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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., Competing Interests: E.C., J.F., S.A., V.B., A.A.J., P.B., K.S., J.R.B., E.B., R.O., K.B., Z.F., T.M.D., R.B., D.L.S., L.A.K., R.P., P.G.K., and C.W. have no conflicts of interest to report. N.M.H., S.P.L., A.M.K., R.S.S., M.A.O., M.J.R., M.D.G., P.C.B., K.P., C.P.D., R.B., and S.P.P. (Psutka) are Editorial Board members of this journal, but were not involved in the peer-review process nor had access to any information regarding its peer-review. N.M.H. receives consulting compensation from AstraZeneca, Merck, BioGears, Seattle Genetics, Mirati, Incyte, RemGen, Janssen, Pfizer, EMD Serono, Verity Pharmaceuticals, Huron Consulting, Guidepoint, Natera, Protara Therapeutics, Astellas Pharma; research support to the institution from HTG Molecular Diagnostics, AstraZeneca, Bristol Myers-Squibb, Genentech, Seattle Genetics, Pieris, Inovio, Principia Biopharm, Incyte, and Ikena Oncology; and speaking honorarium from Medscape. S.P.L. reports clinical trial involvement with Aura Bioscience, FKD, JBL (SWOG), Genentech (SWOG), Janssen (SWOG), Merck (Alliance), QED Therapeutics, UroGen, Vaxiion, Viventia; is on the advisory board or a consultant for Aura Bioscience, BMS, C2iGenomics, Ferring, Incyte, Pfizer/EMD Serono, Protara, Stimit, UroGen, Vaxiion, Verity; has a patent for a TCGA classifier; and receives honoraria from Grand Rounds Urology and UroToday. A.M.K. reports clinical trial involvement with FKD, Merck, Bristol Myers Squibb, Photocure, SWOG, Adolor, Heat Biologics, Janssen, Taris, Seattle Genetics; reports laboratory research with NIH, SPORE, AIBCCR, PCORI; is on the advisory board or a consultant for TMC Innovation, Arquer Diagnostics, Asieris, Astellas, Biological Dynamics, BMS, CG Oncology, Cystotech, Eisai, Engene, Ferring, InCyte, Imvax, Imagin Medical, Janssen, Medac, Merck, Nonagen, Photocure, ProTara, Pfizer, Roche, Seattle Genetics, Sessen Bio, Theralase, US Biotest, Urogen Inc; is on the editorial board of European Urology Oncology; Journal of Urology, UroToday; is president of International Bladder Cancer Network and International Bladder Cancer Group; and has a joint patent with UT MD Anderson Cancer Center for CyPRIT (Cytokine Predictors of Response to Intravesical Therapy). R.S.S. is a consultant for CG Oncology and Verity Pharma; and receives research support from Japanese BCG Laboratories and Merck. N.S. is a consultant for AbbVie, Accord, Alessa Therapeutics, Amgen, Antev, Arquer, Asieris, Astellas, Astra Zeneca, AuraBiosciences, Bayer, BMS, Bioprotect, Boston Scientific, Clarity, Dendreon, Exact Imaging, FizeMedical, CG Oncology, Genentech/Roche, Ferring, Foundation Medicine, ImmunityBio, Incyte, Invitae, Janssen, Lantheus, Lilly, MDX, Merck, Minomic, Myriad, Nonagen, Novartis, Nymox, Pacific Edge, Palette Life, Photocure, Pfizer, PlatformQ, Profound Medical, Promaxo, Propella, Protara, Sanofi, Specialty Networks, Telix, Tolmar, Urogen, and Vessi. M.K. receives grant funding from the American Cancer Society; is a consultant for Merck, Pfizer, Seagen/Astellas, Photocure, Aura, BMS, Nanology, and Janssen; and holds a patent for Nanoparticle Formulations for Enhanced Drug Delivery to the Bladder. M.A.O. receives grant/research/clinical trial support from Abbot Molecular, Photocure, Urogen; and is a consultant or on the advisory board for Fidia Pharmaceuticals, Sesen Bio, Merck, Theralase, and Urogen. G.D.S. is a member of the Clinical Trial Protocol Committee for Merck, BMS, Janssen, CG Oncology, Pfizer, PhotoCure, Fidia, Seagen, Protara; is or has been a scientific advisor /consultant with CG Oncology; PhotoCure; Merck; Taris Biomedical (Now Janssen); Fidia Farmaceuticals; Urogen, Ferring; Fergene, Bristol Myers Squibb; Astra Zeneca; Pfizer, Janssen; Epivax Therapeutics; EnGene Bio; Astellas; SeaGen; Verity Pharmaceuticals, Protara, xCures, Nonagen, Nanology, Imvax, Asieris; and has equity