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

 
• By Sue Sutter

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 controlled trial to support approval in oncology

Key Takeaways

  • US WorldMeds seeks approval of DFMO in neuroblastoma based on a single-arm trial compared with an external control arm, with confirmatory evidence coming from preclinical animal models.

  • FDA has not previously relied upon a single externally controlled trial to support approval in oncology, and consideration of animal model data in the context of confirmatory evidence is unusual

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