Patients protest against possible withdrawal of rare liver disease medicine
The European Medicines Agency (EMA) human medicines committee has triggered outrage among patients by recommending the revocation of a market authorisation for a medicine used in the treatment of a rare liver disease.
The EMA human medicines committee (CHMP) issued a notice (28 June) that the medicine obeticholic acid, known under the name Ocaliva, is no longer recommended for treatment for primary biliary cholangitis (PBC), a progressive incurable autoimmune condition that affects the liver. The condition gradually destroys the bile ducts and can lead to liver failure or a heightened risk of liver cancer.
The EMA’s decision was met with outrage from the PBC foundation and those who feel that the decision is based on a flawed trial and a failure to take account of real-world data.
“We are asking the European Commission to look at the decision and ask them to reverse it for us,” said Robert Mitchell-Thain, chief executive of the PBC Foundation. “We have patients here today who are benefiting from this treatment, who are saying here we are, ask us for the real-world evidence!”
The foundation organised a protest near the European Commission’s Berlaymont headquarters (25 July), to urge the Commission to reject CHMP’s recommendation and to carry out a review of real-world data which they claim proves extensive clinical benefit. They are particularly critical of the randomised control trial, which resulted in many people dropping out to ensure that they had access to the active drug rather than a placebo.
For some patients it is the only available alternative for those who do not respond well to ursodeoxycholic acid (UDCA), another authorised medicine.
Ocaliva was granted conditional marketing authorisation by the EMA in 2016, subject to the results of a randomised clinical trial. Reviewing the results of the trial and taking into account the feedback from experts on liver disease, as well as from people who have experience of living with PBC, the CHMP concluded that the clinical benefits of Ocaliva were not confirmed.
The PBC foundation has received support from the European Association for the Study of the Liver (EASL), who state that, “published real-world evidence evaluating obeticholic acid over several years (at a population level) adds to the body of evidence that contributes to the overall assessment of drug efficacy.” EASL points to significant improvements in biochemical markers and a reduction in clinical events for those with early-stage PBC.
Asked to comment, a European Commission source told Euractiv it had received the EMA’s assessment that the conditional market authorisation for Ocaliva should be revoked and will proceed with a decision on the file within the 67-day deadline set out in the legislation. They would not offer any further comment at this stage, but said that the Commission had full trust in the scientific assessments of EMA.
If confirmed by the Commission, Ocaliva will no longer be authorised for use in the EU. However, the EMA has allowed continued supply through compassionate use or limited named-patient programmes to patients already using the medicine.
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