Engaging with patients, caregivers, clinicians and researchers can provide invaluable insight into developing, maintaining, and completing valuable comparative effectiveness research. Specific to clinical trial design, patient and stakeholder engagement can be applied at multiple steps in the process, including identification of research questions, selection of key trial outcomes including relevance to patients and stakeholders, input on pragmatic and patient-centered trial design, and contextualization of results (see Table).¹ While some of these aspects lend themselves well to structured prioritization, others are best accomplished through semistructured feedback, discussion, and broader consensus building.² For instance, creating a list of topics and/or outcomes to be included may be accomplished with prioritization.³ More nuanced decisions in terms of hypothesis setting or contextualization of results often require more free-form discussion and consensus.⁴ Meanwhile, decisions related to specific elements of trial design (ie, format of patient-facing data, timing of follow-up) may be accomplished via seeking semistructured feedback from the stakeholder team.⁵ In order to better understand the patient voice and provide a mechanism for supporting the kidney stone community, we developed the Kidney Stone Engagement Core (KSEC). KSEC is comprised of 16 individuals, including 8 clinicians and researchers, 6 patients with kidney stones, 1 caregiver of a patient with kidney stones, and 1 patient advocate. We will review our lessons learned in creating and engaging a stakeholder-based team, focused on prioritization of research topics for comparative effectiveness trials in kidney stone disease.

Figure. Stakeholder engagement and Kidney Stone Engagement Core (KSEC) collaboration diagram, defining distinct processes for engagement and KSEC collaborative efforts.

By including a diverse population, we ensured a range of voices represented the breadth of experiences with kidney stone disease. We intentionally sought out representation of youth (patients and caregivers) and those with rare genetic diseases (ie, primary hyperoxaluria) as well as the experiences of patients more representative of the typical epidemiology of kidney stone disease. Recruitment into the KSEC was best accomplished by referral from a trusted source (typically, for the patients or caregivers, their long-standing provider) and personal communication with the KSEC team before signing on to the project.

Regulatory and Logistical Support of a Stakeholder Group

Our team worked closely with our institution in order to define the roles of the KSEC membership. Each KSEC member was contracted through our host institution as an independent contractor (consultant). Within this structure, we were able to follow a standardized process of remuneration already in place for independent contractors and an agreed upon payment schedule. Our engagement team helped to track the hourly engagement of the members to reduce logistical burdens on the stakeholders. Creation and involvement of the KSEC was included in our Institutional Review Board–approved protocol so as to explicitly distinguish the KSEC members from research participants. In our model, KSEC members do not review individualized data, reducing regulatory burden on the group. This model still allows for KSEC to participate in the process related to recruitment materials and review of aggregate results. Introductory meetings and onboarding were important elements to level-set goals and expectations. We chose to use the Fyreworks platform, https://fyreworkstraining.com/, supported by the Patient-Centered Outcomes Research Institute, as a standard educational curriculum for the group. We developed a roadmap, as seen in the Figure, to help guide our team as we worked through each phase of the project.

Maintaining Engagement

Engaging with the KSEC provided valuable insight in working in an engagement-style project. As we moved throughout the project, we developed a better understanding of each member’s time and availability to participate. By seeking KSEC member feedback, we improved our processes to amplify productivity by providing more direct timelines and expectations regarding specific KSEC tasks and responsibilities, recording each bimonthly team meeting, and conducting additional one-on-one meetings for members who wished to have additional feedback. We altered our meeting structure in year 2 of the project to allow for shorter, more frequent meetings acknowledging individual availability may fluctuate from month to month. This flexibility is especially important when including individuals with active urological disease, as health concerns may impede the ability of any 1 member to participate at a given time. Thus, structuring an overlapping representation across the stakeholder group and a flexible workflow allowed us to optimize this varying engagement over time while maintaining the enthusiasm of our core group.

Optimizing Efficient Communication

From specific feedback from the group, we utilized a cloud-based shared drive (Google Docs) so that each KSEC member had access for editing, reviewing, and tracking for all project elements. Additionally, in periods of time-sensitive workflow, we structured smaller group meetings to enhance engagement. We found that the KSEC worked especially well with these small group “sprints” with tasks that were both time-sensitive and nuanced, such as translating our elicited themes into research topics for prioritization.

The Value of Stakeholder Collaboration

KSEC members have found value in the opportunity to make a difference and connect to individuals in the broader kidney stone disease community including those who have similar experiences and/or clinical paths. These individuals find a voice in clinical research, thereby ensuring that their concerns and opinions are being heard by the clinical research communities. Most importantly, this also allowed KSEC members to help guide the direction of future research to ensure that it addresses outcomes most relevant to the patient and other stakeholder communities. Throughout semistructured interviews, focus groups, and prioritization Delphi strategies, the kidney stone community has communicated their enthusiasm towards our project by providing stories, perspectives, and opinions. Additionally, our KSEC members have noted a sense of pride and satisfaction in being involved in our group and providing meaningful input that will benefit the community. Several members have voiced that because of the difficulties and unpredictable nature of their kidney stone disease, the opportunity to participate in the KSEC was an outlet to demonstrate active agency over the disease. As expressed by one of our team members: “…Because of the diversity of our team, the research conducted has the ability to help many people. Patients can refer to the work to assist in their own treatment, caregivers can gain insight, and the medical professionals can use our work to aid in the treatment plans for their own patients. This work allows for the conversation to keep being had and best practices to keep being developed.”