Roadmap Part 1 — Engagement Guidance

Guidance for Forming Successful Multi-Stakeholder PFIC Research Teams

Forming successful multi-stakeholder research teams will be essential to ensure that patient-centered comparative effectiveness research (CER) in PFIC reflects the real-world needs, priorities, and experiences of patients and families.

IMPACT participants identified key stakeholder partners who should be involved, the types of support needed for successful collaboration, and strategies to sustain partner engagement across the lifespan of a research project.

Key Stakeholder Partners for Patient-Centered CER in PFIC

Participants recommended forming a consistent core team that includes:

Patients across ages, disease stages, and geographies
Parents to provide critical caregiver perspectives
Clinicians from both specialty centers and general hospitals
Researchers (clinical and lab)
Statistician to ensure rigorous study design, data collection and analysis

Participants also recommended involving rotating members relevant to specific stages of the project, including:

Psychologist to support patient-centered engagement and address emotional or behavioral aspects of research participation.
Child Life Specialist to help capture pediatric patient voices
Research Assistant to assist with study coordination and logistics
Communications Specialist to support broad dissemination of research findings

Support for Multi-Stakeholder Research Teams

Participants identified several types of support needed to ensure that all stakeholders can work together as equal partners:

Support for pediatric engagement

given that a significant proportion of PFIC research participants will be children and adolescents, use pediatric-specific strategies to ensure meaningful input from younger patients, including:
- child life specialist-led sessions
- play-based methods
- teen advisory boards

Flexible participation options

offer multiple ways to engage – such as core team roles, short-term advisory roles, or rotating participation
allow input asynchronously and schedule meetings at times that accommodate multiple time zones

Clear expectations and structured tasks

define roles, responsibilities, and timelines early
break tasks into manageable parts to support participation

Training on research processes and terms

provide plain-language written guides
share brief (under 10 minute) explainer videos
offer 1:1 mentorship if possible

Opportunities to meet in person

plan at least one in-person meeting early to build trust, strengthen communication, and support collaboration

Support for pediatric engagement

given that a significant proportion of PFIC research participants will be children and adolescents, use pediatric-specific strategies to ensure meaningful input from younger patients, including:

- child life specialist-led sessions
- play-based methods
- teen advisory boards

Compensation for time and effort

offer financial compensation to recognize time contributions and reduce barriers to participation

Strategies to Sustain Partner Engagement

Participants emphasized that building flexibility into the engagement structure is critical, as some studies may span several years, and participants’ availability and capacity may change over time.

To sustain engagement, participants recommended:

Problem

Cost and accessibility of treatments and surgeries

Solution

Patient treatment and self-advocacy education

Problem

Lack of standard care practices for PFIC

Solution

Develop consensus treatment plans

Problem

Lack of designated “centers of excellence”

Solution

Establish PFIC centers of excellence

Problem

Variability in provider knowledge of PFIC subtypes

Solution

Facilitate provider education on the different PFIC diseases and respective treatment choices

Problem

Lack of communication between providers at different centers

Solution

Develop realistic treatment guidance for under-resourced care centers

Problem

Clinicians not involving patients as partners in care decisions

Solution

Develop guidance for patient-centered care decision-making

Problem

Lack of comprehensive evidence regarding patient-centered treatment outcomes

Solution

Patient-centered comparative effectiveness research

Motivations for Participation in Multi-Stakeholder Research Teams

Most IMPACT participants expressed a desire for varying levels of involvement in future multi-stakeholder research teams, ranging from providing occasional advisory input to co-investigator roles.

Participants identified several key motivations for participating in research teams:

ensuring focus on patient and caregiver priorities
improving care outcomes
contributing to community building
contributing to efficient, feasible, and patient-engaged research

For practical tools and worksheets to help you structure patient & stakeholder involvement in study planning, download the Researcher Companion (see p.11)

Engagement Guidance

Communication & Dissemination Strategies

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