Oncology NDAs Based On Single-Arm Studies Run Risk Of FDA Refusal To File
This article was originally published in Pharmaceutical Approvals Monthly
Oncology drug sponsors who seek FDA accelerated approval based upon single-arm studies run the risk of a “refuse-to-file” letter, a new analysis suggests.
You may also be interested in...
Positive Phase III findings support Novartis’ plan for filing the pan-DAC inhibitor in 2014. The firm’s initial BLA for the compound, seeking accelerated approval in Hodgkin’s lymphoma, was rejected by FDA in 2011.
A pivotal trial with a control arm was necessary to evaluate a hepatotoxicity signal with Genentech’s Kadcyla, helping to derail a bid for accelerated approval based on a single-arm Phase II trial. Genentech’s strategy of running trials in different lines of treatment concurrently enabled a quick strategy shift as an ongoing controlled Phase III trial in another line was amended to meet FDA’s regulatory requests.
Response rates of 30%-43% and a 7.6-month median duration of response were sufficiently clinically meaningful to allow FDA approval of Genentech’s basal cell carcinoma treatment on the basis of a non-randomized study.