RE:RE:RE:RE:FDA guides on how AA can be converted to full approval.
March 24, 2023 "While Congress recently passed legislation (IRA) that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval." ....
and as outlined in the FDA's draft guidance.
"In cases wherein historical trials did not specifically evaluate the response rate for the standard of care treatment in a biomarker-selected population of interest (i.e., available therapy is approved for an all-comer population), assessing the new drug compared to the available therapy in the same trial provides a more accurate representation of the efficacy and safety of standard of care in the biomarker-defined cohort of patients"
"This “one-trial” approach maintains efficiency in drug development and can provide early access to a drug using the accelerated approval pathway, while ensuring that a postmarketing trial is fully accrued and well underwayto verify longer term benefit in a timely fashion."