Module232025

Lack of Approved Active Principal Ingredients for Pediatric-Age Patients

Limited Approvals :

o Example: Only 19 of 103 antineoplastic drugs for adults are approved for pediatric use.

o 42% of drugs under pediatric exclusivity lacked pediatric labeling.

o Pediatric drug approvals often face delays of over a decade. Off-Label Prescriptions :

o 38.1% of medications for pediatric patients are off-label.

o Off-label rates: Neonates at least 26%; Outpatients: 2.7% – 51.2%; Inpatients: 9.0% – 79.0%.

o Risks include unsuitable treatments and adverse events. Efforts to Improve :

o I-ACT (2017): Promotes faster access to pediatric medications.

o Pediatric Innovation Forum (2019): Advocates enrolling adolescents in phase III trials. Guideline Support :

o Clinical Practice Guidelines help reduce risks of irrational off-label drug use.

Quality (CMC) Development Considerations

The Organisation for Professionals in Regulatory Affairs