***** 5 September 2024 – LATEST UPDATE *****
We are pleased to share the news that Advanz Pharma have secured a temporary suspension of the revocation of the licence for OCA.
Read more here
***** 4 September 2024 *****
PBC Foundation statement on the European Commission (EC) revocation of conditional
marketing authorisation (CMA) for Obeticholic acid (OCALIVA®) in rare disease Primary
Biliary Cholangitis (PBC)
View our statement here
________________________________________________________________________________________________________________
We are issuing this guidance in response to the announcement made by the European Medicines Agency (EMA) on 28th June 2024 about the future of obeticholic acid (OCA or Ocaliva) in Europe. We understand that this announcement may cause concern, especially amongst people taking the drug. We want to reassure people as much as is possible, clarify the exact position at the moment and ask for your support in challenging what we regard to be an egregious and deeply flawed decision.
It is important to be clear that the decision to withdraw obeticholic acid is not based on concerns about safety. People taking obeticholic acid, or who are about to start it can continue to do so following the discussions with your doctor about the right treatment choice that have always been appropriate.
The decision to remove the drug is also not based on a lack of evidence of beneficial effects in patients but on an arcane and bureaucratic argument about what constitutes acceptable evidence that can be used to prove benefit. In simple terms the clear evidence of benefit provided by doctors and the company that sell the drug has been ignored by the EMA and a single trial placebo (COBALT) – where many people dropped out when they discovered they were in the placebo group in order to take active drug (for perfectly understandable reasons) meaning that the trial was unable to answer the question as to the effects of obeticholic acid- used as a justification for withdrawal. We believe that this is a de facto example of regulators blaming patients for doing what they felt was the right thing for themselves and their families (and something entirely within their rights as trial participants). We regard this as unacceptable.
The EMA were made aware of the views of patients and doctors across Europe in numerous letters and petitions. The EMA, however, chose to ignore those views. We believe that a process which is based entirely on the views of “experts” who have never spoken to a PBC patient let alone looked after one and which ignores the views of the people that the EMA thinks it is “protecting” with its decision making is also completely unacceptable.
Suffice to say we completely reject both the decision and the process that led to it, and will be using all available approaches to challenge it. It is time for your voice to be heard. We will be updating this guidance regularly as events unfold. We will be giving you information about ways you can make your voice heard.
We are aware that all this will cause concern to patients (something even the EMA acknowledge). The following is what we understand to be the exact position. As we said above we will update this guidance as and when new information becomes available
- This only applies to countries in the EU where drug access is regulated by the EMA. Other countries have their own processes (including the UK after Brexit) and we will be lobbying those other regulators to make sure that similarly misguided decisions aren’t made.
- Advanz, the company that sells obeticholic acid in Europe have made it clear that they put the best interests of patients and will continue to make the drug available for people already taking it. This is allowed by the EMA. This means that if you are on obeticholic acid and want to continue taking it you will be able to do so.
- There are several weeks until this takes effect (at the moment it is only a recommendation) and it may well be that that legal action will occur, further slowing implementation. In the mean time anyone for whom obeticholic acid is the right treatment will be able to continue being started on it, and then be covered by the arrangements about ongoing access.
We promise you we are working on this with every resource available to not only overturn this travesty in patient care, but to ensure this is not replicated by other regulators.
If you are interested in supporting this campaign, please email linda@pbcfoundation.org.uk and we shall keep you fully informed of next steps.
If you have not already done so, please add your signature to our first open letter: https://www.change.org/Caring_for_PBC
Yours,
Robert Mitchell-Thain, CEO PBC Foundation
Prof Dave Jones OBE, Chair of PBC Foundation’s Medical Advisory Board
***** UPDATED 1 JULY 2024 *****
Thank you for visiting here and, hopefully, signing our open letter (almost 1500 signatures, so far).
https://www.change.org/Caring_for_PBC
The other thing we need you to do is to email the two European Commission Heads with your personal views on OCA and PBC, asking for European Commission not to accept the recommendation of EMA.
Ms Ursula VON DER LEYEN Ursula.VON-DER-LEYEN@ec.europa.eu Tel. +32 229-56070
Mr Bjoern SEIBERT Head of Cabinet of the President Bjoern.SEIBERT@ec.europa.eu Tel. +32 229-66300
We have been informed that this is one of the most powerful tools the patient community has, and number of emails, irrespective of how well written or how basic, has a role to play.
So please… for the patients who so vitally need OCA in the toolbox of clinicians treating PBC, especially high-risk patients, can you send these people an email?
Feel free to copy me, robert@pbcfoundation.org.uk in also.
Thank you.
Robert
***** UPDATED 2 JULY 2024 *****
Please click HERE to review our open letter in response to the European Reference Networks (ERN) statement
https://rare-liver.eu/news/detail/ocaliva-withdrawal-ern-rare-liver-statement
***** UPDATED 5 JULY 2024 *****
Although we have not received a response to our open letter noted above on 2 July, the PBC Leads at the ERN have issued a response and it can be viewed HERE.
