Patient engagement in a bladder cancer clinical trial is an ongoing, valuable, iterative process that relies on building trusting relationships between various stakeholders, including patients, clinicians, external advisory board (EAB) members, advocacy organizations, and principal investigators (PIs). Patient involvement in studies of bladder cancer may be particularly valuable due to its high recurrence rate, long-term invasive testing, long-term high cost,¹ and limited treatment options, including the life-changing aspects of undergoing radical cystectomy. Best practice statements are readily available for all clinicians.² However, navigating treatment decisions based on patient preferences is particularly more challenging and nuanced than following algorithmic guidelines. Patients, family members, and physicians need evidence about clinical outcomes and subsequent quality of life based on patient-centered, real-world evidence.

In the age of complex and competitive anticancer drug trials, high-quality patient-centered trials examining factors influencing treatment decisions are rare and require dedicated routine patient engagement. The Comparison of Intravesical Therapy and Surgery as Treatment Options (CISTO) is a prospective, observational cohort study of bacillus Calmette-Guérin–unresponsive nonmuscle-invasive bladder cancer patients comparing patient-reported and patient-centered clinical outcomes (eg, recurrence-free survival) between those undergoing radical cystectomy and those receiving bladder-sparing therapies.³ This pragmatic clinical trial will provide evidence to maximize informed decision-making for patients, family members, and clinicians based on patient-reported outcomes and treatment preferences while maintaining rigor in its research methods.⁴ The study illustrates a real-world example of patient engagement in bladder cancer trials.

The CISTO study is fundamentally a patient-centered trial: patients participated in prioritizing research questions identified important outcomes, helped draft the grant proposal, reviewed survey questionnaires, helped establish ways to engage and interest potential patient participants, and brainstormed about recruitment challenges. Future patient engagement will focus on plans for the dissemination of study results to various stakeholders. Creating a patient engagement cohort began with a collaboration between the CISTO research team and the Bladder Cancer Advocacy Network to develop a patient survey network to identify patient research priorities and important stakeholders. Subsequently advocate advisory board (AAB) and EAB members were recruited; a full description of the development of the patient (and public) engagement plan is available elsewhere.⁴

The physician-patient research team has learned valuable lessons in successfully engaging patients in clinical trials. Regular meetings are essential, even if there are no pressing issues to discuss, as this schedule enables relationship-building and trust. Good meeting etiquette is important, such as regular email communication, providing materials and agendas in advance, and respectful leadership and facilitation to allow for full participation. Opportunities for AAB and EAB members to meet each other and invitations to participate in Annual Meetings are also important. Stipends for AAB members also reinforce the importance of patient voices in the study protocol.

Exemplary patient engagement such as this can exist at the national study administrative level, but can only be carried out locally with active study PIs who champion recruitment and patient engagement at a local level. Multiple barriers to patient recruitment to bladder cancer trials exist; specifically, the recruitment and retention of clinical research professionals (CRPs) has received national attention lately. Data indicate an increase in unfilled CRP positions leading to unstable clinical trial recruitment efforts and diminishing patient engagement.⁵ As such, it is important that leadership of any successful bladder cancer trial provide organization, ample operational resources, and suggestions for local strategies to overcome these barriers. Site PIs must remember the first line of patient engagement in a trial starts with CRP personnel, not necessarily routine clinic staff. Thus recruiting, securing, and supporting a highly trained, diverse CRP workforce is essential to success.

Bladder cancer trials, particularly nonmuscle-invasive bladder cancer studies, often provide a unique, long-term patient-provider engagement experience due to frequent treatment failures and progression to clinical trial options. Patient engagement in clinical trial discussions starts at the time of diagnosis when the disease natural history is reviewed. Collaborative efforts and infrastructure between site PIs, CRPs, ancillary workforce, and patients are irrefutably important. Site PIs routinely have busy clinical schedules and serve as a PI on multiple studies. Thus, it is important for patients to understand that the primary contact for management while on study is not necessarily the physician who enrolled them. Engaging patients up front at the time of enrollment to ask questions and set expectations is required. CRPs are critical in educating patients on scheduling, paperwork, time commitment outside of treatment sessions or surgeries, follow-up, etc. Robust institutional research enterprise support, national study support (or industry support), institutional infrastructure, clinical referral base, CRP personnel, and the PI championing the study predict success and optimize patient engagement.

The benefits of patient engagement in bladder cancer studies are many. Pragmatic, well-designed clinical trials must combine patient input, such as patient-reported outcomes and treatment preferences, along with rigorous research methodologies in order to provide evidence to maximize informed decision-making for patients, family members, and clinicians.⁶

The broad inclusion criteria and pragmatic design of trials such as CISTO will also improve the generalizability and usefulness of study results. For example, physicians can use the CISTO study results in decision-making conversations with patients, knowing that the data were generated in partnership with real patients facing similar clinical dilemmas.

From the patient perspective, being a member of the AAB for CISTO has had great benefits. As one AAB member relates, when they agreed to participate in the AAB, they could hardly say the words “bladder cancer.” AAB membership presented a group of individuals who understood and accepted their story, and were open to sharing theirs. To become acquainted with a talented, committed, passionate group of clinicians/researchers dedicated to better understanding bladder cancer, patients with bladder cancer, and the challenging decision-making that bladder cancer requires has been an experience of affirmation and hope. The ability to “give back” and “pay it forward,” while clichéd, have given many patient advocates’ bladder cancer journeys meaning and purpose, because the contributions of patients to CISTO will pay great benefits to future bladder cancer patients, their family members, and their clinical teams.

Acknowledgments

We thank all the members of our CISTO team through the CISTO Collaborative.

Funding: Research reported in this article was funded through a Patient-centered Outcomes Research Institute (PCORI) Award (PCS-2017C3-9380). The statements and opinions in this article are solely the responsibility of the authors and do not necessarily represent the views of the Patient-centered Outcomes Research Institute (PCORI), its Board of Governors, or Methodology Committee.