Closing Remarks
Project IMPACT introduced the PFIC community to patient-centered comparative effectiveness research (CER) and served as the first structured effort to map available PFIC treatments alongside both clinical and patient-reported outcomes.
Using the Treatment Experience App (TEA), interactive learning modules, and focus groups, the project provided foundational knowledge to guide future patient-centered CER efforts in PFIC.
While this work established a strong starting point, there is still much to be done to advance patient-centered research and improve clinical management of PFIC.
We will work to ensure that the insights gained from this project will help shape future studies and foster ongoing collaboration among patients, caregivers, clinicians, and researchers to address the most pressing treatment questions in PFIC.
Acknowledgements
We extend our heartfelt thanks to everyone who made the development of this roadmap possible. Project IMPACT was a truly collaborative effort—grounded in shared learning, mutual respect, and a deep commitment to improving research and care for people living with PFIC.
We are especially grateful to our Project IMPACT team—Melissa Kochanowsky, Gitta Lubke, Alexandra Perez, Jim Squires, Emily Ventura, Emily Perito, and Sarah Brengosz—for your leadership, insight, and steadfast support throughout every phase of project IMPACT.
To all the patients, parents, clinicians, and researchers who participated in the learning modules, focus groups, and Treatment Experience App: we could not have accomplished this project without you. Your experiences, ideas, and feedback shaped every part of this work. Thank you for diving into this effort with honesty, courage, and generosity. Your voices are at the heart of this roadmap and will continue to guide PFIC research for years to come.
We also thank Paolo Catalla and his team from Semi:Formal for their creativity, thoughtful design work, and ability to translate complex ideas into clear, accessible visuals that brought the IMPACT Treatment Experience App to life. Special thanks as well to the team at Scene and Steve Fedak for developing this Roadmap website and helping make this resource easy to navigate, visually engaging, and broadly accessible for all PFIC Network community members.
This project was supported by a Eugene Washington PCORI Engagement Award (Contract #30455) from the Patient-Centered Outcomes Research Institute (PCORI). We are deeply grateful for PCORI’s support and their commitment to building infrastructure for patient-centered comparative effectiveness research in rare diseases.
Thank you to everyone involved—for your time, your trust, and your partnership in shaping the future of research in PFIC.