***** UPDATED 12 JULY 2024 *****
Please continue to sign and share our petition https://www.change.org/Caring_for_PBC we are almost at 1800 signatures!
In addition to the 2 email addresses of the EC Heads
Ms Ursula VON DER LEYEN Ursula.VON-DER-LEYEN@ec.europa.eu
Mr Bjoern SEIBERT Head of Cabinet of the President Bjoern.SEIBERT@ec.europa.eu
Please also email
EC Public Health – stefan.de-keersmaecker@ec.europa.eu
EC Com of Health – stella.kyriakides@ec.europa.eu
EC Press – eric.mamer@ec.europa.eu
This time it’s us that needs you!
Potential protest in Brussels on 25 July, if you can join us please email linda@pbcfoundation.org.uk
View Robert’s vlog’s with more information on our YouTube Channel
Short vlog
Long vlog
***** UPDATED 20 JULY 2024 *****
Our protest in Brussels has been confirmed and approved by the local authorities in Brussels!
Please please come and join us if you can – Thursday 25 July 2024.
We will be protesting 2.30-4pm local time which will include our live webinar at our usual time of 2pm UK time which is 3pm local time.
More information on the protest is here and please contact linda@pbcfoundation.org.uk if you intend to join us, this will help us manage numbers and know to look out for you on the day!
***** UPDATED 22 JULY 2024 *****
Please click here to view our CEO’s open letter to the EC.
The PBC PAG Community has now released a joint statement, click here to view.
Below are some quotes from the PBC Community regarding the ongoing situation with obeticholic acid.
Marco Carbone
“The CHMP has concluded that study 747-302 failed to show that obeticholic acid was more effective than placebo, yet I’d urge them to consider supportive studies and real-world data to confirm the benefits of this treatment. Revoking the license for obeticholic acid could have detrimental consequences for many PBC patients across Europe. It is imperative that the CHMP listens to the voices of patients and consults with PBC experts, patients, and their representatives in this matter.
The decision to withdraw obeticholic acid is not based on safety concerns. Patients currently taking obeticholic acid should continue their treatment in consultation with their doctors until the European Commission make their decision. This decision must be reconsidered to prevent harm to those who rely on this medication.”
Marco Carbone, MD, PhD, Associate Professor of Gastroenterology, Centre for Autoimmune Liver Diseases, University of Milano-Bicocca and on behalf of the PBC Foundation medical advisory board.
Ana Lleo
“I am deeply disappointed by the CHMP’s recommendation to withdraw the licensing approval for obeticholic acid based on the results of study 747-302. The study was designed with a placebo arm while obeticholic acid was already available as part of routine clinical care, this played a particularly large role in the trial failing to meet its clinical endpoints. Further, the CHMP did not consider the wide real-world evidence supporting the drug efficacy, I am greatly concerned about the prospect of not having obeticholic acid as a treatment option for PBC. I hope that the EC will not adopt this decision and will refer the opinion back to the CHMP for re-evaluation. We ask the EMA to engage with the community to find a way forward.”
Ana Lleo, Professor of Medicine at Humanitas University in Italy
Adriaan van der Meer
“Patients with PBC treated with UCDA with an unfavourable prognosis base on a combination of biochemical parameters and/or liver stiffness measures are in need of second-line add-on therapy. This can be obeticholic acid, at present still the only approved second-line treatment, or an off-label PPAR antagonist (most data available for bezafibrate). The choice which option to start with will depend on individual patient characteristics/preferences and drug availability. The more options that are available, the better we can serve all individuals with PBC.”
Adriaan van der Meer, MD, PhD, Hepatologist, Erasmus MC and co-lead ERN PBC working group.
Robert Mitchell-Thain
“It’s impossible to overstate the anxiety and despair experienced by patients suffering from PBC, upon their learning of the CHMP recommendation.
The CHMP’s apparent refusal to give weight to evidence outside of the 747-302 study is difficult to comprehend. The study design – which was required by the EMA – was fundamentally flawed: it asked high-risk patients to voluntarily not take active medicine for a number of years. This is asking the wrong people the wrong question in the wrong century. In a progressive disease, which is potentially fatal, it is completely unethical to ask patients to not take medicines that are publicly available, knowing that real world evidence shows genuine benefit to many patients. Unsurprisingly, there was an enormous drop-out rate in the placebo arm, as patients chose treatment instead, and the trial collapsed. Asking the wrong question is one thing, but for the CHMP to then follow the wrong answers in unforgivable. Whilst the CHMP claims it took into account the patient view and their experience with real world evidence and data, it could not have possibly done so or else it would agree with the enormous public outcry that obeticholic acid has an important part to play in the PBC therapeutic space.
This is a treatment that has been approved for use for seven years, showing clearly that appropriate use of this medicine helps patients in very direct terms. It has a mechanism different from any other medicine available and it is a vital tool in the clinician’s toolbox. To take it away, now, as an option for patients is a colossal step backwards in PBC care. If the CHMP had appropriately consulted patients and expert physicians, this would have been overwhelmingly clear.
