More Drugs Tested in Pediatric Cancer: RACE for Children Act

NEW ORLEANS ― Even within the first year of implementation, the Research to Accelerate Cures and Equity (RACE) for Children Act has made an impact.

"In the year prior to RACE implementation, there were no approvals of therapeutics that required pediatric studies," said Brittany Avin McKelvey, PhD, science policy analyst with Friends of Cancer Research and a childhood cancer survivor. "Within the first year of implementation, almost half of approved therapeutics required pediatric study."

The legislation was passed by Congress in 2017 and took effect in August 2020. It requires that therapeutics that are approved for adult cancers be tested in pediatric cancers if those drugs are directed at molecular targets relevant for pediatric cancers.

The RACE Act also requires testing of therapeutics that are given an orphan drug designation. Such drugs were previously exempt from pediatric trials.

The new findings were presented by McKelvey here at the American Association for Cancer Research (AACR) Annual Meeting 2022.

To evaluate the impact of the RACE Act during the first year of its implementation, her team assessed all the new cancer drugs approved between August 2019 and August 2021.

A total of 19 drugs were identified; 63.2% were approved in the year before the RACE Act took effect, and 36.8% were approved after its implementation. The team suspects that the coronavirus pandemic may have contributed to the lower number of post-RACE approvals.

The researchers found that prior to implementation of the RACE Act, none of the approved adult cancer therapeutics were required to be studied in pediatric populations. But more than 90% of those had molecular targets that would have required that they be studied in pediatric cancers had the RACE Act been in place. The majority of these drugs were exempt because of their designation as orphan drugs.

In the post-RACE group, however, 42.9% of approved drugs are required to be studied in pediatric cancers. One example is infigratinib (Truseltiq), a drug for adult cholangiocarcinoma that targets the protein fibroblast growth factor receptor 2 (FGFR2). Truseltiq is an orphan drug — and thus would have been exempt prior to the RACE Act — but it will now be studied in pediatric patients with advanced or metastatic tumors harboring alterations in FGFR2.

"I find these results encouraging, but it is still very early," said John Maris, MD, an attending physician and professor of pediatrics at the Children's Hospital of Philadelphia in Pennsylvania, who was not involved in the study. "This is only 1 year into implementation, and this legislation will be around for a long time."

In an interview, McKelvey noted that although a handful of clinical trials for pediatric cancers have been launched since implementation of RACE, many drugs are still being waived for pediatric study even when a relevant mechanism of action is present — largely because the extremely low incidence of many childhood cancers makes it impractical or, in some cases, impossible to conduct such studies. "This highlights the need for additional opportunities to help facilitate and encourage robust pediatric studies," she commented.

The main limitation of the study is that it only examined data 1 year after the implementation of the RACE Act; further analysis is needed to determine the full extent of its impact, McKelvey said. "The true measure of success will be determined by whether increased pediatric studies actually translate to label expansions for pediatric patient populations and access to these therapies."

The study was supported by funding from Friends of Cancer Research.

American Association for Cancer Research (AACR) Annual Meeting 2022: Abstract PO.SHP01.01. Presented April 11, 2022.

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Cite this: Diana Kwon. More Drugs Tested in Pediatric Cancer: RACE for Children Act - Medscape - Apr 11, 2022.