Announcing a New PSC Partners Initiative

WHAT Do I Need To Know About WIND-PSC As A Patient Or Caregiver? 

Please read our WIND-PSC overview document for patients and caregivers to learn more. 

To review definitions of natural history data, registries, databases, and studies:

• Review the ROADMAP 101 Warm-up: An Introduction to Natural History Registries and Studies
• Consider watching (or re-watching) the recording of the ROADMAP Natural History 101

WHAT Is The WIND-PSC Initiative?

In 2022, PSC Partners unveiled the Worldwide Integration of Natural History Databases (WIND) project, a PSC Partners-led and sponsored initiative. This project was renamed WIND-PSC in 2023.

WIND-PSC was inspired by the call of our community to  identify new treatment options for PSC and address unmet medical needs of PSC patients. Through the 2020 Our Voices survey, PSC Partners asked patients to choose the most important outcome, beyond a cure, to come from the development of new medications. More than 70% of PSC patients said that the most important area of focus is the development of  treatments to slow the progression of their PSC. With multi-stakeholder support and participation in the WIND-PSC initiative, PSC Partners aims to  accelerate drug development for PSC, taking vital steps to solving this core, patient-identified need.

WIND-PSC aims to create a database to serve as a central location where PSC clinical data and patient-reported symptom experiences from a large prospective cohort of people with PSC may be shared with PSC Partners and the broader PSC research community.

VIEW RECORDING OF THE WEBINAR ABOUT THE WIND INITIATIVE ABOVE.

WIND-PSC Goals

PSC Partners has identified the following three goals for this initiative:

1. Ensure that PSC patient data is accessible and used to best support the patient community, with the primary goal being to support successful drug development. This includes:

• The generation of a minimum protocol for data and sample collection
• Financial support of international PSC registries to support recruitment of 2,000 PSC patients across North America and Europe
• Timely sharing of clinical and symptom data into a centralized database in accordance with a minimum protocol
• Ongoing data sharing, monitoring, and auditing of regulatory-grade natural history data from PSC patients

2. Analyze these data to achieve the three aims of the cohort protocol. These include:

• Aim 1: Data collection to enable the creation of external control arms for use in clinical trials (also known as a synthetic cohort)
• Aim 2: Capture of prospective quality of life (QoL) data to enable the study of PSC symptoms and QoL longitudinally throughout disease progression

3. Expand and leverage the PSC Partners centralized database to meet the continued needs of the PSC community. Some examples include:

• The continued translation of natural history data into real-world evidence (RWE) 
• Patient-friendly clinical trial cohort design
• Investigations of off-label drug use
• Studies of recurrent PSC after transplant

WHY Did PSC Partners Develop the WIND-PSC Initiative?

PSC Partners is expanding its approach to research funding. In addition to the annual call for proposals and grant funding, the organization is launching the International Collaborative Research Network (ICRNetwork) and developing a strategic research plan (SRP). In the development of the SRP, the PSC Partners Board of Directors and staff identified the WIND-PSC initiative as a clear priority towards finding treatments for PSC. Together with the support of both PSC Partners and PSC Partners Canada, the launch of the WIND-PSC initiative is community-funded. 

The funding consortium is complete and made up of individuals, family foundations, and PSC Partners reserve funds, with approximately 40% of the funds coming from Canada and 60% coming from the US. PSC Partners is very grateful for the support of our community funders.

Why was this project prioritized? 

• PSC patients’ lived experience, without effective treatment, is important evidence of the natural course of PSC progression.
• At some future date, when a treatment for PSC is approved, there may be a need for a post-marketing surveillance trial. Such a trial will require a control arm of PSC patients receiving a placebo and not the new treatment. Through the WIND-PSC initiative, there is a possibility for patients from this WIND-PSC cohort to replace part or all of the control arm. This is called an external control arm.
• Why now? A number of therapeutics are in clinical trials for PSC, making now the best time to begin collecting data to serve as an external control arm.
• Why us? Read on!

WHICH Problems Will PSC Partners Aim To Solve For The PSC Community?

PROBLEM: Existing natural history data are segmented and difficult to share.

SOLUTION: PSC Partners will work tirelessly to build collaborations and bring data together.

• Our PSC patient data is precious, and we simply can’t afford to let it sit on the sidelines at individual institutions. Each data point represents a patient’s life and experience, and we hope they each will be used to the greatest extent possible to pursue new treatments for PSC.
• As a neutral third party, PSC Partners is able to work with researchers and their institutions to join data together. 
• In particular, as sponsors and stewards of the WIND-PSC data, PSC Partners and the multi-stakeholder steering committee will enable participating sites to share data in a centralized PSC Partners database to be used for future research. 
• PSC Partners is working with Arbor Research to serve as the data coordinating center and will organize a panel of experts and PSC community members to review and approve data requests. This process will ensure that data is deidentified and shared safely and anonymously.

PROBLEM: Resources are limited, which impedes our ability to recruit patients and collect data.

SOLUTION: PSC Partners, with the support of the PSC community, will invest funding to support research sites and ensure rigorous data collection.

• Across the world, dozens of research institutions have already begun collecting PSC natural history data. Excitingly, these have led to multiple publications and increased our understanding of PSC.
• At the same time, researchers at these sites struggle with limited funding and support of experienced personnel to extract and manage the data. 
• Through the committed $4 million U.S. dollars in community funding, PSC Partners will support our academic partners to bolster their ability to collect, extract, and share the highest quality data into the PSC Partners database.

PROBLEM: Researchers share core values, but creating a unified effort has been a challenge.

SOLUTION: PSC Partners, will serve as an advocate and representative for PSC patients as a neutral third party, uniquely motivated to create a unified effort.

• With so many important areas of ongoing research, dozens of researchers are studying the natural history of PSC. Some natural history registries focus on understanding untreated PSC, some on the relationship between PSC and IBD, and others aim to understand the effects of PSC on quality of life.
• While these seem very different, the registries often collect the same data, which can be united to better answer individual questions and address additional PSC patient needs. 
• With so much going on at individual sites, legal challenges, and limited resources, researchers have struggled to unify data sources and create a shared database. 
• PSC Partners, as a neutral third party, in conjunction with representatives from the PSC patient community, will lead the effort to distribute data within the centralized database and ensure PSC patient needs are heard and addressed.

WHO Is Involved In WIND-PSC? WHERE Can I Join?

WIND-PSC is the inaugural project of the PSC Partners International Collaborative Research Network (ICRNetwork). The ICRNetwork is a place for anyone to volunteer and contribute to ongoing research efforts. The success of WIND-PSC relies on support from patients, caregivers, clinicians, and researchers. 

In order to support drug development, PSC Partners will establish formal partnerships with academic researchers and institutions, and we will keep the community up to date on which sites are ready to enroll patients into the WIND-PSC cohort. 

It is important to know that the goals of WIND-PSC can be accomplished through enrolling just a few thousand dedicated PSC patients who are willing and able to follow-up regularly at dedicated research centers. It’s possible that, given the limited budget, geographical considerations, and numbers of patients and research centers needed to accomplish these goals, not all interested PSC patients will be able to join this initial cohort.  

At PSC Partners, we value every PSC patient's voice and experience and are so inspired by the dedication of our patient community and the interest that many have already shown in joining this effort. Regardless of participation in the WIND-PSC cohort, we encourage all patients to join the PSC Partners Patient Registry, where their participation will contribute priceless input and support to the WIND-PSC project and to other PSC research studies. Through the Registry, PSC Partners may connect PSC patients to ongoing natural history registries, research studies, and clinical trials for PSC. 

Ultimately, PSC Partners hopes that patient participation in the PSC Partners Patient Registry, combined with the current focus of creating a mighty WIND-PSC cohort, will provide a strong foundation for the efforts to expand and enroll all interested patients into observational cohorts in the future.

WHEN Will The WIND-PSC Cohort Begin Recruiting? When Will It End?

PSC Partners is pursuing an aggressive timeline to accomplish the goals of WIND-PSC. While the dates will depend on various factors coming together over the next year, here’s our best estimate:

March 2022: Begin drafting the WIND-PSC protocol with expert researchers. Begin pursuing data sharing agreements

Late 2023: Contract with individual sites and begin enrolling PSC patients at participating research centers

2025: Complete enrollment of 2,000 patients into WIND-PSC

2027: Begin interim analyses and begin using data to support drug development

2030: Complete 5 year follow-up and pursue additional directions for WIND-PSC

2030 - 2050+: Expand and leverage WIND-PSC to address additional needs of the PSC community

WHAT Can I Do To Support WIND-PSC?

REACH OUT

Do you have questions for PSC Partners? Want some more information? Please reach out to us at grants@pscpartners.org. 

SPEAK UP

Excited to join the registry and hear more about the plans for the WIND cohort? We’d love to hear from you on social media: tag us and use the hashtag #WIND-PSC. Also, please share information about WIND-PSC, including a link to this webpage, with your care team. Be on the lookout for more information explaining how PSC Partners plans to bring additional clinicians and researchers into the conversation at this year’s conference.

Together in the fight, whatever it takes!