企业发起的中国儿科临床试验注册现况分析

Analysis of registered industry-sponsored clinical trials of pediatric drugs in China

目的 通过分析企业发起的注册类儿科药物和疫苗临床试验的特征,回顾中国儿科临床试验的发展情况,为推动儿科新药研发提供参考。方法 计算机检索ClinicalTrials.gov和中国临床试验注册中心,检索时限均从建库至2022年9月1日,由企业发起的已完成注册类儿科药物及疫苗临床试验,搜集注册时间、药物产品类型(药物和疫苗)、样本量等相关信息并进行描述性分析。将试验注册时间分为2005―2010年和2011―2022年两个阶段,反映近年来儿科药物临床试验政策的颁布情况。比较两个阶段干预性试验的注册质量和主要特征,探究企业和非企业研究者发起临床试验的结果情况。结果 共收集145项试验,产品类型疫苗占63.4%;随机对照试验(RCT)占68.3%。所有干预性试验平均报告完成度为81.0%,相比于2005―2010年,2011―2022年注册试验平均报告完成度、儿科药物临床试验、多中心试验、RCT和双盲试验的比例均有所提高。企业发起儿科临床试验阳性结果比例大于非企业研究者发起的儿科临床试验。结论 企业发起的已完成的儿科临床试验以疫苗居多。随着儿科临床试验政策的逐步完善,试验注册质量提高。部分试验设计特征发生变化提示试验的质量有所提高但不明显。企业发起的儿科临床试验阳性结果占比更大。未来需要进一步推动儿科临床试验的发展。

Objective To review the characteristics of registered industry-sponsored clinical trials of pediatric drugs and vaccines in China and to provide references for promoting the development of new pediatric drugs. Methods We searched ClinicalTrials.gov and the Chinese Clinical Trial Registry for completed registered industry-sponsored clinical trials of pediatric drugs and vaccines from the database inception to September 11, 2022. Data including the date the trial was first posted, product type(drug or vaccine), sample size, and other information to describe the general characteristics of pediatric clinical trials were collected. The studies were divided into 2 phases based on the trial posted date, 2005―2010 and 2011―2022, reflecting the enactment of pediatric drug clinical trial policies in recent years. The quality of trial registration and the main characteristics of interventional trials in the 2 phases were then compared. Exploring the results attached to industry and non-industry sponsored clinical trials. Results Data for 145 trials were collected, and the largest proportion(63.4%) involved vaccines. Randomized control trial(RCT) was the study type with the highest percentage(68.3%). The average report completion rate for registered interventional trials was 81.0%. Compared with 2005―2010,the percentage of average report completions, pediatric drug clinical studies, multicenter, RCTs, and double-blinded registered trials increased in 2011―2022. The proportion of positive outcomes in pediatric clinical trials sponsored by industries was higher than those sponsored by non-industry. Conclusion The majority of completed pediatric clinical trials sponsored by industries are for vaccines, in line with the promotion of pediatric policies. The quality of trial registration has improved, but not significantly, and some characteristics of trial design have changed. The proportion of positive outcomes in pediatric clinical trials sponsored by industries is higher. And further promotion of pediatric clinical trials is needed.