Pediatric clinical trials are vital for ensuring safe and effective treatments for children, yet they face unique challenges distinct from adult trials. These include ethical and regulatory complexities, age-specific protocol design, small sample sizes, and logistical hurdles in recruitment and retention. Innovative strategies such as adaptive and Bayesian trial designs, pediatric research networks, and active caregiver engagement are helping to address these barriers. Emerging technologies such as digital health tools, electronic health records, real-world evidence, and artificial intelligence/machine learning are transforming trial conduct. Case studies demonstrate how these advancements can overcome traditional obstacles. Moving forward, pediatric research will benefit from stronger stakeholder collaboration, the integration of personalized medicine, and the use of genomics to improve outcomes. This review explores current challenges, practical solutions, and future directions in pediatric clinical trials, advocating for a dynamic research ecosystem tailored to children’s needs.
Upadhye et al. (2026) studied this question.