Journal article
Advancing Clinical Trial Design for Non-Muscle Invasive Bladder Cancer
Bladder cancer (Amsterdam, Netherlands), Vol.9(3), pp.271-286
2023
DOI: 10.3233/BLC-230056
PMCID: PMC11181701
PMID: 38993184
Abstract
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.
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.
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.
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.
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.
Details
- Title: Subtitle
- Advancing Clinical Trial Design for Non-Muscle Invasive Bladder Cancer
- Creators
- Elaine Chang - Center for Drug Evaluation and ResearchNoah M Hahn - Johns Hopkins Greenberg Bladder Cancer Institute, Johns Hopkins School of Medicine, Baltimore, MD, USASeth P Lerner - Baylor College of MedicineJaleh Fallah - Center for Drug Evaluation and ResearchSundeep Agrawal - Center for Drug Evaluation and ResearchAshish M Kamat - The University of Texas MD Anderson Cancer CenterVishal Bhatnagar - United States Food and Drug AdministrationRobert S Svatek - Department of Urology, University of Texas Health San Antonio, San Antonio, TX, USAAdnan A Jaigirdar - Center for Biologics Evaluation and ResearchPeter Bross - Center for Biologics Evaluation and ResearchNeal Shore - Carolina Urologic Research CenterMax Kates - Johns Hopkins University School of MedicineKaren Sachse - Bladder Cancer Advocacy Network, Bethesda, MD, USAJamie R Brewer - Center for Drug Evaluation and ResearchMichael A O'Donnell - University of IowaGary D Steinberg - NYU Langone HealthCharles J Viviano - Center for Devices and Radiological HealthErik Bloomquist - Center for Drug Evaluation and ResearchMaria J Ribal - Uro-Oncology Unit, Hospital Clinic, University of Barcelona, Barcelona, SpainMatthew D Galsky - Tisch Cancer InstituteRichard Oliver - Huntsman Cancer Institute, University of Utah, Salt Lake City, UT, USAPeter C Black - University of British ColumbiaHikmat Al-Ahmadie - Memorial Sloan Kettering Cancer CenterKenneth Brothers - National Cancer Institute Bladder Cancer Task Force, Bethesda, MD, USAKamal Pohar - The Ohio State UniversityColin P Dinney - The University of Texas MD Anderson Cancer CenterZhou Feng - Center for Drug Evaluation and ResearchTracy M Downs - University of VirginiaSima P Porten - University of California, San FranciscoAngela B Smith - UNC Lineberger Comprehensive Cancer CenterRick Bangs - Bladder Cancer Advocacy Network, Bethesda, MD, USASarah P Psutka - University of WashingtonNeeraj Agarwal - Huntsman Cancer InstituteLaleh Amiri-Kordestani - Center for Drug Evaluation and ResearchDaniel L Suzman - Center for Drug Evaluation and ResearchRichard Pazdur - United States Food and Drug AdministrationPaul G Kluetz - United States Food and Drug AdministrationChana Weinstock - Center for Drug Evaluation and Research
- Resource Type
- Journal article
- Publication Details
- Bladder cancer (Amsterdam, Netherlands), Vol.9(3), pp.271-286
- DOI
- 10.3233/BLC-230056
- PMID
- 38993184
- PMCID
- PMC11181701
- NLM abbreviation
- Bladder Cancer
- ISSN
- 2352-3727
- eISSN
- 2352-3735
- Publisher
- IOS PRESS
- Language
- English
- Date published
- 2023
- Academic Unit
- Urology
- Record Identifier
- 9984721139002771
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