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The RACE for children act at one year: progress in pediatric development of molecularly targeted oncology drugs
1Department of Clinical Science, Regor Pharmaceuticals, Inc., Cambridge, MA, USA.
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.
Insights
The Research to Accelerate Cures and Equity (RACE) for Children Act effectively mandates pediatric trials for new molecularly targeted cancer drugs. Early data shows increased pediatric trial requirements, fostering innovation in pediatric cancer therapies.
Area of Science:
- Oncology
- Pediatric Medicine
- Regulatory Science
Background:
- The Research to Accelerate Cures and Equity (RACE) for Children Act of 2017 enables the FDA to mandate pediatric clinical trials for new oncology drugs targeting relevant molecular pathways.
- This legislation aimed to address gaps in pediatric cancer drug development.
Purpose of the Study:
- To evaluate the impact of the RACE Act on the development of molecularly targeted oncology drugs for pediatric cancers.
- Assessing changes in pediatric trial requirements post-RACE Act implementation.
Main Methods:
- Review of FDA oncology drug approvals between August 18, 2020, and August 18, 2021.
- Analysis of drug approval packages, letters, and prescribing information for submission/approval dates, indications, molecular targets, and pediatric trial requirements.
Main Results:
- Of 17 new oncology drugs approved, 5 were submitted after the RACE Act's effective date.
- 60% of post-RACE Act submissions required pediatric trials, compared to none of the pre-RACE Act submissions.
- 83.3% of pre-RACE Act submissions had molecular targets that would have qualified under the Act.
Conclusions:
- The RACE Act appears effective in closing legislative loopholes and promoting innovation for childhood cancer therapies.
- Early evidence indicates a positive impact on the development of targeted oncology drugs for pediatric use.
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