Current Development Status and Consideration for Rare Hemorrhagic Disease Drugs
10.12376/j.issn.2097-0501.2022.04.017
- VernacularTitle:罕见出血性疾病药物研发现状与思考
- Author:
Ling TANG
1
;
Limin ZOU
1
;
Yu DU
1
;
Yueli QI
1
;
Meiyi XIANG
1
;
Zhimin YANG
1
Author Information
1. Center for Drug Evaluation, National Medical Products Administration, Beijing 100022, China.
- Publication Type:Journal Article
- Keywords:
rare diseases;
hemorrhagic diseases;
drug research and development
- From:
JOURNAL OF RARE DISEASES
2022;1(4):461-467
- CountryChina
- Language:Chinese
-
Abstract:
Hemophilia is the only rare hereditary hemorrhagic disorder included in the First Rare Diseases catalogue. However, rare bleeding diseases identified in the clinic are far more common than hemophilia. Most other rare hemorrhagic disorders have less effective treatment than hemophilia. Hemophilia has a history of successful drug development in rare hemorrhagic diseases, and the cycle between clinical research and drug development has been gradually realized. Drug research and pharmaceutical companies can refer to the drug research and development process in the field of hemophilia, learn from the experience of hemophilia drug research and develop treatments. The industry can increase drug development by strengthening basic research, focusing on the value of natural history research, the application of quantitative pharmacological tools and improving the efficiency of drug development to meet the urgent unmet medical needs of patients with rare hemorrhagic diseases.
