Abstract
There are few approved drugs available for the treatment of patients with non-muscle invasive bladder cancer (NMIBC) and none have been approved in the twenty-first century. Four drugs; thiotepa in 1959, BCG Tice in 1989, BCG Connaught in 1990, and valrubicin in 1998, have been approved for the treatment of NMIBC. In addition to these four agents, mitomycin is commonly used off-label as an intravesical treatment for NMIBC. New drugs are needed for the management of NMIBC. This article outlines important aspects of the design and conduct of clinical trials to develop new therapies for these patients and to obtain marketing approval. It includes a discussion of the patient population, BCG-unresponsive disease, and the appropriate endpoints for drug approval. It is hoped that this article will spur drug development in NMIBC within the Center for Drug Evaluation and Research at the Food and Drug Administration.