Identifying Relevant Randomized Clinical Trial Questions for Hospitalized Children


Newly published consensus statement outlines RCT questions ranked according to importance and feasibility.

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While the number of randomized clinical trials (RCTs) for adult participants has increased over time, the number of available RCTs for children has remained mostly stagnant. This leads to uncertain decision-making by pediatric clinicians, who often need to rely on adult studies and expert opinion.1

A consensus statement recently published on JAMA Network Open aimed to address this challenge by identifying the most important and feasible RCT questions for children hospitalized with common conditions.

“There is a lack of randomized clinical trial (RCT) data to guide many routine decisions in the care of children hospitalized for common conditions,” the statement authors wrote. “A first step in addressing the shortage of RCTs for this population is to identify the most pressing RCT questions for children hospitalized with common conditions.”

According to the authors, a 3-stage modified Delphi process was used in a virtual conference series spanning January 1 to September 29, 2022 for the creation of the consensus statement. The participants included 46 individuals from 30 different institutions. In stage 1 of the 3-stage process, participants constructed RCT questions for the 10 most common pediatric conditions leading to hospitalization using condition-specific guidelines and reviews from a structured literature search. In the second stage, RCT questions were refined and scored according to importance, and finally, stage 3 incorporated public comment and feasibility with the prioritization of RCT questions.

This process produced 62 unique RCT questions, “most of which are pragmatic, comparing interventions in widespread use for which definitive effectiveness data are lacking.” In terms of importance and feasibility, questions were scored in a range of 1 to 9 with a median of 5. Of the top 10 selected questions, 6 focused on determining optimal antibiotic regimens for 3 common infections (pneumonia, urinary tract infection, and cellulitis).

“The questions that were prioritized in this process reflect broad goals of improving the value of care delivered to hospitalized children by avoiding unnecessary treatments. Six of the top 10 selected questions focused on determining optimal antibiotic regimens for 3 common infections (pneumonia, urinary tract infection, and cellulitis),” the authors wrote of the results. “There is wide variation in antibiotic prescribing for these infections, raising concerns for potential antibiotic overtreatment and its detrimental effects for children (antibiotic-associated adverse effects), families (stress and costs), and communities (emergence of resistant organisms).”

The authors suggested investigators will likely need to turn away from traditional RCT approaches and use more innovative and efficient processes to address this new, ambitious list of RCT questions. Two designs that might be more feasible are platform and factorial RCTs.

“The single condition entity and similar interventions could lend themselves to a platform or factorial design,” the authors wrote. “RCT questions developed here may also lend themselves to a high-efficiency randomized controlled trial approach, which uses a dispersed enrollment strategy to efficiently conduct pragmatic RCTs.”

Among the limitations of this study was question development being centered around common conditions. The authors acknowledged there are additional questions not condition-based or are focused on less-common conditions.

“In this consensus statement, we have identified the most important and feasible RCT questions for children hospitalized with common conditions. These conditions are responsible for more than three-fourths of pediatric hospitalizations,” the authors concluded. “Answering these pressing questions with RCTs has great potential to improve care and outcomes for hospitalized children.”


1. Coon ER, McDaniel CE, Paciorkowski N, et al. Prioritization of Randomized Clinical Trial Questions for Children Hospitalized With Common Conditions: A Consensus Statement. JAMA Netw Open. 2024;7(5):e2411259. doi:10.1001/jamanetworkopen.2024.11259

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