US WorldMeds’ Neuroblastoma Drug: External Controls, Confirmatory Evidence, And A Concern About Precedent

12 Oct 2023

US FDA officials said the high-quality nature of the patient-level data used in the external control arm, and the use of animal models considered to be translatable, justify reliance upon a nontraditional data package for eflornithine (DFMO) in high-risk neuroblastoma, but some adcomm members worry that an approval will open the door too widely for others to follow.

Ski slope — Some ODAC members worried about the slippery slope effect of an approval based on a single, externally controlled trial. • Source: Shutterstock

The US Food and Drug Administration appears comfortable with the idea of approving US WorldMeds, LLC’s new drug application for eflornithine (DFMO) in high-risk neuroblastoma based upon the results from an externally controlled trial, with animal data as confirmatory evidence.

Such an approval would be groundbreaking. FDA review staff said the agency has not previously relied upon a single, externally...