Medication Repurposing in Pediatric Patients: Teaching Old Drugs New Tricks

Area of Science:

• Pediatric Pharmacology
• Drug Repurposing
• Evidence-Based Medicine

Background:

• Pediatric therapeutics often face gaps, leading to off-label medication use.
• Drug repurposing, a novel use for existing medications, is critical in pediatric care.
• Difficulties in accessing evidence-based information for these uses prompted this review.

Purpose of the Study:

• To characterize innovative off-label medication uses in pediatric patients.
• To identify medications approved by the Food and Drug Administration (FDA) for one indication but used for a different disorder in children.
• To provide a comprehensive listing of repurposed medications specific to pediatric populations.

Main Methods:

• A systematic literature review was conducted to identify repurposed medications in pediatric patients.
• Publications were screened, excluding preclinical data, adult-only use, experimental use, and off-label use of FDA-approved indications with different dosages.
• Evidence quality was assessed using a modified American Academy of Neurology Level of Evidence scale.

Main Results:

• 101 medications were identified for novel off-label pediatric uses: 38 for neonates, 74 for children, and 52 for adolescents.
• Neonates and infants were least likely to receive repurposed medications.
• Strong or intermediate evidence supported 80.2% of the identified repurposed uses, while 19.8% had weak evidence.

Conclusions:

• This study offers a comprehensive catalog of novel, repurposed medication uses exclusively for pediatric patients.
• Further research is required to determine the prevalence of these repurposed uses.
• Pharmacists play a crucial role in evaluating the evidence base for repurposed drug applications in pediatrics.