Q.1 Which year in the history of the organization did each of you get involved and what objectives did you consider vital for the organization to address?
Tara: We started working together in the Spring of 2016. From what I remember, another parent was going to step away from monitoring and managing the pfic.org website she created. She inquired on a closed social media group that she was looking for other PFIC parents to take it over and asked who wanted to help. Melanie, Emily and I were all interested and set up a call with the original website builder. We talked about our struggles as parents in a rare disease community and shared our hopes for what would be helpful for families. While we started with just the website, we dreamed of programming and working to educate others about PFIC.
Melanie: 2018-which is when we officially became 501 C3 non-profit. However, it was in 2017 when I began the process of creating what is now the PFIC network, along with co-founders Emily Ventura, Tara Kearns.
Q.2 From founding PFIC.org in 2002 to the formation of PFIC Network in 2018, what would you describe as the key milestones for each of you, in terms of how the organization is and has developed to address the original objectives?
Tara: A key milestone for me was linking to other parents, face-to-face (via video chat) in 2016; it was the first time I met and interacted with anyone else who knew what PFIC was like. Next, in 2018 when we gained our official non-profit status. And then in 2019, we had our first PFIC Family Conference; this event ignited our community—for the first time, many of us felt connection and hope for answers, options and better outcomes, inspiring many to join our Network as Board members, Parent Ambassadors, and charity organizers. Since then, our organization has continued to grow!
1. Robyn Marceca, who founded PFIC.org back in 2002 made some key connections with medical institutions, researchers, and doctors both in the United States and overseas. This was our first key milestone
2. The formation of the social media group on Facebook in 2007 has played a huge role in bringing the worldwide PFIC community together. With such little research and barely any treatment options the knowledge shared this page became not only supportive but informative as well. Often patients share medical knowledge with other patients, that they will then present to their treating doctor. Begin so rare often our clinicians are at a loss on how to best help a PFIC patient- (This is thankfully changing a huge role has been having our own medical advisory board)
3. Facebook group became the PFIC hub and we began to grow in numbers, rather than a handful of people it began to reach a group of around 200 or more (today our group has 572 members) . Growing numbers combined with a united desperation to have effective treatment options created a momentum- to formalise our needs and to provide advocacy. Hence Emily, Tara and I stepped up..
Q.3 How important is 501c3 for the organization, for our readers outside of the United States, what does this mean in terms of the charitable status of PFIC Network
Tara: This piece is key to our organization being a change agent in the medical community. 501c3 allows us to be a not-for-profit charity organization, meaning we can request donations that are tax free for our organization, but also have tax benefits to those who contribute. It allows us to operate for the good of a greater group, and minimize expenses for many important operations and purchases that help us run—from shipping costs, to supplies, every dollar we save helps us pour it back into the community we serve.
Melanie: Having 501c3 status allows us to operate as an official non-profit/charity in the U.S.A. It has been the most significant step we have taken as it allows us to operate legally and financially as a non-profit organisation. While 501c3 status is set up in the United States we legally have a global reach. This means advocacy, research, educational materials can be provided to any PFIC family or patient around the world, and we take on this role with dedication and commitment.
It does however unfortunately mean that any donations outside of the United States are not tax-deductible. However, we are working on this with partners around the world to find new and creative ways to collaborate. For example, in Australia I am currently collaborating with án established charity ‘Liver kids Australia’ in a joint venture, to allow Australian PFIC patients access to our financial assistance program.
Q.4 In terms of the focus of PFIC Network, how much (say out of 100%) of time and effort is invested in work around research programmes, compared to work involved with educational material development, peer support from family matching and advocacy work?
Tara: Currently, about a third of our time and efforts are placed into research. This includes our efforts assisting families in finding various research studies or drug trials. Additionally, our Executive Board and Executive Director have been working to seek research grant opportunities, as well as gathering information from the Parent Advisory Board to garner information about where research should focus. It is our expectation that research will begin to take up about 50% of our time in the coming year. We have seen a shift occur with our focus—in the beginning much of our time and efforts were on development (website, educational material, boards, etc)—and now as those things have been established, we continue to work to stretch our Network and to develop programs of support.
