The Interpretation of Guideline for Clinical Trials Design of Gene Therapy Products for Rare Diseases(Trial Version)

Abstract

In recent years, driven by policy incentives and regulatory reforms, the research and development 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 Diseases (Trial Version). Now we try to interpret this guideline to provide recommendations for the industry.

Keywords

• rare diseases
• gene therapy
• clinical trials
• review considerations