ABSTRACT
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 tradi-tional 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.