摘要
罕见病单病种发病率极低,病情复杂,诊断难度大,导致罕见病药物临床研发面临诸多困难。为鼓励罕见病药物研发,我国相继出台了一系列鼓励政策。除政策导向以外,加强对罕见病药物研发的科学引导也至关重要。罕见病药物的研发,除应遵循一般药物的研发规律外,更应密切结合罕见病自身特点,探求更科学和精巧的研究方法,助力罕见病药物研发。本文旨在基于我国罕见病药物研发现状及相关政策,阐述当前对于罕见病药物临床研发的思考。
Rare diseases are characterized by extremely low incidence,complex conditions and difficulty in diagnosis.These factors lead to many difficulties in clinical research and development of drugs for rare diseases.In order to encourage the research and development of drugs for rare diseases,China has introduced a series of encouraging policies.In addition to policy orientation,it is also crucial to strengthen scientific guidance to the research and development of drugs for rare diseases.Drug development for rare diseases,in addition to follow the general drug development rules,should more closely combine its own characteristics,explore more scientific and exquisite research methods.To help drug development for rare disease,this article is based on the rare disease drug development status and related policies,expounds the current consideration for the clinical research and drug development of rare disease.
作者
唐凌
张杰
赵伯媛
艾星
王朝云
色日格楞
李远红
杨志敏
TANG Ling;ZHANG Jie;ZHAO Bo-yuan;AI Xing;WANG Zhao-yun;SE Ri-ge-leng;LI Yuan-hong;YANG Zhi-min(Center for Drug Evaluation,National Medical Products Administration,Beijing 100022,China)
出处
《中国临床药理学杂志》
CAS
CSCD
北大核心
2021年第23期3295-3299,共5页
The Chinese Journal of Clinical Pharmacology
关键词
罕见病
药物研发
临床试验
rare disease
drug development
clinical trial
