Accelerating New Treatments for Pediatric Patients

Sometimes, our smallest cancer fighters are met with the biggest challenges, like enduring older, toxic treatments and experiencing persistent side effects that can last for years. Part of this is because regulators often approach kids’ treatment as if they’re tiny adults. But the biology of pediatric cancers is typically distinct from adults’, making targeted research critical.

The patient impact

Since cancer is more common among adults than children, the federal government and drug makers are more likely to invest in research on treatments for adults.

But the rarity of childhood cancer affects more than just research funding. It also means that aren’t always enough patients to fill clinical trials under traditional rules. And, childhood cancer researchers might not have access to the information they need because kids’ data is siloed and difficult to access.

Kids with cancer, then, are left with existing—rather than innovative—treatments that have long-term side effects. But they deserve treatment that is tailor-made for them.

The solution

To make real progress in fighting childhood cancer, we support policies that:

• Sustain and expand funding for research and infrastructure focused on childhood cancers at the National Institutes of Health and other federal agencies
• Secure federal funding for the infrastructure and standards that allow researchers to access and analyze existing data in real time
• Provide financial and regulatory incentives to drug companies that bring a pediatric cancer drug to market
• Urging the FDA to adopt “pediatric-specific guidance” to ensure that federal regulatory decisions are tailored to the biology of childhood cancers
• Partnering with the FDA to build clinical trial designs specifically for kids with cancer