In this issue: • From the Registry Director • Mini-PFDDs • Patient Registry Video Tour • Help Us Grow & Diversify the Registry! • PSC Partners WIND Natural History Project • ROADMAP Wrap-up • PSC Research: Collaborative Efforts
From the Registry Director
Dear Registry Participants,
Excitement is mounting as we approach the 18th PSC Partners Annual Conference, which is being planned in collaboration with Brigham and Women's and Massachusetts General hospitals (Harvard University). We invite all 2000 of you, Registry participants, to join us. The main conference will take place June 2nd-5th, and pre-conference events will start on May 2nd. We can promise you an educational and fun community virtual event! Thanks to our sponsors, there is no required conference fee. Conference registration opens soon, and it only takes a couple of minutes to register. Registration for the pre-conference events is open now. Hope to meet you in one of the virtual community rooms!
The Registry Team's aim with this VIADUCT issue is to update you on your most recent impact on PSC research. Your survey responses were at work during the "mini-PFDDs," which were an offshoot of the Patient-Focused Drug Development Meeting (PFDD). These mini meetings were held for pharmaceutical companies conducting clinical trials for PSC and were a demonstration of the power of your voice.
We would like you to intimately know your Registry. We therefore prepared a Registry tour on video to help you navigate the treasures that are accessible to you as Registry participants.
Who can best report the lived patient experience but the patients themselves? The FDA and European EMA have mandated the integration of the voice of the patient in the drug trial and approval process. We've described PSC Partners' WIND project, which hopefully will fill an important gap.
We are actively seeking ways of reaching communities that are underrepresented in the Registry. You may be able to help the Registry Team increase diversity in the Registry. We provide you with actionable suggestions.
You may have missed some of the webinars in the ROADMAP series paving the way to the 2022 conference. There is great value in watching the recorded videos to find out how these webinars forged relationships among scientists and patients, and how they served to engage scientists in patient projects.
Finally, find out why creating duplicate Registry accounts creates difficulties in curation and why we ask you to avoid starting new accounts if you can't remember your Registry password and/or ID. We are always there to help.
Together, we are creating a world where a PSC diagnosis comes with a cure.
On behalf of the Registry Team, Rachel Gomel, Director, PSC Partners Patient Registry
Your Registry Participation & "Mini-PFDDs"
Your participation in the Registry quietly produces impact that may not be readily visible to all. Recently, one important example was the "mini-PFDD" events which PSC Partners has been holding for pharmaceutical companies conducting clinical trials for PSC.
In 2020, you were asked to complete the "Our Voices Survey," and PSC Partners used your responses to structure the Making Our Voices Heard meeting with the FDA (Patient-Focused Drug Development meeting). Registry participants and PSC community members, 850 in all, offered most valuable information on the impact of PSC on their lives. They also shared what they most wanted improved in their PSC journey.
The PSC Partners planning team, working under the FDA's guidance, very quickly understood the magnitude of this event and its potential. The event was clearly impactful: 1,216 viewers, including pharmaceutical companies, patients, caregivers, and regulators, watched and/or participated in the webinar. FDA representatives who were in the audience wanted us to know that the heart-wrenching PSC stories had moved them to tears throughout the event. Yet it was still too early to know about the specific and unexpected outcomes the PFDD meeting would bring and keeps bringing.
The PFDD was six-hours long and comprehensive. To accommodate targeted audiences, the PSC Partners team created short "mini-PFDD" meetings for pharmaceutical companies conducting clinical trials for PSC. We selected sections from the PFDD recording that would be pertinent to each clinical trial and to the audience that was being targeted. Most often, these audiences had not yet met a single PSC patient.
So far, PSC Partners has held three mini-PFDD meetings. With Mirum Pharmaceuticals currently conducting a Phase 2 clinical trial for pruritus (itching), patient testimonials describing their bewildering pruritus experience were selected from the PFDD meeting video, and in-person (via Zoom) patient panelists shared with Mirum staff the devastating impact of pruritus on their lives.
Later, in November 2021, PSC Partners presented extensive patient feedback from Our Voices Survey at the Mirum Principal Investigator Meeting which took place during the American Association for the Study of Liver Diseases meeting (AASLD).
For Pliant Therapeutics, which is conducting a Phase 2 clinical trial with their anti-fibrotic drug, PSC Partners presented different PFDD video clips from the PFDD meeting that focused on the pain and fear patients felt as their disease progressed, and a new panel of patients generously shared their very personal and painful stories with the Pliant staff.
