Pediatric Trials for Cancer Therapies With Targets Potentially Relevant to Pediatric Cancers
- The Research to Accelerate Cures and Equity (RACE) for Children Act was enacted in 2017.
- The Act authorizes the US FDA to require pediatric studies for new cancer drugs with molecular targets relevant to pediatric cancers.
- An examination of 78 adult cancer drugs approved by the FDA from 2007 to 2017 showed that only 17 (21.8%) had any pediatric labeling information.
- Based on the FDA's Pediatric Molecular Target List, the RACE Act could have potentially increased the proportion of cancer drugs subject to pediatric study requirements from 0% to 78.2%.
- The actual effect of the legislation depends on the frequency of pediatric trial requirements and the timely completion of these trials.
In 2017, Congress enacted the Research to Accelerate Cures and Equity (RACE) for Children Act to stimulate the development of targeted therapies for pediatric cancers (1). The RACE Act closed a legislative gap that had exempted cancer drugs from mandatory pediatric studies.
Specifically, the RACE Act amended the Pediatric Research Equity Act (PREA), which ordinarily authorizes the US Food and Drug Administration (FDA) to require pediatric studies for new drugs, indications, dosage forms, and routes of administration in all relevant pediatric subpopulations (2–4). For sponsors that do not comply with PREA requirements, the FDA can determine that a drug is “misbranded” (5); the FDA can also publicly post noncompliance letters for overdue PREA studies...
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