Comparative Analysis of Orphan Drugs Marketed in the United States and China from 2018 to 2022
10.3870/j.issn.1004-0781.2024.06.007
- VernacularTitle:2018-2022年中国和美国上市孤儿药的对比分析
- Author:
Weixin JIANG
1
;
Shiwei GONG
Author Information
1. 华中科技大学同济医学院药学院,武汉 430030
- Keywords:
Orphan drug;
Rare disease;
Drug quantity;
Drug type;
Innovative drug
- From:
Herald of Medicine
2024;43(6):885-890
- CountryChina
- Language:Chinese
-
Abstract:
Objective To compare the approval status of orphan drugs in China and the United States(the U.S.)from 2018 to 2022,and to further understand the trends and reasons for the differences in the development of orphan drugs between the two countries.Methods Data on orphan drug approvals in China and the U.S.were retrieved from the National Medical Products Administration of China and the U.S.Food and Drug Administration(FDA),and the number,types,Marketing Authorisation Holder(MAH),target diseases,and new drugs marketed in China and the U.S.were classified and compared.Results From 2018 to 2022,426 approvals of orphan drugs in the U.S.and 63 approvals in China.Average annual growth rates of drug approval numbers in the U.S.and China were-4.81％and 8.38％,respectively.The most common types of orphan drugs approved in both countries were antineoplastic drugs and immunomodulators,of which 235(55.16％)were in the U.S.versus 15 types(23.81％)in China.Among the types of rare diseases targeted by these orphan drugs,the U.S.had the most rare tumor diseases(197 types,46.24％),while China had the most rare neurologrical and psychiatric diseases(22 types,34.92％).The U.S.marketed 36 first-in-class orphan drugs with breakthrough therapeutic value.And China marketed 40 innovative new drugs for the treatment of rare diseases.Conclusion The number of orphan drugs marketed in the U.S.has far surpassed that in China,but has shown a slight downward trend,while an increasing trend in China.The concentration of the domestic orphan drug industry in the U.S.is high,while China's orphan drugs still rely on imports.China has made substantial progress in making domestic innovative drugs for rare diseases,but there is a huge gap between China and the U.S.,especially in terms of advanced technology and innovative drugs.Therefore,intensify efforts should be made in the research and development(R & D)of new drugs for rare diseases in China,and actively participate in global synchronized research and development to achieve win-win cooperation.