The mini-PFDDs provided researchers, staff, and coordinators, most of whom have studied the disease but haven't met real patients, an opportunity to ask the patient panelists questions. Among the multitude of positive comments we have received, the one that meant most to us was, "There wasn't a dry eye in the audience," a statement that provided evidence that PSC had left the textbooks and test tubes, and had come to life for these researchers as the cruel, life-shattering, and devastating disease it can be for so many.
The mini-PFDDs have been adding the patient voice to clinical trials. Understanding the patient journey adds flesh and blood to drug development. One researcher said, "After meeting PSC patients, I go to work with a mission. Meeting the patients has added passion to my work."
Through Registry newsletters, we hope to convey the impact your participation has on PSC research. When we ask you to complete surveys or let you know that you may be a match for a clinical trial, your responsiveness changes the PSC research landscape, even if it is not immediately visible to you. For a rare disease such as PSC, no matter how rare, a responsive and engaged community is like a magnet to researchers who trust that they will find a large pool of patients to study. Together we are stronger!
We are grateful for every one of you.
The PSC Voice of the Patient Report Documents the PSC PFDD meeting
This recently completed report summarizes input shared by patients living with PSC and their caregivers during the 2020 PFDD and serves as a resource for all those interested in conducting PSC research and clinical trials.
The link to the report will be available on the FDA website. This comprehensive document includes an 8-page executive summary, a 54-page meeting report, and 100 appendix pages containing full transcripts of testimony as well as audience comments.
Check it out here.
PSC Partners is sincerely grateful to all who opened their hearts and courageously shared their very personal, and often painful stories of living with PSC.
Patient Registry Video Tour: How to Find Data, Trials, and More in the Registry Website
Did you know that Registry participants can view de-identified collective survey data on the Registry website? Or find information about clinical trials? Or access a variety of other resources?
This short video illustrates how to navigate the Registry site so that you can access its many features. Most are viewable by all participants, but collective survey results are only available for the surveys that you have completed – a good incentive to complete them all! Join the tour!
UPDATE (Jan 2024): This video is out of date. Stay tuned for new information on how to navigate the Registry data, which will be available in the Instructional Videos page!
Help us grow and diversify the Registry!
PSC Partners launched the Patient Registry eight years ago, and the attention given it by biotech and pharmaceutical companies have fully confirmed its validity and importance. The Registry, with more than 2,000 participants, has earned credibility as stewards of the patient voice.
However, your medical team may not have heard about the Registry. You can play an important role in serving as ambassador and spreading the information to your medical team and to others with PSC you may know.
Other than increasing numbers, an important Registry goal is to increase participation among underrepresented populations. Our current data would be strengthened by increasing the number of participants of color and ethnicity. Also, though PSC is more common in men, more women than men have joined the Registry. An accurate representation of the PSC patient community is important for trials and studies.
YOU can help increase the number of Registry participants by bringing the Registry to your providers' attention so that they can invite their other PSC patients to join. Also, if you are comfortable with others knowing that you are a Registry participant, take opportunities to encourage other PSC patients to join. You can do this individually or on social media in any PSC groups to which you belong (including PSC Partners groups).
What are key points worthy of sharing?
The Registry is our community's best tool for advancing PSC research.
All the patient information is stored in a secure environment that protects privacy. The Registry is HIPAA* and FISMA** compliant (U.S. government standards).
All personal data is de-identified before it is shared with researchers. No information that could identify you and your family members leaves the Registry.
Registry participants who meet general clinical-trial criteria are contacted by the Registry. The participants can then decide whether they would like to connect with the clinical trial team.
Participants can access and update their own information.
After completing a survey, participants can see the combined, de-identified data for all survey respondents.
Those who participate are making a difference!
If you need more information or have questions about advocating for the Registry, please contact registrycoordinator@pscpartners.org .
We are working on ways to reach a wider PSC community. Your help is one important way to increase diversity in the Registry. You can find more information in our Registry brochure.
*The acronym for Health Insurance Portability and Accountability Act (1996) which was created for the protection of identifiable health information. HIPAA requires the protection and confidential handling of protected health information.
**An acronym for Federal Information Security Management Act is legislation meant to ensure the security of data in the federal government. Annual reviews are conducted to assess the security controls in information systems.
WIND: a New PSC Partners Initiative
PSC Partners is planning the launch of a major new initiative named "PSC Partners WIND."
What is PSC Partners WIND?
WIND, or Worldwide Integration of Natural History Databases, is a PSC Partners-led and sponsored initiative that will make valuable patient data available to researchers. This will give them a better understanding of the lived PSC experience.
The WIND database will serve as a central location where PSC natural history data is securely shared with PSC Partners and with the broader PSC research community. Medical centers from around the world will be invited to contribute clinician-reported natural history data.
Why is the natural history of a disease important?
Natural history refers to the usual course of a disease over time. When natural history is captured, patients are followed over time as their PSC progresses. With a patient's consent, important data, such as treatments, complications, and more, can be stored securely in a natural history registry.
Natural history studies are crucial to PSC research and will one day enable us to get the right treatment to the right PSC patients at the right time.
The main differences between a natural history registry (or database) and a patient registry are the types of data, and how the data is captured. The PSC Partners Patient Registry is a patient-reported registry. A natural history registry includes systematic and regular collection of past or current data from medical records, imaging data, hospitalization records, and lab test results. The systematic capture of these health data over time is key to enabling the completion of natural history studies.
For more on the basics of natural history studies, please consider watching (or rewatching) the recording of the ROADMAP Natural History 101.
What are the main objectives of WIND?
WIND aims to contribute to development of biomarkers which may be used to demonstrate effectiveness of an investigational drug for PSC; provide external control group data for late-stage clinical trials in lieu of placebo groups; and collect various measures of quality of life simultaneously with physician-reported clinical data.
Who is involved in WIND? How can I join?
To support drug development, PSC Partners is working with great urgency to establish formal partnerships with academic researchers and institutions, and we will keep PSC Partners Registry participants and the community updated on which sites are ready to enroll patients into the WIND cohort. Enrollment is targeted to start in 2023. Stay tuned!
Where can I learn more?
If you missed the March 23, 2022, ROADMAP 201 Natural History webinar that featured the introduction of WIND, you can watch the recording. Also, attend the Annual PSC Partners Conference that will be held virtually June 2-5, 2022, to learn more about the WIND initiative. Visit the WIND page of the PSC Partners website for more details.
ROADMAP Wraps Up, Forges New Connections
The PSC Partners ROADMAP webinar series wrapped up in March with the announcement of the PSC Partners WIND project. Ten times over twelve months, the PSC patient/caregiver community and medical/research/industry stakeholders came together to learn, exchange ideas, and raise questions that will spark new research studies in the coming years.
Launched in April 2021, the ROADMAP initiative's primary goal was to accelerate progress to find therapies and a cure for PSC by:
Educating the patient community on the research landscape
Broadening the researcher base and strengthening multi-stakeholder communication
Creating opportunities for researchers and clinicians to engage in meaningful discussions with an informed patient community
In support of that goal, the webinars aimed to build a foundation of knowledge and collaboration leading up to the June 2022 PSC Partners Annual Conference, where next steps will be discussed. Open to patients, researchers, clinicians, and drug developers, the ROADMAP webinars included both educational segments and small-group discussions. The patient community learned about research developments; the PSC researcher base was expanded; and multi-stakeholder communication was strengthened.
Four broad areas of learning and discussion were covered: symptoms and patient-reported outcome measures; research; drug development; and natural history studies, in particular the new WIND project.
You can find a brief video explaining the ROADMAP initiative on the ROADMAP page of the PSC Partners website, in addition to all ROADMAP webinar recordings and accompanying educational materials. These resources are recommended as context for the upcoming PSC Partners June conference.
PSC Research: Collaborative Efforts
Collaboration is key for successfully advancing rare disease research. Research partnerships are efficient and effective because they pool expert resources, increase patient reach, and eliminate research duplication. Several exciting, dedicated collaboratives are part of the global PSC research landscape.
The International PSC study group (IPSCSG)
Founded in Oslo, Norway, in 2010, the IPSCSG membership represents more than 20 countries throughout Europe and North America, as well as Israel, Australia, and Japan. Members come from both basic research and clinical research, and a key aim is to coordinate PSC research projects.
Aligning important PSC research topics efficiently among IPSCSG researchers eliminates redundancy and, ideally, enables enrollment of larger patient cohorts into trials and studies. The group currently has several studies underway.
The IPSCSG established Common Data Elements (CDEs)* for PSC that are now included in their joint patient databases for patient data. The PSC Partners Patient Registry uses a subset of these CDEs in our Clinical Survey for consistency.
In 2016, the PSC Partners annual conference, which was co-hosted by Yale University, was held back-to-back with the first U.S. meeting of the IPSCSG. Several members of the group gave presentations to conference attendees, who also had opportunities to meet informally with IPSCSG members.
Joint plenary session with the IPSCSG - June 26, 2016, Yale University
The PSC Forum
The PSC Forum is part of The Forum for Collaborative Research (The Forum), a public/private partnership that brings together groups of U.S. and European stakeholders interested in specific diseases. These stakeholders include the FDA, EMA (European Medicines Agency), patients/patient advocates, researchers, and industry (pharmaceutical and biotech).
Founded in 1997, The Forum was instrumental in finding effective treatments for Hepatitis C and HIV. In 2016, The Forum was approached by PSC Partners and asked to consider the establishment of a PSC-specific group. The steering committee turned its attention to PSC and launched the PSC Forum in 2017.
In the PSC Forum working groups and larger meetings, stakeholders wrestle with the issues impeding research and drug development for PSC. Working groups address areas such as PSC research endpoints, clinical trial inclusion/exclusion criteria, and patient databases.
Dr. Veronica Miller, director of The Forum and an expert in regulatory science, has become an important partner of PSC Partners, assisting with various projects, including planning and moderating of the 2020 Patient-Led Drug Development forum with the Food and Drug Administration.
The Forum is based in Washington, D.C., and is under the auspices of the University of California Berkeley School of Public Health, where Dr. Miller is an adjunct professor. PSC Partners is a sponsor of the PSC Forum.
Ricky Safer, CEO of PSC Partners, Rachel Gomel, Director of the PSC Partners Patient Registry, and Martine Walmsley, Chair of Trustees, PSC Support, are the patient representatives serving on the PSC Forum steering committee.
The Consortium for Autoimmune Liver Diseases (CALiD)
CALiD has a growing membership of researchers and patient advocates throughout North America and Israel. Its members have an interest or expertise in autoimmune liver diseases, including PSC, primary biliary cholangitis (PBC), and autoimmune hepatitis (AIH).
Dr. Chris Bowlus of the University of California Davis School of Medicine founded CALiD in 2016. Dr. Bowlus is also co-chair of the PSC Partners Scientific and Medical Advisory Committee and medical advisor for the Patient Registry.
CALiD's main mission is to create a coordinated and standardized natural history database combining the efforts of individual institutions.
One of CALiD's collaborative projects has been an analysis of over 2,200 PSC patients to compare the proportion of Black patients with PSC at each CALiD center with the Black population in each center's metropolitan area. The purpose was to identify whether PSC is rare in Black populations and to highlight the problem of minority underrepresentation in many prior PSC research studies, most of which are based on white patients of Northern European descent. Results of the 2018 study can be found here.
PSC Partners has attended CALiD meetings since the collaborative's inception and has witnessed its significant growth through the years.
The growing collaboration among researchers brings hope that that new PSC treatments and an eventual cure are on the horizon. You can read more about each of these groups at the links below.
*Common Data Elements (CDEs) help provide consistency in the way data is collected, formatted, and described.
Complications Caused by Duplicate Registry Accounts
Keeping up with one's online accounts is a challenge in today's internet era. It's easy to lose account access due to lost passwords and obsolete email addresses. Perhaps you've even forgotten your Registry password.
You might be tempted to create a second Registry account if you have forgotten your login information, but please think twice before doing so. Duplicate accounts skew the research data, as each account is counted as a separate patient, and the curator has to search for duplicates.
The Registry team can help get you back into your original account.
If you forgot your login info but still have the same email address, please use the retrieval tool on the login page. If you still have trouble, you can email us at registrycoordinator@pscpartners.org and we will help you.
If you have changed your email address since you joined the Registry, please log in and update it or email the new address to registrycoordinator@pscpartners.org. It will be updated for you. Your email address needs to be up to date to use the retrieval tool on the login page.
Also, your current email address is vital so that the Registry team can reach you with information about your personal account, request health updates, and send you research opportunities when you may qualify.
If you already have two accounts, we are manually merging them, preserving all your survey data. Merging files is detailed, time-consuming work, so please help out by maintaining only one account.
Thank you!
Clinical Trials are Recruiting!
Don't forget to check the Registry Clinical Trials page for updates on active clinical trials!
The 2022 Annual PSC Partners Conference is Almost Here!
The PSC Partners Annual Conference will meet virtually this year, June 2-5, 2022, in collaboration with Dr. Josh Korzenik, Brigham and Women's Hospital, and Dr. Dan Pratt, Massachusetts General Hospital. We are excited to see you in attendance as we bring our community together for important conversations, exciting research updates, and renewed connection via our interactive, virtual platform. Registration opens May 1.
Established in collaboration with the Office of Rare Diseases Research (ORDR), the National Center for Advancing Translational Sciences (NCATS), and the National Institutes of Health (NIH)
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