— Large enhancement in event-free survival, however concerns remain about sources of information
by Charles Bankhead, Senior Editor, MedPage Today
October 4, 2023
After wrangling throughout the day over semantics and a prospective precedent-setting dataset, an FDA advisory committee voted to advise approval of eflornithine (difluoromethylornithine; DFMO) upkeep for high-risk pediatric neuroblastoma in total remission after basic treatment.
By a 14-6 vote, the Oncologic Drugs Advisory Committee (ODAC) chose that the totality of proof provided sufficed to reveal that DFMO upkeep enhanced 2-year event-free survival (EFS). The proof consisted of a single-arm potential scientific trial with an external control group, supplemented by numerous smaller sized medical research studies and convincing preclinical information.
“I believe the basic agreement is that there’s a significant quantity of problem translating this sort of information in an uncommon illness and genuine gratitude of the panel for all the work that entered into this extremely robust analysis,” stated ODAC chair Christopher Lieu, MD, of the University of Colorado Cancer Center in Aurora. “There is basic contract that the totality of the information appear to support the assertion that DFMO does enhance event-free survival, or a minimum of that the outcomes were most likely than not to be something more than simply the outcome of opportunity.”
“There are clear locations of difference within the panel,” Lieu included. “That is whether this kind of information need to actually ever be utilized, offered the issue relating to confounders and predispositions that are simply fundamental to these external controls. There’s a lot of issue from the group about what the future holds for drug advancement and what level of proof the FDA will need in comparable scenarios in the future … There is likewise some dispute in this group about whether a randomized, managed trial is possibly