Assessing Information Gaps Associated with Initial Pediatric Study Plans for New Oncology Drug and Biological...

Area of Science:

• Oncology
• Pediatric Drug Development
• Regulatory Science

Background:

• The Research Acceleration for Cure and Equity (RACE) for Children Act mandates the submission of Pediatric Study Plans (PSPs) for new molecularly targeted drugs and biologics intended for adult cancers.
• These plans must either propose a pediatric investigation or provide justification for a waiver or deferral.
• Ensuring pediatric patient consideration in drug development is a key objective.

Purpose of the Study:

• To conduct a landscape analysis of information gaps in initial Pediatric Study Plan (iPSP) submissions for oncologic new molecular entities in 2021.
• To identify trends in US Food and Drug Administration (FDA) comments during the iPSP review process.
• To provide direction for sponsors in preparing adequate iPSPs.

Main Methods:

• A landscape analysis was performed on initial PSP (iPSP) submissions for oncologic new molecular entities received in 2021.
• Comments from the FDA review process were categorized using nine distinct flags.
• Analysis focused on identifying common information gaps and trends.

Main Results:

• For iPSPs requesting a full waiver, the primary information gap was inadequate justification regarding molecular target relevance.
• Other iPSP plans (deferral, partial waiver, or investigation) frequently showed information gaps in clinical study features, clinical pharmacology, or missing clinical/nonclinical data.
• Trends in FDA comments during initial review were identified.

Conclusions:

• Initial PSP submissions often contain information gaps that require sponsor attention.
• Clearer justification for molecular target relevance is crucial for waiver requests.
• Sponsors need to ensure comprehensive data for clinical study features and pharmacology to meet regulatory requirements for pediatric drug development.