For the last three years the PBC Foundation based in Edinburgh, Scotland, has hosted the International PBC Summit. At the Summit, all stakeholders across the primary biliary cholangitis (PBC) community are brought together to collectively work on ideas that can improve the lives of patients. These stakeholders include patients and patient advocacy groups, clinicians, pharmaceutical industry representatives, specialty nurses and more. I have been lucky and invited to the Summit the last two years to represent the support group I started and facilitate.
At the 2025 International PBC Summit, we chose four primary goals that we wanted to work on, formulating plans to make them a reality. These four ideas are the One-Stop-Shop, Real World Evidence regarding clinical trials, mental health surrounding PBC and the 90:90 program. Although these objectives will take a lot of work, and maybe even years to complete, by working together we can help to meet the unmet needs of those of us with PBC and make our lives better.
A multilingual online resource for all
First is the One-Stop-Shop, a website that both patients and our clinicians can use to find all information regarding PBC. Information will be available in all different languages with individual cultures in mind to help make information accessible to all.
Sometimes it’s hard finding reliable information regarding a rare disease. But this site would have accurate information based off of scientific research and studies. The plan is to have all information regarding PBC available for both patients and their providers to help ensure that patients with PBC are receiving the best care. Instead of having to search all over the internet for various topics related to PBC, everything would be accessible in one location.
This is a wonderful goal that will definitely take time to fully achieve. The biggest thing hindering further advancement of making this a reality is funding. Someone needs to be able to manage the website, keep it updated and provide fixes if something doesn’t work. The groups that worked on this objective have been continuing to work on its advancement since the Summit in April. Though it will likely end up being a long process to reach the final product, it will help so many patients across the globe.
Removing long-term placebos from clinical trials
Real World Evidence is the next objective, and it’s the objective I spent my time working on. I want you to ask yourself and your loved ones a question. Knowing that PBC requires life-long medicinal treatment to slow down disease progression and protect the liver, are you willing to take a placebo for eight years or more so a clinical trial can provide regulators with information regarding a potential treatment? Are you willing to risk liver damage, cirrhosis, the need for transplantation or even death for a clinical trial?
My guess is that you and your loved ones said no. The idea behind Real World Evidence is removing the need for long-term placebo control arms in clinical trials and putting more focus on real world data regarding the treatment and patients. In the PBC community, we have recently seen an issue with Ocaliva not being reapproved for use because of lack of clinical trial data. When patients realized they were on a placebo because of their liver function test results, primarily the ALP not improving, they dropped out of the trial. This issue caused data to be misconstrued.
PBC was formally more diagnosed in middle-aged women. But that’s not the case anymore. Every day more and more people in their 20s and 30s are being diagnosed with PBC. Those of us who were very young at diagnosis are not going to be risking our lives for a regulator to achieve the exact information they want to either approve or not approve a medication. We as patients need to work with the regulators to show them that long-term use of placebos is unethical. It will likely take years to make changes with the FDA and EMA, but it’s necessary. Patients should not be asked to risk their lives just to make a regulator who has no attachment to PBC approve a medication or not.
Mental health support for the PBC journey
Mental health surrounding PBC is a big issue among patients. Finding out you have a rare disease that wants to kill you is scary. When I was first diagnosed, Google told me I’d be dead in 10 years, which would have made me 41 years old. That’s a frightening thought. Being diagnosed with PBC comes with so many challenges that can negatively impact your mental health. I remember feeling despair, feeling incredibly scared and fearful of the future, and experiencing insomnia and severe anxiety. Over time I began to feel a sense of loss. I’m no longer the person I was before I was diagnosed with PBC.
All of these factors can impede your daily thoughts and your daily life. There are so many psychosocial factors at play that making living everyday incredibly difficult. There isn’t much available currently for resources to help us process these feelings and emotions. The goal for this objective is to develop resources that can help us mentally during our journey with PBC. As we know, poor mental health can impact our PBC, as well.
Better care on a global scale
And finally, we continued to work on Project 90:90, which we started to build at the 2024 International PBC Summit. It’s been so rewarding and fun to see this objective progress into reality. This initiative is to Provide Better Care for patients with PBC. The end goal is that 90% of patients receive top quality care standards 90% of the time.
The PBC Foundation had its original launch of the Project 90:90 program in September of 2024, but it continues to be a work in progress. As you can imagine, this is going to be a big community project to reach all parts of the world. Currently we know that many patients with PBC are receiving suboptimal care, which puts patients lives at risk. Implementing this global project will better inform clinicians to help keep our livers healthy and on the right treatment path. I am really looking forward to watching this program continue to grow into what we envision it to be. For more information regarding the Project 90:90 launch, please read about the PBC Foundation’s Project 90:90 launch event.
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