The British Liver Trust has welcomed the decision of the Scottish Medicines Consortium to make Maralixibat available as a treatment for cholestatic pruritus in children with Alagille syndrome.
“Having spent several years campaigning on behalf of families affected, we are delighted that children and young people in Scotland will now be able to access a medicine which alleviates one of the most distressing symptoms of this condition,” said Michelle Wilkins, Head of Children’s and Families’ Services at the Trust.
“We know first-hand the difference this drug makes, as the testimony of one young girl who took part in the trial for the medicine illustrates:
‘As a child, the main thing I recall about Alagilles was the severe itch it gave me. I had so many cuts and scratches that I was often too embarrassed to wear shorts and T-shirts like other children did in the summer. People used to assume that I had eczema, and I was happy to let them think that – I didn’t particularly want to talk about my liver condition. I agreed to take part in a trial for Maralixibat and the medication has changed my life, it’s reduced the itch so much, and unlike when I was younger, I can wear what I want now!’
“Whilst we acknowledge there are other complexities which Alagilles presents, we know that the emotional and psychological impact of pruritus is significant, not only for the child or young person suffering, but from their parents and carers who have to witness it.
“So, there is no doubt that this decision will have a real impact on the quality of life of patients and their families in Scotland. However, we are extremely disappointed that families in the rest of the UK will not have access to this as NICE (National Institute for Health and Care Excellence) has not yet recommended it. It seems extremely unfair that whether a child has access to a drug which could make a huge difference to quality of life for them and their family, is currently down to a postcode lottery.”
Details of the SMC decision can be found here.