The Critical Role of Academic Clinical Trials in Pediatric Cancer Drug Approvals: Design, Conduct, and Fit for Purpose Data for Positive Regulatory Decisions.

Purpose: For decades, academic clinical trials consortia have collaborated to optimize outcomes for childhood cancers through evaluating incremental improvements in conventional mutimodality treatment regimes. There are now increasing opportunities to partner with industry to test new medicines in academic-sponsored trials, but these collaborative studies rarely contribute to marketing authorizations. We addressed why this is the case and sought solutions to enable academic-sponsored trials to directly contribute to the licensing of new medicines.