RACE Act Poised to Advance Pediatric Cancer Research

On August 18, the Research to Accelerate Cures and Equity (RACE) for Children Act went into effect, potentially paving the way for more drug development in pediatric cancers. The legislation will tighten the requirements for testing cancer agents used in adults to treat malignancies in children when there is a shared molecular target. “Those of us who do drug development and research in the field of childhood cancer really hope and expect that the RACE Act will be a game-changer,” says John Maris, MD, of Children's Hospital of Philadelphia in Pennsylvania.

In 2003, the Pediatric Research Equity Act (PREA) authorized the FDA to require studies in children for therapies developed for adults. However, drug developers often received exemptions for testing oncology agents in children because pediatric cancers are rare and tend to occur in different organs.

But with the advent of genomics in cancer research—and the development of agents that target specific molecular alterations—the field began to change. “Since we've recognized that diseases can be molecularly targeted, it's brought forth some new principles, and PREA didn't quite take into account what we know about cancer biology now,” explains Stacey Berg, MD, of Texas Children's Hospital and Baylor College of Medicine in Houston. Specifically, researchers now understand that the same molecular change may make a single treatment relevant in both populations, even if the change causes different cancers.

The RACE Act, passed in 2017, updates PREA by allowing the FDA to require trial sponsors to test molecularly targeted cancer agents developed for adults in children if that same target is present, even if the malignancy occurs in a different organ. The act also eliminates exemptions for testing cancer drugs in rare diseases. “The RACE Act is not a brand-new concept, it's a clarification that makes PREA apply in a sensible way to the current state of oncology drug development,” Berg says.

In 2018, the FDA published a Pediatric Molecular Target List that offers guidance on possible targets (available at www.fda.gov). A 2019 paper concluded that for the 78 cancer agents approved between 2007 and 2017, the RACE Act could have increased drug testing in children from 0% to 78.2% (J Natl Cancer Inst 2020;112:224–8).

Maris says that the challenge is figuring out which agents may be effective. “There has to be collaboration in adjudicating available genomic, proteomic, and preclinical data in order to prioritize which drugs should be moved forward into clinical testing in kids,” Maris says. To this end, the NCI and the Foundation for the NIH established the Pediatric Preclinical Testing Public-Private Partnership for the RACE Act, which includes pharmaceutical companies, the NCI and the FDA, and academia.

Maris adds that the pediatric cancer community has gathered large quantities of detailed clinical and preclinical data on childhood cancers, much of which is not organized in an accessible format. Thus, he sees an opportunity to coordinate the response to the RACE Act with the NCI-led Childhood Cancer Data Initiative (CCDI). “There isn't a place right now where you could go to a single web portal and say, ‘I have a drug that hits this target and I want to know if it's expressed in childhood cancers,’” Maris explains. “There's a hope that we can leverage the CCDI to put together the computational infrastructure to support the RACE Act.”

Although it isn't clear how the RACE Act will affect pediatric cancer research overall, for Berg the bottom line is simple: “It increases the ability to bring potential new anticancer agents to children—and ultimately we hope it leads to more cures.” –Catherine Caruso

For more news on cancer research, visit Cancer Discovery online at http://cancerdiscovery.aacrjournals.org/CDNews.