Melanie: I would say at this point in time 30% of my focus is invested in research programs. However, I hope this expands as research is absolutely where my passion lies, as I know this will create the most change!
However, a severe lack of resources and understanding of PFIC means there are major deficits in peer support and advocacy. In many ways we are playing catch up and trying to juggle all the balls at once. But they do go hand-in-hand, knowing that there is research taking place or there is a patient registry for the potential of research can help us in our peer support efforts i.e it allows an opportunity for families to positively channel their desperation i.e by participating in research or joining our registry.
Q.5 What examples of advocacy work has the organisation undertaken and what are the achievements you are most proud of, following advocacy campaigns
Tara: Wow! I feel like there are too many to name! Some examples are Board participation in advocating at Capital Hill, and participating in FDA outreach opportunities. We have also worked with other Rare Disease Organizations who work with legislators to advocate for needs of the Rare Disease Community. We have also created a PFIC Awareness Day; an entire day to promote awareness, education, and support for the disease. In just our second year of celebrating this day, we more than doubled our first years’ fundraising efforts and brought in more than $40,000 for our organization.
Melanie: Currently we are hosting a series of advocacy and educational webinars on a range of topics, including ambassador training, mental health, the drug development process, PFIC 101, subtypes of PFIC….this is almost ground breaking as we will soon have all our educational webinars led by PFIC experts from around the word available on our website!
The ambassador training is for families and patients around the world. The goal is to empower, support and provide resources to our ambassador so they can advocate and offer support to other PFIC families/patients in their own region.
Another example of advocacy is in June last year, we hosted the very first in person PFIC conference/ meet up, we had 22 families attend!
We also advocate regularly across social media platforms, particularly our public Facebook page has an active and growing following.
We have attended a number of significant rare disease and liver conferences many of which Emily has made presentations to pharmaceutical companies, research groups, she has met with clinicians and connected with other key organisations.
I am most proud of our recently launched patient registry because I know it’s going to make a huge difference! I am proud of the advocacy work I continue to do with the pharmaceutical companies ensuring they understand the unique and nuanced needs of our community. I’m also proud of the peer support I have been able to provide to other parents around the world. I will never stop supporting a parent/patient that reaches out for help because I know the horrendous pain of feeling so helpless and alone- we dearly hope we are changing this.
At the ned of the day I am most proud that my 8 year old daughter who has PFIC 2, is proud of me and can see I am doing everything in my power to ensure she has the highest quality of life possible!
Q.6 How important is socioeconomic evidence, sometimes referred to as ‘real world evidence’, as a resource when undertaking advocacy? Particularly evidence that is drawn from directly from patient and family experience,
Tara: Socioeconomic evidence gives PFIC a human aspect that science alone lacks. This evidence is what helps other connects to the heartbreak and devastation this disease causes families and helps to build empathy, compassion and a desire to work to hard alongside our community to reach better outcomes and opportunities.
Melanie: Having scientific evidence is vital, it legitimises our stories and provides much-needed evidence to prove the commonalities of the burden that all stories express. Our stories are compelling and powerful but combining them with the real world evidence, not only propels research forward but validates our experience.
Q.7 What three key words would you use to help medical resource decision makers what most effectively describes the ‘real world’ of the PFIC patient and family from the perspective of a parent,
Tara: Devastating. Scary. Overwhelming.
Melanie: Helpless. Traumatic. Itch.
Q.8 What was it that encouraged you to become involved in the PICTURE study?
Tara: I believe that whenever I am provided an opportunity to share my experience as a caregiver in order to further research, data, and advocacy, I will do it.
Melanie: The ethical and unbiased approach of the PICTURE study made my decision to be involved very easy. Along with the gathering of data across countries and involving both clinicians and the patient and caregiver experience- this is the exact type of research we need.
Q.9 What can HCD Economics do, to fully support PFIC Network in enabling the network to use the data reported from the study for effective advocacy activities?
Melanie: To break the data down in bite sized layperson terminology will be very helpful for us to advocate to the general community. It will also be very helpful for us to use on our social media platforms to help educate and validate our community. Having measurable data that we can take to our clinicians and researchers is also vital for the progress towards better outcomes for PFIC families and patients.
Also having data that we can use in educational brochures and advocacy materials that is scientific and accessible will be a very powerful tool for us.