What has SMC said?
The Scottish Medicines Consortium (SMC) has accepted marstacimab to help prevent or reduce bleeding episodes in children 12 years and older with severe haemophilia. Haemophilia is an inherited condition that affects blood clotting. In haemophilia A people lack a protein called factor 8 and in haemophilia B people lack a protein called factor 9.
SMC has accepted marstacimab for restricted use. The restriction means that it is accepted for use in patients 12 years and older with severe haemophilia B, who weigh at least 35 kg and who have not developed inhibitors to factor 9 (antibodies that stop factor 9 working). It is not recommended for use in patients with severe haemophilia A.
This document summarises the SMC decision and what it means for patients.
You can find more detailed information about the SMC assessment of marstacimab by looking at the SMC Detailed Advice Document (SMC2759).
What does SMC’s decision mean for patients?
Marstacimab for use in people with haemophilia B 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.
If you have haemophilia A and your healthcare professional thinks you would benefit from marstacimab, they can make a request to prescribe it. All health boards have procedures in place to consider these requests.
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 unable to accept marstacimab for haemophilia A for routine use. The company's evidence of the medicine’s value for money was not strong enough.
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 organisation below can provide more information and support for people living with haemophilia and their families. SMC is not responsible for the content of any information provided by external organisations.
Haemophilia Scotland
0131 295 0016
You can find out more about marstacimab (brand name: Hympavzi®) in the Patient Information Leaflet (PIL) by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website.