Patient-centered Urogynecology Research: Designing a Clinical Trial with a Patient Expert Panel

Objectives: Patient-centered research requires partnering with patient experts to help bridge the gap between health research, policy, and patient-centered practice. Incorporating the patient perspective early in the study design phase shapes health research and treatment guidelines and ensures more meaningful outcomes. The effect of engaging a Patient Expert Panel (PEP) on study design and protocol changes in developing and executing a multi-center randomized trial comparing beta-agonist medication versus onabotulinumtoxinA for the treatment of urgency urinary incontinence (UI) is presented.

Methods: Patient experts were engaged from 5 clinical sites across the U.S to form a Patient Expert Panel (PEP) in the Spring of 2021. Requirements included having urgency UI and being interested in the role. The PEP was engaged on a continuum starting from study conception (submission of letter of intent) through study implementation. Our engagement strategy included Patient-Centered Outcomes Research Institute (PCORI®) rubric components of input, consultation, collaboration, and shared leadership. (1) The objectives of PEP engagement as part of the research team (Figure 1) included providing patient perspective and decision making for: 1. Study design; 2. Primary and secondary outcomes; 3. Study endpoints; 4. Care and research participation barriers; 5. Recruitment strategies; 6. Patient facing and study materials.

Results: The PEP includes a diverse panel of 9 patient experts with urgency UI who were identified by each site’s clinical and/or research team. To date, 10 virtual meetings have been completed, including a training session of research fundamentals as part of co-learning.(1) Key questions were reviewed with the PEP resulting in several protocol changes directly based on their guidance (see Table). Key changes have included: 1. Primary outcome changed from a single outcome (UI symptom severity) to 2 co-primary outcomes (UI symptom severity and treatment satisfaction); 2. Increase in sample size due to co-primary outcomes; 3. Change from planned UI primary outcome questionnaire to alternative based on relevance and ease of understanding; 4. Primary endpoint change from 6- to 3-months; 5. Decreasing other secondary outcome questionnaires from 10 to 3 based on relevance; 6. Identification of barriers, particularly transportation for rural participants, leading to final budget adjustments; 7. Addition of community engagement studios to explore geographic differences in care and research barriers between 5 clinical sites; 8. Modifications of study materials to improve patient appeal.

Conclusions: Early PEP engagement resulted in several substantive protocol and procedural changes addressing relevance of information and ease of understanding of materials for this randomized trial. These changes will improve the efficiency and patient-centeredness of our study and will help to ensure relevance to the intended patient population. PEP engagement will continue throughout this trial's conduct and dissemination phases.

Reference: 1. https://www.pcori.org/sites/default/files/Engagement-Rubric.pdf