New FDA GuidanceThe FDA has incorporated in the new Guidance the requests they made made when Theralase asked the FDA for feedback on the BTD application they are planning.
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"During the conduct of a clinical trial, patients with BCG-unresponsive NMIBC should be followed every 3 months for 2 years, then every 6 months for 2 years, and then annually with cystoscopy, directed biopsies, and urine cytology." page 9 .........................................
"Sponsors should use central pathology review of biopsy specimens and/or cytology for all patients in single-arm trials." page 9
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"For single-arm trials of patients with BCG-unresponsive NMIBC with CIS that use CR rate as the primary endpoint, the lower bound of the 95 percent confidence interval around the observed response rate should rule out a clinically unimportant CR rate. The median duration of CR is also important. A high CR rate is not meaningful if the response duration is short. The sponsor should discuss with the appropriate review division the minimum duration of response prior to the time of NDA or BLA submission. Patients participating in the trial should continue to be followed for the development of a CR and for duration of CR." pages 9-10