The following is a summary of discussion at a U.S. Food and Drug Administration (FDA) public workshop reviewing potential trial designs and endpoints for development of therapies to treat localized prostate cancer.

The workshop focused on the challenge that drug and device development for the treatment of localized prostate cancer has been limited by the large trial sizes and lengthy timelines required to demonstrate an improvement in overall survival or metastasis-free survival and by the lack of agreed-upon alternative endpoints. Additionally, evolving treatment paradigms in the management of localized prostate cancer include the widespread use of active surveillance for patients with low- and some intermediate-risk prostate cancer and the availability of advances in imaging and genomics.

The workshop addressed issues related to trial design in this setting and discussed several potential novel endpoints such as delay of morbidity due to radiation or prostatectomy, and pathologic endpoints such as Gleason Grade Group upgrade.

The workshop provided an open forum for multi-stakeholder engagement to advance the development of effective treatment options in localized prostate cancer. Full workshop proceedings are available online at https://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm608328.htm.

The Journal of urology. 2019 Sep 10 [Epub ahead of print]

Chana Weinstock, Daniel Suzman, Paul Kluetz, John Baxley, Charles Viviano, Amna Ibrahim, Jonathan Jarow, Raejshwari Sridhara, Ke Liu, Peter Carroll, Scott Eggener, Boris Freidlin, Jim C Hu, Maha Hussain, Martin King, Eric Klein, Terry Kungel, Danil Makarov, Peter A Pinto, Brian Rini, Mack Roach, Howard Sandler, Peter N Schlegel, Daniel Song, Kirsten Goldberg, Richard Pazdur, Julia Beaver

U.S. Food and Drug Administration., UCSF Helen Diller Comprehensive Cancer Center., University of Chicago Medicine., National Cancer Institute., Weill Cornell Medicine., Robert H. Lurie Comprehensive Cancer Center Northwestern University Feinberg School of Medicine., Dana Farber Cancer Institute, Harvard Medical Center., Cleveland Clinic., New York University School of Medicine., Cedars-Sinai Medical Center., New York Presbyterian/Weill Cornell Medical Center., Johns Hopkins Medical Institute.

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