The Research to Accelerate Cures and Equity (RACE) Act, implemented in 2020, has significantly impacted pediatric cancer drug development by reducing exemptions from post-approval pediatric testing requirements. A new study published in Pediatrics highlights that more recently approved molecularly targeted adult cancer therapies are now subject to mandatory pediatric post-market studies, ensuring greater inclusion of pediatric populations in cancer drug trials.
Impact of the RACE Act on Pediatric Cancer Drug Testing
Prior to the RACE Act, many cancer drugs were exempt from pediatric testing requirements due to various reasons, including rare disease designations. According to Ian T.T. Liu, MD, JD, MPH, MS, and Aaron S. Kesselheim, MD, JD, MPH, from Brigham and Women’s Hospital and Harvard Medical School, no drugs approved between 2017 and 2020 had mandatory pediatric testing requirements. A significant 73% of these drugs were automatically exempt due to rare disease designations.
The RACE Act, however, closed this loophole. Post-implementation, 62% of drugs had post-approval pediatric testing requirements. Only 38% received waivers due to impractical pediatric populations. Notably, 16 drugs with rare disease designations were no longer automatically exempt, marking a significant shift in regulatory approach.
Earlier Integration of Pediatric Trials
The study also found that pediatric trials are now being integrated earlier in the drug development process. Before the RACE Act, pediatric trials started a median of 0.04 years after FDA approval and concluded a median of 5.4 years after approval. In contrast, trials for drugs approved after the RACE Act began a median of 2.8 years before FDA approval and ended a median of 3.3 years after approval.
Legislative Context and Implications
Congress initially passed the Pediatric Research Equity Act (PREA) in 2003, mandating manufacturers to test drugs in children before approval. However, exemptions were common. The RACE Act amended PREA by removing exemption criteria, ensuring more comprehensive pediatric testing. The FDA began enforcing the RACE Act on August 18, 2020.
"The RACE Act was designed to close a loophole in PREA," the researchers wrote, emphasizing that the legislation has successfully increased mandatory pediatric post-market studies for molecularly targeted adult cancer therapies.
