What has SMC said?
The Scottish Medicines Consortium (SMC) has accepted exagamglogene autotemcel for the treatment of sickle cell disease in patients who are 12 years and older and who have frequent painful crises. People with sickle cell disease have red blood cells that are crescent-shaped (like a sickle) rather than disc shaped. These cells can block blood vessels, causing painful crises. Exagamglogene autotemcel is for treating patients who are suitable for a haematopoietic stem cell transplant (a transplant of the body’s blood producing cells) but do not have a matched donor.
This document summarises the SMC decision and what it means for patients.
You can find more detailed information about the SMC assessment of exagamglogene autotemcel by looking at the SMC Detailed Advice Document (SMC2852).
What does SMC’s decision mean for patients?
Exagamglogene 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.
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 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 living with sickle cell disease and their families. SMC is not responsible for the content of any information provided by external organisations.
Sickle Cell Society
https://www.sicklecellsociety.org
Anthony Nolan
0303 303 0303
You can find out more about exagamglogene autotemcel (brand name: Casgevy®) in the Patient Information Leaflet (PIL) by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website.