摘要
目的探讨评估我国药品生产企业(简称药企)化学创新药研发项目的基准指标。方法收集我国药企在2017年1月1日至2023年12月31日开展的化学新药(含创新药与改良型新药两类)研发项目数据,从药品类型、企业研发实力、所属疾病领域、与已有研究对比等维度分析创新药研发项目的阶段成功率与批准可能性。结果纳入我国995家药企的化学新药,共2008个品种,涉及4068个项目。2008个新药品种中有1595个创新药,413个改良型新药;4068个项目中,有3281个创新药,787个改良型新药。药品类型维度,创新药研发项目从临床试验Ⅰ期到批准上市的可能性为12.69%,低于改良型新药研发项目的31.01%,且两者阶段成功率趋势相反;企业研发实力维度,头部企业的创新药研发项目上述可能性为17.72%,高于非头部企业的11.84%,且头部企业在临床申请和临床试验Ⅲ期阶段的阶段成功率均高于非头部企业;适应证维度,肿瘤类药物的研发热度最高,但上述可能性最低仅有8.34%,各类药阶段成功率以临床试验Ⅰ期差异最大。与已有研究的对比发现,对于中美两国药企在全球研发的化学创新药,我国企业创新药研发项目上市可能性为12.7%,与美国的12.0%基本持平;但在临床试验Ⅱ期阶段的阶段成功率高于美国(47.2%比36.4%)。结论不同维度的阶段成功率与批准可能性能体现我国创新药物研发的总体特征,可作为衡量创新药研发成效的基准指标,辅助政策制定与企业决策。基于此,政府可对罕见病类的药物研发制订更多激励政策;企业也可识别临床需求,结合自身特点和优势制订研发方向与战略规划;从而进一步助力我国药企化学创新药研发进程。
Objective To explore and evaluate the benchmark indicators for the research and development(R&D)projects of chemical innovative drugs of pharmaceutical production enterprises(referred to as pharmaceutical enterprises for short)in China.Methods The data of chemical new drug R&D projects of Chinese pharmaceutical companies between January 1,2017 and December 31,2023 were collected(including two categories:innovative drugs and improved new drugs).The phase success rate and likelihood of approval of innovative drug R&D projects were analyzed from the dimensions of drug type,enterprise R&D strength and disease field and comparison with existing studies.Results A total of 2008 varieties of chemical new drugs from 995 pharmaceutical enterprises in China were included,involving 4068 items.Among the 2008 new drug varieties,there are 1595 innovative drugs and 413 improved new drugs.There are 3281 innovative drugs and 787 improved new drugs among the 4068 items.In terms of drug types,the probability of an innovative drug R&D project proceeding from Phase I of clinical trials to being approved for marketing is 12.69%,which is lower than the 31.01%of an improved new drug R&D project,and the trends of the phase success rates of the two are opposite.In terms of the R&D capabilities of enterprises,the above-mentioned probability for the innovative drug R&D projects of leading enterprises is 17.72%,which is higher than the 11.84%of non-leading enterprises,and the stage success rates of leading enterprises in the stages of clinical application and PhaseⅢof clinical trials are both higher than those of non-leading enterprises.In terms of indications,oncology drugs have the highest R&D popularity,but the above-mentioned probability is the lowest,only 8.34%,and the stage success rates of various drugs have the greatest difference in Phase I of clinical trials.By comparing with existing studies,it is found that for the chemical innovative drugs globally developed by pharmaceutical enterprises in China and the United States,the probability of the innovative drug R&D projects of Chinese enterprises being launched on the market is 12.7%,which is basically the same as that of the United States at 12.0%.However,the stage success rate in PhaseⅡof clinical trials of chinese pharmaceutial interprises is higher than that of the United States(47.2%vs.36.4%).Conclusion The stage success rates and approval probabilities from different dimensions can reflect the overall characteristics of innovative drug R&D in China,and can be used as benchmark indicators to measure the effectiveness of innovative drug R&D to assist in policy formulation and enterprise decision-making.Based on this,the government can formulate more incentive policies for the R&D of drugs for rare diseases;enterprises can also identify clinical needs,formulate R&D directions and strategic plans in combination with their own characteristics and advantages,so as to further boost the R&D process of chemical innovative drugs by Chinese pharmaceutical enterprises.
作者
赵永
王静怡
蒋欣怡
钟华羽
陈欣悦
王涓霁
刘笑笑
张永红
李天泉
ZHAO Yong;WANG Jingyi;JIANG Xinyi;ZHONG Huayu;CHEN Xinyue;WANG Juanji;LIU Xiaoxiao;ZHANG Yonghong;LI Tianquan(School of Pharmacy,Chongqing Medical University,Chongqing,China 400016;Chongqing Kangzhou Big Data<Group>Co.,Ltd.,Chongqing,China 401336)
出处
《中国药业》
2025年第6期1-6,共6页
China Pharmaceuticals
基金
重庆市研究生联合培养基地项目[渝教研发〔2019〕12号]
重庆市研究生科研创新项目[CYS23357]。
关键词
阶段成功率
批准可能性
创新药
研发项目
药品生产企业
phase success rate
probability of approval
innovative drug
R&D project
pharmaceutical production enterprise
