resolution for minimizing the attrition rates is to adopt the strategies of proof of mechanism （POM） and proof of concept （POC） during early phase clinical trials. In the present review, the concept of POM and POC was introduced, their design as well as their effects on new drug develop- ment were discussed; examples were given for demonstration purposes. It is our expectation that those strategies could be incorporated into the conduct of early phase clinical trials, and could be used for accelerating drug devel- opment as well as provide rationale basis for the go/no go decision-making.

关 键 词：机制验证 概念验证 早期临床 I期临床 IIa期临床 

分 类 号：R969[医药卫生—药理学]