Cell and gene therapy(CGT)is a key direction for the future development of the global biopharmaceutical industry and exhibits immense potential applications in the treatment of severe or refractory diseases where traditional treatments are ineffective.In China,due to the unique nature of CGT products,most companies initiate investigator-initiated trials(IIT)at the early stages of research to preliminarily assess the safety and efficacy of the products.However,currently,the results of IIT in China cannot be directly applied and must transition to industry-sponsored clinical trials(IST)conducted by pharmaceutical companies,followed by drug registration and marketing approval processes.Therefore,this paper systematically reviews the regulatory status of CGT and IIT in various countries and regions,including the United States,Europe,and Japan,and analyzes cases of IIT-IST transitions.In conjunction with the development status of China's pharmaceutical industry and the current management of IIT,this paper examines the challenges faced in the transition from IIT to IST in China,and subsequently proposes relevant recommendations,aiming to better allocate clinical research resources for CGT products and promote the development of the CGT industry.
关键词
细胞和基因治疗产品/研究者发起的临床研究/制药企业发起的药物临床试验/国外监管体系/启示
Key words
cell and gene therapy/investigator-initiated trial/industry-sponsored clinical trial/overseas supervision systems/enlightenment
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