Results of IMPACT Published in Peer-Reviewed Journal

We are excited to announce the publication of “Identifying patient-centered outcomes in progressive familial intrahepatic cholestasis: Results from IMPACT” in the Journal of Pediatric Gastroenterology and Nutrition.

IMPACT (Identifying research targets by Merging Patient And Clinician Treatment information) was a two-year project (2023-2025) funded through the Eugene Washington PCORI Engagement Award (EASO-30455) program, an initiative of the Patient-Centered Outcomes Research Institute (PCORI). The project brought together PFIC patients, family members, clinicians, and researchers to build shared knowledge and tools for patient-centered comparative effectiveness research,  a research approach designed to answer the questions that matter most to patients and families living with a condition.

The publication of these results marks an important milestone and a launching point. Building on the foundation IMPACT established, PFIC Network has received a new PCORI Engagement Award (January 2026–December 2027) to form a research consortium to plan future patient-centered comparative effectiveness research studies in PFIC.

Thank you to everyone involved—for your time, your trust, and your partnership in shaping the future of research in PFIC.

Data from the PFIC Network Patient Registry is making news! Liver Disease News recently published an article highlighting the first peer-reviewed paper reporting findings from our registry, covering how pruritus impacts sleep, family quality of life, and the financial burden faced by PFIC families. It’s a great plain-language overview of the paper for anyone who wants to learn more.

In our recent project, IMPACT, people living with PFIC, parents, doctors, and researchers learned together what patient-centered research means. We focused on how research can better address everyday challenges of life with PFIC. During this project, which was funded through the Eugene Washington PCORI Engagement Award program, an initiative of the Patient-Centered Outcomes Research Institute® (PCORI®), our community also shared the questions they would want research to address.

Now, we’re ready for the next step. We’re excited to share that PFIC Network has received funding for a new Engagement Award project.

Our new project, “Building Capacity to Engage in Patient-Centered CER on Progressive Familial Intrahepatic Cholestasis (PFIC),” will run from January 2026 to December 31, 2027.

Together, we will decide which research questions are most important to study first. These will become our PFIC research priorities.

We will also build a PFIC research group made up of people living with PFIC, parents, doctors, and research centers. This group – a patient-centered research consortium – will work together to plan future studies that follow these priorities. Community members like you will help guide the work every step of the way. We’re excited to share more soon and invite you to get involved as this project begins.

Exciting news for young PFIC patients in the United States! The U.S. Food and Drug Administration has approved a label expansion for maralixibat. The expanded label includes use in PFIC patients 12 months and older as well as the higher concentration formulation evaluated in the MARCH Phase 3 study. Congratulations to the Mirum Pharmaceuticals team ?

Exciting new for PFIC patients in Europe!  The European Commission has granted marketing authorization for maralixibat for the treatment of progressive familial intrahepatic cholestasis in patients three months of age and older. 

View Mirum’s press release to find out more!

About Us

Get Involved

The Disease