真实世界证据改善儿童药物临床试验障碍的对策研究

Real world evidence ameliorates barriers to drug clinical trials in children

本文旨在为加快我国儿童药物的研发,促进儿童药物临床试验的顺利开展。通过文献分析,发现我国儿童药物临床试验存在动力不足的问题,主要原因在于伦理要求高、招募工作难以开展、临床试验风险高、时间长、试验设计复杂和评价困难等,因此尝试利用真实世界证据来解决这些难题。真实世界证据的应用可以扭转儿童药物临床试验的困境,但在运用过程中存在挑战。随着真实世界证据的不断发展和完善,未来能为儿童药物临床试验环节的监管决策提供足够的依据,促进真实世界证据在儿童药物临床试验领域的应用。

The aim of the article is to promote the smooth development of clinical trials of pediatric drugs in China and to accelerate their research and development.Through literature analysis,it was found that the motivation of clinical trial of pediatric drug in China was insufficient,mainly due to high ethical requirements,difficulty in recruiting subjects,high risk,long clinical trial time,complicated trial design and difficult evaluation,etc.Thus we try to use real-world evidence to resolve these difficulties.The application of real-world evidence can reverse the dilemma of clinical trials of pediatric drug,but challenges in the application process still exist.With the continuous development and improvement of real-world evidence,it can provide sufficient basis for regulatory decisions in clinical trials of pediatric drugs in the future,and promote the application of real-world evidence in pediatric drug clinical trials.