Pediatric Cancer Studies: Early Results of the Research to Accelerate Cures and Equity for Children Act

What GAO Found

Pediatric cancer is the leading cause of death by disease past infancy among children from birth to age 14 in the United States, but there have been relatively few drugs approved to treat pediatric cancer.

As researchers have improved their understanding of cancer, more drugs have been developed for adult cancers that have a greater potential to also treat pediatric cancers. Recognizing this, and to facilitate the development of new, safe, and effective drugs to treat children, Congress passed, and the President signed into law, the FDA Reauthorization Act of 2017. This act includes a provision commonly known as the Research to Accelerate Cures and Equity for Children Act (RACE Act).

Specifically, the RACE Act requires drug sponsors that intend to apply for approval of certain adult cancer drugs to submit their planned approach for studying the drug in the pediatric population to the Food and Drug Administration (FDA). These studies are designed to yield clinically meaningful pediatric study data about the drug's safety, dosing, and preliminary efficacy to inform potential pediatric uses of the drug.

Early results indicate that the RACE Act has contributed to an increase in the number of planned studies to test certain adult cancer drugs in pediatric patients. Specifically, there have been 32 pediatric studies planned for adult cancer drugs from implementation of the RACE in August 2020 to August 2022, according to FDA data. Prior to the RACE Act, sponsors of most of these drugs would not have been required to conduct pediatric studies. This is because, for example, sponsors developing a drug for a rare condition or for an adult cancer not commonly found in children would not have been required to conduct pediatric studies prior to the RACE Act.

It is too soon to know if the RACE Act will increase the number of drugs approved to treat pediatric cancers. This is because, according to FDA officials, the planned studies are still in the early phases or have not yet begun, so the final study outcomes are not yet known. Additionally, once required RACE Act studies are complete, further studies to test safety and effectiveness may be needed before a drug can be approved for use in pediatric populations. However, FDA officials and nine of the 14 non-federal stakeholders GAO interviewed—including researchers, patient advocates, and drug manufacturers—stated that they expect the RACE Act to have positive effects in furthering pediatric cancer research and drug development. For example, two stakeholders stated that the law had already had positive effects on increasing attention to pediatric cancer drug development.

Enrolling patients in pediatric cancer studies is a long-standing challenge due to limited patient populations, which could be further exacerbated by an increase in planned pediatric studies under the RACE Act, according to FDA officials and six stakeholders GAO interviewed. FDA has taken a number of steps intended to help address this potential challenge. For example, FDA has issued guidance for industry discussing innovative study designs—such as enrolling pediatric patients in a cohort for already existing adult studies.

Why GAO Did This Study

The FDA Reauthorization Act of 2017 includes a provision for GAO to review the effectiveness of the pediatric study requirements enacted by the RACE Act. This report describes the effects of the RACE Act requirements for pediatric studies.

To do this work, GAO reviewed data provided by FDA on certain adult cancer drugs with pediatric study plans starting from the implementation of the RACE Act (August 18, 2020) through August 18, 2022. GAO also conducted interviews with FDA officials and a judgmentally selected sample of 14 nonfederal stakeholders. These 14 were selected to include a broad range of perspectives and include pediatric cancer advocacy groups, researchers, industry groups, and drug sponsors.

For more information, contact Mary Denigan-Macauley at (202) 512-7114 or deniganmacauleym@gao.gov.