Abstract 3365: Pediatric trials and labeling information for newly approved cancer therapies with targets potentially relevant to pediatric cancers
Authors: Hwang Thomas J. , Orenstein Liat , Dubois Steven , Bourgeois Florence 
Published: August 2019

Abstract
Objective: Few new therapies have been approved for pediatric cancers. In 2017, Congress enacted the Research to Accelerate Cures and Equity (RACE) for Children Act, which requires pediatric studies for adult cancer drugs with a molecular target relevant to a pediatric cancer. The pediatric study requirements will apply to new drugs approved by the FDA beginning in 2020. To inform the implementation of this new law, we evaluated pediatric trials and labeling information available for cancer drugs approved by the FDA.
Methods: We identified new adult cancer drugs approved by the FDA from January 2007 to December 2017 using the Drugs@FDA database. New formulations, drugs approved only for pediatric cancers, and imaging and contrast agents were excluded. For each drug, the potential pediatric relevance of the molecular target was determined using the Pediatric Molecular Target List published by the FDA in August 2018. Information on pediatric clinical trials, enrollment, and start and end dates was obtained from ClinicalTrials.gov as of September 2018. Drug labels were examined for pediatric efficacy, safety, or PK/PD and dosing data. A trial potentially open to children was defined as a study for which any participants &lt; 18 years of age were eligible. We also examined pediatric studies, defined as trials for which the midpoint of the eligible age range was &lt; 21 years.
Results: Among the 78 adult cancer drugs approved between 2007 and 2017, 61 (78%) had targets considered potentially relevant for pediatrics under the RACE Act. At the time of approval, 4 (5%) drugs had any pediatric labeling information. As of September 2018, after a median follow-up of 5.1 years (IQR: 2.9-7.0 y) from the date of first FDA approval, 17 (22%) had any pediatric information, and 8 (10%) had a pediatric indication. For these 17 drugs, the median time from first approval to addition of any pediatric information was 1.5 years (IQR: 1.1-4.6 y). Overall, 362 trials identified on ClinicalTrials.gov (total planned enrollment: 57,827 participants) were potentially open to children for 67 of the 78 drugs (86%), including 57 drugs (93%) with targets on the Pediatric Molecular Target List. For these 67 drugs, the earliest planned end date for trials open to children was a median of 3.3 years after first approval (IQR: 0.2-5.5 y). There were 171 solely pediatric studies for 56 (72%) drugs, and the earliest planned trial end was a median of 4.2 years (IQR: 2.3-5.9 y) after first approval.
Conclusions: Less than a quarter of FDA-approved cancer drugs include information on use in children, and most clinical trials open to children are not expected to be completed until several years after FDA approval. Under the RACE Act, most new drugs for adult cancers are likely to be subject to pediatric study requirements, indicating that timely completion of such trials may substantially increase the pediatric data available for cancer therapies.
Citation Format: Thomas J. Hwang, Liat Orenstein, Steven Dubois, Florence Bourgeois. Pediatric trials and labeling information for newly approved cancer therapies with targets potentially relevant to pediatric cancers [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2019; 2019 Mar 29-Apr 3; Atlanta, GA. Philadelphia (PA): AACR; Cancer Res 2019;79(13 Suppl):Abstract nr 3365.