The RACE for Children Act Now in Full Effect
Today is a landmark day for the childhood cancer community: The RACE for Children Act is now fully implemented!
This legislation was signed into law two years ago and is undergoing a thoughtful and deliberative implementation process, with today’s new rules marking an important milestone toward the ultimate goal of accelerating early pediatric evaluation of molecularly targeted cancer drugs.
Background: The 2017 passage of a larger FDA user fees bill included provisions from the RACE Act, which improves opportunities for more studies in childhood cancer by amending the Pediatric Research Equity Act (PREA). PREA - and its partner legislation known as BPCA - provided critically important information on the safe and effective use of medications in the pediatric population. But these laws had a very modest impact on childhood cancer. In fact, exemptions carved out resulted in PREA having virtually no effect in stimulating new treatments for childhood cancer. The RACE Act eliminated those exemptions - paving the way for a sharp increase in the development of novel targeted therapies.
Getting drug companies to test targeted therapies in kids has been a goal for Children’s Cancer Cause for nearly twenty years.
RACE Act provisions hail from a blueprint report that grew out of a working group of the Alliance for Childhood Cancer and the Coalition Against Childhood Cancer in 2015-2016, co-chaired by our founder Susan L. Weiner, PhD.
Our work didn’t stop with passage of the resulting legislation. Children’s Cancer Cause has continued to be at the table, speaking up for our kids. In February, we submitted comments to the FDA Commissioner on the draft guidance for implementation. And in March, we held a webinar focused on the challenges and opportunities around RACE implementation, where we heard from experts how this will change the landscape of pediatric oncology drug development. This webinar addressed how FDA is structuring its regulations, how researchers are structuring trials, and how industry is adapting. The slide presentations are available here.
While this legislation certainly has the potential to substantially increase the number of required pediatric studies, including preclinical evaluations, some experts caution that this legislation is just one piece of a large, complex drug development puzzle.
Our founder Dr. Susan Weiner emphasizes the importance of ongoing collaborative work to maximize the potential for success at every step of the drug development process:
“Giving kids access to new precision cancer drugs depends first on preclinical research to determine whether the drugs are biologically relevant for children’s cancers. We vigorously support public private partnerships now in the works in the U.S. and Europe to carry out these early critical evaluations.”
There is much more work to be done, but today's achievement of full implementation of the RACE Act is something we can all celebrate together as a big victory for our kids. We thank Members of Congress for passing this legislation, the FDA for its careful implementation, and all of you for using your voices and your stories to highlight the need to bring new and better therapies to children battling cancer.