Consideration on"Patient-Centered"Drug Development for Rare Diseases
10.12376/j.issn.2097-0501.2024.02.003
- VernacularTitle:对"以患者为中心"的罕见疾病药物研发的审评考虑
- Author:
Ling TANG
1
;
Xing AI
;
Tian ZHAO
;
Zhimin YANG
Author Information
1. 国家药品监督管理局药品审评中心,北京 100076
- Keywords:
rare diseases;
patient-centered;
drug clinical research and development;
audit and approve
- From:
JOURNAL OF RARE DISEASES
2024;3(2):168-174
- CountryChina
- Language:Chinese
-
Abstract:
The unmet clinical needs of patients with rare diseases persist.Many rare diseases lack effec-tive treatments,and drug development for rare diseases faces greater challenges than that for common multiple diseases.In recent years,the concept of"patient-centered"drug development has been widely adopted.The Center for Drug Evaluation(CDE)of the National Medical Products Administration has successively issued a series of relevant guiding principles,such as the Technical Guidelines for the Implementation of Patient-Centered Clinical Trials,to promote a"patient-centered"drug development model.The implementation of the"patient-centered"approach in rare disease drug research and development,with a focus on patient perspectives and ac-tive engagement,can effectively facilitate a comprehensive understanding of rare diseases and patient needs among drug research and development enterprises,researchers,and regulatory agencies.This approach also en-hances the accuracy and efficiency of rare disease drug research and development.The CDE will continue to pri-oritize the integration of the"patient-centered"concept into rare disease drug research and development,effec-tively enhance the involvement of rare patients in the drug research and development process,and leverage the guiding role of patients'perspectives on drug research and development.
