The RACE for Children Act, Explained for Parents

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Written By Dina Besic

If you spend time in pediatric cancer advocacy, you'll hear a specific piece of legislation come up over and over. The RACE for Children Act.

Most cancer parents I know have heard the name and don't have a clear sense of what it does. This post is the plain-language version.

The one-sentence summary

The RACE for Children Act, passed as part of the FDA Reauthorization Act of 2017, requires certain adult cancer drug developers to plan pediatric cancer studies for their drugs when the drug's molecular target is relevant to a pediatric cancer.

RACE stands for Research to Accelerate Cures and Equity for Children.

Why the RACE Act exists

Before the RACE Act, adult cancer drug developers had no requirement to test their drugs in pediatric populations. Historically, most new cancer drugs were developed for adult cancers, approved for adults, and then, if pediatric use ever happened, it was often years later and sometimes only in off-label or trial form.

This was a problem for two reasons. First, some adult cancer drugs work on molecular targets that are also present in certain pediatric cancers. If a targeted drug is developed for adult non-Hodgkin lymphoma and that drug hits a specific molecular target, and if that target is also present in a rare pediatric cancer, the drug could plausibly help children with that pediatric cancer. Without a requirement to test in pediatrics, that connection often went unexplored for years.

Second, the historical framework was based on clinical indication rather than molecular mechanism. A drug approved for a specific adult cancer wasn't necessarily tested in pediatric cancers with similar molecular biology, because pediatric cancers have different clinical presentations even when the underlying biology overlaps.

The RACE Act was designed to close this specific gap.

What the RACE Act does, mechanically

For new drug applications submitted to the FDA on or after August 18, 2020 (the effective date of the RACE Act's key provisions), the sponsor of an adult cancer drug must submit a Pediatric Study Plan if:

  • The drug is intended for the treatment of an adult cancer, and

  • The drug is directed at a molecular target that the FDA determines to be substantially relevant to the growth or progression of a pediatric cancer.

The Pediatric Study Plan lays out how the sponsor will study the drug in pediatric populations. The FDA reviews the plan and can require modifications. In some cases, the FDA can grant a deferral (allowing the pediatric studies to happen after the adult approval) or a waiver (in specific circumstances where pediatric studies are not appropriate).

This is a significant departure from the historical framework, which exempted adult cancer drugs from pediatric testing requirements altogether.

What the RACE Act does not do

This is where the RACE Act is often misunderstood.

It does not create new funding for pediatric cancer research

The RACE Act shifts the regulatory framework. It does not appropriate new NIH or NCI dollars for pediatric cancer research. The 8 percent federal funding figure is a separate policy issue that requires separate legislative action.

It does not cover all pediatric cancers

The RACE Act applies specifically to adult cancer drugs with molecular targets relevant to pediatric cancers. Pediatric cancers without an adult-drug analog are not directly helped by RACE. Rare pediatric cancers with unique biology still face the pre-RACE research challenge.

It does not guarantee approved pediatric drugs

A Pediatric Study Plan doesn't guarantee that the studies will succeed, or that the drug will end up approved for pediatric use. It requires the sponsor to plan and conduct studies. What comes out of those studies depends on the drug and the science.

It does not shorten pediatric drug approval timelines

The RACE Act adds required studies to the drug development process. In some cases, the pediatric studies can happen in parallel with adult studies. In other cases, they happen after. The overall timeline from drug discovery to pediatric availability is still typically years.

What the RACE Act has done so far

The RACE Act took effect for new drug applications submitted on or after August 18, 2020. Since then, the FDA has been issuing guidance on how sponsors should plan pediatric studies, and drug sponsors have been submitting Pediatric Study Plans as part of new adult cancer drug applications.

A 2023 U.S. Government Accountability Office report noted that early implementation has generated a meaningful increase in pediatric study planning across adult cancer drug submissions. The full impact on pediatric drug approvals will play out over years, as these studies complete and drugs move through the pediatric approval pathway.

The pediatric cancer research community is broadly positive about the direction of the RACE Act while continuing to advocate for stronger enforcement, additional funding for pediatric clinical trials, and companion legislation to fill the gaps RACE doesn't cover.

What families and advocates can do about the RACE Act

1. Support full enforcement

The RACE Act is only as strong as its enforcement. Advocacy organizations track how the FDA is implementing it and press for stronger enforcement where sponsors seek waivers or deferrals inappropriately.

Supporting organizations like the Alliance for Childhood Cancer and ACCO, both of which are active on RACE Act implementation, helps this work.

2. Push for expanded scope

There is ongoing conversation about whether the RACE Act's scope should be expanded to cover additional drug categories or additional pediatric cancer types. Family voice in these conversations matters.

3. Support funding for pediatric clinical trials

RACE requires studies. Studies need infrastructure. NIH and NCI funding for pediatric clinical trial infrastructure is essential to making RACE work in practice. This is a place where federal funding advocacy connects directly to RACE Act effectiveness.

4. Watch for companion legislation

Additional pediatric cancer legislation continues to be proposed and considered in Congress. Sign up for advocacy alerts from the Alliance for Childhood Cancer or ACCO to know when to weigh in on companion bills.

The bigger picture

The RACE Act was a family-advocacy win. It took years of pediatric cancer families and clinicians pushing Congress to change the regulatory framework in a way that adult cancer drug developers had resisted for a long time.

It is one of the reasons I believe advocacy works, even when it takes longer than we'd like. The RACE Act became law because thousands of families over more than a decade made pediatric drug testing a policy issue that Congress could not keep ignoring.

The next fights are similar in shape. Federal research funding. Rare pediatric cancer investment. Survivorship program funding. Every one of these is winnable if the family community keeps showing up.

For the family reading this

You do not need to become a policy expert to be an advocate. You need to know one or two facts, tell your representatives, and keep showing up.

The RACE Act is a good example of what your voice can do when you use it consistently over time.

Dina
Mom of Max | Founder, Maxwell’s Toy Shoppe
Childhood Cancer Advocate 💛

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