China: New Guidelines for Decentralized Clinical Trials for Rare Disease Drug Development

On May 28, 2024, the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) released the Technical Guiding Principles for Applying Decentralized Clinical Trials (DCTs) in Rare Disease Drug Clinical Development. These guidelines aim to ensure the compliance of DCTs and scientific integrity for rare disease drugs.

The Guiding Principles integrate key concepts from ICH E6(R3), focusing on risk-based quality management (RBQM), patient-centricity, data authenticity, reliability, and traceability. This framework focuses on the importance of systematic risk assessments to align with RBQM, promoting the revamp of Good Clinical Practice (GCP) processes in research institutions.

1. Risk-Based Quality Management (RBQM):
   • Emphasis on compliance risk assessments to identify and mitigate potential issues.
   • Recommendations for setting up robust quality management systems and the conduct of risk-benefit assessments, especially concerning digital health technologies.
   • Detailed guidance on developing effective risk control plans to handle potential data quality, privacy, and security risks.
2. Patient-Centricity:
   • Encourages research institutions to redesign GCP compliance processes to prioritize patient rights, safety, and well-being.
   • Specific guidance on involving subjects through informed consent and privacy protection.
   • Emphasis on adapting trial procedures, including informed consent and data collection, to accommodate remote or virtual participation.
3. Vendor Compliance Management:
   • Guidelines for thorough vendor qualification assessments and rigorous performance monitoring.
   • Construction of a compliance evidence chain to document and maintain auditable records of all DCT activities.
   • Emphasis on training and education for vendors to ensure adherence to DCT protocols and regulations.

By adhering to these principles, sponsors, and researchers in China can enhance the quality and reliability of DCT implementation for rare diseases.