Reflecting On Our Journey & Preparing For Our Future

A message from our Executive Director, Emily Ventura:

In preparing to host our 5th PFIC Family & Scientific Conference this April, I have been reflecting on our collective journey as a community. In these reflections, I am filled with gratitude & purpose. When I first began my PFIC advocacy journey, I vividly recall the uncertainty that I felt, both as a leader of a nascent organization and as the mother of a 5 year old girl who had just been transplanted. I knew I wanted to make a difference, yet I had no idea where to begin. Our rare community faced challenges, and I was determined to find a path forward. 

One of the first steps forward I took was to work to live up to the “network” part of our name. I realized we needed to expand our idea of community beyond just PFIC patients and families. My first introduction to the research community was through the parent advocates who paved the way before me, fostering important relationships with those who now sit on our Medical Advisory Board(MAB). These introductions led me to attend my first scientific meeting, the AASLD conference in Boston, four years ago. It was my first opportunity to meet our MAB in person. The dedication these scientists and clinicians had to understanding our rare disease left a profound impression on me and filled me with hope for a brighter future for PFIC families.

This meeting coincided with a formative period for PFIC Network. We had just secured non-profit status and I was ready to share with the scientific and medical communities our first big goals: rebuild the pfic.org website, create lay-friendly educational materials and serve as a resource for community connection and support. During that first meeting, our MAB members expressed a desire to get to know our community and to work to understand our community’s unmet needs. 

Little did I know that the phrase “unmet need” would become so significant and central to our work here at PFIC Network. Through those early discussions with the research and medical communities, the power of storytelling emerged as an important tool to articulate the unmet needs of our community. However, what they helped me to understand is that while sharing our patient and family stories moves people to care about our cause, researchers need data and facts to understand how best to help us. Documenting our needs in a systematic way can help guide scientists on how best to pursue research that produces meaningful  impacts in the lives of PFIC patients and their families.

This past year, the AASLD conference returned to Boston, to the very same convention center as 4 years ago. Since that very first Medical Advisory Board meeting, I am proud to say we have accomplished many of our initial organization goals! Our award-winning patient education brochure is available in 19 different languages and readily accessible to patients through pfic.org. We regularly get messages from newly diagnosed patients all over the world, who no longer have to search far and wide to find support and connection with other PFIC patients. 

This time in Boston, I sat around the table with our Medical Advisory Board members to discuss the many tools that we have put in place for our community to express their unmet needs. We discussed the PFIC Network Patient Registry, Project IMPACT and the Family & Scientific Conference. We discussed the importance of the latest research being communicated directly to families, and brainstormed ways for community members to have a closer look at the latest knowledge and scientific advancements in PFIC:

• The PFIC Network Patient Registry is a powerful tool to share data with researchers. If all those who participate in the registry fill out their follow up survey every 6 months, we can begin to see important trends and share those with researchers, who can then explore new discoveries.

• Project IMPACT is a way for patients and families to connect and build something together with clinicians and researchers. Together, we can unlock treatment experiences and create a research roadmap that meets the needs of the PFIC community.

• The 2024 Family & Scientific Conference will be hosted in partnership with Cincinnati Children’s Hospital and Medical Center. CCHMC will be hosting research workshop sessions where researchers will come together and talk about the latest in PFIC research, including novel PFIC models and gene therapy. In addition to the research workshop and family-oriented sessions, we will be hosting two patient and researcher roundtables, to promote dialogue to better understand how we can empower change together.

These initiatives are all foundational blocks that reflect our commitment to bridging the gap between patients and researchers, with a shared goal of advancing PFIC research. We want to work with the medical and scientific communities to build a truly collaborative research network that values the patient voice and community participation as valued partners.

These initiatives are all foundational blocks that reflect our commitment to bridging the gap between patients and researchers, with a shared goal of advancing PFIC research. We want to work with the medical and scientific communities to build a truly collaborative research network that values the patient voice and community participation as valued partners.

As I contemplate the trajectory of PFIC Network over the past few years and look forward into our future, the significance of addressing the unmet needs of our community remains paramount. Our organization, now more mature and robust, stands at a pivotal juncture. We have the opportunity to forge important partnerships with clinicians and researchers, fostering actionable solutions to our community’s most pressing challenges. Having shared our stories, it is imperative that we come together to identify and prioritize our most pressing needs. This collaborative effort will serve as the catalyst for tangible progress and transformative change within our community. Together, let us continue to advance our cause with unwavering determination and unity.

With Hope,

Emily Ventura

Executive Director and Co-Founder of PFIC Network