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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研究人员发起的试验在中国大陆细胞和基因治疗产品繁荣中的作用和挑战

作为生物医学的前沿技术，细胞和基因治疗（CGT）产品在治疗癌症、罕见疾病和遗传疾病方面显示出巨大的潜力，从而推动了该领域临床研究的重要性。本研究分析了中国大陆科研机构在CGT领域开展的1033项研究者发起试验（iit）的增长趋势和主要特征。结果表明，iit在CGT产品的早期概念验证中发挥了积极作用，有助于获得初步的安全性和有效性数据，并探索CGT产品与其他疗法的结合。此外，本研究还讨论了iit在中国CGT产品开发中的区域分布、治疗领域和面临的挑战。在此基础上，提出了完善监管框架、加强技术指导等政策建议。希望这些措施能够进一步提高iit的效率和质量，充分利用中国大陆庞大的患者基数和丰富的临床资源，支持中国大陆优质CGT产品的发展。

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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.