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 hear more podcasts, subscribe to us on Apple Podcasts, Spotify, or wherever you listen and make sure to leave a rating and review to let others know about the work we're doing. For more from the congressional watchdog, the US Government Accountability Office, visit us at GAO.gov