What has SMC said?
The Scottish Medicines Consortium (SMC) has accepted maralixibat, for treating children aged 2 months and older with cholestatic pruritus (intense itching due to a build-up of bile) caused by Alagille syndrome (ALGS). ALGS is a genetic condition that causes bile to build up in the liver and blood as it does not drain properly from the liver.
SMC has accepted maralixibat for restricted use. The restriction means that it is accepted for use in patients where standard medicines used to treat the condition have not worked.
This document summarises the SMC decision and what it means for patients.
You can find more detailed information about the SMC assessment of maralixibat by looking at the SMC Detailed Advice Document (SMC2806).
What does SMC’s decision mean for patients?
Maralixibat for use as described above is available for prescribing on the NHS in Scotland. Your healthcare professional can discuss with you if it is the right treatment for you or your child.
You can find more information about making decisions about your or your child’s treatment in this booklet called: Medicines in Scotland: What’s the right treatment for me?
More about SMC’s decision
SMC’s decision takes into account a confidential discount offered by the pharmaceutical company. SMC was also able to be more flexible* in its decision-making because the medicine is for a rare condition.
How does SMC make its decision?
SMC carefully considers every new medicine to make sure it benefits patients and is likely to be a good use of NHS resources.
To do this SMC studies the following:
- Evidence from the company about how well the medicine works compared with current treatments available in Scotland, in relation to how much they will cost to buy and use to treat patients.
- Information from patient groups about the potential impact of the medicine on patients and carers.
- Advice from healthcare professionals about any benefits of the new medicine compared to current treatment, along with how the new medicine is likely to be used.
When SMC assesses a medicine it takes account of the needs of all patients in NHSScotland, not only those who may be treated with the medicine.
You can find out more about how SMC decides here: https://www.scottishmedicines.org.uk/how-we-decide/
More information and support
The organisations below can provide more information and support for people with ALGS and their families. SMC is not responsible for the content of any information provided by external organisations.
Children’s Liver Disease Foundation
https://childliverdisease.org
0121 212 3839
The British Liver Trust
https://britishlivertrust.org.uk
0800 652 7330
You can find out more about maralixibat (Livmarli®) in the Patient Information Leaflet (PIL) by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website: https://products.mhra.gov.uk/