Current Status and Prospect of Orphan Drugs for Rare Skin Diseases in China

VernacularTitle:中国皮肤罕见病相关孤儿药现状及展望
Author: Shan ZHANG ^{1
,

2} ; Chao WU ^{1
,

2} ; Zhaorui LIU ^{1
,

2} ; Yimeng GAO ^{1
,

2} ; Jie LIU ^{1
,

2} ; Hongzhong JIN ^{1
,

2}
Author Information

1. Department of Dermatology, State Key Laboratory of Complex Severe and Rare Diseases, National Clinical Research Center for Dermatologic and Immunologic Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences &
2. Peking Union Medical College, Beijing 100730, China.
Publication Type:Journal Article
Keywords: rare skin diseases; orphan drugs; rare disease drugs; accessibility
From: JOURNAL OF RARE DISEASES 2023;2(2):261-272
CountryChina
Language:Chinese
Abstract: Rare diseases are characterized by extremely low incidence rate and small number of patients in total. The drugs used to treat rare diseases are called orphan drugs. Currently, 450 kinds of rare skin diseases have been identified, most of which lack in effective treatments. Supported by the policy-making from the country and pushed by all sectors of the society, drugs for rare skin diseases have been emerging continuously recently in China. This paper reviews the current accessibility of the orphan drugs for skin rare diseases that have been approved in China and in other countries and regions for a better understanding of rare dermatosis and orphan drugs for the diseases.