To standardize the activities of confirmatory clinical research on new biomedical technologies, guide clinical research initiators, clinical research institutions, and researchers to conduct high-quality clinical research scientifically and in a standardized manner, and obtain key clinical evidence for evaluating the clinical application and transformation of new biomedical technologies, the China Biotechnology Development Center organized experts in clinical research, biostatistics, rare disease diagnosis and treatment, regulatory science, and medical ethics to jointly study and draft the "Technical Guidelines for Confirmatory Clinical Research of Biomedical New Technologies" (Draft for Public Comment). 

Objective: To standardize the design and implementation of confirmatory clinical research, produce high-quality evidence, protect the rights and interests of subjects, and facilitate the transition of technology from "research" to "clinical application" (i.e., routine implementation in hospitals). 

Scope of Application:

The focus is on highly personalized biomedical technologies (such as customized CAR-T and gene-editing therapies) used for the treatment of rare diseases. 

Does not replace the registration requirements for drugs or medical devices (those following the drug and medical device path still need to comply with the "Regulations for Drug Registration" and other relevant regulations); 

Emphasize "no rigid stage division" - there are no unified requirements for the research and development stages. Enterprises need to independently assess based on technical risks and disease characteristics when determining "when to enter the confirmatory stage".

Guidelines for Confirmatory Clinical Research on Biomedical New Technologies (Draft for Comment) 

I. Overview

三、Generally speaking

四、Protection of research participants

五、Key points of confirmatory clinical research design

六、Data quality and the traceability of all documents throughout the research process

七、Risk Management

八、Evidence formation for clinical translational application evaluation

九、Appendix

十、Definition of Nouns