Presentation Authors: John Gore*, Erika Wolff, Seattle, WA, Stephanie Chisolm, Robert Lipman, Bethesda, MD, Angela Smith, Chapel Hill, NC
Introduction: Patients are an underrepresented stakeholder in the research process. We partnered with the Bladder Cancer Advocacy Network (BCAN) to develop the Patient Survey Network (PSN) for patient-centered research prioritization. Results highlighted the need for comparative effectiveness research studies among patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). Based on these results, we constructed a research design for a trial entitled Comparison of Intravesical therapy and Surgery as Treatment Options (CISTO). We sought input from individuals with bladder cancer and their caregivers with a survey distributed to the BCAN PSN to query attitudes about randomization, study design, and relevant patient-centered outcomes.
Methods: We conducted a web-based survey of the 1,300-member BCAN PSN. Responses were collected between March 6, 2017, and April 6, 2017. The survey included questions about bladder cancer diagnosis and treatment history, willingness to participate in clinical trials with different study designs, and the relative importance of various outcomes.
Results: A total of 291 individuals previously diagnosed with bladder cancer responded, 201 of whom reported having had NMIBC, and 104 of whom had experienced recurrent NMIBC after BCG treatment. Among all respondents and within the subgroup of patients who experienced recurrent NMIBC after BCG, only 11% reported being willing to consent to randomization for a study of radical cystectomy versus intravesical therapy. The majority (76%) indicated a strong preference to keep their bladders. Therefore, a prospective observational cohort study was selected as the highest quality, most feasible and inclusive study design. Respondents recommended that the most relevant domains with which to compare our two treatments were survival and QOL, ranked in order of: 1) overall survival, 2) risk of spread of the cancer to other parts of the body, 3) overall QOL, 4) impact of the treatments on their caregiver or loved one, 5) urinary QOL, and 6) the costs or financial distress these treatments can incur. These outcomes were included as primary and secondary endpoints in CISTO.
Conclusions: Patient input from the BCAN PSN informed the design of a patient-centered pragmatic trial for bladder cancer, which was funded by PCORI. Our results highlight the importance of involving patients throughout the research process in order to construct feasible study designs whose results will be important to the affected patients.
Source of Funding: PCORI Eugene Washington Engagement Award Contract 1089