HEROIC Trials to Answer Pragmatic Questions for Hospitalized Children

Area of Science:

• Pediatric clinical research
• Health services research

Background:

• The number of pediatric randomized controlled trials (RCTs) has stagnated since 2005, despite rising adult RCTs.
• Common pediatric diseases are underrepresented in RCTs due to barriers like low disease prevalence, funding issues, and complex regulations.
• Existing research has successfully addressed these barriers for rare diseases like pediatric cancer, but not for common childhood illnesses.

Purpose of the Study:

• To propose a novel strategy, High-Efficiency RandOmIzed Controlled (HEROIC) trials, to increase the volume and feasibility of RCTs for common diseases in hospitalized children.
• To outline key components of the HEROIC trials strategy designed for rapid, low-cost participant accrual with minimal site burden.

Main Methods:

• HEROIC trials are multicenter RCTs employing dispersed low-volume recruitment (50-150 sites, 2-10 participants/site).
• Strategies include incentivizing site leads, focusing on pragmatic research questions about widely used interventions, and streamlining regulatory processes (e.g., single IRB, integrated consent).
• The strategy emphasizes scaling to conduct multiple trials concurrently.

Main Results:

• The proposed HEROIC trials strategy facilitates rapid, low-cost accumulation of study participants.
• It aims to minimize the burden on individual research sites through efficient operational models.
• The strategy is designed to be scalable for simultaneous execution of multiple trials.

Conclusions:

• HEROIC trials offer a feasible approach to significantly boost the number of RCTs for common pediatric conditions.
• This strategy can address critical clinical questions and improve healthcare for hospitalized children.
• Implementing HEROIC trials can overcome existing barriers to pediatric clinical trial research.