Volume 2, Issue 1
September 2020
Happy Anniversary Loon Duct Save the Date Oct 23 - PFDD Meeting The Loon Poll Free Webinars We Recommend A Call to Action - Canadian Drug Pricing Crafted by Community - Transplants in the Time of COVID Loon's Feature Person - Sandy V Volunteer Fundraising Have You Heard? News from US PSC Partners Canadian COVID-19 Resources
Many thanks to Loon Duct Leaders Jessica Travis and Meg Ramore for a year's worth of communications to the PSC Canada community! You are so appreciated!! And to all the volunteers who submitted articles, artwork and helped to edit and provide ideas for the Loon Duct. We couldn’t have done it with out you! And thanks to our audience for following along and answering all of Lulu the Loon’s questions in our loon polls.
This is an important opportunity for the FDA, pharmaceutical leaders, researchers, and clinicians to hear directly from patients and to understand the urgent need for treatments and an cure for PSC.
Please plan to virtually attend the
Patient-Focused Drug Development (PFDD) meeting with the FDA

Date: Friday, October 23, 2020
Where: Virtual Online Meeting
Mission: To inform the FDA, drug developers, and researchers about the disease burden of PSC patients.

What is a PFDD meeting?
Patient-Focused Drug Development (PFDD) meetings were developed in response to the FDA's initiative to incorporate the patient voice throughout the drug development process. The FDA recognizes that, "Patients are experts in what it is like to live with their disease or condition and use of available treatments." This is a one-time opportunity for our PSC patient voices to be heard by these important stakeholders and let them know why they should focus their efforts on us. 

Why is the PFDD meeting important for PSC patients, families, and the FDA?
PFDD meetings give the FDA and other key stakeholders, an important opportunity to hear directly from patients and their caregivers about:
  • the symptoms that matter most to them,
  • the impact the disease has on patients' daily lives,
  • the patients' experiences with currently available treatments,
  • and what they hope for from future treatments.

Who will attend the PFDD meeting?
The FDA, drug developers, clinicians, and other researchers will be there to hear what the PSC community has to say. We want the FDA to experience the commitment of our patient community in order to understand the urgent need for treatments and an eventual cure for PSC. Attendance numbers matter! We invite and encourage PSCers from around the world to attend the meeting virtually! That means we hope Canadian PSCers will bring their voices to this meeting! PSC Partners Canada is very involved in the planning and President Mary Vyas will be one of the moderators.

In what ways can you participate?
Attendance numbers matter! We invite and encourage PSCers from Canada and around the world to attend the webcast!
  • Attendees can submit comments online to the FDA during the open comment period of the event.
  • Even if you don't want to speak, being a part of the virtual audience is important to show the strength of our community.
  • We hope all attendees will participate in audience polling during the event.
What can YOU do TODAY to support the PFDD?
• Submit artwork that expresses how living with PSC makes you feel. The artwork will be displayed during the PFDD meeting or included in the final report and will be anonymous. Attach a copy of the artwork and e-mail it to contactus@pscpartners.org
Invite friends and family to attend the meeting and share this flyer with them: download flyer here

Submit a photo if your PSCer was diagnosed before the age of 18, or, if your PSCer is has passed, you can submit a photo of them on their behalf to be shared during the meeting. In order to maintain privacy, the names of those in the photos will remain anonymous. You can submit photos to contactus@pscpartners.org.  

Calling the men! As always, with a male predominance in PSC, we hope the male patients will show up and speak up!
To sign up to attend the PFDD: click here

The view the agenda for the PFDD: click here

The Loon Poll
Lulu has another question for you!
What do you like best about the Loon Duct Newsletter?
The Canadian relevant health information
I feel more connected to the Canadian PSC community
I am better informed about events happening
Lulu the Loon (our mascot)!
Free Webinars
We Recommend!
2020 PSC Partners Seeking a Cure Annual Conference Webinar Series:

6th webinar in the PSC Partners 2020 Conference Series:
Sep 14, 2020: Dr. Elizabeth Pomfret, Univ. of Colorado Anschutz, Dr. Veronica Miller, Univ. of California Berkeley, Dr. Gideon Hirschfield, Univ. of Toronto

5th webinar in the PSC Partners 2020 Conference Series:
Aug 12, 2020: Dr. Cara Mack, Dr. Shikha Sundaram, Dr. Juri Boguniewicz, all from Children's Hospital Colorado 

4th webinar in the PSC Partners 2020 Conference Series:
July 14, 2020: Dr. Lisa Forman, Dr. Michael Kriss, Dr. James Burton, all from University of Colorado

3rd webinar in the PSC Partners 2020 Conference Series:
June 3, 2020: Dr. Gideon Hirschfield, MD, University of Toronto,Dr., Cynthia Levy, University of Miami, Stephen Rossi, PharmD, Clinical Consultant, PSC Partners

2nd webinar in the PSC Partners 2020 Conference Series:
April 29, 2020: The interactive session featured Dr. Jerome Schofferman, a retired physician and post-transplant PSC patient, and Dr. Sally Holland, a psychotherapist and PSC caregiver.

