Five Year Plan
Building on the momentum of project IMPACT
PFIC Network has developed a five-year plan to establish the remaining infrastructure, governance, and partnerships needed to initiate and sustain patient-centered CER in PFIC.
This plan is organized across three phases—short-, medium-, and long-term—each with defined objectives that build upon one another. Together, these efforts will create a durable, patient-led research infrastructure capable of designing and conducting meaningful CER aligned with the priorities of PFIC patients.
Short Term – Building Further Capacity
- form a steering committee of 4–6 PFIC research centers, 4–6 patient family representatives, and 2–3 nursing professionals
- develop a Patient-Centered Research Consortium governance charter that embeds equitable decision-making across all stakeholders
- finalize a prioritized research agenda based on IMPACT findings and additional community input
- publish a consortium development framework for replication by other rare disease groups
Outcomes:
A functioning, patient-centered research consortium with the infrastructure and tools to co-lead future PC-CER studies.
Medium Term – Putting Capacity into Practice
- evaluate and refine consortium processes using member feedback
- transition the Steering Committee into a formal Patient-Centered Research Consortium
- institute additional committees and rotating stakeholder partners
- use the governance charter and research agenda to design and prepare for the first PFIC-specific patient-centered CER study
Outcomes:
The Patient-Centered Research Consortium launches the first multi-center CER study in PFIC aligned with patient priorities and shares its approach as a model for other rare diseases.
Long Term – Sustaining and Scaling the Work
- maintain ongoing Patient-Centered Research Consortium involvement across the full research lifecycle (i.e. review, dissemination, and implementation of study findings)
- update the research agenda using structured community input
- integrate study results into the IMPACT Treatment Experience App and clinical care guidelines to support real-world treatment decision-making
- recruit and onboard new consortium members
Outcomes:
A sustainable, scalable research infrastructure led by patients and embedded in PFIC Network’s operations, advancing patient-centered CER in PFIC and inspiring similar models across the rare disease research landscape.
