从美国1985—2019年新药批准情况看新药研发和审批趋势

Trends of new drug R&D and approval by FDA:an analysis of the data of application of new molecular entities from 1985 to 2019

目的:全面系统了解美国新药批准情况,分析新药研发和审批趋势。方法:以美国FDA的Drugs@FDA数据库为主要数据来源,系统收集美国FDA药品审评研究中心1985—2019年批准的新分子实体信息,对已上市的化学新药和生物新药从创新程度、加快审批途径、治疗领域等方面进行了统计分析。结果:1985—2019年FDA共批准新分子实体药物1096个,其中化学新药930个,生物新药166个。与化学新药相比,生物新药中孤儿药、首创性新药与优先审评新药的比例更高,每年批准数量呈增长趋势。研究期间FDA新药平均审评时间为594 d,随年份的变化呈下降趋势,且生物药、孤儿药、首创性新药和优先审评新药的审评时间显著更短(P<0.01)。不同治疗领域的药物中,抗肿瘤药的平均审评时间最短,仅为268 d。从1985—2019年,新药研发重心逐渐从治疗心血管系统、消化系统和感染性疾病的药物向抗肿瘤药转移。结论:新药审批速度加快,生物药、孤儿药、抗肿瘤药成为新的研发重点。

Objective:To comprehensively and systematically describe the new molecular entity approvals in the United States,and analyze the trends of new drug development and approval.Methods:Statistical analyses of innovation,expedited programs,and indications were conducted based on information of new molecular entities approved between 1985—2019 in the U.S.Food and Drug Administration(FDA)in the Drugs@FDA database.Results:There were totally 1096 new molecular entities approved by FDA between 1985—2019,including 930 chemical and 166 biological products.The proportions of orphan drugs,first-in-class drugs,and drugs approved through priority review in biologics were higher than those in chemical drugs,and the number of biologics approved each year was increasing.The mean drug review time was 594 days,decreasing with year.The review time of biologics,orphan drugs,first-in-class drugs,and drugs approved through priority review was significantly shorter compared to other new molecular entities(P<0.01).The mean review time of antitumor drugs was only 268 days,the shortest among all new drugs within the study period.From 1985 to 2019,the focus of new drug development gradually shifted from the fields of cardiovascular system,digestive system,and infectious diseases to antitumor drugs.Conclusion:The new drug review time has been shortened,and biologics,orphan drugs,and antitumor drugs has become new focus of drug development.