Pediatric Cancer Dataset Gaps, FDA ‘High Bar’ Make Use Of External Control Arms Difficult

The ability to leverage real-world evidence as an external control in pediatric cancer drug trials currently is limited by the quality of the underlying datasets, including a lack of genomic and molecular marker information, and the US Food and Drug Administration’s high bar for use of such data to support regulatory decision-making.

In addition, European data privacy regulations could further complicate efforts to build external control arms using real-world data that are housed outside the US.