药物再定位的发展历程与进展  

The Development and Research Progress of Drug Repurposing

作　　者：王涛[1] 尚靖[1] WANG Tao;SHANG Jing(China Pharmaceutical University,Jiangsu Nanjing 211198,China)

机构地区：[1]中国药科大学,江苏南京211198

出　　处：《中国医药导刊》2022年第4期325-333,共9页Chinese Journal of Medicinal Guide

摘　　要：“安全、有效、质量可控”是新药研发的基本原则。新药研发投入大、风险高、周期长。近年来,世界范围内药物研发面临疾病系统性及如何控制临床风险等理论和技术上新的瓶颈,新药的投入产出比呈明显下降趋势。药物再定位是针对已获批上市或未上市但成分明确、生物活性已知的药物,探寻其潜在的作用机制,扩大其适用范围的过程。与基于新物质新作用机制的原始创新药物研发相比,药物再定位在资金投入、研发周期和风险等方面具有明显优势。本研究结合具体实例,综述药物再定位领域国内外进展情况、相关主要的策略和技术方法,进而对于药物再定位策略的潜在优势领域和我国药物再定位领域的前景及挑战提出思考。"Safety,effectiveness and controllable quality"are the basic principles of new drug research and development,and the research and development of new drug need large ivestment,and have high risk and long cycle.In recent years,drug research and development around the world began to face new theoretical and technical bottlenecks,such as disease systematicness and how to control clinical risks.The input-output ratio of new drugs showed a significant downward trend.Drug repurposing is a process to explore the potential mechanism of action and expand the scope of application of drugs that have been approved for marketing or drugs that have clear ingredients and known biological activities although not yet on the market.Compared with the research and development of original new drug,drug repurposing has obvious advantages in capital investment,development cycle and risk.Combined with specific examples,this paper summarizes the progress,relevant main strategies and technical methods in the field of drug repurposing at home and abroad,and then puts forward some thoughts on the potential advantages of drug repurposing strategy and the prospects and challenges in the field of drug repurposing in China.

关 键 词：药物再定位 药效学 新适应证 新药研发 

分 类 号：R95[医药卫生—药学] R96