Motivation
Why did we start project IMPACT?
At the 2022 PFIC Family & Scientific Conference, patients, parents, clinicians, and researchers gathered to talk about the biggest challenges facing the PFIC community.
In roundtable discussions, parents shared stories of navigating high-risk surgeries like biliary diversion and liver transplant, often without clear or consistent guidance. Many expressed feeling isolated in their decision-making, unable to find real-world data that could help them understand the benefits, burdens, and likely outcomes of each treatment option.
These conversations revealed an urgent need for patient-centered evidence on the real-world benefits and burdens of PFIC treatments to better support care decisions.
PFIC Network recognized patient-centered comparative clinical effectiveness research (CER) as an opportunity to address evidence gaps and enhance the clinical management of PFIC.
What is patient-centered comparative clinical effectiveness research (CER)?
Comparative clinical effectiveness research (CER) is research that compares the outcomes, including possible benefits and harms, of two or more available health care options, to determine what works best for which patients, under what circumstances. Outcomes are the measurable results, positive or negative, of a health care option.
Patient-centered CER compares outcomes that matter most to stakeholders, such as patients and those who care for them, healthcare providers, and healthcare advocates.
Patient-centered CER requires the engagement of patients stakeholders as active partners in research. By sharing their lived experience and expertise, patients and stakeholders influence research to be more patient-centered, relevant, and useful.
The Patient Centered Outcomes Research institute (PCORI) is the leading funder of patient-centered comparative clinical effectiveness research in the United States.
Yet, PFIC lacked the infrastructure to make patient-centered CER possible:
No established mechanisms for patients to help shape research questions or define meaningful outcomes
No process to identify or prioritize research grounded in lived experience
No tools or training to support patient involvement in the research process
To help close these gaps, PFIC Network launched Project IMPACT.
IMPACT was a two-year initiative to build foundational capacity for patient-centered CER in PFIC.
- enable the first patient-centered CER study in PFIC.
- generate real-world evidence that supports families through complex treatment decisions and improves quality of life for those affected by PFIC.
