摘要
目的为优化儿童药物临床试验管理提供参考。方法收集2019年1月至2023年12月南京医科大学附属儿童医院开展的25项儿童药物临床试验资料,分析受试者筛选失败和脱落原因。结果共筛选261例受试者,筛选失败63例(64例次),筛选失败率为24.14%。筛选失败原因包括不符合疾病状态22例次(34.38%),不符合疾病诊断16例次(25.00%),合并其他疾病12例次(18.75%),撤回知情同意7例次(10.94%),不符合方案的其他要求7例次(10.94%)。共脱落21例受试者,脱落率为10.61%。脱落原因包括撤回知情同意(5例,23.81%),监护人自觉无效(5例,23.81%),疫情影响(3例,14.29%),发生安全性事件(3例,14.29%),入组后未治疗(2例,9.52%),不符合方案第二阶段筛选标准(2例,9.52%),失访(1例,4.76%)。结论不符合疾病状态、不符合疾病诊断、合并其他疾病、撤回知情同意为儿童药物临床试验主要筛选失败原因,撤回知情同意、监护人自觉无效、疫情影响、发生安全性事件为主要脱落原因。优化临床试验方案设计和深度知情是改善筛选合格率和脱落率的关键。
Objective To provide a reference for optimizing the management of pediatric drug clinical trials.Methods The data of 25 pediatric drug clinical trials conducted by the Children′s Hospital of Nanjing Medical University from January 2019 to December 2023 were collected to analyze the reasons for subject screening failures and dropouts.Results A total of 261 subjects were screened,with 63 screening failures(64 case times)and a screening failure rate of 24.14%.The reasons for screening failures included failure to meet the disease status(22 case times,34.38%),failure to meet the disease diagnosis(16 case times,25.00%),complicated with other diseases(12 case times,18.75%),withdrawal of informed consent(seven case times,10.94%),and failure to meet other requirements of the protocol(seven case times,10.94%).A total of 21 subjects were dropped out,with a dropout rate of 10.61%.The reasons for dropouts included withdrawal of informed consent(five cases,23.81%),guardian′s perceived ineffectiveness(five cases,23.81%),impact of the epidemic(three cases,14.29%),occurrence of safety incidents(three cases,14.29%),failure to receive treatment after enrollment(two cases,9.52%),failure to meet the second-stage screening criteria of the protocol(two cases,9.52%),and loss to follow-up(one case,4.76%).Conclusion Failure to meet disease status,failure to meet disease diagnosis,complicated with other diseases,withdrawal of informed consent are the main reasons for screening failures in pediatric drug clinical trials.Withdrawal of informed consent,guardian′s perceived ineffectiveness,impact of epidemic,and occurrence of safety incidents are the main reasons for dropouts.The optimization of clinical trial protocol design and thorough informed consent are crucial for improving the screening pass rate and dropout rate.
作者
刘艳
季兴
倪明明
王珊珊
吴文文
李曼
许静
LIU Yan;JI Xing;NI Mingming;WANG Shanshan;WU Wenwen;LI Man;XU Jing(Children′s Hospital of Nanjing Medical University,Nanjing,Jiangsu,China 210008)
出处
《中国药业》
CAS
2024年第24期39-42,共4页
China Pharmaceuticals
基金
江苏省药学会科研项目[H202124]。
关键词
药物临床试验
儿童
受试者
筛选失败
脱落
drug clinical trial
child
subject
screening failure
dropout
