Engaging Patients as Partners in Patient-Centered Comparative Clinical Effectiveness Research

Eugene Washington PCORI Engagement Award

PSC Partners has been awarded funding through the Eugene Washington PCORI Engagement Award Program, an initiative of the Patient-Centered Outcomes Research Institute (PCORI) for a project titled “Building Capacity to Engage in Patient-Led Comparative Clinical Effectiveness Research in Patients with Primary Sclerosing Cholangitis (PSC) at Risk for Cholangiocarcinoma (CCA).”

Understanding PCORI, CER and the Eugene Washington PCORI Engagement Award Program

PCORI is a nonprofit organization with a mission to fund research designed to provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed health care decisions.

Specifically, PCORI funds patient-centered comparative clinical effectiveness research (CER). CER compares the potential benefits and harms of different healthcare options, generating evidence that empowers patients, caregivers and other healthcare decision makers with information to make informed choices that reflect their needs and preferences.

The PSC Partners project is part of a portfolio of projects funded by PCORI to help develop a community of patients, caregivers, clinicians and other stakeholders who are better equipped to engage as partners in all phases of patient-centered CER and to disseminate results of PCORI-funded studies. Through its Engagement Award Program, PCORI is creating an expansive network of individuals, communities and organizations who can leverage their lived experience to influence research to be more patient-centered, relevant and useful.

Project Summary:

Background: There are significant gaps in the research, clinical care, and patient experience related to CCA surveillance in PSC. While the current surveillance standards are suboptimal, we are seeing advances in the testing methods which could significantly improve clinical care and outcomes. However, there is a great need to understand how newer testing strategies compare to current strategies and how they can be implemented into routine care.

Proposed Solution to the Problem: We will work collaboratively with patients, clinicians, and researchers using a comprehensive, engagement-driven stepwise approach to identify questions and concerns most critical to patients at risk for CCA and to assess current CCA surveillance practices and identify gaps in patient–provider communication.

Objectives:

1. Establish a patient-led engagement infrastructure to guide research strategy development related to CCA surveillance in PSC
2. Identify and prioritize potential patient-centered CER questions to support the introduction, evaluation, and implementation of CCA surveillance strategies through a structured, patient multi-stakeholder process
3. Develop and deliver a tailored CER education curriculum for PSC patients, clinicians, and researchers to improve understanding of CER principles and patient-partnered research roles
4. Create a transferable framework for patient-led research prioritization that can be adapted by other rare disease organizations

Activities:

Activity 1 – A patient-led Community Advisory Board (CAB) will be established to serve as the project’s governing and decision-making body and ensure all activities reflect patient priorities and PCORI’s Foundational Expectations.

Activity 2 – PSC Patient Community Feedback Sessions will be conducted to explore the patient journey through the CCA surveillance process and identify key patient-centered outcomes, unmet needs, and evidence gaps related to CCA surveillance and post-diagnosis care in a diverse group of patients and caregivers.

Activity 3 – A detailed community survey will be conducted to expand, validate, and refine insights from the feedback sessions across a broader PSC community.

Activity 4 – A Clinician/Researcher Focus Group will be conducted on CCA Clinical and Research Priorities to compare patient-identified priorities with clinician and researcher perspectives and assess alignment or disconnects from those identified in Activities 2 and 3.

Activity 5 – A CER curriculum for patient, community, and clinician researchers will be developed to build capacity for informed participation in patient-centered CER. The training will emphasize co-learning and position patients and other stakeholders as active contributors rather than passive participants.

Activities 6 and 7 – A Multi-Stakeholder CER Prioritization Consensus Meeting, followed by a post-meeting survey, will finalize and rank the patient-centered CER initiatives related to CCA surveillance.

Expected Outcomes:

• The project builds capacity for patient-centered CER by generating patient-informed research questions, strengthening patient and stakeholder readiness, and aligning perspectives around comparative CCA surveillance strategies.
• These efforts position the PSC community to pursue rigorous, patient-centered CER that addresses real-world decisions and improves outcomes for patients at risk for cholangiocarcinoma.

Project Collaborators:

• PSC Partners Seeking a Cure will lead the project and be responsible for the conduct, reporting, and deliverables associated with the project.
• The Cholangiocarcinoma Foundation (CCF) will be a key collaborator for the project, integrating feedback and priorities from patients diagnosed with CCA. CCF is a global nonprofit organization established in 2006 with the mission to find a cure for bile duct cancer and improve the lives of affected individuals and families.
• The International Primary Sclerosing Cholangitis Study Group (IPSCSG) is a global network of researchers working to advance scientific knowledge as well as improve clinical management and outcomes of PSC. Founded in Oslo in 2010, the IPSCSG coordinates research projects among leading institutions worldwide to avoid redundancy and enable robust, large-scale studies in this rare disease population.

Call To Action:

Your voice is essential to the success of the PCORI Project. PSC Partners is conducting a series of community engagement, education and capacity-building activities, and we invite patients, caregivers, relatives, researchers, and clinicians to join our mailing list for updates and opportunities to get involved.