Investigation on the current situation : Pediatric off-label drug use in Chinese Hospitals

Introduction Off-label prescribing, use of medications outside approved indications, dosages, administration routes, or age groups is common in pediatric clinical practice, largely because of the lack of high-quality clinical trials in children. Although such prescriptions can meet urgent therapeutic needs, particularly in complex or rare pediatric conditions, they also raise significant concerns regarding safety, effectiveness, and medicolegal liability. Limited research has examined the behavioral factors that influence pediatricians’ decisions to prescribe off-label drugs. Aim This study aimed to identify the behavioral determinants of pediatricians’ off-label drug use in Chinese hospitals by applying the Theory of Planned Behavior (TPB) to assess how attitudes, subjective norms, and perceived behavioral control influence prescribing intentions and behaviors. Methods A cross-sectional survey was conducted among pediatricians from 40 hospitals across seven provinces and municipalities in China. A TPB-based questionnaire was developed and refined through expert panel review and pilot testing. Psychometric properties were evaluated using reliability testing and confirmatory factor analysis (CFA). Structural equation modeling (SEM) was used to test the hypothesized relationships among the TPB constructs. Results Most pediatricians acknowledged the necessity (82.4%) and risks (78.9%) of off-label use. Attitudes reflecting perceived benefits, safety concerns, and cost implications significantly predicted behavioral intention (β = 0.32, p < 0.01). Perceived behavioral control, including barriers such as outdated labeling, lack of pediatric formulations, and limited data, also predicted intention (β = 0.30, p < 0.01), but not behavior directly (β = 0.08, p = 0.12). Subjective norms such as institutional expectations and peer influence were positively associated with intention (β = 0.28, p < 0.01). Behavioral intention was the strongest predictor of actual off-label prescription behaviors (β = 0.41, p < 0.001). Most pediatricians (85.2%) supported pharmacists’ involvement in evidence reviews, documentation, and prescription oversight. The SEM demonstrated a good model fit (CFI = 0.94, RMSEA = 0.065, SRMR = 0.045). Conclusion Pediatric off-label prescribing in China is largely intention-driven and shaped by attitudes, perceived control, and professional norms. Interventions targeting these behavioral domains, along with institutional policies and pharmacist collaboration, may enhance the safety, consistency, and regulatory oversight of off-label use in pediatric care.