Remaining Capacity Needs Post-IMPACT
PFIC Network launched project IMPACT to build a foundation for patient-centered CER in PFIC.
IMPACT brought together PFIC patients, parents, clinicians, and researchers to:
Learn principles of patient-centered CER and multi-stakeholder research engagement
Develop tools to support long-term collaboration, including the Treatment Experience App (TEA) and the IMPACT roadmap.
Identify PFIC CER priorities informed by PFIC patients’ lived experiences and clinical insights.
Through educational modules, focus groups, and project team meetings, IMPACT fostered shared knowledge and trust among patients and researchers while producing tools and research priorities that will support future patient-centered CER in PFIC.
Despite the progress made through IMPACT, two critical gaps must be addressed before we can pursue the first patient-centered CER study in PFIC.
Remaining Gaps
No dedicated infrastructure exists to support sustainable, PFIC-specific patient-centered CER.
Currently, no dedicated infrastructure exists to support sustainable, PFIC-specific patient-centered CER. Existing pediatric liver disease consortia mostly aim to advance clinical PFIC research. They do not focus on patient-centered comparative effectiveness research (CER), nor do they engage patients and families as active partners in governance or research design.
Research targets identified by the PFIC community in IMPACT remain unprioritized, with no process to select which to pursue first.
Research targets identified through IMPACT still need to be refined and prioritized through a structured, collaborative process that considers both scientific feasibility and patient-perceived importance.
Looking Ahead
- To address these gaps, PFIC Network has applied for a second PCORI Eugene Washington Engagement Award to support the PATH project (Patient-driven engagement for Advancing Treatment Research).
- PATH will establish the remaining infrastructure needed to launch patient-centered CER in PFIC by forming a multi-stakeholder consortium, creating a governance charter, and ranking the patient-centered CER priorities by scientific feasibility and importance.
