Rare Leader: Emily Ventura, Chief Executive Director, PFIC Network
December 23, 2021
Name: Emily Ventura
Title: Chief executive director
Organization: PFIC Network
Disease focus: Progressive Familial Intrahepatic Cholestasis (PFIC) is a general term that represents a group of rare genetic (inherited) disorders that cause a progressive (increasingly severe) liver disease and can lead to cirrhosis and end-stage liver disease. Often the hallmark feature of this disorder is severe and debilitating pruritus (itching).
Headquarters: Clay City, Kentucky
How did you become involved in rare disease: I have a daughter that has PFIC. She was diagnosed at five months old. At the time of diagnosis, we were not given much information, no type of connection with other patients or families. There was a general lack of support available because of how rare the disease was and there was little information out there. It took about three years until I found any sort of support group. The day that I found the support group—it was on Facebook—my world opened up. Mostly it was a group of people who were asking the same questions. I didn’t necessarily have more answers, but I found relief and hope in the idea that there were just others who understood exactly what we were going through.
Previous career: ICU nurse
Education: Bsn Registered Nursing/Registered Nurse from Carroll University
Organization’s mission: We aim to improve lives of patients and families all over the world. Our activities include peer support, educational opportunities, outreach and awareness, advocacy, and supporting research as we’re growing. We aim to improve the lives of those in our international community.
Organization’s strategy: Peer to peer mentorship, inclusivity, collaboration, and empowering. We work to identify the needs of our patient population, our peers. We have an inclusive environment. The disease is challenging to diagnose. There are multiple different subtypes of the disease, each carrying their own unique burden. There’s an intermittent form of the disease called benign recurrent intermittent cholestasis (BRIC), which presents symptoms that are just as severe and can turn into the progressive disease. Sometimes patients don’t receive an actual diagnosis, but they’re suspected to have PFIC. If they find our community, they found us for a reason. We attempt to be inclusive with our activities because all patients need to be supported. We’re very collaborative. We work with organizations and other patients throughout the world to try to provide resources for each other. Resources for PFIC patients and families are scarce, just as in other rare diseases. We may not ever be able to meet every need our patient community has by ourselves, but we can certainly be resourceful and work with others in the space all over the world to fill the gaps. When you bring it all together on a global scale, we can help support each other. Collaboration is also key in moving treatment options forward. We find strength in numbers by working together as a global community. Last but not least, we aim to empower our community to step up, speak up, and work with us to learn more about our disease. True impact and change will come from within our community, and together we can move mountains.
Funding strategy: We seek grant opportunities from a lot of the rare disease umbrella organizations like Global Genes, Everylife, and NORD when they’re available. We were recently awarded the Rare as One grant with the Chan Zuckerberg Initiative for which we are very proud. We also work with industry partners on grants for activities to try to further our mission as well. In addition to grants, we host community fundraising efforts and events. A few years ago, we empowered the community through a PFIC Awareness Day and hosted a series of lemonade stands to just see how far we could get. Those efforts have grown. There’s been lemonade stands, marathons, bingo nights, and raffles that have been hosted all over the world to try to raise money for PFIC. I’d say this is the most unique and best part of our funding strategy and we are excited to grow our fundraising efforts.
What’s changing at your organization in the next year: We’re growing rapidly. We are starting to focus on supporting research and providing research grant opportunities, opportunities for the patient community and scientific community to come together in collaboration, and to support the rapid growth of research and bringing the patient voice to research efforts. We’ll be taking on a few more staff members, bringing in consultants and advisors who are outside our volunteer board, and building up our volunteer program and community engagement program. We are growing into a sustainable foundation that will continue to identify needs, create solutions, and find strategies to address unmet needs on a larger scale.
Management philosophy: I’m new to the management realm. My early career was as a nurse, and as a nurse, I worked to mentor and educate others, to try to empower others to be good nurses. I feel as though that’s carrying over into managing the foundation. I think mentorship is huge for all those who are working on the cause, both volunteers and staff. Working with people to grow their own abilities, to grow as people, will help to maximize their contribution to the cause. In turn, people will organically buy into the work they do in the organization, feel directly connected to the cause and feel good about the work that they’re doing for and with our community.
