加速药物研发新方式——微剂量给药研究  

New paradigm of accelerating new drug development——microdosing

作　　者：黄河花[1] 江骥[1] 胡蓓[1] 

机构地区：[1]中国医学科学院北京协和医院临床药理中心,北京100730

出　　处：《中国新药杂志》2009年第1期12-16,共5页Chinese Journal of New Drugs

摘　　要：药物研发是一项长时间、难度大、高投入的工程。近年来,新药研发成本逐年增加,经FDA批准上市的新药却呈下降趋势。为加速药物研发,欧洲EMEA在2003年7月[1],美国FDA在2006年1月[2]分别颁布相关指南,提出一种新的药物研发方式:人体微剂量给药研究或称临床试验0期。微剂量研究是首次在人体身上进行的临床药物研究,仅需要少数的受试者,在短时间内给予微剂量新化学实体的研究,其目的不在于治疗或诊断,而是为了及早获得新化学实体的人体药动学信息,协助筛选最优候选化学物。由于是极低剂量给药,极大降低了药物的风险,因此其对临床前研究资料的要求大为减少。合理选择新化学实体进行微剂量研究,可以使患者更早得到安全有效的新药治疗,减少临床试验的损耗,降低研发成本及提高研发效率。Drug development is a long, complex and expensive process. Recently, the expenditure of new drug discovery is increasing yearly. However, the approval number of new drugs by FDA is decreasing, and the actual new drug entering the market is declining. To improve the drug development, European EMEA （July 2003） and US FDA （January 2006） published guidelines on exploratory investigational new drugs, and issued a new drug study method ： microdosing, which is also called Phase 0 clinical trials. Microdosing is a kind of firstly used in human study. Microdosing should involve a very limited number of normal volunteers, exposure to a novel compound at a microdose for a short time period without therapeutic or diagnostic intent; it could allow researchers to quickly establish whether a novel compound has appropriate pharmacokinetic profiles in humans. Owing to the low doses administered and the low risk of toxicity, shorter preclinical packages to support these studies are required. In ap- propriately chosen subjects, microdosing could allow for patients＇ quicker access to safer and more efficacious doses of novel drugs, reduce attrition in clinical trials and facilitate more economical drug development.

关 键 词：微剂量给药研究 临床试验0期 候选化合物筛选 药物研发 药动学 

分 类 号：R969.4[医药卫生—药理学]