Assessment of Oral Anticancer Medicines in Pediatric Patients before the Research to Accelerate Cures and Equity (RACE) Act, 2007-2021.

Abstract

To assess the relationship between availability of pediatric labeling information as well as on-label pediatric safety information and the use of oral targeted anticancer medications (OTAMs) by pediatric patients. We identified all OTAMs first approved by the Food and Drug Administration from January 1, 2007, to September 31, 2021, and assessed their use by children with cancers on the basis of claims data in Optum's deidentified Clinformatics Data Mart from January 1, 2007, to September 31, 2021. Over the study period and across cancer types, 3.8% of patients with childhood cancer received at least 1 OTAM. The majority of OTAMs used (29.4% in 2008 and 51.7% to 88.6% in other years) did not have an indication for the diagnosed pediatric cancers. First OTAM treatment episodes lasted longer (ie, discontinuations happened later) for drugs with pediatric cancer indications and safety information compared with those without the information during the episode of use. From 2007 to 2021, median standard costs of 30-day supplies of OTAMs for patients <18 years old were $6714 and inflation-adjusted, out-of-pocket median spending for a 30-day supply of OTAMs was about $11. Our results show relatively limited yet increasing pediatric OTAM use over time. We document a greater likelihood of earlier discontinuation and significant financial cost for drugs without on-label pediatric cancer indications and safety information. There is a need for a robust regulatory framework to mandate the timely generation of pediatric population-specific efficacy and safety evidence.