基于美国临床试验数据库的罕见病临床试验现状研究及启示

Research Status and Enlightenment of Clinical Trials of Rare Diseases Based on Clinical Trials.gov

目的:探讨国际罕见病临床试验的现状及特点,为促进我国罕见病临床试验管理提供参考和建议。方法:以美国临床试验数据库(Clinical Trails.gov)为基础,全面检索在该平台注册的罕见病临床试验,从注册数量、地域分布、研究类型和进展、设计特点、受试者、研究目的和领域等多个角度,对罕见病临床试验现状和特点展开分析。结果:Clinical Trails.gov中累计注册481项罕见病临床试验;绝大多数试验由发达国家和地区开展;研究类型主要为干预型;无分期、Ⅱ期和Ⅲ期临床试验相对较多;受试者规模多<500例;研究目的以治疗为主,涉及523种罕见病,主要集中在代谢疾病、神经系统疾病等领域。结论:需依据罕见病临床试验特点,探索更多适宜的临床研究新方法和思路;在现阶段,有必要加强罕见病自然史研究;从长远看,需从完善制度、加强人才培养、提高创新能力和积极性等方面加强我国罕见病临床研究。

OBJECTIVE:To probe into the current status and characteristics of clinical trials of rare diseases,so as to provide references and suggestions for promoting the management of clinical trials of rare diseases in China.METHODS:Based on clinical trials.gov,clinical trials of rare diseases were retrieved comprehensively.The status and characteristics of clinical trials of rare diseases were analyzed from the perspectives of the number of registrations,geographical distribution,research types and progress,design characteristics,subjects,research objectives and fields.RESULTS:There were 481 registered clinical trials of rare disease in clinical trails.gov,most of which were hold by developed countries and regions,mainly for interventional research.The main stages were trials without phases,phaseⅡand phaseⅢ.Most of enrollment were under 500 cases.The objective of the research was treatment-oriented,including 523 kinds of rare diseases,mainly for metabolic diseases and neurological diseases.CONCLUSIONS:According to the characteristics of clinical trials of rare diseases,more suitable new methods and ideas for clinical research should be explored.At this stage,it is necessary to strengthen the research on the natural history of rare diseases;in the future,it is necessary to strengthen the clinical research on rare diseases in China from the aspects of perfecting the system,strengthening the training of talents,improving the innovation ability and enthusiasm.