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Updated: Jun 21, 2025
An Orthotopic Bladder Cancer Model for Gene Delivery Studies
Published on: December 1, 2013
Advancing Clinical Trial Design for Non-Muscle Invasive Bladder Cancer
Elaine Chang1, Noah M Hahn2, Seth P Lerner3
1Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA.
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.
Insights
Developing new treatments for non-muscle invasive bladder cancer (NMIBC) is a priority. A workshop highlighted the need for better clinical trial designs to accelerate the approval of effective NMIBC therapies.
Area of Science:
- Oncology
- Urology
- Drug Development
Background:
- Non-muscle invasive bladder cancer (NMIBC) has limited FDA-approved therapies, creating an unmet clinical need.
- Bacillus Calmette-Guerin (BCG) supply shortages emphasize the urgency for alternative NMIBC treatments.
Purpose of the Study:
- To discuss research needs and clinical trial designs for NMIBC therapies.
- To foster collaboration among stakeholders for advancing NMIBC drug development.
Main Methods:
- A public virtual workshop convened by the FDA on November 18-19, 2021.
- Multidisciplinary participation including urologists, oncologists, pathologists, statisticians, scientists, and patient advocates.
- Included invited lectures, panel discussions, and audience engagement.
Main Results:
- A survey revealed 92% of urologists prioritize developing BCG alternatives for high-risk, BCG-naïve patients.
- Key discussions focused on disease state definitions and trial designs for various NMIBC subtypes.
- Considerations included single-arm trial limitations, patient-reported outcomes, and cystectomy avoidance as an efficacy endpoint.
Conclusions:
- The workshop identified critical areas for refining NMIBC clinical trial designs.
- The FDA encourages early discussion of trial proposals with regulatory divisions to expedite NMIBC drug development.
