Considerations For Operationalizing Pediatric Oncology Studies

By Katharine Chorlton

Remarkable progress has been made in our understanding of the genomics of pediatric cancers, and these advancements have led to the recognition that products being studied for use in adult cancer indications may have health benefits for pediatric patients. By closing the orphan drug exemption loophole and enabling earlier discussions with the FDA, the Research to Accelerate Cures and Equity (RACE) for Children Act has the potential to accelerate the development of novel treatment options for children with cancer.

Submitting an initial pediatric study plan

Per the RACE for Children Act, molecularly targeted pediatric cancer investigations should utilize appropriate formulations to yield clinically meaningful study data regarding dosing, safety, and preliminary efficacy for informing potential pediatric labeling. The critical first step in complying with the RACE for Children Act is submitting an initial pediatric study plan (iPSP) outlining a proposed timeline and design of pediatric studies with each Investigational New Drug that falls within the scope of this legislation.