Thoughts on the characteristics of priority review and approval of rare disease drugs in China
CHEN Qian-nan
LIU Xiao-xi
LIAN Gui-yu
HUANG Zhe
Abstract：Objective:To analyze the characteristics of rare disease drugs approved through the priority review and approval process in China from 2018 to 2024,and to provide a reference for optimizing review policies for rare disease drugs and improving drug access.Methods:Based on data from the National Medical Products Administration and Yaozh Data(Drug Intelligence Data)and other sources,a descriptive statistical analysis was conducted on the main features of rare disease drugs approved through priority review and approval.Results:A total of 86 rare disease drugs were included in this study from 2018 to 2024,with an average review and approval duration of 463.55 days.Among them,chemical drugs accounted for 58%,imported drugs accounted for 77.9%,and domestic drugs only accounted for 22.1%.A total of 44 drugs were included in the national medical insurance catalog,and only 4 were pediatric-specific.Conclusion:China has established a multi-pathway accelerated approval system for rare disease drugs,significantly improving the accessibility of rare disease drugs.However,there are still issues such as insufficient supply of domestic innovative drugs,uneven coverage of therapeutic areas,and a shortage of pediatric drugs.
Keywords：rare disease drugspriority review and approvalcharacteristics of drug marketingorphan drugs
Publication Date：2026-07-15
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：6( 1352-1357 )