FDA Panel Will Have To Weigh Pediatric Cancer Drug’s ‘Higher Level’ Of Uncertainty Against Rare, Devastating Disease
US WorldMeds NDA package for eflornithine for high-risk neuroblastoma provides “a higher level of uncertainty than is typically seen in oncology applications,” agency says. Though FDA seems largely satisfied with the sponsor’s externally controlled study, confirmatory evidence may be lacking.
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US WorldMeds’ Neuroblastoma Drug: External Controls, Confirmatory Evidence, And A Concern About Precedent
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.
In 14-6 vote, advisory committee said use of external control comparison to contextualize single-arm trial data demonstrated efficacy and reliance on preclinical data as confirmatory evidence was justified.
Draft guidance describes seven types of confirmatory evidence that can be used with one adequate and well-controlled clinical study to demonstrate substantial evidence of effectiveness, including evidence from expanded access, real-world data, and studies of other drugs in the same class.