CLDF is disappointed to hear that the National Institute for Health and Care Excellence (NICE) has decided to not to recommend maralixibat, the drug developed by Mirum, for the treatment of cholestatic pruritus in Alagille syndrome in patients aged two months and over.
Please note, this is not NICE’s final decision, and the committee have requested comments on the draft guidance document.
Reasons for decision
The NICE committee felt that the clinical evidence presented was not clear enough in relation to how effective maralixibat is at reducing pruritus in the long term. They also concluded that the range of cost-effectiveness estimates presented for maralixibat plus standard care, compared with standard care alone, are likely to be above the range that NICE normally considers an acceptable use of NHS resources.
You can read the full detailed response about the decision in the consultation document via the link below.
https://www.nice.org.uk/guidance/GID-TA10832/documents/consultation-document
What happens next?
This is certainly not over yet! While we fully respect the decisions that NICE make through their in-depth assessments of new treatments, as a patient community we shall continue to provide insight about the impact new treatments could have on children with Alagille syndrome who are suffering from the debilitating and distressing symptom of cholestatic pruritus.
This is where you can help. NICE are calling on all interested parties to provide comments on this decision which will be reviewed in a further meeting to make a final decision. Anyone affected by a childhood liver condition, as well as family friends and medical professionals, can submit their comments.
These are the areas where we feel the consultation would really benefit from your insight:
- The need for effective treatment for cholestatic pruritus in patients with Alagilles – do current standard treatment work for your child?
- Are there other areas where maralixibat could lessen the overall burden on patients, caregivers and NHS resources. For example, the effect pruritus has on your child’s quality of life, impact on family, school attendance, hospital visits needed etc
You can submit your comments following the link below before August 21 2024. Please note that you will need to register or sign in as indicated before you can do so.
Or if you prefer, you can simply respond by submitting a few lines to us about how you feel about this draft decision. Your response can be as short or as detailed as you choose but you must send it to headsupport@childliverdisease.org before Friday 16th August 2024.
Although this treatment is currently only for those with Alagilles, we are calling all our families and young people to take part. This is your chance to make a difference by providing your comments and asking your family, friends and colleagues to submit a response on behalf of everyone affected by a childhood liver condition.
