Fewer cancer drugs exempt from pediatric testing after RACE Act

Key takeaways:

• No drugs approved between 2017 and 2020 were subjected to post-approval pediatric testing requirements.
• After the RACE Act went into effect, 38% of drugs had post-approval testing requirements waived.

Fewer cancer drugs were exempt from pediatric testing requirements after the Research to Accelerate Cures and Equity, or RACE, for Children Act went into effect in 2020, a study showed.

Post-RACE Act pediatric drug trials started and ended earlier than before the legislation was implemented, according to findings published in Pediatrics.

Congress passed the Pediatric Research Equity Act (PREA) in 2003, requiring manufacturers to test drugs in children before approval, but many studies had this requirement waived for a variety of reasons, the researchers wrote. In 2017, the federal government passed the RACE Act, which removed exemption criteria to ensure more cancer drug trials included pediatric populations. The FDA began enforcing the legislation on Aug. 18, 2020.

“We analyzed a cohort of cancer drugs approved between 2017 and 2024, comparing post-approval requirements, pediatric trials and pediatric trial characteristics among molecularly targeted adult cancer drugs before the RACE Act with pediatric trials in similar drugs after the law’s implementation,” Ian T.T. Liu, MD, JD, MPH, MS, and Aaron S. Kesselheim, MD, JD, MPH, from the Program on Regulation, Therapeutics and Law in the division of pharmacoepidemiology and pharmacoeconomics at Brigham and Women’s Hospital and Harvard Medical School, wrote.

Liu and Kesselheim investigated 61 adult cancer drugs, 40 of which were submitted to the FDA for approval before Aug. 18, 2020, and 21 that were submitted after.

None of the drugs submitted before the RACE Act’s effective date were required to conduct pediatric testing. Eleven (28%) had the requirements waived because the low number of children with the disease made testing “impossible or highly impracticable,” and 29 (73%) had rare disease designations and were automatically exempt.

After the RACE Act, 13 (62%) drugs had post-approval pediatric testing requirements, whereas eight drugs (38%) received waivers due to “impossible or highly impracticable” pediatric populations. The researchers noted that 16 drugs had rare disease designations but were not automatically exempt like they would have been before the RACE Act.

There were 58 pediatric trials associated with the drugs approved before the RACE Act. Pediatric trials started a median of 0.04 years after approval (interquartile range [IQR], 3.3 years before to 1.9 years after), and trials concluded a median of 5.4 years after FDA approval (IQR, 1.4-7.7 years).

Among the 21 drugs approved after the RACE Act, there were 19 pediatric trials. Trials began a median of 2.8 years before FDA approval (IQR, 4.3-0.3 years before), and trials ended a median of 3.3 years after approval (IQR, 0.6-4.3 years).

“The RACE Act was designed to close a loophole in PREA,” the researchers wrote. “Compared with cancer drugs approved before the RACE Act, more recently approved molecularly targeted adult cancer therapies had more mandatory pediatric post-market studies and, despite shorter follow-up time, post-RACE Act drugs had similar pediatric testing rates as pre-RACE Act drugs.”