Guiding principles for running an effective organization: Allowing those who are participating to have a voice and allowing everyone to be heard. Setting milestones and working together to create strategic action plans. At the end of the day, let everyone connect with the patient community and see the true impact that they are making.
Best way to keep your organization relevant: I’d say the same thing. Allowing those who are involved to have a voice, community members, regardless of literacy level or geographic location. When you’re looking at disease specific, regardless of subtype or point in your journey, allowing everybody to tell their story, to have a voice, to identify their needs. Everything is relevant. We seek to keep the community connection strong, make sure that everybody is heard, and try to give everybody a seat at the table so that we can address needs from the core as we grow. We are working on a big community engagement project right now to help add structure to this very thing. Allowing community members easy, attainable ways to be involved at various different levels of our organization through councils, committees, advisory boards, governing boards, etc.
Why people like working with you: I’m relatable, and I’m willing to be vulnerable. Sharing is natural for me, even if it’s not positive. I’m probably too honest and genuine when I speak, but I guess it’s easy to connect with and work with people in that way. I also value the people that I work with and for. I’m flexible and understand that everyone has a unique workflow. I value accommodating varying needs when it comes to time, capacity, abilities, etc., to allow people to not be controlled by the work, but rather allow them to work within their capacity.
Mentor: In the rare disease space I have two: Carrie Ostrea [Little Miss Hannah Foundation] and Andra Stratton [Lipodystrophy United] who were two of my earliest and most influential mentors. They helped guide me on my journey to advocacy and helped me to grow and continue to stay with me. I consider them friends now and they’re always there to continue to guide us through this growth.
On the Job
What inspires you: Working with people in the community. If people reach out now, they have a place to reach out to. Sometimes I can help and sometimes I can’t. Sometimes others can help and sometimes they can’t. But the fact that people can reach out and talk to somebody who experiences PFIC, who understands, to provide that point of connection, is extremely hopeful and inspiring. It’s something that I wish I would’ve had back in the early days. I hope that everyone who has a diagnosis of PFIC will be able to find some point of connection.
What makes you hopeful: The patients are speaking up and science is making advancements. I feel as though there’s still a lot of unanswered questions, but patients are bringing a voice to the disease and the scientists and researchers in the space are a great group of people who are willing and able to provide the solutions. I see that growing day-to-day. I feel hope in the advancements that are being made and hope that the patients, as long as people continue to speak up, will continue to get their needs met.
Best organization decision: I would say creating the family conference as our very first programmatic activity, having that connection point in person back in 2018, that was our first program. That helped to build our foundation from the core.
Hardest lesson learned: Being in position to help is wonderful, but you’re not able to help everybody. These are my peers, my friends, I want to be able to help everybody. Being able to understand your position as somebody who connects with patients in this rare community, in the PFIC community, to understand that line and that boundary, it’s hard to be in that position and try to feel hopeful when the other side feels hopeless. That’s an ongoing lesson. I don’t know if I’ll ever completely learn it, but I definitely grow each time we’re facing a challenge with a family.
Toughest organization decision: Establishing the goals, vision, and direction of our patient registry.
Biggest missed opportunity: Building a volunteer program and a solid infrastructure from the very beginning. It’s something that we’re catching up with and some days feels like a scramble to solidify an infrastructure now that we’ve grown. We had an opportunity to do that early and focused on building programs first, because we wanted to provide resources for people. If I could go back in time, I’d focus on the infrastructure first, because then the ability to provide resources and make advancements would feel endless.
Like best about the job: I get to connect with people who aren’t able to connect without us.
Like least about the job: I never turn it off. There’s no off button. I’m also now a businessperson, which is not necessarily my niche.
Pet peeve: Watching someone bite into a popsicle, it gives me the chills.
First choice for a new career: I’d stick with nursing but advance in a research-type direction and go on a path to find a cure for PFIC. That’s the obvious answer. But if I’m being honest, I’d also consider becoming a pastry chef.
Most influential book: Chasing My Cure: A Doctor’s Race to Turn Hope into Action; A Memoir by David Fajgenbaum
Favorite movie: The original Willy Wonka and the Chocolate Factory
Favorite music: I like cello music
Favorite food: Chocolate
Guilty pleasure: Bourbon tastings
Favorite way to spend free time: Rock climbing
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