What has SMC said?
Exagamglogene autotemcel was assessed by the Scottish Medicines Consortium (SMC) and meets the definition of an ultra-orphan medicine, which is a medicine to treat an extremely rare condition.
This document summarises the initial SMC assessment of exagamglogene autotemcel for treating beta-thalassaemia in patients who require regular blood transfusions and are 12 years or older. It is used where a stem cell transplant is appropriate, but there is no suitable donor available.
You can find more detailed information by looking at the SMC ultra-orphan initial assessment (SMC2709).
What does SMC’s decision mean for patients?
The Scottish Government will confirm when exagamglogene autotemcel is available for prescribing on the NHS in Scotland as part of the ultra-orphan pathway.
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?
What has SMC said in this assessment?
• In the clinical study 93% of patients who received exagamglogene autotemcel did not need to have blood transfusions for 12 months or more.
• The clinical study evidence was limited by the small number of patients, the limited time that they were followed up and the lack of a control so there is uncertainty about the long-term efficacy and safety, final results from the study and results from a long-term study are awaited.
• Despite the confidential discount offered by the company, the cost in relation to the health benefits of exagamglogene autotemcel remains high.
How does SMC assess ultra-orphan medicines?
SMC uses a broad assessment framework for ultra-orphan medicines. This is part of the ultra-orphan pathway in NHSScotland.
You can find out more about how SMC assesses medicines in the ultra-orphan pathway here: https://www.scottishmedicines.org.uk/how-we-decide/ultra-orphan-medicines-for-extremely-rare-conditions
More information and support
The organisation below can provide more information and support for people with beta thalassaemia and their families. SMC is not responsible for the content of any information provided by external organisations.
UK Thalassaemia Society
https://ukts.org
0208 882 0011
You can find out more about exagamglogene autotemcel (Casgevy®) in the Patient Leaflet by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website.