Overview and Analysis of Rare Disease Drugs Approved for Market in China,the United States,the European Union,and Japan in 2023
10.12376/j.issn.2097-0501.2024.02.008
- VernacularTitle:2023年度中国、美国、欧盟、日本批准上市罕见病用药概览与分析
- Author:
Shaohong WANG
1
;
Xin LIU
;
Qingyang LIU
;
Yan TANG
;
Bo ZHANG
Author Information
1. 中国医学科学院北京协和医院药剂科,北京 100730
- Keywords:
rare diseases;
orphan drugs;
marketed;
approved drug
- From:
JOURNAL OF RARE DISEASES
2024;3(2):202-213
- CountryChina
- Language:Chinese
-
Abstract:
Objective To summarize and analyze what the listing in market of orphan drugs in China,the United States,the European Union,and Japan in 2023,and to provide empirical reference to Chinese phar-maceutical enterprises,pharmaceutical management,and new drug research and development departments.Methods Collect the 2023 drug market data released by official drug regulatory agencies in four countries and international organization-China,the United States,the European Union,and Japan categorize the market of rare disease drugs and and their indications,approval dates,R&D status in China,component therapeutic are-as,and special review and approval pathways were analyzed.Results Findings in 2023,28 orphan drugs were listed in the United States,mostly anti-tumor related,accounting for 32.1%(9 out of 28);17 orphan drugs were listed in the European Union,anti-tumor related accounting for 47.0%(8 of 17);22 listed in Japan;and 45 listed in China.In 2023,over 70%orphan drugs listed in the European Union and Japan entered the phase of clinical trials/marketing and applications/approved clinical applications in China(86.4%from Japan and 70.6%from European Union).Conclusions Currently,there is no qualification accreditation mechanism for orphan drugs in China.However,in 2023,the number of rare disease drugs listed in China was the greatest among the four countries/international organization.It reflects that China's Catalog for Rare Diseases played a guiding role in drug research and development.Meanwhile,the reform of drug approval and review in China and the issue of rare disease catalogs took place in a short period of time,so the review and approval,as well as re-search and development of rare disease drugs are still in the catching up stage.