关键词: 药品注册, 监管科学, 批准前检查, 许可前检查, 基因治疗, 细胞治疗

Abstract:

Cell and gene therapy （CGT）， as a revolutionary technology in the field of modern biomedicine， provides the possibility of curing diseases such as cancer， genetic diseases， and rare diseases that are difficult to overcome by traditional medicine， through the modification， editing， and delivery of cells or genes. With the successive approval and marketing of products like chimeric antigen receptor T cell （CAR-T） therapy and adeno-associated virus （AAV） gene therapy， this field has witnessed accelerated technological iteration and rapid industrial expansion. The U.S. Food and Drug Administration（FDA） has established a scientifically rigorous and systematic regulatory inspection framework for CGT products. Systematically studying the regulatory requirements， inspection procedures and practical experience of the FDA in pre-approval inspections（PAIs） for CGT products holds significant practical importance for building an inspection system aligned with China's industrial characteristics， enhancing the level of regulatory science， and accelerating the translation process of innovative therapies from the laboratory to clinical application. This research comprehensively reviews relevant U.S. drug registration laws， regulations， and documents， providing an in-depth analysis of the pre-approval inspection process for CGT products， covering the inspection initiation mechanism， the basis and objectives of inspections， the scope and focus of inspections， and the handling and feedback mechanisms for inspection results. Furthermore， it analyzes the regulatory mechanism of U.S. PAI regulatory approach for CGTs through the lens of lifecycle-based oversight. The study aims to learn from internationally advanced regulatory concepts， to offer insights for optimizing China's regulatory framework for such products.

Key words:  , Drug Registration； , Regulatory science； , Pre-approval inspections； , Pre-license inspections； , Gene therapy； , Cell therapy