基于对同情用药认知现状的调研探索在我国开展同情用药研究的思考  

Exploration of Consideration for Compassionate Use in China Based on a Survey of Current Cognition

作　　者：向瑾[1] 周姚 张庆 XIANG Jin;ZHOU Yao;ZHANG Qing(Clinical Trial Center,West China Hospital of Sichuan University;School of Basic Medicine and Clinical Pharmacy,China Pharmaceutical University;Center of Clinical Operations Excellence,IQVIA RDS(Shanghai)Co.,Ltd.)

机构地区：[1]四川大学华西医院临床试验中心 [2]中国药科大学基础医学与临床药学学院 [3]艾昆纬医药科技(上海)有限公司临床运营卓越中心

出　　处：《中国食品药品监管》2024年第4期90-99,共10页China Food & Drug Administration Magazine

摘　　要：目的:根据中美同情用药经验,调研临床试验从业人员的认知和理解现状,对细化和完善我国同情用药制度体系提出建议,从而更好地满足患者的临床需求。方法:使用自愿抽样、方便抽样等非概率抽样方法,基于现有中美同情用药经验,就同情用药的适用条件、申请和审查主体、患者安全管理、审批流程等方面,对临床试验从业人员的认知和理解进行问卷调查。结果:共收集740份有效问卷。调研结果显示,我国临床试验从业人员对我国同情用药的认知程度有待提高。相关企业和机构人员对我国同情用药的认知与理解存在共性和差异。共性主要体现在对同情用药适用人群、“是否可以向药品监管部门提出同情用药申请”的审查主体、严重不良事件报告义务的承担方、同情用药开展地点以及收费问题的认知等方面。差异主要体现在同情用药的药品条件、申请主体、同情用药期间发生不良事件的补偿或赔偿承担方、美国食品药品监督管理局针对紧急情况下单个患者协议和非紧急情况下中等规模患者同情用药的审批流程等方面。结论:现阶段,我国临床试验从业人员对同情用药的了解不足。不同角色的临床试验从业人员对我国同情用药的药品条件、申请和审查主体、患者安全管理以及审批流程等方面的理解差异较大。因此,鼓励加强同情用药相关政策宣传工作,引导更多满足条件的企业开展同情用药计划,让更多潜在患者获益,同时建议针对理解差异较大的内容进一步明确、细化和完善。Objective:To explore the cognitive status and understanding of professionals related to clinical trials based on compassionate use experiences in China and the United States.It provides suggestions for refining and improving China’s compassionate use system to better meet patients'clinical needs.Methods:Non-probability sampling methods,such as voluntary and convenience sampling,are used.A questionnaire survey is conducted with professionals related to clinical trials,focusing on their cognition and understanding of the applicable conditions of compassionate use,the application and review subjects,patient safety management,and the approval process.Results:A total of 740 valid questionnaires are collected.Survey results shows that the awareness of Chinese clinical trial practitioners on compassionate use in China needs to be improved.There are commonalities and differences in cognition and understanding between enterprise practitioners and institutional practitioners.Commonalities are mainly reflected in their cognition of the population suitable for compassionate use,the review subject of applications for compassionate use to the National Medical Products Administration,parties responsible for patient adverse event reporting,places for compassionate use,and fee issues.Differences exist in drug conditions,application subjects,compensation for adverse events during compassionate use,and the FDA’s approval process for compassionate use of individual patients in emergency situations and medium-sized patients in non-emergency situations.Conclusion:Clinical trial practitioners in China have a significant lack of understanding of the country's compassionate use system.Survey results suggest that clinical trial practitioners in different roles have varying levels of understanding of drug conditions,applicants,patient safety management,and approval processes for compassionate use in China.Further policy advocacy work is recommended to guide more qualified enterprises to carry out compassionate use programs to benefit m

关 键 词：同情用药 拓展性临床试验 认知现状 适用条件 申请和审查主体 安全管理 审批流程 

分 类 号：R95[医药卫生—药学]