7 August 2024

Dear Editor,

I write to inform you of a troubling development regarding access to a vital treatment for people living with Primary Biliary Cholangitis. PBC is a rare, autoimmune liver condition. It is progressive, incurable and overrepresented in liver transplantation. It has one licensed first-line therapy, and one second-line therapy. This second-line therapy is at risk of licence revocation by EMA.

The burden of proof in terms of its benefit/risk profile rests on a phase IV double-blind placebo-controlled clinical trial, requested by EMA. The trial failed and its results were described by investigators as “underpowered and potentially biased.”.

We consider EMA’s interpretation of its weak and inconclusive results to be highly questionable. It is increasingly plain that the study was unethical by design. To ask high-risk patients to forego access to an active medicine inevitably leaves them vulnerable to disease progression and changes in liver biochemistry. It makes them less likely to respond to anytherapy and serves to impair their quality of life and life expectancy.

The trial collapsed because of an almost 50% drop-out rate of patients from its placebo-controlled arm. Many wanted access to active therapy already approved in Europe.

While other therapies are emerging, none use the precise biochemical mechanism of this treatment. For some patients, there are no alternatives, while for others, using all the medicines in combination is the only answer.

Real-world evidence shows the treatment works and is supported by consensus among patients, clinicians and academics: yet EMA recommends revoking the licence.

As deeply distressing as this is for the PBC community, it has worrying implications for other drugs and other rare diseases. We must learn, be stronger and support each other.

For this reason, we set up this petition:www.change.org/Caring_for_PBC

When you make impossible demands of patients, clinical trials fail. Patients, facing the burdens that rare diseases present and discovering they are on dummy medicines, are justifiably going to seek access to therapies that can work for them. Why should EMA or the European Commission expect otherwise?

Visitpbcfoundation.org.ukfor more information.

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