The Center for Drug Evaluation (CDE) in China has released the “Technical Guiding Principles for Cell and Gene Therapy (CGT) Product Clinical Communication and Exchange,” addressing the unique considerations of personalized and specific CGT products. Applicants are advised to focus on critical factors such as product characteristics, clinical positioning, target population, dosing, and trial design throughout the clinical trial process.
Pre-Clinical Trial Communication
The principles emphasize the importance of pre-first clinical trial application communication to address and resolve significant technical issues. Applicants are urged to consider safety observation indicators and the definition of dose limiting toxicity (DLT) during dose-finding phases.
Communication Prior to Key/Confirmatory Trials
Communication before key or confirmatory clinical trials is aimed at assessing whether existing data sufficiently supports the proposed trials and discussing the protocols. For new CGT products with high safety risks, if a single-arm trial design is chosen, applicants must provide statistical assumptions for efficacy evaluation and specify the required number of cases in the protocol. The setting of target values in single-arm trials should be well-founded, and the number of cases should satisfy both statistical requirements and safety risk assessments. Applicants are also encouraged to provide an overview of their marketing application plan/strategy.
Pre-Marketing License Application Dialogue
Dialogue before applying for a marketing license for therapeutic biological products focuses on whether existing data aligns with the technical requirements for drug marketing licenses.- Flcube.com
