title: Clinical trial design for the development of new therapies for nonmuscle-invasive bladder cancer: report of a Food and Drug Administration and American Urological Association public workshop.
abstract: OBJECTIVE
  To summarize the discussion at a public workshop, cosponsored by the U.S. Food and Drug Administration (FDA) and the American Urological Association, reviewing potential trial designs for the development of new therapies for non-muscle-invasive bladder cancer (NMIBC). There have been only 3 drug approvals for NMIBC in the last 30 years, and product development for this disease has been stymied by difficulties in trial design and patient accrual.
  
  
  METHODS
  A workshop evaluating potential trial design for the development of therapies for NMIBC was held in San Diego, CA, in May 2013. Invited experts representing all stakeholders, including urology, medical oncology, radiation oncology, industry, and patient advocates, discussed development of products for all risk strata of NMIBC.
  
  
  RESULTS
  The panel responded to specific questions from the FDA, discussing eligibility criteria, efficacy endpoints, and trial design for patients with a mix of high-grade papillary disease and carcinoma in situ, Bacillus Calmette-Guerin (BCG)-refractory disease, and intermediate-risk disease. Panel members also addressed the magnitude of response that would be clinically meaningful for various disease strata and trial design options for perioperative intravesical chemotherapy instillation at the time of resection of bladder tumors.
  
  
  CONCLUSIONS
  Expert commentary provided by panel members will inform a planned FDA guidance on pathways for drug and biologic development for NMIBC and will be discussed at meetings of the FDA's Oncologic Drugs Advisory Committee. FDA intends to develop a set of principles that can be used to promote the development of new products for this disease.
authors: Office of Hematology and Oncology Products, Food and Drug Administration, Silver Spring, MD. Electronic address: Jonathan.jarow@fda.hhs.gov.
authors: Jonathan P
authors: JP
authors: Jarow
authors: Scott Department of Urology, Baylor College of Medicine Medical Center, Houston, TX.
authors: Seth P
authors: SP
authors: Lerner
authors: Office of Hematology and Oncology Products, Food and Drug Administration, Silver Spring, MD.
authors: Paul G
authors: PG
authors: Kluetz
authors: Office of Cellular, Tissue and Gene Therapies, Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD.
authors: Ke
authors: K
authors: Liu
authors: Division of Biometrics V, Office of Biostatistics, CDER, Food and Drug Administration, Silver Spring, MD.
authors: Rajeshwari
authors: R
authors: Sridhara
authors: Memorial Sloan Kettering Cancer Center, Weill Cornell Medical College, New York, NY.
authors: Dean
authors: D
authors: Bajorin
authors: Department of Urologic Surgery, Vanderbilt University Medical Center, Nashville, TN.
authors: Sam
authors: S
authors: Chang
authors: The University of Texas MD Anderson Cancer Center, Houston, TX.
authors: Colin P N
authors: CP
authors: Dinney
authors: Department of Preventive Medicine, USC Norris Comprehensive Cancer Center, USC/Keck School of Medicine, Los Angeles, CA.
authors: Susan
authors: S
authors: Groshen
authors: American Medical Systems, Minnetonka, MN.
authors: Ronald A
authors: RA
authors: Morton
authors: University of Iowa, Iowa city, IA.
authors: Michael
authors: M
authors: O'Donnell
authors: Bladder Cancer Advocacy Network, Bethesda, MD.
authors: Diane Zipursky
authors: DZ
authors: Quale
authors: Johns Hopkins University School of Medicine, Baltimore, MD.
authors: Mark
authors: M
authors: Schoenberg
authors: Dartmouth-Hitchcock Medical Center and Norris Cotton Cancer Center, Lebanon, NH.
authors: John
authors: J
authors: Seigne
authors: National Cancer Institute, Bethesda, MD.
authors: Bhadrasain
authors: B
authors: Vikram
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D006801:Humans; D009361:Neoplasm Invasiveness; D012955:Societies, Medical; D014481:United States; D014486:United States Food and Drug Administration; D001749:Urinary Bladder Neoplasms / Q000473:pathology / Q000628:therapy*; D014572:Urology