The Center for Drug Evaluation (CDE) has released a set of “Guidelines for Clinical Trial Techniques for Human-Derived Stem Cells and Derived Cell Therapy Products,” which are now in immediate effect. These guidelines focus on clinical trials for stem cell-related products that are potential candidates for product approval, distinguishing them from investigator-initiated studies or other forms of research.
Scope and Definition of Human-Derived Stem Cells
The document defines “human-derived stem cells” as encompassing human embryonic stem cells, adult stem cells, and induced pluripotent stem cells. “Human-derived stem cell derived cell therapy products” refer to cell therapy products that originate from human-derived stem cells or mature somatic cell cells.
Clinical Trial Techniques and Processes
The clinical trial techniques outlined in the guidelines include a range of in vitro operations such as collection and transportation of cells from donors, extraction, separation, purification, cultivation, amplification, in vitro stimulation, gene editing or modification, induction of differentiation, cryopreservation and resuscitation, and transportation of stem cell-related products.
Emphasis on Compliance and Data Integrity
The Guiding Principles stress that stem-cell related clinical trials designed for product approval must adhere to general guidance, including China’s Good Clinical Practice (GCP) guidelines and ethical review requirements. Applicants are required to design a rigorous and scientific experimental plan based on the characteristics of stem-cell products to ensure participant safety and obtain reliable clinical trial data. The Principles also provide guidance on subject population, screening, protection, risk control, and other aspects of relevant clinical trials, as well as a series of processes for conducting exploratory/confirmatory clinical trials, long-term follow-up, post market research or monitoring.
Technical Evaluation and Application Submission
The document highlights the necessary conditions for clinical research used to support the market registration of a stem-cell-derived therapy as a drug product, including process and quality consistency, clinical research compliance, and data integrity. The drug review department will conduct a technical evaluation of the filed clinical research results to determine whether the safety, efficacy, in vivo proliferation, survival, and metabolic characteristics of stem-cell related products have met expected clinical goals. Applicants are advised to fully communicate with the drug evaluation department before submitting application materials.-Fineline Info & Tech
