Comparison of the completed and discontinued pediatric drug clinical trials in Mainland China: a cross-sectional analysis based on the data from 2003 to 2023.

Background: Pediatric drug clinical trials are essential for ensuring the accessibility and safety of medications intended for children. In recent years, the Chinese government has implemented various measures to foster the development of pediatric drug clinical trials, and these efforts have yielded noticeable results. This study analyzed pediatric drug trial data from ClinicalTrials.gov and the Drug Clinical Trial Registration and Information Publication Platform. Its primary purpose was to discuss the issues and challenges faced by pediatric drug clinical trials in China and propose potential solutions.

Methods: This study conducted a cross-sectional analysis of concluded pediatric drug clinical trials (completed or discontinued) in mainland China from 2003 to 2023. Descriptive statistics were performed on the included data, and both univariate subgroup analysis and binary logistic regression were utilized to analyze the factors influencing completion duration and trial discontinuation.

Results: A total of 722 concluded pediatric drug clinical trials were extracted, with the overall number increasing annually at an average annual growth rate of 21.5%. We found that there existed imbalance in various aspects such as drug type, diseases, age, and the types and distribution of trial institutions. The analysis of trial durations indicated a progressive reduction in the completion cycles of pediatric drug clinical trials conducted in China, with notable variations across different subgroups. Four variables-trial size, the establishment of Data Monitoring Committees (DMCs), control type, and disease-exerted significant influence on the discontinuation of pediatric drug clinical trials. The primary reasons for trial discontinuation are issues related to safety or efficacy as reflected in trial outcomes (27.2%), as well as adjustments in commercial strategies or trial plans (24.6%).

Conclusions: Despite the ongoing increase in the number of pediatric drug clinical trials in China, significant challenges and imbalances persist across various dimensions. To enhance the quality and efficiency of these trials and collectively advance pediatric drug research and development, it is imperative to refine the existing legal and regulatory frameworks, promote the professionalization and standardization of pediatric drug clinical trials, bolster the application of emerging methods and technologies, such as seamless design and artificial intelligence.