We desperately need the European Commission to reject this opinion and instead return the case to the CHMP for re-evaluation; the lives of patients with PBC, and their families, across Europe quite literally depend on it.” Robert Mitchell-Thain, CEO PBC Foundation
We desperately need the CHMP to reconsider their decision; the lives of people across Europe with PBC quite literally depend on it.”
Robert Mitchell-Thain, CEO PBC Foundation
***** UPDATED 23 JULY 2024 *****
For any media enquiries relating to Obeticholic Acid in Europe, please contact our press office:
Sam Robinson, srobinson@axon-com.com / 07525888458
Please click the link below to view BASL’s statement
Statement on OCA BSG BASL 04 07 2024.pdf
***** UPDATED 24 JULY 2024 *****
Please click the link below to view EASL’s statement
European Medicines Association (EMA) statement on Obeticholic acid for the treatment of primary biliary cholangitis (PBC) European Association for the Study of the Liver (EASL) perspective – EASL-The Home of Hepatology.
***** UPDATED 25 JULY 2024 *****
Please click here to view the statement from The PBC Foundation Board of Trustees.
***** UPDATED 21 AUGUST 2024 *****
IMPORTANT UPDATE REGARDING THE FDA
Today is the deadline to register for the upcoming Open Public Hearing on OCA. Ideally, the patient community needs patients who have benefitted from OCA or who believe it is a vitally important tool in the PBC care pathway for patients.
We are happy to support, help or coach patients who want to take part but do not feel confident enough, yet, to make a presentation to the FDA in their own voice and to make sure their experience is shared.
The due date to register for the OPH is Wednesday, August 21st. We’ve included for you the instructions on how to register. Please remind this deadline to anyone interested in participating.
- Contact Jessica Seo (FDA contact) via GIDAC@fda.hhs.gov; or FDA Advisory Committee Information Line: 1-800-741-8138. If you prefer to submit written comments, you can send them to Jessica Seo at the same email address.
- Include the following details in your correspondence:
- Your name and address
- Docket # FDA-2024-N-3569
- A brief statement of the general nature of the evidence or arguments you wish to present
- The approximate time requested for your presentation
- Whether you plan to participate virtually or in-person
- The deadline to register is on or before August 21, 2024. Your request will be acknowledged by August 22
Updated 19 August 2024
Date Saturday 7 and Sunday 8 September
Location Delta Hotels by Marriott, 111 Glasgow Road, Edinburgh, EH12 8NF
In-person and virtual attendance will be available but please note that not all sessions will be available virtually.
In-person spaces and accommodation are allocated on a first-come, first-served basis, and a registration queue is in place.
Please note that all places with accommodation included are now full, day delegate and virtual attendance are still available.
We will follow up with you by email within 2 working days of your registration.
Subject to availability, the PBC Foundation will provide accommodation the nights of Friday 6 and Saturday 7 September (additional nights can be booked at your own expense directly with the hotel).
How do I register?
Please use this link to register: https://eur.cvent.me/glak9
This link will take you to a simple and easy-to-use registration form. Please make sure you have the following information to hand before starting the form:
- Any hotel requirements
- Dietary or other additional requirements
- Guest information, if applicable (name, contact details, dietary or other requirements)
What happens after I have registered?
You will receive an initial email confirming that your registration has been submitted. We will then follow up with you by email within 2 working days of your registration being submitted with an update.*
*Make sure you keep an eye on your Junk folder too, just in case. Emails will be received from Mo Christie.
EXCITING NEWS!
We are excited to share that Fibroscans will be available for patients throughout the conference!
EVENT APP AND WEBSITE NOW LIVE!
Our event app and website are now live which includes details of the agenda.
You can also view the Agenda here!
WHAT HAPPENS NEXT?
All patients who have registered will be receive an email with further information on the event app and website, how to book a Fibroscan and when our virtual drop-in sessions are to find out more about using the app.
We also need some further information from you regarding time of arrival and which meals you’ll be attending, please respond as noted in the email.
What if I have any questions about the conference or registering?
Please email Mo at mo@pbcfoundation.org.uk, or you can phone the office on +44 (0)131 556 6811.
Whilst we continue to work on translating our new website please note that you can still access our previous website in multiple languages.
Select the language below to view:
Arabic
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Japanese
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A new survey is live on our App.
Pruritus (itch) is a common symptom of PBC that can have a significant impact on quality of life. As you know, the PBC Foundation is very serious about drawing attention to the impact of pruritus. GSK also recognises the huge impact of pruritus and are working hard to develop a treatment to alleviate this condition. We are delighted to collaborate on this survey which will provide a better understanding of your experience with itch and identify the most pressing unmet needs.
All answers are anonymous and will be presented within collective data, e.g. 42% said yes. Survey responses will be collated, owned, protected and published by the PBC Foundation.
The PBC Foundation has a proud history of using the patient experience to improve the patient experience. We intend to use your collected, anonymised answers to campaign for better care in PBC itch. We can only do this when you share your experience and your knowledge. For that, we are grateful.
Apple device: https://apps.apple.com/gb/app/pbc-foundation-self-management/id1225705494
Android device: https://play.google.com/store/apps/details?id=uk.org.pbcfoundation.pbcapp1
