The RACE for children act at one year: progress in pediatric development of molecularly targeted oncology drugs

ABSTRACT

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.

KEYWORDS:

• Oncology drug
• pediatric cancer
• post-marketing study
• regulatory

Declaration of interest

ME Zettler is an employee of Regor Pharmaceuticals Inc. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper received no funding.