摘要
罕见病和孤儿药一直以来倍受关注,因罕见病发生率低和患者分布广的特点,使得疾病的诊断及其治疗药物的研发难度较大,世界各国先后提出多项决策计划政策激励孤儿药的研发。近年来真实世界研究被提出,作为一种新的研究方法,相比于传统的随机对照临床试验,更有助于罕见病的早期发现与诊断,以及孤儿药的临床研究与临床再评价。本文拟对国内外罕见病用药的真实世界数据研究的现状作一综述,并对其发展提出相关建议。
Rare diseases and orphan drugs have always attracted great attention.Due to the low incidence and wide distribution of patients,it is difficult to diagnose the diseases and develop the therapeutic drugs.Countries around the world have put forward a number of decision-making policies to stimulate the research and development of orphan drugs.In recent years,the real world study(RWS)has been proposed as a new research method.Compared with the traditional randomized controlled clinical trials(RCT),RWS is more conducive to the early detection and diagnosis of rare diseases and the clinical research and clinical reevaluation of orphan drugs.This paper reviews the current situation of real world data study on the use of drugs for rare diseases at home and abroad,and puts forward related suggestions for its development in China.
作者
陈烁冰
于锋
CHEN Shuo-bing;YU Feng(School of Basic Medicine and Clinical Pharmacy,China Pharmaceutical University,Nanjing 210098,China)
出处
《国际药学研究杂志》
CAS
北大核心
2019年第9期699-704,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）
关键词
真实世界研究
罕见病
孤儿药
real world study
rare disease
orphan drug
