美国505(b)(2)路径对我国改良型新药设计与开发的启示

Inspiration of the US 505(b)(2)on the Design and Development of Improved New Drugs in China

改良型新药在美国按505(b)(2)路径申报和审评,相关药物的销售市场规模约为仿制药的1.7倍,在满足临床需求的前提下,505(b)(2)药物因竞争压力较小而具有较大的经济效益。近年来,我国仿制药市场增长较为缓慢,越来越多的仿制药企业希望通过布局改良型新药获得新的增长点。同时,随着创新药获批数量的快速增长,越来越多的创新药企业开始利用改良型新药的路径增加产品的适用人群或延长生命周期。然而,改良型新药是一个具有挑战性的全新领域,不仅审批标准较高,而且产品设计的理念和经验尚不成熟,导致相关项目开发失败率较高或市场收益不理想。美国505(b)(2)路径已发展近40年,覆盖2500多个批准文号,且很多产品的市场开拓取得成功,因此美国505(b)(2)路径的设计与开发、药物临床评价及市场开拓对我国具有一定的借鉴意义。本文系统对比了我国改良型新药与美国505(b)(2)路径药品申请的异同,梳理了美国505(b)(2)路径审评审批情况,分析了成功通过505(b)(2)路径上市的产品所具备的共性及其设计思路与亮点,以期为我国改良型新药的业务布局、项目设计和产品开发提供一定的参考。

Improved new drugs are applied and evaluated according to the US 505(b)(2)pathway,and the market of 505(b)(2)products is about 1.7 times that of generic drugs in the USA.While meeting clinical needs,the 505(b)(2)products present significant economic benefits due to less competition.In recent years,due to the slow growth of the Chinese generic drug market,more and more generic pharmaceutical companies are looking to improved new drugs for new growth points.In addition,with the rapid growth of innovative drugs,many innovative pharmaceutical companies are starting to apply this pathway to increase the potential patient population or extend the product lifecycle.However,developing improved new drug is a challenging field for Chinese pharmaceutical companies,with high approval standards and immature product design concepts and experience,resulting in a high failure rate of development or unsatisfactory market returns.In contrast,the US 505(b)(2)has a forty-year history,over 2500 approvals,and many products have obtained successful market performance.Therefore,the design and development,clinical evaluation,and market expansion of the US 505(b)(2)pathway are valuable for reference.This article systematically compares the similarities and differences between China’s improved new drugs and the US 505(b)(2)pathway,reviews 505(b)(2)approvals,including their market performance,design ideas,and highlights,to provide references for the industrial planning,project design,and product development of improved new drugs in China.