What has SMC said?
The Scottish Medicines Consortium (SMC) has not recommended ataluren for treating Duchenne muscular dystrophy (DMD) caused by a faulty copy in the dystrophin gene (known as a nonsense mutation). DMD is a rare genetic disease that gradually causes weakness and loss of muscle function. Ataluren is used in patients who are two years and older and can walk.
This document summarises the SMC decision and what it means for patients.
You can find more detailed information about the SMC assessment of ataluren by looking at the SMC ultra-orphan assessment report (SMC2827).
What does SMC’s decision mean for patients?
Ataluren for use as described above should not normally be prescribed on the NHS in Scotland. Your healthcare professional should talk with you about other treatment options for you or your child.
If your healthcare professional thinks you or your child would benefit from it, they can make a request to prescribe ataluren. All health boards have procedures in place to consider these requests.
You can find more information about making decisions about your or your child’s treatment here: Medicines in Scotland: What’s the right treatment for me?
More about SMC’s decision
SMC was unable to accept ataluren as described above for routine use. The evidence from the company about the benefits the medicine offers was not strong enough to justify the cost of the medicine. In addition, the evidence provided by the company on how well the medicine works, along with how much it would cost to use it, was not strong enough. This was despite using a more flexible approach in the assessment as it 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 Duchenne muscular dystrophy and their families. SMC is not responsible for the content of any information provided by external organisations.
Muscular Dystrophy UK
https://www.musculardystrophyuk.org 0800 652 6352
Action Duchenne
https://www.actionduchenne.org 07535 498 506
You can find out more about ataluren (brand name: Translarna®) 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/