For the last four years, the PBC Foundation based in the U.K. has hosted the International PBC Summit. The Summit brings together all different stakeholders among the primary biliary cholangitis (PBC) community, including patient advocacy groups, pharmaceutical partners, clinicians, specialty nurses, statisticians and more. The goal behind the Summit is for all the stakeholders to work together to come up with ideas to provide better care for patients living with primary biliary cholangitis while putting our patient voices at the front and center. I’ve been lucky enough to be able to attend the last three Summits. This year’s theme was all about collaboration and the impact it can have on patients’ lives.
Improving care with Project 90:90
Over the last four years, there have been four projects that have been started. Project 90:90 is perhaps the most important of them all. Project 90:90 is the goal that 90% of patients living with PBC receive care meeting the clinical guidelines 90% of the time. This is a huge goal, especially considering we want this to occur daily across the globe. However, some countries have already made progress. One example is Portugal, thanks to the collaboration between the PBC Foundation and CBP Portugal.
I am hopeful that one day this is the standard expectation and the level of care that all patients living with PBC experience.
Real-World Evidence: Making clinical trials safer for patients
The second project revolves around bringing more real-world evidence into clinical trials. Currently, regulators require that long-term clinical trials for PBC therapies include placebo arms — meaning patients in the trial can go eight years or more on a placebo, receiving no treatment for PBC. That is a big ask, and a big risk to put on patients. We risk liver failure and even death for long-term placebo clinical trials. It’s unethical.
The goal of the Real-World Evidence project is to work with regulators to come up with some sort of solution that reduces the risk to patients while still providing regulators with the information they need to approve a medication. We also know that patients realizing they are on placebos can increase the chance they drop out of the clinical trial, which can completely skew the data. We saw this happen with Ocaliva, which was removed from the market in the U.S.
Regulations regarding clinical trials haven’t changed in decades; now is the time we can all work together to really put patients first and figure out some sort of compromise that can make both parties happy.
A library of PBC resources
The third project is PBC.International, which was formerly known as the “One Stop Shop.” I am really excited about the progress that has been made with this project. The plan is to develop a repository of all science-based factual information relating to PBC that both patients and clinicians can easily access. This won’t include new content, but instead provide already available resources in one place for quick accessibility. A mock-up design has been made, and funding is needed to help move this project forward and make it available online.
Mental Health and PBC: Helping care for the mind
The fourth project is called Mental Health and PBC, and this project is something that is very important to me. Not only have I struggled with my mental health regarding my diagnosis, but my nonprofit, Friends of the PBC Foundation, is the project manager moving forward.
Our goal is to have a website with instructional videos by trained professionals to address different issues related to mental health. For instance, a patient could watch a short video on breathing exercises to help calm the mind, but could also opt for a video that offers more advanced help.
This project is set up into three phases, and we are currently still in phase 1. We have a survey that was developed by patients and clinicians to help determine how patients’ mental health has been affected by PBC. We know we have a lot of work to do and a lot of funding to raise to make this a reality. But this project will help so many patients around the world during their journey living with PBC.
Liver health beyond PBC
This year we took on a fifth project: Lifestyle Liver Health. Overall liver health is important for patients with PBC. It’s so common to have overlapping issues in combination with PBC, such as fatty liver disease. This project aims to offer resources specific to different liver issues, as well as more information regarding diet. Keeping our livers healthy is so important for those of us living with primary biliary cholangitis.
The takeaway
I really love the International PBC Summit.
My favorite thing about the Summit is the opportunity to connect with other patient advocacy groups from across the world. I have developed true friendships with other patients. All of us working together is how we make real impactful change for the primary biliary cholangitis community.
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