Clinical trial design in non-muscle invasive bladder cancer should depend on whether patients have active disease at the time of enrollment, the FDA said, in a draft guidance that focuses on developing drugs and biologics for disease that has not responded to standard bacillus Calmette-Guerin immunotherapy.
The preferred design for late-phase clinical trials of patients without active disease is a randomized, controlled trial that uses an endpoint of recurrence-free survival, the guidance said. Sponsors should consider randomization by study site, and the effects of individual urologists or investigator sites on outcomes should be examined.
But for active disease, a single-arm trial would also be acceptable. A successful single-arm trial measuring complete responses, not partial responses, could support a marketing application, the agency said. Median duration of response should also be measured. The FDA recommended follow-up with cystoscopy, directed biopsies and urine cytology every three months.