CDE released several guidelines related to clinical trials in DEC2024

Requirements for Writing Pharmaceutical Information in Module 2.3 of Clinical Trial Application for Therapeutic Biological Products (Draft): These draft requirements were developed to instruct applicants on proper Common Technical Document (CTD) format and the appropriate pharmaceutical information to include in Module 2.3 of the application to conduct a clinical trial for a therapeutic biological product. The document includes three appendices outlining the requirements for structure and content of information related to innovative recombinant protein products, antibody-drug conjugates, and biosimilars. CDE is revising these requirements following a period of public feedback.

• Statistical Guidelines for Vaccine Clinical Trials: These guidelines explain key statistical principles in clinical trials for vaccines and provide suggestions on how to design and evaluate a trial with these principles in mind. The guidelines define and describe endpoint evaluation indicators (i.e., immunogenicity and effectiveness) and how to address general statistics-related issues that arise in trial design and evaluation; they also provide advice on cluster randomization and communication with regulatory authorities.

• Technical Guidelines for Model-Guided Dose Exploration and Optimization of Innovative Drugs: These guidelines explain basic principles and considerations for dose exploration and optimization, model-guided research design, and model analysis for exploratory clinical trials of innovative chemical and biological products. They also include considerations for different scenarios such as conducting international multi-center studies, studying combination therapies, expanding indications, and addressing subsequent changes, among others. These guidelines do not apply for starting dose selection as part of a first-in-human trial.

• Guidelines for Estimating Sample Size for Drug Clinical Trials: These guidelines explain the basic considerations for sample size estimation in confirmatory clinical trials conducted as part of product registration. They describe considerations including experimental design, inspections, statistical analysis methods, and expected treatment effects, as well as how to adjust sample sizes if needed. The document also describes Bayesian sample size estimation and sample size sensitivity calculation. 

Original link：

1. 关于公开征求《治疗用生物制品申报临床试验申请模块2.3药学资料撰写要求（征求意见稿）》意见的通知

2. 国家药监局药审中心关于发布《疫苗临床试验统计学指导原则（试行）》的通告（2024年第52号）

3. 国家药监局药审中心关于发布《模型引导的创新药物剂量探索和优化技术指导原则》的通告（2024年第53号）

4. 国家药监局药审中心关于发布《药物临床试验样本量估计指导原则（试行）》的通告（2024年第54号）