Very directly. Very succinctly.  We need your help.

This is the most important thing the Foundation has ever worked on: more important than the surveys, more important than the helpline, more important than the name change.

We are writing a letter which we would like to share with regulators about the patients’ collective views on PBC treatments and about clinical trials in PBC. We need you to sign this letter and to share this letter for others to sign.  We need your voice so that we can all be heard.

As I am writing this, I am fighting back the tears because I know in my very core how vital it is that we, together, drive these changes for PBC patients around the world, for generations to come. This isn’t just about you or I, or now. This is about the thousands of PBC patients in the next generations who need the best possible care in their journey.

We are emphasising two things in this letter: 1) That Obeticholic Acid, aka OCA or Ocaliva continues to have a license, even if it is still conditional, as a treatment for PBC. There is a risk, and we are not entirely sure how big or how small, that the license may be under threat and we need to ensure the regulators know that PBC needs OCA to be part of the toolkit 2) that placebo arm trials stop in the later phases (iii and iv) of drug development for PBC disease management. Regulators still have a belief that placebo arm trials are the best and safest way to conduct these trials, and it is our strong belief that they are not.

1. OCA

There is a lot of real world evidence (i.e. from clinics working in PBC) that OCA makes a difference to those who need it. It is a small percentage of people with PBC, but a significant number who need OCA to improve their chances of a normal life expectancy. As with Urso, some people respond, some not: however, it is a vitally important part of a clinician’s toolkit in treating PBC. (it may actually be used in triple therapy for those difficult to treat cases, which also seems to be the case in the real world, currently)

OCA is not the answer for everyone. Indeed, some patients cannot take OCA because of side effects, or because it is not available in their country. It is clear, however, that OCA is an answer for many patients.

The challenge to OCA is that it was unable to prove in a confirmatory study that it works.  Why? Patients who found out they were on placebo (their liver tests were not improving) and obtained OCA from their clinicians. Do bear in mind, OCA was available to PBC patients in a number of countries, but not to those patients in the placebo arm of this trial. So, patients were benefitting from a new drug, showing the drug worked. So much so, that patients not on the drug (in placebo) got access to the drug and had their liver results improve, showing the drug worked. This meant the placebo arm on the trial managed to miraculously improve their liver tests. Oh, and patients also dropped out the trial.  The drug worked to the point where the company were not able to prove that the drug worked.

Confused??  Yep. We are too…

This is why this letter is so important.

2. Placebo

In short, the traditional way to have a clinical trial is for some patients to be given the new treatment, and some patients don’t.  The patients usually do not know what they have been given. Results come from who benefitted, by how much, and who didn’t. It is a proven scientific method used for many years and has yielded many good results and some amazing therapies.

However, in PBC disease management trials the people taking part are the exact people who should be on a working therapy. These are the people at the greatest risk of disease progression.  As PBC progresses, we know it is more difficult to treat. As biochemistry rises higher and higher, we know it is more difficult to normalise. Not impossible: but more difficult.

Yet, knowing this, regulators ask patients in the high risk group to volunteer to not take any working medicine, adding to their risk of not responding in the future when they do finally take a treatment.  We have an enormous wealth of data from groups such as UK-PBC and the Global PBC Group that can tell us what happens to patients who are not on treatment for years. We no longer need to have any single PBC patient not on a treatment that works.

And so we are calling for change.  We need you to sign this letter. We need to come together: the newly diagnosed, the lived with PBC forever, families, friends, clinicians, industry partners, but mostly you. There is so much more we can add, but we would invite you to contact us directly if you have any thoughts or questions, and chat with us the issues at hand.

So, we beseech you with everything we are: can you go to this link  https://www.change.org/Caring_for_PBC  and sign?

Please.

09 – 12 April | Edinburgh, Scotland 

Following the success of last year’s Summit, we are excited to announce that the PBC Foundation will be hosting its second International PBC Summit. 

The Summit will take place from 09 April until 12 April 2024 in Edinburgh, Scotland. 

We will bring together multiple stakeholders including patient advocacy and support groups, clinicians, industry partners, specialist nurses, and regulatory representatives to consolidate the outcomes of last year’s Summit into this year’s theme: to better use what we already know to improve the PBC patient journey.  

The Summit will offer the opportunity to bring together new knowledge and to leverage that to improve practice in care, treatment, policy, and research. We will be taking ideas and the work from last year and solidifying them into palpable projects with firm multi-stakeholder working groups. 

The Summit will comprise of an engaging mix of presentations, roundtable workshops, and plenty of opportunities for collaboration. 

We look forward to bringing you further updates as they become available. 

Patient Advocacy & Support Groups

Industry Partners

As ever: you asked, we listened…

We are delighted to be sharing with you a set of US-based self-care days, sponsored by Intercept Pharmaceuticals.

These days, between 9.30am and 3.30pm will focus on peer support, on PBC, and on emotional, psychological and physical self-care as you live each day with PBC. The events will be open to patients registered with the Foundation and also caregivers, and will bring a wide range of different tools and techniques to empower you on your PBC journey. As well as learning lots, there will be opportunities to connect with other patients and caregivers from your area, so as to help address any feelings of isolation.

