Advancing Clinical Trial Design for Non-Muscle Invasive Bladder Cancer

Elaine Chang, Noah M. Hahn, Seth P. Lerner, Jaleh Fallah, Sundeep Agrawal, Ashish M. Kamat, Vishal Bhatnagar, Robert S. Svatek, Adnan A. Jaigirdar, Peter Bross, Neal Shore, Max Kates, Karen Sachse, Jamie R. Brewer, Michael A. O'donnell, Gary D. Steinberg, Charles J. Viviano, Erik Bloomquist, Maria J. Ribal, Matthew D. GalskyRichard Oliver, Peter C. Black, Hikmat Al-Ahmadie, Kenneth Brothers, Kamal Pohar, Colin P. Dinney, Zhou Feng, Tracy M. Downs, Sima P. Porten, Angela B. Smith, Rick Bangs, Sarah P. Psutka, Neeraj Agarwal, Laleh Amiri-Kordestani, Daniel L. Suzman, Richard Pazdur, Paul G. Kluetz, Chana Weinstock

Research output: Contribution to journalArticlepeer-review

1 Scopus citations


BACKGROUND: Despite recent drug development for non-muscle invasive bladder cancer (NMIBC), few therapies have been approved by the US Food and Drug Administration (FDA), and there remains an unmet clinical need. Bacillus Calmette-Guerin (BCG) supply issues underscore the importance of developing safe and effective drugs for NMIBC. OBJECTIVE: On November 18-19, 2021, the FDA held a public virtual workshop to discuss NMIBC research needs and potential trial designs for future development of effective therapies. METHODS: Representatives from various disciplines including urologists, oncologists, pathologists, statisticians, basic and translational scientists, and the patient advocacy community participated. The workshop format included invited lectures, panel discussions, and opportunity for audience discussion and comment. RESULTS: In a pre-workshop survey, 92% of urologists surveyed considered the development of alternatives to BCG as a high drug development priority for BCG-naïve high-risk patients. Key topics discussed included definitions of disease states; trial design for BCG-naïve NMIBC, BCG-unresponsive carcinoma in situ, and BCG-unresponsive papillary carcinoma; strengths and limitations of single-arm trial designs; assessing patient-reported outcomes; and considerations for assessing avoidance of cystectomy as an efficacy measure. CONCLUSIONS: The workshop discussed several important opportunities for trial design refinement in NMIBC. FDA encourages sponsors to meet with the appropriate review division to discuss trial design proposals for NMIBC early in drug development.

Original languageEnglish (US)
Pages (from-to)271-286
Number of pages16
JournalBladder Cancer
Issue number3
StatePublished - Sep 25 2023


  • BCG
  • BCG-unresponsive
  • anti-neoplastic agents
  • clinical trial design
  • regulatory

ASJC Scopus subject areas

  • Oncology
  • Urology


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