Cancer Drug Dose Optimization Benefits Include Better Pivotal Trial Retention, Fewer Clinical Holds
US FDA is asking oncology sponsors for randomized studies comparing at least two doses early in development; despite concerns about delaying approval, FDA and industry reps say approach will increase success of registrational trials and reduce likelihood of market withdrawal due to toxicity.
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239 Days: Oncopeptides' Myeloma Drug Pepaxto Comes Off Market Just Months After Accelerated Approval
Withdrawal announcement marks shortest interval ever from accelerated approval to market removal after OCEAN confirmatory trial showed an adverse overall survival trend and US FDA said a hypothesis-generating subgroup analysis would need to be borne out in another study.
FDA’s “Project Optimus” draft guidance on dose optimization is in the works. Until then, randomized dosing studies need to be an IND-level conversation.
FDA’s Rick Pazdur notes that while the agency recently allowed Amgen to conduct a key dose comparison study for its landmark KRAS inhibitor post-market it will be less willing to do this in the future, particularly in settings that are less refractory.