What has SMC said?
The Scottish Medicines Consortium (SMC) has accepted vamorolone for the treatment of Duchenne muscular dystrophy (DMD) in patients who are 4 years old or older. DMD is a rare genetic disease that gradually causes weakness and loss of muscle function.
This document summarises the SMC decision and what it means for patients.
You can find more detailed information about the SMC assessment of vamorolone by looking at the SMC Detailed Advice Document (SMC2721).
What does SMC’s decision mean for patients?
Vamorolone 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: htps://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 0207 250 8240
Duchenne UK
You can find out more about vamorolone (Agamree®) in the Patient Information Leaflet (PIL) by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website.