Background: The Research to Accelerate Cures and Equity (RACE) for Children Act of 2017 authorized the Food and Drug Administration (FDA) to require pediatric clinical trials for new oncology drugs with relevant molecular targets. This study reviewed oncology drug approvals within the first year after the new legislation came into effect, to evaluate the impact on development of molecularly targeted oncology drugs for pediatric cancers.
Research Design And Methods: For new oncology drugs approved by the FDA between 08/18/2020 and 08/18/2021, drug approval packages, letters, and prescribing information were reviewed for the submission and approval dates, indication, and molecular target of the drug, and post-marketing requirements that included pediatric clinical trials.
Results: Within the 1-year period, 17 new oncology drugs were approved, but only 5 had been submitted after 08/18/2020. Three of the 5 (60.0%) had requirements for pediatric trials under the RACE Act. None of the 12 submitted prior to 08/18/2020 had pediatric trial requirements, but 10 (83.3%) had molecular targets that would have made them candidates under the RACE Act.
Conclusions: Early evidence suggests that the RACE Act was effective at closing the loopholes of previous legislation and creating new opportunities for innovation in developing therapies for childhood cancers.
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