Identifying patient‐centered outcomes in progressive familial intrahepatic cholestasis: Results from IMPACT

OBJECTIVES: In rare diseases like progressive familial intrahepatic cholestasis (PFIC), lack of evidence results in treatment variability and strategies for families to engage care teams to understand how variability affects overall health are unknown. Patient-centered comparative effectiveness research (CER) has been identified as an approach to address these challenges, but CER-specific knowledge and infrastructure are lacking. We present the results from IMPACT (Identifying research targets by Merging Patient And Clinician Treatment information), a 2-year capacity-building project for patient-centered CER. METHODS: Six learning modules were adapted from the Patient-Centered Outcomes Research Institute to teach participants about CER and elicit feedback for patient-specific CER priorities and processes in PFIC. Participants completed modules and engaged in focus groups (12 virtual and in-person focus groups). Engagement quality was assessed using a validated patient engagement evaluation tool. RESULTS: Participation in learning modules varied between 16 and 31 patient/family participants (mean 22), and 5 and 8 clinicians/researchers (mean 5.3). Focus group participation varied between 7 and 35 patient/family participants (mean 13.1) and 3-35 clinicians/researchers (mean 7.3). Collated data enabled develop of (1) the treatment experience app (TEA): a web-based, community-built information tool to display relevant, research-based, lay language therapeutic approaches, augmented by patient experience information and (2) the IMPACT roadmap, a manual for future PFIC patient-centered research. Engagement assessments demonstrate consistently strong and steadily improving levels of engagement. CONCLUSIONS: IMPACT successfully engaged key stakeholders to build shared understanding of patient-centered CER, define PFIC-specific CER targets, and develop strategies for equitable and sustainable patient-researcher partnerships.