The Interpretation of Guideline for Clinical Trials Design of Gene Therapy Products for Rare Diseases(Trial Version)
10.12376/j.issn.2097-0501.2024.03.011
- VernacularTitle:《罕见病基因治疗产品临床试验技术指导原则(试行)》解读
- Author:
Xiao LIU
1
;
Xiyuan WU
;
Shuang LU
Author Information
1. 国家药品监督管理局药品审评中心,北京 100076
- Keywords:
rare diseases;
gene therapy;
clinical trials;
review considerations
- From:
JOURNAL OF RARE DISEASES
2024;3(3):345-349
- CountryChina
- Language:Chinese
-
Abstract:
In recent years,driven by policy incentives and regulatory reforms,the research and devel-opment of rare disease drugs in China has begun to show a promising future.As of March 2024,more than 30 gene therapy products for rare diseases in China have been approved for clinical trials.In order to guide the clinical trial design of gene therapy products for rare diseases,Center for Drug Evaluation,National Medical Products Administration has issued Guideline for Clinical Trials Design of Gene Therapy Products for Rare Disea-ses(Trial Version).Now we try to interpret this guideline to provide recommendations for the industry.
