This month, Pediatrics is debuting a new section for “Health Policy” articles, which “provide policy-relevant, pediatric-focused data to inform decision-making by local, state, and federal policymakers; private and public insurers; and healthcare leaders.”
The first article for this new section is entitled “The RACE Act and Pediatric Trials of Adult Cancer Drugs,” by Ian Liu, MD, JD, MPH, MS, and Aaron Kesselheim, MD, JD, MPH, from Harvard Medical School (10.1542/peds.2024-066920).
The authors point out that, although the Pediatric Research Equity Act (PREA), which requires drug manufacturers to test all new drugs in pediatric patients before receiving approval, was passed by Congress in 2003, in reality this testing may not happen because these drugs are indicated for rare diseases (these drugs are exempt from pediatric testing) or for diseases that do not generally occur in children. For instance, most adult cancer drugs were exempted from pediatric testing until 2017, when Congress passed the Research to Accelerate Cures and Equity (RACE) for Children Act. Because many modern cancer drugs now target molecular pathways that may underlie both pediatric and adult disease, this testing is critical.
Drs. Liu and Kesselheim analyzed Food and Drug Administration (FDA) data to compare timing and other characteristics of pediatric trials of adult cancer drugs before and after the RACE Act. They found that drugs submitted for FDA approval after the RACE Act were more likely to have requirements for pediatric post-approval testing. Importantly, while pre-RACE Act cancer drugs started pediatric trials a median 0.04 years following FDA approval, post-RACE Act cancer drugs started these trials a median 2.8 years prior to approval.
The authors conclude that there have been important changes to pediatric testing of cancer drugs since the RACE Act was passed, and these changes will benefit pediatric cancer patients. They also note that the RACE Act may serve as a template for policies that may accelerate pediatric testing of other drugs.