The 7th and final PSC Partners 2020 Conference Webinar will be held in November, so keep an eye out on social media Facebook, Instagram and check the PSC Partners Seeking a Cure website often for the latest updates.  
Other Webinars:
UHN Centre for Living Organ Donation, Toronto
Living Donation Week 2020
A week-long series of recorded webinars on living donation
Youtube Playlist from the event: click here.

American Association for the Study of Liver Disease (AASLD)
For those PSC patients who also have Autoimmune Hepatitis
Webinar: Treatment Strategies for Autoimmune Hepatitis Beyond First Line Therapy 
Oct. 14, 12pm - 1pm ET: A discussion about alternative treatment options for autoimmune hepatitis and a review of their efficacy. Learn about updates to the treatment paradigm from the new 2019 guidelines!
To register just click here.

The Liver Meeting, Digital Experience TLMdX: American Association for the Study of Liver Disease (AASLD)
This digital version of the annual AASLD Liver Meeting (intended for hepatology professionals) is offered this year with special patient pricing ($50 if you register before Oct 31). Content will not be geared to lay public, but this is a source of liver research updates.
Nov. 13-16. More information at https://tlmdx.aasld.org/

A Call to Action Regarding Drug Pricing in Canada
Currently there are no drugs approved specifically for PSC. But clinical trials are ongoing, and we hope that new drugs will be shown to be effective and will become available in Canada. All new drugs coming to the Canadian market must comply with Canadian rules and guidelines, including pricing guidelines, and pricing guidelines have recently been updated. A call to action to the rare disease community has recently been issued by CORD (Canadian Organization for Rare Disorders). CORD believes that the revised guidelines are of concern in that they could potentially provide disincentives for drug companies from bringing new drugs for rare disease to Canada.

CORD is a national network for organizations representing individuals with rare disorders. CORD’s purpose is to provide a way for these individuals to work together as a common voice to ensure that health care policies and systems are created with the needs of those with rare diseases in mind. PSC Partners Canada is a member organization of CORD. 

The Patented Medicine Prices Review Board (PMPRB) is the public agency responsible for setting the guidelines that affect maximum allowed drug pricing in Canada. The new guidelines propose to set the maximum list price of drugs to not be higher than the median price of 11 comparative countries. Most Canadians, including those with rare diseases, can agree on wanting reasonably priced medicines. The main concern raised by CORD has to do with the fact that the new PMPRD “proposes to restrict the actual selling price of all new medicines to a single arbitrary standard, without regard for severity of condition, unmet need or the added benefits over existing therapy.” For the rare disease community, especially for those diseases with no treatments at all, CORD anticipates the guidelines will be a huge disincentive to bring new drugs into Canada.

The previous guidelines were designed in the mid 1980s when the drug market was dominated by companies making small molecule drugs for common ailments. But now, over 30 years later, the market is dominated predominantly by drug companies who are creating biologic and genetic therapies for specific illnesses. Some of the changes proposed by the PMPRB are:
For more information about the guidelines please visit the Government of Canada website here

The bottom line is that it is very hard to know if and how these proposed changes might affect drugs for PSC coming into Canada. One of the main criticisms of CORD is that the proposed changes do not have a separate pricing mechanism for drugs for rare diseases.

To learn more and to provide input, visit https://www.fightforourlives.ca/.
CORD is asking for constituents to contact their local MP. You can find more, including suggested language, at the fightforourlives.ca site.

Crafted by Community
Content created by members of the Canadian PSC community
COVID-19 Impacts on Liver Transplant in Canada
By: Kristian Stephens

In March of this year, as the COVID-19 pandemic was developing, hospitals across Canada hit pause on many of their surgeries including transplant procedures, involving both live and deceased donors, unless the recipient patient's life was at risk. Some transplant surgeries were cancelled for mainly two reasons: (1) to ensure that enough hospital beds and hospital equipment were available to handle any influx in patients due to COVID-19 and (2) that once transplanted, the individual would have a weakened immune system due to anti-rejection drugs - not an ideal situation during this COVID-19 pandemic.  

Although some transplants involving deceased donors continued during the pandemic, the number of transplanted patients decreased nationwide (Figure 1). For instance, the president and chief executive at Trillium Gift of Life Network, Ronnie Gavsie, said that between March 15 and May 10, 61 deceased-donor transplants occurred in Ontario. That compares with 170, more than double, such surgeries during the same period last year.  
Source: Unpublished draft data is from Canada Blood Services and Provincial Organ Donation Organizations.

In addition, the number of available organs for transplant has dropped. Because of the potential for disease transmission from donor to recipient, organs from people who died from COVID-19 aren’t useable, and all other donors must be carefully screened and tested.

Now that we are past the first wave of COVID-19 cases, hospitals are resuming transplant surgeries. However, as we are entering in the second wave of COVID-19, BC Transplant states that a decision about whether to proceed with a live donation and transplant right now “must balance [between] the risk the virus poses to potential recipients with the risk of delaying transplantation.” They add, it also depends upon the prevalence of COVID-19 in an area and whether a hospital has the capacity to support a transplant recipient who would need to recover in the intensive care unit. 

For more information on how current COVID-19 protocols are impacting transplant surgeries in your area, please consult your local Organ Transplant Centre. 
The Loon's Feature Person: Sandy VonOstrand
This is Sandy VonOstrand from Richmond, British Columbia. Sandy attended the University of Arizona in Tucson, is a golfer, and has been teaching herself Spanish over the last two years. She found PSC Partners Seeking a Cure online about 12 years ago and has been involved ever since. When asked what she enjoys the most about being a part of the PSC community, she had this to say: “ [It’s a] great place to go to get reliable information. I believe by working together we can find a way to cure this disease and PSC Partners is made up of people who have the passion, energy, experience, and knowledge to find a cure for this terrible disease.”
When her son was diagnosed with PSC, Sandy said it had a way of “spurring me on to do something.” “I believe it is easiest to raise the large chunks of money when people are first diagnosed and everyone is asking what they can do to help.”

Sandy’s first foray into fundraising for PSC research was before PSC Partners Canada was formed. She knew that fundraising for a Canadian organization that could write tax receipts for Canadain donors would make a difference, so Sandy worked with the Canadian Liver Foundation, and convinced them to make a policy exception and divert all funds that Sandy raised to PSC-specific research. Sandy and her event was able to fund the following study: Pediatric PSC-IBD: Optimizing colitis monitoring strategies to enhance liver disease outcomes.” 
So many friends were sorry to hear about the diagnosis and a fundraiser gave them the opportunity to come together to help out also. Sandy wanted the fundraiser she planned to be minimum outlay of cost so all the money collected could go to research. A large local pub was the perfect answer. She promoted it in a fun way so that 100 people bought $25.00 tickets to come for a burger and an enjoyable evening out. While they were there they could participate with a raffle and the silent auction. Because these were friends of the family and she knew what kinds of things they would be interested in paying money for, her silent auction items were very popular and almost everything went for the full value. 
Once PSC Partners Canada was formed, Sandy used this same format two additional times to raise funds for PSC Partners Canada. She planned a two-year break in between each event. Now she is looking at some other options for fundraising as well as promoting registration for organ donation in the community.

In 2018, Sandy was recognized by PSC Partners Canada with the Community Dedication Award for her continuing work towards raising funds for research towards a cure! Together, Sandy’s three community events have raised over $60,000 for PSC research. 

Thank you for all of your wonderful work Sandy!
We're Looking for Volunteers!
Sandy is just one of many who have helped to raise money for PSC Partners Seeking a Cure Canada. Although we are all adjusting to the new world we’re living in, there is still a need for support to help raise funds for PSC.

PSC Partners Canada has not stopped supporting the PSC community during this time, and we need your help to continue doing so. We are currently looking for volunteers to help with our fundraising efforts… NO EXPERIENCE NECESSARY!

If you’re looking for a way to get involved this is your opportunity! Please contact us at: contactus@pscpartners.ca or simply join us for our brainstorming session on November 1st. Check out the details below!
PSC Partners Canada • Fundraising Brainstorming Session
Sunday, November 1st at 6:00PM EST.

