医疗机构开展细胞治疗临床试验的思考

Reflections on the conduct of cell therapy clinical trials in medical institutes

目的本文介绍"以患者为中心细胞治疗研究管理模式",期望对国内医疗机构开展细胞治疗临床试验有所启示。方法通过总结北京大学深圳医院应对开展细胞治疗研究面临的技术评估、伦理评估风险以及规范实施等挑战的具体策略,探索建立以患者为中心的,多方共同决策的研究管理体系的相关思考与实践经验。结果"以患者为中心的细胞治疗研究管理模式"通过多方共同决策与管理,进行充分技术评估、有效伦理审查以及有组织的科研管理,确保研究结果可靠,不仅能满足患者迫切的临床治疗需求,也促进规范开展创新技术。结论"以患者为中心"的研究管理模式适用于细胞治疗临床试验,值得进一步评价与应用。

Objective This paper introduces the concepts of"patient-centered cell therapy research management model",to provide reference for domestic medical institutions which conduct cell therapy clinical trials.Methods We reviewed and summarized the experiences of conducting cell therapy at Peking University Shenzhen Hospital,including challenges and response plans regarding to the technology assessment,ethical evaluation risks and regulatory compliance.According to which,this paper aims to explore the reflections and practical experience of establishing a patient-centered,multi-stakeholder shared decision-making research management system.Results The"patient-centered cell therapy research management model"ensures the reliability of research results through multi-stakeholder engagement in decision-making and management,adequate technical evaluation,effective ethical review and harmonized scientific research management,which not only meets the urgent health needs of the patient,but also promotes the standardized development of emerging technologies.Conclusions The"patient-centered cell therapy research management model"is tailored for the cell therapy research,it is important to promote its further assessment and applications.