Importance: Pediatric medication is a challenging issue globally. Promoting trials of medications for children and implementing measures to encourage innovation for addressing unmet medical and health needs are important.
Objective: To explore the recent landscape of pediatric clinical trials of new investigational drugs conducted by pharmaceutical enterprises in China from 2013 to 2022 to provide insight into pediatric drug development in the pharmaceutical industry and regulatory policy formulation.
Methods: We performed a cross-sectional observational investigation of pediatric clinical trials registered from January 1, 2013, to December 31, 2022, on the Registration and Information Disclosure Platform for Drug Clinical Trials, the official registration platform established in 2013 for trials of new investigational drugs initiated by biopharmaceutical enterprises. Trials that included pediatric participants (under 18 years old) were retrieved, and their relevant characteristics were extracted and analyzed.
Results: In total, 895 pediatric clinical trials were collected, accounting for 5.1% of the total registered clinical trials initiated prior to January 1, 2023. The overall average annual growth rate for the number of pediatric clinical trials was 12% (P < 0.001). Phase III trials accounted for the highest proportion (49.1%, 439). Of the 895 trials included, 736 (82.2%) were domestic trials, and 159 (17.8%) were international multicenter trials. In terms of tested drugs, investigations of biological products accounted for the largest proportion of trials (67.4%, 603). Among pediatric clinical trials, studies of vaccines accounted for the largest proportion of trials (41.0%, 367), followed by trials for rare diseases (17.2%, 154). Furthermore, geographical distribution analysis revealed that the largest and smallest numbers of trials were conducted in North China (35.7%, 320) and Northeast China (0.8%, 7), respectively.
Interpretation: The growth trends for industry-sponsored clinical trials involving children illustrate the progress and increasing capability of pediatric drug development achieved in China since 2013. Current challenges and potential areas of focus for policymakers and stakeholders include investigating orphan drugs for rare diseases according to the unique epidemiological characteristics of Chinese children, expanding the scope of pediatric clinical trials, and improving the uneven geographical distribution of leading research centers.
Keywords: Geographic distribution; Pediatric clinical trials; Pediatric medication; Rare disease.
© 2024 Chinese Medical Association. Pediatric Investigation published by John Wiley & Sons Australia, Ltd on behalf of Futang Research Center of Pediatric Development.