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

The Research Acceleration for Cure and Equity (RACE) for Children Act requires sponsors to submit a Pediatric Study Plan (PSP) with a proposed pediatric investigation of new molecularly targeted drugs and biologics that are intended for treatment of adult cancers, and whose target is relevant to pediatric cancer or provide a justification for a plan to request a deferral or waiver of the required investigation. A landscape analysis was performed to identify trends in information gaps associated with a sponsor's first initial PSP (iPSP) submission for oncologic new molecular entities received in 2021. Comments sent to sponsors by the US Food and Drug Administration (FDA) during the review process of each evaluated iPSP were categorized using nine flags relating to different portions of the PSP. For iPSPs that included a plan for a full waiver request, the most common information gap was inadequate justification based on molecular target relevance. All other sponsor proposed plans (deferral and/or partial waiver or investigation) were found to have information gaps related to clinical study features, clinical pharmacology, and/or missing clinical or nonclinical data. This landscape analysis of iPSPs shows the trends in comments that often occur during initial review and may help to provide sponsors with more direction for preparing an adequate iPSP to fulfill statutory requirements aimed at ensuring pediatric patients are considered in the development of new molecularly targeted drugs.