stock/options with Epivax Therapeutics, Urogen, CG Oncology, Engene Bio. M.J.R. has intellectual property with Fina Biotech and is or has been a meeting participant or lecturer for Roche, AstraZeneca, and Bayer. M.D.G. receives research funding from Bristol Myers Squibb, Novartis, Dendreon, Astra Zeneca, Merck, Genentech; and is on the advisory board or a consultant for Bristol Myers Squibb, Merck, Genentech, AstraZeneca, Pfizer, EMD Serono, SeaGen, Janssen, Numab, Dragonfly, GlaxoSmithKline, Basilea, UroGen, Rappta Therapeutics, Alligator, Silverback, Fujifilm, Curis, Gilead, Bicycle, Asieris, Abbvie, Analog Devices. P.C.B is a member of the advisory board or equivalent with AbbVie, AstraZeneca, Astellas, Bayer, BMS, EMD-Serono, Ferring, Fergene, Janssen, Merck, miR Scientific, Nonagen, NanOlogy, Pfizer, Photocure, Prokarium, Protara Therapeutics, QED Bioscience, Roche, Sanofi, Sesen Bio, STIMIT, TerSera, Tolmar, Urogen, Verity; is a member of a Speaker’s bureau with Janssen, Minogue, Ferring, TerSera, Pfizer; has received a grant or honorarium from iProgen; and shares a patent with Veracyte. H.A. provides consultation to AstraZeneca, Flare Therapeutics and Paige.AI. K.P. receives consulting fees from Photocure. C.P.D. reports compensation for Scientific/Advisory Committee Member for AstraZeneca Pharmaceuticals, UroGen Pharma (formerly Theracoat Ltd.); consulting fees from STIMIT Corporation; consulting fees from General Atlantic; board member position for DF/HCC Kidney Cancer SPORE Advisory Board; research funding from Cancer Prevention Research Institute of Texas (CPRIT), Department of Defense (DoD), and NIH/NCI. S.P.P. (Porten) receives research support from Photocure, consulting compensation from Pacific Edge, grant funding from PCORI, BCAN, AHRQ, Genentech, Merck, and is on the advisory board for AstraZeneca. A.B.S. receives grant funding from PCORI, BCAN, AHRQ, Genentech, and Merck. S.P.P. (Psutka) reports being on Practice Guidelines Committee (Upper Tract Urothelial Carcinoma) of the American Urological Association (AUA), being AUA Core Curriculum Sr. Editor (2019-2023) and Senior Consultant (2023-); is or has been on Scientific Advisory Boards for Merck (Past), ImmunityBio (Past), Janssen (Current); has received travel funding/honoraria from Medtronic (Past), AstraZeneca (Past); receives research funding from PRIME Education, INC, Bladder Cancer Advocacy Network, National Institute on Aging, and is on the editorial boards for European Urology. N.A. receives research funding to institution from Arnivas, Astellas, Astra Zeneca, Bavarian Nordic, Bayer, Bristol Myers Squibb, Calithera, Celldex, Clovis, Crispr, Eisai, Eli Lilly, EMD Serono, Exelixis, Genentech, Gilead, Glaxo Smith Kline, Immunomedics, Janssen, Lava, Medivation, Merck, Nektar, Neoleukin, New Link Genetics, Novartis, Oric, Pfizer, Prometheus, Rexahn, Roche, Sanofi, Seattle Genetics, Takeda, and Tracon., (© 2023 – The authors. Published by IOS Press.)

Over the past 20 years, cancer patient advocacy groups have demonstrated that patient engagement in cancer care is essential to improving patient quality of life and outcomes. Bladder cancer patient advocacy only began 10 years ago in the United States, but is now expanding around the globe with non-profit organizations established in Canada, the United Kingdom and Italy, and efforts underway in Australia. These organizations, at different levels of maturity, are raising awareness of bladder cancer and providing essential information and resources to bladder cancer patients and their families. The patient advocacy organizations are also helping to advance research efforts by funding research proposals and facilitating research collaborations. Strong partnerships between these patient advocates and the bladder cancer medical community are essential to ensuringsustainability for these advocacy organizations, increasing funding to support advances in bladder cancer treatment, and improving patient outcomes.