Although the number of randomized controlled trials (RCTs) published each year involving adult populations is steadily rising, the annual number of RCTs published involving pediatric populations has not changed since 2005. Barriers to the broader utilization of RCTs in pediatrics include a lower prevalence of disease, less available funding, and more complicated regulatory requirements. Although child health researchers have been successful in overcoming these barriers for isolated diseases such as pediatric cancer, common pediatric diseases are underrepresented in RCTs relative to their burden. This article proposes a strategy called High-Efficiency RandOmIzed Controlled (HEROIC) trials to increase RCTs focused on common diseases among hospitalized children. HEROIC trials are multicenter RCTs that pursue the rapid, low-cost accumulation of study participants with minimal burden for individual sites. Five key strategies distinguish HEROIC trials: (1) dispersed low-volume recruitment, in which a large number of sites (50-150 hospitals) enroll a small number of participants per site (2-10 participants per site), (2) incentivizing site leads with authorship, training, education credits, and modest financial support, (3) a focus on pragmatic questions that examine simple, widely used interventions, (4) the use of a single institutional review board, integrated consent, and other efficient solutions to regulatory requirements, and (5) scaling the HEROIC trial strategy to accomplish multiple trials simultaneously. HEROIC trials can boost RCT feasibility and volume to answer fundamental clinical questions and improve care for hospitalized children.

<p>Children with complicated appendicitis, osteomyelitis, and complicated pneumonia have historically been treated with postdischarge intravenous antibiotics (PD-IV) using peripherally inserted central catheters (PICCs). Recent studies have shown no advantage and increased complications of PD-IV, compared with oral therapy, and the extent to which use of PD-IV has since changed for these conditions is not known. We used a national children's hospital database to evaluate trends in PD-IV during 2000-2018 for each of these three conditions. PD-IV decreased from 13% to 2% (risk ratio [RR], 0.15; 95% CI, 0.14-0.16) for complicated appendicitis, 61% to 22% (RR, 0.41; 95% CI, 0.39-0.43) for osteomyelitis, and 29% to 19% (RR, 0.63; 95% CI, 0.58-0.69) for complicated pneumonia. Despite these overall reductions, substantial variation in PD-IV use by hospital remains in 2018.</p>