Early Discontinuation, Results Reporting, and Publication of Pediatric Clinical Trials

Abstract

Background and objectives: Unique ethical, epidemiological, and economic factors are barriers to performing research in children. The landscape of pediatric clinical trials, including drivers of completion and timely dissemination of results, is not well understood. We aimed to characterize the prevalence of and factors associated with early discontinuation, results reporting, and publication of pediatric clinical trials registered at ClinicalTrials.gov.

Methods: Cross-sectional analysis of clinical trials enrolling participants <18 years old registered at ClinicalTrials.gov from October 2007 to March 2020. Multivariable logistic regressions were performed to assess the association between trial characteristics and primary outcomes. Publication data were obtained through PubMed, ClinicalTrials.gov, Embase, and Scopus.

Results: Overall, 11.1% trials were stopped early, with recruitment failure being the predominant reason for discontinuation. Only 23.5% of completed trials reported results, and 38.8% were published within 3 years of completion. Rates of discontinuation and publication significantly improved over the study period. Among funding sources, government-sponsored trials (adjusted odds ratio [aOR], 0.72; 95% CI, 0.47-0.97) and academic trials (aOR, 0.64; 95% CI, 0.50-0.82) had lower odds of discontinuation compared with industry trials and were more likely to be published (government: aOR, 1.94 [95% CI, 1.52-2.48] academic: aOR, 1.61 [95% CI, 1.35-1.92). Academic trial investigators were the least likely to report results (aOR, 0.34; 95% CI, 0.31-0.52).

Conclusions: Early discontinuation and nonreporting/nonpublication of findings remain common in registered pediatric clinical trials and were associated with funding source and other trial features. Targeted efforts are needed to support trial completion and timely results dissemination toward strengthening evidence-based pediatric medicine.