Meeting ID: 811 8662 2399 • Passcode: 692838

As we all continue to grapple with significant issues during this unprecedented time in our world, at PSC Partners Seeking a Cure Canada, we have never stopped our fight against primary sclerosing cholangitis.

Understandably, donations have slowed during this difficult time while everyone deals with their own challenges. PSC Partners' efforts to find a cure have not slowed. We are asking for your support, so that we may continue serving the PSC community. Together, we will find a cure.
Idea Bulb
Fundraising Ideas from Sandy V!

Internet Searches and Online Shopping: Did you know that you can earn a penny for PSC Partners, each time you search for something on the internet? It may not sound like very much but it adds up quickly, especially if lots of us are doing it. Sign up at goodsearch.com to use their yahoo search engine. Identify PSC Partners Seeking a Cure as the organization you want to support. The pennies go to American PSC Partners - we are all working together! Sandy has earned $133.00 over the years. Imagine if all of us were doing that? You can also sign up for goodshop.com. Participating businesses will give a percentage to the charity of your choice. For a list of Canadian businesses can be found here (for example, Apple Canada, Bed Bath & Beyond, Walmart Canada, Staples, SportChek, Pizza Pizza, Old Navy, Gap, Home Depot).

Unexpected Opportunities: This has been a challenging year with a lot of changes and unforeseen circumstances. Everyone has been affected and supporting PSC Partners during this has been difficult for various reasons, but consider this: the government has provided many individuals with some financial relief to help during this time. Although some people are really hurting during this pandemic, others may find they are saving money because of receiving some unexpected funds or simply because they aren’t travelling. If you or others you know are comfortable financially, the idea of donating some of the money they may have received or saved to www.pscpartners.ca may be a welcomed one. For some, this year may provide them with an opportunity to help when they normally wouldn’t. 
US Affiliate: Have You Heard the Great News?
  • PSC Partners launched an improved website for more effective and impactful dissemination of vital information to our community. The go-to source for PSC patient info!

  • The upgrade and launch of the new PSC Partners Registry is complete. See below for details, and don't miss the short video message about the registry from Dr. Gideon Hirschfield.

  • Rare as One project: Rachel G. and Mary V. from the Canadian board of directors are both part of the PSC Partners Rare as One team. Participation in this two-year grant with a cohort of 30 rare disease organizations is supporting capacity-building and thinking big as we plan for the future. Check out more here.

Upgraded Patient Registry!

The new registry website has a new look and feel, is easier to navigate in languages other than English, and has multiple surveys. Surveys can be updated annually or whenever there is a change in health, treatment, or your personal information changes. Users can now navigate the site, join, and take the surveys on their mobile device or tablet.

If you are newly diagnosed, or have not yet joined the patient registry, please join. The registry is a powerful way to have your patient experience heard by researchers and is a good way to learn if you might be eligible for a clinical trial. PSCPartnersRegistry.org 

If you have already joined, thank you! Please spread the word to others and encourage them to take part!

If you have any questions, please contact the PSC Patient Registry at RegistryCoordinator@pscpartners.org
Ask the PSC Expert
Joanne Hatchett, PSC Partners Medical Science Liaison, sat down for a short zoom interview with Dr. Gideon Hirschfield. Watch here.

Dr. Hirschfield is the Lily and Terry Horner Chair in Autoimmune Liver Disease Research at the Toronto Centre for Liver Disease, Toronto General Hospital and Professor of Medicine in the Division of Gastroenterology at the University of Toronto. He also serves on the PSC Partners Scientific Medical Advisory Committee.

The discussion focused on the Our Voices survey, clinical trials, and the value of the patient-led PSC Partners Patient Registry. Thanks Dr. Hirschfield!
Watch for the “You’re Not Alone Zone” posts on social media and come have some fun with the PSC Community!

Each post gives you an opportunity to share something with your PSC pals, whether it be a photo of your favourite pet, some ideas for a game night with family and friends, or some laughs with some of your PSC peers in a zoom meeting.

It’s a great way to meet some new people or just let others get to know you a little better. We may be separated, but we’re not alone!
Find the "You're Not Alone Zone" on Facebook, Twitter and Instagram
Our mission is to provide education and support to PSC patients, families and caregivers and to raise funds to research causes, treatments and potential cures for primary sclerosing cholangitis.

PSC Partners Seeking a Cure Canada
A Canadian registered charity affiliated with
PSC Partners Seeking a Cure

585 Dundas St East, Suite 300 • Toronto, Ontario • M5A 2B7 • Canada