Uniting Research Efforts to Create a Future Without PSC

PSC Partners International Collaborative Research Network

“Together in the fight, whatever it takes!” is a rallying cry for our community of PSC patients, caregivers, loved ones, and friends. For decades, PSC researchers and clinicians have continued the fight, investigating various treatments for PSC and working tirelessly to improve quality of life and outcomes for their patients. PSC Partners has been inspired by the “Collaborative Network Model” defined by the Castleman Disease Collaborative Network (CDCN) in 2016. This model is described as a formal connection of key stakeholders with the same goal and core values: identifying treatments and a cure for rare diseases. In the spirit of this model, PSC Partners now invites researchers, clinicians, and industry partners to join our ranks as we unite our efforts through the PSC Partners ICRNetwork (International Collaborative Research Network) PSC Partners will officially launch the ICRNetwork at the Annual Conference in June! Stay tuned for more information.

At PSC Partners, we continue to collaborate with PSC experts through the Research Grant program, Scientific/Medical Advisory Committee, externally-led Patient-Focused Drug Development (PFDD) Forum with the FDA, our annual patient/caregiver conferences, attendance at national and international scientific meetings, and more.

Through the ICRNetwork, we will champion the patient voice as we work with our partners around the world to:

• integrate international databases
• spark collaboration
• and facilitate patient-driven research projects and patient-focused drug development

Check out the sections below to preview our journey.

Downloadable Handouts

PATIENT COMMUNICATION: DRAFT RESEARCH AGENDA

DRAFT RESEARCH AGENDA

We hope these documents will serve as educational references and resources that you may come back to frequently as you consider your own hopes and needs for PSC research. Don’t forget that YOU are part of this Collective Network!

A Patient-Prioritized Research Agenda And Strategic Research Plan

The PSC Partners ICRNetwork will support the development of a Strategic Research Plan (SRP) with the goal of accelerating research towards treatments and a cure for PSC. The first step is to develop a patient-prioritized research agenda. PSC Partners has collected hundreds of research questions directly from PSC patients and caregivers through the 2021-2022 ROADMAP Initiative (Research Opportunities and Development Meetings to Accelerate Progress). In addition, the PSC Partners team has been meeting with expert PSC researchers to capture the most important research questions across various areas of PSC research. 

With hundreds of questions voiced by PSC patients, caregivers, researchers, and clinicians, PSC Partners will next look to the ICRNetwork to support the organization and prioritization of research questions into a draft research agenda. This draft research agenda will be presented and discussed during the PSC Partners Annual Conference this June 2-5, 2022.

Following the conference, PSC Partners will formulate a formal Strategic Research Plan (SRP) to guide the efforts of the PSC Partners ICRNetwork. With a SRP in place, PSC Partners will pursue specific patient-driven research projects over the next five years.

Patient-Driven Research Projects

The PSC Partners Research Grant program will continue to support promising PSC research directions. With the implementation of the SRP, PSC Partners will expand efforts and capacity, and the ICRNetwork will drive forward key research projects prioritized by PSC patients. In our first round of prioritization, PSC Partners has focused on supporting drug development through the initiation of two multi-year projects: the Patient-Reported Outcome Measure (PROM) Project and natural history initiative (WIND). The PSC Partners Board of Directors decided to pursue both of these projects based on the needs voiced by PSC patients through the Our Voices survey and the Patient-Focused Drug Development (PFDD) Forum.

• PSC Partners WIND: Soon, we’ll be sharing more information about PSC Partners new natural history initiative, called the Worldwide Integration of Natural History Databases (WIND). Be sure to check out the recording from the Natural History 101 and join us for the Natural History 201 on March 23rd to learn more about this exciting new project!

• PSC Partners PROM Project: The Patient-Reported Outcome Measure (PROM) project aims to ensure that the PSC patient voice and symptom experience are heard loud and clear during clinical trials. Attend the PSC Partners Annual Conference this June to learn more about this project!

Pursuing The Most Important PSC Research Through the ICRNetwork

These research projects will meet key unmet needs for the PSC community, but our work together does not end here!

With the SRP in place, fundraising will be the next step to meet the budget of each research project. Fundraising isn’t the only way to get involved at this stage. Research projects cannot be completed without precious samples and health data donated by PSC patients. Your personal health data, blood, and tissue donations make research possible!

Together with the best experts to perform the research, our community funding and samples will propel research forward. Key findings from research projects will be shared openly with the full community of PSC patients, caregivers, researchers, clinicians, industry partners, and regulatory agencies. With further understanding of PSC, we’ll begin to ask new questions and pursue additional research, continuing the cycle. Ultimately, we hope that our collaborations will lead to patient-focused drug development and the identification of new treatments for PSC.

Patient-Focused Drug Development For PSC

While the aim of our SRP is to identify new treatments for PSC, these new treatments must first be rigorously studied in clinical trials. By initiating the PROM and WIND natural history initiative now, PSC Partners hopes we will be well-positioned to see meaningful improvement in PSC outcomes in future clinical trials.

Patients are at the core of all PSC Partners projects. PSC Partners strives to ensure a positive patient experience in clinical trials by providing the patient perspective to clinical trial design, as we recognize that no one knows the experience of living with PSC better than PSC patients. Through the ICRNetwork, patients and their caregivers will continue to help guide the development of clinical trials for PSC and respond to the Food and Drug Administration (FDA) requirement to pursue patient-focused drug development.

Looking To The Future: The PSC Partners Annual Conference, June 2-5, 2022

The PSC Partners Annual Conference will look a bit different this year with the launch of the PSC Partners ICRNetwork. With this growing focus on patient-driven research, PSC Partners will offer opportunities for patients and caregivers to meet and form connections with researchers and clinicians, who will share key research updates and welcome input from PSC patients and caregivers. PSC Partners welcomes all conference attendees to join a session detailing the PSC Partners research agenda. We will then provide the opportunity to join working groups to guide the development of the SRP.

Additionally, there will be educational opportunities for everyone in our PSC community, no matter where you are in your PSC journey. As always, you can expect to have fun, relax, and laugh as you reconnect virtually with friends, or meet new people, from the PSC community. Opportunities will include engaging social events, peer-to-peer sessions, small group chats, and more, from June 2nd - 5th, 2022.

This year, the conference doesn’t end on June 5th! With support from our conference collaborators, Dr. Josh Korzenik and Dr. Dan Pratt of Mass General Brigham, PSC Partners will organize additional virtual sessions covering popular conference topics between July and December 2022. We’ll welcome expert researchers and clinicians to present and answer your questions through a dedicated Q&A general session. Some topics include PSC and IBD, symptom management, the basics of liver transplant, and an overview of ongoing clinical trials. 

More conference information coming soon!

We Want to Hear What You Think!

Please email us at contactus@pscpartners.org with feedback, questions, or suggestions.

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