有关促进国内罕见病药物临床试验的几点建议  

Suggestions for promoting domestic clinical trials of medicinal products for rare diseases

作　　者：白桦[1] 张抒扬[1] BAI Hua;ZHANG Shu-yang(Clinical Pharmacology Research Center,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences,Beijing 100032,China;Peking Union Medical College Hospital,Chinese Academy of Medical Sciences,Beijing 100730,China)

机构地区：[1]中国医学科学院北京协和医院临床药理研究中心

出　　处：《国际药学研究杂志》2019年第9期679-684,共6页Journal of International Pharmaceutical Research

基　　金：国家重点研发计划精准医学研究重点专项“罕见病临床队列研究”资助项目（2016YFC0901500）,国家重点研发计划精准医疗伦理、政策法规框架研究资助项目（2017YFC0910101）;中国医学科学院医学与健康科技创新工程资助项目（2016-I2M-1-002）

摘　　要：无论罕见病还是常见病,缩短药物研发上市的时间是各方的共同需求,这对于大多数尚无有效治疗方法的罕见病而言尤为重要。美国罕见病药物相关激励政策出台时间最早,因此罕见病药物临床试验数量最多,中国的临床试验数量正在逐渐增加。罕见病的药物临床试验以罕见肿瘤为主,目前多为Ⅱ期试验。临床试验作为孤儿药研发中的重要环节,除需要政策支持,在试验设计和实施中还存在很多问题,如对自然病程和发病机制缺乏了解、无法实施随机对照试验、有待更为新颖的试验设计和敏感的终点指标、入组困难、存在伦理冲突等。这些问题的解决有待政府部门、制药企业、医疗机构、患者组织等多方努力才能共同实现。Regardless of rare or common diseases,it is a common desire to shorten the time period of drug development.This is of particular importance for rare diseases because the most of rare diseases are lack of sufficient therapeutic methods.The incentive policy for the development of rare disease drugs(also called orphan drugs)was first adopted by the United States in the Orphan Drug Act in 1983,and correspondingly,most of clinical trials of the orphan drugs are seen in the United States.The clinical trials of orphan drugs are also increasingly on the rise in China.The main target diseases of the major portion of orphan drugs in clinical trials are mainly the rare cancers,and most of them are in the phaseⅡtrials.The clinical trial is a particular step in the orphan drug development.Besides the need of policy support,it still suffers from severe challenges in the clinical trial design and the clinical trial implementation,including the insufficiency in the understanding of natural history and mechanism of the diseases,the impossibility to conduct the randomized controlled clinical trial,the requirement for innovated design and more sensitive outcome measures to evaluate efficacy,the difficulty in enrolling patients to participate in the trials,and the ethical conflict.The solutions to these problems will require the joint efforts of all parties involving the government,pharmaceutical companies,medical institutions,and patient organizations.

关 键 词：罕见病 孤儿药 临床试验 伦理 

分 类 号：R954.4[医药卫生—药学] R969.4