The purpose of this guidance is to assist sponsors in the clinical development of drugs, including biologics, for the treatment of patients who have bacillus Calmette-Guerin (BCG)-unresponsive nonmuscle invasive bladder cancer (NMIBC). The definition described by Lerner et al. 2015 is used to identify the patient population with BCG-unresponsive disease. This guidance is intended for pharmaceutical sponsors, the academic community, and the public and provides a framework, based on current Food and Drug Administration (FDA) thinking, to facilitate the development of drugs to treat this patient population. The pathological diagnosis and staging, risk stratification, and trial design, including assessment of appropriate clinical endpoints, are discussed. These issues were discussed at the Food and Drug Administration/American Urological Association Bladder Cancer Workshop held on May 6, 2013, and in more recent publications (Jarow, Lerner, et al. 2014; Jarow, Maher, et al. 2015).

Many of the general principles elucidated in this guidance also apply to development of drugs for other forms of nonmuscle invasive bladder cancer. Nevertheless, the specific recommendations for trial design and endpoints contained herein may not necessarily apply, and sponsors are encouraged to discuss development plans with the FDA for drugs intended to treat other forms of NMIBC or for muscle invasive, locally advanced, or metastatic bladder cancer.

This guidance does not contain discussion of the general issues of statistical analysis or clinical trial design. Those topics are addressed in the ICH guidances for industry E9 Statistical Principles for Clinical Trials and E10 Choice of Control Group and Related Issues in Clinical Trials, respectively.

Posted on the FDA website on 17 November 2016