The Role and Challenges of Investigator-Initiated Trials in the Cell and Gene Therapy Products Boom in Mainland China

IF 3.1 3区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL

Cts-Clinical and Translational Science Pub Date : 2025-02-12 DOI:10.1111/cts.70148

Yifan Yang, Lianlian Bian, Yuan Cheng, Yan Xu, Hui Shao, Jian Rao, Sixiang Ge, Jifang Gong, Min Jiang, Xiaoyu Zheng, Lijun Liu, Shihui Ma, Xuan Liu, Tao Cheng, Chenyan Gao

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Abstract

As cutting-edge technologies in biomedicine, cell and gene therapy (CGT) products demonstrate immense potential in treating cancer, rare diseases, and genetic disorders, thereby driving the importance of clinical research in this area. This study analyzes the growth trends and key characteristics of 1033 Investigator-Initiated Trials (IITs) conducted by mainland Chinese institutions in the CGT field. The results show that IITs have played a positive role in the early proof-of-concept of CGT products, helping to obtain preliminary safety and efficacy data, and exploring the combination of CGT products with other therapies. Additionally, this study discusses the regional distribution, therapeutic areas, and challenges faced by IITs in the development of CGT products in China. Based on these findings, policy suggestions are proposed to optimize the regulation of IITs in mainland China, such as improving regulatory frameworks and enhancing technical guidance. It is hoped that these measures will further improve the efficiency and quality of IITs, fully utilize the large patient base and abundant clinical resources, and support the development of high-quality CGT products in mainland China.

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来源期刊

Cts-Clinical and Translational Science 医学-医学：研究与实验

CiteScore

6.70

自引率

2.60%

发文量

234

审稿时长

6-12 weeks

期刊介绍： Clinical and Translational Science (CTS), an official journal of the American Society for Clinical Pharmacology and Therapeutics, highlights original translational medicine research that helps bridge laboratory discoveries with the diagnosis and treatment of human disease. Translational medicine is a multi-faceted discipline with a focus on translational therapeutics. In a broad sense, translational medicine bridges across the discovery, development, regulation, and utilization spectrum. Research may appear as Full Articles, Brief Reports, Commentaries, Phase Forwards (clinical trials), Reviews, or Tutorials. CTS also includes invited didactic content that covers the connections between clinical pharmacology and translational medicine. Best-in-class methodologies and best practices are also welcomed as Tutorials. These additional features provide context for research articles and facilitate understanding for a wide array of individuals interested in clinical and translational science. CTS welcomes high quality, scientifically sound, original manuscripts focused on clinical pharmacology and translational science, including animal, in vitro, in silico, and clinical studies supporting the breadth of drug discovery, development, regulation and clinical use of both traditional drugs and innovative modalities.