Children's clinical cancer trials: what needs to change to allow children access to new cancer drugs?

Abstract

Despite numerous advances with the incorporation of multimodal treatment, children with cancers have a major unmet need to access new drugs. Still, a reduced number of new targeted drugs is evaluated in pediatric patients and very few of them progress into late phase trials and clinical use. Changes required include: increased collaboration between all stakeholders, improved understanding of disease biology and its incorporation into early clinical trials, faster and more efficient early and late clinical trials, better incentives for pharmaceutical companies and improving access to new drugs across the globe.

Keywords: Phase I; childhood cancer; children; clinical trials; drug development; pediatric medicines; targeted drugs.