International status of orphan drug registration review system and the enlightenment to China

YANG Run-feng; WANG Qian; TIAN Kan

doi:10.20251/j.cnki.1003-3734.2025.09.003

Abstract：The registration and review of orphan drugs,as an important part of new drug marketing,affect its accessibility to a certain extent.Currently,the registration and review systems for orphan drugs have been relatively well-established in some developed countries.For instance,the United States,the European Union and Japan have all introduced relevant legislation for rare diseases and orphan drugs.They established specialized regulatory agencies,orphan drug qualification recognition mechanisms,special review and approval channels,and introduced review fee waiver policies to accelerate the marketing process of orphan drugs.The registration and review system for orphan drugs in China was established relatively late,which still faces many challenges in the implementation.Through a literature review,this paper conducts a comparative analysis of the registration and review systems for orphan drugs in different countries.In combination of China's specific national conditions,it suggests that relevant departments can improve the registration and review system for orphan drugs from five aspects:clarifying the definition of rare diseases and the recognition criteria for orphan drugs,establishing orphan drug qualification recognition mechanisms,setting up specialized orphan drug review departments,refining the review and approval system for orphan drugs,and improving the registration system for rare diseases,which will further enhance the accessibility of orphan drugs.