Why childhood cancers get so few new drugs, and the law trying to fix it
Childhood cancers are biologically distinct from adult ones, and for years that let new cancer drugs bypass children entirely. The RACE for Children Act changed the rules.
In brief
Why childhood cancers get few new drugs, and how the RACE for Children Act requires paediatric studies based on a drug’s molecular target.
Survival from childhood cancer has improved dramatically over the past half century, mostly through better use of old chemotherapy drugs. New drugs have been scarce. For a long time, most targeted cancer therapies were approved for adults without ever being studied in children.
The loophole
Under the Pediatric Research Equity Act, paediatric studies were tied to the indication a drug was approved for. Many adult cancers, such as lung or breast cancer, rarely or never occur in children. A drug for adult lung cancer could therefore receive a waiver, even if its molecular target was relevant to a childhood cancer.
RACE for Children
The RACE for Children Act, passed in 2017 and in effect from August 2020, closed that gap. It requires paediatric investigation of new cancer drugs when their molecular target is substantially relevant to the growth or progression of a paediatric cancer, regardless of the adult indication. The FDA maintains lists of molecular targets considered relevant to childhood cancers to guide these decisions.
The European Union is moving in a similar direction as part of its pharmaceutical reform, with paediatric plans based on mechanism of action.
Why biology matters
Childhood cancers are often driven by different events from adult cancers: fewer mutations overall, more gene fusions and developmental pathways gone wrong. That makes some targeted drugs highly relevant, such as those against particular fusion proteins, and others irrelevant.
Tumour-agnostic approvals have shown the potential. Larotrectinib, approved in 2018 for solid tumours with NTRK gene fusions in adults and children, demonstrated that a drug defined by its target rather than its tumour type could reach children from the start.
What still holds things back
Small patient numbers, the need for age-appropriate formulations and the long-term toxicity of treatments in growing children all remain. Collaborative platforms that bring together academics, industry, regulators and parents aim to prioritise which drugs to test first, so that limited patient numbers are used where they matter most.
