我国风湿性疾病药物临床试验现状分析  

Analysis of drug clinical trials for rheumatic diseases in China

作　　者：王晓霞[1] 王洪[1] 何佳莉 刘丹[2] 吴珮涵 刘桂海 Wang Xiaoxia;Wang Hong;He Jiali;Liu Dan;Wu Peihan;Liu Guihai(Drug/Medical Device Clinical Trial Institution Office in the Second Hospital of Shanxi Medical University,Taiyuan 030001,China;Graduate Departments of Shanxi Medical University,Taiyuan 030001,China)

机构地区：[1]山西医科大学第二医院药物/医疗器械临床试验机构办公室,太原030001 [2]山西医科大学研究生院,太原030001

出　　处：《中华风湿病学杂志》2020年第4期240-246,共7页Chinese Journal of Rheumatology

摘　　要：目的:分析总结2013年1月至2019年11月,国内风湿性疾病药物临床试验登记情况。方法:检索国家食品药品监督管理局药品审评中心(CDE)官网"药物临床试验登记与信息公示平台",获得数据,进行分类统计。结果:①国内风湿性疾病领域药物临床试验数量逐年递增,共计525项。适应证主要包括弥漫性CTD、脊柱关节炎、代谢性疾病及退行性变四大类,以RA(189项)、痛风(122项)、OA(89项)、AS(60项)及SLE(39项)为主;②Ⅱ~Ⅲ期临床试验的药物主要是生物制剂DMARDs,生物等效性试验大多数为化学药品;③国内自主研发的化学创新药和生物制品创新药涵盖了BLyS/APRIL、CD22、JAK1、S1P1及BTK等作用靶点;④生物类似药以阿达木单抗和托珠单抗为参照药占比最多,适应证主要集中在RA;⑤国际多中心临床试验逐年增多,适应证以RA及SLE居多。结论:①我国风湿性疾病药物临床试验整体数量近年来呈现迅速增长趋势;②化学创新药和生物制品创新药研发是趋势;③国内生物类似药研发取得了重要成果,有望通过外推适应证惠及更多患者。Objective To analyze and summarize the drug registration clinical trial for rheumatic diseases in China from January 2013 to November 2019.Methods The website of CDE was searched to obtain the data and statistical analysis was conducted.Results①The number of drug clinical trials for rheumatic diseases in China increased year by year and the total was 525.The registered indications mainly included diffuse connective tissue disease,spondylo arthritis,metabolic diseases and degenerative diseases,including rheumatoid arthritis(RA)(189),gout(122),osteoarthritis(OA)(89),ankylosing spondylitis(AS)(60)and systemic lupus erythematosus(SLE)(39).②The phaseⅡ-Ⅲclinical trials were mainly for biological disease-modifying antirheumatic drugs(bDMARDs).Most of the bioequivalence test(BE)were chemical drugs.③The domestically developed chemicals and biological included BLyS/APRIL,CD22,JAK1,S1P1 and BTK.④Adalimumab and tocilizumab accounted for the largest proportion of biosimilar drugs.The indications were mainly RA.⑤The number of international multi-center clinical trials increased year by year,with the indications mainly for RA and SLE.Conclusion①The overall number of drugs trials for rheumatic diseases in China has been increasing rapidly in recent years.②The research and development of chemicals and biologics is the trend.③Significant achievements have been made in the research and development of biosimilars in China and it is expected to benefit more patients by broaden the indications.

关 键 词：药物 临床试用 临床试验 风湿性疾病 

分 类 号：R95[医药卫生—药学]