FDA's ODAC Votes in Favor of Drug to Reduce Neuroblastoma Relapse Risk
The Food and Drug Administration (FDA) made a precedent-setting decision last week when its Oncologic Drug Advisory Committee (ODAC) voted in favor of a new drug to reduce relapse risk in children with high-risk neuroblastoma who are in remission. The 14-6 vote was particularly notable because it is the first time the FDA has approved a new oncologic agent based on an externally controlled trial rather than on a randomized phase 3 clinical trial.
The Committee determined that DFMO (eflornithine hydrochloride), produced by US WorldMeds, showed sufficient evidence of patient benefit to outweigh its risks. Children who received DFMO for two years after the completion of their neuroblastoma treatment experienced a higher rate of “event-free survival” (i.e., their cancer did not return) than the historical relapse rate seen in this population (85% vs 70%). The small population size and the poor survival data made a Phase 3 randomized trial infeasible, according to Advisory Committee members, and gave the Agency flexibility to “consider an external control design in this circumstance,” according to FDA’s Diana Bradford, MD.
Committee members expressed concern that the externally controlled clinical trial comparison might loosen FDA’s standards to approve agents without the gold standard comparison that a randomized Phase 3 trial can provide. However, members complimented both the company and the FDA for their careful analysis of the data presented at the meeting.
Neuroblastoma is a very rare cancer seen mostly in infants and very young children, with approximately 700-800 new diagnoses in the U.S. every year. Children with high-risk neuroblastoma have among the lowest survival rates of pediatric cancers, especially after relapse. This unmet medical need was a major factor in the committee’s approval of DFMO.
Learn more about DFMO and the Committee’s decision: