Definitions, End Points, and Clinical Trial Designs for Bladder Cancer

On behalf of the Society for Immunotherapy of Cancer (SITC) and the International Bladder Cancer Group (IBCG), we are pleased to announce the publication of the “Definitions, End Points, and Clinical Trial Designs for Bladder Cancer: Recommendations From the Society for Immunotherapy of Cancer and the International Bladder Cancer Group,” in the Journal of Clinical Oncology. These recommendations provide a detailed clinical trial design guide to maximize the chance of capturing the benefit of medical therapies for each unique stage of bladder cancer, from low-risk non-muscle invasive bladder cancer (NMIBC) to metastatic disease.

SITC and IBCG formed a partnership to develop Expert Panel recommendations in response to a significant unmet need that still exists for standardized trials designed for new urothelial carcinoma (UC) treatments specific to stage and treatment approach. In this new manuscript, the Expert Panel discusses standardization of key clinical design elements to identify optimal treatment options and improve outcomes for patients. By providing standardized criteria, the paper also aims to benefit the field by allowing cancer researchers to develop more comparable and robust data sets for meta-analyses. 

Key Bladder Cancer Stage-Specific Recommendations:

  • Rationally selected endpoints
  • Eligibility criteria
  • Response measurement
  • Statistical analyses (including minimal value for effect and sample size considerations)
  • Pathology considerations 

Companion Education 

To help provide a deeper understanding of the recommendations, SITC hosted a webinar on March 18, 2024, from 4–5 p.m. CST with the manuscript authors serving as faculty for the program. Participants joined experts to discuss the publication in-depth, ask questions and explore topics such as: non-muscle and muscle invasive bladder cancer, metastatic unresectable and locally advanced disease.

Moderators

  • Matthew D. Galsky, MD – Mount Sinai
  • Ashish Kamat, MD, MBBS - University of Texas MD Anderson Cancer
    Center

Faculty Speakers

  • Andrea Apolo MD — National Cancer Institute/Center for Cancer Research
  • Shilpa Gupta, MD — Cleveland Clinic Taussig Cancer Institute
  • Seth Lerner, MD, FACS — Baylor College of Medicine
  • Sarah P. Psutka — University of Washington

Learning Objectives

  1. Understand appropriate definitions and end points that are relevant to the understanding of existing trials in each unique stage of bladder cancer, from low-risk non-muscle invasive bladder cancer (NMIBC) to metastatic disease.
  2. Understand nuances of rationally selected endpoints, eligibility criteria, response measurement, statistical analyses (including minimal value for effect and sample size considerations), and pathology considerations
  3. Apply these key clinical design elements and standardized criteria to their clinical trials (and practice) to identify optimal treatment options and improve outcomes for patients.
  4. Apply these criteria to develop comparable and robust data sets for meta-analyses. 

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Tweet 1 graphic alt - front page publication

SITC-IBCG Bladder Cancer Clinical Trial Design Expert Panel

CHAIR

Matthew D. Galsky, MD 
Mount Sinai Medical Center

CHAIR

Ashish M. Kamat, MD, MBBS, FACS
The University of Texas MD Anderson Cancer Center

Members

Andrea B. Apolo, MD — National Cancer Institute/Center for Cancer Research

Marek Babjuk, MD, PhD — Charles University, Praha

Trinity J. Bivalacqua, MD, PhD — University of Pennsylvania

Peter Black, MD, FACS, FRCSC — University of British Columbia

Roger Buckley, MD, FRCSC — North York General Hospital

Matthew T. Campbell, MD, MS — The University of Texas MD Anderson Cancer Center

Eva Comperat, MD, PhD — Sorbonne University

Jason Efstathiou, MD, PhD, DPHIL — Massachusetts General Hospital

Petros Grivas, MD, PhD — University of Washington

Shilpa Gupta, MD — Cleveland Clinic Taussig Cancer Institute

Neil J. Kurtz, MD — BCAN Representative

Donald Lamm, MD, FACS — BCG Oncology, AZ

Seth P. Lerner, MD, FACS — Baylor College of Medicine

Roger Li , MD — H Lee Moffitt Cancer Center

David J. McConkey, PhD — Johns Hopkins Greenberg Bladder Cancer Institute

Joan Palou Redorta, MD, PhD — Fundació Puigvert

Thomas Powles, MBBS, MD, MRCP — Queen Mary University of London

Sarah P Psutka , MD — University of Washington

Neal Shore, MD, FACS — Carolina Urologic Research Center

Gary D. Steinberg, MD — Perlmutter Cancer Center - New York University Langone Medical Center

Richard Sylvester, ScD — EAU NMIBC Guidelines Panel

J. Alfred Witjes MD, PhD — University Radboud

Arjun V. Balar, MD — Perlmutter Cancer Center - New York University Langone Medical Center

Other Clinical Resources