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.
FDA officials sought to tamp down concerns that approval would encourage other sponsors to rush forward with externally controlled trials, saying the DFMO application is an unusual case involving high-quality, patient-level data and there remains a high bar for considering whether an external control data source is fit for purpose.

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

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