Title: Efforts to Promote Pediatric Cancer Research

Description: Pediatric cancer is among the leading cause of death by
disease among children in the United States. But there have been
relatively few new drugs approved for treatments. What are the efforts
to expand research for developing new treatments? We find out from GAO's
Mary Denigan-Macauley.

Related GAO Work: GAO-23-105947, Pediatric Cancer Studies: Early Results
of the Research to Accelerate Cures and Equity for Children Act 

Released: January 2023

[Music]

[Mary Denigan-Macauley] The good news is that there have been more
planned pediatric studies, and one would hope that some treatments will
come out of that.

[Holly Hobbs:] Hi, and welcome to GAO's Watchdog Report. Your source for
news and information from the U.S. Government Accountability Office. I'm
your host, Holly Hobbs. Pediatric cancer is among the leading cause of
death by disease among children in the United States. Last year alone,
about 10,000 new cases of cancer were diagnosed in children. While
survival rates have improved, some pediatric cancer survival rates
remain low and there have been relatively few new drugs approved for
treatments. Today, we'll talk with GAO's Mary Denigan-Macauley about
recent efforts to expand pediatric cancer treatment options and improve
survival rates. Thanks for joining us.

[Mary Denigan-Macauley:] Thanks for having me, Holly.

[Holly Hobbs:] So, Mary, it's devastating news when families get a
cancer diagnosis for their children. Why are there so few drug treatment
options for pediatric cancers?

[Mary Denigan-Macauley:] You're right, Holly. It is devastating. And as
a parent, I can't begin to imagine how I'd feel if my son was diagnosed
with a brain tumor that wasn't treatable. There are a number of reasons
why there are so few treatments, but most importantly is that most
cancers occur in adults. And so that's where the research is really
focused.

[Holly Hobbs:] So dumb question, but what's the difference between like
a child cancer treatment and an adult cancer treatment?

[Mary Denigan-Macauley:] It's not a dumb question at all. What's most
important is that developing drugs for children is different than it is
for adults. As the oncologists that we interview liked to say, they're
not just mini-adults. And so this means that there are different risks
that need to be taken into consideration. So, for example, if I'm 85
years old with cancer, I might think differently about the risks that
I'm willing to assume to help to overcome that particular cancer and the
treatment options that I'll choose. If a child is 5 years old and has
their life ahead of them, then the risks might be different. And so you
have to weigh those risks. The other thing is that children can't give
consent and it's a family decision. So that's something else that needs
to be taken into consideration.

[Holly Hobbs:] So what's being done to promote new drug treatments?

[Mary Denigan-Macauley:] So what we looked at was a law that came into
effect in 2020 called the RACE Act. And the purpose really was to
leverage potential adult cancer treatments that might also work in
children. And the law specifically says that if you have a drug that may
help treat cancers in children, then the researchers need to do
pediatric clinical trials—even if that drug is for a cancer that kids
don't really get. So, for example, if there was a drug that was being
developed for cervical cancer, but that drug potentially has the ability
to work in a child's cancer, then there’re pediatric trials have to be
done.

[Holly Hobbs:] So has this effort worked? Has the RACE Act resulted in
more drug treatment options?

[Mary Denigan-Macauley:] So in terms of the drug treatment options, it's
really just too early to say. Drug development can take a long time. It
will be years before we really know the full impact of this new law. The
good news, though, is that there have been more planned pediatric
studies, and one would hope that some treatments will come out of that.

{MUSIC}

[Holly Hobbs:] So Mary just told us that Congress approved the RACE Act
to promote pediatric cancer research, calling on adult-cancer treatment
researchers to include children when there could be benefits for kids.
But that we don’t yet know the impact of this law. So, Mary, we talked
to a number of stakeholders on this issue, including researchers. What
did they tell us needs to happen to better promote pediatric cancer
research?

[Mary Denigan-Macauley:] So the stakeholders we spoke with are very
optimistic and they're excited about the potential for new treatment. At
the very least, it's begun a dialogue, right? Now we're having a
dialogue, we’re creating potentially new treatments for children. So
first and foremost, that's very important. On the more challenging side,
it's difficult to find the number of children to be able to enroll in
the clinical trials. So on the positive note, it's great that we don't
have that many children that enrollment is really high, but you do need
a certain amount to be able to conduct these clinical trials. And so
they are coming up with innovative ways to overcome this particular
challenge to make sure that the trials are still good.

[Holly Hobbs:] And last question, what's the bottom line of this report?

[Mary Denigan-Macauley:] So the bottom line is that there are some
efforts underway. It's too early to know how effective the RACE Act will
be on actually developing treatments because drug development does take
a while. But they are working diligently to see what they can do to help
save these kids.

[Holly Hobbs:] That was Mary Denigan-Macauley discussing GAO's recent
review of pediatric cancer research. Thanks for your time, Mary.

[Mary Denigan-Macauley] Thanks for having me, Holly.

[Holly Hobbs:] And thank you for listening to the Watchdog report. To
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