Research to Accelerate Cures and Equity (RACE) for Children Act
The small population of children with cancer traditionally has provided little market incentive for the biopharmaceutical industry to develop new pediatric oncology drugs. Childhood cancer remains the leading cause of disease-related death in our children, and new and better therapies are sorely needed for children battling cancer.
In 2002, Congress passed a pair of laws - the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) - that provide critically important information on the safe and effective use of medications in the pediatric population. While BPCA and PREA yielded important new safety and labeling information for other children’s diseases, the laws had a very modest impact on childhood cancer. In fact, exceptions carved out have resulted in PREA having virtually no effect in stimulating more treatments for childhood cancer.
The RACE Act aims to eliminate those exemptions and improve opportunities for more studies in childhood cancer by:
Requiring that companies developing cancer drugs do PREA studies of their drugs in children when the molecular target of their drug is relevant to a children's cancer; and
Ending the exemption of PREA obligations for cancer drugs with orphan designations if the molecular target of their drug is relevant to a children's cancer.
On August 19, 2017 President Trump signed the RACE Act into law, a hard-fought achievement celebrated by the childhood cancer community. RACE Act provisions hailed 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. Childhood cancer stakeholders - including the Children's Cause - were involved in intensive bipartisan, bicameral negotiations with Congressional champions to make these recommendations a reality.
The RACE Act took full effect in August 2020, an important milestone toward the ultimate goal of accelerating early pediatric evaluation of molecularly targeted cancer drugs. The RACE Act has the potential to substantially increase the number of required pediatric studies, including preclinical evaluations, although some experts caution that it 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.”
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.
Blog Update, February 8, 2023: First Two Years of RACE Act Evaluated in New GAO Report
Health Policy Study from Pediatrics, September 4, 2024: The RACE Act and Pediatric Trials of Adult Cancer Drugs (Ian T. T. Liu, MD, JD, MPH, MS; Aaron S. Kesselheim, MD, JD, MPH)