What has SMC said?
The Scottish Medicines Consortium (SMC) has accepted atidarsagene autotemcel for treating metachromatic leukodystrophy (MLD). MLD is a rare inherited condition caused by faults in the ARSA gene. Atidarsagene autotemcel is used for treating children with late infantile or early juvenile forms of MLD who don’t yet have symptoms. It is also used for children with the early juvenile form of MLD who have some initial symptoms but can still walk independently and do not yet have signs of mental deterioration.
This document summarises the SMC decision and what it means for patients.
You can find more detailed information about the SMC assessment of atidarsagene autotemcel by looking at the SMC ultra-orphan assessment report (SMC2886).
What does SMC’s decision mean for patients?
Atidarsagene autotemcel 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 treatment here: 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 very 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:
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 MLD and their families. SMC is not responsible for the content of any information provided by external organisations.
Alex TLC
https://alextlc.org 020 7701 4388
MPS Society
https://mpssociety.org.uk 0345 389 9901
MLD Support Association UK
https://www.mldsupportuk.org.uk 07414 529392
ArchAngel MLD Trust
https://www.archangel.org.uk
You can find out more about atidarsagene autotemcel (brand name: Libmeldy®) 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/