Examining how study teams manage different viewpoints and priorities in patient-centered outcomes research: Results of an embedded multiple case study.

INTRODUCTION: Limited evidence exists about which patient and stakeholder engagement practices support or hinder study teams as they negotiate different viewpoints in decisions about the design and conduct of patient-centered outcomes research. METHODS: We applied a multiple-embedded descriptive case study design for six studies funded by the Patient-Centered Outcomes Research Institute (PCORI). We interviewed 32 researchers and stakeholder partners, including patients, caregivers and clinicians, and reviewed documents related to each study (e.g., publications, and progress reports submitted to PCORI). FINDINGS: Overall, researchers reported that incorporating different viewpoints was a strength or opportunity to learn rather than something to be avoided or dreaded. Across cases, different viewpoints and priorities, often related to ethical or pragmatic considerations, emerged between researchers and stakeholders, between stakeholder groups (e.g., patients and clinicians) or within groups (e.g., amongst researchers). Examples of navigating different viewpoints arose across study phases. The length of time to resolve issues depended on how strongly people disagreed and the perceived importance or impact of decisions on the study. All cases used collaborative decision-making approaches, often described as consensus, throughout the study. Interviewees described consensus as using negotiation, compromise or working towards an agreeable decision. To encourage consensus, cases actively facilitated group discussions with an openness to diverse opinions, remained flexible and open to trying new things, referenced a ground rule or common goal and delegated decisions to partners or smaller workgroups. When viewpoints were not easily resolved, cases used different approaches to reach final decisions while maintaining relationships with partners, such as elevating decisions to leadership or agreeing to test out an approach. No one engagement structure (e.g., advisory group, coinvestigator) stood out as better able to manage different viewpoints. Teams adjusted engagement structures and behaviours to facilitate an overall culture of inclusion and respect. Partners acknowledged the intentional efforts of researchers to incorporate their perspectives, navigate challenges and communicate the value of partner input. CONCLUSION: By using collaborative decision-making in the early stages and throughout the study, researchers built trust with partners so that when decisions were difficult to resolve, partners still felt listened to and that their input mattered. PATIENT OR PUBLIC CONTRIBUTION: Members of the PCORI Patient Engagement Advisory Panel in 2019-2020 provided input into the design of the study, including the research questions and approaches to data collection and analysis.

Stakeholder-driven principles for advancing equity through shared measurement.

OBJECTIVE: To advance equity by developing stakeholder-driven principles of shared measurement, which is using a common set of measurable goals that reflect shared priorities across communities and systems, such as health care, public health, and human and social services. DATA SOURCES: From October 2019 to July 2021, we collected primary data from leaders in cross-systems alignment, measurement, and community engagement-including community members and community-based organization leaders-across the United States. STUDY DESIGN: In partnership with equity and community engagement experts, we conducted a mixed-methods study that included multiple formative research activities and culminated in a six-week, stakeholder-engaged modified-Delphi process. DATA COLLECTION: Formative data collection occurred through an environmental scan, interviews, focus groups, and an online survey. Principles were developed using a virtual modified Delphi with iterative rapid-analysis. Feedback on the final principles was collected through virtual focus groups, an online feedback form, and during virtual presentations. PRINCIPAL FINDINGS: We developed a set of five guiding principles. Measurement that aligns systems with communities toward equitable outcomes: (1) Requires upfront investment in communities; (2) Is co-created by communities; (3) Creates accountability to communities for addressing root causes of inequities and repairing harm; (4) Focuses on a holistic and comprehensive view of communities that highlights assets and historical context; and (5) Reflects long-term efforts to build trust. Using an equity-focused process resulted in principles with broad applicability. CONCLUSIONS: Leaders across systems and communities can use these shared measurement principles to reimagine and transform how systems create equitable health by centering the needs and priorities of the communities they serve, particularly communities that historically have been harmed the most by inequities. Intentionally centering equity across all project activities was essential to producing principles that could guide others in advancing equity.

Conceptual Framework for Patient-Reported Outcome Measures in Clinical Trials of Skeletal Muscle Cramping Experienced in Dialysis: A Kidney Health Initiative Workgroup Report.

Skeletal muscle cramping is a common and bothersome symptom for patients on maintenance dialysis therapy, regardless of modality, and it has not been prioritized for innovative assessments or treatments. Research to prevent or treat skeletal muscle cramping in patients receiving dialysis is hindered by poorly understood pathophysiology, lack of an accepted definition, and the absence of a standardized measurement method. The Kidney Health Initiative, a public-private partnership between the American Society of Nephrology and US Food and Drug Administration, convened a multidisciplinary workgroup to define a set of patient-reported outcome measures for use in clinical trials to test the effect of new dialysis devices, new KRTs, lifestyle/behavioral modifications, and medications on skeletal muscle cramping. Upon determining that foundational work was necessary, the workgroup undertook a multistep process to elicit concepts central to developing the basis for demonstrating content validity of candidate patient-reported outcome measures for skeletal muscle cramping in patients on dialysis. The workgroup sought to (1) create an accepted, patient-endorsed definition for skeletal muscle cramping that applies to all dialysis modalities, (2) construct a conceptual model for developing and evaluating a skeletal muscle cramping-specific patient-reported outcome measure, and (3) identify potential questions from existing patient-reported outcome measures that could be modified or adapted and subsequently tested in the dialysis population. We report the results of the workgroup's efforts, provide our recommendations, and issue a call to action to address the gaps in knowledge and research needs we identified. These action steps are urgently needed to quantify skeletal muscle cramping burden, assess the effect, and measure meaningful changes of new interventions to improve the experience of patients receiving dialysis and suffering from skeletal muscle cramping.

Understanding the Influence and Impact of Stakeholder Engagement in Patient-centered Outcomes Research: a Qualitative Study.

BACKGROUND: Engaging patients and other stakeholders as partners in research offers promise in improving the relevance and usefulness of research findings. OBJECTIVE: To explore the influence and impact of patient and other stakeholder engagement on the planning and conduct of comparative effectiveness research studies. DESIGN: Qualitative study with virtual, hour-long semi-structured interviews. PARTICIPANTS: Fifty-eight researchers and fifty-one partners from a diverse purposeful sample of fifty-eight studies funded by the Patient-Centered Outcomes Research Institute (PCORI). APPROACH: Content and thematic analysis of interview data. KEY RESULTS: Described as an integral, long-term part of the research process, engagement influenced all aspects of the design and execution of studies. Partner influence was also dynamic and iterative, taking different forms over the course of the study. Across studies, we identified 387 discrete examples of influence and classified each as one of five types of influence, derived inductively from the interview data: co-producing, redirecting, refining, confirming, and limited. Most projects exhibited multiple types of influence, with 50 researchers and 41 partners reporting two or more types of influence within a project. Of the 387 examples of stakeholder influence, 306 had at least one reported impact on the study. Such impacts included changes to reflect the needs and preferences of patients or clinicians, as well as impacts on study feasibility, study quality, engagement scope or quality, and study relevance. Both researchers and partners identified multiple types of impact within projects, with 42 researchers and 38 partners reporting two or more types within a project. Because of these observable impacts, researchers and partners described engagement as worthwhile. CONCLUSIONS: Findings provide insights for funders and institutions supporting engagement, measurement efforts, and clinical researchers aiming to conduct engaged research and observe similar influences and impacts in their own studies.