Bad News

[DonnaBoll]

this was an email I received yesterday .....

Dear colleague

I am writing to you today with some disappointing, and deeply concerning news. Today (28th June), the EMA will announce that the licence for OCA use in Europe is being revoked. Advanz have, with our support, been engaged in a long, and frankly painful approvals process which has now reached its denouement. In essence, the EMA accept that there is a need for second-line therapies in PBC, accept that OCA is safe when used appropriately (importantly), accept that it improves biochemical tests that are themselves associated with disease severity and risk in PBC but given the failure of COBALT (the confirmatory trial) to show benefit the drug must be withdrawn.

They will not consider any of the real-world evidence from multiple sources, clinician experience and patient views (expressed collectively in multiple letters). Nor will they accept the arguments as to why COBALT was not able to show effects (high degree of unblinding with patients going onto active drug whilst still being included as placebo recipients, together with the issues of focusing (at the behest of EMA I might add) on more advanced patients who would be expected to reach endpoints during the trial but are, we all believe, much less likely to respond to therapy). In an act of supreme irony the EMA accept that this will cause real concern and distress to patients who have responded to the therapy (a therapy which, remember, they believe has no actions…….)

Obviously, there are parallel processes going on in other jurisdictions whose regulators might take a different view (especially around RWE) and this is not yet legal in Europe (meaning that European colleagues can continue to prescribe the drug to new and existing patients). I understand there is now legal action pending about concerns with the process which means that there is going to be further short-term uncertainty.

We are all bound to experience high levels of patient anxiety once this is announced and we all must do what we can to reassure people. We will obviously be putting out appropriate information to patients through the Foundation. Advanz have committed to make the drug available on a compassionate basis for patients wishing to continue when and if withdrawal happens so there is no reason for patients on the drug to worry. We need to reiterate that this is a decision about evidence of efficacy that is acceptable to EMA rules NOT about real work efficacy or safety.

I am sure that I speak for all of you in saying that this isn’t why we went into academic medicine.

I will write again next week than the immediate future may be a little clearer

Best wishes

Dave

Professor David Jones OBE

Director, NHIP Academy

Director, Newcastle Centre for Rare Disease

Professor of Liver Immunology, Newcastle University

Hon Consultant Hepatologist, Newcastle upon Tyne Hospitals

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