What has SMC said?
The Scottish Medicines Consortium (SMC) has accepted nusinersen for the treatment of patients with 5q spinal muscular atrophy (SMA). SMA is a very rare muscle wasting condition caused by a mutation in a gene called SMN1 located on chromosome 5q.
SMC has accepted nusinersen for restricted use. The restriction means that it is accepted for use in patients with type 2 or type 3 (later onset), 5q SMA that is causing symptoms.
This document summarises the SMC decision and what it means for patients.
You can find more detailed information about the SMC assessment of nusinersen by looking at the SMC ultra-orphan assessment report (SMC2805).
What does SMC’s decision mean for patients?
Nusinersen 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 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 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:
- 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 SMA and their families. SMC is not responsible for the content of any information provided by external organisations.
SMA UK
01789 267520
Muscular Dystrophy UK
https://www.musculardystrophyuk.org
0800 652 6352
You can find out more about nusinersen (brand name: Spinraza®) in the Patient Information Leaflet (PIL) by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website.