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Can we improve the efficiency of early phase trials in pediatric oncology?

Esther Blanco & Darren Hargrave

Despite significant improvement in survival for childhood cancer, there remains an urgent need for novel therapies for poor risk cancers and to reduce the burden of treatment for survivors. We analyzed the efficiency of published early phase clinical trials over the past two decades and found a modest increase in the number of both trials and agents studied, with a significant move from cytotoxic chemotherapy to oral based targeted agents. However, there has been limited adoption of new design methodologies. We believe that combined Phase I/II studies with an initial doseescalation (proof of mechanism) phase followed by an integrated Phase II (proof of concept) to demonstrate direct clinical benefit could greatly improve the efficiency of early clinical trials in pediatric oncology.