These build on a raft of events in the UK, and we very much look forward to welcoming you in these cities:

Booking is essential so if you would like to attend please click the link for the city above and complete the form.

These sessions are part our registered community services so if you haven’t registered with the Foundation, you can do so here for free: https://www.pbcfoundation.org.uk/members/login-register/

If you are having log-in issues, please contact robert@pbcfoundation.org.uk

Statement from the PBC Foundation Medical Advisory Board

Since our last advice about Covid, there have been major developments. Although infections are still prevalent, the infections tend to be less severe, a smaller proportion of patients are admitted to hospital because of Covid and deaths from Covid are far fewer. Vaccination has been very effective and safe and treatments for those with Covid are far more effective in preventing serious illness and death. However, despite the relaxations in social activities, Covid has not gone away; infections are occurring, and, although for most people, the infection is mild, it may be serious and long Covid can affect many people.

Thus, while there is a great deal to celebrate, Covid has not gone away and will continue to affect us.  People with PBC, especially those with advanced disease, and those on immunosuppression are among those who are at greater risk. Note that UDCA and obeticholic acid are not immunosuppressive agents.

Advice from the Medical Advisory Committee includes:

Get yourself vaccinated: if you are eligible for vaccination, whether first, second, third or booster, take full advantage of the offer and get vaccinated. There are very few side effects from the vaccine and these contrast with the many, proven benefits to yourself and others: vaccinated people are less likely to get severe disease, less likely to require hospital admission, less likely to die from Covid and less likely to infect others.

Take sensible precautions:

• if you are in an enclosed space, consider wearing an appropriate mask, keep your distance where possible and ensure adequate ventilation

• Continue to use hand washing and sanitisation

• Avoid contact with people known to have Covid

Do go out and meet others: maintaining physical activity and keeping our mental health are important too.

For the clinically vulnerable

This group includes those who are on immunosuppressive drugs and those with advanced liver disease; if you are in this group, guidance includes:

• considering whether you and those you are meeting have been vaccinated – you might want to wait until 14 days after everyone’s most recent dose of a Covid-19 vaccine before being in close contact with others

• asking friends and family to take a rapid lateral flow antigen test before visiting you

• asking home visitors to wear face covering

• avoiding enclosed crowded spaces

Work for those who are immunosuppressed: HSE Guidance for England includes the following:

• UK government public health guidance advises people who are immunosuppressed or clinically extremely vulnerable, to work from home if they can. If they cannot work from home, they should talk to their employer about any temporary arrangements that could be made to reduce the risks.

• Employers have responsibilities to their employees who are immunosuppressed or clinically extremely vulnerable or live with people who are in these categories. Employers can help to support these workers by explaining clearly how they are managing the risks from Covid-19, asking and responding to any concerns they may have and making sure everyone works safely. Factors that should be included in addressing the employee’s safety are travel to and from work as well as in the work place.

Please note that the situation regarding Covid-19 is continually changing and advice given by the UK’s 4 nations varies. The advice given above is current at the time of writing, but further details should be obtained from the national bodies.

More information is available at:

https://www.gov.uk/government/collections/coronavirus-covid-19-list-of-guidance

https://www.gov.scot/collections/coronavirus-covid-19-guidance/

https://gov.wales/coronavirus

https://www.nidirect.gov.uk/articles/coronavirus-covid-19-regulations-and-guidance-what-they-mean-you

Professor James Neuberger, Chairman of the PBC Foundation Medical Advisory Board, shares an important message with us.  Click here to view: https://youtu.be/YVyNetPmOk4

We are delighted to inform you that within the Health Awareness section of today’s edition of the Guardian, there are several articles about “Your Liver” including a contribution by our CEO, Collette Thain MBE about Primary biliary cholangitis. To view Collette’s contribution click here: https://bit.ly/2vXrjVK

by Dr George Mells

CymaBay Therapeutics, a drug company focussed on developing medications for liver diseases including primary biliary cholangitis (PBC), recently announced that it had stopped all studies of a potential new medication called Seladelpar. This is because, in a recent clinical trial of Seladelpar in patients with non-alcoholic fatty liver disease, some of the people taking the drug developed inflammation of the liver. Seladelpar was a potential new medication for PBC, so it is disappointing that all studies of this drug have been stopped. On the other hand, patient safety is critical: if there is any suggestion that a medication might be harmful, it is better to err on the side of caution and stop people from taking that drug. CymaBay’s actions are a testament to the rigour with which clinical trials are conducted, and people with PBC will no doubt be relieved that the potentially harmful effects of Seladelpar were picked up during the trial phase.

Some people with PBC might have taken part in a previous clinical trial of Seladelpar. These people have nothing to worry about. They were monitored very closely during the clinical trial, and any problems were identified and addressed at the time of the trial. If they are worried, however, they should get in touch with their liver doctors.  

For further information, please see: https://ir.stockpr.com/cymabay/press-releases/detail/476