PBC Foundation Update regarding Ocaliva

[DonnaBoll]

Obeticholic Acid in Europe

29/6/2024

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 [e-mail] 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: 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).

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 [e-mail] Tel. [phone]

Mr Bjoern SEIBERT Head of Cabinet of the President [e-mail] Tel. [phone]

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, [e-mail] 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

rare-liver.eu/news/detail/o...