RE:RE:RE:FDA guides on how AA can be converted to full approval.Furthermore, patient trial sizes may be smaller for those patients who have been identified by a biomarker .... ( so the Phase 3 breast cancer study may be much smaller than 200-300 patients as recently stated)- and as outlined in the FDA's draft guidance.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."